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1、Outlook 2024A Health Care Metamorphosis The Latest Scrip 100 Pharma RankingsM&A,Financing And R&D Data AnalysesC-Suite Insights On Future Trends December 2023|In Vivo|3 OUTLOOKTABLE OF CONTENTSOUTLOOK4SCRIP 100 16BUSINESS28GENERICS&BIOSIMILARS 40LEADERSHIP52MANUFACTURING60MEDTECH70POLICY®ULATION

2、80R&D88WORLD VIEW 100An unsteady geopolitical situation in eastern Europe,worldwide inflation and the politicization of drug pricing in the US all made their mark on 2023.Making predictions about the biopharma world is always a tricky endeavor,but looking ahead into 2024 reveals an uptick in deal-ma

3、king and perhaps the return of larger scale M&A,as big pharma feels the pressure to fill pipeline gaps at a faster rate.2024 will perhaps be a transition year as biopharma and medtech companies get through the worst of the market turmoil.Obesity and CNS diseases are creating fresh growthareas for bi

4、g pharma.And oncology has bounced back after being overshadowed in the R&D setting by COVID-19.Technological developments hold a lot of promise,but also bring a lot of confusion about best uses.The speed of development for artificial intelligence tools is outpacing the health care sector.For medtech

5、s,as well as an unstable financial market,environmental demands are adding pressure.Meanwhile,health care systems are grappling with rapid change in the structure of care delivery and how to adjust payment models to facilitate the ongoing move to care delivered in the ambulatory setting and at home.

6、Outlook 2024 includes exclusive interviews,data,features and industry league tables for Scrip 100,Medtech 100 and Generics Bulletins Top 50.LUCIE ELLIS-TAITT,EXECUTIVE EDITOR THANK YOU TO OUR SPONSORSIN VIVO EXECUTIVE EDITORLucie Ellis-TaittIN VIVO MEDTECH EDITORAshley YeoEDITORS-IN-CHIEFIan Haydock

7、Eleanor MaloneRyan NelsonDenise PetersonEXECUTIVE DIRECTOR Karen ColemanHEAD OF CUSTOM CONTENTAndrea CharlesMANAGING EDITORS,CUSTOM CONTENTMaire GerrardJanelle HartMANUFACTURING EDITORAndrew WarmingtonCONTRIBUTORSFrancesca BruceElizabeth CairnsDaniel ChancellorHeidi ChenEdwin Elmhirst Anju Ghangurde

8、Kevin GroganJoseph HaasNielsen HobbsMandy JacksonCatherine Kelly Chloe KentJessica MerrillAmanda MicklusShardha MillingtonTimothy PangBarnaby PickeringDean RudgeDavid WallaceMarion WebbDESIGNERSKosh NaranNancy PhamAll stock images in this publication courtesy of unless otherwise stated.Customer Serv

9、ices:Tel+44(0)203 480 7600 or (US)Toll Free:1 888 670 8900Email:To subscribe,visit:https:/ advertise,contact:Copyright 2023 Pharma Intelligence UK Limited(Citeline),a Norstella companyConnections to Spark innovationWhatever your marketing plan for 2024,werehere to helpIf youre looking to elevate you

10、r lead generation,brandawareness or market analysis,look no further thanPharma Ignite.As Citelines marketing insights division,our team ofindustry-leading analysts,editors,and partners providecustom marketing campaigns,utilizing Citelines world-renowned suite of products.You wont find a morecomprehe

11、nsive solution anywhere else.Custom ContentLive Events&Webinars Market Research&AnalysisAdvertising&AwarenessTailored Outreach: 2023|In Vivo|5 OUTLOOK4|In Vivo|December 2023 OUTLOOKAn unsteady geopolitical situation in eastern Europe,worldwide inflation and the politicization of drug pricing in the

12、US all made their mark on 2023.Making predictions about the biopharma world is always a tricky endeavor,but looking ahead into 2024 reveals an uptick in deal-making and perhaps the return of larger scale M&A,as big pharma feels the pressure to fill pipeline gaps at a faster rate.The unprecedented de

13、mand for new treatments in obesity has meant a couple of companies have fuller pockets and,in turn,more cash to put to work.The industry is moving closer to a large and extended patent cliff,a period of time from 2025 to the end of the decade when many of pharmas biggest revenue generating products

14、are facing loss of exclusivity(see Exhibit 1).While the maturation of understanding around diseases such as obesity and long-awaited novel approvals in Alzheimers will start to fill the gap,there is not sufficient pipeline growth to make up the difference.According to Evaluate Pharma,there is$14.1bn

15、 in US sales at risk in 2024.As well as a look at deal-making in the year ahead,In Vivo has highlighted a number of key development areas to watch in 2024 alongside anticipated clinical trial readouts.Deal-Making In 2024At Novembers BIO-Europe Fall conference,held in Munich,Ipsens EVP,chief business

16、 officer,Philippe Lopes Fernandes,highlighted the challenging market in 2023.He told delegates during a panel discussion on navigating biopharma deal-making,“It has been a challenging market,especially for public companies.Thank god for the biotech CEOs they are not all public,and right now it is mu

17、ch better to be a private biotech than a public one.The market is crazy,but the fundamentals are right.”Bradley Hardiman,senior director,Astellas Venture Management,described the situation for companies looking for deals and raising funds as“tightly controlled at the moment.”“We hear about dry powde

18、r,venture capital funds,but there is still fear in the market and we need to flip that confidence level,”Hardiman said.“Dry powder on its own is pointless,but lets make some fireworks and do some deals.Pharma is very active but there is pressure on our share price:we are not immune to what is going

19、on in the market.”SVP,head of global business development&alliance management at Merck KGaA,Matthias Mllenbeck,noted that M&A deals had focused on“post-proof of concept”assets in 2023.But he expects more earlier stage deals in 2024.“The number of targets with totally de-risked assets is limited,”he

20、said.“You will need to move into more earlier space,you will need to take more risk,despite having all of these uncertainties in the market.”Mllenbeck was cautious about the deal-making environment in 2024.He predicts a slower rebound for the market.“It will be a tough time ahead of us.The financing

21、 environment will likely not change dramatically.We will not go back to a money-for-free set-up,which will also drive industry consolidation at all levels.If I talk with our friends at the law firms,they are pretty busy not with doing stuff for the buy side,but preparing for mergers and reverse merg

22、ers at the moment,to get the cash to the assets that people believe should be invested in.”New Growth Markets Obesity and CNS diseases are creating fresh growth areas for big pharma(see Exhibit 2).In the first half of 2023,sales of Novo Nordisks Wegovy increased by 367%to DKK12.08bn($1.70bn)and anal

23、ysts expect these figures to go through the roof in years to come,especially as Novo Nordisk expands its manufacturing capacity to address the current shortage of the drug.At a Q3 2023 event in Copenhagen,Mads Krogsgaard Thomsen,CEO of the Novo Nordisk Foundation,said Novo Nordisk could be forgiven

24、for not predicting such an impact as Wegovy was the first entrant into“a market that didnt exist,”given that previously the only clinical option for obese people was bariatric surgery.Other therapeutic options for treating obesity are emerging and the cardiovascular-metabolic(CVM)space has been rein

25、vigorated in recent years.According to recent data presented at the Sachs Biotech in Europe Forum,clinical-stage cardiometabolic assets account for around 7%of the overall industry R&D pipeline.The top five diseases by number of studies under the cardiometabolic R&D umbrella are diabetes,NASH,hypert

26、ension,obesity and heart failure,which collectively represent about 50%of the clinical-stage CVM pipeline.Biopharma Embraces New Markets And New Tech BY LUCIE ELLIS-TAITT,EXECUTIVE EDITORNew markets,a patent cliff,an M&A rebound and clinical trial catalysts.How will it all play out for the biopharma

27、 sector in 2024?2023 Total Sales924,659-10,269-2,752-36,33610,7486,13593,557985,7422023 Patent Expiry Losses2024 Patent Expiry LossesOther LossesGrowth From 2023 LaunchesGrowth From 2024 LaunchesOther Growth2024 Total SalesExhibit 1:2024 Drivers And BrakesExhibit 2:Therapy Area Growth 18161412108642

28、0-2-10-5052019-2023 CAGR2024-2028 CAGR10152025BloodRespiratoryEndocrineImmunomodulatorsGastro-IntestinalCardiovascularMusculoskeletal GenitourinaryOncologyCentral Nervous SystemDermatologySystemic Anti-infectivesSensory OrgansSource:Evaluate Pharma Source:Evaluate Pharma 6|In Vivo|December 2023Decem

29、ber 2023|In Vivo|7 OUTLOOK OUTLOOKNovo Nordisk is also expanding its CVM pipeline.In October 2023,the company announced it was acquiring ocedurenone for uncontrolled hypertension,with potential application in cardiovascular and kidney disease,from KBP Biosciences for up to$1.3bn.Ocedurenone is an or

30、ally administered,small molecule,non-steroidal mineralocorticoid receptor antagonist that is being tested in the Phase III CLARION-CKD trial in patients with uncontrolled hypertension and advanced chronic kidney disease.Novo Nordisk expects to initiate Phase III trials for ocedurenone in additional

31、cardiovascular and kidney disease indications in the coming years.In the year ahead,a number of clinical trial readouts are expected for drugs targeting Alzheimers disease(AD).CNS diseases have seen renewed interest following the approval of new treatments for Parkinsons and Alzheimers disease.The u

32、nmet need in is huge,as is the market potential for drugs able to demonstrate a slowing of progression in symptoms or those able to tackle underlying causes of CNS conditions.The landmark,yet controversial,US approval of Biogen and Eisais anti-amyloid antibody Aduhelm came despite Phase III clinical

33、 trial data resulting in one positive and one negative study.In its announcement of this groundbreaking approval,the US Food and Drug Administration(FDA)acknowledged there was some uncertainty around the data,but reiterated that the treatment was the first to show a benefit in amyloid plaque reducti

34、on,thus targeting the underlying disease pathology rather than masking symptoms.The FDAs accelerated approval of Aduhelm based on amyloid reduction rather than cognitive improvement lowered the bar for approval,though market access barriers remain.In July 2023,Eisais Leqembi(lecanemab)became the fir

35、st anti-amyloid antibody to gain full approval from the FDA,after receiving accelerated approval from the agency in January the same year.Though competitor Eli Lillys donanemab may have a slight efficacy advantage,physicians may favor Leqembis better safety profile.A traditional approval decision fo

36、r donanemab is expected by the end of 2023.Despite uncertainties around efficacy and barriers to access,the high unmet need may ultimately create a lucrative market for anti-amyloid antibodies.If positive,data from subcutaneous formulations of anti-amyloid antibodies may generate excitement about a

37、more desirable formulation coming down the pipeline.Datamonitor Healthcare analyst Pamela Spicer told In Vivo,“Eli Lillys next-generation plaque-removing antibody remternetug targets the same pyroglutamate residue as donanemab but is designed to avoid the level of anti-drug antibodies.”Lilly has ini

38、tiated the Phase III TRAILRUNNER-ALZ 1 study evaluating a subcutaneous injection and an intravenous infusion of remternetug.A subcutaneous formulation of Leqembi is also being developed.Spicer highlighted biomarker results presented from a Phase I/IIa study of Biogens BIIB080 in patients with mild A

39、D as“the most exciting data to emerge from 2023.”BIIB080 is an antisense oligonucleotide designed to reduce concentrations of MAPT messenger RNA and thus reduce the production of all tau species within the CNS.During the study,patients on placebo maintained relatively stable levels of CSF tau,wherea

40、s patients on BIIB080 demonstrated a dose-dependent reduction in CSF tau over the three-month treatment period.In the two highest dose cohorts,CSF tau continued to decrease after treatment was discontinued,though patients on the lower doses saw a rebound in their CSF tau levels once treatment stoppe

41、d.With regard to the tau PET imaging results,which reflect aggregated forms of tau in the brain,patients that received placebo demonstrated a slight increase from baseline in tau across the majority of brain regions assessed.This is consistent with natural disease progression.For treated patients,th

42、ose on the highest dose showed a slight reduction in tau burden across all brain regions.Although tau tangles,along with beta-amyloid plaques,are considered hallmark pathological features of Alzheimers disease,tau-based strategies remain underrepresented in the late-phase clinical pipeline.Cancer Re

43、takes Top SpotEach year in its Clinical Trials Roundup,Citelines Trialtrove team analyzes the top 10 diseases for clinical trial activity to get a view of where research efforts are taking place.The most recent dataset looks at all trials in the full year of 2022.After a two-year reign,COVID-19 fina

44、lly gave its number one spot back to an oncology disease(unspecified solid tumor,566 trials),though it continues to exert its presence in a close second place(563 trials).Looking more broadly at therapeutic areas,oncology continued to be the top-ranking development area with a clear lead,even though

45、 its trial initiations were down by 10%.Within oncology,bladder,prostate and ovarian cancers will potentially gain more attention in 2024.Datamonitor Healthcare analyst Millie Gray told In Vivo that although bladder cancer is a very difficult disease to treat,“there has been a hub of research around

46、 this indication and the work is finally coming to fruition.”She noted that in 2023,Merck&Co.s Keytruda(pembrolizumab)plus Seagens Padcev(enfortumab)met its primary endpoint in the Phase III EV-302 trial,becoming the first targeted combination therapy to show an overall survival benefit over the cur

47、rent first-line standard of care,carboplatin or cisplatin in combination with gemcitabine.“This will become the new standard of care and will change the treatment paradigm,”Gray said.In prostate cancer,there is excitement around Novartis AGs radioligand therapy Pluvicto(lutetium vipivotide tetraxeta

48、n)and its use in wider prostate cancer indications.The product won its first US approval in March 2022 for the treatment of PSMA-positive metastatic castration-resistant prostate cancer(mCRPC)patients who had previously been treated with both androgen-receptor pathway inhibitor(ARPI)therapy and taxa

49、ne-based chemotherapy,based on the VISION study.Data are expected in the first half of 2024 from the Phase III PSMAddition trial,which is looking at Pluvicto in the metastatic hormone-sensitive prostate cancer indication(mHSPC).This lucrative setting is mainly comprised of de novo metastatic prostat

50、e cancer patients,and is heavily dominated by hormonal therapies such as Xtandi and abiraterone.“Competition here will be fierce but an approval will widen Pluvictos reach in prostate cancer,bringing it a step closer to being ubiquitous across prostate cancer settings,”Gray said.Another key readout

51、from the PSMAfore trial,looking at patients in the pre-chemo setting,will likely lead to a pre-chemo(but post next-gen hormonal therapy)approval sometime in 2024,further expanding Pluvictos reach.Also,a well-established standard of care,Xtandi,is expected to gain approvals in the high-risk biochemic

52、ally recurrent setting based on data from the Phase III EMBARK trial.Gray noted that a regulatory nod for Xtandi in this setting“could change how a large proportion of prostate cancer patients are treated.”Datamonitor analyst Ellie Davenport also spotlighted the first-line advanced ovarian cancer se

53、tting as an area likely to gain prominence in 2024.This indication is expected to see PD-1 and PARP inhibitor combinations gain approvals from 2024.The Phase III DUO-O trial investigating Imfinzi plus Lynparza is the only trial to read out so far,but more are expected in 2024.Currently,only PARP inh

54、ibitors,bevacizumab and chemotherapies are treatment options for untreated advanced ovarian cancer.“The launches of PD-1/PD-L1 inhibitor combinations have the potential to shift the first-line treatment paradigm through the introduction of new options for patients without a BRCA1/2 mutation or HRD d

55、eficiency,”Davenport told In Vivo.(See Table 1 for late-stage cancer trial data expected in 2024.)Drug/DeveloperClinical Trial Analysts Comments Enhertu/Daiichi SankyoPhase III DESTINY-Breast06HR+/HER2-low breast cancer patients who have progressed on endocrine therapy in the metastatic setting.Posi

56、tive data will cement its use as the leading ADC for HR+/HER2-low breast cancer,where it already has an approval based on the Phase III DESTINY-Breast04 trial.Imfinzi/AstraZenecaPhase III NILEImfinzi in combination with standard of care chemotherapy and Imfinzi in combination with tremelimumab and s

57、tandard of care chemotherapy versus standard of care chemotherapy alone in patients with unresectable locally advanced or metastatic bladder cancer.Keytruda is currently leading ICI in this setting.Krazati/Mirati Phase III KRYSTAL-12Krazati and JDQ443 are being tested in patients with previously tre

58、ated KRASp.G12C-mutated NSCLC.Patients with KRAS-positive NSCLC were previously deemed undruggable and responded poorly to immunotherapy treatments.If either Krazati or JDQ443 can demonstrate a survival benefit in a Phase III trial,they will likely become the treatment of choice.JDQ443/NovartisPhase

59、 III KontRASt-02See aboveJemperli combinations/GSKPhase II/III COSTARGSK is testing a triplet of cobolimab in combination with Jemperli and docetaxel,as well as a doublet of Jemperli plus docetaxel,against docetaxel alone in the Phase III COSTAR Lung trial in patients with advanced NSCLC whose disea

60、se has progressed on previous PD-1/PD-L1 inhibitor treatment.The post-immunotherapy setting is a setting of high unmet need and represents a large commercial opportunity as there is currently no standard of care,and often patients are treated with chemotherapy.“Dry powder on its own is pointless,let

61、s make some fireworks.”Bradley Hardiman,Astellas Venture ManagementTable 1:Select Oncology Trial Readouts To Watch For 8|In Vivo|December 2023December 2023|In Vivo|9 OUTLOOK OUTLOOKDrug/DeveloperClinical Trial Analysts Comments patritumab deruxtecan/Daiichi SankyoPhase III HERTHENA-Lung02The HER3-di

62、rected ADC patritumab deruxtecan is the first anti-HER3 monoclonal antibody in development for the treatment of NSCLC.Daiichi Sankyo is investigating patritumab deruxtecan in the Phase III HERTHENA-Lung02 trial in patients with EGFR-mutated advanced or metastatic NSCLC whose disease has progressed f

63、ollowing treatment with a third-generation EGFR inhibitor.Tagrisso,a third-generation EGFR inhibitor,is estimated to capture 6070%of the first-line advanced or metastatic EGFR-positive NSCLC market,and a standard of care treatment following disease progression is yet to be determined.Typically,patie

64、nts are not retreated with EGFR inhibitor monotherapy,meaning chemotherapy is often the choice of treatment and these patients are lacking an active and tolerable targeted treatment.Opdivo/BMSPhase III CheckMate 9DXTesting Opdivo in the adjuvant setting for the treatment of early-stage HCC.Currently

65、,early-stage HCC patients are primarily treated with liver transplants or surgical resection,or with locoregional therapies if they are not candidates for surgery.Imfinzi/AstraZenecaPhase III EMERALD-2Testing Imfinzi with or without bevacizumab as an adjuvant therapy in patients with HCC who are at

66、high risk of recurrence after curative hepatic resection or ablation.As with Opdivo,there is an unmet need for an efficacious but tolerable therapy for these early-stage HCC patients.Closing The Door On COVID?For many people,the pandemic is being placed firmly in the past as new worries take precede

67、nt the challenging economic climate,upcoming election years and tougher regulation,as a few examples.However,winter of 2023 will be a key test for countries that have seen declining levels of COVID-19 following successful vaccine programs.The biopharma sector is demonstrating a mixed response,with s

68、ome companies investing in advanced vaccine options while others step away from COVID R&D.With the Omicron variant now the dominant strain globally,accounting for 99%of new cases in the US and Europe,efficacy against this strain is paramount to long-term commercial potential.So far,Pfizer/BioNTechs

69、Comirnaty and Modernas Spikevax have shown the most robust efficacy data against the Omicron variant,with vaccine efficacy of 90%against hospitalization and death after a third booster dose,though protection wanes considerably after four months.Inactivated vaccines have played a crucial role in prim

70、ary vaccination series in China,India,Russia,and other emerging markets.However,as domestically produced vectored,protein subunit and recently mRNA vaccines reach emerging markets,inactivated vaccines are expected to progressively lose market share.Many pharma companies appear to be moving away from

71、 COVID R&D.Assessing the clinical trial landscape for industry-sponsored trials shows that in 2022 the number of trial initiations decreased by 7%overall.However,when excluding COVID-19 trials from this analysis,this reduces to a 4%decline,reflecting the industrys survival mode since 2021.“We have b

72、een seeing significant pipeline attrition as minor players have reallocated resources to other,more profitable indications from approximately 2021 onwards,”noted Datamonitor Healthcare analysts Natasha Boliter and Charlotte Holmes.“This phenomenon is particularly encouraged by the speed of COVID-19

73、mutation,demanding annual,variant-specific vaccines,and the excellent efficacy results(exceeding 90%)of currently marketed assets.However,we are also seeing pipeline discontinuations from larger vaccine manufacturers.This was heralded by Sanofi in 2021,which suspended development of its own mRNA vac

74、cine,despite reporting positive Phase I/II results,stating that the program was no longer commercially viable given the dominance of the other mRNA vaccines.”(see Exhibit 3).There is uncertainty even for giants in the COVID-19 vaccine industry.Pfizer,for example,is encountering constraints in the CO

75、VID-19 market,with predicted revenues for 2023 less than 60%of the preceding year.“In light of this,Pfizer has announced an enterprise-wide cost realignment program in response to the unpredictable demand caused by the transition from the government-sponsored pandemic phase to the privately or indiv

76、idually funded endemic phase,”Boliter and Holmes highlighted.“Currently,it is uncertain what this will mean for its pan-respiratory programs or its future seasonal,variant-specific vaccines.”There is still R&D interest in COVID,but the pipeline today consists of mainly next-generation mAbs,variant-s

77、pecific adaptations of currently marketed vaccines and the pan-respiratory combination vaccines.Looking ahead into the first half of 2024,AstraZeneca is expected to announce efficacy data from the Phase I/III SUPERNOVA study for its next-generation mAb AZD3152.Similarly,Invivyd is expecting primary

78、endpoint data from the Phase III CANOPY trial of its next-generation mAb VYD222 by early 2024.Generative AIArtificial intelligence was a buzzword in almost all panel discussions at the November 2023 BIO-Europe conference.There is a lot of promise,but also a lot of confusion about best uses.The speed

79、 of technology development in AI is outpacing biopharma.Generative AI,building upon advances in deep learning,is both a promising and a concerning technology.“If harnessed securely and ethically,leveraging multi-modal data,such as text,images,and videos,generative AI can help pharmaceutical companie

80、s identify unmet clinical needs and expedite clinical planning and execution strategies,”said Luca Parisi,Citelines director of clinical analytics and data science.In both drug discovery and repurposing,generative AI can play a role in respectively devising novel molecules and elucidating relationsh

81、ips that may inform drug repositioning.“Generative AI-powered drug repurposing efforts may include both approved drugs in certain indications and help in capitalizing on those drugs that did not make it through Phase II studies in some indications but could be better suited for treating other indica

82、tions,”Parisi said.He also highlighted the potential of leveraging real-world data,especially electronic health records and medical images.Here,generative AI“can help to titrate treatments on a subject-specific basis,accelerating the transformational paradigm of personalized medicine and the impact

83、it can bring to providing subject-specific,lifesaving or life-enhancing treatments faster.”Despite the potential uses and clear excitement around generative AI tools,there are challenges for using the technology in a health care setting.“It is crucial to ensure HIPAA,GDPR(where applicable),and GxP c

84、ompliance by design,thus leveraging appropriate infrastructure and technologies to guarantee that data security and confidentiality,and patient privacy are adhered to,”Parisi warned.“Furthermore,considering the scale of the data required to train such large generative AI models,appropriate analyses

85、to detect and minimize biases and ensure representativeness in the underlying data are of paramount importance to provide clinically relevant,accurate and reliable recommendations to design more recruitable,diverse,inclusive clinical trials,and inform operational workflows throughout the clinical tr

86、ial lifecycle objectively.”The key challenges ahead towards a fruitful,sustainable adoption of generative AI tools are:1.data quality,given the scale of the data required;2.achieving a seamless integration of such advanced technologies in clinical workflows;3.tackling ethical considerations by desig

87、n;4.ensuring replicability and reproducibility at all stages;5.clinical validation of the outputs derived from these technologies.The main expectation for the biopharma sector is to accelerate the continuum of the drug development pipeline and clinical trial lifecycle,increasing both time and cost s

88、avings.A Health Care Metamorphosis Biopharma is on the edge of a new era driven by the need to replenish pipelines and the evolution of technology.In 2024,the sector will see 70 key launches from around 65 drug brands.A notable proportion of these launches have the potential to shift treatment pract

89、ices.AstraZenecas Imfinzi is just one example,with five label expansions expected in the coming year.After a period of easy fund raising and busy deal-making in 2020-2021,tough times have hit the sector.But there is positivity for the volume of M&A deals to rise again,even if the financial markets d

90、o not bounce back as quickly as some might hope in the coming 12 months.Regulatory changes will have an impact from 2024 into the coming few years,in the form of both intentional and unintended consequences.Astellass Hardiman summarized the state of the sector:“We have seen a myriad of challenges ov

91、er the course of time.As a testament to our industry,we always overcome these I am confident and optimistic that we can again.”2020$4.46bn$96.38bn$105.24bn$39.26bn$30.43bn$26.76bn$24.07bn$22.72bn$22.03bn20212022202320242025202620272028Exhibit 3:Worldwide Sales For COVID-19 Treatments And Prophylaxis

92、Source:Evaluate Pharma December 2023|In Vivo|11 OUTLOOK10|In Vivo|December 2023 OUTLOOKApril 2024 sees another milestone in the fast-developing environmental compliance agenda for medtech and health care products suppliers.That is when the UKs National Health Service ratchets up its Net Zero Commitm

93、ent(NZC)requirements on suppliers,bringing into scope procurement contracts worth under 5m($6m).Suppliers in this bracket will have to demonstrate a commitment to net zero and comply with Carbon Reduction Plan demands.Since April 2023,the NHS has required contracts valued over 5m to publish a CRP fo

94、r their Scope 1 and 2 emissions.Very small contracts remain out of scope.It underlines how the environmental sustainability stakes and costs are rising for medtech manufacturers.The debate on how or if medtech manufacturers can pass on any of the additional costs is in full swing,but there is no obv

95、ious or easy answer.Compliance with sustainability needs remains voluntary for medtechs,but it is also non-negotiable.Scope 3 Will Need A Lot of WorkThe Commonwealth Fund reports that the US has set a 2045 net zero emissions target for certain health-related government facilities and buildings,but i

96、t is the model of NHS England(NHSE)that has set the standard in becoming the first health system to introduce Scope 1,2 and 3 compliance targets for all suppliers.Specifically,2040 is the year by which NHSEs own carbon emissions must reach net zero,and 2045 is the deadline for net zero carbon compli

97、ance across the providers entire supply chain.The supply chain accounts for 62%of NHSs GHG emissions.“There is lots of work needed to get there,”said NHSE head of sustainable procurement Alexandra Hammond.Her remit at the national provider extends to net zero,social value and the modern slavery agen

98、da.Within the GHG footprint that the NHS does not directly control,10%of emissions come from medical equipment and 20%from the pharma sector.From 2030,only those suppliers who can demonstrate progress through published reports and continued carbon emissions reporting will qualify for NHS contracts.I

99、nterim sustainability performance thresholds until 2030 are being applied under the NHS supplier roadmap.The compliance momentum is speeding up and circularity “designing out waste”has taken a seat at the medtech R&D table.ESG strategies have rapidly become indispensable for medtechs that intend to

100、continue operating freely in the global market be it attracting investment,working with partners,luring future talent or responding to the evolving customer preference and market trend.Accordingly,many medtechs with long experience of embedding Corporate Social Responsibility commitments into their

101、operating rationale were beginning to factor“E in ESG”principles into future business strategies before the UNs 2015 Paris climate agreement set wider society a target of limiting global warming to 1.5%this century.But where CSR has been open to greenwashing,the more data-driven and business complia

102、nce-oriented ESG principles cannot ethically or actually be circumvented.What Medtech Leaders Are Saying And DoingIn Vivos“E in ESG”series has sampled the different approaches that a selection of the industrys leaders employ Royal Philips,Johnson&Johnson,Thermo Fisher,GE HealthCare to name just a fe

103、w and has documented the sectors burgeoning scientific standards and compliance reporting needs.Each player in the fragmented medtech ecosystem will experience different pressures in how they achieve sustainability and compliance with the various internal goals they set for themselves in what is at

104、present still classed as self-regulating activity.There is no“cookie cutter”way of approaching sustainability in such a varied industry,although the principles are enshrined.Sustainabilitys demands were at first perceived as a constraint on medtech innovation,but things have actually worked the othe

105、r way.That is the view of Philips global head of sustainability Robert Metzke.He said Philips R&D department was motivated by the challenge of meeting heightened environmental goals.Circularity can in fact must create value for medtechs to remain competitive in the sector.Thermo Fishers global head

106、of sustainability and CSR Meron Matthias set the tone,telling In Vivo:“Theres so much potential for innovation across this space,and also many gaps to fill to allow us to reach our goals faster.”She is sure that there is a major innovation boom ahead.“There are technologies we need that do not exist

107、 yet,”she said in May 2023.But compliance must be watertight,because ESG litigation is already on the rise.Life sciences and healthtech companies must ensure tight management of risks to avoid any potential for regulatory enforcement,civil litigation,criminal sanctions and reputational harm.Sharehol

108、der activism and ESG due diligence are prominent litigation risks.Recent history shows that,in environmental and social matters,Europe tends to lead the agenda.In early 2023,the European Commission released the Net-Zero Industry Act as part of its Green Deal Industrial Plan for scaling up manufactur

109、ing of clean technologies in the EU.Its provisions and recommendations are designed to help the EU achieve its 2030 climate and energy targets.The EU Corporate Sustainability and Due Diligence Directive(CSDDD)will start to be put into place in EU member states as of the second quarter of 2024,and wi

110、ll have a two year or so lag before coming into force.Precisely when the CSDDD will apply to companies of different sizes and differing nature of activities is the subject to ongoing debate at EU institution level,legal firm CMS told the industry in fall 2023.The directive will introduce a new basis

111、 of civil liability.Not directly related,the European Health Data Space initiative,a regulation governing the use of electronic health data,will be implemented across the EU in two or so years.This is another area where the EU has a strong advantage.New Approach To MDR Sought But the enthusiasm with

112、 which medtechs have embraced the sustainability challenge is all but absent when the global industry reflects on the major ongoing compliance shift of recent years:the requirements of the EU medical device regulations.Here,it remains a case pragmatism,paying for compliance,defending technical files

113、,and,in many cases relinquishing safe and effective products and portfolios that the heightened compliance specifications coupled with lack of system readiness have rendered non-financially viable.Many companies have dropped portfolio staples,frustrated by the costly requirement to revalidate tried

114、and trusted products under the EU Medical Device and IVD Regulations.Germanys medtech industry told In Vivo during Medica 2023 that it believes that by the time regulatory change has bedded in,some 30%of products will have been dropped and 10%of companies will have closed.June 2024 sees the European

115、 Parliament elections,and the industry is keen to present its case for a more pragmatic MDR well in advance of the new intake of MEPs assuming their seats in Strasbourg and Brussels.Industry association MedTech Europes scathing view is that the MDR has not achieved the EUs goal of delivering a trans

116、parent,predictable and sustainable regulatory framework that supports innovation and the growing use of AI.That was its position in fall 2023,on issuing a position paper.Calls for a single EU authority to designate and oversee notified bodies have not gone away.Some medtechs suggest the European Med

117、icines Agency should have a bigger role in medtech.Many companies call for the early technical and clinical conversations on innovations between with regulatory stakeholders that the US FDA system provides.EU innovation has begun to vote with its feet.Companies that once prioritized the EU for devic

118、e launches are now increasingly taking the commercial decision to focus resources on the US market.But two countries on the fringes of the EU,whose major export markets are the EU,have been able to take matters into their own hands.Their local medtech industries are seeing the future possibilities.B

119、eing outside the single European market was not what medtech businesses in the UK and Switzerland would have chosen.But now,a sense of goal-oriented purpose is evident relating to the in-development UK medtech statutory instruments and Switzerlands readiness to open the door to FDA-approved medtech

120、innovations.The political and regulatory developments in both Environmental demands,the evolution of care models,new delivery technologies and AI tools are influencing how medtechs must approach innovation.Shaping A Medtech Environment Where Technology Leads The WayHealth Care Delivery Transformatio

121、n And Sustainability Modeled As Opportunities For InnovationBY ASHLEY YEO,EXECUTIVE EDITOR12|In Vivo|December 2023December 2023|In Vivo|13 OUTLOOK OUTLOOKCITELINE.COMTRANSFORMING THE WAY YOU SEE THE WORLD Comprehensive pharma news and analysis that goes beyond the headlinesEasily understand the impa

122、ct of global pharma news on your business with Scrip.Discover Scrips direct access to industry decision makers,reliable competitive intelligence,and best-in-class proprietary data.Register for your free,no-obligation,7-day trial.Go behind the headlines with Scrips worldwide team of industry experts.

123、Access up-to-the-minute insights and analysis of the events and topics in pharma that affect you most:Licensing opportunities Clinical trials updates Product lifecycle value chain info Partnership deals R&D activity Company developments Updates and analysis for multiple therapeutic areas.JN6920 Outl

124、ook 2024-Commercial Adverts-Scrip.indd 12023/11/24 12:48countries will be worth tracking in 2024,as much for the way the national regulatory codes and markets are reshaped as for how these breakaway initiatives are perceived in the much larger EU 27 market.One big prize for the UK would be EU mutual

125、 recognition,but in the meantime,its program of statutory instruments as the basis for its sovereign,post-EU,medtech regulatory system should be ready for parliamentary debate in late 2024 ahead of planned system readiness in 2025.Notable has been the readiness of the UK to embrace the IMDRF,interna

126、tional regulatory recognition and reliance,and potentially MDSAP.The use of FDA-approved devices in Swiss patients would not happen for a few years,but the parliamentary wheels are in motion,and when it happens,questions will be asked as to why the EU allowed its regulatory system to become innovati

127、on-unfriendly.Shift In Health Care Delivery Gathers PaceHealth care systems are grappling with rapid change in the structure of care delivery and how to adjust payment models to facilitate the ongoing move to care delivered in the ambulatory setting and at home.Germany,Europes largest medtech market

128、 and industry base,is introducing hybrid diagnosis-related groups as a means to fund more care outside the hospital.At the same time,it is overhauling its two-decades old system of DRGs with changes starting in 2024 that will replace flat rate,case-based payments for inpatient care with a combinatio

129、n of funding from a provisional payment pot as well DRGs.Hospitals will be relieved of the pressure to generate more income by maximizing procedure volumes.That is the theory,but the hospitals are first demanding a law that tackles underinvestment in hospital resources by the federal states.Globally

130、,as care delivery evolves,medtech innovation will increasingly be adopted in community and primary care settings,particularly with the advent of wearables,remote technology and telemetry.Companies will require sound data infrastructures,and developers of artificial intelligence-based products will r

131、equire multimodal data sets.Providers will have to respond accordingly.NHS England has set up a secure data environment(SDE)as a data and research analysis platform to give approved researchers and projects secure access to pseudonymized health care data.Reacting or even leading the digitally enable

132、d shift to ambulatory care will call for a different risk appetite and require a different kind of workforce capability.At the same time,the trend towards lower availability of healthcare professionals to serve the growing demand for care in the post-COVID era has put pressure on providers to the ex

133、tent that the embedding of interoperable AI capabilities clinical and operational workflows especially in cardiology and radiology is becoming a fact of life.AI In Health Care So it is no surprise that AIs approval ratings were at an all-time high in 2023.Philips annual Future Health Index revealed

134、that 39%of health care leaders are already investing in AI for critical decision support and 37%intend to invest in AI for automating documentation,scheduling patients or other operational efficiencies.AI is a tool that,for the present,is desired and feared in equal measure by HCPs,and the mantra th

135、at it will not replace clinicians and will act as only as second pair of eyes for radiologists may well all be true.But OpenAIs ChatGPT,released in 2022,took the debate to another level in 2023 when it became crystal clear that generative AI and its ability to create(almost)human texts,deliver answe

136、rs,advise and produce realistic images of fictional people,setting and events was moving in one direction only.Medtech can exploit AIs value in supporting innovative and targeted health care technologies,like the digital twin.Siemens Healthineers managing board member Elisabeth Staudinger is an enth

137、usiastic proponent of digital twinning,a technique that integrates data sources to model a digital representation of an individual.The enthusiasm surrounding the potential for AI to produce markedly better patient outcomes is dampened by impending regulatory structures yet to be put in place in the

138、EU.The EU AI Act the worlds first comprehensive AI act is a horizontal cross-industry regulation.It has sparked frustration among an industry that fears in the worst of all scenarios a system of double regulation for medical devices the MDR and the AI Act.In the run up to Medica 2023,German medtech

139、capital equipment manufacturers association ZVEI dismissed the need for another layer of potentially conflicting AI regulation for medtech in the EU.Technology Is The AnswerThe AI Act gained support from the German,French and Italian governments in late November and is on a pathway to adoption.How d

140、isruptive it will be to medtech innovation is a wait-and-see issue,even with its concept of regulatory sandboxes for innovators of AI-enabled technologies.Durable up to a point the medtech industry learns to live with such thorny issues.Manufacturers are still counting their wounds from recent exper

141、iences,ranging from COVID-19 to geopolitical conflicts,supply chain disruption,rising inflation and the increasing shortage of skilled staff.The ongoing challenges of health system transformation,the need to factor in digitally enabled remote diagnosis and therapy,reimbursement shifts,the consumer d

142、evice and wearables revolution and personalized and convenient care have also become fixtures on the agenda.The MDR(and IVDR)has been the standout bte noire for many years,but it has a rival now in sustainability compliance.Market access barriers and challenges will certainly push medtech manufactur

143、ers to the limit again in 2024.Industrys trump card is,as ever,innovation,aided by big data,cloud computing and augmented reality-based surgical platforms designed with AI and machine learning.Medtech must adjust to changing environments,but technology innovation is always the common denominator.14|

144、In Vivo|December 2023December 2023|In Vivo|15 OUTLOOK OUTLOOKFrom less tolerance for failure as pressure mounts on pipelines to ongoing consequences of regulatory changes,such as the introduction of US Inflation Reduction Act,life sciences companies will face a number of challenges in the coming yea

145、r.Precisely how health care systems deliver novel medicines is also high on the list of concerns.Industry experts from across the wider Norstella business shared their predictions going into 2024.Ashley Schwalje,senior director of Clinical Solution Consulting at Citeline,told In Vivo she was expecti

146、ng less tolerance for clinical trials that are not producing results.“Every trial is of increased importance,”she noted.Big pharmas are approaching a lengthy patent cliff,where best-selling brands will face heavy generic competition.This is driving a“fail fast mentality,”Schwalje said.The longer tha

147、t investment remains at suppressed levels,the more attritional the industry will be.“This is not necessarily a bad thing,”said Daniel Chancellor,thought leadership and consulting director at Evaluate,noting that few drugs that enter clinical trials eventually gain approval.“Concentration of investme

148、nt around fewer companies,platforms and assets that are more differentiated is better for long-term innovation.Nevertheless,picking the winners from the losers at early stages of R&D is as much luck as calculated risk.While an overfunded industry during 2020-2021 undoubtedly created some bloat,we ar

149、e now seeing an overcorrection and that risks some ideas going underfunded.”At the individual biotech level,a clear vision is required on how to create value for the end customer,whether that is patients,potential partners or investors.“Pharma is unequivocal in looking for assets that are first-in-c

150、lass or best-in-class,with a measurable patient impact,so adopting a development roadmap with this shared mindset is essential,”said Chancellor.In the absence of this,raising capital will be a challenge considering the huge number of other companies vying for investment,he noted.“This must also occu

151、r alongside careful consideration of costs and alternative means of funding such as partnerships.”At the same time,industry is also witnessing innovation outpace the rate of health care delivery system evolution,experts from The Dedham Group warned.“Without recognition of insufficiencies and opennes

152、s to change across payer and provider channels,novel testing and treatment options will continue to face limitations reaching patients,”said James Pisano and Jen Klarer,both partners at the specialty consulting firm.The Dedham Group highlight three key challenges in 2024 for access to medicines in t

153、he US:1.Sustained,Insufficient CMS Reimbursement:Provider sites are increasingly strained by insufficient government reimbursement,limiting patient access to appropriate care.CMS reimbursement is also slow to change and lacks recognition of its inability to adequately accommodate novel treatment opt

154、ions.Selecting the best sites and investigators(based on more sophisticated data models).Despite the increased use of AI,other digital tools are falling out of favor.Schwalje expects the interest in decentralized clinical trials to“die down”in 2024.Virtual trials were ramped up in the midst of COVID

155、,but the need for DCTs has calmed as the world moves on from the pandemic.AI is spilling into other areas of health care,such as managed care.“Payers experience significant infrastructure challenges so one might imagine them to be slower adopters to technological innovation,”said Dinesh Kabaleeswara

156、n,senior vice president of consulting&advisory services at MMIT.He noted that events across other industries had encouraged payers“to have conversations within their organizations on the applications of ChatGPT and AI to their day-to-day activities.”Innovation At A Cost“Increasing R&D costs,while gr

157、eat for bringing new therapies to the market,also pass on a percentage of these costs to patients through payers imposing higher premiums and stricter restrictions in access,”Kabaleeswaran warned.“As costs tend to increase,we should not lose sight of the most important stakeholder in the industry th

158、e patient.”The introduction of the Inflation Reduction Act in the US will also have an impact on the cost of innovation.“What assets will be cut because companies cannot afford the R&D investments?”asked Citelines Schwalje.According to a 2023 survey by PhRMA of its member companies,78%of respondents

159、 expect to cancel early-stage pipeline projects that no longer make sense given the short timelines before medicines could be subject to government price setting.Experts from Panalgo also highlighted the IRA as a critical issue in 2024,noting that“CMS is becoming the de facto US health technology ag

160、ency.”Chancellor noted that pharma has so far been powerless to shape the IRA.Although,with numerous challenges in play it will be interesting to see whether any legal arguments hold water.“Regardless of outcome,R&D and commercial decisions taken today must reflect the reality that the pricing envir

161、onment in the US is getting tougher,and the IRA may just be the tip of the iceberg.”Each new high-cost drug launch puts strains on budgets and can lead to increased premiums.In 2024,the conversation must shift towards value for money.“This will play out in real-time considering the eye-wateringly hi

162、gh revenue forecasts for the GLP-1 class,”Chancellor gave as an example.Looking just at Novo Nordisks glucagon-like peptide 1 agonist Wegovy(semaglutide),approved for the treatment of diabetes and obesity,the drug is expected to see worldwide sales of around$8.6bn in 2024.BY LUCIE ELLIS-TAITT,EXECUT

163、IVE EDITOR2.Constraints In Offering Novel Treatments:Provider sites are struggling to evolve treatment capabilities at a pace which matches novel product releases(e.g.,psychiatrist observation for psychedelics,inpatient bed availability for patient-specific treatment with cell-based therapies).3.Slo

164、w,Inconsistent Payer Coverage Of Genetic Testing:As opportunities for personalized medicine evolve,payers are unable to develop clear,comprehensive,and consistent coverage policies for novel patient identification techniques,even when targeted treatment options are available.Amidst these challenges,

165、“some provider sites and payers are staying ahead of the curve with increased resource allocation and forward-looking consideration of needs to enable access to emerging innovation,”Pisano and Klarer noted.Also,biopharma companies“are increasingly allocating resources toward market access to anticip

166、ate patient access challenges and develop stakeholder education plans,resources and patient services to address needs,”they added.New Tech And Patient GoalsOn a brighter note,industry spectators are watching a few important trends as we head into 2024,particularly the greater use of artificial intel

167、ligence to speed up decision-making.Also,with a renewed focus on health equity after the worldwide COVID-19 pandemic,experts expect that there will be a stronger emphasis on diversity in health care in the coming years.Looking at clinical trials specifically,Schwalje said,“Rubber will meet the road

168、on diversity in 2024.”Final guidance on DEI in clinical trials is expected from the US Food and Drug Administration in 2024 or the early part of 2025.“Pharma companies will tap deeper into real-world data to understand patient behaviors and social determinants of health to craft clinical trials that

169、 fit the needs of patients and create omnichannel,more personalized patient engagement strategies,”she noted.“Clinical trial diversity is part of a bigger picture related to health equity this will become a major part of the conversation,”Schwalje predicts.Alongside a focus on diversity and inclusio

170、n,Schwalje expects an increased emphasis on clinical trial patient experience in 2024.Some companies have already made waves in this area.Moderna,as an example,has a team dedicated to site and patient experiences.AI tools will play their part in helping biopharma companies reach the right patients.C

171、ompanies are starting to use AI to augment decision making and make smarter,more refined decisions related to:Building more precise,measured patient cohorts/segments;Designing clinical trial protocols with greater confidence;andIndustry experts from Norstella,whose data and technologies support deci

172、sion making from R&D through to market access,shared their worries going into 2024.2024202120222023Worries And Hopes For Health Care Delivery“Trial diversity is part of a bigger picture related to health equity.”Ashley Schwalje,Citeline SCRIP 100 SCRIP 10016|In Vivo|December 2023December 2023|In Viv

173、o|17In drug development,Pharmaceutical and Biotechnology organizations are leaning into data-driven insights to aid decision-making and lower the burden on physicians and patients to conduct and participate in clinical research.The paradigm has shifted even more since the global pandemic to adopt mo

174、re decentralized methods of data generation versus traditional clinical study methods.Traditional clinical studies can be costly,time consuming and burdensome on patients and sites.As a result,researchers are looking for new and innovative solutions to answer challenging research questions.One of th

175、ese solutions is Clinical Trial Tokenization.The adoption of using real world data(RWD)and enhanced insights through analytics and artificial intelligence(AI)is rapidly increasing and in the United States(US),the 21st Century Cures Act encourages use of RWD or medical data in generating evidence or

176、Real World Evidence(RWE)in regulatory submissions.Tokenization is the production of a unique encrypted token,or de-identifier in the place of Personal Identifiable Information(PII).It is already used in many industries,such as real estate,commercial analytics,finance and banking to gain insight into

177、 consumer behaviors.When employed in the life sciences,tokenization can be used for clinical research and development to enhance evidence generation.Streamlined And Efficient Approach To Generating Evidence Clinical trials already play a crucial role in advancing drug development by evaluating the s

178、afety and efficacy of new treatments.However,one aspect that often presents a challenge is the generation of longitudinal data showing what occurs for participating patients before,during and after the clinical study concludes.By harnessing the power of tokenization,drug developers can adopt a secur

179、e and efficient method to evaluate the long-term safety and effectiveness of treatments.Clinical trial tokenization is revolutionizing long-term participant monitoring even when patients move,change care practices,have study fatigue or discontinue study participation.By tokenizing participant PII fo

180、r consented patients,and matching to a unique identifier,researchers can link to patient records in a privacy-protected manner.These records may include medical claims data,electronic health records,laboratory data,treatment and follow-up records.Tokenization ensures that data remains accessible for

181、 long-term analysis.There could be a possibility to eliminate some of the cumbersome paper-based systems,and/or reduce the number of study site visits.By aggregating tokenized patient data within a proven privacy protected framework,researchers achieve a streamlined and efficient approach to generat

182、ing enhanced clinical evidence.This approach can be particularly helpful where the research requirements include regulatory-mandated long-term follow-up portion of up to 15 years for treatments such as gene and cellular therapy products.Tracking Effectiveness And Safety Of Treatment In Long-Term Fol

183、low-Up StudiesLong-term follow-up in clinical trials is essential for tracking the effectiveness and safety of treatments over an extended period.Tokenization enables the creation of unique tokens for each participant,which can be used to track and monitor their health outcomes in a decentralized ma

184、nner.Researchers can efficiently collect and analyze long-term data,identifying any potential trends in adverse events,disease progression or concomitant drugs administered.When longitudinal real world data is accessible,visibility of events that may have gone unnoticed during the trial phase,or hap

185、pened long after the study concluded,can be analyzed.For example,tokenized data can capture an adverse event(AE)occurring in the study population years after the investigational drug is approved and on the market.In addition,researchers can evaluate the patient population taking the investigational

186、treatment through the RWD capture and compare this to the general population not taking the drug.Enhancing Evidence Generation With Clinical Registries Traditional registry and natural history studies,whether publicly or privately funded,support the development of improved treatments for patients.Th

187、ese often-lengthy studies are a key initiative for collection and storage of real world information pertaining to a specific disease.A potential benefit for inclusion of tokenization in registries is the provision of de-identified trends and additional enhanced evidence that may answer research ques

188、tions in a specific patient population.Through tokenized data,researchers can compare patients with a given diagnosis or patient profile to the general population by looking at concomitant medicines,AEs,procedures,and diagnoses codes.RWD may lend insight into diagnosis patterns,pre-existing conditio

189、ns or risk factors.This secondary evidence generated can lead to more treatments and understandings surrounding a specific condition.Through analytics and reporting over the larger patient population,we can further extrapolate insights that occur in a specific gender or age(i.e.,.025%of the pediatri

190、c female population has a comorbidity of type 1 diabetes).Supporting Regulatory Discussions On Post-Marketing Commitments Regulatory agencies(e.g.,FDA,EMA)will sometimes request or mandate a post approval surveillance study(PASS)or require additional safety and efficacy studies for an approved produ

191、ct.Although many factors are captured in the drug approval process,there may be additional evidence gleaned about real world treatment patterns after the clinical controlled environment has ceased.There is a benefit in evaluating the safety and effectiveness of treatments on patients taking approved

192、 drugs.Tokenized data can further enhance evidence and generate support for safety and efficacy.This is particularly important in cases of pediatric or vulnerable patient populations.Sponsors may use RWE to support discussions with regulators,as this data supports and can increase significantly the

193、sheer volume of data to be considered.Balancing The Risks And BenefitsSponsors conducting any type of study,including long-term follow-up studies,must also delicately balance risks and benefits when using new innovative technologies in clinical trials.Some risks to be considered and managed include:

194、Ensuring appropriate patient consent,privacy and data security Increased financial costs to implement novel technologies Inadvertently unblinding a clinical trial Lack of adherence to Good Clinical Practice(GCP However,the potential benefits of deploying clinical trial tokenization are far reaching:

195、Capability to leverage insights for product development,positioning and performance The ability to support launch strategies with deeperunderstanding of product value Supplementary data for regulatory discussions/submissions Increased insight and ability to assess diversity,equity andinclusion(DEI)g

196、oals in clinical trialsSummaryClinical trial tokenization has the potential to revolutionize long-term study participant follow-up in medical research.By leveraging the power of this technology,tokenization offers streamlined data management,enhanced privacy and security,efficient tracking of long-t

197、erm outcomes,lessens the burden to sites and patients and facilitates collaboration among researchers.When considering including Clinical Trial Tokenization to research efforts,it is important to collaborate with a trusted partner who understands the rigor of research and development within the fram

198、ework of GCP and regulatory parameters.It takes more than a token to lead to meaningful evidence generation.Deploying Clinical Trial Tokenization requires an expert in consent and patient privacy,clinical technologies,GCP,General Data Protection Regulation(GDPR),security and compliance guidelines.As

199、 this innovative approach continues to gain traction,it holds the promise of transforming the way clinical trials are conducted,benefiting both researchers and participants in the quest for better health care solutions.For more information on how to deploy clinical trial tokenization in your study,p

200、lease contact www.ICON Use Cases For Tokenizing Clinical TrialsTokenization enables the creation of unique tokens for each participant,which can be used to track and monitor their health outcomes in a decentralized manner.By Melissa McDonald,Director of Operations,eClinical Development and Delivery,

201、Clinical Trial Tokenization,ICON plc SCRIP 100December 2023|In Vivo|19 SCRIP 10018|In Vivo|December 2023 SCRIP 100BY JESSICA MERRILL,SENIOR EDITORPfizer dominated the Scrip 100 rankings of the top pharmaceutical companies in the world based on full year 2022 pharmaceutical revenues,driven by its COV

202、ID-19 success,but Merck&Co.,AstraZeneca and Novo Nordisk each rose on the leaderboard,powered more generally by business fundamentals.It remained an atypical year for the pharmaceutical industry as some big pharma companies continued to generate unusual growth in 2022 from the sale of COVID-19 vacci

203、nes and treatments,and others faced challenging comparisons against the prior year as the pandemic phase of the COVID-19 outbreak wound down.Ten of the companies in the top 20 pharmaceutical rankings in the Scrip 100 sold COVID-19 products that either padded the top line or chipped away at growth in

204、 2022.Across the Scrip 100,total pharmaceutical revenues reached$1tn.Pfizer remained the biggest winner on the COVID-19 front,with sales of its vaccine Comirnaty,shared with BioNTech,and antiviral Paxlovid driving unprecedented growth for the company.Pfizers 2022 biopharmaceutical sales grew 24%to a

205、n astounding$98.99bn,but excluding sales of Comirnaty and Paxlovid,the companys top line only grew 2%operationally.Other big sellers for Pfizer included the Prevnar franchise of pneumococcal vaccines,which continue to be a steady earner.June 2021 saw the first approval of Prevnar 20,which lifted the

206、 franchise sales by around$1bn in 2022.However,potential competition for Pfizer in the pneumococcal vaccine space has been emerging particularly in infants from the likes of Merck and Vaxcyte.However,the April 2023 approval of Prevnar 20 in infants may allow Pfizer to continue to dominate the market

207、 which it has held for so long.Ibrance,Pfizers CDK 4&6 inhibitor,was also a major contributor to the 2022 sales total,pulling in over$5bn.According to Evaluate consensus forecasts 2022 was the last year before the drug begins to see a decline in its sales-losing out to competing products such as Lil

208、lys Verzenio and Novartiss Kisqali.Now Pfizer is facing an even more challenging comparison in 2023 as demand for COVID-19 vaccinations and treatments has continued to decline.Pfizer recently lowered its financial forecast by$9bn,citing lower than expected sales of Comirnaty and Paxlovid,and impleme

209、nted a$3.5bn cost reduction program.The companys revised guidance foresees 2023 revenues of$58bn-$61bn,a dramatic decline from the COVID-19 high of 2022,but still well above the companys pharma revenue base before the pandemic.Pfizer ranked seventh in the Scrip 100 in 2021,on 2020 pharmaceutical rev

210、enues of$41.9bn,after spinning out its established products business into a new company that is now Viatris.A big question on the minds of Pfizer investors is how the post-pandemic COVID business will eventually settle out and what annual demand for boosters and treatment will look like long term.Th

211、at could make a difference for Pfizer as it heads toward a patent cliff in the middle part of the decade like many of its peers.Other companies across the pharmaceutical sector were also impacted by gains and dips driven by the sale of COVID-19 products.Those include vaccine makers like BioNTech and

212、 Moderna,and to a smaller extent Johnson&Johnson and AstraZeneca,as well as manufacturers of antivirals like Gilead Sciences and Merck&Co.and developers of monoclonal antibody treatments like Eli Lilly&Co.,Regeneron Pharmaceuticals,and GlaxoSmithKline.Gilead,for example,was negatively impacted in 20

213、22 as use of its antiviral Veklury(remdesivir)tapered off later in the pandemic as newer antiviral alternatives like Paxlovid reached the market.The companys FY 2022 revenues were flat at$26.98bn and the company held steady on the Scrip 100 at number 13.Excluding Veklury,Gileads product sales increa

214、sed 8%.Lilly experienced a smaller headwind from the sale of its COVID-19 antibodies which boosted revenues in 2021 and 2022 but were less effective against newer variants of the virus that emerged in 2022,which correlated with revenues tapering off.Lillys 2022 pharmaceutical revenues grew 1%to$28.5

215、4bn,as COVID-19 antibody sales declined 10%for the year and 96%in the fourth quarter.Lillys ranking on the Scrip 100,like Gileads,also remained steady versus the prior year at number 12.Rounding Out The Top 5AbbVie:2022 represented the peak of Humiras sales bringing in over$21bn,just over a third,of

216、 Abbvies sales.The anti-TNFa antibody is currently the biggest selling drug of all time having made$219bn in sales from its launch in 2003 to the end of 2022.However,after an exceedingly long lifetime,Humira came off patent in the US in January 2023.Although the patent may have expired and Humira is

217、 forecast to lose a third of its sales in 2023 it still maintains a commanding grip on the US market.Current consensus forecasts predict the drug will pull in over$14bn in 2023 retaining its position as Abbvies biggest seller.Skyrizi was Abbvies second largest seller over the course of 2022 and also

218、 the companys largest growth driver.The psoriasis drug generated$5.1bn in sales in 2022 with$2.2bn of that representing new sales over 2021s annual tally.Rinvoq,another autoimmune agent,also counts among Abbvies top growth drivers.Unfortunately for Abbvie Skyrizi and Rinvoq had disappointing starts

219、to 2023 with both drugs missing their first quarter estimates.However,both of these products have seen label expansions recently,including Crohns disease for Rinvoq in the US and Europe in Q2 2023.Looking ahead to 2023s top-line pharma sales predictions for Abbvie reveals that current consensus is a

220、 fall of around$4bn from the 2022 total to$54bn.The loss of exclusivity on its flagship asset is a huge blow to Abbvie and its sales line.Whilst this loss is being partially offset by the growth of new immunology products investors are continuing to ask questions about potential M&A targets to try a

221、nd boost performance.Pfizer held the number one spot on the Scrip 100 on the strength of its COVID-19 products,while some others faced challenging financial comparisons in 2022 because of COVID-19 headwinds.Industry Leaderboard Gains And Dips Were Still Marked By C VID-19BY EDWIN ELMHIRST,DATA JOURN

222、ALISTProductIndication2022HumiraArthritis,rheumatoid$6.7bnCrohns disease$6.2bnArthritis,psoriatic$2.1bnPsoriasis$3.9bnAnkylosing spondylitis$1.1bnUlcerative colitis$1.2bnSkyriziPsoriasis$4.6bnCrohns disease$0.1bnArthritis,psoriatic$0.5bnImbruvicaNon-Hodgkin lymphoma(NHL)$0.9bnLeukaemia,chronic lymph

223、ocytic(CLL)$2.3bnWaldenstrms macroglobulinaemia$0.2bnBotoxOveractive bladder$0.6bnCervical dystonia$0.6bnHyperhidrosis$0.3bnMigraine$1.2bnMuscle spasticity$0.1bnFacial wrinkles/Nasolabial folds$2.6bnRinvoqArthritis,rheumatoid$2bnEczema/Dermatitis$0.2bnAnkylosing spondylitis$0.1bnUlcerative colitis$0

224、.1bnArthritis,psoriatic$0.2bnOtherOther$16.8bn2022 Sales Portfolio BreakdownSource:Scrip 100;Evaluate2021$56.2bn$58bn$54.1bn20222023(estimated)AbbVies Pharma SalesSkyriziRinvoqHumiraBotox CosmeticVraylar$2.23bn$0.87bn$0.54bn$0.38bn$0.31bnTop 2022 Growth DriversAdditional sales in 2022 versus 2021.20

225、21$79.56bn$98.99bn$64.7bn20222023(estimated)Pfizers Pharma SalesPaxlovidPrevnar 13ComirnatyPanzygaVyndaqel$18.9bn$1.1bn$1.0bn$0.5bn$0.4bnTop 2022 Growth DriversAdditional sales in 2022 versus 2021.20|In Vivo|December 2023December 2023|In Vivo|21 SCRIP 100 SCRIP 100J&J:remained steady moving from 202

226、1 into 2022 seeing a modest$900m growth in top line pharmaceutical sales to bring it into third place.J&J appears to have a slightly more diverse top end portfolio compared to Pfizers reliance on COVID-19 and AbbVies aging Humira.The psoriasis antibody Stelara and multiple myeloma drug Darzalex were

227、 J&Js two biggest earners over 2022,bringing in$9.7bn and$8bn respectively.Stelaras patent is expected to expire around 2025 and the sales are forecast to continue to climb until then.Along with Xarelto and Imbruvica(partnered with Bayer and AbbVie,respectively),Stelara has made it onto the 2026 pri

228、ce negotiation list for the IRA.However,as can be seen by looking ProductIndication 2022StelaraPsoriasis$3.4bnCrohns disease$3.8bnArthritis,psoriatic$0.1bnUlcerative colitis$2.4bnDarzalexMultiple myeloma$8bnInvega SustennaSchizophrenia$3bnTremfyaPsoriasis$2.7bnXareltoThrombosis,deep vein(DVT)$1bnStr

229、oke prophylaxis,secondary to atrial fibrillation(AF)$1bnStroke prophylaxis,secondary to acute coronary syndrome(ACS)$0.5bnOtherOther$24.3bn2021DarzalexInvega TrinzaErleadaStelaraTremfyaAdditional sales in 2022 versus 2021.$53.6bn$54.5bn$1.95bn$0.63bn$0.59bn$0.59bn$0.54bn$55.9bn20222023(estimated)J&J

230、s Pharma SalesTop 2022 Growth Drivers2022 Sales Portfolio BreakdownSource:Scrip 100;Evaluateat J&Js drivers over 2022,Darzalex is shaping up to be a fine replacement lead asset.The anti-CD38 antibody grew by nearly$2bn over 2022 and current forecasts show that growth rate continuing for at least ano

231、ther five years.In fact,Darzalex is currently predicted to be the fifth bestselling drug in the world come 2028,according to Evaluate.Johnson&Johnson is,according to consensus,due to have another year of growth in 2023 with estimates showing around$1.5bn in new sales.This increase is largely driven

232、by the growth of Darzalex but also from continued increase in sales from products such as the anti-psoriasis injectable Tremfya and prostate cancer hormone treatment Erleada.Merck&Co:the company benefited from sales of its COVID-19 antiviral Lagevrio in 2022,but to a lesser extent than Pfizer did fr

233、om sales of Paxlovid,which quickly emerged as the market leader in the US.Lagevrio generated$5.68bn in 2022,delivering a substantial boost to the companys pharmaceutical revenues,which grew 22%,to$52bn.The company also had a solid performance within its base business.Excluding Lagevrio,Mercks consol

234、idated sales,including animal health,still grew 12%,on the continued strength of the oncology cornerstone Keytruda,which grew 22%and the vaccine Gardasil,which also grew 22%,rebounding after vaccinations had waned during the pandemic.That solid performance pushed Merck into the number four slot on t

235、he Scrip 100,leapfrogging Bristol Myers Squibb,Roche and Novartis,from its number seven ranking last year.The jump represents Mercks highest rank on the leaderboard since 2018,when it also ranked fourth,and the company appears well positioned for another year of solid growth in 2023.Keytruda continu

236、ed to deliver success for the company in 2022 and shows no sign of slowing down as it smashed its second-quarter expectations in 2023.The PD-1 inhibitor raked in almost$21bn in sales over 2022 and with the reducing demand for COVID-19 products will likely retake its crown as the best-selling drug in

237、 2023.Mercks go-wide strategy with Keytruda has certainly paid off as the drug was,at the end of 2022,approved across 38 settings.This continued expansion allowed for Keytruda to remain one of Mercks biggest growth drivers adding a further$3.8bn of new sales in 2022.The HPV vaccine Gardasil has also

238、 turned out to be a major asset for Merck.The vaccine brought in nearly$6.9bn in 2022 a 23%increase from 2021.Gardasil was also reported to be outperforming guidance in the Q2 2023 results.These sales are largely driven by increased demand from outside the US.In particular there has been a surge of

239、sales in China.Despite the better-than-expected performance for Mercks top products current forecasts for 2023 show that in terms of top-line pharma sales next year may bring modest growth for Merck.The company is predicted to post around$52.8bn in pharma sales.As with many other companies making sa

240、les from COVID-19 related products the reduced demand for these assets will impact Merck.Lagevrios sales are expected to fall by$4.7bn next year which is the biggest brake for Mercks sales.In addition,forecasts show sales losses of around 30%for both Januvia and Janumet.2021LagevrioKeytrudaGardasilV

241、arivaxVerquvo$45.3bn$52bn$4.7bn$3.8bn$1.2bn$0.3bn$0.2bn$52.8bn20222023(estimated)Merck&Cos Pharma SalesTop 2022 Growth Drivers2022 Sales Portfolio BreakdownProductIndication 2022KeytrudaNon-small cell lung cancer(NSCLC)$10.1bnMelanoma$2.3bnHead&neck cancers$1.8bnRenal cell carcinoma(RCC)$1.6bnBladde

242、r cancer$1.3bnBreast cancer$1.1bnGastro-intestinal adenocarcinoma$1.1bnColorectal cancer$0.9bnOesophageal cancer$0.3bnPancreatic cancer$0.2bnHodgkin lymphoma$0.1bnNeuroendocrine tumour$0.1bnUterine cancer$0.1bnCervical cancer$0.0bnHepatoma,liver cancer$0.0bnSquamous cell carcinoma$0.0bnGardasilCervi

243、cal cancer prophylaxis$6.9bnLagevrioCOVID-19 treatment$5.7bnJanuviaDiabetes,type 2$2.8bnJanumetDiabetes,type 2$1.7bnOtherOther$10.0bnUlcerative colitis$0.1bnArthritis,psoriatic$0.2bnOtherOther$16.8bnSource:Scrip 100;EvaluateNovartis:of the top five Scrip 100 companies by pharma sales Novartis has mo

244、st diversity in its sales-the Swiss companys revenue is not as dominated by one or two products.At the end of 2022 it highlighted eight core drugs marked for multi-blockbuster potential.Two of these,Cosentyx and Entresto,are already its top-selling drugs,each making up around 9%of total sales.Cosent

245、yx,the anti-IL-17 monoclonal antibody,brought in$4.8bn over the course of 2022 and was Novartiss biggest earner.Nevertheless,the immunosuppressant does not feature in the top growth drivers for 2022 the company stated that growth for Cosentyx was partly offset by high US revenue deductions.However,c

246、urrent consensus forecasts show a return to growth for Cosentyx in 2023.Novartiss second biggest seller for 2022 was the chronic heart failure drug Entresto.Entresto earned$4.6bn over the year and was also the companys biggest growth driver,adding$1.1bn in new sales.According to Evaluate data Novart

247、is is forecast to be 2023s top company in the cardiovascular space by sales,bringing in$8bn,of which$6bn are derived from Entresto.Kesimpta was another high-growth product for Novartis over 2022.The multiple sclerosis drug,launched in September 2020,saw growth of$700m during 2022 and is also forecas

248、t to 2021$51.2bn$50.1bn$53.5bn20222023(estimated)Novartiss Pharma SalesAdditional sales in 2022 versus 2021.22|In Vivo|December 2023 SCRIP 100 SCRIP 100be one of Novartiss biggest growth drivers for 2023 with an annual growth of 83%.2023 is currently forecast to be a year of large growth for Novarti

249、s with pharma sales of over$55bn predicted.This lift,driven by many of the products already mentioned,will push the company up the rankings and into the top three as things stand.Novartis has,in the past few years,had to battle investor concerns over impending patent expiries but seems to be coming

250、out the other side with a broad pipeline of innovative medicines promising growth.Still,the companys pharmaceutical business is poised to become even smaller going forward with the recent spinout of ProductIndication Level 32022CosentyxPsoriasis$3.6bnAnkylosing spondylitis$0.6bnArthritis,psoriatic$0

251、.6bnEntrestoChronic heart failure(CHF)$4.6bnPromactaThrombocytopaenic purpura,idiopathic(ITP)$1.2bnHepatitis C induced thrombocytopaenia$0.9bnGilenyaRelapsing-Remitting MS(RRMS)$2.0bnTasignaLeukaemia,chronic myeloid(CML)$1.9bnOtherOther$24.8bn2022 Sales Portfolio BreakdownSource:Scrip 100;EvaluateEn

252、trestoKesimptaKisqaliPluvictoScemblix$1.1bn$0.72bn$0.29bn$0.27bn$0.15bnTop 2022 Growth Driversits Sandoz generic drug unit into a separate company,which will leave Novartis positioned as a leaner innovative biopharma.Sandoz generated$2.3bn in 2022,so the separation could leave Novartis facing anothe

253、r decline on the leaderboard the following year.Sandoz was spun out of Novartis in October 2023 as a standalone generics and biosimilars specialist.Climbing The Leaderboard AstraZeneca was another standout on the Scrip 100,moving up one spot to number eight on the leaderboard on 18%pharmaceutical re

254、venue growth,with growth coming partly from a full year of sales from Alexion Pharmaceuticals added to the top line.Alexion sales were added to AstraZenecas revenues for the first time as of July 2021,following the completion of the acquisition.AstraZenecas COVID-19 vaccine,Vaxzevria,while not autho

255、rized in the US,was still used in other markets and added$3.92bn to the balance sheet in 2021,so a tapering off in 2022 remained a headwind as seen with other COVID-19 vaccine developers.Novo Nordisk was one winner that moved up the leaderboard from number 16 to number 15 without any COVID-19 presen

256、ce to impact sales.The companys strong double-digit growth came solely from business fundamentals,driven by its strength in diabetes and obesity.Unable to keep up with the demand for its semaglutide franchise branded as Ozempic and Wegovy the company appears on track to continue the climb up the lea

257、derboard in the years ahead.Moving DownMercks rise to number four pushed Novartis,Roche and BMS each down one slot to number five,six and seven respectively,on the Scrip 100.The change reflects a gradual descent for Novartis and Roche,which ranked as the number one and two biggest pharmaceutical com

258、panies in the world,respectively,on the Scrip 100 two years ago.Last year,they ranked fourth and fifth respectively,as Pfizer and AbbVie assumed top spots.All three companies have experienced slower growth recently,with Roche and BMS being impacted by the loss of exclusivity of big-selling brands li

259、ke Roches Avastin and Herceptin and BMSs Revlimid and Roche also experiencing a headwind in 2022 from lower sales of Actemra,which was used during the pandemic to treat severe COVID-19.The Next Leaderboard A look at the top 10 prediction for full year 2023s pharma sales shows there is little shake-u

260、p.Pfizer is still predicted to lead the market although the gap is closing from 2022.Whilst positions two to five are still occupied by the same companies as in the 2022 rankings there has been some shuffling as to who comes in where.Novartis is predicted to take third place whilst Merck,although ow

261、ning the biggest predicted drug in 2023,may potentially fall back to fifth.However,many eyes are now on the two companies at the bottom of this top 10.Novo Nordisk and Eli Lilly are experiencing massive growth due to demand for their type 2 diabetes and obesity drugs and they may well feature much h

262、igher come 2024 and beyond.See more data onlinehttps:/ 2024The Scrip 100 universe gathers 2022 financial performance data and compares the activities of the top biopharma businesses,ranked by pharmaceutical sales.Top 10 Companies By R&D Spend20202021NovartisPfizerPfizerRocheAbbVieAbbVieAbbVieJ&JJ&JJ

263、&JNovartisMerck&CoMerck&CoRocheNovartisBMSBMSRochePfizerMerck&CoBMSSanofiSanofiAstraZenecaGSKAstraZenecaSanofiTakedaGSKGSK202212345678910Roche12345678910PfizerMerck&CoAstra-ZenecaBMSNovartisEli LillySanofiBayerJohnson&Johnson$14,727m$14,603m$13,829m$11,818m$9,762m$9,509m$8,485m$7,191m$7,067m$6,925mA

264、sia 45Europe 30RoW 3US 22Count Top 100Asia 1Europe 9RoW 0US 10Count Top 20WHO GETS IN?Top 100 companies based on pharmaceutical sales only for fiscal year 2022Combined Pharma Sales Of Top 100Combined Pharma Sales Of Top 20 1,224,693Number Of People Employed By Top 20Combined Pharma SalesPfizer domin

265、ated the Scrip 100 rankings of the top pharmaceutical companies in the world based on full year 2022 pharmaceutical revenues,driven by its COVID-19 success.COVID-19 BoostRoche remains top of the table for another year,but it spent less on R&D compared to FY 2021.Sumitomo Dainippon Pharma,GSK and Abb

266、Vie dropped out of the top 10 list.$755.0bn$1,036.1bnPharma Sales RankingAdditional sales in 2022 versus 2021.24|In Vivo|December 2023December 2023|In Vivo|25 SCRIP 100 SCRIP 100Scrip 100 RankingCompanyCountryPharma Sales($m)1PfizerUnited States98,9882AbbVieUnited States58,0543Johnson&JohnsonUnited

267、States52,5634Merck&CoUnited States52,0055NovartisSwitzerland48,2746RocheSwitzerland47,7457Bristol Myers SquibbUnited States46,1598AstraZenecaUnited Kingdom44,3519SanofiFrance39,95610GlaxoSmithKlineUnited Kingdom36,27411TakedaJapan30,84212Eli LillyUnited States28,54113Gilead SciencesUnited States27,2

268、8114AmgenUnited States26,32315Novo NordiskDenmark25,06516BayerGermany20,28717Boehringer IngelheimGermany19,45418Moderna,Inc.United States18,43519BioNTech SEGermany18,24120ViatrisUnited States16,21821TevaIsrael14,92522Regeneron PharmaceuticalsUnited States12,17323AstellasJapan11,62924BiogenUnited Sta

269、tes10,17325CSLAustralia10,13626Vertex PharmaceuticalsUnited States8,93127Otsuka PharmaceuticalJapan8,71428Fresenius SE&Co.KGaAGermany8,27229Merck KGaAGermany8,26130Bausch HealthCanada812431EisaiJapan5,70132Sun PharmaceuticalIndia5,51233UCBBelgium5,41634Grifols,S.A.Spain5,27535ServierFrance5,13836Abb

270、ott LaboratoriesUnited States4,91237Shanghai Fosun Pharmaceutical GroupChina4,56938Sino BiopharmaceuticalHong Kong4,28439Sumitomo Dainippon PharmaJapan4,25440Mitsubishi Tanabe PharmaJapan4,10041STADAGermany4,00142Shanghai Pharmaceutical Group Co.,Ltd.China3,98343CSPC Pharmaceutical Group Ltd.Hong Ko

271、ng3,90844Asahi Kasei PharmaJapan3,80545Jazz PharmaceuticalsIreland3,65946Horizon Therapeutics plcIreland3,62947IncyteUnited States3,39448IpsenFrance3,18849Jiangsu Hengrui Medicine Co.Ltd.China3,13950Dr ReddysIndia3,13151AurobindoIndia2,983Scrip 100 RankingCompanyCountryPharma Sales($m)52CiplaIndia2,

272、89853ChiesiItaly2,89754Sichuan Kelun PharmaceuticalChina2,81555LundbeckDenmark2,58456Joincare Pharmaceutical Group Industry Co.,Ltd.China2,55257Ferring PharmaceuticalsSwiterland2,39958Samsung BioLogicsSouth Korea2,33159Endo InternationalIreland2,31960OnoJapan2,25961Shandong Buchang Pharmaceuticals C

273、o.,Ltd.China2,22662Zydus Lifesciences(earlier Cadila Healthcare)India2,21963Amneal PharmaceuticalsUnited States2,21264Baxter InternationalUnited States2,12665SantenJapan2,07466LupinIndia2,07267Genmab A/SDenmark2,06768Harbin Pharmaceutical Group Co.,Ltd.China2,05569BioMarin PharmaceuticalUnited State

274、s2,04770Hikma PharmaceuticalsUnited Kingdom2,04371Seattle Genetics Inc.(Seagen)United States1,96272RecordatiItaly1,95373Kyowa Hakko KirinJapan1,94474United TherapeuticsUnited States1,93675MallinckrodtIreland1,91476Livzon Pharmaceutical GroupChina1,88077Shijiazhuang Yiling Pharmaceutical Co.,LtdChina

275、1,86578Swedish Orphan Biovitrum ABSweden1,86579KRKASlovenia1,80980GrunenthalGermany1,79181CelltrionSouth Korea1,77482Glenmark PharmaceuticalsIndia1,65483Topcon Corp.Japan1,65184ExelixisUnited States1,61185TowaJapan1,59986SawaiJapan1,53487Meiji HoldingsJapan1,51188Leo PharmaDenmark1,50789Sinovac Biot

276、ech Ltd.China1,49390Neurocrine Biosciences,Inc.United States1,48991BioconIndia1,41092CR Double-Crane Pharmaceuticals Co.,LtdChina1,40693Jiangsu Hansoh PharmaceuticalChina1,39794Yuhan CorpSouth Korea1,37995GC Biopharma(Green Cross)South Korea1,32996BeigeneChina/Cayman Islands1,25597Shenzhen Hepalink

277、Pharmaceutical Group Co.,Ltd.China1,23598KPC Pharmaceutical Inc.China1,23399Zhejiang Huahai Pharmaceutical Co.,Ltd.China1,219100Torrent PharmaceuticalsIndia1,215The Scrip 100 ranking is based on Citelines analysis of fiscal year 2022 prescription pharmaceutical sales data for the top 100 biopharmace

278、utical companies.For more information contact:Lucie.E.ICON is the worlds largest pure-play clinical research organisation.From molecule to medicine,we advance clinical research by providing outsourced development and commercialisation services to pharmaceutical,biotechnology,medical device and gover

279、nment,and public health organisations.We develop new innovations,drive emerging therapies forward and improve patient lives.ICON offers the most comprehensive suite of integrated clinical development services in the industry.Weve designed fully customisable solutions to help our customers achieve th

280、eir goals across a seamless delivery model,spanning their products entire lifecycle.Our focus is on delivering Healthcare Intelligence to customers to address the full spectrum of clinical development challenges,not just point-of-service delivery.The synthesis of our experience,expertise,best practi

281、ces,technology and data provides patient centric processes,commercially optimised for global success,and is driving transformation of trials to improve R&D ROI.ICON has established relationships with a majority of the worlds top pharmaceutical and biotech companies,offering:Globally scaled expertise

282、 and solutions for all customers and patients.ICON is:World leader in Functional Service Provision(FSP)Global number 2 in full-service Ph 2/3 Clinical Research Global number 2 in Early Phase Clinical Research Global number 3 in Late Phase&RWE Global number 4 in Central&Speciality Laboratory Services

283、 Clinical focus:no ownership from parent organisation and no distractions from near adjacencies,means we are completely committed to customers clinical development programmes.Flexible partnership models and governance structures ensure transparent communications.Regardless of the size of your organi

284、sation or your project,we work your way.Strategy-driven delivery:At ICON,we know that clinical research now requires a more comprehensive,strategy-driven delivery.Our approach is to proactively guide clients towards the most effective,efficient solution across all modalities of research.We commence

285、client engagements with a strategic consultation,delivered by our team of over 700 consultants,to identify custom solutions,eliminate process white space and identify efficiencies and savings.This delivers across three areas:Robust asset development consulting for a deeper understanding of developme

286、nt pathways,asset acquisition/transfer options and access to scientific expertise across disciplines for ad hoc support.A network of international experts delivering superior regulatory and quality assurance strategies,submissions,and support to expedite drug and medical device development and manag

287、e ongoing global compliance.Integrated solutions to demonstrate the value of products and support global brand success from dedicated real-world evidence,pricing,market access,reimbursement,health economics,and medical communications experts.This consultative-led approach,the breadth of our capabili

288、ties and advanced digital and data capabilities enables us to deliver seamless,integrated services.Consultative lead engagementAsset DevelopmentRegulatoryCommercial PositioningSeamless,integrated service deliveryAdvanced digital and data capabilitiesEmpirically-based guidance and built-to-spec model

289、s and solutions across every phase of clinical research and lifecycle managementIntelligent,informed decisions to drive actions that accelerate the development of drugs and devicesThe worlds leading CROPowered by Healthcare IntelligenceGovernmentBiotechPharmaMedical DevicePublic HealthConsumer Healt

290、hDecentralised clinical trial:Clinical research should engage with patients wherever they are.ICON has all the service components to deliver DCTs and the experience and expertise to provide integrated,customised solutions.Our integrated decentralised clinical trial solutions can help customers to ac

291、hieve better outcomes,while maximising recruitment and retention of diverse patient populations.Site and patient recruitment:Our site networks,patient recruitment expertise,in-home services and site resourcing services unlock access to millions of patients.Patients are at the heart of everything we

292、do at ICON.We provide the most comprehensive and connected patient journeys across the largest and most diverse patient populations.ICON streamlines the clinical trial process,accelerating study startup,and ensuring patient recruitment and retention meet or exceed targets.ICON offers customers enhan

293、ced access to a larger global pool of more diverse patients through its global site network(Accellacare),specialised oncology network(Oncacare),a paediatric site network,in-home services and a network of five Phase I clinical research units across the United States and Europe.Speed to market:An exte

294、nsive services portfolio,digital and technology capabilities,combined with flexible delivery and models allow us to reduce development time and costs.Quality:The quality of our work is vital to our mission of bringing better medications to patients around the world.We are committed to maintaining,su

295、pporting,checking,and improving our quality systems to exceed the quality standards demanded by our clients,patients and regulatory authorities.ICONs Quality Management System(QMS)comprises our mechanisms for ensuring that all our services are performed to the highest ethical standards,conform to al

296、l relevant regulatory requirements and satisfy contractual obligations.Emerging therapies:ICON offers deep experience in the unique challenges of developing emerging treatments such as immuno-oncology and other cell and gene therapies,with several approved treatments already on the market.Advanced d

297、igital and data capabilities:Innovation at ICON is focused on the factors that are critical to our clients.Investing heavily in AI,machine learning and RPA capabilities,our portfolio of digital solutions and platforms are specifically developed for the needs for clinical research.Our enterprise clin

298、ical informatics framework ingests,integrates and interrogates the full spectrum of data sources,and assets for rich,real-time data-visualisation and actionable insights to significantly enhance the efficiency and productivity of clients development programmes.Data insights can include experiential

299、data paired with external data,benchmark data on different models of research,predictive algorithms and continuous performance evaluation,whilst tokenisation of data extends reusability.ICON is driving transformation of trials to improve R&D ROI and support a future landscape of many more trials del

300、ivered in shorter timeframes,but conversely of greater operational and scientific complexity.We strategically and proactively solve todays challenges without losing sight of their impact tomorrow.ICON28|In Vivo|December 2023December 2023|In Vivo|29 BUSINESS BUSINESSLongitudinal Study BackgroundSince

301、 2014,Catenion have annually assessed R&D productivity and corporate growth of the top biopharma companies,assembling a unique dataset to address fundamental questions around performance and success models.While our annual review focuses primarily on the performance of individual companies,in this a

302、nalysis we dissected the patterns that have played out over longer periods of time identifying groups of companies that belong to similar performance phenotypes.To provide the full longitudinal view,this study only includes companies that were consistently in our top 30 rankings over the past 10 yea

303、rs.Six Different Performance Phenotypes Identified Our longitudinal analysis uncovered six different performance phenotypes(See Exhibit 1).To evaluate the impact of these phenotypes on company performance,we created an exchange-traded fund(ETF)for each phenotype,giving equal weight to each company(S

304、ee Exhibit 2).Almost all ETFs achieved an upside compared to the start of our rankings in 2014 with significant gains over the Standard&Poors(S&P)500 pharma index,which failed to increase value over the 10-year period.The value increases per ETF reflect our ranking and phenotypes,with the strongest

305、performance observed for the clusters League Of Its Own and Turn-Arounds.Novo In A League Of Its OwnNovo Nordisk is the only company that consistently featured in the top five since 2014,as such defining a league of its own.Novo was in the right place at the right time with its focus on diabetes and

306、 peptide-based drugs,in an era where diabetes and its co-morbidities have reached epidemic proportions.Novo is also the company that has historically relied the most on internal strengths.Internally developed assets such as semaglutide and its various formulations account for over 90%of value.The gl

307、ucagon-like peptide-1(GLP-1)agonist class is the gift that keeps on giving.After type 2 diabetes mellitus and obesity,there has recently been a Phase III win in chronic kidney disease that could have a huge impact on how it is treated.We wait and see whether this unprecedented run of success can out

308、live Novos hypergrowth,as Novo is expected to double in size from 2022 to 2026.One thing is guaranteed:Novo will not end up as a takeover candidate,as the majority of voting shares are owned by Novo Holdings A/S that itself is owned by the non-profit Novo Foundation.Despite failures to enter new the

309、rapeutic areas(TAs),Novo keeps trying new things illustrated by recent significant mergers and acquisition(M&A)deals,including RNA company Dicerna for$3.3bn in 2021 and small molecule-focused Forma Therapeutics for$1.1bn in 2022.Investments in Novo stock back in 2014 would have generated a return of

310、 563%until now(See Exhibit 2).Big Pharma Turn-Arounds:AstraZeneca&Eli LillyIn the start of our ranking,both Eli Lilly and AstraZeneca(AZ)were underperforming big pharma companies with an uncertain future.The fate of AZ changed around 2017 when it broke into the top 10,culminating at#1 in 2019.For AZ

311、,the solution had not only been a strong re-focusing on a few TAs,such as oncology,cardio-metabolic and respiratory and recently rare diseases through the Alexion acquisition but an equally strong focus on governance and decision-making.Its 5R decision framework has since served as a template for ma

312、ny other struggling large pharma organizations.AZ is also a prime example of a company that never completely gave up on internal research,while emphasizing deals and M&As in parallel.Both high-value assets Tagrisso and Imfinzi have not been in-licensed.The solution for Lilly has been its focus on me

313、tabolic diseases and creating an industry-leading GLP-1 agonist franchise,competing with Novo.Ironically,Lillys success can be attributed to indications that were viewed as financially toxic by investors just a few years ago,such as obesity and Alzheimers disease.Investment in both companies in 2014

314、 would have returned an astonishing 572%by now(See Exhibit 2).Failed To Scale:Gilead&BiogenThe Failed To Scale phenotype includes Gilead and Biogen,but one could also add Celgene,as without the acquisition by Bristol Myers Squibb(BMS),Celgene would have faced the same challenge.All three companies h

315、ad a very focused model at one point that led to their success.Gilead focused on virology,Biogen on multiple sclerosis(MS)and Celgene on hematologic cancers.After contributing to curing hepatitis C(HCV)through drugs such as sofosbuvir,Gilead started to seek growth in the oncology area,and embarked o

316、n expensive acquisitions,including Kite Pharma for$11.9bn in 2017 and Immunomedics for$21bn in 2020.Simultaneously,it faced increasing growth challenges in its core business of human immunodeficiency virus(HIV)and HCV.The company contributed to the fight against COVID by launching the nucleotide ana

317、logue remdesivir,but sales are declining rapidly as COVID has become endemic.Biogen also wanted to diversify beyond its historic focus on MS into Alzheimers disease.After some missteps,it finally found its stride with the approval of lecanemab.The more interesting strategic question is:why is it so

318、hard to scale R&D productivity when companies go through periods of hypergrowth?Some answers lie in the fact that creative functions often suffer as organizations grow larger and require more bureaucracy and processes.Additionally,decision-making relies more on“aligned”and highly polished views as c

319、ompanies add hierarchical layers.The Recent Ups:Daiichi Sankyo,Eisai&PfizerFor Pfizer,the recent upsurge after many years of underperformance is almost entirely driven by COVID and the success of its messenger RNA(mRNA)vaccine Comirnaty with BioNtech and its own protease inhibitor Paxlovid.Consideri

320、ng dwelling COVID sales we do not expect Pfizer to keep its position.Pfizer is an example of how difficult it is to scale innovation,itself being the result of decades of mega M&As.The comeback of Eisai is almost exclusively driven by Alzheimers,where the sustainability remains.Longitudinal Global B

321、iopharma R&D Productivity And Growth Ranking28|In Vivo|December 2023December 2023|In Vivo|29Markus Thunecke,Erika Kuchen and Alexander WallrothCONFIDENTIALExhibit 1 Performance phenotypes based on Catenions longitudinal R&D productivity rankingNovember 10,2023Recent UpsLaggardsIts Own League Failed

322、To ScaleTurn-AroundsUps&Downs130R&D rank(lower is better)Exhibit 1:Performance Phenotypes Based On Catenions Longitudinal R&D Productivity RankingsSource:CatenionCONFIDENTIALExhibit 2 ETFs of performance phenotypes anchored at the start of Catenions longitudinal studyNovember 10,2023Presentation tit

323、le 3Year68%572%563%-2%43%229%-1.6%Performance(%)60050040030020010002014201520162017201820192020202120222023Exhibit 2:ETFs Of Performance Phenotypes Anchored At The Start Of Catenions Longitudinal Analysis Source:CatenionIts Own LeagueTurn-aroundsRecent UpsUps And DownsLaggardsFailed To ScaleS&P 500C

324、ONFIDENTIALExhibit 2 ETFs of performance phenotypes anchored at the start of Catenions longitudinal studyNovember 10,2023Presentation title 3Year60050040030020010002014201520162017201820192020202120222023 BUSINESS30|In Vivo|December 2023December 2023|In Vivo|31 BUSINESS BUSINESSStrategically,the mos

325、t interesting company of this group is Japanese Daiichi Sankyo whose success is almost entirely down to one drug class antibody-drug-conjugates(ADCs).The success of Enhertu and the massive deal Daiichi struck with AZ on the asset speak for itself.Further validation recently came from Merck&Co,who pi

326、cked up three ADCs for a$4bn upfront payment.Of the three companies in the“Recent Ups”phenotype,Daiichi is the one most likely to remain in a top position over the next few years.The Largest Group Of All:Ups&Downs Throughout The YearsA disappointing performance comes from Novartis,demonstrating agai

327、n the challenges of maintaining top performance and innovation at that scale.When Novartis first set up shop in Boston with its Novartis Institute for Biomedical Research back in 2002,it claimed that it had“re-invented the grammar of drug discovery.”Twenty years later it is fair to say that these at

328、tempts have proven unsuccessful.Roche chose a different path compared to its Basel rival and decided to maintain two separate Research and Early Development organizations,one consisting of the former Genentech R&D organization,the other representing the former Roche.The decision not to integrate the

329、 two may be questioned by proponents of synergies,however economies scale do not really exist beyond a small threshold in creative functions.Since 2014,Roche had a much better overall performance compared to Novartis with even a few showings in the top 10 of the yearly R&D productivity ranking.Inves

330、tment into an equally weighted ETF of this phenotype would have achieved a modest+68%over the 10-year period,though still superior to the S&P pharma index.The LaggardsDespite comparably poor R&D productivity,several companies in this group nevertheless had a satisfactory company growth performance a

331、nd the ETF of these companies still delivered a+43%.There are a few factors that can help sustain performance in the absence of superior R&D productivity such as a resilient portfolio of marketed products consisting of biologics and products that have a second life in emerging markets,cost-cutting w

332、ith or without M&A,and a diversified business base.Common Factors Of OutperformersThe factors that most strongly correlate positively with R&D productivity in our longitudinal study are a high degree of therapeutic focus and a strong share of organically developed assets(See Exhibit 3).Irrespective

333、of the TAs chosen,a high degree of focus lays the foundation for good science by allowing R&D teams to build deep and competitive expertise.This expertise not only supports the development of organic assets but also the evaluation of external opportunities.Growth potential and focus can be achieved along several dimensions,such as TA,specific indications,modalities,and/or successive improvements o