生物制药行业展望：影响行业发展的关键趋势

生物制药行业在2021年实现了巨大发展。目前全球已有近50亿人接种了新冠疫苗¹，首批支持体内CRISPR疗法安全性和效果的临床数据也于去年发布²，全球有128家专注于治疗性产品的生物制药公司在全球接连市³。

展望未来，生物制药企业的管理者们将在商业化、融资以及产品组合规划等各个方面遇到一系列挑战和变革。本报告中，我们总结出五个将对生物制药行业产生巨大影响的关键趋势：

1. 先进疗法的增长

2. 全球定价压力持续存在

3. 商业化模式不断演变

4. 患者诊断和生物标志物检测增多

5. 融资格局的变化

L.E.K.对目前影响生物制药领域的全球动态、进展以及关键趋势进行了研究和分析：

先进疗法的增长

先进疗法在过去五年中实现了迅速增长，而在2021年，先进疗法更是实现多个“首次”：中国批准了首个CAR-T疗法；美国批准了首个靶向BCMA的CAR-T疗法⁴；Intellia公布了首批支持体内CRISPR疗法安全性和效果的临床数据；2021年基因治疗、细胞免疫治疗/细胞治疗和组织工程领域的融资创历史记录，筹集资金超231亿美元⁵。在2022年，临床、政策法规以及商业化等方面的里程碑式发展将对越来越多样化的先进疗法的发展轨迹产生不同的影响。

值得关注的关键进展包括：

• 吉利德（Yescarta）和百时美施贵宝（Breyanzi）正竞相将CAR-T应用于早期的大型B细胞淋巴瘤的治疗。吉利德已向美国食品药品管理局（FDA）、欧洲药品管理局（EMA）以及其他监管机构提交二线用药的申请⁶，百时美施贵宝也向FDA提交了二线用药的申请⁷。一旦获批，这些疗法的市场潜力将得到进一步扩大。

• 美国目前只有两种基因疗法获得了批准，但这一数字可能会在今年翻数倍，多个疗法可能会在今年进入审批里程碑。例如：蓝鸟生物正在等待FDA批准其β地中海贫血基因疗法（beti-cel）⁸以及脑肾上腺脑白质营养不良（CALD）基因疗法（eli-cel）⁹的生物制品许可申请；拜玛林用于治疗严重A型血友病的基因疗法（valoccogene roxaparvovec）目前正在接受EMA的审核，并可能会很快向FDA提交申请¹⁰；其他正在计划或可能向FDA和/或EMA提交申请的还包括用于治疗有输血依赖的β地中海贫血和严重镰状细胞病的CRISPR和Vertex的CTX001¹¹，以及CSL Behring和uniQure合作开发的用于治疗严重至中度血友病B的基因疗法etranacogene dezaparvovec¹²。这些临床申请能够为未来获批所需的安全性和有效性的要求提供参考。生物制药企业针对这些疗法的市场准入、商业化生产以及普及等方面所采取的下一步措施也将对该领域具有指导意义。

• RNA治疗管线再次兴起的趋势可能会在2022年得以延续。预计将会发布的后期临床试验结果包括：Alnylam的RNAi疗法Onpattro在转甲状腺素蛋白淀粉样变心肌病（ATTR-CM）中的作用¹³，以及Ionis和阿斯利康合作开发的配体偶联反义药物eplontersen在遗传性运甲状腺素蛋白淀粉样变性多发性神经病（hATTR-PN）中的作用¹⁴。此外，该管线还可以扩展到对RNA编辑、tRNA、环状RNA和mRNA等其他作用机制的研究中^15,16。

• 生物制药公司正越来越多地关注蛋白质降解，即通过PROTACs等小分子来破坏之前被认为难以在药理学上靶向的致病蛋白质¹⁷。从2020年到2021年，临床前和临床阶段的蛋白质降解剂管线增加了近70%¹⁸，而2022年该领域预计将持续进展。

以上所罗列的先进疗法的最新进展并不全面，例如我们还有望看到对异体细胞治疗的持续投资以及中国细胞和基因治疗管线的迅速增长，但这些案例都显示了先进疗法的显著进步，包括新机制的探索、新疾病领域的探索、新市场的加速发展以及商业化道路上重要先例的开创

全球定价压力持续存在

各国政府、支付方和消费者一直在对全球药品价格施加压力。美国的药品福利管理机构（PBMs）和支付方正在协商更大的折扣，自2018年以来，尽管名义出厂价上涨，但净价格却在下降¹⁹（图1）。支付方也一直在对他们认为过于昂贵的药品的医保覆盖范围进行限制，例如：美国医疗保险和医疗补助服务中心（CMS）最近提出对渤健的阿尔茨海默氏症药物Aduhelm的保险覆盖范围加以限制，只有参加了获批的临床试验的患者才适用²⁰。定价压力也可能来自于行业内新进入的竞争者，例如：以EQRx为代表的采取“低成本me-too”战略的公司计划将其产品的定价比竞争对手低50%-70%²¹。EQRx已经建立了至少5个已进入临床阶段的产品管线²²，并与CVS健康、Geisinger、加州蓝盾、北卡罗莱纳的蓝十字蓝盾、Horizon以及英国的NHS达成了合作或谅解备忘录²³。该公司提交第一批上市申请文件的时间仍有待观察，因为他们目前两个领先资产的关键临床数据，一个来自于中国的研究，另一个则来自于预计将于今年开始的美国的研究²⁴。此外，生物仿制药的管线也在加速进展，可能很快就会给众多重磅药物的特许经营带来更大的竞争。

拜登政府提出了一系列有关控制药品价格的政策建议，例如：医疗保险药品价格谈判、对价格涨幅高于通货膨胀的药物进行惩罚、让仿制药和生物仿制药更早加入竞争、提高回扣透明度^25,26。其中一些条款包括在最新的“重建美好未来”法案（Build Back Better bill）²⁷中，但由于国会两党僵持不下，这些条款能否获批、以何种形式获批都还是未知数。尽管如此，两党敦促药品定价改革的压力依然存在，改革可能会以某种形式小规模推进。

除了美国，全球各国政府也在2021年推出了新的控制药品价格的措施或计划。例如：德国新政府推出的一项计划将药品的自由定价期从12个月缩短至6个月，还增强了国家医疗保险限制药品价格的权力，并将报销药品价格冻结计划的期限延长至2022年以后²⁸；日本首次实施了“off-year”药物降价，并开始对昂贵的创新药物进行成本效益评估²⁹；通过与中国国家医疗保障局（NHSA）谈判新列入国家医保药品目录的药品平均降价61.7%，这是2017年以来幅度最大的降价³⁰。

展望未来，生物制药公司必须更加谨慎地去平衡价格和市场准入，而以价值为导向/按效付费合同等其他定价模式将是实现这一目标的重要工具。

商业化模式不断演变 

新冠疫情的暴发、医院系统的整合、制度决策的增多、新的先进治疗药物的出现等一系列综合因素正在不断促进生物制药公司商业化模式的演变。未来新产品上市预计将在以下方面进一步发展：

• 缩小销售团队，线上和线下的互动方式相结合。传统的以销售代表为主导的商业化模式正在被更灵活的方式所取代，即减少销售代表数量、增加线上接触点。新冠疫情的暴发加速了这一转变，这种趋势预计将得以持续：在GlobalData 2021年5月的一项调查中，66%的医生预计后疫情时代将会采用线下和线上相结合的互动方式³¹。制药公司也采取了相应的行动：安进去年裁减了500个工作岗位，主要是美国的销售人 员³²；今年1月辉瑞也紧随其后裁剪了销售人员，但并未公布裁减规模³³。这种转变意味着销售代表的角色需要重新定义，并且需要对他们进行再培训，从而带来更好地客户体验。人工智能和机器学习将被越来越多地用于支持这种互动方式，因为该类技术能够在现场报告模型完成之前就探测到会影响商业化部署决策的市场动态变化。成功将变得更加微妙，因此销售团队更加需要在正确的时机通过正确的渠道向客户分享清晰且有吸引力的内容，并考虑医生的喜好，同时保持联系。

• 更多地关注医疗系统而不是单个决策者。“客户”和“决策者”的定义已经发生了根本性转变，生物制药公司不得不重新审视其商业化模式。例如：美国医院系统不断整合，医生的比例越来越大，而决策者的比例则相反³⁴。因此，产品上市团队越来越多地将目标对准大型医疗系统中的机构决策者，诺华为其PCSK9抑制剂Leqvio所制定的上市策略就是一个很好的例子：为了确定适合使用该药物的患者，诺华更希望与美国约200家医院系统合作，而不是将重点放在作为决策者的单个医生身上³⁵。诺华公司还与英国NHS签订了一项与人口健康管理项目相关的商业协议（由Medicines公司签署，该公司后被诺华收购），为高风险患者提供这种药物³⁶。这种更协调、更中心化的规划策略预计将会变得越来越普遍。

• 动员定点治疗中心使用先进药物。细胞和基因疗法需要采取与传统小分子疗法和生物制剂完全不同的商业化模式。诺华、吉利德和Spark等早期进入者已经建立了定点治疗中心网络³⁷，以管控质量和不良事件，并监督相关的双向生产物流。这种模式的成功需要的步骤包括网站注册、激活、持续的支持以及强大的医学科学联系，但对传统的广泛销售策略的依赖要小得多。细胞和基因治疗领域预计将会在未来十年里愈渐成熟，例如：美国到2030年可能会有60种以上持久³⁸细胞与基因治疗产品适应症获批³⁹，届时公司必须改变以往的习惯，采取不同的方式动员治疗中心使用这些先进药物。

患者诊断和生物标志物检测增多

用于筛查、诊断、治疗选择和药物反应监测的生物标志物策略将成为药物上市策略中越来越重要的组成部分。过去五年中的四年，个性化药物在获得FDA药物评估和研究中心（CDER）批准的治疗性新分子实体中至少占到了三分之一，而在2005年这一比例仅为5%（图2）⁴⁰。

在肿瘤学领域，生物标志物在患者旅程中的作用正变得越来越复杂和精妙：

• 随着液体活检的进步，更广泛的筛查和早期诊断成为可能。例如：Exact Sciences⁴¹和Grail⁴²正在开发或已经开发出基于血液的多癌早期检测方法。这种检测方法的广泛应用能够帮助生物制药公司让进入治疗模式的患者人数增加。

• 液体活检技术的进步也可能为辅助治疗带来更好的反应监测，并可能催生新的治疗方案。例如：美国CMS也正在覆盖基因检测公司Natera的Signatera方法，该方法通过ctDNA检测和分析对微小残留病变（MRD）进行监测，用于II-III期结直肠癌的术后，以防出现复发。使用该方法的诊断平均要比使用现有方法的诊断早8.7个月⁴³。

• 在治疗选择方面，肿瘤类型正在根据生物标记物的不同细分为更小的亚型。NSCLC就是最典型的例子，另外在乳腺癌、前列腺癌和结肠直肠癌领域中也有这种趋势。肿瘤亚型治疗的管线可能会继续扩大，而随着使用相关疗法的患者群体变小，生物制药公司可能需要制定更高的价格来支撑收入潜力。

生物标志物也将扩展应用到肿瘤以外的领域，尤其是神经、免疫和心血管等疾病领域。例如：在早期甚至出现症状之前，为阿尔茨海默病和非酒精性脂肪性肝炎患者提供负担得起的非侵入性检测是非常有必要的。诊断公司正在努力开发这样的检测，生物制药公司也开始将其纳入临床试验。例如：C2N Diagnostics的血液检测 PrecivityAD能够识别阿尔茨海默氏症患者的脑淀粉样斑块状态，该检测正被用于卫材的一项关于阿尔茨海默病药物lecanemab（一种抗淀粉样单克隆抗体）的临床前试验中⁴⁴。

生物制药产品上市成功的关键因素中，与诊断公司建立伙伴关系、确保检测被医保覆盖、促进检测的普及以及对医生和患者的教育将变得越来越重要。从转化医学阶段开始，产品规划团队必须将诊断策略明确纳入其开发和上市计划中，并且只有在为目标患者群体找到价格合理、易于注射、经过验证且被广泛采用的生物标志物以后才能退出。

融资格局的变化

西方市场的广泛共识似乎是政府的财政支持将减少，鼓励社会资本以及其他融资方式。2020年和2021年见证了全球生物科技企业的IPO热潮，而在2022年该领域的IPO活动预计将放缓⁴⁵。2021年美国生物科技公司的IPO中，约80%去年年底的价格低于发行价，纳斯达克生物科技指数在2021年没有产生任何回报，而标准普尔500指数的回报率则高于25%⁴⁶。许多特殊目的收购公司（SPAC）表现不佳，美国证券交易委员会（SEC）可能会出台新规，以消除SPAC的部分优势⁴⁷。随着美国股票市场面临挑战，我们预计风险资本投资将保持强劲势头，特许权货币化、债权融资等其他非摊薄融资选择预计将增加。

香港交易所已成为全球第二大生物科技公司融资中心⁴⁸。自2018年推出新的上市制度以来，该公司的IPO规模大幅增加，但随后其上市制度在2021年下半年经历了一次调整，可能会影响到2022年的公开融资。亚洲风险资本市场可能会继续保持增长势头，其他融资机制预计仍将较为少见。

结论

综合来看，这些趋势正在推动整个医疗生态系统的根本性转变。为了在不断变化的市场环境中获胜，行业领导者必须对这些趋势进行监测，并调整其公司战略，以做出与组合投资、新产品上市策略、融资以及如何与行业参与者展开合作等方面相关的决策。L.E.K.一直以来都在密切关注所有关键趋势的发展，能够帮助广大生物制药公司有效解决其战略需求。

如欲了解更多，请联系lifesciences@lek.com。

尾注
¹The New York Times. “Tracking Coronavirus Vaccinations Around the World.” Accessed Feb. 11, 2022. https://www.nytimes.com/interactive/2021/world/covid-vaccinations-tracker.html 

²Intellia Therapeutics. “Intellia and Regeneron Announce Landmark Clinical Data Showing Deep Reduction in Disease-Causing Protein After Single Infusion of NTLA-2001, an Investigational CRISPR Therapy for Transthyretin (ATTR) Amyloidosis.” https://ir.intelliatx.com/news-releases/news-release-details/intellia-and-regeneron-announce-landmark-clinical-data-showing

³Nature Reviews Drug Discovery. “No shortage of fresh IPO capital in 2021.” https://www.nature.com/articles/d41573-022-00018-0 

⁴Alliance for Regenerative Medicine. “Regenerative Medicine in 2021: A Year of Firsts and Records.” http://alliancerm.org/wp-content/uploads/2021/08/ARM-H1-2021-Report.pdf 

⁵Alliance for Regenerative Medicine. “Regenerative Medicine: New Paradigms.” https://alliancerm.org/wp-content/uploads/2022/01/SOTI-Presentation-FINAL.pdf

⁶Gilead Sciences. “Yescarta® CAR T-Cell Therapy Quadruples Median Event-Free Survival Duration Over Standard of Care in Second-Line Relapsed or Refractory Large B-Cell Lymphoma.” https://investors.gilead.com/news-releases/news-release-details/yescartar-car-t-cell-therapy-quadruples-median-event-free

⁷Bristol Myers Squibb. “U.S. Food and Drug Administration (FDA) Accepts for Priority Review Bristol Myers Squibb’s Supplemental Biologics License Application for Breyanzi (lisocabtagene maraleucel) as a Second-Line Therapy for Relapsed or Refractory Large B-cell Lymphoma. https://news.bms.com/news/corporate-financial/2022/U.S.-Food-and-Drug-Administration-FDA-Accepts-for-Priority-Review-Bristol-Myers-Squibbs-Supplemental-Biologics-License-Application-for-Breyanzi-lisocabtagene-maraleucel-as-a-Second-Line-Therapy-for-Relapsed-or-Refractory-Large-B-cell-Lymphoma/default.aspx

⁸bluebird bio. “bluebird bio Submits Biologics License Application (BLA) to FDA for betibeglogene autotemcel (beti-cel) Gene Therapy for Patients With ß-Thalassemia Who Require Regular Red Blood Cell Transfusions.” https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-submits-biologics-license-application-bla-fda

⁹bluebird bio. “bluebird bio Announces FDA Priority Review of Biologics License Application for eli-cel Gene Therapy for Cerebral Adrenoleukodystrophy (CALD) in Patients Without a Matched Sibling Donor.” https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-announces-fda-priority-review-biologics-license-0

¹⁰BioMarin Pharmaceutical. “BioMarin Announces Stable and Durable Annualized Bleed Control in the Largest Phase 3 Gene Therapy Study in Adults with Severe Hemophilia A; 134-Participant Study Met All Primary and Secondary Efficacy Endpoints at Two Year Analysis.” https://investors.biomarin.com/2022-01-09-BioMarin-Announces-Stable-and-Durable-Annualized-Bleed-Control-in-the-Largest-Phase-3-Gene-Therapy-Study-in-Adults-with-Severe-Hemophilia-A-134-Participant-Study-Met-All-Primary-and-Secondary-Efficacy-Endpoints-at-Two-Year-Analysis 

¹¹Vertex Pharmaceuticals, accessed on Seeking Alpha. “Vertex Pharmaceuticals Incorporated’s (VRTX) CEO Reshma Kewalramani Presents at 40th Annual JPMorgan Healthcare Conference — Transcript.” https://seekingalpha.com/article/4478807-vertex-pharmaceuticals-incorporatedsvrtx-ceo-reshma-kewalramani-presents-40th-annual 

¹²CSL Behring. “uniQure and CSL Behring Announce Primary Endpoint Achieved in Hope-B Pivotal Trial of ETRANACOGENE DEZAPARVOVEC Gene Therapy in Patients with Hemophilia B.” https://www.cslbehring.com/newsroom/2021/hope-b-gene-therapy-for-hemophilia-b-topline-results 

¹³Alnylam Pharmaceuticals. “40th Annual J.P. Morgan Healthcare Conference.” https://alnylampharmaceuticalsinc.gcs-web.com/static-files/a152a390-748b-46df-92bc-5c064c0bad3c 

¹⁴Ionis Pharmaceuticals. “Corporate Overview.” https://ir.ionispharma.com/static-files/ebc240f3-749c-436c-93cd-53a03ae8c425. “AstraZeneca and Ionis sign deal to develop and commercialise eplontersen.” https://www.astrazeneca.com/media-centre/press-releases/2021/astrazeneca-ionis-to-collaborate-on-eplontersen.html

¹⁵Pharmaceutical Technology. “Mapping the RNA Therapeutics R&D Landscape in 2022.” https://www.pharmaceutical-technology.com/analysis/mapping-the-rna-therapeutics-rd-landscape-in-2022/ 

¹⁶Chemical and Engineering News. “RNA-Editing Race Intensifies as Big Pharma Buys In.” https://cen.acs.org/pharmaceuticals/drug-discovery/RNA-editing-race-intensifies-Big/99/i39

¹⁷Drug Discovery Today. “The Rise and Rise of Protein Degradation: Opportunities and Challenges Ahead.” https://www.sciencedirect.com/science/article/pii/S1359644621003640 

¹⁸Informa Pharma Intelligence. Citeline. “Pharmaprojects.” Accessed January 2022. https://citeline.informa.com/; Query: “Mechanism Of Action is Protein degrader” and “Global Status is Pipeline”.  

¹⁹Drug Channels. “Tales of the Unsurprised: Brand-Name Drug Prices Fell for the Fourth Consecutive Year.” https://www.drugchannels. net/2022/01/tales-of-unsurprised-brand-name-drug.html; Drug Channels. “Surprise! Brand-Name Drug Prices Fell (Again) in 2020.” https://www.drugchannels.net/2021/01/surprise-brand-name-drug-prices-fell.html

²⁰Centers for Medicare & Medicaid Services. “CMS Proposes Medicare Coverage Policy for Monoclonal Antibodies Directed Against Amyloid for the Treatment of Alzheimer’s Disease.” https://www.cms.gov/newsroom/press-releases/cms-proposes-medicare-coverage-policy-monoclonal-antibodies-directed-against-amyloid-treatment

²¹Business Insider. “Investors Aren’t Buying EQRx’s Pitch for ‘Radically Lower’ Drug Prices. The CEO of the $3 Billion Startup Tells Us Why She’s Doubling Down Despite a Stock Drop.” https://www.businessinsider.com/eqrx-ceo-on-spacs-drug-pricing-and-2022-growth-plans-2022-1

²²EQRx. “Progress and Pipeline.” Accessed Feb. 13, 2022. https://www.eqrx.com/progress-and-pipeline/#pipeline

²³EQRx. “New Pharma — 40th Annual J.P. Morgan Healthcare Conference Presentation.” https://investors.eqrx.com/static-files/9a9e69da-28f3-4b5e-9386-3fd5dd7f8c04

²⁴EQRx. “EQRx presentation delivered at the 40th Annual J.P. Morgan Healthcare Conference on Monday, January 10, 2022 at 3:45 PM.” https://investors.eqrx.com/static-files/9de5fee9-6b1a-4355-8084-ea57fa7927d6

²⁵Secretary Xavier Becerra, U.S. Department of Health and Human Services, Office of the Assistant Secretary for Planning and Evaluation. “Comprehensive Plan for Addressing High Drug Prices.” https://aspe.hhs.gov/sites/default/files/2021-09/Competition%20EO%2045-Day%20Drug%20Pricing%20Report%209-8-2021.pdf

²⁶U.S. Department of Health and Human Services. “Biden-Harris Administration Improves Transparency and Oversight of Prescription Drug and Medical Costs.” https://www.hhs.gov/about/news/2021/11/17/biden-harris-administration-improves-transparency-and-oversight-of-prescription-drug-and-medical-costs.html

²⁷The White House. “President Biden Announces Prescription Drug Pricing Plan in Build Back Better Framework.” https://www.whitehouse.gov/briefing-room/statements-releases/2021/11/02/president-biden-announces-prescription-drug-pricing-plan-in-build-back-better-framework/

²⁸Covington. Inside EU Life Sciences. “New German Government Plans Significant Changes with Impact on the Healthcare, Life Sciences and Food Sector.” https://www.insideeulifesciences.com/2021/11/29/new-german-government-plans-significant-changes-with-impact-on-the-healthcare-life-sciences-and-food-sector/

²⁹FiercePharma. “Japan Sees ‘Drug Lag’ as Foreign Pharmas Pass Up the Market amid Pricing Pressure, Industry Group Warns.” https://www.fiercepharma.com/pharma-asia/japan-sees-drug-lag-as-foreign-pharmas-skip-market-amid-pricing-pressure-industry-group

³⁰Pharma DJ. “China’s NRDL Explained: 67 New Drugs, 62% Price Cut, 80% Success Rate and $47,000 Annual Cap.” http://www.pharmadj.com/en/cms/detail.htm?item.id=1abe2390597111ecbee6fa163e42049a

³¹GlobalData Healthcare, Pharmaceutical Technology. “Virtual Sales Reps’ and Physicians’ Engagement to Remain a New Standard Post- Covid-19.” https://www.pharmaceutical-technology.com/comment/virtual-engagement-covid-pharmaceutical-companies/

³²FiercePharma. “Amgen Cuts 500 U.S. Jobs, Primarily Sales Reps, as Covid-19 Speeds a Shift to Digital.” https://www.fiercepharma.com/marketing/amgen-cuts-several-hundred-u-s-jobs-primarily-sales-reps-as-covid-19-speeds-a-shift-to#

³³Reuters. “Exclusive: Pfizer to Cut U.S. Sales Staff as Meetings with Healthcare Providers Move to Virtual.” https://www.reuters.com/business/healthcare-pharmaceuticals/exclusive-pfizer-cut-us-sales-staff-meetings-move-virtual-2022-01-11/

³⁴American Medical Association. “Policy Research Perspectives: Recent Changes in Physician Practice Arrangements: Private Practice Dropped to Less Than 50 Percent of Physicians in 2020.” https://www.ama-assn.org/system/files/2021-05/2020-prp-physician-practice-arrangements.pdf

³⁵The Wall Street Journal. “Novartis Rethinks Sales Strategy for New Cholesterol Drug Launch.” https://www.wsj.com/articles/novartis-rethinks-sales-strategy-for-new-cholesterol-drug-launch-11637510400

³⁶FiercePharma. “JPM: Novartis Teams up with NHS England in ‘World-First’ Access Deal for PCSK9 Hopeful Inclisiran.” https://www.fiercepharma.com/pharma/novartis-teams-up-nhs-england-world-first-access-agreement-for-inclisiran

³⁷Novartis Pharmaceuticals. “Kymriah® Treatment Center Locator.” https://www.us.kymriah.com/treatment-center-locator/ Kite Pharma, A Gilead Company. “Find a Large B-Cell Lymphoma YESCARTA® Authorized Treatment Center.” https://www.yescarta.com/lbcl/ find-a-treatment-center/ Spark Therapeutics. “Patient Support Services.” https://mysparkgeneration.com/patient-support.html

³⁸Defined as those with potential for at least 18 months of treatment effects; based on cell and gene therapy pipeline from December 2019.

³⁹NEWDIGS FoCUS Project. MIT. “Updated Projection of US Durable Cell and Gene Therapies Product-Indication Approvals Based on December 2019 Development Pipeline.” https://newdigs.mit.edu/sites/default/files/NEWDIGS-Research-Brief-2020F207v51-PipelineAnalysis.pdf

⁴⁰The Personalized Medicine Coalition. “The Personalized Medicine Report — 2020: Opportunity, Challenges, and the Future.” https://www.personalizedmedicinecoalition.org/Userfiles/PMC-Corporate/file/PMC_The_Personalized_Medicine_Report_Opportunity_Challenges_and_the_Future.pdf

The Personalized Medicine Coalition. “Personalized Medicine at FDA: The Scope & Significance of Progress in 2020.” https://www.personalizedmedicinecoalition.org/Userfiles/PMC-Corporate/file/PM_at_FDA_The_Scope_Significance_of_Progress_in_2020.pdf

The Personalized Medicine Coalition. “Personalized Medicine at FDA: A Progress & Outlook Report.” https://www.personalizedmedicinecoalition.org/Userfiles/PMC-Corporate/file/PM_at_FDA_The_Scope_and_Significance_of_Progress_in_2019.pdf

The Personalized Medicine Coalition. “Personalized Medicine at FDA: A Progress & Outlook Report.” https://www.personalizedmedicinecoalition.org/Userfiles/PMC-Corporate/file/PM_at_FDA_A_Progress_and_Outlook_Report.pdf

The Personalized Medicine Coalition. “Personalized Medicine at FDA: 2017 Progress Report.” https://www.personalizedmedicinecoalition.org/Userfiles/PMC-Corporate/file/PM_at_FDA_2017_Progress_Report.pdf

The Personalized Medicine Coalition. “Personalized Medicine at FDA: 2016 Progress Report.” https://www.personalizedmedicinecoalition.org/userfiles/pmc-corporate/file/pm-at-fda.pdf

The Personalized Medicine Coalition. “Personalized Medicine at FDA: 2015 Progress Report.” https://www.personalizedmedicinecoalition.org/Userfiles/PMC-Corporate/file/2015_Progress_Report_PM_at_FDA.pdf

⁴¹Exact Sciences. “Multi-Cancer Early Detection Blood Test.” https://www.exactsciences.com/Pipeline-and-Data/Multi-Cancer-Early-Detection

⁴²GRAIL. “Grail Confirms Q2 2021 Introduction of Galleri, First-of-Kind Multi-Cancer Early Detection Blood Test.” https://grail.com/press-releases/grail-confirms-q2-2021-introduction-of-galleri-first-of-kind-multi-cancer-early-detection-blood-test/

⁴³Natera. “Natera Receives Final Medicare Coverage for Its Signatera™ MRD Test in Stage II-III Colorectal Cancer.” https://www.natera.com/company/news/natera-receives-final-medicare-coverage-for-its-signatera-mrd-test-in-stage-ii-iii-colorectal-cancer/

⁴⁴Being Patient. “New Era for Alzheimer’s Trials: Blood Tests Bring Younger Participants.” https://www.beingpatient.com/c2n-alzheimers-bloodtest-in-clinical-trials/

⁴⁵BioPharma Dive. “As Three Biotechs Head to Wall Street, a Battered Sector Braces for a Pullback.” https://www.biopharmadive.com/news/biotech-ipo-performance-investors-2021/616808/

⁴⁶Nasdaq. “2021 Year in Review: What’s New with the Innovators of the Nasdaq Biotechnology Index?” https://www.nasdaq.com/articles/2021-year-in-review-whats-new-with-the-innovators-of-the-nasdaq-biotechnology-index

⁴⁷Evaluate Vantage, Evaluate. “Evaluate Vantage 2022 Preview.” https://info.evaluate.com/rs/607-YGS-364/images/Vantage%202022%20Preview%20Report_Final.pdf

⁴⁸HKTDC Research. “Biotechnology, Medical & Healthcare Industry in Hong Kong.” https://research.hktdc.com/en/article/MzEzOTQ1MjMz

Conclusion

Combined, these trends are leading to fundamental shifts across the healthcare ecosystem. To win in this evolving landscape, industry leaders will have to monitor these trends and adapt their corporate strategies to make decisions regarding portfolio investments, new product launch strategies, capital formation and partnerships with industry participants. L.E.K. Consulting is closely monitoring developments across all these trends and can help biopharmas effectively respond to each of these strategic imperatives.

The authors of this Executive Insights would like to acknowledge the following L.E.K. Managing Directors for their insights and contributions: Helen Chen, Adrienne Rivlin, Stephanie Newey, Jonathan Kfoury and TJ Bilodeau.

Endnotes
¹Informa Pharma Intelligence. Citeline. “Pharmaprojects.” Accessed January 2022. https://citeline.informa.com/; Query: “Mechanism Of Action is Protein degrader” and “Global Status is Pipeline”.

²Defined as those with potential for at least 18 months of treatment effects; based on cell and gene therapy pipeline from December 2019.