Potential policy reforms to strengthen the accelerated approval pathway
Publication: Journal of Comparative Effectiveness Research
Abstract
The accelerated approval pathway for new drugs in the United States is often praised but faces growing criticism of whether it is finding the appropriate balance between uncertainty, access and cost. To support efforts to strengthen the pathway, this paper provides an analysis of key concerns and the advantages and disadvantages of ten potential policy reforms – those achievable through the US FDA action alone, and those that would require a combination of government, payer and life science industry actions. Accelerated approval sits at the heart of many of the controversies regarding drug approvals and pricing, and this analysis provides perspectives on how best to strengthen the pathway within the broader landscape of an innovative US healthcare system.
Formats available
You can view the full content in the following formats:
References
1.
FDA. Guidance for industry: expedited programs for serious conditions – drugs and biologics (2014). www.fda.gov/media/86377/download
2.
Beaver JA, Howie LJ, Pelosof L et al. A 25-year experience of US Food and Drug Administration accelerated approval of malignant hematology and oncology drugs and biologics: a review. JAMA Oncol. 4(6), 849–856 (2018).
3.
Ladd V. The FDA’s accelerated approval process is working. Morning Consult. (2021). https://morningconsult.com/opinions/fdas-accelerated-approval-process-working/
4.
Friends of Cancer Research. Optimizing the use of accelerated approval (2021). https://friendsofcancerresearch.org/sites/default/files/2020-11/Optimizing_the_Use_of_Accelerated_Approval-2020.pdf
5.
National Breast Cancer Coalition. Call to action online – April 5, 2021 – National Breast Cancer Coalition (2021). www.stopbreastcancer.org/call-to-action-online-april-5-2021/
6.
Naci H, Smalley KR, Kesselheim AS. Characteristics of preapproval and postapproval studies for drugs granted accelerated approval by the US Food and Drug Administration. JAMA 318(7), 626–636 (2017).
7.
Dhruva SS, Redberg RF. Accelerated approval and possible withdrawal of midodrine. JAMA 304(19), 2172–2173 (2010).
8.
Herrup K, Goulazian J. Bad medicine: aducanumab is a lackluster drug with a high price tag. Stat News (2021). www.statnews.com/2021/06/08/aducanumab-lackluster-drug-high-price/
9.
Karlawish J. If the FDA approves Biogen’s Alzheimer’s treatment, I won’t prescribe it. Stat News (2021). www.statnews.com/2021/05/30/if-the-fda-approves-biogens-alzheimers-treatment-i-wont-prescribe-it/
10.
Kesselheim AS, Avorn J. The FDA has reached a new low. The New York Times (2021). www.nytimes.com/2021/06/15/opinion/alzheimers-drug-aducanumab-fda.html
11.
Kaltenboeck A, Mehlman A, Pearson SD. Strengthening the accelerated approval pathway: an analysis of potential policy reforms and their impact on uncertainty, access, innovation, and costs. Institute for Clinical and Economic Review (2021). https://icer.org/wp-content/uploads/2021/04/Strengthening-the-Accelerated-Approval-Pathway-_-ICER-White-Paper-_-April-2021.pdf
12.
FDA. The biomarker qualification process: a roadmap for requestors (2021). https://fda.report/media/107876/The-Biomarker-Qualification-Process–A-Roadmap-for-Requestors.pdf
13.
Amur S, LaVange L, Zineh I, Buckman-Garner S, Woodcock J. Biomarker qualification: toward a multiple stakeholder framework for biomarker development, regulatory acceptance, and utilization. Clin. Pharmacol. Ther. 98(1), 34–46 (2015).
14.
FDA. Biomarker qualification: evidentiary framework (2021). www.fda.gov/regulatory-information/search-fda-guidance-documents/biomarker-qualification-evidentiary-framework
15.
McShane LM. Concepts and case study template for surrogate endpoints workshop. Biomarkers Consortium – Workshop: Defining an Evidentiary Criteria Framework for Surrogate Endpoint Qualification (2018). https://fnih.org/sites/default/files/final/pdf/6-McShane-Case%20Study%20Overview.pdf
16.
Wallach JD, Egilman AC, Dhruva SS et al. Postmarket studies required by the US Food and Drug Administration for new drugs and biologics approved between 2009 and 2012: cross sectional analysis. BMJ (Clin. Res. Ed.) 361, k2031 (2018).
17.
Cure SMA. Our strategy (2021). www.curesma.org/our-strategy/
18.
FDA. A guide to drug safety terms at FDA. (2012). www.fda.gov/media/74382/download
19.
FDA. Prescribing information for Keytruda (2020). www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf
20.
Sullivan HW, O’Donoghue AC, David KT, Patel NJ. Disclosing accelerated approval on direct-to-consumer prescription drug websites. Pharmacoepidemiol. Drug Saf. 27(11), 1277–1280 (2018).
21.
Gellad WF, Kesselheim AS. Accelerated approval and expensive drugs – a challenging combination. N. Engl. J. Med. 376(21), 2001–2004 (2017).
22.
Dauner DG, Dauner KN, Peterson ALH. FDA’s unimproved enforcement of postmarketing requirements and commitments: implications for providers and patients. Res. Social Admin. Pharm. 16(6), 844–847 (2020).
23.
DiMagno SSP, Glickman A, Emanuel EJ. Accelerated approval of cancer drugs – righting the ship of the US Food and Drug Administration. JAMA Intern. Med. 179(7), 922–923 (2019).
24.
Detela G, Lodge A. EU regulatory pathways for ATMPs: standard, accelerated and adaptive pathways to marketing authorisation. Mol. Ther. Meth. Clin. Dev. 13, 205–232 (2019).
25.
Dolan R. Understanding the Medicaid Prescription Drug Rebate Program. KFF, CA, USA (2019). https://files.kff.org/attachment/Issue-Brief-Understanding-the-Medicaid-Prescription-Drug-Rebate-Program
26.
Sachs RE, Donohue JM, Dusetzina SB. Confronting state Medicaid drug spending pressures. JAMA 324(18), 1831–1832 (2020).
27.
Medicaid and Chip Payment and Access Commission. April 2021 MACPAC Public Meeting (2021). www.macpac.gov/public_meeting/april-2021-macpac-public-meeting/
28.
Medicaid and Chip Payment and Access Commission. High-cost specialty drugs: review of draft chapter and recommendations (2021). www.macpac.gov/wp-content/uploads/2021/04/High-Cost-Specialty-Drugs-Review-of-Draft-Chapter-and-Recommendations.pdf
29.
Centers for Medicare and Medicaid Services. Billing 340B modifiers under the hospital outpatient prospective payment system (OPPS) (2018). www.cms.gov/medicare/medicare-fee-for-service-payment/hospitaloutpatientpps/downloads/billing-340b-modifiers-under-hospital-opps.pdf
30.
Pallone F Jr. H.R.3 – 116th Congress (2019–2020): Elijah E. Cummings Lower Drug Costs Now Act (2019). www.congress.gov/bill/116th-congress/house-bill/3
31.
Kaltenboeck A, Bach PB. Value-based pricing for drugs: theme and variations. JAMA 319(21), 2165–2166 (2018).
32.
Kaltenboeck A. Pharmaceutical products and their value: lessons learned and the path ahead. Value Health 23(4), 421–424 (2020).
33.
Value-Based Contracts: 2009 - Q4 2019 (2020). www.phrma.org/Fact-Sheet/Value-Based-Contracts-2009-Q4-2019
34.
Garattini L, Curto A. Performance-based agreements in Italy: ‘Trendy Outcomes’ or Mere Illusions? Pharmacoeconomics 34(10), 967–969 (2016).
35.
Kaltenboeck A, Bach PB. Outcomes-based drug contracts do not move us closer to value - morning consult. Morning Consult (2021). https://morningconsult.com/opinions/outcomes-based-drug-contracts-not-move-us-closer-value/
Information & Authors
Information
Published In
Pages: 1177 - 1186
PubMed: 34427103
Copyright
© 2021 Future Medicine Ltd.
History
Received: 29 July 2021
Accepted: 5 August 2021
Published online: 24 August 2021
Keywords:
Topics
Authors
Funding Information
ICER receives support from pharmaceutical and health insurance companies for its Policy Leadership Forum program, including the annual ICER Policy Summit. Please find details of this support in the Financial Disclosure section below.
Metrics & Citations
Metrics
Article Usage
Article usage data only available from February 2023. Historical article usage data, showing the number of article downloads, is available upon request.
Citations
How to Cite
Potential policy reforms to strengthen the accelerated approval pathway. (2021) Journal of Comparative Effectiveness Research. DOI: 10.2217/cer-2021-0184
Export citation
Select the citation format you wish to export for this article or chapter.
Citing Literature
- Tu My To, Stacey Kowal, James D. Chambers, William B. Wong, Real-World Clinical Oncology Outcomes Associated with the Accelerated Approval Pathway, Cancer Research Communications, 10.1158/2767-9764.CRC-25-0225, 6, 1, (191-200), (2026).
- Huseyin Naci, Mahnum Shahzad, Peter Murphy, Yichen Zhang, Rebecca Costa, Joseph S Ross, Anita K Wagner, Costs and benefits of early access to new cancer drugs through the US Food and Drug Administration’s accelerated approval pathway: retrospective observational study and economic evaluation, BMJ Medicine, 10.1136/bmjmed-2025-001934, 4, 1, (e001934), (2025).
- Sharon Phares, Mark Trusheim, Sarah K Emond, Steven D Pearson, Managing the challenges of paying for gene therapy: strategies for market action and policy reform in the United States, Journal of Comparative Effectiveness Research, 10.57264/cer-2024-0118, 13, 12, (2024).
- Adrian Towse, Elisabeth Fenwick, It Takes 2 to Tango. Setting Out the Conditions in Which Performance-Based Risk-Sharing Arrangements Work for Both Parties, Value in Health, 10.1016/j.jval.2024.03.2196, 27, 8, (1058-1065), (2024).
- William Wong, Stacey Kowal, Tu My To, Achal Patel, David Veenstra, Louis Garrison, Meng Li, The value of the accelerated approval pathway: real-world outcomes associated with reducing the time between innovations, Future Oncology, 10.2217/fon-2023-0514, 20, 16, (1099-1110), (2024).
- Daniel Tobias Michaeli, Thomas Michaeli, Sebastian Albers, Tobias Boch, Julia Caroline Michaeli, Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy, The European Journal of Health Economics, 10.1007/s10198-023-01639-x, 25, 6, (979-997), (2023).
- Boshen Jiao, Estimating the Potential Benefits of Confirmatory Trials for Drugs with Accelerated Approval: A Comprehensive Value of Information Framework, PharmacoEconomics, 10.1007/s40273-023-01303-5, 41, 12, (1617-1627), (2023).
- Rosanna Tarricone, Helen Banks, Oriana Ciani, Werner Brouwer, Michael F Drummond, Reiner Leidl, Nicolas Martelli, Laura Sampietro-Colom, Rod S. Taylor, An accelerated access pathway for innovative high-risk medical devices under the new European Union Medical Devices and health technology assessment regulations? Analysis and recommendations, Expert Review of Medical Devices, 10.1080/17434440.2023.2192868, 20, 4, (259-271), (2023).
- Yichen Zhang, Huseyin Naci, Anita K. Wagner, Ziyue Xu, Yu Yang, Jun Zhu, Jiafu Ji, Luwen Shi, Xiaodong Guan, Overall Survival Benefits of Cancer Drugs Approved in China From 2005 to 2020, JAMA Network Open, 10.1001/jamanetworkopen.2022.25973, 5, 8, (e2225973), (2022).
- Jeromie Ballreich, Mariana Socal, Charles L. Bennett, Martin W. Schoen, Antonio Trujillo, Andrew Xuan, Gerard Anderson, Medicare Spending on Drugs With Accelerated Approval, Annals of Internal Medicine, 10.7326/M21-4442, 175, 7, (938-944), (2022).