Improving the Use of FDA Advisory Committees

Daval, C. Joseph Ross; Kesselheim, Aaron S.; Sarpatwari, Ameet

doi:10.1056/nejmp2206492

Cited by 7 publications

(8 citation statements)

References 3 publications

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“…As numerous observers have noted, how the FDA generally applies evidentiary thresholds or the rationale behind its conclusions are too often unclear 65–68 . It is often difficult to discern the criteria the FDA uses to refer drugs to its advisory committees and its reasoning in “discordant” cases in which the agency goes against advisory committee votes 65,69 . Janiaud and colleagues conducted a study of “close call” decisions made by the FDA in the context of high levels of uncertainty and criticized the FDA's use of “bespoke” rationales for decisions, the “balkanization and fragility of institutional knowledge” and “no mechanism to find or tradition to cite similar cases when weighing evidence.” They advocate for additional use of case law and precedents to address weak institutional memory and new databases on prior decisions to support such reforms.…”

Section: Improving the Handoff: Options And Recommendationsmentioning

confidence: 99%

Improving Food and Drug Administration–Centers for Medicare and Medicaid Services Coordination for Drugs Granted Accelerated Approval

NEUMANN,

CRUMMER,

CHAMBERS

et al. 2023

Milbank Quarterly

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Policy Points The increasing number of drugs granted accelerated approval by the Food and Drug Administration (FDA) has challenged the Medicare program, which often pays for expensive therapies despite substantial uncertainty about benefits and risks to Medicare beneficiaries. We recommend several administrative and legislative approaches for improving FDA–Centers for Medicare and Medicaid Services (CMS) coordination around accelerated‐approval drugs, including promoting earlier discussions among the FDA, the CMS, and drug companies; strengthening Medicare's coverage with evidence development program; linking Medicare payment to evidence generation milestones; and ensuring that the CMS has adequate staffing and resources to evaluate new therapies. These activities can help improve the integrity; transparency; and efficiency of approval, coverage, and payment processes for drugs granted accelerated approval. ContextThe Food and Drug Administration (FDA)’s accelerated‐approval pathway expedites patient access to promising treatments. However, increasing use of this pathway has challenged the Medicare program, which often pays for expensive therapies despite substantial uncertainty about benefits and risks to Medicare beneficiaries. We examined approaches to improve coordination between the FDA and Centers for Medicare and Medicaid Services (CMS) for drugs granted accelerated approval.MethodsWe argue that policymakers have focused on expedited pathways at the FDA without sufficient attention to complementary policies at the CMS. Although differences between the FDA and CMS decisions are to be expected given the agencies’ different missions and statutory obligations, procedural improvements can ensure that Medicare beneficiaries have timely access to novel therapies that are likely to improve health outcomes. To inform policy options and recommendations, we conducted semistructured interviews with stakeholders to capture diverse perspectives on the topic.FindingsWe recommend ten areas for consideration: clarifying the FDA's evidentiary standards; strengthening FDA authorities; promoting earlier discussions among the FDA, the CMS, and drug companies; improving Medicare's coverage with evidence development program; tying Medicare payment for accelerated‐approval drugs to evidence generation milestones; issuing CMS guidance on real‐world evidence; clarifying Medicare's “reasonable and necessary” criteria; adopting lessons from international regulatory‐reimbursement harmonization efforts; ensuring that the CMS has adequate staffing and expertise; and emphasizing equity.ConclusionsBetter coordination between the FDA and CMS could improve the transparency and predictability of drug approval and coverage around accelerated‐approval drugs, with important implications for patient outcomes, health spending, and evidence generation processes. Improved coordination will require reforms at both the FDA and CMS, with special attention to honoring the agencies’ distinct authorities. It will require administrative and legislative actions, new resources, and strong leadership at both agencies.

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Section: Improving the Handoff: Options And Recommendationsmentioning

confidence: 99%

Improving Food and Drug Administration–Centers for Medicare and Medicaid Services Coordination for Drugs Granted Accelerated Approval

NEUMANN,

CRUMMER,

CHAMBERS

et al. 2023

Milbank Quarterly

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“…13 , 14 The question of how the FDA chooses whether to convene an advisory committee arose after it declined to reconvene its vaccine advisory committee before authorizing a third dose of the BNT162b2 COVID-19 vaccine (Pfizer-BioNTech) for the general population after that committee had declined to endorse that authorization a few months earlier. 15 A previous study of negative FDA advisory committee votes found that the proportion of new drugs reviewed by an advisory committee before approval declined dramatically from over half in 2010 to 6% in 2021. 16 A separate recent analysis found 86% alignment with advisory committee recommendations on 133 approval decisions from 2017 to 2022 but did not address the frequency of meetings or the timing of decisions.…”

Section: Introductionmentioning

confidence: 99%

Association of Advisory Committee Votes With US Food and Drug Administration Decision-Making on Prescription Drugs, 2010-2021

et al. 2023

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ImportanceThe US Food and Drug Administration (FDA) often relies on independent advisory committees when making decisions about the approval of prescription drugs or their withdrawal from the market. These committees provide the FDA with valuable insight and an opportunity to build public trust through transparent deliberation, but recent controversies have raised questions about the optimal use of FDA advisory committees.ObjectiveTo assess the frequency, purposes, and voting outcomes of human drug advisory committees convened from 2010 to 2021 and the FDA’s corresponding actions.Design, Setting, and ParticipantsThis qualitative study used a manual review of meeting summaries prepared by FDA staff for the 18 human drug advisory committees active at any time from January 1, 2010, to December 31, 2021, as well as FDA announcements and press releases, drug labels and approval data, industry publications, and company press releases.Main Outcomes and MeasuresOutcomes of votes on regulatory questions were recorded using meeting minutes. Alignment of FDA action with advisory votes for new drugs and indications was judged as of 1 year after the vote was held and as of November 30, 2022.ResultsThe FDA held 409 human drug advisory committee meetings from 2010 to 2021. Committees were convened less frequently over time, from a high of 50 in 2012 to a low of 18 in 2020 and 2021. Much of this decrease occurred at committee meetings involving votes on initial approvals, which declined from a high of 26 in 2012 to a low of 8 in 2021. Overall, FDA regulatory actions aligned with 262 of 298 advisory committee votes on initial approvals, supplemental approvals, withdrawals of approval, and safety actions (88%). Approval followed 142 of 147 positive votes for initial approvals (97%) and 33 of 36 positive votes for supplemental indications (92%), while nonapproval followed 40 of 60 negative votes on initial approvals (67%) and 18 of 21 negative votes on supplemental indications (86%).Conclusions and RelevanceIn this qualitative study, there was consistent alignment between advisory votes and FDA action across years and subject areas, but the number of meetings decreased over time. Discordance between FDA actions and advisory committee votes was most frequently an approval after a negative vote. This study demonstrated that these committees have played a key role in the FDA’s decision-making process but that the FDA sought independent expert advice less frequently over time even as it continued to follow it. The role of advisory committees in the current regulatory landscape should be more clearly and publicly defined.

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“…Although not required to, it convenes these expert panels when facing difficult decisions. 1 In this case, the subject was 3,4methylenedioxymethamphetamine (MDMA), a Schedule I controlled substance, which shows promise for treating posttraumatic stress disorder.For decades, the nonprofit Multidisciplinary Association for Psychedelic Studies (MAPS) pursued research on MDMA with psychotherapy, a combination it calls MDMA-assisted therapy (MDMA-AT). In 2014, MAPS formed a wholly owned, for-profit subsidiary, the MAPS Public Benefit Corporation, which took over drug development.…”

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confidence: 99%