Literature DB >> 35696021

Aspiring to Reasonableness in Accelerated Approval: Anticipating and Avoiding the Next Aducanumab.

Emily A Largent1, Andrew Peterson2, Jason Karlawish3,4,5, Holly Fernandez Lynch3.   

Abstract

The US Food and Drug Administration's decisions about drug approval-though guided by science, as well as relevant statutes, regulations, and guidance documents-reflect normative judgments about how the agency should exercise its discretion. This is particularly true in the context of the "accelerated approval" pathway, where the agency must balance speeding to market drugs for patients with unmet needs before they have been proven to work and ensuring confidence about the benefits and risks of those drugs. A key challenge in evaluating normative judgments such as these is that reasonable people can disagree, rendering it difficult to proclaim with certainty that a particular decision is right or wrong. Therefore, we propose that it is preferable to ask whether a decision is reasonable. A decision is reasonable when it transparently, comprehensively, and fairly balances the interests of affected parties, within the parameters of the decision maker's legal authority. If a decision achieves these three qualities, it can be viewed as legitimate and worthy of trust and confidence, regardless of whether one agrees with the particular outcome. We recommend that the Food and Drug Administration adopt procedural protections to promote reasonableness in four domains affecting accelerated approval decisions: pathway gatekeeping, endpoint selection, stakeholder engagement, and deliberation. This should aid the agency in minimizing controversies, such as that surrounding the 2021 approval of aducanumab (Aduhelm; Biogen).
© 2022. The Author(s), under exclusive licence to Springer Nature Switzerland AG.

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Year:  2022        PMID: 35696021      PMCID: PMC9247014          DOI: 10.1007/s40266-022-00949-8

Source DB:  PubMed          Journal:  Drugs Aging        ISSN: 1170-229X            Impact factor:   4.271


  25 in total

Review 1.  Three dimensions of the amyloid hypothesis: time, space and 'wingmen'.

Authors:  Erik S Musiek; David M Holtzman
Journal:  Nat Neurosci       Date:  2015-06       Impact factor: 24.884

2.  The limits of acceptable political influence over the FDA.

Authors:  Holly Fernandez Lynch; Steven Joffe; Matthew S McCoy
Journal:  Nat Med       Date:  2021-02       Impact factor: 53.440

3.  An Appropriate Use of Accelerated Approval - Aducanumab for Alzheimer's Disease.

Authors:  Billy Dunn; Peter Stein; Robert Temple; Patrizia Cavazzoni
Journal:  N Engl J Med       Date:  2021-07-28       Impact factor: 91.245

4.  US Food and Drug Administration Approval of Aducanumab-Is Amyloid Load a Valid Surrogate End Point for Alzheimer Disease Clinical Trials?

Authors:  Vincent Planche; Nicolas Villain
Journal:  JAMA Neurol       Date:  2021-11-01       Impact factor: 18.302

5.  Aducanumab and the Business of Alzheimer Disease-Some Choice.

Authors:  Jason Karlawish
Journal:  JAMA Neurol       Date:  2021-11-01       Impact factor: 18.302

6.  Two Randomized Phase 3 Studies of Aducanumab in Early Alzheimer's Disease.

Authors:  S Budd Haeberlein; P S Aisen; F Barkhof; S Chalkias; T Chen; S Cohen; G Dent; O Hansson; K Harrison; C von Hehn; T Iwatsubo; C Mallinckrodt; C J Mummery; K K Muralidharan; I Nestorov; L Nisenbaum; R Rajagovindan; L Skordos; Y Tian; C H van Dyck; B Vellas; S Wu; Y Zhu; A Sandrock
Journal:  J Prev Alzheimers Dis       Date:  2022

Review 7.  Assessment of the Clinical Benefit of Cancer Drugs Receiving Accelerated Approval.

Authors:  Bishal Gyawali; Spencer Phillips Hey; Aaron S Kesselheim
Journal:  JAMA Intern Med       Date:  2019-07-01       Impact factor: 21.873

8.  Approval of Aducanumab for Alzheimer Disease-The FDA's Perspective.

Authors:  Billy Dunn; Peter Stein; Patrizia Cavazzoni
Journal:  JAMA Intern Med       Date:  2021-10-01       Impact factor: 21.873

9.  Aggregated Tau Measured by Visual Interpretation of Flortaucipir Positron Emission Tomography and the Associated Risk of Clinical Progression of Mild Cognitive Impairment and Alzheimer Disease: Results From 2 Phase III Clinical Trials.

Authors:  Ming Lu; Michael J Pontecorvo; Michael D Devous; Anupa K Arora; Nicholas Galante; Anne McGeehan; Catherine Devadanam; Stephen P Salloway; P Murali Doraiswamy; Craig Curtis; Stephen P Truocchio; Matthew Flitter; Tricia Locascio; Marybeth Devine; Jennifer A Zimmer; Adam S Fleisher; Mark A Mintun
Journal:  JAMA Neurol       Date:  2021-04-01       Impact factor: 18.302

10.  The amyloid hypothesis on trial.

Authors:  Simon Makin
Journal:  Nature       Date:  2018-07       Impact factor: 49.962

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