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Raising the bar for using surrogate endpoints in drug regulation and health technology assessment

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journal contribution
posted on 12.11.2021, 16:08 by Dalia Dawoud, Huseyin Naci, Oriana Ciani, Sylwia Bujkiewicz
In June 2021, the US Food and Drug Administration
granted accelerated approval to aducanumab for
treating Alzheimer’s disease based on the drug’s
amyloid reducing effects. This was despite evidence
from several earlier studies that shrinkage of
β-amyloid protein plaques does not predictably delay
cognitive impairment.1 The controversial decision
has drawn attention to the use of surrogate
endpoints—laboratory values, radiographic images,
or other physical measures that may serve as
indicators of clinical outcomes such as symptom
control or mortality—in clinical trials of new drugs.2
In fact, the approval of aducanumab is only the latest
example of growing regulatory reliance on surrogate
endpoints, even though their use can cause problems
for patients, clinicians, drug regulators, and health
technology assessment bodies.
We argue for more selective use of surrogate
endpoints when evaluating new drugs, restricting
their use to chronic diseases, especially when
collecting data on patient relevant clinical outcomes
requires trials with unattainably long follow up.

History

Citation

BMJ 2021; 374 doi: https://doi.org/10.1136/bmj.n2191 (Published 16 September 2021) Cite this as: BMJ 2021;374:n2191

Author affiliation

Department of Health Sciences, University of Leicester

Version

AM (Accepted Manuscript)

Published in

BRITISH MEDICAL JOURNAL

Volume

374

Publisher

BMJ Publishing Group

issn

1759-2151

eissn

0959-8138

Copyright date

2021

Available date

12/11/2021

Language

English