Intended for healthcare professionals

Rapid response to:


Drugs with FDA accelerated approval often have weak evidence, study finds

BMJ 2017; 357 doi: (Published 14 June 2017) Cite this as: BMJ 2017;357:j2905

Rapid Response:

Accelerated approval of drug on weak evidence is not the worst.

Naci, from the London School of Economics, commenting on his analysis showing evidence that drugs with FDA accelerated approval often have weak evidence, claimed: “research practices on these drugs rarely meet the information needs of patients, doctors, and other decision makers in healthcare systems,”.(1,2)

First, this is not news.(3,4) Moreover post-marketing studies are performed in only two-thirds of cases and when performed it is with a median delay of 4 years.(4)

One can understand that stakeholders could rely on hope, too much, and that finding a prudent middle ground to protect patient interests represents an immense tension for drug regulators.

Nevertheless accelerated approval is associated with unreasonable delays in market withdrawal, even in the case of drug-related deaths.(5) For lack of efficacy, the process is even slower: drotrecogin alfa was not withdrawn for 10 years after initial approval and bevacizumab was approved for metastatic breast cancer in February 2008 under the FDA accelerated program and the license was not revoked until November 2011.

No one can understand how the system can have been so wrong for so long: FDA goes too fast for approval and too slow for withdrawal, the European Agency too.(6,7)

1 Dyer O. Drugs with FDA accelerated approval often have weak evidence, study finds. BMJ 2017;357:j2905.

2 Naci H, Wouters OJ, Gupta R, Ioannidis JPA. Timing and characteristics of cumulative evidence available on novel therapeutic agents receiving Food and Drug Administration accelerated approval. Milbank Q2017;357:261-90.

3 Kim C, Prasad V. Cancer drugs approved on the basis of a surrogate end point and subsequent overall survival: an analysis of 5 years of US Food and Drug Administration approvals. JAMA Intern Med 2015;175:1992-1994.

4 Johnson JR, Ning YM, Farrell A, Justice R, Keegan P, Pazdur R. Accelerated approval of oncology products: the food and drug administration experience. J Natl Cancer Inst 2011;103:636-644.

5 Braillon A, Menkes DB. Balancing accelerated approval for drugs with accelerated withdrawal. JAMA Intern Med 2016;176:566-7.

6 Davis C, Lexchin J, Jefferson T, Gøtzsche P, McKee M. "Adaptive pathways" to drug authorisation: adapting to industry? BMJ 2016;354:i4437.

7 Bolland MJ, Grey A. Ten years too long: strontium ranelate, cardiac events, and the European Medicines Agency. BMJ 2016;354:i5109.

Competing interests: No competing interests

20 June 2017
alain braillon
senior consultant
University Hospital. 80000 Amiens. France