Is the FDA Too Conservative or Too Aggressive?: A Bayesian Decision Analysis of Clinical Trial Design / Vahid Montazerhodjat, Andrew W. Lo.

Format:

Book

Author/Creator:

Montazerhodjat, Vahid.

Contributor:

National Bureau of Economic Research.

Lo, Andrew W.

Series:

Working Paper Series (National Bureau of Economic Research) no. w21499.

NBER working paper series no. w21499

Language:

English

Physical Description:

1 online resource: illustrations (black and white);

Other Title:

Is the FDA Too Conservative or Too Aggressive?

Place of Publication:

Cambridge, Mass. National Bureau of Economic Research 2015.

Summary:

Implicit in the drug-approval process is a trade-off between Type I and Type II error. We explore the application of Bayesian decision analysis (BDA) to minimize the expected cost of drug approval, where relative costs are calibrated using U.S. Burden of Disease Study 2010 data. The results for conventional fixed-sample randomized clinical-trial designs suggest that for terminal illnesses with no existing therapies such as pancreatic cancer, the standard threshold of 2.5% is substantially more conservative than the BDA-optimal threshold of 27.9%. However, for relatively less deadly conditions such as prostate cancer, 2.5% is more risk-tolerant or aggressive than the BDA-optimal threshold of 1.2%. We compute BDA-optimal sizes for 25 of the most lethal diseases and show how a BDA-informed approval process can incorporate all stakeholders' views in a systematic, transparent, internally consistent, and repeatable manner.

Notes:

Print version record

August 2015.