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http://ssrn.com/abstract=1492909
 
 

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Accounting for Differences Among Patients in the FDA Approval Process


Anup Malani


University of Chicago - Law School; University of Chicago Pritzker School of Medicine; Resources for the Future; National Bureau of Economic Research (NBER)

Oliver Bembom


University of California, Berkeley

Mark Van der Laan


University of California, Berkeley

October 22, 2009

U of Chicago Law & Economics, Olin Working Paper No. 488
U of Chicago, Public Law Working Paper No. 281

Abstract:     
The FDA employs an average-patient standard when reviewing drugs: it approves a drug only if the average patient (in clinical trials) does better on the drug than on control. It is common, however, for different patients to respond differently to a drug. Therefore, the average-patient standard can result in approval of a drug with significant negative effects for certain patient subgroups (false positives) and disapproval of drugs with significant positive effects for other patient subgroups (false negatives). Drug companies have a financial incentive to avoid false negatives. After their clinical trials reveal that their drug does not benefit the average patient, they conduct what is called post hoc subgroup analysis to highlight patients that benefit from the drug. The FDA rejects such analysis due to the risk of spurious results. With enough data dredging, a drug company can always find some patients that benefit from their drug.

This paper asks whether there workable compromise between the FDA and drug companies. Specifically, we seek a drug approval process that can use post hoc subgroup analysis to eliminate false negatives but does not risk opportunistic behavior and spurious correlation. We recommend that the FDA or some other independent agent conduct subgroup analysis to identify patient subgroups that may benefit from a drug. Moreover, we suggest a number of statistical algorithms that operate as veil of ignorance rules to ensure that the independent agent is not indirectly captured by drug companies. We illustrate our proposal by applying it to the results of a recent clinical trial of a cancer drug (motexafin gadolinium) that was recently rejected by the FDA.

Number of Pages in PDF File: 39

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Date posted: October 24, 2009  

Suggested Citation

Malani, Anup and Bembom, Oliver and van der Laan, Mark, Accounting for Differences Among Patients in the FDA Approval Process (October 22, 2009). U of Chicago Law & Economics, Olin Working Paper No. 488; U of Chicago, Public Law Working Paper No. 281. Available at SSRN: http://ssrn.com/abstract=1492909 or http://dx.doi.org/10.2139/ssrn.1492909

Contact Information

Anup Malani (Contact Author)
University of Chicago - Law School ( email )
1111 E. 60th St.
Chicago, IL 60637
United States
773-702-9602 (Phone)
773-702-0730 (Fax)
HOME PAGE: http://www.law.uchicago.edu/faculty/malani/
University of Chicago Pritzker School of Medicine
Chicago, IL 60637
United States
Resources for the Future
1616 P Street, NW
Washington, DC 20036
United States
National Bureau of Economic Research (NBER)
1050 Massachusetts Avenue
Cambridge, MA 02138
United States
Oliver Bembom
University of California, Berkeley
310 Barrows Hall
Berkeley, CA 94720
United States
Mark Van der Laan
University of California, Berkeley
310 Barrows Hall
Berkeley, CA 94720
United States
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