Off-label prescribing

How frequently are pharmaceuticals used off label?  Perhaps more than you think.  Although these figures are a bit dated, Tabarrok (2000) details the extent of off-label prescribing in the U.S. as follows: According to a study by the U.S. General Accounting Office, 56 percent of cancer patients have been given non-FDA-approved prescriptions, and 33 percent…

The Voice of the Patient

Did you ever wonder what is is like having lung cancer?  Or narcolepsy?    What factors are most important to patients when receiving treatment for these diseases? The FDA is working to collect these answers to help guide their drug approval process.  The FDA’s “Voice of the Patient” aims to “…more systematically gather patients’ perspectives…

FDA Hedges

Pharmaceutical companies face major risk.  There is risk that the drugs they are researching don’t work (e.g., lack of efficacy) or are not safe.  There is risk that health insurers or government payers will not cover their treatment.  And there is risk that the FDA will not approve a drug after a Phase III clinical trial.…

Drug approval and reimbursement when clinical trials use surrogate endpoints

An interesting paper from some of my colleagues at Precision Health Economics: Approval of new drugs is increasingly reliant on “surrogate endpoints,” which correlate with but imperfectly predict clinical benefits. Proponents argue surrogate endpoints allow for faster approval, but critics charge they provide inadequate evidence. We develop an economic framework that addresses the value of…

How to regulate precision medicines

Currently, pharmaceutical treatments that are used in the U.S. need to gain an approval from the FDA.  The FDA’s approval is contingent on a demonstration of efficacy and safety in a randomized controlled trial (RCT).  However, precision medicine makes the standard FDA approval problematic. As described in Breckenridge et al. (2016), in the precision medicine…