FDA sets standards for Real-World Evidence

The 21st Century Cures Act, enacted December 2016, mandated that the FDA consider how to use real-world evidence (RWE) to bring innovative treatments to market faster. To meet this statutory requirement, the FDA has provided guidance outlining its policies for using RWE to support the approval of a new drug indication. What is real-world evidence?…

Economics of the Pharmaceutical Industry

Darius Lakdawalla has an very interesting review article in the Journal of Economic Literature on the Economics of the pharmacuetical industry.  Do read the whole thing, but below I have listed some highlights. A model of firm R&D decisions Lakdawalla uses a simple model based on Nordhaus (1969) to derive some important implications about pharmaceutical…

Should we move from patents to prizes?

An interesting article by Charles Silver and David A. Hyman argues that we should do away with patents for pharmaceuticals and move to a prize based system.  They write in Vox: A well-designed prize regime would lower drug prices by eliminating drug monopolies, yet it would also create the necessary incentives for innovation, including incentives to…

How Scott Gottlieb is transforming the FDA

The Economist has an article on the evolution of FDA regulation of pharmaceutical treatments.  Brining a drug to market costs billions of dollars.  FDA commissioner Scott Gottlieb aims to cut this cost by expediting the drug approval process in three key ways. First, he wants the agency to rethink how much information the FDA demands…

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…

Advancing the Discussion on Real-World Evidence

With the FDA’s introduction of new guidelines surrounding the use of real-world evidence (RWE) in medical device regulatory decisions, FDA Commissioner Scott Gottlieb advances the argument for the utility of RWE. In fact, the FDA is currently considering the role of RWE in evaluating pharmaceutical treatments. Despite much debate over what part RWE should play in…

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…