Unbiased Analysis of Today's Healthcare Issues
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…
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…
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.…
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…
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…
The American Journal of Managed Care has an interesting special issue on treatment for the hepatitus C virus (HCV). Jay Bhattacharya provides the introductory commentary, and starts off with the following. Although the medical prospects facing patients with hepatitis C virus (HCV) have never been better, the prospect of gaining access to a cure is another…
Dana Goldman–my colleague at PHE and a professor at USC–offers three suggestions on how to prevent generic products from increasing their prices drastically as occurred in the EpiPen saga. In Stat News, he makes three recommendations: First, Congress should mandate that the Federal Trade Commission report on the availability of all such drugs and devices…
Aaron Carroll of the Incidental Economist draws on a column from his colleague Austin Frakt in The Upshot to explain why there is not more research into off-label uses of patented or generic drugs.
Approximately one in five prescriptions for drugs in the US are for off label use. In some cases, this may be inapprorpiate as there is typically limited clinical evidence supporting off label use. In other cases, off label use is approrpiate. Clinical trials rarely enroll children or pregnant women and thus medictions are often not…
The answer may be yes. One of the big inpediments to value-based pricing of pharmaceuticals was that any discount given to any single organization based on outcomes needed to be reflected in the Medicaid price. Since outcomes are subject to random noise, there will inevitably be health plans that end up getting a low price due to worse than expected…