Unbiased Analysis of Today's Healthcare Issues

Archive for the 'Regulation' Category

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 […]

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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 […]

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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 […]

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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. […]

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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 […]

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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 […]

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Dismal Arithmetic of Hepatitis C Treatment?

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 […]

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How to prevent another EpiPen controversy

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 […]

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Incentives for investing in “off label” trials

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.  

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Off Label Drug Use

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 […]

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