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Hepatitis C

FDA Paper Addresses Concerns Over Hep C Drug Approvals

A recently released statement from the FDA’s Division of Antiviral Products in the Center for Drug Evaluation and Research addresses public concerns regarding the approval of several new treatment options for chronic hepatitis C.

Recently approved treatment options for hepatitis C—including several interferon (IFN)-free or IFN and ribavirin-free treatment regimens—have displayed improved efficacy and safety profiles, as well as shorter treatment duration.
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In response, several public groups have raised concerns regarding the evidence used to support the approvals of these new options.

To address these concerns, the FDA has released a paper titled, “Direct-Acting Antiviral Drug Approvals For Treatment Of Chronic Hepatitis C Virus Infection: Scientific And Regulatory Approaches To Clinical Trial Designs,” in order to “provide increased transparency to various stakeholders about FDA's scientific approaches and regulatory processes that supported drug development and marketing approval of direct acting antiviral agents for the treatment of hepatitis C.”1

“FDA’s approach to evaluation of recent hepatitis C drugs underscores the Agency’s flexibility in considering innovative or alternative trial designs for drugs that have demonstrated highly promising outcomes in early phase development,” said lead author Poonam Mishra, MD, deputy director for Safety, Division of Antiviral Products. 2

“Expedited approaches can be used without compromising efficacy standards for drugs that demonstrate breakthrough therapy potential.”

Methods discussed within the paper include:

  • Use of a validated surrogate (sustained viral response) for a primary endpoint
  • Shortening the timepoint for measuring the surrogate by 12 weeks
  • Use of historical controls when clinically appropriate
  • Use of modeling when scientifically sound to extend treatment indications to subpopulations not fully evaluated in clinical trials1

The full paper was published in the journal Heptology.

—Michael Potts

References:

  1. Mishra P, Murray J, Birnkrant D. Direct-acting antiviral drug approvals for treatment of chronic hepatitis C virus infection: Scientific and regulatory approaches to clinical trial designs. Heptology. June 2015 [epub ahead of print]. DOI: 10.1002/hep.27880.
  2. FDA. FDA addresses concerns on approval of drugs to treat chronic hepatitis C [press release]. June 1, 2015. http://www.wiley.com/WileyCDA/PressRelease/pressReleaseId-118518.html