FDA Overhaul Bill Passes House Panel, Moves to Floor Vote

The ayes had it with the House Energy & Commerce Committee last week, with all 51 members voting to pass the 21st Century Cures Act.

The measure, H.R. 6, would extend the rare pediatric disease priority review voucher program from its current sunset date in March 2016 through December 2018. Critics of the provision have argued that the less than three-year extension could discourage sponsors that aren’t in the latest stages of development from continuing their research programs.

The bill approved by the committee also would authorize — but not fund — the FDA $15 million to run pilot programs aimed at expanding the Sentinel surveillance system in fiscal years 2016 to 2010 to include evidence from observational studies, registries and real-world therapeutic use. The information, which would complement safety information from electronic health records and claims data, could be used to support new drug indications and postmarket study requirements.

Other provisions in the massive overhaul bill would encourage broader use of observational studies, patient registries and actual therapeutic use to support approvals of new drugs and enhance postmarket surveillance and allow for greater use of innovative statistical methods, such as adaptive clinical trials and Bayesian statistics, in clinical trial designs. Sponsors could seek approval for new “qualified” indications of an existing drug or biologic based on qualified data summaries.

The draft also directs the FDA to issue guidance on acquiring clinical evidence on different categories of drugs outside randomized clinical trials.

Speeding Approval

To speed access to novel therapies, drugmakers would be guaranteed FDA approval if intermediate endpoints in an agreed to development plan were met. In addition, drugmakers of products granted breakthrough, fast-track or qualified infectious disease product (QIDP) status could make them available via a corporate expanded access policy for compassionate use following the start of Phase II or III safety studies.

The FDA would have to develop guidance on how it would interpret and use adverse event data related to compassionate use.

The measure adopted last week includes an amendment from Reps. Anna Eshoo (D-Calif.) and Leonard Lance (R-N.J.) that would exempt FDA user fees from sequestration — a provision Eschoo said was intended to ensure that private dollars are not locked up.

During the markup, several lawmakers proposed and then withdrew amendments to the bill, including one from Rep. G.K. Butterfield (D-N.C.) that would allow the FDA to grant a sponsor a rare pediatric disease priority review voucher after the December 2018 cut-off, as long as the company’s application had been designated as treating a rare childhood disease prior to the expiry date. 

Rep. Jan Schakowsky (D-Ill.) proposed requiring drugmakers to disclose the full costs of their drug development programs, including any NIH funding that would offset the company’s costs. This is especially important in light of the proposed extra six months of exclusivity provided companies that develop rare disease indications for existing drugs, she said.

Schakowsky called out Gilead Sciences’ blockbuster hepatitis C cure Sovaldi (sofosbuvir), which costs $84,000 for a 12-week treatment, as an example of a game-changing drug whose cost is astronomical — especially since Gilead bought the company that owned Sovaldi for billions of dollars. 

Rep. Bobby Rush (D-Ill.) also wanted to require NIH, and in particular, the National Institute on Minority Health and Health Disparities, to draw up concrete plans to include more minorities in clinical trials. Well-publicized and well-studied diseases such as heart disease, stroke and diabetes often disproportionately affect minorities, and this needs to be reflected in clinical studies, he said.

While all three amendments were withdrawn at the markup, agreement between the sponsors and committee chairman Fred Upton (R-Mich.) could translate into manager’s amendments before the bill hits the House floor or be proposed during floor debate.

No comparable measure exists in the Senate. — Lena Freund