21st Century Cures Act Sails Through House Committee

21st Century Cures Act Sails Through House Committee

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

The bill, H.R. 6, released May 19, encourages greater use of observational studies, patient registries and real-world therapeutic use to support approvals of new drugs and improve postmarket surveillance.

Such methods could be used to assess a drug’s benefits and risks and advance patient-centered care — tapping into local considerations, community values and mechanisms to protect vulnerable populations. The act would direct the FDA to develop guidance to attain clinical evidence on different types of products outside randomized clinical trials.

It would also allow broader use of innovative statistical methods in clinical trial designs, including more use of adaptive clinical trials and Bayesian statistics. The FDA would also be tasked with establishing pilot programs to make “strategic linkages” between data captured in the Sentinel drug safety system and other public health data sources.

Under a streamlined data review program, sponsors could seek approval for a new “qualified indication” of an existing drug or biologic based on submission of “qualified data summaries.” The bill doesn’t clarify how indications would be qualified, other than that they must treat a form of cancer.

This provision is a very big deal, says Peter Pitts, president of the Center for Medicine in the Public Interest. Beyond the design of the trial, it also speaks more specifically to the type of evidence that the FDA can accept.

To speed up drug development and get novel therapies to market faster, companies and the FDA could agree on accelerated approval development plans, assuring approval if intermediate endpoints were met.

To advance development of targeted therapies, the bill includes a new section on precision medicines. The FDA would have to issue guidance defining what a “precision medicine” is and the requirements for developing and testing such products.

One drawback, however, is the reduced rare pediatric disease priority review voucher period. While previous versions of the bill had allowed the FDA to hand out these vouchers until June 2022, the current bill cuts that period to December 2018.

A rare pediatric disease priority review voucher allows a drugmaker to secure a six-month review of a subsequent drug, following approval of a drug that treats a rare childhood disease. The program, authorized under the 2012 FDA Safety and Innovation Act, is set to expire in March 2016.

The measure would require the Government Accountability Office to look at the indications approved for each drug awarded a priority review voucher and whether the voucher affected the sponsor’s decision to invest in developing drugs to treat or prevent rare pediatric diseases. The comptroller general also would have to conduct an analysis of drugs approved using the voucher, including whether they address unmet needs or represent an advance over available therapies.

Shortening the voucher reauthorization period by four years may have the unintended effect of discouraging drugmakers from developing rare disease treatments, since companies need certainty when developing drugs for small patient populations, says Kurt Karst, a partner at D.C. law firm Hyman, Phelps & McNamara. If the program is cut, as the current measure proposes, firms in the early stages of drug development might not have time to qualify for a voucher and recoup their investment.

During the full committee markup, Rep. G.K. Butterfield (D-N.C.) offered, then withdrew, an amendment that would allow the FDA to grant vouchers to sponsors after the cut-off date if the application in question had already been designated for a rare childhood disease.

Rep. Bobby Rush (D-Ill.) also withdrew an amendment that would require the National Institute on Minority Health and Health Disparities to draw up plans to include more minorities in clinical trials. He noted that 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 trials.

Committee chairman Fred Upton (R-Mich.) agreed to continue working with the lawmakers on these issues, suggesting new manager’s amendments could be introduced into the bill before it hits the House floor.

The committee plans to get the bill to the House floor this month and have it on the president’s desk by the end of the year. The Senate has no comparable measure.

Read the bill at www.fdanews.com/05-20-15-21CCbill.pdf. — Lena Freund

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