21st Century Cures Act Clears U.S. House

Last week, the House of Representatives approved the 21st Century Cures Act by a vote of 344 to 77, moving focus to the Senate where efforts have begun to develop comparable legislation.

Sen. Lamar Alexander (R-Tenn.), chairman of the Senate HELP Committee, has said his committee is crafting legislation. “The HELP Committee is working on a bipartisan basis on this initiative and currently has five working groups meeting weekly on a variety of FDA and NIH issues relating to drug and device innovation, from research to approval,” spokeswoman Margaret Atkinson says.

Peter Pitts, president of the Center for Medicine in the Public Interest, says the Senate could pass legislation by the fourth quarter of this year.

The massive FDA overhaul bill, H.R. 6, 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 2020 to include evidence from observational studies, registries and real-world therapeutic use. The information could be used to support new drug indications and postmarket study requirements.

The bill also would encourage broader use of observational studies, patient registries and actual therapeutic use to support approvals of new drugs and enhance postmarket surveillance, and would allow for greater use of Bayesian statistics and other innovative statistical methods in clinical trial designs.

To speed access to novel therapies, drugmakers would be guaranteed FDA approval if investigational products met intermediate endpoints in an agreed-to development plan.

Extra Exclusivity

H.R. 6 also provides an extra six months of marketing exclusivity when drugmakers adapt existing products to treat rare diseases — beyond the seven years of exclusivity drugs get when granted orphan drug status. And it extends the rare pediatric disease priority review voucher program through December 2018. The program is currently set to expire in March 2016.

The bill also would give hospitals an additional Medicare payment when they use new antibiotics. On Friday, the House approved, by a voice vote, just one amendment affecting drugmakers — a provision requiring the Centers for Disease Control and Prevention to study whether incentivizing the use of new antibiotics will lead to antibiotic resistance and cause these lifesaving drugs to be less effective.

The amendment, cleared for consideration by the House Rules Committee on Wednesday, applies to so-called DISARM drugs — new antimicrobials approved to treat an infection for which there is an unmet medical need and a high rate of mortality or severe illness.

The White House welcomed the additional support for biomedical research and innovation, but said it is concerned about providing additional funding for the FDA without addressing sequestration more broadly. The new responsibilities for the FDA outlined in H.R. 6 exceed the monies authorized for the agency in the president’s FY 2016 budget. The administration also raised concerns over how the provision for extending drug exclusivity beyond current law could affect drug costs.

Consumer advocacy group Public Citizen echoed that concern, saying the six-month exclusivity extension would further delay generic entry of drugs into the market and deny patients access to affordable, lifesaving drugs. — Jonathon Shacat