By Clinical Informatics News Staff

January 28, 2015 | The House Committee on Commerce and Energy has published a draft version of the 21st Century Cures Act, a sweeping bill containing numerous changes to the FDA's powers to review and approve drugs and medical devices. While many amendments are to be expected as the bill winds its way through the full House and Senate, a combination of rare bipartisan support — an earlier piece of legislation included in the draft bill attracted nearly 100 cosponsors, evenly split between Democrats and Republicans — and the President's stated interest in increased federal support for new categories of therapies makes it appear likely that some version of the legislation will be enacted in the 114th Congress. At the same time, the current draft bill appears to have lost some Democratic support; ranking member of the Commerce and Energy Committee Rep. Frank Pallone, Democrat of New Jersey, said in a press release, "I am disappointed that the discussion document released today by [Republican] Chairman [Fred] Upton does not reflect true bipartisan collaboration."

Many of the bill's proposals build on successful recent experiments at the FDA, including breakthrough therapy designations that give accelerated timelines to the most promising new drugs, new clarification around patient-centered outcomes and surrogate endpoints that help pharma companies use alternative metrics to support their submissions, using well-curated public data rather than new clinical trials to support certain claims, and market vouchers that reward companies for pursuing certain indications with transferrable benefits to future submissions. Among other measures, the 21st Century Cures Act would: introduce a new tradable voucher for one year of market exclusivity, given to companies who develop new antibiotics; introduce a category of "breakthrough devices" parallel to breakthrough therapies (notably, the FDA definition of devices includes diagnostic tests); let the FDA approve some therapies based on early stage trial results, with the option to revoke approval as trials continue; and codify the use of variant databases and the scientific literature in approving new tests for genetic mutations.

The draft bill also includes some measures the FDA is likely to be wary about, including opening up Twitter and other character-limited web platforms to drug marketing, and making it easier for terminally ill patients to receive unapproved drugs on a "compassionate use" basis. Among the most controversial measures of the draft bill will be the introduction of 15 years of market exclusivity for new drugs with at least one unmet need. That length is unprecedented and is sure to be opposed by the generic drug industry and critics of pharmaceutical companies, especially as unmet needs are broadly defined in the draft.

The entire draft bill, or discussion document, can be read here. Meanwhile, the Regulatory Affairs Professionals Society has ongoing analysis of the bill's provisions here, which is well worth reading.