21st Century Cures: One Step Forward and Two Back?

In mid-May, the House Energy and Commerce Committee unanimously approved the 21^st Century Cures Act, a non-partisan law that brought to fruition the year-long non-partisan efforts of Congressman Fred Upton (R-MI) and Congresswoman Diana DeGette (D-CO). The expectations are that the act’s provisions will allow for rapid breakthrough innovations in healthcare by supporting basic research as well as the drug development and approval process.

In response to this approval, a perspective in the New England Journal of Medicine authored by physicians—experts in healthcare policy and pharmacoeconomics—at Harvard evaluated the implications of the act. The authors welcomed the support promised by 21^st Century Cures to funding basic research efforts of the National Institutes of Health (NIH), saying this would “counteract the effects of sequestration and budget cuts that have reduced the purchasing power of the NIH to its lowest level in years.”

While the bill also hoped to accelerate the drug approval process, the authors emphasize that the FDA is already quite adept at moving breakthrough therapies quickly through the process (an average time of 6 to 10 months) to allow for rapid access. They highlight 1 improvement or allowance of the act: the use of nontraditional trial designs such as adaptive designs and the use of Bayesian methods that would be particularly useful for oncology drugs, but warn against the use of shorter trial periods or the use of clinical experience for approval.

With rising global concerns on emergent bacterial resistance to existing antibiotics, the act allows for approval of antibiotics and antifungals, without evidence from a conventional clinical trial, in case of a “serious or life-threatening infection,” say the authors.

Finally, with respect to medical devices, which have always been a topic of contention among stakeholders, the new law provides several useful amendments to validate and standardize devices, but the authors warn that “political forces have introduced provisions that could approve less safe or ineffective drugs and devices.”

Avorn J, Kesselheim AS; 21^st Century Cures Act — will it take us back in time? [published online]. N Engl J Med. doi:10.1056/NEJMp1506964.