Revised 'Cures' Proposal Seeks to Simplify Clinical Trials, Speed Approvals

Among the multiple proposals for stimulating drug development and revising regulatory processes, the 21^st Century Cures initiative includes several provisions designed to streamline clinical research and the amount of data required to gain FDA approval of certain indications. The draft measure issued with bipartisan support by the House Energy & Commerce (E&C) Committee promotes the development of new biomarkers and other drug development tools and offers a number of strategies for advancing precision medicine and speeding breakthrough drugs and medical devices to patients. It includes a program to quickly develop new antibiotics for limited use by certain patients and for narrow conditions, utilizing fast and small clinical trials.

Biopharma companies lost a number of earlier provisions that provided added exclusivity for certain new therapies. E&C Republicans say they will continue to seek ways to restore some of these incentives for developing important new drugs, but there’s strong opposition from many Democrats. Also controversial is a proposal under the heading of “modern trial design and evidence development” that would encourage biopharma companies to use “clinical experience” rather than data from randomized trials to support approval of new indications. The measure also permits FDA to quickly approve certain additional drug uses based on data summaries, rather than the full data package. FDA would hold a public meeting and issue guidance documents to assist sponsors in incorporating adaptive trial designs and Bayesian statistical modeling into study protocols, and a new pilot program would explore additional ways of using data from FDA’s Sentinel System to access real-world evidence.

There’s less dispute, though, over another section of the Cures proposal that would streamline clinical trials by supporting broader use of centralized institutional review boards, particularly for medical device studies. And in the interest of avoiding redundant regulation that can delay enrollment and protocol review, the legislators would exempt FDA-regulated clinical trials from also meeting requirements set by the Common Rule.

While FDA officials stated general support for many of these provisions at the E&C Committee hearing on April 30, they raised concern that the legislation would require FDA to establish new programs and issue more guidance without providing any additional resources. This latest E&C proposal has “significant resource implications for FDA,” stated Janet Woodcock, director of the Center for Drug Evaluation and Research (CDER), pointing out that added mandates could divert resources from application review and biomarker development. CDER’s new drug review process is now “going at full speed, and we’d like to keep it that way,” Woodcock commented, noting that “there’s always a trade-off” in putting out new guidances and getting reviews done. Being able to offer timely advice to biopharma companies helps accelerate new drug development, she noted, but support for multiple advisory meetings “would be the first to go” if the agency gets further stretched on resources.

Woodcock also expressed reservations about a proposal that encourages pharma companies to communicate economic information to formulary committees and payers. Drug marketers also gained a change in the "Sunshine" disclosure program to make it easier to distribute journal articles and medical textbooks to physicians. In response to a query about why FDA doesn’t let sponsors “tweet” about a new drug with a link to full safety and efficacy information, Woodcock observed that manufacturers have “a different stake in presenting information than the agency.” She agreed to work with the legislators to develop a “common-sense” approach to social media communications about drugs.

A main feature of the new Cures proposal is its significant boost in funding for the National Institutes of Health (NIH). While FDA gains no specific additional resources, the legislators grant NIH an added $5 billion in funding over three years to achieve a budget of $35 billion in 2018. And there’s another $2 billion a year to support an NIH Innovation Fund. The NIH funding increase is key to generating bipartisan support for the package, and opens the door to  action on some kind of compromise measure by summer.