Colorado U.S. Senator Michael Bennet’s bipartisan bill to help bring breakthrough drugs to patients who need them more quickly has been included in a Senate committee plan introduced today. 

The Senate Health Education Labor and Pension Committee unveiled a bipartisan plan to advance important medical and bioscience innovations through reforms to the U.S. Food and Drug Administration (FDA). It includes the Advancing Breakthrough Therapies for Patients Act, introduced by Bennet, along with Senators Orrin Hatch (R-UT) and Richard Burr (R-NC).

The committee’s Drug Approval and Patient Access Plan is a discussion draft of a bill and is one component of a larger FDA reauthorization.

“We’re seeing major breakthroughs in drugs and other treatments for debilitating and terminal diseases, but we’re not always getting them to patients though the most efficient and safe pathways. This bipartisan plan is a good starting point to ensure patients have access to safe, innovative treatments as quickly as possible,” said Bennet. “The inclusion of the new “breakthrough” designation provides the flexibility to allow home-run treatments that show great promise early can reach patients more quickly. It also strikes a careful balance between providing regulatory certainty for developers of these breakthrough treatments and maintaining the level of drug safety and efficacy patients expect and deserve.”

The Drug Approval and Patient Access discussion draft aims to enhance accelerated patient access to new medical treatments by modernizing FDA’s Accelerated Approval pathways and creating a new “breakthrough” designation to provide more flexibility and certainty for developers of new medicines intended to address serious or life-threatening diseases or conditions.

The Bennet-Hatch-Burr Advancing Breakthrough Therapies for Patients Act, which is included in the plan, would expedite U.S. Food and Drug Administration (FDA) approval and provide more flexibility when a drug or treatment shows dramatic responses early in development, while still ensuring drug safety and efficacy. For patients, this proposal would allow FDA the ability to move toward more innovative clinical trials, such as minimizing the number of patients enrolled in trials with a placebo and shortening the duration of trials, when scientifically appropriate.

This bill amends the Food, Drug, and Cosmetic Act to require FDA to facilitate the development and expedite the review of breakthrough drugs. Breakthrough drugs are drugs intended for a serious or life-threatening disease or condition where preliminary clinical evidence indicates that they may demonstrate substantial improvement over existing therapies. 

If the FDA determines that a drug meets the criteria to be designated as a “breakthrough therapy” then the agency shall take such actions as are appropriate to expedite the development and evaluation of the drug, including establishing an interactive development process;  providing timely advice to the sponsor regarding the plan to develop the drug; enlisting a collaborative, cross-disciplinary approach by senior managers and experienced review staff; and minimizing the number of patients enrolled in and shortening the duration of a clinical trial. A breakthrough therapy may still seek fast-track product designation, accelerated approval, or priority review.