Bill Would Expedite FDA Approval and Increase Clinical Trial Flexibility of Drugs and Treatments That Show Dramatic Early Responses
U.S. Senators Michael Bennet (D-CO), Orrin Hatch (R-UT) and Richard Burr (R-NC) today introduced a bipartisan bill to help bring breakthrough drugs and treatments to patients who need them more quickly.
The Advancing Breakthrough Therapies for Patients Act 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 towards more innovative clinical trials, such as minimizing the number of patients enrolled in trials and shortening the duration of trials, when scientifically appropriate.
“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,” Bennet said. “I have heard from countless Coloradans that the FDA needs to modernize its regulatory science to keep up with 21st century science and technology. Our bill provides the flexibility in approval mechanisms so home-run treatments that show great promise early on 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.”
“This common sense legislation ensures that patients across the country can receive breakthrough treatments faster and more efficiently than ever before,” Hatch said. “Time and again we’ve seen regulators in Washington fail to keep up with the industries they are tasked to oversee. This bipartisan bill ensures that when it comes to treating patients suffering with cancer or other devastating illnesses, science and patient care will not be slowed down by government red tape.”
“As our understanding of diseases and conditions has advanced, targeted cutting-edge medical therapies are increasingly being developed,” Senator Burr said. “We owe it to patients to provide a clear pathway for expediting the development and review of breakthrough therapies so promising innovation can reach patients as soon as possible. Clarifying the regulatory pathway for breakthrough therapies for innovators and the FDA will help fulfill the potential of these therapies for America’s patients, while spurring future generations of life-saving treatments.”
The Advancing Breakthrough Therapies for Patients Act is supported by several organizations, including Friends of Cancer Research, a cancer research think tank and advocacy organization, and the National Venture Capital Association (NVCA), the preeminent trade association for venture capitalists.
“Friends of Cancer Research applauds Senators Bennet, Hatch and Burr for this bipartisan legislation focused on strengthening FDA's ability to get new treatments to patients suffering from diseases like cancer,” said Dr. Jeff Allen, Executive Director of Friends of Cancer Research. “With the passage of this bill, and the establishment of a Breakthrough Therapy Designation, FDA will be able to more actively keep pace with scientific progress, and more quickly engage with developers, ensuring that the most promising new therapies are reaching patients as safely and efficiently as possible.”
“NVCA's members invest in and help develop life-saving treatments for some of our most devastating diseases. But too often the time, cost and uncertainty of meeting regulatory requirements prevent venture capitalists from investing in promising new therapies,” said Jonathan Leff, member of the NVCA’s Board of Directors and Managing Director of the healthcare investing practice at Warburg Pincus. “This bill encourages regulators and drug developers to work together to break bottlenecks in drug development and shorten the path to market, which will help unlock investment and deliver breakthrough treatments to the patients who need them most.”
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.