The result of this understanding is an emerging paradigm shift for the development of new medicines. This paradigm is beginning to yield drugs that may have much more of a positive impact on the lives of these patients than traditional therapies.
Over the past year, our organizations - Friends of Cancer Research and the Engelberg Center for Health Care Reform at Brookings - have convened a series of workshops to explore potential scientific strategies that FDA and drug researchers could use to address this new paradigm. We found that FDA and the scientists involved in product development have already taken a number of promising steps to respond to these new trends in medical product development. Working with FDA, we also identified some critical further steps to make sure that the development and regulatory processes for breakthrough drugs are aligned with the rapid progression of science.
Recently, to ensure that FDA can actively and quickly engage, Senators Bennet (D-Colo.), Hatch (R-Utah) and Burr (R-N.C.) introduced a bill in the Senate to establish a Breakthrough Product Designation.
The Breakthrough Product Designation has two main goals: 1) to reduce the total development time and cost of the most promising "breakthrough" treatments; and 2) to minimize the number of patients that would be given a "control" regimen or a currently available treatment that doesn't work well. This legislation does not alter current FDA standards and any new drug would continue to be required to fully demonstrate both safety and efficacy.
Upon designation, the FDA and the drug sponsor would engage in intensive interactions to collaboratively construct and agree upon a development strategy for the drug. This could include the use of a variety of innovative clinical trial designs, or specific strategies for accumulating further evidence on the breakthrough drug after it is approved.
In order to be most effective, the Breakthrough Product designation would be given as early as possible, so that the sponsor and FDA can discuss ways to condense traditional, often lengthy multi-phase trials into rigorous and efficient protocols to cut that time down. This designation would allow FDA to prepare for and implement a consistent strategy for potentially revolutionary medicines. It would signal to developers that, for the most promising products to battle severe illness and disease, the FDA will actively marshal these products through the course of development - and would help them identify the most efficient ways to develop their drugs.
McClellan is the director of the Engelberg Center for Health Care Reform and senior fellow in economic studies at The Brookings Institution, and served as commissioner of the Food and Drug Administration. Sigal is chairperson and founder of Friends of Cancer Research, a cancer research think tank and advocacy organization based in Washington, DC.