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Washington Update: Mark McClellan & Ellen Sigal Op-Ed Supporting FDA Breakthrough Designation

Washington Update: Mark McClellan & Ellen Sigal Op-Ed Supporting FDA Breakthrough Designation


Former FDA Commissioner Dr. Mark McClellan and Friends Chair Dr. Ellen Sigal Pen Op-Ed Supporting Breakthrough Designation


Over 45 Organizations Sign Congressional Support Letter for Breakthrough Therapies Designation to be Included in PDUFA V


  • March 26, 2012: The “Advancing Breakthrough Therapies for Patients Act of 2012” was introduced by Senators Bennet (D-CO) Hatch (R-UT) and Burr (R-NC). For an overview of the bipartisan Senate legislation: CLICK HERE
  • April 4, 2012: The “Breakthrough” legislation was included in a plan released by the Senate’s Health Education Labor and Pension Committee (HELP). The committee’s bipartisan “Drug Approval and Patient Access Plan” is a discussion draft of a bill and is one component of the larger FDA re-authorization. For a copy of the discussion draft CLICK HERE
  • April 16, 2012: Over 45 organizations signed a support letter, sent to the House Energy and Commerce Committee, asking that a Breakthrough Therapy designation be included as part of the upcoming reauthorization of the Prescription Drug User Fee Act (PDUFA). For a copy of the letter CLICK HERE
  • April 20, 2012: An op-ed supporting the “Breakthrough” designation, authored by Dr. Ellen Sigal, Chair & Founder of Friends of Cancer Research and Dr. Mark McClellan, former FDA Commissioner and Director of the Engelberg Center for Health Care Reform at Brookings, was published in The Hill. 


The Hill: Getting drugs to market place faster

By Mark McClellan, Brookings Institution and Ellen Sigal, Friends of Cancer Research


Science is progressing at a rapid pace. The human genome sequence has been completed and evidence on how diseases occur at the molecular level is growing by the day. Researchers and clinicians have been steadily working to use this data to make personalized medicine become a reality. However, with all of this new information, these prospective breakthroughs aren’t yet reaching patients as quickly as needed. In the coming year alone, cancer will claim the lives of over 550,000 Americans. To address this incredible burden, and to ensure that patients have access to the most beneficial treatments, the process for turning these scientific insights into safe and effective treatments needs to accelerate as well.


Our progress in understanding the specific pathways of disease has identified hundreds of new targets for potentially life-saving drugs that hold the potential to treat individual patients much more effectively.


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.


The regulators at the FDA must be fully engaged in this shift in development, which may involve a sharpening focus on particular subsets of a disease.  When a new drug or drug combination shows extreme activity very early, like these new targeted agents are capable of doing, new approaches are needed that focus on the most efficient mechanisms to get a potential medical breakthrough to the relevant patients – and that may require significant revisions in the way such drugs are tested for safety and effectiveness under the FDA’s oversight.


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.