The bipartisan bill 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. 

“Through the bipartisan ‘Breakthrough’ legislation, Senators Bennet, Hatch and Burr are showing great leadership to address the real needs of patients suffering from diseases like cancer, “said Dr. Ellen Sigal, Chair and Founder of Friends of Cancer Research. 

• For an overview of the legislation from Senators Bennet, Hatch, Burr: CLICK HERE
• For a copy of the full legislation: CLICK HERE
• For an overview of the Breakthrough Concept from the 2011 Friends/Brookings Conference: CLICK HERE

The “Breakthrough” proposal originated through the November 10, 2011, Friends of Cancer Research (Friends) and the Engelberg Center for Health Care Reform at Brookings Conference on Clinical Cancer Research. A panel from this conference addressed potential new approaches that would speed up the process for FDA approval of drugs that show large treatment effects early in development, while still ensuring drug safety and efficacy.

On Thursday, March 8, 2012, Friends Executive Director Dr. Jeff Allen testified before the House Energy and Commerce, Subcommittee on Health, where he discussed the need to examine opportunities to expand the accelerated approval process, and introduced a “Breakthrough Therapies” designation for new therapies that show great benefit early in development. To read Friends of Cancer Research Congressional Testimony: CLICK HERE  

Dr. Sigal introduced Senator Michael Bennet (D-CO) as a “champion for FDA and Cancer Research.” Sen. Bennet, in delivering the opening remarks, underscored the importance of discussing new approval pathways to speed treatments to patients in a faster and safer way, particularly with the upcoming reauthorization of the Prescription Drug User Fee Act legislation in Congress.  He praised the bipartisan nature of the discussion and highlighted the need for patients, as well as the scientific community, to have confidence in the future of drug approvals in a rapidly changing scientific environment.

Senator Bennet Delivers Opening Remarks                                           (L-R) Dr. Ellen Sigal, Dr. Mark McClellan

Dr. Jeff Allen, Friends Executive Director, gave a brief overview of a proposal for a “Breakthrough Therapies” designation for the FDA. The designation, which originated from a panel at the annual Friends-Brookings Conference on Clinical Cancer Research, would trigger close communication between FDA and the drug sponsor, to develop trial designs to shorten or combine traditional phases of development, expedite the pathway to approval and avoid giving larger numbers of patients a potentially harmful or ineffective drug as part of a control arm.

Dr. Woodcock then began the panel discussion by summarizing some of the FDA’s current mechanisms for expediting drug development and review;, accelerated approval, fast-track designation, and priority review status, and pointing out that 80 drugs have been approved under accelerated approval since 1992, 29 of them cancer drugs.

Full Panel during Senate Briefing                                                              (L-R) Jonathan Leff, Howard Scher, Janet Woodcock

Dr. McClellan asked the panel where drug development is headed as a result of new scientific discoveries and progress.  Dr. Scher stated that genetics and biomarkers have changed the way many have looked at and classified certain diseases, using prostate cancer, once thought of as a site specific disease but now known to comprise up to 30 different diseases, as an example. 

The panel discussed current proposals to enhance the FDA, including enhanced accelerated approval and the breakthrough therapy designation. Dr. McClellan asked Dr. Woodcock what she thought of the proposal and whether a mechanism like “breakthrough” would be beneficial.  Dr. Woodcock stated that it would be helpful and that there should be an “all hands on deck” by the FDA when significant is seen early so that a potential breakthrough can be tested and developed as rapidly as possible. She stressed the need for FDA and developers to design trials that are ethical and able to yield information as rapidly as possible.  Dr. Scher agreed and said that anytime you can get everyone together and on the same page, things get done faster.  

Dr. Sigal also reiterated this point and went further to say that “since people, especially in Washington, tend to work in silos, collaboration, communication and clarity are of the utmost importance to alleviating the confusion in all communities about what the agency will or will not do.” 

Mr. Leff went on to discuss the opportunities and challenges to investors in light of these new discoveries.  While the scientific developments present opportunities themselves, Mr. Leff said that high costs and time delays getting drugs through the regulatory cycle are the main barriers to new investment and there is a growing trend toward new areas of investment including health information technology and social media. 

Asked later what investors are looking for to reverse this trajectory, Mr. Leff replied that looking at new approaches and ways to speed up the process is the right conversation to be having, but also later cautioned the audience that it is important to not forget about the many drugs not designated breakthrough therapies moving through the pipeline or lose focus on other treatments because they are not considered to be breakthroughs. 

Dr. Sigal emphasized the importance of continued development of targeted therapies and getting safe and effective treatments to patients, who “at the end of the day want treatments options that will work for them.”