Robert J. Beall, PhD, President and Chief Executive Officer Cystic Fibrosis Foundation

Transcription

1 Name and Organization: Robert J. Beall, PhD, President and Chief Executive Officer Cystic Fibrosis Foundation Subject of Testimony: Food and Drug Administration Funding for Fiscal Year 2013 Point of Contact: Mary Dwight, Vice President of Government Affairs On behalf of the Cystic Fibrosis Foundation and the approximately 30,000 people with cystic fibrosis (CF) in the United States, we are pleased to submit the following testimony to the House of Representatives Appropriations Subcommittee on Agriculture, Rural Development, Food and Drug Administration, and Related Agencies requesting sufficient funding for the Food and Drug Administration in Fiscal Year This testimony urges the Committee to provide the Food and Drug Administration the funding it needs to quickly and efficiently review treatments for CF and other rare diseases and encourages the FDA to reach out on a more systematic basis to outside experts early in the drug development process. Additionally, the CF Foundation urges the Committee to support collaborative efforts by the FDA and the National Institutes of Health, such as the Regulatory Science Initiative and the FDA-NIH Joint Leadership Council. Collaboration between the NIH and FDA has the potential to help move innovative new drugs more quickly through the development process and into the hands of patients. In particular, the Foundation wishes to commend the speed with which the FDA approved Kalydeco, a breakthrough treatment for cystic fibrosis that is the first to address the underlying genetic cause of the disease for 1,200 people with CF who carry a specific genetic mutation. The agency reviewed and approved Kalydeco s New Drug Application in only three 1

2 months - one of the fastest approvals of any drug in the history of the agency. The speed with which this review was conducted is a testament to the FDA s commitment to collaboration with Vertex Pharmaceuticals, Kalydeco s developer, and the Cystic Fibrosis Foundation, as well as its commitment to the patients who already are benefiting from the drug. The science behind Kalydeco has opened exciting new doors to research and development that may eventually lead to a cure for all people living with CF. ABOUT CYSTIC FIBROSIS Cystic fibrosis is a life-threatening genetic disease for which there is no cure. People with CF have two copies of a defective CFTR gene, which causes the body to produce abnormally thick, sticky mucus that clogs the lungs and results life-threatening lung infections. This mucus also obstructs the pancreas, preventing pancreatic enzymes from assisting in the breakdown of food and the absorption of nutrients. The mission of the Cystic Fibrosis Foundation is to find a cure for cystic fibrosis and improve the quality of life for people living with the disease. This is accomplished by funding life-saving research and working to provide access to quality care and effective therapies for people with CF. Through the Foundation s efforts, the life expectancy of a child with CF has doubled in the last 30 years. Although real progress toward a cure has been made, the lives of young people with CF are still cut far too short. The promise for people with CF lies in research. The CF Foundation has raised and invested hundreds of millions of dollars in private money to help develop CF drugs and therapies and nearly every CF drug available today was made possible because of the Foundation s 2

3 support. The Foundation accredits a nationwide network of over 110 CF care centers that has been widely recognized as a national model for specialized treatment of a disease. SUSTAINING FUNDING FOR RARE DISEASE DRUG REVIEW AT THE FDA Funding for Rare and Orphan Disease Drug Review In order to encourage swift review of drugs for CF and other rare diseases, we urge the Committee to recommend sufficient funding for the Food and Drug Administration, particularly the Center for Drug Evaluation and Research (CDER) s Office of New Drugs, in Fiscal Year To be effective, the FDA needs an adequate number of reviewers with the appropriate skills and expertise to evaluate therapies for rare diseases like cystic fibrosis. Additional support for the FDA through increased funding not only ensures that the nation has a safe and effective supply of drugs and devices, but also that the agency can give the necessary attention to reviewing therapies that treat small populations and serve specific unmet medical needs. It is more critical than ever that Congress significantly increase funding for the Center for Drug Evaluation and Research (CDER) at the FDA and for the agency as a whole in FY2013 so that it can meet its statutory obligations in a timely manner. Accelerating the Rare Disease Drug Review Process at the FDA The Cystic Fibrosis Foundation applauds the FDA and Associate Director for Rare Diseases Dr. Anne Pariser in particular for their attention to rare disease drugs and sensitivity to the unique challenges posed by the evaluation of these treatments. As we reap the benefits of the mapping of the human genome, treatments like Kalydeco are being developed that target smaller and smaller populations. This aspect of personalized 3

4 medicine holds the promise to treat or cure rare diseases and subsets of more common diseases that plague millions of Americans. However, as the scientific landscape changes, it is important that the FDA has access to the expertise it needs to swiftly review innovative new treatments. FDA review officials have taken steps to improve access to scientific expertise during the review of therapies that treat rare diseases, and FDA leaders and review staff have been willing to engage in constructive dialogue to address the challenges of rare disease review. The agency has taken part in productive conversations with researchers and patients at the CF Foundation, including with many of the world s foremost experts on cystic fibrosis, on the development and review of potential therapies to treat cystic fibrosis and on topics separate from specific drug review, such as improving tools for Patient Reported Outcomes (PROs). In particular, the collaboration showcased during the review of Kalydeco is an excellent example of how the FDA, a drug sponsor, patients and external experts can work to effectively evaluate new drugs and accelerate the approval process. However, in some cases the opportunity for public comment is not available if the product in question is not the subject of an advisory committee. In all cases, this public comment period occurs very late in the review process. While FDA review divisions do conduct some consultations with external experts separate from the advisory committee process, the complexity and diversity of applications for rare disease therapies suggest that the agency would benefit from more regular consultation with extramural experts early in the review process. The Cystic Fibrosis Foundation asks that the Committee encourage the FDA to reach out on a more systematic basis to outside experts early in the drug development process. One such strategy Congress is considering is the proposed Expanding and Promoting Expertise in Review of Rare Treatments (EXPERRT) Act, H.R CFF strongly supports the 4

5 EXPERRT Act, which establishes a program to facilitate FDA outreach to external experts earlier and throughout the drug review process on issues such as unmet medical need, genetically targeted treatments, disease severity, clinical trial design and patient demographics. Additionally, the CF Foundation urges the Committee to support collaborative efforts by the Food and Drug Administration and the National Institutes of Health, such as the Regulatory Science Initiative and the FDA-NIH Joint Leadership Council. Collaboration between the NIH and FDA has the potential to help move innovative new drugs more quickly through the development process and into the hands of patients by ensuring that the FDA has the resources, strategies, and tools it needs to efficiently review and regulate drugs in this ever changing scientific landscape. As treatments like Kalydeco are being developed to target specific genetic mutations and smaller and smaller populations, it is important that the FDA has the expertise it needs to quickly move these drugs through the review process. **************** The Cystic Fibrosis Foundation s unique and successful drug development model for creating treatments for a rare disease has helped create a robust pipeline of potential therapies to fight cystic fibrosis. The Food and Drug Administration has played a critical role in this process, working with the Foundation as they review treatments and move them into the hands of patients. Encouraged by our successes, we believe the experience of the CF Foundation in clinical research can serve as a model of drug discovery and development for research on other orphan diseases and we stand ready to work with the FDA and congressional leaders. On behalf of the Cystic Fibrosis Foundation, we thank the Committee for its consideration. 5