cystic fibrosis (CF) in the United States, we are pleased to submit the following testimony to the

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1 Robert J. Beall, Ph.D. Cystic Fibrosis Foundation President and Chief Executive Officer 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 Appropriations Committee s Subcommittee on Agriculture, Rural Development, Food and Drug Administration, and Related Agencies on our funding requests for fiscal year In order to encourage efficient review of drugs for cystic fibrosis and other rare diseases, we urge the Committee to prioritize the Food and Drug Administration (FDA) in fiscal year 2014 by providing the highest possible funding level for this critical agency. We urge special consideration and support for the Center for Drug Evaluation and Research (CDER), its Office of New Drugs (OND), and the Office of Orphan Products Development (OOPD). The Food and Drug Administration Sufficient Funding for Swift Drug Review The Cystic Fibrosis Foundation requests that the Committee provide robust funding for drug review at the Food and Drug Administration in fiscal year The FDA has broad and significant responsibilities, regulating roughly 25 percent of consumer spending, and its responsibilities grow each year. In light of this, we are significantly concerned about the impact of the recently-enacted sequester, which results in a loss of about $208 million, 5.1 percent of the agency s budget. This figure includes sequestration of user fees for prescription drugs. The Foundation strongly opposes sequestering these funds, which are collected from industry for the express purpose of supporting drug review and are critical to the efficient review of rare disease

2 treatments. It is vital that the FDA have the funding it needs to ensure that the nation has a safe and effective supply of drugs and devices and that the agency can give the necessary attention to reviewing therapies that treat small patient populations and serve specific unmet medical needs. The Cystic Fibrosis Foundation applauds FDA Commissioner Dr. Margaret A. Hamburg, CDER Director Dr. Janet Woodcock, and Associate Director for Rare Diseases Dr. Anne Pariser for their sensitivity to the challenges posed by the evaluation of rare disease treatments and support for swift rare disease drug review and approval. Last year s approval of Kalydeco, a groundbreaking cystic fibrosis treatment called the most important drug of 2012 by Forbes Magazine, is an example of this support. Reviewed and approved in only three months, Kalydeco was one of the fastest approvals in the history of the FDA. Kalydeco s review is a testament to the agency s efficiency and its commitment to patients who live everyday with serious chronic diseases. Kalydeco, developed by Vertex Pharmaceuticals in cooperation with the Cystic Fibrosis Foundation, is the first treatment to target the underlying genetic cause of cystic fibrosis. It is effective in those with a particular CF mutation, impacting about 4 percent of the cystic fibrosis population. Other treatments like Kalydeco that target the root cause of the disease in larger portions of the CF population are moving quickly through the pipeline. Phase 3 clinical trials are underway to study a combination of Kalydeco and a new compound, VX-809, which would treat those with the most common CF mutation, affecting about 50 percent of those with CF in the United States. Additionally, the CF Foundation has significantly expanded its research investments with leading pharmaceutical companies, including Pfizer, Genzyme and Vertex, to accelerate the discovery and development of new drugs that will help more CF patients. 2

3 As innovative, genetically-targeted treatments like Kalydeco are developed, it is important that the FDA has the resources it needs to support a sufficient amount of reviewers and retain those with appropriate and extensive expertise in rare diseases in order for new drugs to be evaluated effectively, efficiently and without delay. The review of rare disease drugs involves myriad issues not faced in the evaluation of treatments for more common ailments. For example, treatments akin to Kalydeco target specific genetic mutations. However, there are over 1,000 mutations of cystic fibrosis and some of these mutations may impact very small patient populations, creating challenges in designing clinical trials. In some cases it may be necessary to consider alternate approaches, including accelerating classification of biomarkers, testing combinations of drugs in populations that might include patients with several different CF mutations and the development and testing of single and combination therapies in so-called n of 1 trials (trials consisting of a single patient). Experienced FDA personnel who understand the complexities of rare disease clinical research contribute to a more nimble review process and cultivate an environment where more novel approaches are considered. Providing the FDA with sufficient resources is the only way the agency can retain the high caliber workforce it needs to review the life-saving drugs of tomorrow. The Food and Drug Administration Safety and Innovation Act (FDASIA) In July 2012, the President signed the Food and Drug Administration Safety and Innovation Act (FDASIA) into law. This legislation included many provisions with the potential to speed the development and review of rare disease treatments, but it cannot be implemented effectively unless the FDA has adequate resources. 3

4 One provision in particular outlined in FDASIA is a new pathway for Breakthrough Therapies. This new pathway was created to expedite the development and review of a potential new medicine intended to treat a serious or life-threatening disease or condition, which evidence indicates could demonstrate substantial improvement over existing therapies. The first two Breakthrough Therapy designations were awarded to Vertex Pharmaceuticals for Kalydeco monotherapy (use of Kalydeco by people with certain CF mutations not evaluated in prior studies) and the combination regimen of Kalydeco and VX-809 currently being studied in clinical trials. This additional pathway has the potential to help move new treatments quickly to CF patients, but the FDA needs sufficient funding to implement it effectively. Another provision, recommended by the Cystic Fibrosis Foundation for inclusion in FDASIA, encourages FDA consultation with external experts regarding the review of rare disease and genetically targeted drugs, authorizing the Secretary to maintain a list of experts to consult on rare disease topics. These topics include the unmet medical need associated with rare diseases, an assessment of the benefits and risks of therapies to treat rare disease and the design of clinical trials for rare disease populations and subpopulations. As we reap the benefits of the mapping of the human genome and the creation of innovative models for advancing drug development, FDA outreach to external rare disease experts will be more important than ever. An example of the importance of this type of outreach can be found in the swift approval of Kalydeco. Throughout Kalydeco s review, the Cystic Fibrosis Foundation and renowned CF experts worked closely with Vertex and the FDA, providing valuable insight on very specialized issues. We believe this contributed to an efficient review and approval, a testament to what can be achieved when all interested stakeholders collaborate. 4

5 Collaboration Leads to Innovation The CF Foundation urges the Committee to support funding for collaborative efforts between 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 FDA and NIH can help move innovative new drugs more quickly through the development process and into the hands of patients by providing an opportunity to share resources, strategies, and tools, streamlining the development process from the laboratory to FDA review and enhancing the regulation of drugs in this ever-changing scientific landscape. About Cystic Fibrosis and the Cystic Fibrosis Foundation Cystic fibrosis is a rare genetic disease that causes the body to produce abnormally thick mucus that clogs the lungs and results in life-threatening infections. This mucus also obstructs the pancreas and stops natural enzymes from helping the body break down and absorb food. The Cystic Fibrosis Foundation s mission is to find a cure for CF and improve quality of life for those living with the disease. Through the Foundation s efforts, the life expectancy of a child with CF has doubled in the last 30 years and research to find a cure is more promising than ever. The Foundation s research efforts have helped create a robust pipeline of potential therapies that target the disease from every angle. Nearly every CF drug available today was made possible because of the Foundation s support and our ongoing work to find a cure. *** Once again, we urge the Committee to make funding for the Food and Drug Administration a priority in fiscal year 2014, and stand ready to work with the Committee and Congressional leaders on the challenges ahead. Thank you for your consideration. 5