On behalf of the Cystic Fibrosis Foundation (CFF) and the 30,000 people with cystic

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1 Robert J. Beall, Ph.D. President and Chief Executive Officer Cystic Fibrosis Foundation On behalf of the Cystic Fibrosis Foundation (CFF) and the 30,000 people with cystic fibrosis (CF) in the United States, we submit the following testimony to the House Appropriations Committee s Subcommittee on Labor, Health and Human Services, Education, and Related Agencies on our funding requests for fiscal year The Foundation requests the highest possible funding level for the National Institutes of Health (NIH), particularly the National Center for Advancing Translational Sciences (NCATS) and programs under its jurisdiction, including the Cures Acceleration Network (CAN) and the Clinical and Translational Science Awards (CTSA). Collaboration and Innovation: The Future of Drug Development In previous public forums, Chairman Kingston has expressed his view that NIH must use its taxpayer-funded resources wisely. As is demonstrated in the testimony below, NIH does use appropriated funds effectively by supporting programs that promote efficiency and innovation in drug discovery and encouraging collaboration across sectors. Many of these effective, collaborative ventures aim to translate basic research into promising potential treatments, speeding the discovery of therapies for those with serious illnesses like cystic fibrosis. We urge you to ensure that these critical programs are sufficiently funded and receive the support they need. For those with rare genetic diseases like CF, treatments and cures cannot wait. As an example of the NIH s cooperative, innovative approach, in February the agency announced the establishment of the Accelerating Medicines Partnership (AMP), a joint venture

2 between NIH, pharmaceutical companies, and several non-profit organizations to characterize biomarkers and distinguish biological targets that are most likely to respond to new therapies. The AMP will begin with three to five year pilot projects in Alzheimer s disease, type 2 diabetes, rheumatoid arthritis and systemic lupus erythematosus. Through this cross-sector partnership, NIH and industry partners share expertise, resources, and data in order to speed the development of treatments. Furthermore, industry partners have agreed to make AMP data and analyses available to the biomedical community for use in future study. Drug development is risky, expensive, and time-consuming, and there is a 95% failure rate for drug candidates. This kind of cross-sector partnership aims to reduce the time, cost, and risk of drug development by sharing resources so diseases can be analyzed in ways that drug companies have not been able to do on their own. Importantly, industry will fund one-half of the $230 million budget while NIH will provide the other half. The federal money used for this project acts as seed money, a jumping off point for private sector investment in drug discovery for serious diseases. This type of cooperative approach saves taxpayer funds in the long run and can save lives. While AMP is not administered by the National Center for Advancing Translational Sciences (NCATS), this NIH center spearheads similarly innovative programs that encourage collaboration, improve the process by which diagnostics and therapeutics are developed, and improve the efficiency of the translation of basic scientific discoveries into new therapies. For example, the Cures Acceleration Network (CAN), a program under the umbrella of NCATS, funds a variety of initiatives designed to address scientific and technical challenges that hinder transitional research. For instance, CAN provides funding for the Tissue Chip for Drug 2

3 Screening Initiative, a joint project with the Defense Advanced Research Projects Agency (DARPA) and the Food and Drug Administration (FDA) to develop 3-D human tissue chips. These chips, composed of diverse human cells and tissues, mimic how drugs interact with the human body. If successful, these chips could make drug safety and efficacy assessments possible at an earlier stage in drug development, enabling investigators to concentrate on the most promising new drugs. Unfortunately, CAN has been chronically underfunded. Since its inception as part of the Patient Protection and Affordable Care Act in 2010, it has been funded at approximately $10 million per year for fiscal years 2012, 2013, and We urge the Committee to provide at least the funding level requested in the President s fiscal year 2015 budget $29.8 million. CAN needs additional funding for projects that will help move new treatments to patients. Similarly, the Clinical and Translational Science Awards (CTSA) program in the NCATS Division of Clinical Innovation demonstrates NCATS innovative, collaborative approach. This program supports a national consortium of more than 60 medical research institutions that work together on research. Its goals are to accelerate the process of translating laboratory discoveries into treatments for patients, train a new generation of researchers, and engage communities in clinical research efforts. Institutional CTSA awards provide academic homes for translational sciences and support research resources needed by local and national research communities to improve the quality and efficiency of all phases of translational research. They also support the training of clinical and translational scientists and the development of all disciplines needed for a robust translational research workforce. 3

4 CTSA funds have the potential to be used in new ways. For example, CTSA s academic homes can serve as a platform for sharing patient registry data. As the CF Foundation has seen with its Therapeutics Development Network of clinical trial sites, the sharing of patient registry information, including demographics and health outcomes, among sites is integral to conducting CF research. This strategy could be beneficial in the wider disease community. A Culture of Collaboration: The Cystic Fibrosis Model The Cystic Fibrosis Foundation has long been engaged in partnerships with industry and supports a collaborative network of care centers and clinical trial sites. As such, CFF knows firsthand that this type of cooperation can lead to the targeted treatments that change the face of many life-threatening diseases. Because drug research and development is a lengthy, expensive and risky process, CFF pioneered a successful venture philanthropy business model to drive drug development for this rare disease. By collaborating with pharmaceutical companies and providing financial, scientific, and clinical support in order to de-risk the development process, CFF speeds development of much-needed treatments. Through its venture philanthropy model, the Foundation is able to invest in promising CF research and 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 ongoing work with researchers and the pharmaceutical industry to find a cure. In January 2012, the Food and Drug Administration approved Kalydeco, a groundbreaking cystic fibrosis drug developed by Vertex Pharmaceuticals in partnership with the CF Foundation. This targeted drug is the first to address the underlying genetic cause of cystic fibrosis in a subset of the CF population. 4

5 Kalydeco was approved in only 3 months, one of the fastest approvals in the FDA s history. According to Margaret A. Hamburg, M.D., Commissioner of the FDA, The unique and mutually beneficial partnership that led to the approval of Kalydeco serves as a great model for what companies and patient groups can achieve if they collaborate on drug development. Throughout Kalydeco s review, the Cystic Fibrosis Foundation and renowned CF experts worked closely with Vertex Pharmaceuticals and the FDA, providing valuable insight on specific issues related to CF, clinical research on CF treatments, and other issues related to the product and its review. We believe that this collaborative process contributed to a more efficient evaluation, and is a testament to what can be achieved when stakeholders collaborate across sectors on critical drugs for patients. Akin to AMP, the Cystic Fibrosis Foundation also recognizes the profound importance of data sharing, which is a critical way to enable efficient drug development. The Cystic Fibrosis Foundation Therapeutics Development Network (TDN) of clinical trial centers has accumulated data from over 40 cystic fibrosis (CF) studies in the last 15 years. This data resides in a repository specifically meant to facilitate sharing among our research community. *** As the Committee determines its funding levels for fiscal year 2015, we request your attention to the critical nature of NIH s work and the innovation it supports, and urge robust funding for this important agency. The CF Foundation stands ready to work with the Committee, NIH, and Congressional leaders on the challenges ahead. Thank you for your consideration. 5