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Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary 8 Considering Next Steps As noted by both Hamburg and Goodman, a wealth of scientific and regulatory knowledge already exists at FDA. There have been a number of examples of successful use of regulatory science and collaborative models at the agency level. Likewise, several recent initiatives—the Food and Drug Administration Amendments Act, the creation of the Office of the Chief Scientist, increased funding and recruitment, and enhanced FDA–NIH interaction—represent efforts to move in the direction of scientific regulatory decision making. On February 24, 2010, HHS Secretary Katherine Sebelius announced a new partnership between FDA and NIH to spur the development, evaluation, and approval of new medical products. The partnership focuses particularly on strengthening regulatory science research. The FDA–NIH collaboration represents the type of partnership advocated by workshop speakers. As HHS Deputy Secretary Corr stated: NIH is the world’s leader in biomedical research and FDA is the gold standard across the world in the evaluation and approval of drug products and other medical products. Secretary Sebelius and I are confident that, with the two dedicated leaders we have in Peggy Hamburg, one of the nation’s foremost advocates for public health, and in Francis Collins, one of the nation’s most distinguished scientists, they will lead us into a new era in which we can promote and protect public health by realizing the promise of science. The primary goal of the FDA–NIH Collaboration Initiative is to improve and harmonize the functions of two interrelated disciplines,
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Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary regulatory and translational science, through the expertise available in FDA and NIH for efficiently and effectively guiding biomedical discoveries into safe therapies for the public. It will be led by a newly established Joint FDA–NIH Leadership Council,1 which will be charged with including regulatory considerations in biomedical research and vice versa. As a part of the Initiative, FDA and NIH issued a joint Request for Applications making available $6.75 million dollars for work in regulatory science over the course of three years. “We now have a special opportunity—and responsibility—to harness advances in science and technology to support our efforts. We are working in collaboration with the best minds and research institutions available, so that we can better develop and utilize new tools, standards and approaches needed to properly assess the safety, effectiveness and quality of products currently in development or already on the market,” said Hamburg at the announcement of the initiative (NIH, 2010). IMMEDIATE NEXT STEPS FOR FDA Goodman stated that FDA must proceed with the development of regulatory science regardless of external barriers. He outlined the following key implementation steps for first creating an internal support system for a regulatory science infrastructure at the agency: Leadership to strengthen and support science and promote innovation at FDA—Both the Commissioner’s Office and leadership at FDA centers are supportive of promoting science and innovation. There is interest across the agency in multicenter working groups, scientific guidance, and sharing of resources. Goodman also announced the creation a new Office of Science and Innovation within the Office of the Chief Scientist to coordinate such efforts. Excellence in professional development—Professional development is essential to a scientific career and needs to be built in at the agency level. Goodman acknowledged that some FDA staff are unable to attend professional meetings because of either funding or time constraints. Given the success of staff colleges at each FDA center, Goodman encouraged the development of training programs at NIH and local universities, as well as online programs for staff. Recruitment and retention of outstanding scientists—Building regulatory science at FDA will require that the agency recruit and 1 Available at http://www.fda.gov/ScienceResearch/SpecialTopics/RegulatoryScience/ucm201654.htm (accessed September 24, 2010).
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Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary retain top talent. To reverse its high turnover rate the agency will need to continue and expand fellowship programs such as its present Commissioner’s Fellowship Program2 and other tools such as its scholar in-residence sabbatical programs. BUILDING A FRAMEWORK: THEMES FROM THE WORKSHOP Weak regulatory science is not a problem just for the FDA; it is a shared problem that requires a shared solution. Several workshop participants called for development of a common framework for enhancing regulatory science. A number of individual suggestions were made during the workshop about the initial steps toward building a national regulatory science infrastructure. They are compiled here as part of the factual summary of the workshop, and should not be construed as reflecting consensus or endorsement by the workshop, the Forum, or the National Academies. They are as follows: Enable emerging technologies to strengthen regulatory science. Massive amounts of new data are expected to come from emerging technologies in such areas as rare adverse events, genomics, and biostatistics. Each of these areas presents unique opportunities for regulatory science, as well as the potential for serious adverse consequences if the gaps between scientific and regulatory needs are not bridged. A strategic plan is critical. While many of the elements of regulatory science are already instilled in the functioning of FDA, a strategic approach will be necessary going forward. The blueprint should account for the numerous complexities that will be faced, should acknowledge the need for priority setting, and should involve all stakeholders. Approach barriers to enhanced regulatory science one at a time. By addressing issues of resources, sustainable funding, recruitment and retention of talent, incentives for the scientific workforce, and building and utilization of IT, the foundation for a regulatory science infrastructure can be laid. Consider existing efforts to bolster regulatory science. Centers of excellence models, public–private partnerships, and federal col- 2 The Commissioner’s Fellowship Program offers an opportunity for health professionals and scientists to receive training and experience at the FDA for a two-year period. More information is available at http://www.fda.gov/AboutFDA/WorkingatFDA/FellowshipInternshipGraduateFacultyPrograms/CommissionersFellowshipProgram/default.htm (accessed August 11, 2010).
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Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary laborative initiatives offer numerous lessons for developing a standard mechanism for regulatory science. Several successful models of collaborative networks already exist, such as the Tufts Center for the Study of Drug Development, DC-based UCSF Center for Drug Development Science, European Center for Pharmaceutical Medicines, European Innovative Medicines Initiative, and further analysis of these initiatives can be useful in developing an implementation plan for building regulatory science within FDA. Leverage informatics and existing network capabilities. Various industry sectors have devoted considerable resources to development of surveillance, which could be leveraged through cross-sector and cross-disciplinary partnerships. Educate the public. Public education is an important component to raise awareness and understanding about the complexity of clinical research and the drug development process, and about the need for a strong regulatory science to support drug development. There is a general lack of understanding, by not only the public but also by academia, about the roles of NIH and FDA, respectively, in supporting regulatory science for drug development. Patient advocacy voices are a key element in providing models and input for energizing the public about the need for enhanced regulatory science. Actively engage public policy community. The public policy community is a strong force for enacting reform and should be considered a partner in the efforts to boost regulatory science. As demonstrated by improvements in HIV/AIDS therapies and cancer care, an effort as substantial as the establishment of a regulatory science infrastructure cannot succeed without the understanding and support of the public policy community. FDA and other stakeholders will need to involve and communicate effectively with patient advocates, policy makers, and the public in its endeavors to move forward with this effort. Secure sustainable support for regulatory science. Although FDA’s FY 2011 proposed budget includes an allotment for regulatory science activities, establishing a regulatory science infrastructure will not happen overnight and will require steady funding, workforce, and leadership for realization. Many workshop participants acknowledged that simultaneously developing in-house expertise, taking advantage of available funding mechanisms, and coordinating with external experts will be a massive undertaking. Building a regulatory science infrastructure will require tremendous commitment, resources, and an agreed-upon blueprint to be successful.