Strengthening a Workforce for Innovative Regulatory Science in Therapeutics Development Workshop Summary (2012) / Chapter Skim
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2 The Importance of Innovative Regulatory Science
2 The Importance of Innovative Regulatory Science
Pages 5-18
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From page 5... ...
In the opening session of the workshop, representatives of the four key "locuses" of the development and practice of innovative regulatory sci ence for therapeutics development (FDA, NIH, the pharmaceutical industry, and academia) described these perspectives.
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Andrew Dahlem, Vice President and Chief Operating Officer, Lilly Research Laboratories, Eli Lilly & Co., summarized some of the challenges and needs facing industry in a radically changed drug development environment. Ralph Snyderman, Chancellor Emeritus, Duke University, discussed therapeutics development in the broader context of personalized medicine.
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From page 7... ...
Small business plays an important role in the product development ecosystem, but small businesses today are often undercapitalized and are struggling with the complexities of the therapeutic product development cycle. FDA has been engaged in discussions with companies along the size spectrum -- from small biotechnology to large pharmaceuticals -- to provide advice on how to move ideas forward suc cessfully.
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From page 8... ...
SOURCE: Seyfert-Margolis, 2011. Presentation at IOM workshop on Strengthen ing a Workforce for Innovative Regulatory Science in Therapeutics Development.
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From page 9... ...
For the first time in its his tory, FDA has a budgetary line item for regulatory science and has issued direct funding solicitations for projects in reproductive toxicology and biomarker research and qualification. The FDA Strategic Plan for Regulatory Science FDA has issued a strategic plan for regulatory science that was crafted with input from all of its centers yet takes an agency-wide, rather than center-specific, perspective (FDA, 2011)
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From page 10... ...
Stimulate innovation in clinical evaluation and personalized medicine. FDA seeks to develop and refine clinical trial designs, end points, and analysis methods; leverage existing and future clinical trial data; identify and qualify biomarkers and study end points; increase the accuracy and consistency, and reduce interplatform variability, of analytical methods to measure biomarkers; and develop a "virtual physiologic patient." Support new approaches to improve product manufacturing and quality.
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From page 11... ...
She commented that there is a need for regula tory science in thinking about how to understand, regulate, and enable clinical trials using human embryonic stem cells. She noted that FDA has embraced this innovative technology and enabled it to move forward, with three clinical trials currently being conducted in the United States.
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From page 12... ...
In addition, the leadership council created cooperative research grants to advance translational regulatory science, and four projects totaling approximately $9 million over 3 years have been funded under this initiative: • Creating an organ on a chip that functions as a heartlung micro machine model to test the safety and efficacy of drugs • Designing innovative, adaptive clinical trials for evaluating drugs and devices used in the emergency care of patients with acute neurological illness or injury • Developing an understanding of how nanoparticles interact with the complement system and developing a model that can predict which nanoparticles might activate the complement cascade • Developing a novel strategy to predict ocular irritancy NCATS and NIH Training Initiatives The NIH-proposed National Center for Advancing Translational Sciences (NCATS) , if appropriated and stood up, would assume responsibility for the agency's regulatory science initiatives, in a shift from the distributed function currently residing in NINDS and the National Institute of Diabetes and Digestive and Kidney Diseases.5 The vision and mission of NCATS have evolved over the year that the proposed new center 5 NCATS was formally established by Congress in the FY 2012 Omnibus Appropriations Bill, signed into law on December 23, 2011 (after the date of the workshop)
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From page 13... ...
Landis noted that NIH currently carries out a training function through the institutes and centers. The broad training environment cre ated by NIH-funded programs operated by the institutes and centers represents a real opportunity for producing scientists and physicianscientists who can think about regulatory science, she said.
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From page 14... ...
First, industry needs timely and predictable reviews of NDAs, which could be achieved through enhanced collaboration among industry, academia, and FDA. Second, Dahlem called for unbiased third-party assessments of benefits and risks, especially when drug candidates move from the preclinical setting to human clinical trials.
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For those diseases, intervention typically occurs late, when the underlying pathology makes reversibility difficult and therapy expensive. Today, new technologies are seeking to enable earlier interventions, before primary symptoms develop.
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Moving forward, he said, these activities will include regulatory science and the development of new tools for measuring the safety and efficacy of not only drugs but also the accompanying diagnostics that are being developed. Snyderman analogized the development of a discipline of regula tory science to efforts to advance translational research and the conduct of clinical trials within academic medical centers.
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Sigal remarked that a greater sense of urgency is needed to reinforce that regulatory science is crucial to delivering therapies for patients. She remarked that the agency has not had sufficient resources to accomplish the science needs and added that the resources that are needed to advance regulatory science include not just a budget line item but also a sup portive scientific ecosystem to continue the collaborative advancements, notwithstanding a heavily resource-constrained environment.
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From page 18... ...
This exercise would include identifica tion of specific regulatory science competencies and definition of collaborative mechanisms established to connect academic training programs to relevant programs at NIH and FDA.
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