Advancing Regulatory Science for Medical Countermeasure Development Workshop Summary (2011) / Chapter Skim
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1 Introduction
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plays a central and crucial role in domestic preparedness through its regulation of drugs, biologics, medical devices, and radiation-emitting products. However, recent reports have highlighted the need for improved regula tory science to help address the novel regulatory issues the agency faces in its review and approval of many MCMs (e.g., ethical or practicality barriers to conducting standard clinical trials, balancing benefit and risk for products that would be used only under dire circumstances)
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Speakers were asked to introduce their comments by providing a brief overview of scientific advances or emerging technologies holding promise to facilitate development of MCMs, and then to focus on what regulatory science advances are needed to address gaps in currently available tools to predict and evaluate product safety, efficacy, and quality. They were also asked to identify how innovative regulatory science methodologies can address emerging tech 2 As part of the PHEMCE review, and at the request of the Secretary of HHS and the HHS Assistant Secretary for Preparedness and Response, the IOM's Forums on Drug Discovery, Development, and Translation and Medical and Public Health Preparedness for Catastrophic Events collaborated to host a workshop in February 2010, which addressed challenges facing the PHEMCE.
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This workshop summary identifies key issues and raises awareness of opportunities and challenges in advancing regulatory science underpinning regulatory decision making about MCM products. After first providing context and background, the summary presents enterprise stake holder perspectives (federal and private sector)
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With regard to MCMs, enhanced regulatory science is needed to reduce uncertainty and provide clear regulatory pathways for MCM development. Exercising the principles of regulatory science requires underlying capacity and expertise at FDA, Goodman added.
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. Goodman also emphasized the importance of engaging the agency early in the MCM development process, so FDA regulatory science can be brought to bear in a highly interactive manner with the product spon sor.
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. Based on the identified barriers and challenges, the review highlighted a variety of opportunities for improvement, the first of which BOX 1-2 Highlights of Keynote Address CHALLENGES TO MCM DEVELOPMENT • Increasing costs of product development • Low success rates • Uncertainties throughout the process • Uncertain market for MCM products • Highly variable time frames for product development, production, and mo bilization in response to an emergency • Limited application, thus far, of systems biology, new technologies, and high-level computational science approaches to product development (ap proaches that are routinely applied at the basic science level)
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"You get the system that you reward," Korch said, so moving forward, the MCM enterprise needs to reward flexibility, fresh thinking, and a vision of how to prepare against low-probability, high-consequence events. OVERVIEW OF THE FDA MCM INITIATIVE The mission of the FDA MCM initiative is to promote the development and availability of MCMs by establishing clear regulatory path ways based on the best available science, explained Luciana Borio, acting director of the Office of Counterterrorism and Emerging Threats at FDA.
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presented their views on the current state of MCM regulatory science and the needs and opportunities for development of MCM regulatory science. Second, a series of panel discussions, anchored by presentations, was held that reviewed novel scientific methodologies in MCM development and identified highpriority MCM regulatory science needs and opportunities.
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Due to their crosscut ting nature, resonating themes and "big ideas" are listed below as a means of orienting the subsequent workshop summary, which summarizes the discussions underlying and supporting these themes. The themes and "big ideas" listed below are not inclusive of the many individual suggestions that were made throughout the workshop and are summarized elsewhere in this workshop summary.
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Work toward approval of an MCM product based on safety and ■ efficacy data from an animal model but with information on clinical dosing. • App" technology for public education and communication, sur " veillance, response, and adverse event monitoring holds promise.
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Gerald Parker, deputy assistant secretary to the secretary of defense for chemical and biological defense at the DoD, emphasized that "perhaps complacency may be our biggest threat." He noted that it may be more useful to acknowledge that the issues are "high probability, high consequence" ones, and that there is a need to appreciate the sense of urgency associated with this threat to create the important social drivers needed to effect change. Sidebar: Regulatory Framework for Review and Approval of MCMs Although the focus of the workshop was regulatory science, there was also significant discussion of the closely related topic of the regulatory framework for re view and approval of MCMs (which is being addressed in Pillar 3 of the FDA MCM initiative)
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Note: Some participants referred to such a mechanism of provisional approval prior to an emergency as a "pre-EUA." However, Michael Kurilla, director of the Of fice of BioDefense Research Affairs and associate director of Biodefense Product Development at NIAID, explained that the term pre-EUA is used by FDA to refer to a submission from a sponsor regarding potential future emergency use of a product, for the purposes of allowing FDA to begin to formulate draft EUA documentation, so that these drafts could be finalized more quickly should an emergency be declared. He noted that there has been variability as to when in the development of a product it could be considered eligible for data to be submitted for pre-EUA status, and what the pre-EUA package should look like is vague and ill defined.
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establishes a regulatory process for FDA approval of certain new drugs and biologics used to reduce or prevent the toxicity of CBRN substances based on animal data, when adequate and well-controlled efficacy studies in humans cannot be ethically conducted and field trials are not feasible. See Guidance for Industry, Animal Models -- Essential Elements to Address Efficacy Under the Animal Rule (Draft Guidance)
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