Regulatory Science and Innovation: FDA s Role in Transformation of the MCM Enterprise Jesse L. Goodman, MD, MPH Chief Scientist and Deputy Commissioner for Science and Public Health (Acting), presentation to IOM Review February 24, 2010
Critical Moment Multiple insights from accomplishments and limitations of BioShield, pandemic investments, and 2009 H1N1 experience Galvanized public, policymaker and administration interest Clear public health & national security needs History to build on of bipartisan engagement on MCM and pandemic preparedness History of teamwork, personal relationships and commitment of HHS, CDC, FDA, NIH, DOD, others at all levels to achieve outcomes
Overview Four proposed key principles, going forward: End to end partnering, highly interactive and collaborative engagement - outcomes oriented management Regulatory science: capacity and knowledge More agile platform, multi-use technologies Policies that meet public health needs And what else is needed to succeed? These principles themselves are multi-use & can be applied, resources permitting, broadly to EID and more
What Has Worked- examples? H1N1 response Previous investments/planning, including at FDA Excellent public-private partnerships and interagency collaboration and communication FDA staff rapidly, collaboratively responded in ICS mode, vaccine development/licensure, new manufacturing sites, rapid lot release, EUA s for drugs/dxs, now transitioning to approval paths Many changes multiuse and can be institutionalized (e.g. safety monitoring, delivery, communication), as can approaches/culture MCM Where dedicated leadership and teams with right members, strong science, success has occurred
Interactive Engagement and Effective Management Institutionalize best practices Outcomes oriented partnership with all parties End to end team approach, information sharing Start early, continue throughout development FDA at the table, engagement and communication connect to review divisions, OCET/OCS engaged Define possible uses, pathways, scientific gaps Regulatory requirements to reduce uncertainty Include animal studies, models, assays, manufacturing Early, periodic problem identification throughout Defined plans, resources/responsibilities, and timelines to address all gaps and solve problems Oversight/review of progress at high levels
Regulatory Science Develop, assess and provide tools, methods, models, standards, guidance, pathways to evaluate product safety, efficacy and quality Laboratory, population, clinical, manufacturing and behavioral sciences and scientists/capacity FDA sees what works and what does not across multiple products and stakeholders Unlike work typically performed in academia or by individual sponsors and becomes available to all Key elements: leadership, coordination, training, development, targeted research and collaboration Enhances quality and integrity of decisions Empowers engagement throughout the development & evaluation process to catalyze and enable change, internally and externally
Regulatory Science: Examples Preclinical Modernize safety testing Better markers and models to predict product efficacy Clinical Develop and use biomarkers and surrogate endpoints Develop and use adaptive and other flexible clinical trial designs to improve or speed evaluation Validate new diagnostic and other tests, including POC, to inform deployment and utilization of MCMs Infrastructure and information science to evaluate products rapidly, including in emergencies Manufacturing and Quality Facilitate more rapid scale up of production Improve quality and stability of products Better rapid methods for purity, potency, quality, contamination
Platform & Multi-use Technologies Vaccine (e.g. recombinant, VLP, vectored, DNA), diagnostic (e.g. high throughput NAT, Ag), monoclonal platforms, can be rapidly adaptable to address new pathogens: e.g. blood screening Typically more scalable, faster Need concrete experience with real products to provide enhanced predictability of results and reduce regulatory requirements Will not perform for all pathogens & diseases Unlikely to alone solve incentive issues, but focus should be on products with commercial use or potential, helps provide warm base
Policy for Public Health Needs EUA can be cumbersome, need to better prepare prior to emergencies Animal rule need to re-examine experience, and scientific needs and realities Expand consideration of accelerated approval approaches (requires underlying science of likely surrogates) Should there be other approaches or status short of full approval?
What else is needed? Transparency and trust Effective communication, behavioral science Collaboration and partnerships Resources and incentives
Vision The potential of innovation is realized to prevent and treat disease and reduce suffering globally, mitigate pandemics, prepare for and respond to emerging infectious disease threats and bioterrorism. Nimble, rapid generation of effective countermeasures protects health and stability.
Thanks! Contact: jesse.goodman@fda.hhs.gov 301-796-4880