Innovation for Healthier Americans Anh Nguyen, MD, MBA Health Policy Fellow US Senate Committee on Health, Education, Labor, & Pensions

An Evolution of Ideas

21st Century Cures US House of Representatives' Energy and Commerce Committee's Subcommittee on Health released a new bipartisan draft bill in April 2015 Designed to dramatically alter the ways pharmaceutical and medical device products are regulated in the US

21st Century Cures Discovery, Development, Delivery Putting Patients First By Incorporating Their Perspectives Into The Regulatory Process And Addressing Unmet Needs Building The Foundation For 21st Century Medicine, Including Helping Young Scientists Modernizing Clinical Trials Accelerating The Discovery, Development, And Delivery Cycle And Continuing 21st Century Innovation At NIH, FDA, CDC, And CMS Modernizing Medical Product Regulation

21st Century Cures -Improving Access to Innovative Products-

Innovation for Healthier Americans Released January 29, 2015 It Takes Too Long and Costs Too Much to Develop Medical Products for US Patients From Bench to Bedside: The Role of Basic Science in New Medical Products Opportunities to Improve Clinical Trials What Does the “Gold Standard” Look Like in the 21st Century and Beyond? Regulatory Science: Why FDA Must Be Prepared to Review Medical Products in the 21st Century and Beyond Medical Innovation and American Competitiveness

It Takes Too Long and Costs Too Much to Develop Medical Products for US Patients Demand exists to accelerate and improve the discovery and development processes Many patients with no treatment options await new therapeutic options Difference in knowledge at FDA and the fast pace of biomedical innovation may stifle innovation in American medicine

From Bench to Bedside: The Role of Basic Science in New Medical Products NIH plays a vital role in its support of basic research Systemic inefficiencies exists, that do not allow us to fully leverage the value of US Research Create an environment that increases scientific discovery and medical product development

Opportunities to Improve Clinical Trials The issues facing clinical trials— spiraling costs, high failure rates, administrative inefficiencies, the rise of precision medicine, and regulatory hurdles—are not new Some of this inefficiency stems from unpredictable and inconsistent development requirement standards of the FDA review process Flexibility in the design of clinical trials and data sharing has the potential to accelerate medical product development and reduce costs

What Does the “Gold Standard” Look Like in the 21st Century and Beyond? The FDA must keep up with the fast pace of science as it fulfills its mission to ensure efficient access to needed safe & effective medical products GAO found that FDA has not fully implemented practices for effective strategic planning and management to deal with the new wave of innovation Does Congress need to re-examine the FDA’s current infrastructure and processes?

Regulatory Science: Why FDA Must Be Prepared to Review Medical Products in the 21st Century and Beyond After 10 years of funding many projects to improve medical product development are these projects translating into products reaching patients more quickly?

Medical Innovation and American Competitiveness The US remains the global leader in innovative drugs and devices However, it faces increasing global competition US requires a comprehensive response from legislators and regulators to ensure superiority in medical product development Build a regulatory system that is efficient, predictable, streamlined, and aligned to the needs of a globalized medical product industry

Progress Thus Far… Environment Process Goals

Thank You

Incentives for Data Sharing

Incentives for Serious Diseases and Unmet Needs