The Bipartisan Policy Center is encouraged by recent efforts in the House and Senate to advance medical innovation legislation that is expected to deliver new, safe, and effective treatments and cures to patients. Today there are 10,000 known diseases in the U.S., but only 500 have established treatments. The urgent need to find cures for life-threatening diseases such as Alzheimer’s, cancer, Parkinson’s, and many other diseases calls for policies that modernize the discovery, development, and delivery of drugs and devices. This year BPC launched a new initiative, FDA: Advancing Medical Innovation, led by former Senate Majority Leader Bill Frist, M.D. and former Representative Bart Gordon, to address this challenge and submitted recommendations to Congress. As the Senate Health, Education, Labor, and Pensions (HELP) Committee works to finalize and release draft legislation in the coming weeks, BPC recommends three priority areas that we believe are ready for bipartisan consensus in the near term:
1. Improving scientific expertise and capacity at the U.S. Food and Drug Administration (FDA)
In order to accelerate the development of new, safe, and effective treatments and cures, FDA must have adequate scientific expertise, staffing, and other resources (including effective use of public-private partnerships) to successfully carry out provisions of medical innovation legislation. BPC recommends several reforms to improve FDA scientific expertise and capacity, many of which could be implemented without new funds and could improve the agency’s ability to carry out its mission.
2. Enhancing health IT interoperability and increasing regulatory clarity
The U.S. has invested more than $30 billion on electronic health record incentive programs, and yet the majority of systems are not able to routinely exchange electronic patient information. Interoperability and electronic information sharing play a critical role in improving the cost, quality, and patient experience of care. Also, there is regulatory uncertainty regarding federal agency authority associated with the oversight of health information technology (IT). BPC recommends adopting a common set of standards to improve interoperability, creating incentives to promote their widespread adoption, and recognizing and requiring independent validation to ensure interoperability. BPC also recommends clarifying regulatory authority related to health IT.
3. Expanding the use of data and development tools to improve medical product development and FDA approval processes, including:
- Using real world evidence drawn from claims, clinical, and patient-generated data to improve the development and delivery of drugs and devices. To lay the groundwork for use of these new data sources, FDA should develop and implement a framework for evaluating these sources as well as methods for generation and use of real world evidence for randomized, pragmatic trials and post-market activities.
- Expanding the qualification and use of drug development tools, including biomarkers and patient-reported outcomes, to develop safer and more effective medical products, and increase the efficiency and effectiveness of the drug development process.
- Incorporating patient perspectives into benefit-risk assessment by clarifying the process and timelines for submission of patient preference data and methodologies for collection and assessment of data for benefit-risk.
We urge Congress to sustain the momentum and bipartisan will to enact realistic and meaningful medical innovation legislation. Effective policy solutions require balance and compromise. We believe legislation is not only possible but probable, given the thorough groundwork already accomplished and the long history of bipartisan achievements by the Senate HELP and House Energy and Commerce committees. BPC looks forward to continuing to work with policymakers as they develop medical innovation legislation this year, and work toward reforms in 2017 that may become part of reauthorizing prescription drug and medical device user fee legislation.