From an article by Joann Donnellan – Today there are 10,000 known diseases in the United States, but only 500 have established treatments. Life-threatening diseases such as Alzheimer’s, cancer, heart disease, and many others lack effective treatments or cures. The urgent need to find cures is impeded by the process of bringing promising drugs and devices to market. A new report by the Bipartisan Policy Center (BPC) recommends a series of policy actions that Congress can take to help reduce the time and cost of developing and delivering safe and effective medical products to patients. Such actions could considerably increase the competitiveness of U.S. companies in the global marketplace.
The report, Advancing Medical Innovation for a Healthier America, was developed over the past six months by BPC with guidance from co-chairs former Senate Majority Leader Bill Frist, M.D. and former Congressman Bart Gordon, along with an expert advisory committee. Its recommendations focus on improving the medical product development process to more rapidly and cost-effectively bring safe and effective cures to patients, increasing regulatory clarity, strengthening the FDA’s ability to carry out its mission, and enhancing investment in medical products to address unmet public health needs.
“It’s time to take action to significantly advance medical innovation in the United States,” said former Senate Majority Leader Bill Frist, M.D. “Americans cannot afford to rely on 20th century methodologies for treatments when the world is on the cutting edge of new medical technologies. Federal agencies must be equipped to keep the U.S. at the forefront of medical innovation.”
The report highlights that, in the U.S., expensive development, lengthy approval processes, and the lack of regulatory clarity slows U.S. patient access to new medical technologies. Currently, it takes an estimated $2 billion dollars and up to a decade to bring a new drug to the marketplace. The challenges of medical product development hamper the competitiveness of U.S. companies globally.
From 2007 to 2012, private-sector investment in medical innovation in the U.S. declined by nearly 13 billion dollars, while investments increased around the world. The U.S. share of the world’s total R&D investment declined from 50 percent in 2007 to 44 percent in 2012. “This decline in investment is significant,” said former Congressman Bart Gordon. “Improvements in the medical product development process, greater regulatory clarity, and increased harmonization of international standards need to be top priorities so we can reverse this trend and improve our nation’s global competitiveness.”
BPC’s recommendations to Congress include:
- Improving the Medical Product Development Process
- Accelerate the generation and use of more relevant evidence—including data from both clinical and patient experience–to support post-approval study requirements, approval of new indications for existing medical products, and ultimately improved clinical trials for regulatory review.
- Assure greater harmonization of international standards and requirements.
- Improve the interoperability of health information technology (IT).
- Clarify and allow increased sharing of scientific information regarding off-label use of approved medical products.
- Improve and expand the qualification and use of new drug development tools, including biomarkers and patient-reported outcomes, and assure that patient perspectives are included in the benefit-risk assessment associated with regulatory decision-making.
- Increasing regulatory clarity
- Assess the rapidly evolving field of precision medicine and develop a regulatory framework that promotes innovation, protects patient safety, and is flexible enough to accommodate rapid changes in science.
- Improve regulatory clarity associated with combination products.
- Clarify that health IT should not be regulated as a medical device. Recognize independent bodies that can develop and assure compliance with consensus standards and facilitate patient safety reporting to continually improve the development, implementation and use of health IT.
- Clarify the regulatory authority associated with and require a regulatory framework for laboratory-developed tests.
- Improve the regulatory framework for regenerative medicine.
- Strengthening the FDA’s ability to carry out its mission
- Modernize human resources-related systems and processes to improve FDA’s scientific expertise and capacity, including significantly improving recruiting, hiring, and retention practices; more effectively addressing conflict of interest concerns; and enabling staff to attend scientific conferences and meetings.
- Expand expertise and capacity through greater and more effective use of public-private partnerships in areas such as creating a framework and virtual infrastructure for developing better, more relevant evidence; developing and qualifying drug development tools; and developing guidance generally.
- Improving patient access to medical products for unmet and public health needs
- Accelerate the development of antibiotics for use in limited populations.
- Improve the processes associated with early patient access to medical products.
- Increase incentives to develop medical products for unmet medical needs.
“The policy recommendations in this report will accelerate the delivery of much-needed, safe and effective cures to patients,” said BPC Director of Health Innovation Janet Marchibroda. “They will also provide FDA with the tools and support they need to succeed in this rapidly evolving field, and ultimately will improve the global competitiveness of American companies.”
BPC recognizes that the recommendations could require additional resources not available within the FDA’s current budget. If additional funding is required, BPC recommends FDA first review and prioritize existing programs and reallocate funds as appropriate. If further funding is required, BPC suggests additional resources be provided through reductions in federal spending programs, increased user fees, new revenues, or a combination of all three.