Washington, D.C. — Today, the amount of electronic health care data is rapidly growing due to the widespread adoption of electronic health records and the popularity of digital health tools, such as wearable devices, biosensors, apps, and remote patient monitoring. A new report released by the Bipartisan Policy Center offers recommendations for leveraging this data, while protecting patient privacy, to improve the Food and Drug Administration (FDA) regulatory decision-making, as well as value-based payment for drugs and biologics.
The report, developed under the guidance of former FDA commissioners Dr. Robert Califf, Dr. Andrew von Eschenbach, and Dr. Mark McClellan, and former Senate Majority Leader Dr. Bill Frist, explains how these new data sources can help bring safe and effective therapies more quickly to patients by improving the evidence base for evaluating medical products and value-based payment arrangements. New medical products, including small molecule drugs and biologics, such as cell and gene therapies, are showing great promise for patients with cancer, neurodegenerative diseases, and other diseases with no current cure.
“The use of real-world data and evidence is expanding to inform the discovery, development, and delivery of new therapies for patients, but there are many steps we can take to accelerate progress” said McClellan, founding director of the Duke-Margolis Center for Health Policy. “Real-world data can provide the basis for badly needed additional evidence about the risks and benefits of many therapies, and which ones are best for particular patients.”
“We are living in an age of remarkable scientific progress,” said von Eschenbach, president of Samaritan Health Initiatives. “It is imperative that we support this progress by continually examining and improving the way we evaluate and bring new, effective therapies to patients as quickly as is safely possible. We believe our recommendations are important steps toward reaching that goal.”
The report points out that while considerable progress has been made in using real-world data, there is more to do. In response to the 21st Century Cures Act, the FDA has been working to advance its real-world evidence program to support both pre-market evaluation and post-market approval study requirements. At the same time, value-based payment arrangements have been a theme across many policy proposals to improve the affordability of drugs and biologics, and participation in these arrangements by payers and manufacturers is on the rise.
While regulatory and payment decision-making would greatly benefit from electronic data in hospitals and health systems, physician practices and clinics, laboratories, pharmacies, radiology centers, health plans, registries, and with patients themselves, challenges remain.
“Bringing new data sources to bear, while assuring patient privacy, can help us tackle the accessibility and affordability of drugs in an era of rising health care costs, declining life expectancies, and concerns about disparities in our health care system,” said Califf, professor of medicine and cardiology at Duke School of Medicine.
BPC’s recommendations include:
- Congress should assure adequate funding of real-world evidence activities by funding the FDA’s FY 2020 $60 million budget request for a new medical data enterprise.
- FDA should develop and publish timely guidance on real-world evidence.
- The Department of Health and Human Services (HHS) should improve access to data to support real-world evidence needs by adopting electronic health information export and application programming interface (API) provisions contained in recently released proposed rules, collaborating to develop HL7 FHIR® implementation guides, and making both Medicare data and mortality information more readily available.
- ONC should improve the reliability and relevance of data by adopting the U.S. Core Data for Interoperability (USCDI) and requiring real-world testing of interoperability as a condition of certification.
- FDA should develop guidance to accelerate the use of digital tools in the collection of data from patients to support the entire medical product development life cycle.
- FDA should expand its efforts to explore the utility of artificial intelligence to support real-world evidence needs.
- FDA and the Centers for Medicare and Medicaid Services (CMS) should expand and accelerate pilot and demonstration projects to evaluate the use of real-world evidence generated from new data sources for the evaluation of both drugs and biologics—including cell therapies—for both regulatory approval and payment.
- Congress should advance a federal data privacy framework that establishes baseline protections and addresses entities handling health information that are not covered under HIPAA to help assure privacy and confidentiality. Privacy policies should also take into account the real-world evidence needs of regulatory evaluation and value-based payment arrangements.
- FDA should continue to advance innovative, new models of drug development, including adaptive pathways that leverage real-world data. The FDA should also continue with its plans to create a new office of drug evaluation science.
- HHS should create safe harbors to provide regulatory certainty regarding value-based payment arrangements and expand upon existing safe harbors to enable donation or cost-sharing of software supporting real-world evidence needs.
- Congress and HHS should expand and support the CMS workforce to support evaluation of new emerging therapies, as well as the implementation of value-based payment arrangements for drugs and biologics.
- CMS and the FDA should increase collaboration on the generation and use of real-world evidence to support both value-based payment and regulatory evaluation needs. Public and private sector stakeholders should also work together to advance collaborative models of evidence development that bridge current silos.
“Congress and HHS have laid the groundwork for significant progress on real-world evidence,” said Janet Marchibroda, BPC fellow. “By improving regulatory clarity, increasing funding, launching pilot projects, improving access to and standardizing data, and building capacity and forging stronger partnerships between CMS and the FDA, the U.S. can significantly improve the way that it evaluates and pays for drugs and biologics and supports access for patients.”