When the 21st Century Cures Act became law in 2016, the legislation boldly funded the FDA’s real-world evidence (RWE) program in an effort to promote innovation in life sciences. This investment has proven prescient given new challenges ushered in by COVID-19, when we confront the reality of fewer randomized controlled trials (RCTs) amid the increased urgency to understand safety and effectiveness of available treatments.
As the legislation’s sponsors, Reps. Diana DeGette (D-Colo.) and Fred Upton (R-Mich.), consider an update to Cures, they can broaden the use of RWE and help deliver treatments to patients efficiently and at a lower cost, even in today’s landscape.
First, lawmakers should invest in RWE capabilities not just at the FDA but across the federal health care landscape to provide generalizable evidence on clinical care. Due to factors ranging from population selection to imperfect adherence, RWE can represent broader populations and treatment experiences than those included in RCTs.
Second, as the FDA and the CMS adopt RWE in their processes, Congress should create an interagency working group to explore how to consistently turn real-world data (RWD) into regulatory-grade RWE, and how to use it to inform coverage decisions. Congress has an important role to encourage a shared learning process around RWE adoption.
Third, lawmakers should require RWE studies on clinical effectiveness for each Medicare Part D protected class to determine access and formulary design. Since 2006, Medicare Part D has required sponsors to include drugs in six specific categories on their formularies, with few exceptions. However, people over 65 or with multiple comorbidities are not often represented in RCTs. Therefore, CMS would benefit from using RWE to increase transparency around the real-world clinical effectiveness of these interventions and to continually refine recommendations.
Fourth, Cures should invest in FDA’s continual refinement of the Sentinel System, which monitors the safety of regulated medical products using pre-existing electronic health care data from multiple sources. By adding new data types and partners, strengthening targeted linkages, and broadening access to de-identified electronic health record data, Congress can enhance Sentinel’s safety analysis capabilities, accelerate access to a broader RWD set, broaden the Sentinel user base to expand its reach nationally and advance regulatory science.
Fifth, Congress should make regulatory review documents publicly available. The lessons learned from RWE used in submission packages are valuable for regulators, biopharma and others as they explore where RWE can best support regulatory decisions. If researchers can access data points like the therapeutic area involved, average review time and speed of approval for RWE submissions, we could better understand the role of RWE in regulatory decision-making.
Finally, Cures should mandate the use of RWE software platforms for regulatory submissions. When conducting RWD analyses, it is vital for researchers to ensure reliability, transparency and reproducibility of their studies and results. The most effective way to do so is through a scientifically validated software platform, which can connect one or more RWD sources, allow researchers to define and document all steps of an analysis and grant audit permissions to reviewers to check their work. This will cut down on review time for regulators and improve their output.
Sustaining the innovative momentum of the 21st Century Cures Act is a job for everyone in the industry: legislators, regulators, payers, providers, technologists, manufacturers, data providers and patients. Updating Cures to include these key priorities will help us lay the common foundation and wider doorway to build our shared knowledge, empowering better, faster decision-making on the treatment questions that matter most.
Carolyn Magill is the CEO of Aetion, the digital health company that turns real-world data into the regulatory-grade evidence needed to inform health care’s most critical decisions: which treatments work best, for whom, when, and how much we should pay for them.
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