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Cures 2.0: Discussion draft signals impact on FDA, creation of ARPA-H

More than four years after the 21st Century Cures Act was signed into law, Reps. Diana DeGette (D-CO) and Fred Upton (R-MI) on Tuesday circulated a discussion draft of their proposed Cures 2.0 legislation, setting the stage for negotiations on the long-awaited legislative package.
In addition to proposing new programs and enhancements for the US Food and Drug Administration (FDA) and the Centers for Medicare and Medicaid Services (CMS), the 127-page draft bill would create the $6.5 billion Advanced Research Projects Agency for Health (ARPA-H) the Biden administration called for in its 2022 budget proposal. A concept paper outlining the White House’s vision for ARPA-H was published in Science alongside the release of the discussion draft.
The proposed bill has also expanded on the original Cures Act’s aim of boosting research and development and streamlining regulatory processes to having a greater focus on delivering new treatments to patients and providing education and support to patients and their caregivers. (RELATED: Upton and DeGette begin work on Cures Act repriseRegulatory Focus 25 November 2019)
“The federal government has amazing resources at its disposal and now is the time to put the full weight of those resources to use to cure some of the world’s most devastating diseases, such as cancer, diabetes, Alzheimer’s and more. Developing and delivering new lifesaving cures is a mission that must unite all of us,” said DeGette and Upton, who introduced the final version of the 2016 bill.
The discussion draft contains more than 10 provisions that would involve or directly impact the FDA, including participation on a committee focused on antimicrobial innovation; providing Congress with an updated report and action plan on diversity in clinical trials; and calling on the agency to submit reports or issue guidance on other topics such as digital health, real world evidence, and cell and gene therapies.
Other FDA-related provisions include the creation of grants for novel clinical trial designs and other drug development innovations and improving communications between FDA and CMS on “transformative new therapies.”
The proposed bill would also modify FDA’s breakthrough therapy and regenerative medicine advanced therapy (RMAT) programs to allow sponsors to request designation before or after a new drug application (NDA) or biologics license application (BLA) is submitted. Additionally, the draft bill calls for FDA to issue guidance clarifying how, and by when, sponsors must submit chemistry, manufacturing, and controls (CMC) information for products subject to one of FDA’s expedited pathways.
As written, the bill would amend postapproval study requirements for products granted accelerated approval to allow sponsors to submit other types of data to verify and describe a product’s benefit in addition to postapproval studies, including “clinical evidence, patient registries, or other sources of real world evidence.”
“Like the original Cures Act, Cures 2.0 reflects a determination to address challenges and advance bold ideas which might otherwise become missed opportunities. We are pleased to see DeGette and Upton highlighting the importance of many pressing issues: achieving clinical trials that reflect the diversity of the patient population; leveraging real-world data to create closer alignment between medical progress and patient need; soliciting patient and research ecosystem input on how best to achieve the Biden Administration’s vision for ARPA-H; and advancing solutions for individuals confronting ‘long COVID,’” said Research!America CEO Mary Woolley.