Reps. Fred Upton, R-WI, and Dianna DeGette, D-CO, are exploring ways to include some of the ideas for their second iteration of the 21st Century Cures Act into legislation currently in the works to tackle the coronavirus epidemic, the pair said on call with reporters this week.

The bipartisan duo, which shepherded the 21st Century Cures Act into law in 2016 against the odds of getting any major FDA reform outside of a user fee reauthorization package, released a “concept paper” this week that outlines their plans for the follow-up legislation, which they first announced work on in November 2019.

The first section of the concept paper includes many provisions that were added in response to the COVID-19 crisis, a DeGette spokesperson told the Pink Sheet. Other provisions are likely ideas the pair would have pushed for anyway, but now may be seen as having extra urgency because of the global health situation.

DeGette said that she and Upton are working with House Energy and Commerce Committee leaders Reps. Frank Pallone, D-NJ, and Greg Walden, R-OR, to put together a memo of Cures 2.0 ideas that could be incorporated into an anticipated fourth COVID-19-related bill currently in the works by Congress.

Some of the Cures 2.0 ideas “can easily be slotted right in,” DeGette said, though she acknowledged that other issues with less of a COVID connection are likely not ready for prime time, such as real-world data measures and payment reforms that are still being crafted. Having the COVID vehicle is likely a good thing for the parts that are ready to go as getting Cures 2.0 done has been seen as an even harder lift than the first bill.

Antibiotic, Manufacturing Sections Likely Targets For COVID Bill

Antibiotic reimbursement is one priority for Upton and DeGette that could hitch a ride on the COVID-19 legislation. The concept paper proposes improving U.S. pandemic preparedness and response through support of antimicrobial resistant commercialization challenges, noting that secondary bacterial infections often result from viral outbreaks and are one of the leading causes of death. Any antibiotics developed would also help patients in non-pandemic conditions, they add.

The lawmakers want to give HHS’s Assistance Secretary for Planning and Response “the resources and regulatory authorities necessary to fix the commercial market for new antibiotics.” This would include providing federal support “for both premarket and post-market costs that can lead to company bankruptcy.”

This wouldn’t be the first time legislators tried to use COVID to address the antibiotic pipeline challenges. The Senate tried to include a provision to boost hospital reimbursement for antibiotics in the third coronavirus relief package in March, but the House axed that attempt.

Besides antibiotics, the Cures concept paper also ties its push for modernized and expanded domestic drug manufacturing to pandemic preparedness, making it a likely target for the upcoming coronavirus legislation. Upton and DeGette propose requiring HHS to implement a national testing, data sharing infrastructure, vaccine, therapeutic and medical supply readiness strategy to mitigate future pandemics that would include a drug manufacturing component, in particular continuous manufacturing.

The lawmakers also want to create a program that could help patients with rare diseases deal with increased health care expenses due to COVID-19 and future pandemics. “Financially vulnerable rare disease patients do not have the ability” to bear additional costs related to changes in treatment locations or disruptions in services caused by a disease outbreak, they said.

Upton and DeGette propose creating a COVID-19 specific fund for rare disease patients and then establishing a federal grant program for organizations that would help support access to and the cost of medical care during future pandemics.

On the press call, DeGette also highlighted the concept paper’s push for clinical trial diversity as policies that could make it into COVID-19 legislation, citing the virus’s disproportionate impact on minorities. She and Upton are calling for Medicaid to cover the routine care costs of clinical trial participation for patients with life-threatening conditions.

“The financial pain caused by the COVID-19 pandemic has made enacting this policy even more urgent,” the concept paper says. They also want HHS to do more promotion of clinical trials and they want HHS to convene a task force to make clinicaltrials.gov more patient friendly.

FDA-Centric Policies May Have To Wait

The FDA-titled section of the concept paper is largely filled with proposals that are less likely to be tacked on to the coronavirus work.

The concept paper is pushing for FDA to coordinate its work on digital health technologies across its centers and requests a guidance on the use of digital endpoints for regulatory review, including the validation and qualification of digital endpoints and biomarkers. They also want the guidance to address the acceptance of decentralized trials and the use of digital health technologies in these trials, and the use of digital health technologies in patient-focused development product. The pair also want FDA to harmonize their digital heath work with foreign regulators.

The lawmakers also propose authorizing funds for FDA to provide grants to groups working on innovative clinical trial design, including work on complex adaptive and Bayesian designs and grants for patient-focused drug development work.

They also ask for HHS to issue a “consistent and clear regulatory framework for the recognition and utilization of real-world evidence” and for FDA to issue guidance on how real world evidence could be used to as part of post-approval commitments for breakthrough therapy-designated drugs and products that are approved via accelerated approval. The guidance could include acceptable endpoints and outcomes measures, data quality standards, and data transparency results. Finally, they want HHS to establish a public-private task force to develop ways to encourage patients to engage with real-world evidence generation.

For breakthrough therapies, the lawmakers want to make a technical correction to the first 21st Century Cures Act to require FDA to provide the benefits of this designation or the regenerative medicine advanced therapy designation, when sponsors have collected scientifically valid preliminary clinical evidence outside of the U.S. even if they don’t have an active investigational new drug application approved by FDA. Currently, FDA will only review designation requests if there is an active IND.

They also propose mandating FDA and CMS automatically commence communication on breakthrough products once the designation is granted to help speed coverage decisions.

More Feedback Wanted On Reimbursement Reform

The lawmakers are still asking for public input on many of their policies directed at the payment system.

Their ultimate aim seems bold: “We are interested in efforts to modernize the entire coverage and care delivery system in the United States,” they write.

They ask some very broad questions like whether the current coverage and reimbursement approaches for new medical products and technologies are adequate to keep up with the pace of innovation and what coverage issues exist for patients transitioning from private insurance to Medicare. They also hone in on potential coverage issues that may exist for cell and gene therapies, medical products for small patient populations, coverage of genomic sequencing and coverage of breakthrough-designated products.