U.S. lawmakers propose faster clinical trials for rare disease treatments

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Faster rare disease trials

Highlights

The Faster Clinical Trials Act, introduced by Reps. Jake Auchincloss and Morgan Griffith, seeks to accelerate rare disease clinical trials by establishing a reusable trial site network and encouraging computer simulations.
The legislation proposes expanding remote participation through Zoom, electronic medical records, and wearables, aiming to increase access for patients hindered by geographic barriers.
The bill allocates up to $75 million annually for five years to the FDA, with supporters targeting a bipartisan Senate version and a likely vote next year.

FDA-backed trial overhaul takes shape

As first reported by CNBC, Democratic Rep. Jake Auchincloss and Republican Rep. Morgan Griffith are introducing the Faster Clinical Trials Act to make studies faster to launch and easier for patients to join. The bill comes one week after the FDA unveiled a rare disease initiative aimed at speeding approvals and would create a Center for Clinical Trial Innovation at the agency to coordinate reusable clinical trial site networks.

The measure is designed to move away from a system in which trial infrastructure is built for a single study and then dismantled. It also encourages drug developers to use computer simulations in early-stage development and seeks to address common issues that lead the FDA to suspend trials, reducing the risk of delays.

The legislation would also let more patients take part from home or from their doctors' offices rather than traveling to major research centers. Researchers could monitor participants through Zoom calls, electronic medical records and wearable devices, broadening access for patients who live far from traditional trial sites.

Access gains could extend beyond rare diseases

Supporters say the approach could help rare disease patients, who are often difficult to recruit because some conditions affect only small numbers of people. The bill says trial networks should prioritize rare and chronic diseases, while also supporting the use of electronic health records to build natural history databases that may serve as control groups in place of placebo arms when those are impractical or unethical.

Griffith said the model could also help patients in southwest Virginia, including people seeking access to studies for black lung disease, who may live two hours or more from medical centers that host trials. Mark McClellan, founding director of the Duke-Margolis Institute for Health Policy at Duke University, which helped craft the bill, said the goal is to let patients enroll, receive treatment and be monitored with minimal travel.

There are still limits to the model. Intensive treatments such as gene therapies would still need to be administered at academic medical centers, although newer advances may allow patients to return home the same day and be followed remotely during a trial.

The bill directs as much as $75 million a year to the FDA for five years. Griffith said it likely will not come to a vote until next year, while Auchincloss's office said it is working with a bipartisan group of senators on a similar Senate measure later this year.

Our earlier article on AstraZeneca’s AI-driven partnership with insitro explained how the companies plan to use the “Virtual Human” platform to identify targets for MASH and cardiomyopathies and advance them to development-candidate stage. We noted that AstraZeneca would then take those programs into funded clinical trials, highlighting the industry-wide push to use AI to cut guesswork and accelerate early drug discovery.

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