New bipartisan bill aims to speed up clinical trials for rare disease treatments

One week after the Food and Drug Administration unveiled a new initiative for speeding up the approval of rare diseases treatments, new legislation is being introduced to reimagine the way that drugs are studied.

The bipartisan bill, which will be introduced today by Democratic Rep. Jake Auchincloss and Republican Rep. Morgan Griffith, would make it easier for more patients to participate in clinical trials and reduce the time it takes to get them started. The so-called "Faster Clinical Trials Act" would also encourage drugmakers to use computer simulations in early-stage drug development and address some of the common reasons why the FDA suspends clinical trials to avoid delays.

Typically, clinical trials assemble various people at specific locations. When the trial ends, the infrastructure is dismantled and everyone goes their separate ways, even though many things are the same from trial to trial for a particular disease or type of treatment. The legislation aims to create a network of reusable clinical trial sites.

"So you're not recreating the wheel every time you're trying to do a new drug," Auchincloss said in an interview with CNBC Cures. "You have a platform, and now you're just trying one thing after another."

Griffith said the bill would benefit patients beyond those with rare diseases, including residents of his southwest Virginia district who want to participate in studies of new treatments for black lung disease. Some of his constituents live a two-hour drive or longer from medical centers that typically host clinical trials.

"We're trying to bring the clinical trial to the patient instead of the model today of bringing the patient to the clinical trial," he said in an interview.

The legislation comes just days after the FDA announced its own plan to accelerate new treatments for rare diseases. Under the new Rare Disease Framework Initiative, the agency said it will be more flexible in evaluating therapies for small patient populations.

The FDA's plan will give drugmakers "very clear rules of the road that are going to last a long time," former FDA Commissioner Scott Gottlieb said in an interview on CNBC's Squawk Box on Oct. 19.

The new legislation creates a "Center for Clinical Trial Innovation" at the FDA to coordinate networks of clinical trial sites. It also lays out specific instructions to the FDA's Rare Disease Innovation Hub, like giving it the authority to consult with drugmakers about their concerns and provide help to the agency's review teams.

The bill creates a scenario where patients may get an experimental drug in their regular doctor's office instead of traveling to a clinical trial site. Trial researchers could track their progress through Zoom calls, electronic medical records or wearable technology.

"The idea is to make it as easy as possible for the patient to stay right where they are," said Mark McClellan, founding director of the Duke-Margolis Institute for Health Policy at Duke University, which helped craft the legislation. "To enroll in the trial, take the drug and be monitored and get the follow-up data without having to travel."

The legislation directs as much as $75 million in annual funding to the FDA over the next five years. The clinical trial networks would prioritize rare and chronic diseases. The goal is to cut the cost of trials and the time it takes to enroll participants by at least 30%. Auchincloss compares it to the pioneering clinical trial model that the Cystic Fibrosis Foundation built in the 1990s to study the disease.

The idea of "point-of-care" clinical trials has been around for more than a decade. The Department of Veterans Affairs ran the first one in 2010 by comparing two treatment regimens for patients with diabetes. Today it's used widely in Australia. But it hasn't gained much traction in the United States, where the system for studying drugs was designed to take place at specific clinical trial sites. New technology now makes it possible to bring drug studies to a patient's doorstep.

The idea could benefit rare disease patients. By expanding point-of-care trials, it could be easier to find patients—a common problem for rare diseases that may only affect a few dozen people. Also, many families of rare disease patients spend years creating databases, known as natural history studies, so they can be used as control groups in trials instead of giving patients a placebo, which is often impractical and unethical. Under the bill, those databases could be created with electronic health records.

There are limits to the approach. Intensive treatments, like gene therapies, will still need to be given at academic medical centers, McClellan said. But new scientific advances – like in vivo gene editing – allow patients to leave the hospital the same day. If they're in a clinical trial, they could go home and be monitored remotely, he said.

Griffith said the bill likely won't move forward for a vote until next year. Auchincloss's office said that they are working with a bipartisan group of senators to introduce a similar measure in the Senate later this year.

Auchincloss said he expects the house measure will pass because it appeals to a wide range of people — from drugmakers and patient groups that want faster and cheaper clinical trials, to followers of the MAHA movement who want greater access to experimental treatments.

"There's something in here for everyone to like," Auchincloss said.