Rep. Robin Kelly admits the word “diversity” has scared some of her fellow Republicans away from her efforts to improve representation in clinical trials.
“It’s been a little rough across the aisle,” Kelly said at a recent STAT event. “I don’t want to put a broad brush on everyone, but…they feel like the government is sticking their nose into something a little too much.”
The Illinois Democrat has already successfully secured a GOP co-sponsor with her bill, the 2022 NIH Clinical Trial Diversity Act, which would require clinical trial sponsors to write formal diversity plans when they apply for funding from the National Institutes of Health. . If passed, the bill would also hold drug companies accountable for a clear strategy to recruit and retain people who are grossly underrepresented in trials. It would also apply anti-discrimination rules to clinical trials.
The goal, she explained, is to put some of the burden on the pharmaceutical industry to help address issues of transportation, childcare and work responsibilities, as well as distrust of the system. that may prevent underrepresented groups from participating in clinical trials.
“It just can’t be the same old the same way,” she said.
Kelly, who has hinted that she will have a Senate co-sponsor to announce soon, is “hopeful but not counting on the bill passing by December,” she said.
Kelly and several other speakers at STAT’s event made it clear that improving diversity in clinical trials can be particularly challenging in the area of rare diseases. Because a particular rare disease affects so few people, it is difficult to find enough patients who meet the enrollment criteria for a particular trial. And people with a certain rare disease are widely dispersed and live with a variety of symptoms, subtypes and stages of the disease.
With all the limitations surrounding clinical trials and rare disease research, patients are often misdiagnosed or diagnosed late despite the warning signs. Sometimes they’re in critical condition in the ER before a doctor tests for the disease — if the doctor even knows they need to test for a rare condition.
Jay Scott Randell, a dentist and parent of two children with Barth syndrome who also spoke at the event, knows this all too well. Barth is characterized in part by an enlarged heart and muscle weakness. Randell recalls a heartbreaking phone call he received in 2005: Michael, one of his 21-month-old triplets, was in the emergency room with congestive heart failure. Providers were unable to introduce an IV because the infant’s cardiovascular system had collapsed.
Unlike another of his triplet brothers, Michael was not diagnosed with any illness at birth. The doctor told his parents he was “perfectly fine”. Michael was not diagnosed until his parents saw a doctor who had treated another child with Barth syndrome.
In a later session, STAT lead writer Ed Silverman explored what makes ultra-rare diseases like Barth so hard to treat, even when high rare disease drug prices make new drugs so profitable. Another panel with Washington correspondent Sarah Owermohle discussed next steps a week after Food and Drug Administration advisers voted in favor of a new drug to treat another rare disease, ALS.