Rep. Robin Kelly admits that the word “diversity” has scared some of her Republican colleagues away from her effort to improve representation in clinical trials.
“It’s been a little tough on the other side of the aisle,” Kelly said at a recent STAT event. “I don’t want to put one broad brush on everybody, but … they feel like the government’s putting their nose in something a little bit too much.”
The Illinois Democrat has already managed to get one GOP co-sponsor on board with her bill, the NIH Clinical Trial Diversity Act of 2022, which would require clinical trial sponsors to write formal diversity plans when they apply for funding from the National Institutes of Health. If it passes, the bill would also hold pharmaceutical companies responsible for a clear strategy to recruit and retain people largely 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 drug industry to help address the transportation issues, child care and work responsibilities, and mistrust in the medical system that can keep underrepresented groups out of clinical trials.
“It just cannot be the same old same way,” she said.
Kelly, who hinted that she’ll 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 especially hard in the rare disease space. Since any particular rare disease affects such few people, it’s tough to find enough patients who fit 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 limits around clinical trials and research on rare diseases, many times patients are misdiagnosed or receive a late diagnosis despite warning signs. Sometimes they’re in critical condition in the emergency room before a doctor tests for the disease — if the doctor even knows 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 muscular weakness. Randell recalled a harrowing phone call he got in 2005: Michael, one of his 21-month-old triplets, was in the ER with congestive heart failure. Providers couldn’t get an IV in because the infant’s cardiovascular system had collapsed.
Unlike another of his triplet brothers, Michael hadn’t been diagnosed with any medical condition at birth. The doctor told his parents he was “perfectly fine.” Michael didn’t get diagnosed until his parents saw a doctor who happened to have treated another child with Barth syndrome.
In a later session, STAT senior writer Ed Silverman probed into what makes ultra-rare diseases like Barth so difficult to treat, even when high drug prices for rare diseases make new drugs so profitable. Another panel with Washington correspondent Sarah Owermohle touched on next steps a week after advisers to the Food and Drug Administration voted in support of a new drug to treat another rare disorder, ALS.