‘All the tech in the world doesn’t solve this’: Rare disease experts push biopharma on equity

Over the past few years, the biopharmaceutical industry has revved up efforts to diversify clinical trials. But clinical trials for rare diseases are still too often homogeneous. Rare disease experts at the Milken Institute Future of Health Summit on Tuesday had words of warning for biopharma: Don’t let equity efforts peter out.

Pharmaceutical companies can’t keep developing trials for rare disease treatments without solving for equity, said Tamar Thompson, head of corporate affairs at Alexion, AstraZeneca’s rare disease division. “We need to put this as a challenge to the CEOs and think about the environmental, social, and governance aspects of this as well.”

When asked by STAT’s Nicholas St. Fleur how to ensure the momentum around equity doesn’t fizzle out, panelists agreed that CEOs had to back up their talk with actions inside and outside the company.


“All the tech in the world doesn’t solve this right now,” said Nicole Boice, executive director of nonprofit Rare-X. “It’s more about what we can learn and then listen and reassess and reevaluate, because it is really about understanding these lived experiences and challenges that many of us haven’t.”

Pharmaceutical companies need to be aware of how communities’ needs differ, the panelists said. In some cases, language barriers might be a large obstacle to enrolling in clinical trials for certain rare diseases. In other communities, challenges might involve the time and money required to travel to the far-away centers that treat a given rare disease.


Income barriers are often a major issue, particularly for marginalized communities. When children deal with rare genetic diseases that manifest from a young age, their caregivers often have to quit their jobs, further compounding health inequities with financial disparities, Thompson said.

Diversifying rare disease research is a tougher feat when advocacy groups themselves aren’t diverse. In the rare disease space, these groups have traditionally enjoyed close relationships with researchers, said Donna Cryer, CEO of the Global Liver Institute, a patient advocacy nonprofit. But if the advocacy groups’ boards, staff, and events aren’t diverse, then their outreach, education, and ultimately the research they help advance won’t be diverse enough, she said.

Pharma also needs to invest in rare diseases that disproportionately affect Black people, the experts agreed, not just “white men’s diseases” or conditions such as cystic fibrosis that are more prevalent among white people.

“Look at all of the money and the infrastructure that went into finding … very strong treatments for [cystic fibrosis] when the patient population is probably three to four times more in the sickle cell space, and one has to ask why,” said Thompson. “We can continue to say that it’s hard to find patients or [other] challenges. At some point, they become excuses.”

Pharmaceutical executives might also take a hard look at how their internal operations can stymie diversity efforts. They need to bridge silos in their company so staff with closer community connections can share knowledge about particular groups, Cryer said. These are the employees who might, for instance, already know about the “promotora” community health worker model, she added.

Companies should also solicit direct feedback from marginalized communities, said Mary McGowan, CEO of nonprofit Foundation for Sarcoidosis Research (FSR), citing a recent national survey in which FSR asked Black respondents their thoughts on clinical trials.

As for the issue of how to incentivize communities’ participation, that might be the wrong question to ask, Cryer said, to raucous agreement from fellow panelists.

“The right question, I feel, is more, ‘How can we create a clinical research enterprise that people want to participate in, that they feel reflects them, that showcases the questions … that came from the community?’” Cryer said. “How do we get a research system that has earned the trust of underrepresented people?”

Source: STAT