Better collaboration is needed among patient advocacy groups and biotech

In a recent fireside chat at the Rare Disease Summit in Philadelphia, Karin Hoelzer, senior director, patient advocacy at Biotechnology Innovation Organization (BIO), and Tricha Shivas chief strategy officer at the Foundation for Sarcoidosis Research explored the role patient advocacy groups play in accelerating and improving the development of therapies for rare diseases.

For the Foundation for Sarcoidosis Research, Shivas said one of the challenges of working with biotech and pharma is the understanding of the patient population. While sarcoidosis affects about 175,000 people in the US, the foundation primarily focuses on the approximately 30% of that population whose condition requires complex treatment beyond basic interventions or observation, she noted.

Referring to an earlier panel, the panelists mentioned using claims data and IDC codes to understand disease populations, according to Shivas.

“Claims data doesn’t always tell the story,” she said. “The data that they’re getting is not fully comprehensive, or sometimes has a lot of misinformation, misdiagnosis, and other components that are really shaping how trials are designed.

Hoelzer gave her perspective on how biotech companies can better collaborate with patient advocacy organizations to gain a deeper understanding of the patient population for whom they are developing drugs, particularly in clinical trial design.

Drawing on her past experience in patient advocacy as the senior director of policy and regulatory affairs at the National Organization for Rare Diseases, she stressed that understanding diseases grows as patients connect, uncovering new symptoms and highlighting underdiagnosis and subpopulations.

“The reason why biotech really should start partnering with the patient community from the beginning is because nobody knows the disease better than the patient community that is living with the disease day in, day out,” Hoelzer said.

“There are so many examples where working with the patient community really helps understand how to design a clinical trial so that it fits into their life, so that we have good recruitment and retention, and that we have outcomes that we measure that actually matter to the patient community as we go,” she continued.

Foundation for Sarcoidosis Research has taken proactive steps to bridge the gap between patients and biotech companies, according to Shivas. They’ve implemented a three-step approach: training patients to be effective advisors, understanding the landscape of clinical trial-ready clinicians, and optimizing their role in the regulatory and recruitment processes for clinical trials.

"It’s not a one-time conversation, which I think for us, has been a really big learning curve,” Shivas said. “Everything about understanding the trials in our space requires consistent conversations about what are those barriers, what are those challenges.”

The success of this approach is evident in the Foundation for Sarcoidosis Research’s recent achievements. “This year alone, between 2024 and early 2025, we have completed four clinical trials in our space for enrollment, working really closely with our pharmaceutical partners,” she said.

“Being able to do that and work closely with our partners to make that possible has been a long journey, but something that we now think we have a mechanism for doing that we consistently revamp, but is really important,” she continued.

Shivas raised the issue of maintaining biotech and pharma commitment to rare disease research beyond individual clinical trials, especially when trials fail, as this can create long-lasting barriers.

“I think that’s unfortunately too universal within the rare disease community,” Shivas said, posing the question, “How is it that we can get biotechs and pharma to start thinking about the investment beyond the moment of a particular trial, still staying in that clinical trial space when trials are not successful, or when the learnings from those trials are maybe guiding in a particular way?”

In response, Hoelzer said, “Starting early and not thinking about the interactions in a transactional way. Really partnering because of shared priorities and shared goals but really taking that long view and thinking about that longer-term partnership, I think that is very important.”

DepositPhotos/AndreyPopov