A clinical researcher applies a bandage to a study participant.

How to connect patients with rare diseases to clinical trials

Experts say a wider diverse pool of patients needs to be given a chance to participate in clinical research to get rare disease drugs faster to market.

By Manasi Vaidya, Clinical Trials Arena

Rare disease clinical research is no longer an afterthought for pharmaceutical companies with rapid approvals and increased investment in this space. But conducting trials for conditions that affect relatively few patients still remains challenging compared to conventional ones.

The journey that rare disease patients take to find care greatly differs from those who have common diseases like hypertension, said Jay Russak, senior director, Clinical operations, at the biotech Keros Therapeutics. The company is developing rare disease drugs currently in Phase I and Phase II studies. On average, it takes seven years to get a diagnosis in the rare disease community, he added. Russak said the question then becomes, “how do we identify patients who are lost in the system”.

Last week, at the Clinical Trials in Rare Diseases 2023 conference in Princeton, New Jersey, patient advocates, clinical investigators and biotech representatives gathered to discuss factors influencing clinical research in rare diseases. Primary amongst them was the difficulty in identifying patients with rare diseases.

Firstly, patients need to be asked. Often, marginalised groups do not participate in clinical trials not only because of mistrust due to historical events like the Henrietta Lacks case, but also because they are not asked, said Jenifer Ngo Waldrop, executive director of the nonprofit Rare Disease Diversity Coalition. Read more …