How Rare Disease Patients Weigh Risks and Benefits of a Therapy

Patients with a rare disease may face debilitating, degenerative, and even life-threatening conditions, often with little treatment options. Their willingness to accept risks in the use of a therapeutic that may provide them benefit may lead to a different calculus than what regulators might consider. A study published at the end of May in the Orphanet Journal of Rare Diseases sheds light on how rare disease patients might weigh the potential benefits and risks of a therapy. We spoke to Thomas Morel, lead author of the study and a research fellow at Leuven University in Belgium, about his findings, what factors change a patient’s willingness to assume risks, and how this work might advance the discussion about the need to better consider patients’ perspectives in the regulatory process.