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.
Daniel S. Levine is an award-winning business journalist who has reported on the life sciences, economic development, and business policy issues throughout his 25-year career. He founded Levine Media Group in 2013 to provide strategic communications to life sciences companies. He is host of The Bio Report and RARECast podcasts, a senior fellow at the Center for Medicine in the Public Interest, and a member of the advisory board of the California Biotechnology Council.