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Patient organization involvement and the challenge of securing access to treatments for rare diseases: report of a policy engagement workshop

Sturdy, S   Mikami, K

Research Involvement and Engagement   3 (14) 1-13

DOI: https://doi.org/10.1186/s40900-017-0065-z

September 2017

https://www.ncbi.nlm.nih.gov/pubmed/29062539

Patients with rare diseases often help to develop new treatments for their conditions. But once developed, those treatments are sometimes priced too high for many patients to access them. We became aware that this is a problem in the course of a social science research project that examines the place of rare diseases in health policy. We therefore organized a two-day workshop to try and understand why this problem occurs and what might be done about it. The people who participated in our workshop were: representatives of rare disease patient organizations, experts in matters of drug regulation and assessment of new health technologies, consultants involved with companies producing treatments for rare diseases, and social scientists researching related issues.