HTA Evidence in Rare Diseases: Just Rare or Also Special?
Anirban Basu, Simu K. Thomas, Richard H. Chapman, Jason Spangler
Abstract
Manufacturers of orphan drugs face several obstacles in meeting health technology assessment requirements because of poor availability of natural history data, small sample sizes, single-arm trials, and a paucity of established disease-specific endpoints. There is a need for specific considerations and modified approaches in health technology assessments that would account for the challenges in orphan drug development. Multistakeholder collaborations can benefit patients, their families, and the broader society and reduce the inequity faced by patients with rare diseases.
Topics & Concepts
Orphan drugHealth economicsQuality of Life ResearchMedicinePublic healthHealth technologyHealth administrationRare diseasePharmacoeconomicsHealth services researchBusinessCost–benefit analysisMEDLINEHealth careHealth policyFace (sociological concept)Drug approvalWork (physics)Risk analysis (engineering)Genomics and Rare DiseasesHealth Systems, Economic Evaluations, Quality of LifeBiotechnology and Related Fields