Access to Orphan Medicines: A Case for Change
MAP BioPharma has published a report on access to rare disease medicines, developed with support of a Steering Group of MAP Online members*. The report finds that the assessment route for orphan medicines has a significant impact and that NICE’s Single Technology Appraisal does not deliver equal access for patients, with routine availability often delayed due to inflexible, inappropriate assessment processes.
*The Steering Group members are Amicus Therapeutics, AveXis, bluebird bio UK, Chiesi Limited, Gilead Sciences Inc, Kyowa Kirin International and Santhera Pharmaceuticals. Each company made an equal financial contribution to support this work. MAP retains editorial control