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HTA programme response to the challenges of dealing with orphan medicinal products: process evaluation in selected European countries

Elena Nicod, Lieven Annemans, Anna Bucsics, Anne Lee, Sheela Upadhyaya, Karen Facey
April 2017

Following the Advance-HTA project, various new initiatives have been developed to specifically address commonly encountered issues relating to health technology assessment (HTA) of orphan medicinal products (OMPs). Reviewing these initiatives across England, Scotland and at the European-level, this study aims to understand why they were established and whether they resolved the challenges around OMPs. A literature review was conducted and the new HTA programmes were critiqued against a conceptual framework of clinical, regulatory, and economic challenges for OMPs. The new initiatives acknowledge the need for different approaches for assessing the value of OMPs and employ similar criteria across countries. However, questions still remain about whether they sufficiently capture value and manage uncertainty in clinical practice.

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