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Pharmaceuticals

Patient Access: Regulatory, HTA, and Patient Perspectives on an Orphan Drug – Case Study of Erythropoietic Protoporphyria

Tuesday, May 7, 2024
08:30 – 09:30 CEST
Location: B 07 + B 08
Learning Level: Expert
Description: The different evidence requirements for an EU-wide marketing authorisation recommendation by the European Medicines Agency (EMA) and for a national reimbursement decision can be a key obstacle for patients’ access to innovative new medicines. Using a case study from Germany, this session will look at the pathway of an approved orphan drug for the treatment of erythropoietic protoporphyria (EPP) within the benefit assessment by the G-BA – the German decision-making health technology assessment (HTA) body. The underlying challenges will be explained from the different perspectives of the EMA, the G-BA and an EPP patient. The interdependencies between the opinions of the Committee for Orphan Medicinal Products (COMP), the Committee for Medicinal Products for Human Use (CHMP) and the G-BA will be outlined and the benefits of parallel EMA-HTA discussions will be highlighted. The involvement of the EPP patient in both regulatory and HTA discussions will be analysed, including in the selection of endpoints and measurement scales. The implications of these approaches for the national benefit assessment in Germany will be discussed in the light of the upcoming EU Joint Clinical Assessment.

Learning Objectives:

  • Describe which EU regulatory decisions affect the national HTA decision in Germany and the benefits of parallel EMA-HTA discussions.
  • Adapt the clinical development strategy of orphan medicinal products to the patient needs, regulatory and HTA requirements.
  • Explain the role of patient involvement in both regulatory and HTA discussions for this orphan medicinal product.