Recommended methods for the collection of clinical expert judgment in rare diseases: Generating evidence to support reimbursement of orphan drugs
These recommendations create a blueprint for developers of rare disease therapies to conduct high-quality clinical expert judgment studies. Hence, developers can present more robust evidence to inform key areas of uncertainty in reimbursement decisions, where empirical evidence is unavailable.
Authors
- Isabelle Newell
- Dina Janković (ORCID: https://orcid.org/0000-0002-9311-1409)
- Sheela Upadhyaya
- Fabian Schmidt (ORCID: https://orcid.org/0000-0002-6807-7464)
- L. H. DUNNING (ORCID: https://orcid.org/0000-0002-0098-2572)
- Michela Meregaglia (ORCID: https://orcid.org/0000-0003-0092-5970)
- Carlos González Malla
- Kevin F. Wilson
- Karen Facey (ORCID: https://orcid.org/0000-0001-6670-5271)
- Sari D Wright
- Eric Low (ORCID: https://orcid.org/0000-0001-8600-3357)
- N. B. Chapman
- Annabel Griffiths
- Chloe Zentai
Institutions
- Universidad de Buenos Aires (AR)
- National Institute for Health and Care Excellence (GB)
- Enzo Life Sciences (United States) (US)
- Recordati (Ireland) (IE)
- Institute of Life Sciences (IN)
- RED Consulting (Norway) (NO)
- Recordati (Italy) (IT)
- Costello Medical Consulting (United Kingdom) (GB)
- University of York (GB)
- Bocconi University (IT)
- Newcastle University (GB)
- University of Edinburgh (GB)
Publication Details
- Journal
- International Journal of Technology Assessment in Health Care
- Published
- 2025-01-01
- DOI
- https://doi.org/10.1017/s0266462325100457
- Primary Topic
- Genomics and Rare Diseases
- Type
- article
- Field-Weighted Citation Impact
- 0.00