Academic Journal
Integrating Equity in Topic Selection for Orphan Medicinal Products: Policy Implications for Health Technology Assessment in Rare Diseases.
| Τίτλος: | Integrating Equity in Topic Selection for Orphan Medicinal Products: Policy Implications for Health Technology Assessment in Rare Diseases. |
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| Συγγραφείς: | Hosseini F; Peninsula Technology Assessment Group (PenTAG), University of Exeter, Exeter, UK., Mobinizadeh M; Health Management and Economics, National Institute for Health Research, Tehran University of Medical Sciences, Tehran, Iran., Padula WV; Department of Pharmaceutical and Health Economics, Alfred E. Mann School of Pharmacy and Pharmaceutical Sciences, University of Southern California, USC Schaeffer Center, 635 Downey Way (VPD), Los Angeles, CA, 90089, USA. padula@usc.edu.; The Leonard D. Schaeffer Center for Health Policy and Economics, University of Southern California, Los Angeles, CA, USA. padula@usc.edu.; Stage Analytics, Suwanee, GA, USA. padula@usc.edu. |
| Πηγή: | Applied health economics and health policy [Appl Health Econ Health Policy] 2026 Jul; Vol. 24 (4), pp. 647-654. Date of Electronic Publication: 2026 May 17. |
| Τύπος έκδοσης: | Journal Article; Review |
| Γλώσσα: | English |
| Στοιχεία περιοδικού: | Publisher: Open Mind Journals Ltd Country of Publication: New Zealand NLM ID: 101150314 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1179-1896 (Electronic) Linking ISSN: 11755652 NLM ISO Abbreviation: Appl Health Econ Health Policy Subsets: MEDLINE |
| Imprint Name(s): | Original Publication: Auckland, N.Z. : Open Mind Journals Ltd., c2002- |
| Ιατρικοί όροι (MeSH): | Rare Diseases*/drug therapy , Technology Assessment, Biomedical*/organization & administration , Orphan Drug Production*/economics , Health Policy* , Health Equity*, Humans ; Diversity, Equity, Inclusion ; Policy Making ; Decision Making |
| Περίληψη: | Health technology assessment (HTA) informs evidence-based decision-making for resource allocation in healthcare; however, the topic selection phase that determines which technologies proceed to full assessment is underexplored and often lacks fairness and transparency, particularly disadvantaging orphan medicinal products (OMPs) for rare diseases. We drew on selectively identified literature and illustrative HTA experiences to advance an opinion-driven argument about equity in topic selection for OMPs, grouping insights into three domains: structural challenges, conceptual frameworks and policy strategies. Structural barriers in topic selection include a lack of institutionalised, participatory processes, overreliance on prioritisation criteria implicitly aligned with population-wide impact and minimal engagement with stakeholders representing the rare disease community, resulting in the systematic under-prioritisation of OMPs. Procedural and value frameworks, including multi-criteria decision analysis (MCDA), evidence-informed deliberative processes (EDPs), accountability for reasonableness (A4R), equity weighting and Health Equity Impact Assessment (HEIA), can help agencies incorporate social value and fairness before formal appraisal, but remain unevenly operationalised. Policy strategies such as equity-oriented horizon scanning, open nomination systems, and special pathways for OMPs are highlighted as feasible approaches to promote fairness. Achieving equity in HTA topic selection for OMPs requires both policy and cultural change, and embedding pluralistic value frameworks, engaging stakeholders and adopting specific policy tools are essential steps towards ensuring that rarity is met with heightened ethical attention rather than exclusion. (© 2026. The Author(s).) |
| Competing Interests: | Declarations. Funding: The authors did not receive support from any organisation for the submitted work. Conflict of interest: Authors MM and FH have no conflict of interests to declare. Unrelated to the submitted work, WVP declares grant support from PhRMA Foundation and AbbVie and also declares personal fees and equity holdings from Stage Analytics. |
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| Entry Date(s): | Date Created: 20260517 Date Completed: 20260613 Latest Revision: 20260803 |
| Update Code: | 20260804 |
| PubMed Central ID: | PMC13260230 |
| DOI: | 10.1007/s40258-026-01048-0 |
| PMID: | 42143656 |
| Βάση Δεδομένων: | MEDLINE |
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