title: HTA programme response to the challenges of dealing with orphan medicinal products: Process evaluation in selected European countries.
abstract: Challenges commonly encountered in HTA of orphan medicinal products (OMPs) were identified in Advance-HTA. Since then, new initiatives have been developed to specifically address issues related to HTA of OMPs.
  
  
  
  This study aimed to understand why these new HTA initiatives in England, Scotland and at European-level were established and whether they resolve the challenges of OMPs. The work of Advance-HTA was updated with a literature review and a conceptual framework of clinical, regulatory and economic challenges for OMPs was developed. The new HTA programmes were critiqued against the conceptual framework and outstanding challenges identified.
  
  
  
  The new programmes in England and Scotland recognise the challenges identified in demonstrating the value of ultra-OMPs (and OMPs) and that they require a different process to standard HTA approaches. Wider considerations of disease and treatment experiences from a multi-stakeholder standpoint are needed, combined with other measures to deal with uncertainty (e.g. managed entry agreements). While approaches to assessing this new view of value of OMPs, extending beyond cost/QALY frameworks, differ, their criteria are similar. These are complemented by a European initiative that fosters multi-stakeholder dialogue and consensus about value determinants throughout the life-cycle of an OMP.
  
  
  
  New HTA programmes specific to OMPs have been developed but questions remain about whether they sufficiently capture value and manage uncertainty in clinical practice.
authors.affiliation: Centre for Research on Health and Social Care Management, Bocconi University, Via Roentgen 1, 20126 Milan, Italy; Department of Social Policy, London School of Economics and Political Science, Houghton Street, WC2A 2AE, London, United Kingdom. Electronic address: elena.nicod@unibocconi.it.
authors.forename: Elena
authors.initials: E
authors.lastname: Nicod
authors.affiliation: Department of Public Health, Ghent University, De Pintelaan 185, 4K3, 9000 Gent, Belgium. Electronic address: lieven.annemans@Ugent.be.
authors.forename: Lieven
authors.initials: L
authors.lastname: Annemans
authors.affiliation: Department of Finance, University of Vienna, Oskar-Morgenstern-Platz 1 (6th floor), 1090 Vienna, Austria. Electronic address: anna.bucsics@univie.ac.at.
authors.forename: Anna
authors.initials: A
authors.lastname: Bucsics
authors.affiliation: Scottish Medicines Consortium, Delta House (8th floor), 50 West Nile Street, G1 2NP Glasgow, Scotland, United Kingdom. Electronic address: annelee@nhs.net.
authors.forename: Anne
authors.initials: A
authors.lastname: Lee
authors.affiliation: Centre for Health Technology Evaluation, National Institute for Health and Care Excellence, 10 Spring Gardens, SW1A 2BU London, United Kingdom. Electronic address: sheela.upadhyaya@nice.org.uk.
authors.forename: Sheela
authors.initials: S
authors.lastname: Upadhyaya
authors.affiliation: Usher Institute of Population Health Sciences and Informatics, University of Edinburgh, Nine Edinburgh BioQuarter, 9 Little France Road, EH16 4UX Edinburgh, Scotland, United Kingdom. Electronic address: k.facey@btinternet.com.
authors.forename: Karen
authors.initials: K
authors.lastname: Facey
mesh_terms: D004739:England; D005060:Europe; D006291:Health Policy; D006801:Humans; D009965:Orphan Drug Production / Q000191:economics*; D019057:Quality-Adjusted Life Years; D035583:Rare Diseases; D012606:Scotland; D013673:Technology Assessment, Biomedical / Q000379:methods* / Q000458:organization & administration*; D035501:Uncertainty
keywords: England; Europe; Rare diseases; Scotland; Technology assessment; Uncertainty; Value of life