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Appraisal of Orphan Medicinal Products (OMPs)


Develop guidance on novel approaches to appraise Orphan Medicinal Products (OMPs), recognizing that they are often high cost products that have limited evidence base with high uncertainty (and intense stakeholder scrutiny).

  • Create a database of HTA appraisal and decision-making processes for OMPs in individual countries

  • Optimize evidence and knowledge input to HTA appraisal processes for OMPs

  • Improve use of patient-reported outcome measures (PROMs) in appraisal of OMPs

  • Guide implementation of Outcome Based Managed Entry Agreements to help demonstration of value of OMPs. 

  • Deliverable D10.1: HTA Appraisal Framework Suitable for Rare Disease Treatments (Orphan Medicinal Products) [PDF]
  • Deliverable D10.2: Guidance on Use of Patient Reported Outcome Measures in HTA Appraisals of Rare Disease Treatments [PDF]

  • Deliverable D10.3: Guidance on Use and Implementation of Outcome-Based Managed Entry Agreements for Rare Disease Treatments [PDF]

  • Country vignettes: HTA appraisal/reimbursement processes for rare disease treatments in most EU and EEA Member States, Canada and New Zealand have been documented. These country vignettes are now publicly available HERE
  • Outcomes-Based Managed Entry Agreements (OBMEA) Tools to support use and implementation with a rare disease treatment:

    • Checklist to determine if an OBMEA is appropriate [PDF]

    • Template for public documentation of an OBMEA data collection agreement [WORD DOC]

    • Terms of reference template for a monitoring committee responsible for overseeing implementation of an OBMEA [WORD DOC]

    • Patient group submissions template for re-appraisal after an OBMEA [WORD DOC]

  • Dataset of the characteristics of AIFA registries up to 2019: Xoxi, Entela. (2021). Characteristics of AIFA registries up to 2019 [Data set]. Zenodo.



  • Facey KM, Rannanheimo P, Batchelor L, Borchardt M, de Cock J (2020). Real-world evidence to support Payer/HTA decisions about highly innovative technologies in the EU – actions for stakeholders. International Journal of Technology Assessment in Health Care 1–10 []

  • Nicod E, Whittal A, Drummond M et al. Are supplemental appraisal/reimbursement processes needed for rare disease treatments? An international comparison of country approaches. Orphanet J Rare Dis 15, 189 (2020) []

  • Whittal A, Nicod E, Drummond M, Facey K. Examining the impact of different country processes for appraising rare disease treatments: a case study analysis. International Journal of Technology Assessment in Healthcare. 2021 (in press) []

  •  Meregaglia M, Whittal A, Nicod E, Drummond M. 'Mapping' health state utility values from non-preference-based measures: a systematic literature review in rare diseases. Pharmacoeconomics. 2020 Jun;38(6):557-574. []

  •  Meregaglia M, Nicod E, Drummond M. The estimation of health state utility values in rare diseases: overview of existing techniques. Int J Technol Assess Health Care. 2020 Oct;36(5):469-473 []

  • Whittal A, Meregaglia M, Nicod E. The use of patient-reported outcome measures in rare diseases and implications for health technology assessment. The Patient. 2021 Jan (open access, in press) []

  • Facey K, Espin J, Kent E et al. Implementing outcomes-based managed entry agreements for rare disease treatments: Nusinersen and Tisagenlecleucel. PharmcoEconomics. July 2021. []


Università Commerciale Luigi Bocconi

Centre of Research on Health and Social care Management (CERGAS)


University of Edinburgh

Usher Institute

Research team

Karen Facey (PI)

Elena Nicod (PI)

Michael Drummond

Michela Meregaglia

Amanda Whittal 

Trenholme Junghans


In collaboration with:

Sheela Upadhyaya (NICE)

Entela Xoxi (UCSC)


February 2021 – Webinar #2 on Outcomes-Based Managed Entry Agreements for Rare Disease Treatments, video below (skip along to 03:23 minutes for the start)

March 2021 – Webinar #3 ​​​​on Better Use of Patient-reported Outcome Measures and Health State Utility Values in Appraisal of Rare Disease Treatments

May 2021 – Webinar #4 ​​​​on The IMPACT-HTA Framework suitable for Rare Disease Treatments

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