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. 


A mixed methods approach will be used throughout this work package working closely with HTA decision makers to develop outputs that will be useful to them.

  • Map HTA appraisal processes for OMPs and ultra-OMPs in cancer vs non-cancer sub-groups – including use of PROMS, MEAs and process for pricing negotiations

  • Document evidence considered, appraisal process and decision-making frameworks for HTA appraisals of specific OMPs and ultra-OMPs in different jurisdictions

  • Undertake formal participant observation of appraisal committees discussing OMPs and ultra-OMPs

  • Review how disease specific and generic PROMs are presented in appraisals of OMPs and Ultra-OMPs as evidence of clinical effectiveness and how they are used in economic modelling

  • Use OMP and ultra-OMP HTA case studies to explore disease specific PROM modelling issues for utility mapping

  • Document key elements of publicly available Outcome Based Managed Entry Agreements for OMPs and ultra-OMPs

  • Run workshops with all stakeholders to explore data exchange and collaborative implementation issues in MEAs of OMPs and ultra-OMPs

  • Interviews with HTA decision makers to ascertain processes, issues and test new proposals.

  • This work package will draw in work from WP2/WP7 on use of non-randomized data, WP4 on social costs, WP5 on EQ5D and WP7 on MCDA.


For the HTA Appraisal of OMPs (and Ultra-OMPs).

  • A framework that guides individual jurisdictions or cross-country collaborations on the HTA appraisal, which takes account of issues beyond cost effectiveness and supports robust, accountable decision-making across countries in Europe.

  • Guidance on interpretation of disease specific and generic PROMs in HTA and their use in economic modelling

  • A toolkit to support implementation of Outcome Based Managed Entry Agreements.

HTA appraisal/reimbursement processes for rare disease treatments:
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.

Research Update

January 2019 – IMPACT HTA 2nd Project Meeting (Presentation PDF)

Communique – WP10 Summary (PDF)

June 2019 – International overview of appraisal systems for rare disease treatments – HTAi (Presentation PDF)

June 2019 – Appraisal of RDTs workshop – HTAi (Presentation PDF)

October 2019 – Real-world evidence to support HTA/payer decisions about highly innovative technologies (PDF)

January 2020 – IMPACT HTA 3rd Project Meeting


Università Commerciale Luigi Bocconi


University of Edinburgh

Principal Investigators

Karen Facey

Elena Nicod

This project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 779312