Orphan Drugs—Exploring the Challenges of Pricing, Reimbursement, and Funding

Glasgow, Scotland, UK—6 November 2017—ISPOR, the professional society for health economics and outcomes research (HEOR), held several sessions on the topic of orphan drugs at its 20th Annual European Congress, currently being held 4-8 November 2017 in Glasgow, Scotland, UK.

Europe’s ORPH-VAL Principles and Orphan Drug Pricing and Reimbursement:
This morning an issue panel, “How to Improve Consistency of Orphan Drug Pricing and Reimbursement in Europe? Application of the ‘ORPH-VAL’ Principles in Germany, France, and the United Kingdom”[IP2], was moderated by, Lieven Annemans, PhD, MSc, Interuniversity Center for Health Economics Research, Ghent University—Brussels University, Ghent, Belgium. Panelists included Karina Ehrig, PhD, Federal Joint Committee, Berlin, Germany; Francois Meyer, PhD, Haute Autorité de Santé, Saint-Denis, France; and Sheela Upadhyaya, MSc, National Institute for Health and Care Excellence, London, UK.

In Europe, rare diseases represent a major public health issue. Orphan medicinal product legislation was introduced in 2000 that led to an increase in the development of orphan drugs. Patient access to these medicines, however, is imperfect. Access varies partially due to pricing and reimbursement challenges with orphan drugs and the different approaches toward access by the various countries in the region.

The European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL), a multistakeholder group of rare disease experts in Europe, published a set of principles for value assessment and funding processes in rare diseases in March 2017. Speakers discussed how pricing and reimbursement for orphan drugs in Germany, France, and the UK align with these principles and opportunities for improvement.


Sustainable Funding and Fair Pricing for Orphan Drugs:

This afternoon a workshop, “Sustainable Funding and Fair Pricing for Orphan Drugs: What Are the Solutions?,” [W5] was held with discussion leaders, Martina Garau, MSc, Office of Health Economics, London, UK; Michael Drummond, MCom, DPhil, University of York, Heslington, York, UK; Saskia Knies, PhD, National Health Care Institute, Diemen, The Netherlands; and Olivier Ponet, Baxalta, Paris, France.

Panelists discussed the options that exist to make funding of valuable orphan drugs sustainable for health care systems while also providing ‘fair’ reward to manufacturers that invest in these areas of high unmet need. Martina Garau, MSc began the session with an overview of the rate of health technology assessment approval and reimbursement of orphan drugs across Europe. Saskia Knies, PhD provided insights from the current Dutch approach of assessing and appraising orphan drugs. Michael Drummond, MCom, DPhil proposed a new method that can be used to adjust the cost effectiveness threshold to reflect the difference between the population size of orphan and non-orphan treatments. And Ulf Staginnus, MBA provided the manufacturers perspective, illustrating the cost drivers and risks associated with research and development of medicines for rare conditions.

Additional information on the ISPOR 20th Annual European Congress can be found here. Released presentations from the congress can be found here. Interested parties can follow news and developments from the congress on social media using the hashtag #ISPORGlasgow.



ISPOR, the professional society for health economics and outcomes research (HEOR), is an international, multistakeholder, nonprofit dedicated to advancing HEOR excellence to improve decision making for health globally. The Society is the leading source for scientific conferences, peer-reviewed and MEDLINE-indexed publications, good practices guidance, education, collaboration, and tools/resources in the field.
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