Published Nov 1, 2016

Vienna, Austria—1 November 2016—The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) offered a thought-provoking issue panel, Valuing Transformative Medicines in Rare Diseases: Methods and Madness, at the Society’s 19th Annual European Congress in Vienna, Austria. Paul Hodgkins, PhD, Vice President, Vertex Pharmaceuticals, Boston, MA, USA moderated the session. Panelists included:

• Ron Akehurst, DSc, Strategic Director, BresMed Health Solutions, Sheffield, UK
• Alastair Kent, OBE, Director, Genetic Alliance UK, London, UK
• Maarten Postma, PhD, Professor Pharmacoeconomics, Pharmacy Department, University of Groningen, Groningen, The Netherlands

Innovative treatments for rare diseases are often transformative for patients. This is especially true if the therapy is the first to treat a disease’s underlying cause, said moderator Paul Hodgkins. It can, however, be difficult to demonstrate clinical benefit and cost effectiveness due to a number of research challenges such as small sample sizes in clinical trials and the lack of good outcome measures. The question was raised, “Why should rare and/or ultra-rare conditions be treated any differently in health technology assessment than more common conditions?” Panelists examined the pros and cons of adhering to current methods and evidence standards versus employing alternative methodological approaches and data sources in rare disease treatment evaluation. Alastair Kent, OBE discussed the importance of patient values and priorities when developing rare disease treatments and outcome measures. ISPOR offers a number of working groups and resources related to this topic, including:

• Clinical Outcomes Assessment in Rare Disease Clinical Trials—Emerging Good Practices Task Force
• Patient Centered Special Interest Group
• Rare Disease Special Interest Group
• Rare Disease Terminology and Definitions—A Systematic Global Review: Report of the ISPOR Rare Disease Special Interest Group (Value in Health 2015; 18:906-14)
• Patient Representatives Roundtable

Additional information on the ISPOR 19th 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 conference on social media using the hashtag #ISPORVienna.

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