Mission

To identify issues in the rare disease environment so that all stakeholders can effectively address key challenges and more effectively establish the value of new and existing diagnostics and therapeutics.

Background

Rare disease is a rapidly expanding area of research and clinical development. Advancements in genetic understanding and other scientific breakthroughs have led to improved identification of rare conditions and possible pathways for improving rare disease diagnosis and treatment, as well as stratifying relatively common diseases into many rarer ones.

As clinicians are better able to diagnose specific rare diseases, new treatments are becoming available for clinical development. However, treatments for rare diseases are typically available at much higher "per patient" cost, which (along with other factors) create challenges to payers, providers and patients.


Work Products




Manuscripts & Reports




Leadership



Chair-Elect

Joel Iff, PhD

Senior Director Market Access, Sarepta Therapeutics
Cambridge, MA, United States

Chair

Sheela Upadhyaya

Life Science Adviser - Speciality Rare Diseases, NICE
London, LON, United Kingdom

Past Chair

Jamie O'Hara

CEO, Health Economics
Daresbury, CHW, United Kingdom

Working Groups



Member Engagement


These activities allow for a variety of members to participate and also facilitates disseminating content.

Co-Chairs:

Kapil Khambholja

CSO, Genpro Research Inc
Waltham, MA, United States

Eric Low, MSc

Independent Healthcare Consultant, Eric Low Consulting
Haddington, ELN, United Kingdom

Antony Martin

Principal Health Economist, QC Medica
York, NYK, United Kingdom

Questions or ideas? Please send an email to rarediseaseSIG@ispor.org

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