Mission
To identify key challenges in the evaluation of rare disease technologies and develop solutions that will support healthcare decision-making and foster equitable access for patients.
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.
Goals
Identify and prioritize key challenges in the evaluation of new rare disease technologies, and explore existing barriers to equitable patient access globally
Develop strategies and patient-centered solutions that can mitigate existing market access challenges and improve access decision-making for new and existing rare disease technologies
Manuscripts & Reports
Leadership
Chair-Elect
Carina Righetti, MSc, BSc
Chair
Joel Iff, PharmD, PhD
Past Chair
Sheela Upadhyaya
Operational Lead
Charles Brown
Working Groups
Member Engagement
These activities allow for a variety of members to participate and also facilitates disseminating content.
Co-Chairs:
Sushmitha Inguva, MS, BS
Kapil Khambholja
Eric Low, MSc
Antony Martin, MSc, BSc
James Wu, MSc, MPH
Key Project
Definition and Consistent Approaches to Integrating Evidence-Based Patient Advocacy in Rare Disease Health Technology Assessment (HTA)
Co-Chairs:
Suchita Chaudhari
Mohit Jain, MBA, PhD, BA
Sheela Upadhyaya
Questions or ideas? Please send an email to rarediseaseSIG@ispor.org