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.
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.
Manuscripts & Reports
Carina Righetti, MSc, BSc
Joel Iff, PharmD, PhD
These activities allow for a variety of members to participate and also facilitates disseminating content.
Sushmitha Inguva, MS, BS
Eric Low, MSc
James Wu, MSc, MPH
Definition and Consistent Approaches to Integrating Evidence-Based Patient Advocacy in Rare Disease Health Technology Assessment (HTA)
Mohit Jain, MBA, PhD, BA
Questions or ideas? Please send an email to rarediseaseSIG@ispor.org