The small patient populations of rare diseases, the limited natural history of these conditions, and the lack of long-term experience with new treatments all contribute to the difficulty in determining the value of rare disease therapies. The issue can be further complicated by whether value is calculated in a single payer system, or a multi-payer system. Recently the Innovation and Value Initiative issued a report with the Everylife Foundation for Rare Diseases from a long-term project to bring together stakeholders to explore patient-centered outcomes across rare diseases to inform those discussions. We spoke to Rick Chapman, chief scientific officer of the Innovation and Value Initiative, about the challenges of assessing the value of rare disease therapies, the role qualitative data should play in value assessments, and the recommendations from the report.
Creating a Playbook for Bespoke Gene Therapies
Finding Answers for Undiagnosed Patients with Rare Genetic Diseases
How a Familiar Face Can Lead to a Rare Disease Diagnosis
Bringing the Trial to the Patient
Targeting Rare and Chronic Kidney Diseases
Addressing the Delivery Challenges of Genetic Medicines
Targeting Tissues Throughout the Body with RNA Therapies
Delivering Rare Disease Therapies to Patients in Need
An Advocate's Journey through Diagnosis, Loss, and Hope
Using Model Systems to Find Drugs to Repurpose for Rare Diseases
How a Small and Young Foundation Catalyzed Research into a Rare Disease
Overcoming the Limitations of Conventional Cell Engineering
Experimental Therapy for Rare Endocrine Disorder Offers Hope of Improved Care
Forging Gene Therapy Capacity and a Pipeline at the Same Time
Using A Natural DNA Repair Process to Improve Genetic Medicines
Empowering Rare Disease Patients with their Own Health Records
Targeting CNS Disease with Gene Therapies
A New Therapy Offers A Different Approach to Inhibiting the Complement System
Bringing Aberrant Proteins Back into the Fold
A Cat who Curates Rare Mice
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