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.
Putting the Patient at the Center of Rare Disease Clinical Trials
Creating a Toolkit to Accelerate the Development of Gene Editing Therapies
PTC Looks to Advanced Therapies
Going to Extreme Lengths
Everything’s up to Date in Kansas City, at Least When It Comes to Genomics
Choosing the Right Viral Vector for a Gene Therapy
A Software Platform to Give Patients the Tools to Build Treatments for Rare Genetic Diseases
Side Effects: The Toll a Rare Disease Can Take on a Family in Pursuit of a Cure
Realizing the Potential of CRISPR Gene Editing
Understanding the Economic Toll of Rare Disease in the United States
The Promise of Gene-Based Therapies for Neurodegenerative Conditions
Moving Beyond Viral Vectors for Gene Therapies
Acquisitions Help Jazz Build Toward Commercial Crescendo
Powerful Gene Editing Approach Offers the Promise of Correcting a Range of Rare Diseases
A Child's-Eye-View of Clinical Trials
Using Nanoliposomes to Make Cancer Therapies Safer and More Effective
Accelerating Treatments for Rare Disease through Data Sharing
Treating the Root Cause of Sickle Cell Disease
Targeting a Common Pathway in Genetic Forms of Obesity
After Pruning Orchard, Gaspar Focuses on High Value Opportunities
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