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.
Examining the Legislative Landscape for Rare Disease Drug Development
How a Drug Setback Became a Patient Community’s Gain
Helping Regulators and Drug Developers Understand the Challenges of Living with Fabry Disease
Addressing Racial Disparities in a Rare Blood Cancer
How New Sequencing Technology Promises to Alter the Diagnostic Odyssey
Leveraging Technology to Empower Patients and Decentralize Clinical Trials
Equipping The Next-Generation Rare Disease Patient Advocate
How a Family Raced from Diagnosis to Experimental Gene Therapy in Three Years
How One Advocate Went from Losing His Hearing to Being Heard
Building a Pipeline of Therapies to Treat Rare Mineralization Disorders
Advancing an Oral Alternative to Infused and Injected Therapies for HAE
Embracing the Promise of Patient-Centered Biotechnology
Learning to Live with Loss
Using Cryptocurrency to Tap into the Wisdom of Crowds
A Therapy for a Rare Neurodegenerative Disease Moves Towards Regulatory Review
Transforming the Treatment of Neuromuscular Diseases with Next-Gen Oligonucleotides
Teaching the Immune System to Let Medicines Do Their Job
Designing Clinical Trials with the Patient in Mind
Keeping Clinical Trials Running Smoothly
A Vision for Patient-Centric Gene Therapy Development
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