HEOR models are only as credible as the inputs behind them. In rare diseases, those inputs are frequently incomplete: natural history is poorly characterized, endpoints are inconsistent, and small, fragmented patient populations amplify uncertainty in both clinical and economic parameters (Grand et
al., 2024; Pearson et al., 2018).
Real-world evidence (RWE) underpins modern health economics and outcomes research (HEOR), especially when traditional clinical trials are infeasible in rare diseases. Small patient populations, varying symptoms, and uncertain etiology can limit trial insight and complicate endpoint selection.
100 rare disease treaters in the US and the UK shared their biggest challenges in treating and managing rare disease patients.
MedPanel asked 100 rare disease treaters in the US and the UK if pharmaceutical companies should be involved in rare disease patient advocacy groups.
In what care setting do doctors see patients with rare diseases? How do they keep up with treatment advances? Can pharmaceutical companies support rare disease treaters and the patient community? Those were just some of the questions addressed by exclusive research carried out by Cambridge, Massachusetts-based healthcare market research company
MedPanel has always been an industry leader in conducting market research in the rare disease space and now we’re formalizing those efforts to support the growing needs of our customers – introducing RareDiseaseMatters.com. During Rare Disease Week, February 24th through the 28th in Washington, DC, MedPanel will be formally launching