PD60 Leveraging Real-World Evidence For Rare Disease And Oncology Health Technology Assessments: Insights From Pulse Infoframe Inc.’s Hybrid Approach
Daniel Lewi, Femida Gwadry-Sridhar · 2025
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Description
Daniel Lewi and Femida Gwadry-Sridhar wrote a 2025 report on using real-world evidence in reviews of rare disease and cancer drugs. Such reviews are hard: patient counts are small, data are thin, and the right measures of success are unclear. The authors describe a hybrid approach built by a firm called Pulse Infoframe.
The approach starts with patients, then mixes site-based and remote data gathering. Patient reports and clinical registries feed one platform in a standard format. One case backed payment for a treatment for uveal melanoma, a rare eye cancer, in several regions. Data from the platform were paired with early access program data to show unmet need. The report argues that patient-first data can make reviews of rare drugs fairer and faster.
Catalog record
| CREATOR | Daniel Lewi, Femida Gwadry-Sridhar |
|---|---|
| YEAR | 2025 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 2 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |