Abstract
To address this general difficulty, as well as various difficulties specific to impacts of COVID-19, we describe the approach of using natural history studies for comparative effectiveness analysis used in rare disease drug approval applications. This article is divided in three parts: substantial evidence standards, virtual matched control methodology, and inappropriateness of existing methods such as propensity score methods.
| Original language | American English |
|---|---|
| Journal | |
| State | Published - Apr 8 2020 |
UN SDGs
This output contributes to the following UN Sustainable Development Goals (SDGs)
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SDG 3 Good Health and Well-being
Disciplines
- Biostatistics
- Environmental Public Health
- Epidemiology
- Public Health
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