Current Advances in Clinical Trials for Rare Disease Populations: Spotlight on the Patient

Curr Clin Pharmacol. 2021 Mar 16. doi: 10.2174/1574884716666210316120615. Online ahead of print.ABSTRACTCharacterized by small, highly heterogeneous patient populations, rare disease trials magnify the challenges often encountered in traditional clinical trials. In recent years, there have been increased efforts by stakeholders to improve drug development in rare diseases through novel approaches to clinical trial designs and statistical analyses. We highlight and discuss some of the current and emerging approaches aimed at overcoming challenges in rare disease clinical trials, with a focus on the ultimate stakeholder, the patient.PMID:33726656 | DOI:10.2174/1574884716666210316120615

Source: Current Clinical Pharmacology - March 17, 2021 Category: Drugs & Pharmacology Authors: Erica Winter Scott Schliebner Source Type: research