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Current Reviews in Clinical and Experimental Pharmacology

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ISSN (Print): 2772-4328
ISSN (Online): 2772-4336

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Review Article

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

Author(s): Erica Winter* and Scott Schliebner

Volume 17, Issue 1, 2022

Published on: 16 March, 2021

Page: [39 - 45] Pages: 7

DOI: 10.2174/1574884716666210316120615

Price: $65

Abstract

Characterized 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.

Keywords: Rare disease, small populations, clinical trials, patient-centric, innovation, drug development.

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