Efficacy and safety of compassionate use for rare diseases: a scoping review from 1991 to 2022.

ABSTRACT:

Background

Compassionate use is a system that provides patients with expedited access to drugs which has not yet been approved, but currently in clinical trials. The investigational drugs have been authorized for compassionate use in cases involving patients suffered from life-threatening diseases and with no alternative treatments. For instance, patients afflicted with highly heterogeneous rare diseases are eligible for treatment assistance through the compassionate use program. This study aims to investigate the characteristics of compassionate use in the context of rare diseases, evaluate the efficacy and safety of compassionate use for rare diseases, and analyze the marketing approval of investigational drugs.

Methods

The case reports/case series of compassionate use wer

SUBMITTER: Wu J 

PROVIDER: S-EPMC10685565 | biostudies-literature | 2023 Nov

REPOSITORIES: biostudies-literature