C-Path’s Rare Disease COA Consortium is pleased to announce that “Approaches to the Assessment of Clinical Benefit of Treatments for Conditions That Have Heterogeneous Symptoms and Impacts: Potential Applications in Rare Disease” was published online in the April 2023 issue of Value in Health. This publication identifies assessment approaches used in clinical trials to address heterogeneous manifestations that could be relevant in rare disease research and Food and Drug Administration-approved labeling claims that used these approaches.
Access here: https://doi.org/10.1016/j.jval.2022.11.012
