Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.
Duchenne Parent Project, Veenendaal, The Netherlands.
Nucleic Acid Ther. 2022 Apr;32(2):118-122. doi: 10.1089/nat.2021.0048. Epub 2021 Oct 1.
The drug development process is a long and arduous one, especially for rare diseases. Patient and patient representatives can and should be involved in this process from an early stage, since they have the perspective of living with a disease on a daily basis and can best identify which symptoms are the largest burden and which benefits would be more important to them. In this perspective, we outline how patients can be involved optimally in drug development. We outline success factors such as finding the right partners, bilateral education, having realistic expectations, and an open and honest dialog with all stakeholders.
药物研发过程漫长而艰辛，特别是针对罕见病。患者及其代表应从早期就参与到这一过程中，因为他们每天都在与疾病作斗争，最能识别哪些症状是最大的负担，以及对他们来说哪些获益更为重要。从这个角度出发，我们概述了如何让患者最佳地参与到药物研发中。我们概述了成功的因素，如找到合适的合作伙伴、双向教育、具有现实的期望以及与所有利益相关者进行开放和诚实的对话。
