Department of Medicine, University of California at Davis School of Medicine, Sacramento, CA 95817, USA.
Osteoarthritis Cartilage. 2011 May;19(5):478-82. doi: 10.1016/j.joca.2010.09.013. Epub 2011 Mar 23.
To respond to a pre-specified set of questions posed by the United States Food and Drug Administration (FDA) on defining the disease state to inform the clinical development of drugs, biological products, and medical devices for the prevention and treatment of osteoarthritis (OA).
An Osteoarthritis Research Society International (OARSI) Disease State working group was established, comprised of representatives from academia and industry. The Working Group met in person and by teleconference on several occasions from the Spring of 2008 through the Autumn of 2009 to develop consensus-based, evidence-informed responses to these questions. A report was presented at a public forum in December 2009 and accepted by the OARSI Board of Directors in the Summer of 2010.
An operational definition of OA was developed incorporating current understanding of the condition. The structural changes that characterize OA at the joint level were distinguished from the patients' experience of OA as the 'disease' and 'illness', respectively. Recommendations were made regarding the evaluation of both in future OA clinical trials. The current poor understanding of the phenotypes that characterize OA was identified as an important area for future research.
The design and conduct of clinical trials for new OA treatments should address the heterogeneity of the disease, treatment-associated structural changes in target joints and patient-reported outcomes.
针对美国食品和药物管理局（FDA）提出的一系列预先设定的问题进行回应，以确定用于预防和治疗骨关节炎（OA）的药物、生物制品和医疗器械的疾病状态，从而为其临床开发提供信息。
成立了一个由学术界和工业界代表组成的骨关节炎研究协会国际（OARSI）疾病工作组。该工作组于 2008 年春季至 2009 年秋季期间多次举行现场会议和电话会议，以达成基于共识和循证的回应。2009 年 12 月在一次公开论坛上提交了一份报告，并于 2010 年夏季被 OARSI 董事会接受。
制定了一个包含当前对该疾病理解的 OA 操作性定义。关节水平上的结构性变化与患者对 OA 的体验分别被定义为“疾病”和“疾病”。建议在未来的 OA 临床试验中评估这两个方面。目前对 OA 表型的认识不足被确定为未来研究的一个重要领域。
新 OA 治疗方法的临床试验设计和实施应考虑到疾病的异质性、目标关节的治疗相关结构性变化以及患者报告的结局。
