Therapeutic Team Leader Psychiatry, Janssen Scientific Affairs, LLC, 1125 Trenton-Harbourton Rd-A32404, Titusville, NJ 08560
J Clin Psychiatry. 2014 Dec;75(12):1388-93. doi: 10.4088/JCP.13m08965.
Public health considerations require that clinical trials address the complex "real-world" needs of patients with chronic illnesses. This is particularly true for persons with schizophrenia, whose management is frequently complicated by factors such as comorbid substance abuse, homelessness, and contact with the criminal justice system. In addition, barriers to obtaining health care in the United States often prevent successful community reentry and optimal patient management. Further, nonadherence to treatment is common, and this reinforces cycles of relapse and recidivism. Long-acting injectable antipsychotic therapy may facilitate continuity of treatment and support better outcomes, particularly in patients who face these challenges. Clinical trials with classical explanatory designs may not be the best approaches for evaluating these considerations. We describe the design and rationale of a novel trial that combines both explanatory and pragmatic design features and studies persons with schizophrenia who face these challenges.
The Paliperidone Palmitate Research in Demonstrating Effectiveness (PRIDE) study is a prospective, open-label, randomized, 15-month study conducted between May 5, 2010, and December 9, 2013, comparing long-acting injectable paliperidone palmitate and oral antipsychotic medications in subjects with schizophrenia (according to DSM-IV criteria). Investigators and subjects had broad flexibility for treatment decision-making, thus making it a model that better reflects real-world practice. The primary end point was time to treatment failure, defined as arrest/incarceration psychiatric hospitalization; suicide; treatment discontinuation or supplementation due to inadequate efficacy, safety, or tolerability; or increased psychiatric services to prevent hospitalization. This end point was adjudicated by a blinded event monitoring board. Patients were followed to the 15-month end point, regardless of whether they were maintained on their initial randomized treatment. This article provides some of the reasoning behind the authors' choices when combining features from both explanatory and pragmatic approaches to this trial's design.
The PRIDE study incorporates real-world design features in a novel, prospective, comparative study of long-acting injectable and oral antipsychotics in persons with schizophrenia who have had recent contact with the criminal justice system. Insights provided should help the reader to better understand the need for more real-world approaches for clinical studies and how a broader approach can better aid clinical treatment and public health decision-making.
ClinicalTrials.gov identifier: NCT01157351.
公共卫生考虑要求临床试验解决慢性疾病患者复杂的“真实世界”需求。对于精神分裂症患者来说，这一点尤其正确，他们的管理经常因合并物质滥用、无家可归和与刑事司法系统接触等因素而变得复杂。此外，在美国获得医疗保健的障碍常常阻碍成功的社区重新融入和最佳的患者管理。此外，治疗不依从很常见，这加剧了复发和累犯的循环。长效注射抗精神病药物治疗可能有助于治疗的连续性，并支持更好的结果，特别是对于面临这些挑战的患者。具有经典解释性设计的临床试验可能不是评估这些考虑因素的最佳方法。我们描述了一种新型试验的设计和原理，该试验结合了解释性和实用性设计特征，并研究了面临这些挑战的精神分裂症患者。
棕榈酸帕利哌酮研究证明疗效（PRIDE）研究是一项前瞻性、开放标签、随机、15 个月的研究，于 2010 年 5 月 5 日至 2013 年 12 月 9 日进行，比较了长效注射型棕榈酸帕利哌酮和口服抗精神病药物在精神分裂症患者中的疗效（根据 DSM-IV 标准）。研究者和受试者在治疗决策方面具有广泛的灵活性，因此使其成为更好地反映真实世界实践的模型。主要终点是治疗失败的时间，定义为逮捕/监禁精神病住院；自杀；由于疗效、安全性或耐受性不足而停药或补充治疗；或增加精神科服务以预防住院。该终点由盲法事件监测委员会裁定。无论患者是否维持最初的随机治疗，都将随访至 15 个月的终点。本文提供了作者在将解释性和实用性方法的特征结合到试验设计中时的一些推理。
PRIDE 研究将现实世界的设计特征纳入了一项新颖的前瞻性比较研究，研究了近期与刑事司法系统接触的精神分裂症患者长效注射剂和口服抗精神病药物的疗效。提供的见解应有助于读者更好地理解临床研究中更现实世界方法的必要性，以及更广泛的方法如何更好地帮助临床治疗和公共卫生决策。
ClinicalTrials.gov 标识符：NCT01157351。
