Institute of Epidemiology and Social Medicine, University of Muenster, Muenster, Germany.
MS Forschungs- und Projektentwicklungs-gGmbH, German MS Register, Hannover, Germany.
Mult Scler. 2021 Oct;27(12):1852-1863. doi: 10.1177/1352458520985118. Epub 2021 Jan 20.
Newly approved, drug-modifying therapies are associated with still unknown adverse events, although clinical trials leading to approval have strict inclusion and exclusion criteria and analyse safety and efficacy.
The aim of this study was to analyse the eligibility of multiple sclerosis (MS) patients treated in routine care into the phase III clinical trial of the respective drug.
In total, 3577 MS patients with 4312 therapies were analysed. Patients with primary-progressive MS were excluded. Inclusion and exclusion criteria of phase III clinical trials in relapsing-remitting MS were adopted and subsequently applied. A comparison in clinical and sociodemographic characteristics was made between patient who met the criteria and those who did not.
83% of registered patients would not have been eligible to the respective phase III clinical trial. Relapse was the single most frequent criterion not fulfilled (74.7%), followed by medication history (21.2%).
The majority of MS patients treated in routine care would not have met clinical trials criteria. Thus, the efficacy and safety of therapies in clinical trials can differ from those in the real world. Broader phase III inclusion criteria would increase their eligibility and contribute to a better generalizability of the results in clinical trials.
新批准的药物修饰疗法与尚未知晓的不良反应相关，尽管导致批准的临床试验具有严格的纳入和排除标准，并分析安全性和疗效。
本研究旨在分析在常规治疗中接受治疗的多发性硬化症 (MS) 患者进入各自药物 III 期临床试验的资格。
共分析了 3577 名 MS 患者的 4312 种治疗方法。排除原发性进展型 MS 患者。采用并随后应用复发缓解型 MS 的 III 期临床试验的纳入和排除标准。对符合和不符合标准的患者进行临床和社会人口统计学特征的比较。
注册患者中有 83%不符合各自的 III 期临床试验标准。复发是最常见的未满足标准（74.7%），其次是药物治疗史（21.2%）。
在常规治疗中接受治疗的大多数 MS 患者不符合临床试验标准。因此，临床试验中的治疗效果和安全性可能与实际情况不同。更广泛的 III 期纳入标准将提高其资格，并有助于提高临床试验结果的普遍适用性。
