Schlag Anne Katrin, Zafar Rayyan R, Lynskey Michael T, Athanasiou-Fragkouli Alkyoni, Phillips Lawrence D, Nutt David J
Drug Science, London, United Kingdom.
Department of Brain Sciences, Faculty of Medicine, Imperial College London, London, United Kingdom.
Front Psychiatry. 2022 Nov 3;13:1027159. doi: 10.3389/fpsyt.2022.1027159. eCollection 2022.
Randomised controlled trials (RCTs) have long been considered the gold standard of medical evidence. In relation to cannabis based medicinal products (CBMPs), this focus on RCTs has led to very restrictive guidelines in the UK, which are limiting patient access. There is general agreement that RCT evidence in relation to CBPMs is insufficient at present. As well as commercial reasons, a major problem is that RCTs do not lend themselves well to the study of whole plant medicines. One solution to this challenge is the use of real world evidence (RWE) with patient reported outcomes (PROs) to widen the evidence base. Such data increasingly highlights the positive impact medical cannabis can have on patients' lives. This paper outlines the value of this approach which involves the study of interventions and patients longitudinally under medical care. In relation to CBMPs, RWE has a broad range of advantages. These include the study of larger groups of patients, the use of a broader range and ratio of components of CBMPs, and the inclusion of more and rarer medical conditions. Importantly, and in contrast to RCTs, patients with significant comorbidities-and from a wider demographic profile-can also be studied, so providing higher ecological validity and increasing patient numbers, whilst offering significant cost savings. We conclude by outlining 12 key recommendations of the value of RWE in relation to medical cannabis. We hope that this paper will help policymakers and prescribers understand the importance of RWE in relation to medical cannabis and help them develop approaches to overcome the current situation which is detrimental to patients.
长期以来，随机对照试验（RCTs）一直被视为医学证据的黄金标准。对于基于大麻的药用产品（CBMPs）而言，这种对随机对照试验的关注导致英国出台了非常严格的指导方针，限制了患者的获取途径。目前人们普遍认为，关于CBPMs的随机对照试验证据尚不充分。除了商业原因外，一个主要问题是随机对照试验不太适合对全植物药物进行研究。应对这一挑战的一个解决方案是使用真实世界证据（RWE）以及患者报告的结果（PROs）来扩大证据基础。此类数据越来越多地凸显了医用大麻对患者生活可能产生的积极影响。本文概述了这种方法的价值，该方法涉及在医疗护理下对干预措施和患者进行纵向研究。对于CBMPs，真实世界证据具有广泛的优势。这些优势包括对更大规模患者群体的研究、使用更广泛的CBMPs成分范围和比例，以及纳入更多和更罕见的医疗状况。重要的是，与随机对照试验不同，还可以研究患有严重合并症且来自更广泛人口统计学特征的患者，从而提供更高的生态效度并增加患者数量，同时大幅节省成本。我们通过概述关于真实世界证据在医用大麻方面价值的12项关键建议来得出结论。我们希望本文将有助于政策制定者和开处方者理解真实世界证据在医用大麻方面的重要性，并帮助他们制定方法来克服当前对患者不利的局面。
