The value of real world evidence: The case of medical cannabis

Abstract

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.

Cannabis for endometriosis‐related pain and symptoms: It's high time that we see this as a legitimate treatment

“…Their appearance at the top of hierachies of evidence is inappropriate; and hierarchies, themselves, are illusory tools for assessing evidence.They should be replaced by a diversity of approaches that involve analysing the totality of the evidence base'. 20 As researchers who are undertaking randomised controlled trials in medicincal cannabis and endometriosis, we do not suggest that the current evidence is conclusive, nor does it supplant well-designed placebo-controlled trials. However, it is compelling and does raise some important questions around how evidencebased medicine (EBM) is currently being practised in Australia and New Zealand.…”

“…medical practitioners fail to include the clinical experience of fellow colleagues currently prescribing medicinal cannabis, or the evidence from patient's lived experience utilising cannabis therapeutically as being important in clinical decision making, simply citing a lack of evidence to justify their hesitation to prescribe. 20 If the practice of EBM is to be true to its guiding principles, then all three pillars need to be taken into consideration, particularly in diseases such as endometriosis where symptom management is often challenging. Given that current management strategies often lack effectiveness and/or have intolerable adverse effects for many, medicinal cannabis should be considered as a potential treatment option.…”

“…11,17,18 In a recent publication, the importance of considering the use of real-world evidence when it comes to medicinal cannabis was outlined, citing limitations associated with randomised controlled trials, and that evidential supplementation with long-term pragmatic studies, N = one study, case studies/series and basket protocols should more fully be investigated. 20 While we agree that well-designed, placebo-controlled trials, or comparative effectiveness trials are important and urgently needed in this area, they are by no means an essential prerequisite prior to prescription. In fact, there have been over 50 medicines licensed by the Food and Drug Administration and/ or European Medicines Agency between 1999 and 2014 without randomised controlled trial data.…”

“…In fact, there have been over 50 medicines licensed by the Food and Drug Administration and/ or European Medicines Agency between 1999 and 2014 without randomised controlled trial data. 20 Sir Michael Rawlins, former head of the Medicines & Healthcare Products Regulatory Agency and the National Institute for Health and Care Excellence (NICE), goes further, stating that 'Randomised controlled trials, long regarded as the "gold standard" of evidence, have been put on an undeserved pedestal. Their appearance at the top of hierachies of evidence is inappropriate; and hierarchies, themselves, are illusory tools for assessing evidence.They should be replaced by a diversity of approaches that involve analysing the totality of the evidence base'.…”

“…In a recent publication, the importance of considering the use of real‐world evidence when it comes to medicinal cannabis was outlined, citing limitations associated with randomised controlled trials, and that evidential supplementation with long‐term pragmatic studies, N = one study, case studies/series and basket protocols should more fully be investigated 20 …”