Having worked on both sides of evidence-based medicine, previously in the laboratory researching cancer cell biology, and in my current role conducting statistical analysis and systematic literature reviews, I have seen first-hand the value that clinical judgement can bring to evidence-based medicine.
This article by Professor J D Spence1 piqued my interest, as it discusses how an overzealous application of evidence-based medicine can lead to inappropriate decisions that restrict patients’ access to treatments.
Health technology assessment bodies around the world require robust and comprehensive evidence to support the efficacy of a new treatment. Systematic literature reviews, which we conduct here at Mtech Access, are widely recognised as the ‘gold standard’ methodology to find this evidence. This methodology aims to gather relevant evidence for a given research question and evaluate the reliability of the evidence. This is just one area where evidence-based medicine plays a crucial role in a new treatment’s journey from the laboratory to the patient.
For some treatments, e.g. rare diseases, evidence may be limited or unavailable. Perhaps very few clinical trials have been conducted, or there are no existing treatments on the market for the disease in question.
In the article, Professor Spence, discusses a variety of scenarios where the inappropriate use of evidence-based medicine can lead to limited decision making. Two areas that stood out for me were:
- Withholding a therapy from patients whose disease subgroup was not included in the trial – I have seen strict applications of evidence-based medicine that have meant, for instance, that because a trial was conducted in adults, the results could not be said to apply to children aged 16–18, meaning that this patient group (teenage cancer) could not access the treatment. The article cites a similar example for elderly patients.
- Misunderstanding of the data by those with little clinical expertise can lead to the use of clinically irrelevant endpoints or effect sizes that are clinically meaningless. Blind acceptance of the numbers from complex analyses, without clinical input from experts in the field, can lead to conclusions that do not reflect the treatment’s true efficacy, or the patient experience.
The article concludes that whilst evidence-based decision making is critical in ensuring patient safety and the development of effective treatments, it should be tempered by clinical judgement. Going forwards it is important to debate whether clinical judgement can help to fill evidence gaps for diseases that are unlikely to receive funding for clinical trials. Is it ever appropriate to assume evidence is transferable?
This balance of evidence and clinical common sense, in the interest of patients, is something that I have looked to investigate throughout my career. It is a passion I share with my new colleagues across Mtech Access. Joining in the summer, I was delighted to learn more about our links with healthcare professionals and how our team draw on these throughout the market access and reimbursement process.
Via our network of contracted associates from across the NHS and other healthcare settings, we are able to engage clinical experts, currently working with patients, who can review the evidence generated by our systematic review team and provide thoughtful insight in the presence of evidence gaps.
It is highly valuable to have this resource on tap, so we can ensure our evidence base is as robust as possible, combining the best of evidence-based medicine with clinical insight.
- Spence JD. The need for clinical judgement in the application of evidence-based medicine. BMJ Evidence-Based Medicine 2020; 25:172-177. http://dx.doi.org/10.1136/bmjebm-2019-111300