To find the safest and most effective medical treatments for illnesses, clinical trials are vital. In the year 2014/15 over 618,000 people took part in clinical research studies in the UK . Clinical trials offer the opportunity to ‘test’ medicines in humans to find out whether they work and allowing for direct comparison of different medicines that could be implemented to treat a condition. Trials also allow for assessment of potential side effects that a medicine might produce, meaning they can be evaluated for both the quality of medicinal effect and the coupled negative side effects.
There are serious risks associated with testing new, incompletely understood substances in humans and regulation of clinical trials needs to pre-empt and avoid any risk to those participating in research. Hosting clinical trials also offer member states the opportunity to gain access to innovative medicines whilst additionally promoting research in their nations. EU legislation is a useful tool to establish a level of safety and regulation that applies throughout the EU that ultimately facilitates the fastest evaluation of new and necessary medicines.
The first piece of European legislation to apply to all clinical trials occurring within the EU was the EU Clinical Trials Directive, implemented in 2004, which acts to protect and ensure the safety of patients who participate in clinical trials. The Directive is soon to be superseded by the EU Clinical Trials Regulation, which acts to amend issues with its legislative predecessor, by maintaining rigorous protection of patients, whilst promoting the EU as a location for clinical trials by streamlining the application process, and increasing transparency of trials by implementing EU-wide databases for recording clinical trials.
It was thought by some that the Clinical Trials Directive had a chilling effect on the number of clinical trials occurring in the EU because of disproportionate regulatory requirements. In an attempt to alleviate this regulatory burden the new Regulation offers eased guidelines governing certain clinical trials – such as in trials comparing the impact of two already authorised medicines for a condition – and improves the speed of setting up clinical trials.
Increased transparency in clinical trials is important. By improving access to clinical trial data (the new regulations will only apply to new trials) the EU has ensured that patients and physicians can evaluate the potential use of a treatment using all evidence, good and bad. Additionally, increased transparency will promote publication of null or negative results: when the expected outcome of the study does not occur, or the results are inconclusive. Publication of negative results is important, as they help to reduce redundant or dangerous repetition of investigation.
The UK’s membership of the EU and the automatic adoption of the Clinical Trials Regulation also fosters cross-border research within EU member states, through harmonisation of application and reporting procedures. The Regulation removes discrepancies between authorisation procedures meaning application is streamlined, and co-ordination of rules for the conduct of trials throughout the EU mean that studies can be harmonised and performed in parallel across institutions. A key benefit of this Regulation will be the ease of access to larger patient groups for clinical trials which arises as a result of being able to tap cross-border patient populations and initiate multi-institutional studies. This is especially important for rare disease populations, which are by definition thinly spread, as only a handful of people in every 10,000 will be affected. By expanding the size of the population from which we can draw patients with a particular condition – as would occur with multi-centre, cross-border studies – EU membership has enabled clinical trials for rare diseases to be more easily carried out, helping to promote investigation of orphan drugs and treatments for rare conditions.
Beyond providing the evidential basis for the efficacy of treatments for particular conditions, clinical trials provide an accelerated access to promising new treatments that may not be available from physicians. Thus, individuals with conditions that are currently without treatment are offered the opportunity to access innovative new interventions. Additionally, as a member of the EU, with the added potential benefits of existing under the Clinical Trials Regulation, it is likely more clinical trials will be drawn to the EU and the UK – increasing the volume of innovative and experimental treatments available. Indeed, the Alkaptonuria (AKU) Society in the UK, which supports those with AKU, a rare disease that causes severe, early-onset osteoarthritis, are part of a European wide consortium called DevelopAKUre, who are holding clinical trials for nitisinone as a potential treatment.
Currently AKU does not have a treatment and although some individuals have had access to nitisinone there is an enormous inequality of access in the UK and the rest of the world. DevelopAKUre is drawing on expertise from all over Europe, with partners assisting in provision of patient support, clinical trials sites, medical monitoring, drug supply and analysis. This co-operation throughout the EU is helping to deliver a clinical trial that may provide the first treatment for AKU. Critically, due to the size of the clinical trials, funding obtainable from individual consortium partner countries was insufficient to support the proposed plans; however, EU wide collaboration has permitted access to an EU grant awarded by the European Commission’s Seventh Framework Programme (FP7), establishing one of the first patient-led clinical trials. Thus, European Commission (EC) initiatives are not only promoting the clinical trial environment throughout EU member states but also providing opportunities for patient and academic research groups to hold clinical trials. This second point is especially important as this allows for greater variation in the organisations – currently predominantly pharmaceutical companies – supporting the authorisation of medicines, which in turn may influence variation in the clinical targets for trialled medicines and the motivation behind getting certain treatments to market. Thus, in instances where the potential uptake of a treatment may be small and as a consequence its profitability is low – as would be the case with medicines for rare diseases – it is still possible to establish a clinical trial for a treatment.
Clinical trials are integral for understanding the impact and efficacy of new and existing medicines for treating the myriad of conditions that affect people throughout Europe and the rest of the world. As a member of the EU, we present the UK is an attractive site for clinical trials, as part of a region filled with phenomenal research institutions providing unparalleled access to co-operative expertise, patient groups and patient populations, where individuals who kindly offer themselves to advance medicine do so in the safest environment.