This is a pivotal moment for clinical trial regulations

Globally, clinical research is governed by guidelines established almost three decades ago. And Brexit is a looming issue for trials in the UK. Wellcome’s Policy team is working with partners to address both problems in two key pieces of work launched this week.

A man being vaccinated in an Ebola vaccine trial in Liberia

Credit: National Institutes of Health / Science Photo Library

There are growing concerns among the international research community about the existing clinical trial regulations.

Before a promising scientific discovery can become a new life-saving and life-enhancing treatment it must be tested in patients.

Each year thousands of patients across the world take part in clinical trials, accessing new interventions sooner and ensuring that they are safe and effective. It perhaps goes without saying that the trials themselves must be safe and effective.

But among the international research community, there have been growing concerns over whether existing regulations meet the needs of modern science and research in all countries.

Globally, most clinical research is governed by a set of guidelines first established almost three decades ago. They were primarily designed for the pharmaceutical industry and its time that we look to a create a new more inclusive system.

And, as we look to remove one set of barriers from clinical trials, another issue is on the horizon for those that take place in the UK – Brexit.

Wellcome’s Policy team have been working with partners to address both problems in two key pieces of work launched this week.

Bringing the community together to improve clinical research 

Researchers face a complex system of regulations, ethical guidelines and international standards when setting up clinical trials. When this works well, it creates a supportive environment for research by protecting people, building public trust and driving innovation. When it goes badly, it creates confusion, costs and delays.

This complex system is holding back clinical research, so to create a more supportive environment Wellcome is launching a joint initiative with the African Academy of Sciences and the Bill and Melinda Gates Foundation to develop new good practice guidelines for clinical research.

To develop the new guidelines we’re funding an independent team to develop the guidelines that will transform how clinical research is governed.

This new set of guidelines are meant to sit alongside those first established in 1990 by the International Council for Harmonisation (ICH) of Technical Requirements for Pharmaceuticals for Human Use. Its aim was to provide a single set of international guidelines to ensure that the rights, safety and wellbeing of subjects in trials testing new pharmaceuticals are protected, and that clinical trial data are meaningful.

But we’re concerned that the guidelines are being used more and more for studies that they weren’t designed to cover. For example, a study designed to look at what sepsis looks like in low resource settings needed to follow the ICH guidelines. These require the researchers to set out what the outcomes will be, but how can they researchers do this when the aim of the study is to uncover what isn’t already known?

Using guidelines when inappropriate increases the cost and burden of these studies, making the research less efficient. 

This is why we need to develop new guidelines that fill the vacuum, so that researchers use the right guidelines for the right study.

Working with the research community and patients will ensure that they are inclusive, transparent and adaptable to create an environment that ensures new treatments and interventions benefit patients quickly and safely.

To achieve this, we’re now looking for an inspiring and experienced leader to make this ambitious new initiative happen.

A looming challenge for UK clinical trials

But, while we are making plans to lift one barrier to clinical trials, we are concerned about another issue on the horizon for those taking place in the UK – Brexit.

At the moment the UK and EU share common regulations that makes trials easier across these borders. Together, we have the critical mass of expertise and patients needed to conduct robust trials – with a total population larger than the USA. The importance of this is shown by the current extent of UK and EU collaboration: 40 per cent of UK-based trials also have a site in the EU.

As the UK and Europe continue to discuss their future relationship, the final decision will have a huge impact on the future of clinical trials. In a report published earlier this week,  Brexit and Beyond: clinical trials [PDF 969KB], we set out a number of scenarios of what that relationship could and should look like.

Assessing these options, the best route is for the UK to have full participation in the EU clinical trials system on a similar basis to Member States. This would benefit the UK and EU, but it isn’t an easy goal to achieve. To make it happen, both sides will need to be pragmatic in negotiations. If this option isn’t possible, the UK should continue in close alignment in the short-term and then consult widely on the long-term options. 

In the event the UK leaves the EU without a deal, there will be uncertainty over the legal arrangements for clinical trials. This is likely to disrupt current and future trials, potentially discouraging new investment by international pharmaceutical companies. It's vital we avoid a no deal outcome.

International collaboration is central to modern clinical trials and it is becoming even more important that they reach more patients. We have to make this collaboration easier and not harder – shared rules and processes are an important way of doing this. We’re ambitious for what we can achieve by reducing barriers through the joint initiative on good clinical practice – and working hard to make sure that Brexit doesn’t create new ones.

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