Adaptive trials: the pathway for new treatments?



Standardised reporting and better knowledge exchange could be key to wider use of adaptive trials, according to our latest FORUM report.

In January 2019, the Academy, the MHRA, the MRC and NICE held a FORUM workshop exploring the use of adaptive clinical trials and the advantages they may have when assessing the safety and efficacy of new medicines and medical interventions.

Randomised controlled trials (RCTs) are the primary source of evidence for showing that medicines are safe and effective. While highly valued, RCTs can be expensive and time-consuming, which can have an impact on the end costs of new medicines. More adaptive and flexible approaches enable changes to be made to the trial as it progresses, allowing trials with unique parameters (such as those dealing with rare diseases or small patient populations) to be conducted quickly and efficiently.

Adaptive trials rely on sophisticated algorithms and statistics to simulate possible outcomes of a medical trial. As the potential outcomes of a trial are narrowed by the gathered data, adaptations are made to the trial design. This ensures that the trial can maximise its chances of answering its original research questions.

Adaptive trials can have multiple advantages over standard RCTs. They can allow for new drugs to be incorporated into a trial as they are developed, or to more quickly determine the safest and most effective dose. They can also ensure that the right number of patients are recruited into the trial as it progresses. This has the potential to both reduce the cost of the trial, and to allow results to be gathered more quickly.

Despite their potential benefits, adaptive trial designs are still not widely used. Our new FORUM report, ‘Adaptive trials – acceptability, utility and versatility’  highlights some of the key reasons why this might be:

  • Misconceptions that adaptive trials are more complex and expensive than standard RCTs and that regulators and funders are less willing to accept adaptive designs.
  • Cultural differences between industry, academia and the NHS result in a lack of knowledge exchange and collaboration, which is essential to innovation in adaptive trial design.
  • Funding mechanisms not flexible enough to account for the infrastructure and staffing required for adaptive trials.
  • Involving multiple sponsors in trials raises challenges for intellectual property and maintaining trial integrity.
  • A highly skilled and informed clinical research workforce, including statisticians and computer scientists, is needed through industry, academia and the NHS to allow widespread delivery of adaptive RCTs.

Whilst these reasons each represent a significant challenge to teams considering adopting an adaptive trial design, our report also highlights key ways in which these challenges might be tackled. These include:

  • Knowledge exchange: Through secondments or shadowing staff already running adaptive trials, researchers could gain key insights and experience into how adaptive trials work. Through such crucial skills exchanges, the pool of trained researchers can expand.
  • Dedicated infrastructure: By acquiring more funding for research proposing to use an adaptive trail design, we can encourage the use of such designs in future research. Also, developing algorithms that could be used by other researchers would help to alleviate costs for future adaptive trials.
  • Standards for reporting: Using a standardised reporting system for adaptive trials would allow for researchers to build upon each other’s work, creating a wider and more knowledgeable research base. Effective reporting could also help researchers using adaptive trails for the first time, allowing them to understand the challenges that others have faced to anticipate them in their own trial.

Adaptive RCTs are finding new uses and are likely to become widespread in clinical research. This could result in better medicines developed faster and at lower cost. Adaptive trials could have wide and varied uses within the clinical and non-clinical setting, bringing significant benefits for patients and the NHS.

The full report from the workshop is now available to view and download online.

The FORUM is the Academy’s neutral and independent platform for individuals across academia, industry, the NHS, Government, charity, regulatory and the wider healthcare sector to meet and take forward national discussions on scientific opportunities.

Find out more about the FORUM on our website.

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