Public Consultation: New Guidance for Smarter Randomized Controlled Trials
Aisha Mazhar
Nick Medhurst
Martin Landray
Good Clinical Trials Collaborative
G

ood randomized controlled trials (RCTs) play a central role in generating the evidence needed to inform the development and implementation of health interventions. However, useful evidence from good RCTs is often lacking. This can be because the RCTs were never done, because those that were done failed to produce scientifically robust and clinically relevant answers, or because the results were never published.

The pandemic has particularly highlighted the need to be better at generating good-quality evidence more quickly. A prominent study, Trends in COVID-19 therapeutic clinical trials, estimated that only 5% of clinical trials for COVID-19 therapies were designed to be capable of producing robust results. It demonstrates the wasted effort and missed opportunity, resulting in delays or outright failure to identify effective interventions.

The Good Clinical Trials Collaborative (GCTC) has developed a new guidance to promote and enable good RCTs. The inclusive and broad-based guidance, which is available in five languages, will help researchers make RCTs smarter and better inform healthcare strategies. The collaborative is currently seeking input to shape the guidance through a public consultation until September 30. We invite you to get involved and share this opportunity with any organizations or individuals with an interest in RCTs.

Need for New Guidance

It must be made easier to conduct good RCTs that actually inform future care. One of the most significant hurdles to the design and execution of good randomized controlled trials is the overinterpretation of complex clinical trial guidelines. Too often there is undue focus on fixed rules rather than fundamental principles, process rather than product, and a tendency to mistake more documentation for better quality. Others have highlighted the variability in actual and perceived requirements.

But it is implausible that a single set of operational rules and checklists will suit all purposes. Rather, what is needed is new guidelines which are based on the underpinning scientific and ethical principles of randomized controlled trials. We need guidelines that genuinely guide – guidelines that allow flexibility to meet the needs of specific trials in specific circumstances and enable innovation so that the trials of tomorrow are better than the trials of today.

The Good Clinical Trials Collaborative

The Good Clinical Trials Collaborative (GCTC) was formed in June 2020 to develop new guidance to promote and enable good RCTs. It is supported by the Wellcome Trust, the Bill and Melinda Gates Foundation, and the African Academy of Sciences and has a global perspective that spans all health interventions (including drugs, vaccines, medical devices, digital technologies, and diagnostics).

The GCTC has brought together a wide range of individuals and organizations with an interest in and role to play in the design, delivery, analysis, and reporting of RCTs, and in implementing the results. This includes those who fund, regulate, design, deliver, or are responsible for RCTs, those who provide audits and quality-assurance functions, research organizations, ethicists, clinicians, participants, and lay health advocates. It includes those from a wide variety of sectors (industry, academia, government, charitable, nongovernmental organizations, participant and public groups) and settings (including higher and lower income countries around the world).

The new guidance has been developed to be:

  • Based on key scientific principles and ethical principles that really matter when it comes to designing, conducting, and regulating a RCT. The guidance is designed to enable those involved in RCTs to work out for themselves how to design and deliver their RCT in their setting.
  • Clear, concise, consistent, and proportionate to the context and setting in which RCTs are conducted, recognizing that there are risks associated with both usual clinical practice and a lack of reliable evidence on the effects of an intervention.
  • Forward looking, fostering innovation in health interventions and trial methods, including the appropriate use of routine healthcare data, technologies, and designs.
  • Flexible, widely applicable, usable, and durable across disease areas, intervention types, development phases, trial designs, geographies, and time.

The guidance aims to contribute toward a regulatory system that enables critical and rational thinking, in which trials are structured to meet standards of scientific and ethical excellence. The guidance focuses on what matters when designing, conducting, and regulating a RCT.

Get Involved

The Draft Guidance is open to public consultation until September 30. The consultation aims to strengthen the guidance and ensure that the final version is inclusively developed and applicable for a variety of settings. The draft guidance is currently available in English, and translations in Arabic, Chinese, Japanese, Spanish, and French will be available in the coming weeks. We invite you to provide input and would be delighted if you would share this opportunity with any organizations or individuals with an interest in randomized controlled trials and their role in improving health.

Through this guidance we hope to shape a future in which improvements in patient care and public health are driven by robust evidence from high-quality randomized controlled trials.

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