Is the Clinical Trials Enterprise broken and how can we fix it?

A report of the inaugural JAMA Summit

Wien, 17.06.2024 – Text: Catarina Carrão

The inaugural Journal of the American Medical Association (JAMA) Summit 1 was held last October and focused on clinical trials, the basis of evidence-based medicine. It was meant to address the provocative questions: Is the Clinical Trials Enterprise Broken? And How Can It Be Fixed?

As such, an international group of invited clinical researchers, funders, regulators, policymakers, innovators, and industry representatives scrutinized and pondered the challenges of and potential solutions for the clinical trials enterprise. The discussions held at the two-day summit have now been published in a special communication 2, pointing to problems with the design and conduct of Randomized Clinical Trials (RCTs), as well as the issue of trial results not translating over to clinical practice, presenting solutions that need to be urgently overcome.

Siloed stakeholders

Has underscored in the publication, clinical trialists and clinicians have been working independently; and their separate missions, incentives, and infrastructures have been siloed which has resulted in inefficiency in the performance of trials and limitations with their scope and impact. Despite the 40,000 RCTs registered every year on, usually treatment guidelines released by the USA Preventative Services Task Force and by medical societies are often based in expert opinions and, in fact, few reference high quality RCTs in their recommendations.

Lack of translation

As stated by Angus et al (JAMA Network, 2024) 2, when randomized controlled trials (RCTs) include participants who do not reflect the broader patient population for a particular condition, the findings can be overly specific. The care instructions used within the RCT might not be incorporated into everyday clinical practice, and the results may not align with the priorities of patients and society. Conversely, RCTs can offer general insights that benefit the overall patient group but may be detrimental to specific subsets of patients. As such, the siloed nature of both clinical trial ecosystems and health care delivery systems means that nor clinicians are implementing RCT results, nor RCTs are answering questions that would lead to improvements in the clinical setting. Even when strong RCT evidence exists, it is frequently not followed in practice, which leads to poor quality of care. These issues are even more pressing given the lessons learned from the COVID-19 pandemic, where heightened awareness of the disproportionate impact of poor access to optimal care on vulnerable populations revealed an unprecedented opportunity for improvement. In fact, the platform trials that were conducted during the COVID-19 pandemic were embedded within clinical care and permitted the study of multiple therapeutic interventions simultaneously, with the results applicable to patient care in short order. These platform trials are examples of the types of novel approaches to clinical trials that are needed to realign and integrate all clinical trial stakeholders.

The four areas of improvement

The authors argue that four areas of improvement need to be urgently addressed, particularly in the USA ecosystem. First, there is a need for greater clarity to ensure appropriate regulation and oversight of science implementation, quality improvement, embedded clinical trials, and learning health systems. Second, study designs that improve statistical and logistical efficiency and lower the burden on participants and clinicians, allowing trials to be smarter, safer, and faster need to be widely adopted. Third, in order to use and integrate electronic health records more efficiently in RCTs, a wider adoption of standards and procedures is needed to guarantee that health data are sufficiently reliable and accurate so that it can be transferred responsibly and efficiently across various platforms and organizations. Fourth, financial and nonfinancial incentives are needed to align all stakeholders in clinical trials and healthcare delivery systems.


The future of clinical medicine will continue to be greatly influenced by the data derived from RCTs, but trials will need to be accomplished more quickly, efficiently, and cheaply than they are today. There is now an open opportunity to improve RCT design and best serve the needs of patients and clinicians.

Image: Stock Photo

Further Reading:

All references assessed 07 June 2024.



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