Last week, the World Health Organization (WHO) introduced new global guidance to enhance the design, conduct, and oversight of clinical trials across countries of all income levels.
The initiative seeks to strengthen country-led research and development (R&D) ecosystems, accelerating access to new, safe, and effective health interventions that are both more accessible and affordable worldwide.
For the first time, WHO is offering comprehensive recommendations to national health authorities, regulatory bodies, funders, and other key stakeholders on optimizing clinical trials. The guidance addresses significant challenges such as poor trial design, lack of participant diversity, inadequate infrastructure, and bureaucratic inefficiencies, all of which contribute to delays and higher costs—factors that can ultimately cost lives.
Global inequalities in clinical trials remain a pressing concern. In 2022, there were 27,133 trials conducted in high-income countries (HICs) compared to only 24,791 trials in 131 low- and middle-income countries (LMICs). Often, LMICs are included in trials due to high disease burdens, yet the resulting data is frequently used to obtain health intervention approvals in HICs, leaving LMICs underserved by the very trials they contribute to.
“Strengthening country-led research and development, and embedding clinical trials into routine health services, will ensure faster and more equitable access to safe and effective treatments, improving global health outcomes,” said Dr. Jeremy Farrar, WHO’s Chief Scientist. “This new guidance promotes more diverse trial participation, moving away from a one-size-fits-all approach and ensuring broader representation in research.”
The lack of diversity in clinical trials has led to gaps in evidence, with less than 5% of trials in 2022 including pregnant women, and only 13% involving children. This underrepresentation can reduce the quality of evidence and hinder access to treatments for these groups, leading to cautious medical practices that may negatively affect their health. Moreover, this gap can make underrepresented populations less likely to trust and accept health interventions.
The guidance offers practical advice on designing trials to include pregnant and lactating women, recognizing their unique healthcare needs. It encourages involving at-risk populations from the earliest stages, emphasizing the importance of assessing safety first. Procedures for obtaining informed consent and assent are particularly crucial when trials involve children.
Another key recommendation is to prioritize patient, participant, and community engagement in the planning, execution, and dissemination of clinical trials. This approach aims to align research with public health needs and maintain trust in health systems.
WHO’s guidance also focuses on building stronger national R&D ecosystems, backed by sustainable financing, to promote better decision-making and faster access to health innovations. The aim is to create more effective national and global health research environments.
Developed in response to World Health Assembly resolution WHA 75.8, the guidance was the result of an extensive consultation process, involving nearly 3,000 stakeholders from 48 countries. It covers a wide range of health interventions, including pharmaceuticals, vaccines, diagnostics, nutrition, cognitive and behavioral therapies, preventive care, digital health approaches, and traditional or herbal treatments.
