WHO Releases New Guidance to Enhance Clinical Trials and Improve Global Health Equity

Dr. Jeremy Farrar, WHO’s Chief Scientist, emphasized the importance of strengthening country-led R&D and integrating clinical trials into routine clinical and public health services.

The World Health Organization (WHO) has unveiled new guidance aimed at improving the design, execution, and oversight of clinical trials across all income levels. This initiative seeks to bolster country-led research and development (R&D) ecosystems, facilitating quicker access to safe and effective health interventions worldwide.

For the first time, the WHO offers targeted recommendations for national health authorities, regulatory bodies, funders, and others on optimizing clinical trial facilitation to generate robust evidence for health interventions. The guidance addresses significant challenges, including suboptimal trial designs, limited participant diversity, inadequate infrastructure, and bureaucratic inefficiencies that waste time, resources, and ultimately lives.

A critical issue highlighted is the stark disparity in clinical trial distribution between high-income countries (HICs) and low- to middle-income countries (LMICs). In 2022, there were 27,133 trials conducted in 86 HICs compared to only 24,791 in 131 LMICs. Often, LMICs are targeted for clinical trials due to their higher disease burdens, yet the data generated frequently supports the authorization of health interventions in HICs without benefiting the LMICs themselves.

Dr. Jeremy Farrar, WHO’s Chief Scientist, emphasized the importance of strengthening country-led R&D and integrating clinical trials into routine clinical and public health services. “This new guidance aims to improve the diversity of trial participants to ensure research benefits the broadest range of people possible, decisively moving away from a one-size-fits-all approach,” he stated.

Current statistics reveal a significant lack of representation in clinical trials, with less than 5% including pregnant women and only 13% involving children. This lack of diversity compromises the quality of evidence available, impacting care and access to necessary interventions. Underrepresentation can foster hesitancy among these groups toward health recommendations, further endangering their health outcomes.

To address these gaps, the guidance includes practical steps for establishing trials that adequately involve pregnant and lactating women, who have distinct healthcare needs. It emphasizes involving at-risk populations from the trial's inception and prioritizing their safety through comprehensive evaluations of similar interventions and expedited pre-clinical studies.

Patient, participant, and community engagement is stressed as essential to organizing clinical trials, ensuring that research aligns with public needs and preserves trust. Furthermore, the guidance seeks to strengthen national R&D ecosystems through sustainable financing, thereby enhancing decision-making processes and expediting access to health innovations.

Developed in response to World Health Assembly resolution WHA 75.8, this guidance emerged from an extensive and inclusive process involving nearly 3,000 stakeholders from various sectors across 48 countries. It encompasses trials for various health interventions, including pharmaceuticals, vaccines, diagnostics, nutritional strategies, cognitive and psychological interventions, preventive care, digital health approaches, and traditional or herbal treatments.