The World Health Organization (WHO) has released a series of papers in The Lancet detailing the work that needs to be done to run more efficient clinical trials on a global scale, building on earlier guidance.
The organisation announced the Clinical Trials in Global Health series during a webinar held on Friday 28 March, which includes six research papers that delve further into recommendations made in the WHO’s “Guidance for best practices for clinical trials” released in September 2024.
The WHO’s guidance focuses on several key areas including global “research waste” and diversity in clinical trials, with the guidance going beyond race, gender and sexuality.
The webinar heard from the authors of each paper as they discussed the need for greater support to ensure efficient and effective research on a global scale.
Underrepresentation at heart
One of the key themes of the WHO guidance is ensuring underrepresented communities have greater access to clinical trials, including those from low- and middle-income countries, paediatric patients, and pregnant and lactating mothers.
Dr. Lillian Mutengu, programme manager for the Science for Africa Foundation, and is one of the authors of the paper “Better engagement, better evidence: working in partnership with patients, the public, and communities in clinical trials with involvement and good participatory practice”.
The paper discusses the need for harmonisation between stakeholders in clinical trials to try and improve accessibility to research in low- and middle-income countries.
Mutengu said that for principal investigators (PIs), the paper recommends they provide leadership and ensure that patient, public and community engagement is embedded in operations. From a sponsor perspective, Mutengu said that she recommends they provide training, planning and requirements for monitoring and stakeholder engagement.
Dr. Martina Penzzato, who leads the Global Accelerator for Paediatric Formulations in the Research for Health Department of WHO’s Science Division, and one of the authors of “Strengthening the paediatric clinical trial ecosystem to better inform policy and programmes” said that clinical trials that focus on children in their early stage of life remain rare.
The key takeaway from the research is that there needs to be alignment across global, regional and national priorities to work on the most pressing needs in paediatric health, especially in low- and middle-income countries.
“Researchers, policymakers, funders and communities, of course, are the centre. As we move forward, I guess I’d like to encourage everyone to reflect on your role in this collective effort and whether you are a researcher who is designing a new trial or a policymaker developing the necessary frameworks – more contribution will indeed help shape the future of child health research,” said Penzzato.
Dr. Teesta Dey, WHO Medical Consultant in Maternal and Perinatal Health, spoke about the research paper “Advancing maternal and perinatal health through clinical trials: key insights from a WHO global consultation”. The paper aims to improve research access into pregnant and lactating women. Dey said that currently, 98% of medicines that patients rely on during pregnancy have not been tested for safety or efficacy in pregnancy.
“We really don’t have enough data to know whether many medical products are safe or even if they work during pregnancy. This puts a huge burden on both the healthcare providers and women who have to make decisions about the risks and benefits of treatment,” said Dey. “Without having the data to guide them, it means that pregnant women may end up getting suboptimal care, use medications off label or take medicines without knowing if they’re safe or effective.”
Strong primary care can bolster research
Another theme from the WHO guidance was the importance of primary care in the research setting. Dr. Chris Butler authored one of the Lancet papers on this topic. “It is estimated that about 75% of health gains from attaining suitable development goals could be achieved through primary care. Primary healthcare is a whole of society approval, it addresses the broader determinants through multi-sector policy and action, and it empowers individuals to take charge of their own health,” Butler explains.
The University of Oxford professor helped author the paper, “Democratising clinical trials research to strengthen primary health care”. He said there is a need for a global primary care alliance to ensure all patients are able to gain access to research and to strengthen research on a global scale.
Transparency and collaboration needed from stakeholders
The final two papers focused on the need for transparency, firstly from sponsors reporting clinical trial data, and secondly from regulatory agencies to ensure there is alignment globally.
Dr. An-Wen Chun spoke about the paper “Reporting summary results – recommendations perspectives and importance on how to report trials”. He stated how only half to two-thirds of clinical trial results are published in journals and fewer than half are published in public registries.
Chun laid out three ways that trial reporting should be made more transparent. “Firstly, investigators and sponsors should ensure that the results for all of their clinical trials are reported in trial registries within 12 months and this timeframe aligns with the 2015 WHO statement on public disclosure of results in registries.
“Secondly, we call for the funders of registries and governments to ensure that these platforms are adequately resourced to implement the new WHO guidance with structured data fields and adequate quality control measures and the WHO trials registry platform is working with registries internationally and nationally to implement this new guidance.
“And thirdly. Relevant parties including relevant parties including funders, journals and regulators should implement policies requiring adherence to the new WHO guidance and the reporting of the eight new WHO recommended items related to summary results in registries. Only with these actions can we ensure public access to a complete body of evidence from clinical trials to inform patient care and global health,” Chen said.
Finally, Dr. Marco Cavaleri from the European Medicines Agency (EMA) spoke about a paper focusing on ethics and regulation. The paper, entitled “A roadmap for fostering timely regulatory and ethics approvals of international clinical trials in support of global health research systems”, investigates how regulators should be trying to align themselves to assist with sponsors running global studies.
“One important aspect that we discussed in order to advance the entire framework is the concept of a single ethics committee per country because this will really streamline how ethics committee will come up with decisions around approving clinical trials in each individual country,” Cavaleri said. “We fully recognise that there is still a role for local ethics committees, but we should not duplicate efforts here. We should look into simplification, into having a process that is more streamlined and able to deliver the type of review that is needed to protect participants.”
The WHO has spoken out recently about the need for more funding into certain research efforts including tuberculosis (TB) after the US announced its withdrawal from the organisation as well as a funding pull of United States Agency for International Development (USAID) which supported research into TB, human immunodeficiency virus among other disease globally, with most funding being directed toward low- and middle-income countries.
