The World Health Organization (WHO) has recently released new guidelines aimed at improving the design, conduct, and oversight of clinical trials worldwide. The guidance represents a significant effort to strengthen research and development (R&D) ecosystems across countries of all income levels, with the goal of ensuring more equitable access to safe and effective health interventions. While several of ESMO’s key recommendations have been reflected in the final document, significant gaps remain, particularly regarding the prioritization of cancer and rare diseases. Given the substantial and increasing global burden of cancer, stronger attention to oncology in the clinical trials landscape will be essential for moving forward.
Key Outcomes of the WHO Guidelines:
The new guidelines offer practical recommendations for national health authorities, regulatory bodies, funders, and other stakeholders on enhancing the quality and inclusivity of clinical trials. The document addresses several pressing challenges, such as:
- Improving trial design and participant diversity: The guidance acknowledges the underrepresentation of certain populations, including those with rare diseases, and stresses the need for more inclusive trials. It emphasizes the importance of diversity to ensure trial results are generalizable and applicable to groups with the highest disease burden.
- Strengthening country-led R&D ecosystems: The guidelines aim to embed clinical trials within routine healthcare settings to reduce global disparities in research and facilitate equitable access to new health interventions.
- Addressing infrastructure gaps and bureaucratic inefficiencies: By proposing measures to streamline clinical trial processes, the guidance seeks to reduce the time and cost associated with bringing new interventions to market.
Several of ESMO's recommendations are present in the final guidelines, with some notable alignments:
- Inclusivity and Addressing Underrepresented Populations: ESMO's call for greater inclusivity is well-reflected. The WHO guidelines explicitly mention the need to improve representation in trials for groups such as people with rare diseases, genetic subtypes, and those affected by high-burden conditions. The document recognizes the risks posed by limited diversity in clinical trials, which can result in less generalizable findings and inequitable access to interventions.
- Acknowledgment of Specific Trial Challenges in Oncology and Rare Diseases: While oncology is not a focal point, the guidelines do make references to oncological and rare disease trials, acknowledging the complexities involved. For example, they highlight the difficulties in enrolling large numbers of participants for rare disease trials and mention specific trial types, such as early-stage drug development or single-arm trials, which may apply to oncology and rare diseases.
ESMO notes with concern, however, that the guidelines have a limited focus on cancer, and stresses that cancer research and the unique needs of oncology trials should receive further attention, including in the implementation of the guidelines. It is also crucial to develop targeted incentives and dedicated frameworks for rare cancer trials. These areas remain critical for improved clinical trial practices. ESMO will continue to advocate for these essential aspects to become more prominent in R&D ecosystems globally to ensure that clinical trials better meet the needs of all patients, especially those with rare cancers.
For more information on the WHO guidelines and ESMO's ongoing policy efforts, please visit our ESMO public policy webpage.