The Cancer Drug Development Forum (CDDF) held its annual conference in the Netherlands from February 3–5, 2025. Under the title “Challenges, Advances, and Open Questions in Global Cancer Drug Development and Clinical Trials,” the workshop focused on diversity and real-world evidence in anticancer clinical trials, taking very much a worldwide perspective.
The mission of the CDDF is to provide open-minded discussion among all stakeholders in oncology drug development—academics, colleagues from industry, regulators (eg, European Medicines Agency, Medicines and Healthcare Products Regulatory Agency, and the U.S. Food and Drug Administration), patient advocates, and payers—in Europe health technology assessors.
Challenges in Collecting Hard Evidence From the Real World
Despite the current global political climate, this workshop provided a series of excellent, frank discussions and positive recommendations for the future. The first session focused on the concept of diversity. It is well recognized that industry-led clinical trials tend to concentrate on selective patient populations to demonstrate statistically significant efficacy as rapidly as possible. Although such an approach may lead to a successful marketing authorization, the challenge for doctors, patient groups, and health technology assessors is that the subsequent real-world experience often involves a much more diverse population of patients for whom the outcome of a new intervention may be considerably different, both regarding efficacy and toxicity, compared with the results of the published trials.
“Diversity is at the heart of providing the best possible care for the widest population of patients in our various societies.”— JOHN F. SMYTH, MD
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We all seek to optimize and accelerate the process of licensing and access but recognize there are competing elements in the various stages involved. Doctors, patients, and especially health technology assessors emphasise that newly licensed medicines need to be relevant to the “real-world” situation, and there is increasing support for collecting good-quality, real-world data as soon as possible after a new medicine becomes available.
This CDDF workshop discussed some of the challenges in collecting hard evidence from the real world. These discussions overlapped with the diversity issue, since the process of collecting good-quality, real-world data is influenced by different patient populations—age, enthusiasm to take part, ability to access and use modern communication devices—essential for patient-reported outcomes.
Key Factors at Play in Clinical Trials
Our diversity discussions focused on the effects of age, sex and ethnic origins. Age is an increasingly important aspect of clinical trial design, recognizing an ever-increasing age of the cancer population, and the complications of age affecting organ function (eg, renal, cardiac, and hepatic). This adds to the reality of comorbid conditions that may require medication not for cancer.
There are many examples of differences in both efficacy and toxicity between the sexes, and it has long been appreciated that differences in outcome exist between different ethnic groups (eg, Black vs White, Asian vs European). All of these different factors need to be taken into account when evaluating a new cancer intervention.
This CDDF workshop was designed several months before the U.S. Presidential election. But, to put it mildly, the participants expressed their deep dismay at the announcement from the new administration that diversity initiatives were to be halted.
Surely we simply cannot ignore diversity. Many of us wish to preserve a global approach to cancer research. Recognizing the challenges only emphasizes the essential relevance of diversity, which is at the heart of providing the best possible care for the widest population of patients in our various societies.
DISCLOSURE: Dr. Smyth reported no conflicts of interest.
Dr. Smyth is Emeritus Professor of Medical Oncology at the University of Edinburgh and founding member of the Cancer Drug Development Forum.
Disclaimer: This commentary represents the views of the author and may not necessarily reflect the views of ASCO or The ASCO Post.
