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‘’We should include diverse patient populations in first-in-human clinical trials.’’ That is what PREPARE researchers Lieke van Kempen, Nienke de Graeff and Martine de Vries from reNEW Leiden argue in a recently published commentary for BMJ Journal of Medical Ethics.
A growing number of regenerative therapies, including those developed by reNEW, are advancing to clinical trials. The question of who should be included in these early trials remains a subject of debate, often at the intersection of scientific priorities and ethical considerations.
A fifth criterium
In this context, a recent paper by Kögel et al. outlines an approach towards patient inclusion on the basis of four criteria. They specifically focus on first-in-human trials of cardiac xenotransplantation and recommend including patients who have (1) a high medical need, (2) the capacity to benefit from participating in first-in-human research, (3) have a ‘real’ choice between enrolling in the trial and alternative treatment options and (4) no clear record of non-compliance.
The PREPARE researchers agree with these criteria but advocate for a fifth criterion: the inclusion of diverse patient populations, particularly regarding sex, age, ethnicity, and socioeconomic background. They present two arguments for their claim.
Win-win situation
“By including diverse patient populations in first-in-human trials the potential risks and benefits of participation are more equally distributed, making the trial more ethically just,’’ says van Kempen. The researchers stress that excluding patients who are deemed ‘vulnerable’ should be avoided. van Kempen: “Instead of categorically excluding patients, researchers should design their trial in ways that would decrease patients’ vulnerabilities, granting them access to innovative treatments too.”
The authors then argue that including diverse patient populations will also strengthen the trials’ scientific value. “Including diverse populations in first-in-human trials enhances the scientific value of the data by assessing safety and efficacy amongst various populations, identifying potential differences in treatment response.’’
Progressive insight
The importance of including diverse patient populations is also illustrated by the historical effect of excluding women and ethnic minority groups from clinical trials. This has for instance led to large knowledge gaps in the context of cardiovascular disease and has had a huge impact on the care women and patients from ethnic minorities currently receive.
“Only when diverse patient populations are included in early clinical trials, we can prevent the same mistakes and ensure scientifically sound and ethically just advancements in regenerative therapies,“ concludes van Kempen.
