Background: As clinical trials have limited enrollment, it is vital to establish objective methods for subject selection to address medical needs and to ensure equitable access to potentially life-altering therapeutic options.

Aims: To describe our preliminary steps in the development of an objective clinical decision-making process for the selection of pediatric patients with hemophilia for participation in therapeutic trials.  

Methods: This clinical decision-making process was developed using an iterative design with our inter-disciplinary team.

Results: The team identified need to structure the process with a numerical matrix in an attempt to minimize subjectivity. Random selection was also discussed. The first iteration identified areas of unmet clinical needs including venous access, bleeding, joint health. The second iteration included the addition of social and treatment support needs including home infusion and transportation. Following review, the team recognized the need to include patient safety measures such as adherence to treatment recommendations and attendance. The final step addressed barriers to participation. These barriers were identified in consultation with team members and Equity/Diversity Specialists for equitable inclusion of participants from marginalized or racialized communities, those who may experience reduced family medical literacy, interest in, access to or enrollment on previous clinical trials. Throughout this process the team recognized the importance of inter-disciplinary participation and the need for a defined structure to generate discussion.

Conclusions: This was our first attempt at developing a decision support process which included a numerical matrix followed by structured team discussion, to address equity for participation in pediatric clinical trials. Next steps will involve further consultation with Equity-Diversity Specialists and other stakeholders.  This approach is an ongoing iterative process and may be applicable to clinical trials in rare chronic diseases; other therapeutic areas may find random selection sufficient to mitigate selection bias and equity of opportunity.

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ISTH Congress Abstracts - https://abstracts.isth.org/abstract/development-of-decision-making-process-to-support-equitable-participant-selection-in-pediatric-hemophilia-trials/