Development of decision-making considerations to support equitable patient selection in paediatric haemophilia trials

Abstract Background Clinical trials for investigational haemophilia treatments such as gene therapy offer a potentially life-changing opportunity to those who are selected for enrolment. However, the number of enrolment slots available for these trials is often greatly exceeded by the number of eligible patients. Many of the strategies that are commonly used to select candidates for participation can be highly unsystematic, inequitable, and subjective. A more rigorous set of criteria is therefore needed to evaluate each candidate's suitability for trial participation in order to eliminate bias in selection and fulfill the ethical principle of justice. Aims To review current knowledge and issues in patient selection for paediatric haemophilia clinical trials with competitive availability, and to develop a more objective standard for decision-making that takes into account the needs of all involved parties. Methods A literature search on the ethics of trial participant selection and the practice of fairly distributing limited medical resources was conducted to identify previous literature and best practices in the area. A list of essential decision-making considerations was then designed to guide the selection of paediatric participants for haemophilia therapy trials through iterative group discussions between a diverse team of health professionals at McMaster Children's Hospital, Hamilton, ON, Canada. Results Current practices in resolving this ethical issue are highly heterogenous, although there are some common themes and recommendations. The three main criteria supported by the team and the literature search for inclusion in the considerations were: medical need, need for support, and potential safety considerations for the patient. Three measures for evaluating each criterion were developed and added for consideration during the decision-making process. The role of patient selection in meeting the scientific aims of the trial was also considered. Conclusion Attempting to create an equitable, systematic decision-making procedure for clinical trial participant selection involves a wide variety of competing values and ethical considerations, and discrepancies between recommendations are commonplace. The criteria presented here are intended to be used as a guideline to assist the equitable selection of paediatric patients for participation in haemophilia clinical trials with highly limited enrolment, although it may have some applicability to other areas of clinical research or therapeutic areas concerned with the allocation of scarce medical resources. Next steps should involve speaking with patients, community members and other stakeholders in order to include their perspectives. Assessment of medical need, potential support needs, and safety considerations form the basis of criteria for discussions around how to make enrolment in paediatric haemophilia clinical trials more equitable © Shutterstock