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

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