Clinical trials are how investigational drugs, devices, and other medical interventions are tested for safety and efficacy before being authorized for use. Individuals with life-limiting disorders – or, in the case of pediatric patients, the adults who care for them – may view these trials as offering the best chance of altering the trajectory of their conditions. Yet clinical trials are intended primarily as research, not treatment and they have restrictive criteria for participation. For some patients who are ineligible for clinical trials, non-trial access to the investigational medical products, including promising gene therapies, may be provided. But such non-trial access raises ethical concerns.
In this talk, Alison Bateman-House will review several of these concerns. First, how can we balance the needs of patients who may benefit from non-trial preapproval access with the need of a larger number of patients for expeditious conduct of robust clinical trials, in order to bring new medicines to market. Next, how can non-trial preapproval access be done equitably? Finally, how should we handle the thorny issue of access that occurs within families in which one affected sibling is eligible for a trial and another is not? The ethical problems raised by preapproval access to gene therapies for children with life-limiting disorders are not scientific problems but rather moral and social ones, with no clear-cut answers. Nevertheless, they are urgent questions, as gene therapy trials are underway for numerous life-limiting conditions and parents of the children unable to enroll in these are seeking non-trial preapproval access.