Ethics of gene and cell therapy development for neurologic disorders.

In this chapter, I provide a condensed overview of nine recurring policy and ethical challenges encountered with the development of gene and cell therapies for neurologic disease. These include the question of when to initiate first-in-human trials, the ethics and policy of expanded/special access, the conduct of individualized therapy trials, subject selection in trials, designing trials for negative results, unintended effects of interventions on personal identity, comparator choice in randomized trials, consent and therapeutic misestimation, and cost and access for effective therapies. Broadly speaking, I argue that early in their development, the justification of risk in trials of gene and cell therapies derives from the social and scientific value of a trial and not the therapeutic value for trial participation. This generates strong imperatives to justify, design, and report trials appropriately and select patient populations that incur the least burden and opportunity cost for trial participation. Late in intervention development, policy makers must contend with the fact that proven effective interventions will almost certainly amplify strains in healthcare budgets as well as the ethical justifications standing behind reimbursement decisions.