Why We Need a Dynamic Right to Withdraw in Individualized Antisense Oligonucleotide Therapy Development

Individualized therapies for ultra-rare diseases blur the lines between clinical care and medical research, creating emotional and practical entanglements. These entanglements, in the absence of clear ethical guidelines, put pressure on the fundamental right to withdraw. Conventional ethical guidelines treat withdrawal as absolute, unconditional, complete, and immediate; yet, in personalized therapies, abrupt withdrawal can mean significant scientific and societal losses. We argue for a shift: a dynamic approach to the right to withdraw, treating it not as a one-time decision but as an evolving dialogue throughout therapy development. Through reconstructing the patient journey, we highlight how bodily involvement, uncertainty, mutual dependence, and resource intensity vary across development phases. As these aspects evolve, the right to withdraw assumes different meanings. Shaping the right to withdraw as a dynamic allows for the exploration of patient perspectives and motivations and for balancing patient autonomy with the social and scientific value of individualized therapies. Such an approach turns the concept of the right to withdraw into an avenue for patient engagement and addressing early concerns, preventing any unnecessary adverse effects of withdrawal.