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

Area of Science:

• Medical Ethics
• Personalized Medicine
• Rare Disease Research

Background:

• Individualized therapies for ultra-rare diseases merge clinical care and research.
• This blurs ethical lines and complicates the right to withdraw.
• Current guidelines view withdrawal as absolute, potentially causing scientific and societal loss.

Purpose of the Study:

• To propose a dynamic model for the right to withdraw in personalized therapy development.
• To re-evaluate withdrawal not as a final decision but an ongoing dialogue.
• To balance patient autonomy with the value of individualized therapies.

Main Methods:

• Reconstruction of the patient journey through therapy development.
• Analysis of evolving factors: bodily involvement, uncertainty, dependence, and resource intensity.
• Examination of how these factors influence the meaning of withdrawal.

Main Results:

• The right to withdraw evolves in meaning throughout therapy development.
• A dynamic approach acknowledges varying patient perspectives and motivations.
• This model facilitates patient engagement and early concern resolution.

Conclusions:

• Shifting to a dynamic right to withdraw is crucial for personalized therapies.
• This approach respects patient autonomy while preserving scientific and societal gains.
• It transforms withdrawal from a point of conflict into an opportunity for engagement.