Understanding individualised genetic interventions as research-treatment hybrids

Area of Science:

• Biomedicine
• Genetics
• Medical Ethics

Background:

• Millions suffer from rare and ultrarare genetic conditions with limited treatment options.
• The development of Milasen in 2019 marked a significant advancement in personalized genetic medicine.
• Milasen's unique application raised critical questions regarding its classification and oversight.

Purpose of the Study:

• To analyze the conceptual, ethical, and regulatory challenges posed by individualized genetic interventions.
• To determine the appropriate classification of therapies like Milasen within biomedical domains.
• To explore the implications of this classification for development, assessment, funding, and regulation.

Main Methods:

• Case study analysis of Milasen's development and administration.
• Examination of existing regulatory frameworks for human subjects research and clinical innovation.
• Conceptual analysis of individualized genetic therapies.

Main Results:

• Individualized genetic therapies, exemplified by Milasen, challenge traditional research versus treatment dichotomies.
• These therapies represent a "research-treatment hybrid" category.
• This hybrid classification has significant epistemological and pragmatic consequences.

Conclusions:

• At least some individualized genetic therapies should not be categorized strictly as research or treatment.
• Recognizing these therapies as research-treatment hybrids is crucial for appropriate oversight, funding, and regulation.
• This new understanding is essential for advancing personalized medicine ethically and effectively.