RESPONSIBLE THERAPIES FOR BLOOD DISEASES
Due to the possible dramatic improvement of the lives of patients and families with rare hereditary blood diseases, bringing iPSC-based therapies with curative potential comes with great responsibility towards donors, patients, clinicians and society as a whole. In line with the requirements of Responsible Research & Innovation (RRI), we introduce a normative framework of four criteria to guide the development and implementation of these therapies: the 4A framework.
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Our work contains connecting and contributing to the work of all the researchers within our project, who must make the therapies Available. We discuss with patients, families and their organizations, and the cell donors for whom the options must be Acceptable in terms of ethics and practicality. We talk to clinicians and other care providers for whom the therapies must be Accessible. Finally, we meet with the parties involved in decision making in health care policy for whom the therapies must be Affordable. By interviewing all these stakeholders, we aim to give meaning to the 4A framework and develop different scenarios about the application and implementation of the iPSC-based therapies.
Louisanne van Hooff is a PhD student working on the TRACER 3 project. She focuses on responsible research, and within that context creates an ethical and social normative framework to be incorporated in iPSC therapies for sickle cell anaemia and Diamond-Blackfan anaemia. She has a background in qualitative social sciences.