This project develops design guidelines to responsibly govern the institutional and ethical aspects of local bedside development and in-hospital production of personalised medicines.

The availability and access of innovative therapies is decreasing, because they are more and more expensive. This project focuses on novel, high-tech solutions in local production of therapies for treatment of rare diseases in hospital pharmacies, known as ‘magistral biopharmaceutical production.’ It uncovers the relevant institutional and ethical aspects and aligns these in an early phase of design with the technological development of a proof-of-concept for this new approach to personalised medicine.

The affordability of innovative therapies for seriously debilitating diseases is increasingly putting pressure on the sustainability of our healthcare system. Personalised medicine development has been associated with improvements in terms of efficacy and safety, but also with increased costs.

New model for developing medicines

To deal with the issue of affordable and personalised medicine, this project studies a novel high-tech approach of bedside development and in-hospital production of medicines. This approach consists of an innovative configuration of miniaturised, flexible production of medicines, and data infrastructures that ensure swift data exchange on technological performance. The locus and nature of responsibilities is changing in this new model for developing medicines, leading to various regulatory, risk governance and ethical innovation challenges.

This project aims to study to what extent these personalised medicines can be developed while taking into account institutional and ethical issues from an early stage onwards. Questions posed include:

• How to evaluate and safeguard quality and safety issues;
• How to ensure adequate protection of patients ‘rights and interests through voluntary informed consent to treatment.

Preliminary results

So far the research team has identified several potential benefits and concerns and related enabling and impeding factors, which they are currently exploring further together with stakeholders. The researchers will work on the identification of the roles and responsibilities of the different stakeholders involved. Also, the views and attitudes of these stakeholders about the nature of the personalised medicine practice will be articulated. What are their ideas, problem definitions, operational and ethical objections and reflections?

Based on the information obtained the researchers will construct different, possible scenarios for the future of this new medicine practice, which will be used as input for discussion in focus groups to learn about (in)congruencies and to explore opportunities to align visions. They will also create a process map that presents these scenarios.

personalised medicine, rare diseases, hospital pharmacies, magistral biopharmaceutical production, magistral biopharmaceutical production, safety, informed consent, patient protection, affordable medicines, scenario study, cost reduction

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