Research Project

Understanding Patient Preferences and Their Importance in the Medicinal Product Life Cycle: A case study of Myotonic Dystrophy and Mitochondrial Disorders.

Principal Investigators: Dr Grainne Gorman

Other staff members involved: Prof. Robert McFarland and Julie Murphy

Project Details

Presently, there is no consensus on how and when to best incorporate patient preferences into the decision-making processes that govern the development and testing of medicinal products. The IMI project PREFER (‘Patient Preferences in Benefit and Risk Assessments during the Drug Life Cycle’) launched in 2016 and will provide recommendations for different stakeholders.

In this case study, we will explore and elicit myotonic dystrophy and mitochondrial disorders patient preferences. We will assess patient and caregivers’ unmet clinical needs and attitude to risk related to neuromuscular treatments.

An initial set of interviews and focus groups will inform the larger quantitative study (i.e. online survey) to be conducted later in the year.

Overall, this study will inform decision-makers and other stakeholders interested in these diseases of the unmet clinical need priorities identified by patients and caregivers, as well as the treatment associated risks they would be prepared to undertake.

Contact:  [email protected]

Sponsor/funder: This work has received support from the EU/EFPIA Innovative Medicines Initiative-2 Joint Undertaking PREFER grant n° 115966.

Collaborators: PREFER has 32 partners. The consortium is co-ordinated by Uppsala University, and managed together with Novartis that acts as project leader.