PREFER: first two years

2018-11-12

The PREFER consortium met in Leuven on October 25-26 this year. Celebrating our achievements so far, and outlining strategies for years to come. We used the opportunity to plan the next big step: our upcoming clinical patient preference studies! 

Two keynote speakers set the stage for a panel discussion about how patient preference studies can help include the patient perspective in decision-making. Peter Mol from the European Medicines Agency gave a regulator’s perspective on using patient preferences in decision-making. Followed by Nathalie Seigneuret from the Innovative Medicines Initiative, who spoke about the importance of sustainability and impact.

The panel consisted of Nele Caeyers (patient expert), Rachael Ward (patient), Margaret Varley (caregiver), Peter Mol (regulator), Marco Petschulies (HTA), Nathalie Seigneuret (funder), and Conny Berlin (industry). The main message from the panellists was that patient preference studies can be valuable, if conducted in a robust and valid way. And that people who take part in them need to have support, and also should be informed about how their contributions have been used.  

Patient preference studies can tell us what patients want from their treatment. This can guide researchers deciding which medical products they should continue to develop. Patient preference information could also indicate what to measure in clinical trials. The panel agreed that supporting patients and research participants should include educating them about the expectations, scope, and terminology used in the study. 

PREFER is one of many initiatives on including patient perspectives in medical product development and market access. Another Innovative Medicines Initiative (IMI) project, working with patient engagement, is PARADIGM. In the spirit of collaboration, PREFER and PARADIGM have signed a memorandum of understanding, outlining how the projects will collaborate and share ideas. To celebrate this agreement, we invited PARADIGM coordinator Mathieu Boudes, who spoke about their approach to giving patients a voice. Together, PREFER and PARADIGM will share efforts to support patient engagement.

We are now at a stage where we can start planning for our recommendations on when and how to include patient preferences in medical product decision-making. We have identified the needs and concerns of patients, physicians, pharmaceutical industry, HTA bodies, payers, regulators, and academic researchers. Based on what they told us, we identified a number of research questions. The next step is to explore them in case studies on three patient groups: people with rheumatoid arthritis, lung cancer, and neuromuscular disorders.

However, three studies alone cannot satisfy all the needs and concerns of our stakeholders. Therefore, during the meeting, PREFER issued a call for project partners to contribute studies from their own companies and organisations. So far, three private partners (Merck Sharp & Dohme, and Pfizer with Eli Lilly and Company) have answered, and are contributing two case studies. And more are coming!

At the end of the meeting, attendees brainstormed about different helpful and meaningful ways to continue our exchange with the pharmaceutical industry, regulators (from Europe and the US), HTA bodies, payers, reimbursement agencies, patient organisations and the IMI. To make sure our stakeholders understand the value of knowing when and how to use patient preference studies, and for PREFER to understand the needs and concerns of stakeholders, we need to continue listening. And for our work to be meaningful, we must continue creating with, and for, stakeholders.

By Josepine Fernow & Kristin Bullok

PREFER consortium members at the second annual project meeting in Leuven, October 2018
PREFER consortium members and stakeholders at Leuven meeting. Photo: Anna Holm