Patient preferences: why, how and when
Patient preferences can give us information that is critical for developing medical treatments. But they can also tell us how much risk patients think is acceptable for a given benefit. The methods to find out, or ‘elicit’ patients preferences are there, but decision makers are not sure how to assess and use them.
Most patients have to accept treatments that come with both benefits and harms. The pharmaceutical industry, regulatory authorities, health technology assessment bodies, reimbursement agencies and patient organisations all agree that their perspective, or what is known as ‘patient preferences’, needs to be part of decision making on benefit and risk. But how? When? And what are the regulatory requirements for preference studies?
There are many methods to elicit patient preferences and large amounts of scientific literature. What is missing is a structured approach that takes all these perspectives into account. The PREFER project will find out what the key stakeholders think is important.
FINDING OUT WHAT STAKEHOLDERS WANT
We will start with a literature review, interviews and focus group meetings with patient organisations, physicians, regulatory authorities, health technology assessment bodies, industry experts and academics on their key concerns, needs, expectations and desires on the assessment and use of patient preferences. This work will help us determine what needs to be addressed in the next step, where we conduct clinical case studies and simulations of preference studies. This work ensures that the final recommendations from this project addresses the issues all stakeholders raise.
IDENTIFYING METHODS AND CRITERIA FOR TESTING
The literature review will identify methods for preference elicitation, education and psychological measures methods. We will look at both quantitative and qualitative methods. The review will be based on a set of criteria to determine which methods are suitable at different decision points in the drug life cycle. The result will be a report on candidate methodologies and assessment criteria to go forward for testing in the next project step.
People working on methodology
The work on methodologies is led by Erasmus University Rotterdam together with Sanofi, in collaboration with Uppsala University and Janssen.
- Esther de Bekker-Grob, Public lead for methodology, Erasmus University Rotterdam
- Juhaeri Juhaeri, Private lead for methodology, Sanofi
- Ulrik Kihlbom, Public co-lead for methodology, Uppsala University's Centre for Research Ethics & Bioethics (CRB)
- Bennett Levitan, Private co-lead for methodology, Janssen R&D.
PATIENT REPRESENTATIVES IN METHODOLOGY WORK
The patient input in the methodology workstream is led by the International Alliance of Patients’ Organizations (IAPO) with representatives from the European Cancer Patients Coalition (ECPC), European Patients Forum (EPF) and Muscular Dystrophy UK (MDUK).
- Charis Chirvalaki, ECPC
- Ziede Burage, IAPO
- Kathi Apostolidis, ECPC
- Valentina Strammielloas, EPF
- Jenny Sharpe, MDUK
RESEARCHERS WHO ARE DOING THE WORK
- Karin Schölin Bywall, Uppsala University
- Mats G. Hansson, Uppsala University
- Ulrik Kihlbom, Uppsala University
- Ardine de Wit, UMC Utrecht
- Bas Donkers, Erasmus University Rotterdam
- Chiara Whichello, Erasmus University Rotterdam
- Esther de Bekker-Grob, Erasmus University Rotterdam
- Jorien Veldwijk, Erasmus University Rotterdam
- Maureen Rutten-van Mölken, Erasmus University Rotterdam
- Vikas Soekhai, Erasmus University Rotterdam
- Isabelle Huys, Katholieke Universiteit Leuven
- Rosanne Janssens, Katholieke Universiteit Leuven
- Gwenda Simons, University of Birmingham
- Karim Raza, University of Birmingham
- Marie Falahee, University of Birmingham
- Larissa Valormendez, University of Erlangen
- Gabriella Praveltoni, European Institute of Oncology
- Giulia Marton, European Institute of Oncology
- Ketti Mazzocco, European Institute of Oncology
- Laura Vergani, European Institute of Oncology
- Paolo Guiddi, European Institute of Oncology
- Geert Vander Stichele, MindBytes
- Sarah Verschueren, MindBytes
- Kathi Apostolidis, European Cancer Patient Coalition
- Cecilia Jimenez Moreno, Newcastle University
- Irina Cleemput, Belgian Health Care Knowledge Centre
- Andreas Brueckner, Novartis
- Mireille Muller, Novartis
- Martin Gebel, Bayer
- Stephen Heaton, Bayer
- Antje Tockhorn, Eli Lilly
- Becky Noel, Eli Lilly
- Greg Anglin, Eli Lilly
- Bennett Levitan, Janssen
- Eva Katz, Janssen
- Filip Mussen, Janssen
- Rachael DiSantostefano, Janssen
- Zhong Yuan, Janssen
- Tarek Hammad, Sanofi
- Juhaeri Juhaeri, Sanofi
- Vaishali Patadia, Sanofi
- Berkeley Phillips, Pfizer
- Leo Russo, Pfizer
- Stephen Watt, Pfizer
- Cathy Anne Pinto, Merck, Sharpe & Dome
- Jürgen Kübler, CSL Behring
- Oliver Guenther, Merck KGaA
- George Quartey, Roche
- Emma Du Four, Abbvie
- Esteban Herrero-Martinez, Abbvie
- Jerzy Tyczynski, Abbvie
- Kawaldip Sehmi, International Alliance of Patient Organisations
- Sonja Potenze, International Alliance of Patient Organisations
Assessing methods
PREFER started its work with a literature review, interviews and focus group meetings with patient organisations, physicians, regulatory authorities, health technology assessment bodies, industry experts and academics on their key concerns, needs, expectations and desires on the assessment and use of patient preferences.
Through clinical case studies
PREFER ran three core patient preference studies looking at lung cancer, rheumatoid arthritis and neuromuscular disorders. Both academic and industry partners have added studies to the PREFER portfolio that has helped cover different disease areas, methods and research questions.
.png)
Developing recommendations
By the middle of 2019, PREFER drafted recommendations and asked stakeholder advisory groups to test them to see how they work for other disease areas and decision points. Final recommendations were presented in 2022.
