Developing recommendations
The point of PREFER is to develop evidence-based recommendations on how and when to include patient perspectives on benefits and risks of medicinal products. The aim is to support development of guidelines for industry, Regulatory Authorities and HTA bodies.
We begin by defining the scope of the recommendations and preparing operational requirements and best practices for conducting clinical case studies. By the middle of 2019, we will have drafted recommendations and asked stakeholder advisory groups to test them to see how they work for other disease areas and decision points. By the middle of 2021 we expect to have a refined draft. Final recommendations will be presented in the spring of 2022.
Our recommendations will be based on the results of our work with methodology and clinical case studies.
People developing PREFER recommendations
The work drafting recommendations is carried out by a large group of researchers from universities, industry, patient organisations and HTA bodies. Most of them have already been involved in the work on methodologies and case studies leading up to this.
The work is led by representatives from KU Leuven and CSL Behring, together with the Belgian Health Care Knowledge Centre (KCE) and Novartis Pharma.
- Isabelle Huys, Deputy coordinator and public lead for recommendations, KU Leuven
- Jürgen Kübler, Private lead for recommendations, CSL Behring
- Irina Cleemput, Public co-lead for recommendations, Belgian Health Care Knowledge Centre (KCE)
- Mireille Muller, Private co-lead for recommendations, Novartis
PATIENT INPUT TO RECOMMENDATIONS WORK
The patient input to the work on recommendations is led by the European Cancer Patients Coalition (ECPC) together with Muscular Dystrophy UK (MDUK) and the European Patients Forum (EPF).
- Antonella Cardone, ECPC
- Jenny Sharpe, MDUK
- Valentina Strammielloas, EPF
RESEARCHERS WHO ARE DOING THE WORK
- Ulrik Kihlbom, Uppsala University
- Ardine de Wit, UMC Utrecht
- Esther de Bekker-Grob, Erasmus University Rotterdam
- Jorien Veldwijk, Erasmus University Rotterdam
- Isabelle Huys, Katholieke Universiteit Leuven
- Rosanne Janssens, Katholieke Universiteit Leuven
- Elise Schoefs, Katholieke Universiteit Leuven
- Gwenda Simons, University of Birmingham
- Karim Raza, University of Birmingham
- Marie Falahee, University of Birmingham
- Ketti Mazzocco, European Institute of Oncology
- Geert Vander Stichele, MindBytes
- Sarah Verschueren, MindBytes
- Kathi Apostolidis, European Cancer Patient Coalition
- Klevisa Ceka, European Cancer Patient Coalition
- Aina Laura Errando, European Cancer Patient Coalition
- Juan Jose Ventura, European Cancer Patient Coalition
- Cecilia Jimenez Moreno, Newcastle University
- Grainne Gorman, Newcastle University
- Irina Cleemput, Belgian Health Care Knowledge Centre
- Nancy Thiry, Belgian Health Care Knowledge Centre
- Kate Adcock, Muscular Dystrophy UK
- Mireille Muller, Novartis
- Laurent Ecochard, Novartis
- Isabelle Naeije, Novartis
- Andrii Danyliv, Novartis
- Sheila Dickinson, Novartis
- Susan Talbot, Amgen
- Lucia D'Apote, Amgen
- Pujita Vaidya, Amgen
- Carmit Strauss, Amgen
- Taisa Paluch-Kassenberg, Astellas
- Stephen Heaton, Bayer
- Nicola Charpentier, Bayer
- Charalabos-Markos Dintsios, Bayer
- Montse Soriano Gabarro , Bayer
- Isabelle Stoeckert, Bayer
- Kim Quaintance-Lunn, Bayer
- Bennett Levitan, Janssen
- Tarek Hammad, Sanofi
- Juhaeri Juhaeri, Sanofi
- Mireille Collombat, Actelion Pharmaceuticals
- Monika Brand, Actelion Pharmaceuticals
- Berkeley Phillips, Pfizer
- Leo Russo, Pfizer
- Stephen Watt, Pfizer
- Annlouise Assaf, Pfizer
- Eline van Overbeeke, Pfizer
- Cathy Anne Pinto, Merck, Sharpe & Dome
- Emuella Flood, AstraZeneca
- Jürgen Kübler, CSL Behring
- Quazi Ataher, CSL Behring
- George Quartey, Roche
- Evan Davis, Roche
- Sohail Mulla, Roche
- Thomas Brookland, Roche
- Esteban Herrero-Martinez, Abbvie
- Valentina Strammiello, European Patient Forum
- Kawaldip Sehmi, International Alliance of Patient Organisations
- Sonja Potenze, International Alliance of Patient Organisations
- Meredith Smith, Alexion
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.
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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.
