Giving patients a voice in drug development


The patient perspective is important in all medical research, and particularly in drug development. This month, a public private research initiative called PREFER, is launched to assess when and how patient preferences on benefits and risks should be incorporated in decisions on medicinal products.

Drugs are developed for patients and there is an emerging consensus that they should be involved in determination of benefit-risk considerations during the life cycle of medicinal products. The patient voice is becoming increasingly important, not only for the companies that develop new therapies, but also for the authorities that assess, regulate and decide which drugs are effective, well tolerated and cost-effective for patients and the community.

Industry, regulatory authorities, health technology assessment bodies, reimbursement agencies and patient organisations are in agreement regarding the high value of patient preferences. However, there is little guidance on conducting and using such studies in pharmaceutical industry, regulatory and reimbursement decision environment. PREFER will provide a set of systematic methodologies and recommendations to assess, engage and include patient perspectives during the development, approval, and post-approval of new therapies.

Mats G. Hansson, Professor of Biomedical Ethics, Uppsala University
Mats G. Hansson, Uppsala University

PREFER is a five year project funded by the Innovative Medicines Initiative (IMI), to evaluate different preference elicitation methods and test them in a clinical setting.

“Almost all decisions taken in research, trials and approval somehow affect patients, which is why we involve patients directly as partners in the PREFER project. We have also created patient advisory groups to make sure the project gets input from a wider patient community.” says Mats G. Hansson, Professor of Biomedical Ethics at Uppsala University’s Centre for Research Ethics & Bioethics (CRB).

Connu Berlin, Global Head of Quantitative Safety and Epidemiology at Novartis Pharma
Conny Berlin, Novartis Pharma AG

“We have put a lot of effort into building close collaborations between academic and industry researchers, health and technology assessment bodies, payers, regulators and patients to investigate how patient preference studies can best address everybody’s needs. We believe that PREFER will provide recommendations that can support the development of guidelines for industry, regulatory authorities and HTA bodies for all medicinal products” says Conny Berlin, Global Head of Quantitative Safety and Epidemiology at Novartis Pharma AG.

PREFER will conduct patient preference studies in three disease areas where patient and clinical research partners have expertise: cancer, rheumatoid arthritis and neuromuscular disorders. Industry partners will provide additional patient preference studies, covering disease areas from their fields of expertise.

About the public-private research collaboration

Uppsala University and Novartis lead a new public-private research project "Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (PREFER)" under the Innovative Medicines Initiative (IMI). IMI is a partnership between the European Union’s Horizon 2020 programme and the European pharmaceutical industry (represented by EFPIA, the European Federation of Pharmaceutical Industries and Associations).

Mats G. Hansson, Professor of Biomedical Ethics at Uppsala University’s Centre for Research Ethics & Bioethics (CRB) and Conny Berlin, Global Head of Quantitative Safety and Epidemiology at Novartis Pharma AG are leading this public-private research collaboration.

The academic research is supported by a 6 million € grant from IMI. The pharmaceutical industry contributes the same amount as financial or human resources. The 33 partners include: 10 academic research institutions, 16 pharmaceutical companies, 4 patient organisations, 1 Health Technology Assessment body and 2 small and medium sized enterprises, all adding their experience and perspectives to the project.

Contact and more information:

To find out more, please visit the PREFER website:

Co-ordinator: Mats G. Hansson, Professor of Biomedical Ethics, Centre for Research Ethics & Bioethics (CRB), Uppsala University. E-mail:, phone: +46 18 4716197, mobile: +46 763 412050

Project Leader: Conny Berlin, Global Head Quantitative Safety & Epidemiology Novartis. E-mail:, phone: + 41 61 3248736, mobile: + 41 798451615


The Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (PREFER) project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 115966. This Joint Undertaking receives support from the European Union's Horizon 2020 research and innovation programme and EFPIA. The contents of this text reflects the PREFER project's view and not the view of IMI, the European Union or EFPIA.

PREFER partners:

Academic institutions: Uppsala University (Co-ordinator), Erasmus MC - University Medical Centre (EMC), Erasmus University Rotterdam, European Institute of Oncology, National Cancer Research Center G Paolo II, Bari, University Medical Centre Utrecht, University of Birmingham, University of Erlangen, University of Leuven, University of Newcastle upon Tyne

Patient organisations: European Cancer Patients Coalition (ECPC), European Patient Forum (EPF), International Alliance of Patients’ Organizations (IAPO), Muscular Dystrophy UK (MDUK)

HTA-Bodies: Belgian Health Care Knowledge Centre (KCE)

Small and medium sized enterprises: MindBytes, Steinbeißer Project Management

Pharmaceutical Industry: Novartis Pharma (Project Leader), AbbVie, Actelion Pharmaceuticals, Amgen, Astellas Pharma Europe, AstraZeneca, Bayer Pharma, CSL Behring, Eli Lilly and Company, Janssen, Merck KGaA, MSD, Pfizer. Roche, Sanofi, Takeda Development Centre Europe

Last modified: 2021-11-10