Patients receive a voice in drug development

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Researchers participate in European research project with patient preference study

The patient perspective is important in all medical research, and particularly in drug development. This is reflected in the European research initiative Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (PREFER) in which a research group led by Dr. A. Huber at the Department of Medicine 3 is involved. The aim of the initiative, which has received both public and private funding, is to collect information on patients’ preferences and expectations with regard to the benefits and risks of medications for inclusion in guidelines on drug development for industry, regulatory authorities, and assessment bodies. The research group based at the Department of Medicine 3 is supporting the initiative by developing and conducting studies of preferences among patients who have or are at risk of developing rheumatoid arthritis.

Drugs are developed for patients and there is an emerging consensus that they should be involved in the identification 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 organizations are in agreement regarding the importance of patient preferences. However, there is little guidance on how to conduct studies on this topic that can be used in the pharmaceutical industry, or in decisions on regulations and reimbursement policies. PREFER aims at addressing this by providing a set of systematic methodologies and recommendations for assessing patient perspectives and including them during the development, approval, and monitoring of new therapies.

The working group based at Universitätsklinikum Erlangen will contribute to the project by conducting a clinical study of patient preferences among patients who have or are at risk of developing rheumatoid arthritis. Patients with chronic inflammatory rheumatic diseases are usually treated with medication, which is often expensive, throughout their lives. In light of this, it is particularly important that patient’s expectations with regard to the effect and success of medication, as well as their acceptance of potential risks and side effects, are understood. Dr. A. Hueber, head of the working group, explains:

We cannot improve the process of drug development and use without considering patient opinion. The aim of our project is to test the methodologies and recommendations developed as part of PREFER in a clinical study.

Project co-ordinator M. G. Hansson, Professor of Biomedical Ethics at Uppsala University’s Center for Research Ethics and Bioethics (CRB), continues explaining:

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.

The PREFER project has received six million euros of funding for a period of five years from the Innovative Medicines Initiative 2 Joint Undertaking. This Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation program and the European Federation of Pharmaceutical Industries and Associations (EFPIA). The pharmaceutical industry is matching the amount of financial and human resources provided by the Innovative Medicines Initiative 2. The 33 partners involved in the project include 11 European universities, 16 pharmaceutical companies from Europe and the USA, 4 patient organizations, and 2 small and medium-sized enterprises.

Further information

Dr. A. Hueber, PhD

Tel.: +49 9131/85-39109

PREFER partners


  • Uppsala University (Project coordination)
  • Erasmus MC – University Medical Centre (EMC), Erasmus University Rotterdam
  • European Institute of Oncology, Istituto Tumori ‘Giovanni Paolo II’
  • University Medical Centre Utrecht
  • University of Birmingham
  • University of Erlangen
  • University of Leuven
  • University of Newcastle upon Tyne

Patient organizations:

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

Further partners:

  • Belgian Health Care Knowledge Centre (KCE)
  • MindBytes
  • Steinbeißer Project Management

Pharmaceutical industry:

  • Novartis Pharma (Project management)
  • AbbVie
  • Actelion Pharmaceuticals
  • Amgen
  • Astellas Pharma Europe
  • AstraZeneca
  • Bayer Pharma
  • CSL Behring
  • Eli Lilly and Company
  • Janssen Pharmaceutical
  • Merck KGaA
  • Merck, Sharpe & Dohme
  • Pfizer. Roche
  • Sanofi
  • Takeda Development Centre Europe