PREFER Focuses on Patient Preferences

Kristin Bullok
Benefit-Risk Management Scientist, Global Patient Safety
Eli Lilly and Company


atient engagement in medical product development has become a priority across patient groups, regulators, and industry. Presentations and communications proliferate about what patient preferences are and why it is important to understand them. But patient preference research needs practical steps to realize its goal; namely, the systematic inclusion of patient preference information and data into medical product decision-making. The Innovative Medicines Initiative (IMI) project Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (PREFER) is helping in several major ways.

Engaging patients and their representatives in medical product research through patient preference studies is considered important. But at the same time, complex questions remain regarding methods used to elicit patient preferences and how to integrate these preferences into making research decisions. Previous projects and initiatives, including those from the FDA and EMA – plus earlier IMI projects such as Pharmacoepidemiological Research on Outcomes of Therapeutics (PROTECT), the European Patients Academy on Therapeutic Innovation (EUPATI), and the Medical Device Innovation Consortium (MDIC) – provide a solid foundation for developing such tangible recommendations.

PREFER is a five-year public-private research project working to develop evidenced-based recommendations on how and when to assess and use patient preference in medical product decision-making that can be codified into guidances for industry, regulatory, and health technology assessment (HTA) organizations.

Compared with previous initiatives, PREFER has taken a broader approach by engaging the multiple perspectives of all stakeholders (through four patient organizations, nine academic groups, sixteen industry organizations, one HTA body, two small/medium size companies, plus other scientific and regulatory experts), and by performing case studies within the project to inform its final recommendations.

Shifting Patient Engagement from Theoretical to Practical

PREFER project contributors are working to shift patient engagement (PE) from the theoretical to the practical; to illuminate and share how to make patient preference studies more scientifically robust; and to lay down a clear path for these studies throughout the medical product development lifecycle. Deputy Project Director Becky Noel, drawing from her public health background, is drawn to these practical approaches when considering policy needs. She knows, for example, that patient preference studies can assist quantitative benefit-risk assessments requiring the use of values and weights, like mutli-criteria decision-analysis (MCDA). “But where do the weights come from?” she asks. “In a time of increased transparency for patients, with their greater inclusion in drug development and benefit-risk decision-making, shouldn’t those values and weights come directly from patients?” These can come directly from patients through scientifically sound and representative patient preference studies.

Key stakeholders are systematically involved in PREFER and their feedback is incorporated throughout the project. Here are some ways in which academic and industry partners are working together and interacting with additional stakeholders:

  • Patients and representatives inform action on such practical aspects as communication within patient groups, and by participants in PREFER case studies.
  • A recent PARADIGM-PREFER collaboration provides additional ways to meaningfully engage patients from concept, to developing patient-facing materials, to communicating research results.
  • Regulatory authority and HTA representatives meet regularly with PREFER leadership through Scientific Advisory Board meetings to review empirical and simulation case studies; this input helps provide relevant study designs.
  • Non-PREFER scientific experts in collection and analysis of patient preference data, including the International Association for Health Preference Research (IAHPR), provide additional perspectives through their scientific and ethical advisory boards and review of academic case studies.

Four PREFER Work Packages

Within PREFER, four work packages (WP) closely collaborate to generate data and develop final recommendations.

  • WP1 provides strategic direction, project management, and communications support throughout the project, including development and maintenance of PREFER’s work after the project completes.
  • WP2 generates results to understand stakeholder needs through psychological instruments that investigate preference heterogeneity, and methodological questions of most interest to stakeholders.
  • WP3 develops, conducts, and compiles results from empirical studies by industry and academia, and simulated case studies, which aim to address stakeholders’ needs and answer prioritized questions related to the conduct and use of patient preferences studies in making decisions.
  • WP4 leads writing the final recommendations based on the entire project results.

Output to Date

PREFER began in 2016 and is already more than halfway complete. PREFER Research is already providing practical output for medical product decision-making. Its complete list of publications includes:

PREFER results to date, including two case studies and the influence that patients have on this project, will be presented in the session Patient Preferences in Decision-making and the PREFER Project: Past, Present, and Future on Wednesday, June 28 at the upcoming DIA Global Annual Meeting 2019 in San Diego.

From the Author

PREFER seeks to understand how to incorporate patient preferences in a representative and scientifically robust manner to support the systematic and consistent inclusion of these preferences in healthcare product development. This future state of consistent inclusion of patients and their preferences–by ALL stakeholders–continues to energize and increase my efforts within PREFER. The potential from practical outputs of PREFER, like recommendations on clinical study quality, checklists and other tools, supports the view that PREFER will ultimately lead to this future state.

The PREFER project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement N° 115966. This Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA.