Patient Preferences and the PREFER project

REFER is a project from the Innovative Medicines Initiative (IMI). "IMI is the world's biggest public-private partnership in the life sciences. See more

What is the PREFER project?

PREFER is a project from the Innovative Medicines Initiative (IMI). “IMI is the world’s biggest public-private partnership in the life sciences. It is a partnership between the European Union (represented by the European Commission) and the European pharmaceutical industry (represented by EFPIA, the European Federation of Pharmaceutical Industries and Associations).”

PREFER will establish recommendations to support development of guidelines for industry, Regulatory Authorities and HTA bodies on how and when to include patient perspectives on benefits and risks of medicinal products. The final recommendations from PREFER on patient preference studies coming in March 2022.

PREFER organised a webinar on patient preferences to present and discuss their results, below is our summary and you should definitely watch the recordings.

What are patient preferences?

As defined by the FDA, Patient Preferences means,

“Qualitative or quantitative assessments of the relative desirability or acceptability to patients of specified alternatives or choices among outcomes or other attributes* that differ among alternative health interventions”

*By attributes it is meant benefits, risks, uncertainties

Patient preferences are not to be confused with Patient Reported Outcomes, which is heath data collected directly from patients, without any comparison with the alternatives. Patient preferences can be used by Industry, Regulators, HTA/payers, academics, physicians, patients to inform decision-making regarding topics like benefit/risk assessment, labeling, early access, and developing relevant endpoints. In certain situations, patient preferences are incredibly sensitive and important, as for example, when multiple treatments exist and there is no option that is clearly superior for all patients.

What are patient preference studies?

Patient preference studies typically follow these steps:

  1. Design
  2. Conduct
  3. Communication of results

And include both quantitative (surveys to larger populations) and qualitative methods (literature review, focus groups, interviews)

Preference studies help all stakeholders understand patients’ views on what matters, how much it matters, and which trade-offs patients are willing to make. They can be used by physicians to create informed decision making tools to be used with patients to align decision with what is most important to them.

Here are some examples of patient preference studies from the PREFER project:

Patient Preferences to Assess Value IN Gene Therapies: Protocol Development for the PAVING Study in Hemophilia

Patient Preferences in Rare Diseases: A Qualitative Study in Neuromuscular Disorders to Inform a Quantitative Preference Study