By Silke Schoch, Senior Associate, Programs

On April 18, Dr. Pauline McNulty, Vice President of Patient-Reported Outcomes and Dr. Bennett Levitan, Senior Director of Benefit-risk Assessment at Johnson & Johnson presented during, Measuring Patient Experiences: Distinguishing Between Patient-Reported Outcomes (PROs) and Patient Preferences – the latest in our Clinical Outcome Assessment (COA) webinar series. Their presentation introduced the crucial differences between patient-reported outcomes (PROs) and patient preferences in both definition and application.

PROs and patient preference studies are two different, but complementary ways of capturing patients’ experiences. PROs provide insights on how patients feel and function in their everyday lives, whereas preferences reflect what patients choose when presented with different options (e.g., treatment attributes or desired outcomes). PROs reflect how a patient feels or functions and they are reported directly by the patient. On the other hand, patient preferences measure what matters to patients, how much it matters, and what tradeoffs patients are willing to make between benefits, harms, and other aspects of treatment.

Patient-Reported Outcomes

• Patient Preferences
• PROs reflect what actually happened to a patient when on a treatment
• Preference reflecs how patients believe they would react to a treatment
• A PRO can describe how patients felt or reacted to a treatment they experienced
• But, a PRO cannot describe how patients will react to a treatment they have not experienced
• A preference study can be conducted among patients who have not used a treatment (the treatment may not yet exist)
• But, a preference study does not tell how people responded to a treatment even when done with those who had the treatment
• PRO measures are included as endpoints in clinical trials
• Preference surveys generally cannot be trial endpoints – and are often conducted in panels outside trials
• PRO instrument development can inform the design of a patient-preference study
• Preference studies can inform the methods for scoring a PRO instrument

Understanding the difference between these two different types of data is key for the patient community as regulators and other health care stakeholders are increasingly interested in both to inform their decision-making. For example, the FDA’s Center for Devices and Radiological Health (CDRH) released guidance on patient preference information in 2016. Later this year, FDA’s Center for Drug Evaluation and Research (CDER) will release draft guidance on PROs as part of their Patient-Focused Drug Development Initiative. The patient community plays a key role in developing both PRO and preference studies.