Strengths and Limitations of Patient Preferences to Inform Regulatory Decisions

Baltimore, MD, USA—May 23, 2018—ISPOR, the professional society for health economics and outcomes research, held its third plenary session, “Examining the Role of Patient Preferences to Inform Regulatory Decisions,” this morning at ISPOR 2018 in Baltimore, MD, USA.

Healthcare research is increasingly seeking to better understand patients’ views related to treatment alternatives (eg, the relative desirability of harms, risks, benefits, etc). In this session, the panel debated the strengths and limitations of stated-preference research and the extent to which patients’ views on benefit-risk tradeoffs for medical therapies can be relied upon to inform regulatory decisions.

Shelby D. Reed, PhD, ISPOR President (2017-2018), Duke University, Durham, NC, USA moderated the session. Speakers included Jeff Shuren, MD, JD, Center for Devices and Radiological Health, US Food and Drug Administration, Silver Spring, MD, USA; Matt Reaney, MSc, Sanofi, Guildford, Surrey, UK; and Bennett Levitan, MD, PhD, Janssen Research and Development, Titusville, NJ, USA.

The US Food and Drug Administration (FDA) user fee agreements (MDUFA IV and PDUFA VI) require the FDA to develop systematic approaches to build fit-for-purpose tools to collect meaningful patient input that can be incorporated into regulatory review. Additionally, the Center for Drug Evaluation and Research (CDER) and the Center for Devices and Radiological Health (CDRH) have taken different but complementary approaches to incorporate the views of patients in research. CDRH has worked to understand quantitative preference elicitation methods to ascertain patients’ views about benefits and risks of medical products. In contrast, CDER has focused on qualitative preference elicitation methods.

Dr Shuren stressed that patients are at the heart of the FDA’s work. He noted that for many years medicine focused on the length of a person’s life and only later—after actually listening to patients—began focusing on their quality-of-life. FDA now captures patient input through patient preference information (PPI) and patient-reported outcomes (PROs). Dr Shuren pointed out that patients often have different views of the tradeoffs of a health intervention than regulators, but patients with dissimilar characteristics can also have different views on these tradeoffs. These differing viewpoints should be considered in decision making at the patient level.

Mr Reaney noted that PPI can be extremely relevant. There is a conflict, however, between stated preferences and patient experience, as it is difficult for people to imagine a context they have not yet experienced. He believes that PPI has its place, but that actual patient experience information should be considered when it is available.

Dr Levitan identified 3 types of PPI—attributes (what matters), relative importance (how much it matters), and tradeoffs (what tradeoffs patients are willing to make between benefits, harms, etc). He pointed out that it can be helpful to look at the gaps between clinician and patient beliefs, noting that one preference survey identified a large gap between clinician and patient caretaker beliefs on the importance of specific endpoints. Dr Levitan noted that while people can agree on facts, they often disagree on values. He closed by underscoring the goal for preference studies is to elicit and to better understand what those values may be.

Additional information on ISPOR 2018 can be found here. Released presentations from the conference can be found here. Interested parties can follow news and developments from the conference on social media using the hashtag #ISPORBaltimore.


ISPOR, the professional society for health economics and outcomes research (HEOR), is an international, multistakeholder, nonprofit dedicated to advancing HEOR excellence to improve decision making for health globally. The Society is the leading source for scientific conferences, peer-reviewed and MEDLINE®-indexed publications, good practices guidance, education, collaboration, and tools/resources in the field.
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