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  1. Advancing Regulatory Science

Methods to capture post-market patient preference information

CERSI Collaborators: Mayo Clinic: Markus Bendel, MD; Juan Britto, MBBS, MSc (PI); Elizabeth Golembiewski, PhD; Ian Hargraves, PhD (PI); Victor Montori, MD, MSc; Nilay Shah, PhD; Angela Sivly, CCRP; Nafisseh Warner, MD
Yale University: Joseph Ross, MD, MHS; Erica Spatz, MD, MHS

FDA Collaborators: Susan Bersoff-Matcha, MD, PhD; Kaveeta Vasisht, MD, PharmD

CERSI In-Kind Collaborators: Montserrat León (Universitat Autònoma de Barcelona, Barcelona, Spain)

Project Start Date: October 16, 2019
Project End Date: July 31, 2023

Regulatory Science Challenge

Patient preferences may strongly influence the use of medical products after they are approved for market. While the FDA encourages the incorporation of patient preference information in the regulatory process, it is mostly incorporated in the pre-market evaluation process. Preferences that patients use in situations where they need to make actual treatment decisions to adopt (or not) a marketed medical product may be different than their preferences in circumstances where there is no need for, or possibility of, adopting the product. Identifying preferences that affect patients’ decisions to use a medical product is necessary for patient’s priorities to influence regulatory processes. Methods for identifying post-market decisional preferences are still early in development.

Project Description and Goals

This project aimed to conduct an initial assessment of three methods for identifying patient preferences present in decision making visits with a clinician (an encounter): a direct from encounter (DFE) method–a third party observing and analyzing the encounter; and two immediately post-encounter methods (IPE)–interviewing patients immediately after decision making conversations with clinicians, and a self-reported survey completed immediately after the encounter. The goal of this project was to provide initial real-world evidence of the strengths and weaknesses of DFE and IPE patient preference identification methods that can be used to inform future development of these methods. The study compares the number and nature of preferences identified through each method, the certainty that identified preferences reflect actual patient preferences, the effort required to employ each method, the ability of each method to account for varying clarity of preference expression, and patient gender related differences in preference identification. A preference is considered as a verbal or textual indication that some decision related topic may be desirable or undesirable in some way.

Goal 1: Design interim DFE and IPE methods and instruments, iteratively refined in real-world decision-making contexts.
Goal 2: Characterize differences between DFE and IPE methods when they are used to identify real-world patient preferences at play in making decisions to use marketed medical products.

The decision of whether to use a spinal cord stimulator (SCS) for management of chronic pain was used as a case, utilizing adult outpatient consultations or education visits at the Pain Clinic at Mayo Clinic, Rochester MN. For Goal 1, 30 patient-clinician encounters were used to develop, and field test the coding/analysis schemes to be used with video-recorded encounters (DFE), an interview guide and coding scheme (IPE), and a survey (IPE). In Goal 2, these instruments were used to identify patient preferences in encounters so the three methods could be compared.

For goal 2, 13 encounters were video recorded, and participants were randomized to complete either an interview or survey immediately following their encounter. Findings from this study revealed differences between the methods regarding the ability to account for clarity of preference expression, the number and nuance of preferences expressed, certainty that expressions represented preferences, and the effort required to employ the methods. Participant gender appeared to have little effect on each method’s ability to identify preferences. The interview and video data suggest that women may attribute preferences to different issues than men, however the number of participants was too small to draw definitive conclusions.

Research Results

On average, when a video recording of an encounter was analyzed, researchers were able to identify more preferences than they could identify from the participant’s survey or interview data. However, it was less clear which of the preferences found in the video recording affected the participants’ final decision.

In contrast, from a completed survey, researchers could clearly identify which preferences affected the participant’s decision, however, the survey did not ask the participant about their preferences regarding many of the topics that only emerged during the participant’s and clinician’s video-recorded conversation.

On average, by interviewing a participant after their conversation with their clinician, researchers were able to identify more preferences than they could from the participant’s survey but fewer than they could from the video recording. The proportion of a participant’s identified preferences that clearly affected the participant’s decision was higher in interviews than in video recordings.

The study also found that participants frequently offered different reasons for their preferences in video recordings, interviews, and surveys.

Research Outcomes

Methods are needed to determine which preferences affect patients’ real-time decisions to use an approved medical product. Choice of methods to be used in studies to identify patients’ preferences at the point decision making should balance on a case-by-case basis:

  • The resources available to support the differential effort that video analysis, interviews, and surveys require.
  • The extent to which it is necessary that the set of identified preferences be comprehensive.
  • The extent to which it is necessary to comprehensively identify the different motivations/attributions that patient’s use to justify their preferences.

Results of this study have been published in the Journal Patient Preference and Adherence (DOI: https://doi.org/10.2147/ppa.s431378) and were presented at the 2024 CERSI Scientific Symposium at Stanford University.

 

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