A community-engaged approach to quantifying caregiver preferences for the benefits and risks of emerging therapies for duchenne muscular dystrophy

Holly L. Peay, Ilene Hollin, Ryan Fischer, John F P Bridges

Research output: Contribution to journalArticle

Abstract

Background There is growing agreement that regulators performing benefit-risk evaluations should take patients' and caregivers' preferences into consideration. The Patient-Focused Drug Development Initiative at the US Food and Drug Administration offers patients and caregivers an enhanced opportunity to contribute to regulatory processes by offering direct testimonials. This process may be advanced by providing scientific evidence regarding treatment preferences through engagement of a broad community of patients and caregivers. Objective In this article, we demonstrate a community-engaged approach to measure caregiver preferences for potential benefits and risks of emerging therapies for Duchenne muscular dystrophy (DMD). Methods An advocacy oversight team led the community-engaged study. Caregivers' treatment preferences were measured by using best-worst scaling (BWS). Six relevant and understandable attributes describing potential benefits and risks of emerging DMD therapies were identified through engagement with advocates (n = 5), clinicians (n = 9), drug developers from pharmaceutical companies and academic centers (n = 11), and other stakeholders (n = 5). The attributes, each defined across 3 levels, included muscle function, life span, knowledge about the drug, nausea, risk of bleeds, and risk of arrhythmia. Cognitive interviewing with caregivers (n = 7) was used to refine terminology and assess acceptability of the BWS instrument. The study was implemented through an online survey of DMD caregivers, who were recruited in the United States through an advocacy group and snowball sampling. Caregivers were presented with 18 treatment profiles, identified via a main-effect orthogonal experimental design, in which the dependent variable was the respondents' judgment as to the best and worst feature in each profile. Preference weights were estimated by calculating the relative number of times a feature was chosen as best and as worst, which were then used to estimate relative attribute importance. Results A total of 119 DMD caregivers completed the BWS instrument; they were predominately biological mothers (67.2%), married (89.9%), and white (91.6%). Treatment effect on muscle function was the most important among experimental attributes (28.7%), followed by risk of heart arrhythmia (22.4%) and risk of bleeding (21.2%). Having additional postapproval data was relatively the least important attribute (2.3%). Conclusions We present a model process for advocacy organizations aiming to promote patient-centered drug development. The community-engaged approach was successfully used to develop and implement a survey to measure caregiver preferences. Caregivers were willing to accept a serious risk when balanced with a noncurative treatment, even absent improvement in life span. These preferences should inform the Food and Drug Administration's benefit-risk assessment of emerging DMD therapies. This study highlights the synergistic integration of traditional advocacy methods and scientific approach to quantify benefit-risk preferences.

Original languageEnglish (US)
Pages (from-to)624-637
Number of pages14
JournalClinical Therapeutics
Volume36
Issue number5
DOIs
StatePublished - May 1 2014

Fingerprint

Duchenne Muscular Dystrophy
Caregivers
Therapeutics
Pharmaceutical Preparations
United States Food and Drug Administration
Cardiac Arrhythmias
Muscles
Patient Preference
Terminology
Nausea
Research Design
Mothers
Organizations
Hemorrhage
Weights and Measures

Keywords

  • benefit-risk assessment
  • caregiver
  • choice behavior
  • Duchenne muscular dystrophy
  • patient preferences

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)
  • Medicine(all)

Cite this

A community-engaged approach to quantifying caregiver preferences for the benefits and risks of emerging therapies for duchenne muscular dystrophy. / Peay, Holly L.; Hollin, Ilene; Fischer, Ryan; Bridges, John F P.

In: Clinical Therapeutics, Vol. 36, No. 5, 01.05.2014, p. 624-637.

Research output: Contribution to journalArticle

Peay, Holly L. ; Hollin, Ilene ; Fischer, Ryan ; Bridges, John F P. / A community-engaged approach to quantifying caregiver preferences for the benefits and risks of emerging therapies for duchenne muscular dystrophy. In: Clinical Therapeutics. 2014 ; Vol. 36, No. 5. pp. 624-637.
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