PMID- 30259384
OWN - NLM
STAT- MEDLINE
DCOM- 20190712
LR  - 20200309
IS  - 1178-1661 (Electronic)
IS  - 1178-1653 (Linking)
VI  - 12
IP  - 2
DP  - 2019 Apr
TI  - How Do Members of the Duchenne and Becker Muscular Dystrophy Community Perceive a 
      Discrete-Choice Experiment Incorporating Uncertain Treatment Benefit? An 
      Application of Research as an Event.
PG  - 247-257
LID - 10.1007/s40271-018-0330-8 [doi]
AB  - BACKGROUND: Best-worst scaling methods have been used in several Duchenne and 
      Becker muscular dystrophy (DBMD) studies to quantify patient and caregiver 
      priorities and preferences and promote patient-focused drug development (PFDD). 
      We sought to assess the extent to which different members of the DBMD community 
      would accept a discrete-choice experiment (DCE) that incorporates uncertainty 
      regarding individual-level benefit. METHODS: A community advisory board 
      encouraged the development and testing of a DCE to further examine treatment 
      preferences in DBMD and to facilitate the inclusion of a policy-relevant 
      uncertainty attribute. The DCE assessed preferences across a primary outcome 
      (muscle strength) and several risks (uncertainty regarding treatment benefit, 
      kidney damage risk, and fracture risk). The single instrument was tested among 
      adult patients, caregivers, and professionals at the national Parent Project 
      Muscular Dystrophy annual meeting. The DCE was analyzed using conditional logit. 
      Instrument acceptability was evaluated using a previously developed set of 
      questions assessing ease of understanding and answering, and if answers reflected 
      the respondents' real preferences. We proposed a 75% agreement rate as a 
      threshold of acceptability, and used a Z score to assess if this was met, 
      exceeded, or rejected. RESULTS: A total of 161 people completed the survey 
      including 9 patients, 87 caregivers, and 65 professionals. Patients reported high 
      acceptability across all evaluation items (p values > 0.21). Caregivers and 
      professionals exceeded the benchmark of acceptability on understanding and 
      reflecting real preferences (p < 0.001). Professionals met the benchmark 
      (p = 0.08) for ease of answering, but caregivers did not (p < 0.01). DCE results 
      demonstrated that all groups made meaningful trade-offs, with patients being less 
      tolerant of risks than either caregivers or professionals (p < 0.001), and with 
      no significant difference between caregivers and professionals (p = 0.46). 
      CONCLUSIONS: This study demonstrates the acceptable application of a single 
      instrument across a multi-stakeholder population that used a complex preference 
      method and included a policy-relevant uncertainty variable. Ease of answering was 
      lowest among caregivers, but a post-hoc analysis revealed that it was most 
      difficult for those with children under the age of 10, while those with older 
      children met the threshold. The success of this study has laid the foundation for 
      a global study of DBMD preferences using this method.
FAU - Bridges, John F P
AU  - Bridges JFP
AD  - Department of Health Policy and Management, The Johns Hopkins Bloomberg School of 
      Public Health, 615 N. Wolfe St, Baltimore, MD, 21205, USA.
AD  - Department of Health, Behavior, and Society, The Johns Hopkins Bloomberg School 
      of Public Health, 615 N. Wolfe St, Baltimore, MD, 21205, USA.
FAU - Tsai, Jui-Hua
AU  - Tsai JH
AD  - Department of Health Policy and Management, The Johns Hopkins Bloomberg School of 
      Public Health, 615 N. Wolfe St, Baltimore, MD, 21205, USA.
FAU - Janssen, Ellen
AU  - Janssen E
AD  - Department of Health Policy and Management, The Johns Hopkins Bloomberg School of 
      Public Health, 615 N. Wolfe St, Baltimore, MD, 21205, USA.
FAU - Crossnohere, Norah L
AU  - Crossnohere NL
AD  - Department of Health, Behavior, and Society, The Johns Hopkins Bloomberg School 
      of Public Health, 615 N. Wolfe St, Baltimore, MD, 21205, USA. ncrossn1@jhu.edu.
FAU - Fischer, Ryan
AU  - Fischer R
AD  - Parent Project Muscular Dystrophy, 401 Hackensack Avenue, 9th Floor, Hackensack, 
      NJ, 07601, USA.
FAU - Peay, Holly
AU  - Peay H
AD  - Department of Health, Behavior, and Society, The Johns Hopkins Bloomberg School 
      of Public Health, 615 N. Wolfe St, Baltimore, MD, 21205, USA.
AD  - Parent Project Muscular Dystrophy, 401 Hackensack Avenue, 9th Floor, Hackensack, 
      NJ, 07601, USA.
AD  - RTI International, 701 13th St NW #750, Washington, DC, 20005, USA.
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PL  - New Zealand
TA  - Patient
JT  - The patient
JID - 101309314
SB  - IM
MH  - Adolescent
MH  - Adult
MH  - *Choice Behavior
MH  - Female
MH  - Humans
MH  - Male
MH  - Middle Aged
MH  - Muscular Dystrophy, Duchenne/*drug therapy
MH  - Perception
MH  - Policy
MH  - *Research
MH  - Risk Assessment
MH  - *Uncertainty
MH  - Young Adult
EDAT- 2018/09/28 06:00
MHDA- 2019/07/13 06:00
CRDT- 2018/09/28 06:00
PHST- 2018/09/28 06:00 [pubmed]
PHST- 2019/07/13 06:00 [medline]
PHST- 2018/09/28 06:00 [entrez]
AID - 10.1007/s40271-018-0330-8 [pii]
AID - 10.1007/s40271-018-0330-8 [doi]
PST - ppublish
SO  - Patient. 2019 Apr;12(2):247-257. doi: 10.1007/s40271-018-0330-8.