PMID- 30098675
OWN - NLM
STAT- MEDLINE
DCOM- 20180925
LR  - 20191210
IS  - 1524-4733 (Electronic)
IS  - 1098-3015 (Linking)
VI  - 21
IP  - 8
DP  - 2018 Aug
TI  - Patient-Reported Outcome Measures in the Food and Drug Administration Pilot 
      Compendium: Meeting Today's Standards for Patient Engagement in Development?
PG  - 967-972
LID - S1098-3015(18)30184-0 [pii]
LID - 10.1016/j.jval.2018.01.004 [doi]
AB  - BACKGROUND: In 2016, the Food and Drug Administration (FDA) released a Pilot 
      Clinical Outcome Assessment Compendium (COA Compendium) intended to foster 
      patient-focused drug development (PFDD). However, it is unclear whether patient 
      perspectives were solicited during development or validation of the included 
      patient-reported outcome (PRO) measures. OBJECTIVE: To examine the pedigree of a 
      sample of measures included in the COA Compendium. METHODS: PROs included in 
      chapters 1 or 2 of the COA Compendium were extracted and three reviewers 
      independently searched PubMed and Google to identify information on measure 
      pedigree. Data on method and stage of measure development where patient 
      engagement took place were documented. RESULTS: Among the 26 evaluated PRO 
      measures, we were unable to identify information on development or validation on 
      nearly half the sample (n = 12). Among the remaining 14 measures, 5 did not 
      include any evidence of patient engagement; 2 engaged patients during concept 
      elicitation only; 1 engaged patients during psychometric validation only; and 6 
      engaged patients during both concept elicitation and cognitive interviewing. 
      Measures either previously qualified or submitted for qualification were more 
      likely to include patient engagement. CONCLUSIONS: For the FDA Pilot COA 
      Compendium to fulfill its purpose of fostering PFDD, it needs fine-tuning to 
      reflect today's standards, improving transparency and facilitating clear 
      identification of included measures so that the level of patient engagement, 
      among other factors, can be properly assessed. Suggested improvements include 
      identifying clinical trials that correspond to the COA Compendium's use in drug 
      development; more clearly identifying which measure is referred to; and including 
      only those measures that already qualified or undergoing qualification.
CI  - Copyright (c) 2018 ISPOR-The Professional Society for Health Economics and Outcomes 
      Research. Published by Elsevier Inc. All rights reserved.
FAU - Oehrlein, Elisabeth M
AU  - Oehrlein EM
AD  - Department of Pharmaceutical Health Services Research, University of Maryland 
      School of Pharmacy, Baltimore, MD, USA. Electronic address: 
      eoehrlein@umaryland.edu.
FAU - Perfetto, Eleanor M
AU  - Perfetto EM
AD  - Department of Pharmaceutical Health Services Research, University of Maryland 
      School of Pharmacy, Baltimore, MD, USA; National Health Council, Washington, DC, 
      USA.
FAU - Love, T Rose
AU  - Love TR
AD  - Department of Pharmaceutical Health Services Research, University of Maryland 
      School of Pharmacy, Baltimore, MD, USA.
FAU - Chung, Yujin
AU  - Chung Y
AD  - University of Maryland School of Pharmacy, Baltimore, MD, USA.
FAU - Ghafoori, Parima
AU  - Ghafoori P
AD  - University of Maryland School of Pharmacy, Baltimore, MD, USA.
LA  - eng
PT  - Journal Article
DEP - 20180309
PL  - United States
TA  - Value Health
JT  - Value in health : the journal of the International Society for Pharmacoeconomics 
      and Outcomes Research
JID - 100883818
SB  - IM
MH  - Humans
MH  - Outcome Assessment, Health Care/*methods
MH  - Patient Participation/*methods
MH  - *Patient Reported Outcome Measures
MH  - Pilot Projects
MH  - United States
MH  - United States Food and Drug Administration/organization & administration
OTO - NOTNLM
OT  - Clinical Outcome Assessment (COA)
OT  - Measure Development
OT  - Patient Engagement [Method]
OT  - Patient-focused drug development (PFDD)
EDAT- 2018/08/14 06:00
MHDA- 2018/09/27 06:00
CRDT- 2018/08/13 06:00
PHST- 2017/09/07 00:00 [received]
PHST- 2017/12/05 00:00 [revised]
PHST- 2018/01/14 00:00 [accepted]
PHST- 2018/08/13 06:00 [entrez]
PHST- 2018/08/14 06:00 [pubmed]
PHST- 2018/09/27 06:00 [medline]
AID - S1098-3015(18)30184-0 [pii]
AID - 10.1016/j.jval.2018.01.004 [doi]
PST - ppublish
SO  - Value Health. 2018 Aug;21(8):967-972. doi: 10.1016/j.jval.2018.01.004. Epub 2018 
      Mar 9.