PMID- 25773565
OWN - NLM
STAT- MEDLINE
DCOM- 20150615
LR  - 20220408
IS  - 1524-4733 (Electronic)
IS  - 1098-3015 (Linking)
VI  - 18
IP  - 2
DP  - 2015 Mar
TI  - Implications of the minimal clinically important difference for health-related 
      quality-of-life outcomes: a comparison of sample size requirements for an 
      incontinence treatment trial.
PG  - 292-8
LID - S1098-3015(14)04760-3 [pii]
LID - 10.1016/j.jval.2014.11.004 [doi]
AB  - BACKGROUND: Sample size calculations for treatment trials that aim to assess 
      health-related quality-of-life (HRQOL) outcomes are often difficult to perform. 
      Researchers must select a target minimal clinically important difference (MCID) 
      in HRQOL for the trial, estimate the effect size of the intervention, and then 
      consider the responsiveness of different HRQOL measures for detecting 
      improvements. Generic preference-based HRQOL measures are usually less sensitive 
      to gains in HRQOL than are disease-specific measures, but are nonetheless 
      recommended to quantify an impact on HRQOL that can be translated into 
      quality-adjusted life-years during cost-effectiveness analyses. Mapping 
      disease-specific measures onto generic measures is a proposed method for yielding 
      more efficient sample size requirements while retaining the ability to generate 
      utility weights for cost-effectiveness analyses. OBJECTIVES: This study sought to 
      test this mapping strategy to calculate and compare the effect on sample size of 
      three different methods. METHODS: Three different methods were used for 
      determining an MCID in HRQOL in patients with incontinence: 1) a global rating of 
      improvement, 2) an incontinence-specific HRQOL instrument, and 3) a generic 
      preference-based HRQOL instrument using mapping coefficients. RESULTS: The sample 
      size required to detect a 20% difference in the MCID for the global rating of 
      improvement was 52 per trial arm, 172 per arm for the incontinence-specific HRQOL 
      outcome, and 500 per arm for the generic preference-based HRQOL outcome. 
      CONCLUSIONS: We caution that treatment trials of conditions for which 
      improvements are not easy to measure on generic HRQOL instruments will still 
      require significantly greater sample size even when mapping functions are used to 
      try to gain efficiency.
CI  - Copyright (c) 2015 International Society for Pharmacoeconomics and Outcomes 
      Research (ISPOR). Published by Elsevier Inc. All rights reserved.
FAU - Halme, Alex S
AU  - Halme AS
AD  - Faculty of Medicine, University of Montreal, Montreal, QC, Canada.
FAU - Fritel, Xavier
AU  - Fritel X
AD  - Faculty of Medicine and Pharmacy, University of Poitiers, Poitiers, France.
FAU - Benedetti, Andrea
AU  - Benedetti A
AD  - Departments of Medicine, Biostatistics and Occupational Health, McGill 
      University, Montreal, Quebec, Canada; Departments of Epidemiology, Biostatistics 
      and Occupational Health, McGill University, Montreal, Quebec, Canada; Respiratory 
      Epidemiology and Clinical Research Unit, McGill University Health Center, 
      Montreal, QC, Canada.
FAU - Eng, Ken
AU  - Eng K
AD  - Independent Consultant, Ottawa, ON, Canada.
FAU - Tannenbaum, Cara
AU  - Tannenbaum C
AD  - Faculties of Medicine and Pharmacy, University of Montreal, Montreal, QC, Canada. 
      Electronic address: cara.tannenbaum@umontreal.ca.
LA  - eng
PT  - Comparative Study
PT  - Journal Article
PT  - Randomized Controlled Trial
PT  - Research Support, Non-U.S. Gov't
DEP - 20150128
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  - Aged
MH  - Female
MH  - *Health Status
MH  - Humans
MH  - *Quality-Adjusted Life Years
MH  - Sample Size
MH  - Single-Blind Method
MH  - Treatment Outcome
MH  - Urinary Incontinence/*epidemiology/*psychology/therapy
OTO - NOTNLM
OT  - clinical trials
OT  - minimal clinically important difference
OT  - quality of life
OT  - sample size
OT  - urinary incontinence
EDAT- 2015/03/17 06:00
MHDA- 2015/06/16 06:00
CRDT- 2015/03/17 06:00
PHST- 2014/03/26 00:00 [received]
PHST- 2014/09/10 00:00 [revised]
PHST- 2014/11/13 00:00 [accepted]
PHST- 2015/03/17 06:00 [entrez]
PHST- 2015/03/17 06:00 [pubmed]
PHST- 2015/06/16 06:00 [medline]
AID - S1098-3015(14)04760-3 [pii]
AID - 10.1016/j.jval.2014.11.004 [doi]
PST - ppublish
SO  - Value Health. 2015 Mar;18(2):292-8. doi: 10.1016/j.jval.2014.11.004. Epub 2015 
      Jan 28.