PMID- 36130315
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20221213
IS  - 2372-952X (Print)
IS  - 2372-952X (Electronic)
IS  - 2372-952X (Linking)
VI  - 9
IP  - 4
DP  - 2022 Oct 26
TI  - Interpreting Evaluating Respiratory Symptoms(TM) in COPD Diary Scores in Clinical 
      Trials: Terminology, Methods, and Recommendations.
PG  - 576-590
LID - 10.15326/jcopdf.2022.0307 [doi]
AB  - Accurately interpreting scores on patient-reported outcome (PRO) measures is 
      essential to understanding and communicating treatment benefit. Over the years, 
      terminology and methods for developing recommendations for PRO score 
      interpretation in clinical trials have evolved, leading to some confusion in the 
      field. The phrase "minimal clinically important difference (MCID)" has been 
      simplified to "minimal important difference (MID)" and use of responder 
      thresholds to interpret statistically significant treatment effects has 
      increased. Anchor-based derivation methods continue to be the standard, with 
      specific variations preferred by regulatory authorities for drug development 
      programs. In the midst of these changes, the Evaluating Respiratory Symptoms in 
      COPD (E-RS:COPD) was developed and qualified for use as an endpoint in chronic 
      obstructive pulmonary disease (COPD) drug development programs. This paper 
      summarizes the evolution of terminology and method preferences for the 
      development of recommendations for interpreting scores from PRO measures used in 
      clinical trials, and how these changes are reflected in the E-RS:COPD 
      recommendations. The intent is to add clarity to discussions around PRO endpoints 
      and facilitate use of the E-RS:COPD as a key efficacy endpoint in clinical trials 
      of COPD.
CI  - JCOPDF (c) 2022.
FAU - Leidy, Nancy K
AU  - Leidy NK
AD  - Evidera, Bethesda, Maryland, United States.
FAU - Bushnell, Donald M
AU  - Bushnell DM
AD  - Evidera, Bethesda, Maryland, United States.
FAU - Thach, Chau
AU  - Thach C
AD  - Novartis Pharma AG, Basel, Switzerland.
FAU - Hache, Carolina
AU  - Hache C
AD  - Novartis Pharma AG, Basel, Switzerland.
FAU - Gutzwiller, Florian S
AU  - Gutzwiller FS
AD  - Novartis Pharma AG, Basel, Switzerland.
LA  - eng
GR  - Novartis/Switzerland
PT  - Journal Article
PL  - United States
TA  - Chronic Obstr Pulm Dis
JT  - Chronic obstructive pulmonary diseases (Miami, Fla.)
JID - 101635411
PMC - PMC9718578
OTO - NOTNLM
OT  - Evaluating Respiratory Symptoms (E-RS) in Chronic Obstructive Pulmonary Disease 
      (COPD)
OT  - PROs and clinical trials
OT  - interpretation recommendations for PRO measures
OT  - patient-reported outcome (PRO) measure
OT  - symptomatic relief
COIS- NKL and DMB are employed by Evidera | PPD who received funding from Novartis to 
      conduct the review. FSG, CH, and CT are employed by Novartis. The development of 
      figures in this paper was supported by Novartis, as part of a contract with 
      Evidera for a related body of work.
EDAT- 2022/09/22 06:00
MHDA- 2022/09/22 06:01
PMCR- 2022/09/21
CRDT- 2022/09/21 17:32
PHST- 2022/09/22 06:00 [pubmed]
PHST- 2022/09/22 06:01 [medline]
PHST- 2022/09/21 17:32 [entrez]
PHST- 2022/09/21 00:00 [pmc-release]
AID - 10.15326/jcopdf.2022.0307 [doi]
PST - ppublish
SO  - Chronic Obstr Pulm Dis. 2022 Oct 26;9(4):576-590. doi: 10.15326/jcopdf.2022.0307.