Patient-Reported Outcome Measures in the Food and Drug Administration Pilot Compendium: Meeting Today’s Standards for Patient Engagement in Development?

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 pat...

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Vydané v:	Value in health Ročník 21; číslo 8; s. 967 - 972
Hlavní autori:	Oehrlein, Elisabeth M., Perfetto, Eleanor M., Love, T. Rose, Chung, Yujin, Ghafoori, Parima
Médium:	Journal Article
Jazyk:	English
Vydavateľské údaje:	United States Elsevier Inc 01.08.2018 Elsevier Science Ltd
Predmet:	Clinical assessment Clinical Outcome Assessment (COA) Clinical outcomes Clinical research Clinical standards Clinical trials Cognitive ability Cognitive interviews Drug development Drugs Elicitation FDA approval Food Identification Interviews Management Measure Development Patient Engagement [Method] Patient handling Patient-focused drug development (PFDD) Patients Pedigree Quantitative psychology Transparency Validity Clinical Outcome Assessment (COA) Patient-focused drug development (PFDD) Patient Engagement [Method] Measure Development
ISSN:	1098-3015, 1524-4733, 1524-4733
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Abstract	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. To examine the pedigree of a sample of measures included in the COA Compendium. 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. 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. 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.
AbstractList	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. 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.BACKGROUNDIn 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.To examine the pedigree of a sample of measures included in the COA Compendium.OBJECTIVETo examine the pedigree of a sample of measures included in the COA Compendium.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.METHODSPROs 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.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.RESULTSAmong 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.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.CONCLUSIONSFor 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. 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. To examine the pedigree of a sample of measures included in the COA Compendium. 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. 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. 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.
Author	Perfetto, Eleanor M. Love, T. Rose Ghafoori, Parima Chung, Yujin Oehrlein, Elisabeth M.
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BackLink	https://www.ncbi.nlm.nih.gov/pubmed/30098675$$D View this record in MEDLINE/PubMed
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Cites_doi	10.1016/j.jchf.2016.04.010 10.1111/j.1365-2869.1994.tb00114.x 10.1007/s11882-003-0100-z 10.1016/S0735-1097(00)00531-3 10.1111/j.1524-4733.2007.00272.x 10.1007/s40271-016-0183-y 10.1111/j.1524-4733.2009.00603.x 10.1097/MLR.0000000000000273 10.1007/s40271-015-0159-3 10.1016/j.jvsv.2015.03.003 10.1016/j.jval.2017.05.015 10.1186/1477-7525-8-89 10.1056/NEJMp1110652 10.1016/j.clinthera.2007.09.005 10.1016/j.cgh.2007.02.004 10.1177/0268355515595193 10.1001/archopht.1990.01070030090035 10.1258/phleb.2008.008018 10.1111/apt.13703 10.1016/j.jval.2014.08.1897 10.1016/j.jval.2011.06.013 10.1016/j.jval.2012.01.010 10.1016/j.jval.2011.06.014
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References	Kleinman, Talbot, SchÃler (bib17) 2014; 17 Canestaro, Edwards, Patrick (bib21) 2016; 44 Abelson, Chambers, Smith (bib29) 1990; 108 Benjamin, Vernon, Patrick (bib9) 2017; 20 Food and Drug Administration. Pilot Clinical Outcome Assessment Compendium (COA Compendium) Version 1. Available from Wright, Paty, Turner-Bowker, Bradbury (bib27) 2016; 9 (bib3) 2009 [Accessed July 19, 2017]. Paty, Turner-Bowker, Elash, Wright (bib26) 2016; 31 DeMuro, Clark, Mordin (bib31) 2012; 5 Perfetto, Burke, Oehrlein, Epstein (bib6) 2015; 53 Monk, Reynolds, Kupfer (bib33) 1994; 3 Todd, Wright (bib23) 2015; 3 Abelson, Loeffler (bib30) 2003; 3 Vasquez, Munschauer (bib25) 2008; 23 Paty, Elash, Turner-Bowker (bib28) 2017; 10 Newman, Feldman (bib20) 2011; 365 [Accessed June 1, 2017]. Frost, Reeve, Liepa (bib18) 2007; 10 Miles, Huberman, Saldana (bib8) 1994 [Accessed June 25, 2016]. International Society for Quality of Life Research. ISOQOL commentary on FDA Compendium. Available from Letter to FDA: Re: Docket No. FDA-2015-N-5106: Clinical Outcome Assessment Compendium. Available from Rothman, Burke, Erickson (bib12) 2009; 12 Patrick, Burke, Gwaltney (bib10) 2011; 14 Green, Porter, Bresnahan, Spertus (bib15) 2000; 35 Food and Drug Administration. Roadmap to patient-focused outcome measurement in clinical trials. Available from Psotka, von Maltzahn, Anatchkova (bib14) 2016; 4 Food and Drug Administration. Pilot Clinical Outcome Assessment Compendium (COA Compendium) Webinar (March 8, 2016). Available from Patrick, Burke, Gwaltney (bib11) 2011; 14 Dupont, Jiang, Belkind-Gerson (bib34) 2007; 5 (bib13) 2006 Food and Drug Administration. Clinical Outcome Assessment Qualification Program submissions. Available from [Accessed June 29, 2016]. (bib24) 2012 Rahman, Rizwan, Waycaster, Wall (bib32) 2007; 29 Food and Drug Administration. Enhancing the development and use of patient-reported outcomes in drug development: July 16, 2014 meeting summary. Available from Doward, Gnanasakthy, Baker (bib19) 2010; 8 Todd (10.1016/j.jval.2018.01.004_bib23) 2015; 3 Patrick (10.1016/j.jval.2018.01.004_bib11) 2011; 14 Rahman (10.1016/j.jval.2018.01.004_bib32) 2007; 29 DeMuro (10.1016/j.jval.2018.01.004_bib31) 2012; 5 10.1016/j.jval.2018.01.004_bib22 Rothman (10.1016/j.jval.2018.01.004_bib12) 2009; 12 Vasquez (10.1016/j.jval.2018.01.004_bib25) 2008; 23 Dupont (10.1016/j.jval.2018.01.004_bib34) 2007; 5 Psotka (10.1016/j.jval.2018.01.004_bib14) 2016; 4 Miles (10.1016/j.jval.2018.01.004_bib8) 1994 10.1016/j.jval.2018.01.004_bib7 Green (10.1016/j.jval.2018.01.004_bib15) 2000; 35 10.1016/j.jval.2018.01.004_bib5 Paty (10.1016/j.jval.2018.01.004_bib26) 2016; 31 10.1016/j.jval.2018.01.004_bib4 Kleinman (10.1016/j.jval.2018.01.004_bib17) 2014; 17 10.1016/j.jval.2018.01.004_bib2 10.1016/j.jval.2018.01.004_bib16 10.1016/j.jval.2018.01.004_bib1 Newman (10.1016/j.jval.2018.01.004_bib20) 2011; 365 Wright (10.1016/j.jval.2018.01.004_bib27) 2016; 9 Patrick (10.1016/j.jval.2018.01.004_bib10) 2011; 14 Abelson (10.1016/j.jval.2018.01.004_bib30) 2003; 3 Benjamin (10.1016/j.jval.2018.01.004_bib9) 2017; 20 Frost (10.1016/j.jval.2018.01.004_bib18) 2007; 10 Perfetto (10.1016/j.jval.2018.01.004_bib6) 2015; 53 (10.1016/j.jval.2018.01.004_bib3) 2009 Canestaro (10.1016/j.jval.2018.01.004_bib21) 2016; 44 Monk (10.1016/j.jval.2018.01.004_bib33) 1994; 3 (10.1016/j.jval.2018.01.004_bib13) 2006 Doward (10.1016/j.jval.2018.01.004_bib19) 2010; 8 Abelson (10.1016/j.jval.2018.01.004_bib29) 1990; 108 Paty (10.1016/j.jval.2018.01.004_bib28) 2017; 10 (10.1016/j.jval.2018.01.004_bib24) 2012
References_xml	– volume: 108 start-page: 84 year: 1990 end-page: 88 ident: bib29 article-title: Conjunctival allergen challenge: a clinical approach to studying allergic conjunctivitis publication-title: Arch Ophthalmol – volume: 17 start-page: A569 year: 2014 ident: bib17 article-title: Cdad-Daysymsâ: a new patient-reported outcome tool for publication-title: Value Health – volume: 10 start-page: 51 year: 2017 end-page: 63 ident: bib28 article-title: Content validity for the VVSymQ(R) Instrument: a new patient-reported outcome measure for the assessment of varicose veins symptoms publication-title: Patient – volume: 14 start-page: 967 year: 2011 end-page: 977 ident: bib10 article-title: Content validity—establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO Good Research Practices Task Force report: part 1—eliciting concepts for a new PRO instrument publication-title: Value Health – volume: 10 start-page: S94 year: 2007 end-page: S105 ident: bib18 article-title: What is sufficient evidence for the reliability and validity of patient-reported outcome measures? publication-title: Value Health – year: 2006 ident: bib13 article-title: Patient-reported outcome measures: use in medical product development to support labeling claims publication-title: Draft guidance. – reference: Letter to FDA: Re: Docket No. FDA-2015-N-5106: Clinical Outcome Assessment Compendium. Available from: – volume: 12 start-page: 1075 year: 2009 end-page: 1083 ident: bib12 article-title: Use of existing patient-reported outcome (PRO) instruments and their modification: the ISPOR Good Research Practices for Evaluating and Documenting Content Validity for the Use of Existing Instruments and Their Modification PRO Task Force report publication-title: Value Health – volume: 29 start-page: 1950 year: 2007 end-page: 1956 ident: bib32 article-title: Pooled analysis of two clinical trials comparing the clinical outcomes of topical ciprofloxacin/dexamethasone otic suspension and polymyxin B/neomycin/hydrocortisone otic suspension for the treatment of acute otitis externa in adults and children publication-title: Clin Ther – volume: 31 start-page: 481 year: 2016 end-page: 488 ident: bib26 article-title: The VVSymQ(R) instrument: use of a new patient-reported outcome measure for assessment of varicose vein symptoms publication-title: Phlebology – volume: 8 start-page: 89 year: 2010 ident: bib19 article-title: Patient-reported outcomes: looking beyond the label claim publication-title: Health Qual Life Outcomes – reference: . [Accessed July 19, 2017]. – volume: 3 start-page: 111 year: 1994 end-page: 120 ident: bib33 article-title: The Pittsburgh Sleep Diary publication-title: J Sleep Res – volume: 53 start-page: 9 year: 2015 end-page: 17 ident: bib6 article-title: Patient-focused drug development: a new direction for collaboration publication-title: Med Care – volume: 365 start-page: 2447 year: 2011 end-page: 2449 ident: bib20 article-title: Copyright and open access at the bedside publication-title: N Engl J Med – volume: 35 start-page: 1245 year: 2000 end-page: 1255 ident: bib15 article-title: Development and evaluation of the Kansas City Cardiomyopathy Questionnaire: a new health status measure for heart failure publication-title: J Am Coll Cardiol – volume: 3 start-page: 258 year: 2015 end-page: 264 ident: bib23 article-title: Durability of treatment effect with polidocanol endovenous microfoam on varicose vein symptoms and appearance (VANISH-2) publication-title: J Vasc Surg Venous Lymphat Disord – volume: 44 start-page: 313 year: 2016 end-page: 331 ident: bib21 article-title: Systematic review: patient-reported outcome measures in coeliac disease for regulatory submissions publication-title: Aliment Pharmacol Ther – reference: Food and Drug Administration. Pilot Clinical Outcome Assessment Compendium (COA Compendium) Webinar (March 8, 2016). Available from: – reference: Food and Drug Administration. Clinical Outcome Assessment Qualification Program submissions. Available from: – reference: Food and Drug Administration. Enhancing the development and use of patient-reported outcomes in drug development: July 16, 2014 meeting summary. Available from: – start-page: 24 year: 2012 end-page: 28 ident: bib24 article-title: Validation of new patient/clinician-reported outcome tool—capturing the visual impact of varicose veins – reference: . [Accessed June 29, 2016]. – reference: International Society for Quality of Life Research. ISOQOL commentary on FDA Compendium. Available from: – reference: . [Accessed June 1, 2017]. – volume: 5 start-page: 443 year: 2012 end-page: 448 ident: bib31 article-title: Reasons for rejection of patient-reported outcome label claims: a compilation based on a review of patient-reported outcome use among new molecular entities and biologic license applications, 2006–2010 publication-title: Value Health – year: 1994 ident: bib8 article-title: Qualitative Data Analysis: A Methods Sourcebook – volume: 14 start-page: 978 year: 2011 end-page: 988 ident: bib11 article-title: Content validity—establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO Good Research Practices Task Force report: part 2—assessing respondent understanding publication-title: Value Health – volume: 5 start-page: 451 year: 2007 end-page: 456 ident: bib34 article-title: Treatment of travelers’ diarrhea: randomized trial comparing rifaximin, rifaximin plus loperamide, and loperamide alone publication-title: Clin Gastroenterol Hepatol – reference: . [Accessed June 25, 2016]. – volume: 20 start-page: 838 year: 2017 end-page: 855 ident: bib9 article-title: Patient-reported outcome and observer-reported outcome assessment in rare disease clinical trials: an ISPOR COA Emerging Good Practices Task Force Report publication-title: Value Health – volume: 23 start-page: 259 year: 2008 end-page: 275 ident: bib25 article-title: Venous Clinical Severity Score and quality-of-life assessment tools: application to vein practice publication-title: Phlebology – reference: Food and Drug Administration. Roadmap to patient-focused outcome measurement in clinical trials. Available from: – year: 2009 ident: bib3 publication-title: Guidance for Industry. Patient-reported outcome measures: use in medical product development to support labeling claims. – volume: 4 start-page: 791 year: 2016 end-page: 804 ident: bib14 article-title: Patient-reported outcomes in chronic heart failure: applicability for regulatory approval publication-title: JACC Heart Fail – volume: 3 start-page: 363 year: 2003 end-page: 368 ident: bib30 article-title: Conjunctival allergen challenge: models in the investigation of ocular allergy publication-title: Curr Allergy Asthma Rep – reference: Food and Drug Administration. Pilot Clinical Outcome Assessment Compendium (COA Compendium) Version 1. Available from: – volume: 9 start-page: 335 year: 2016 end-page: 348 ident: bib27 article-title: Psychometric evaluation of a new patient-reported outcome (PRO) symptom diary for varicose veins: VVSymQ(R) Instrument publication-title: Patient – ident: 10.1016/j.jval.2018.01.004_bib7 – volume: 4 start-page: 791 year: 2016 ident: 10.1016/j.jval.2018.01.004_bib14 article-title: Patient-reported outcomes in chronic heart failure: applicability for regulatory approval publication-title: JACC Heart Fail doi: 10.1016/j.jchf.2016.04.010 – volume: 3 start-page: 111 year: 1994 ident: 10.1016/j.jval.2018.01.004_bib33 article-title: The Pittsburgh Sleep Diary publication-title: J Sleep Res doi: 10.1111/j.1365-2869.1994.tb00114.x – ident: 10.1016/j.jval.2018.01.004_bib5 – volume: 3 start-page: 363 year: 2003 ident: 10.1016/j.jval.2018.01.004_bib30 article-title: Conjunctival allergen challenge: models in the investigation of ocular allergy publication-title: Curr Allergy Asthma Rep doi: 10.1007/s11882-003-0100-z – year: 2009 ident: 10.1016/j.jval.2018.01.004_bib3 publication-title: Guidance for Industry. Patient-reported outcome measures: use in medical product development to support labeling claims. – volume: 35 start-page: 1245 year: 2000 ident: 10.1016/j.jval.2018.01.004_bib15 article-title: Development and evaluation of the Kansas City Cardiomyopathy Questionnaire: a new health status measure for heart failure publication-title: J Am Coll Cardiol doi: 10.1016/S0735-1097(00)00531-3 – ident: 10.1016/j.jval.2018.01.004_bib1 – volume: 10 start-page: S94 year: 2007 ident: 10.1016/j.jval.2018.01.004_bib18 article-title: What is sufficient evidence for the reliability and validity of patient-reported outcome measures? publication-title: Value Health doi: 10.1111/j.1524-4733.2007.00272.x – volume: 10 start-page: 51 year: 2017 ident: 10.1016/j.jval.2018.01.004_bib28 article-title: Content validity for the VVSymQ(R) Instrument: a new patient-reported outcome measure for the assessment of varicose veins symptoms publication-title: Patient doi: 10.1007/s40271-016-0183-y – volume: 12 start-page: 1075 year: 2009 ident: 10.1016/j.jval.2018.01.004_bib12 article-title: Use of existing patient-reported outcome (PRO) instruments and their modification: the ISPOR Good Research Practices for Evaluating and Documenting Content Validity for the Use of Existing Instruments and Their Modification PRO Task Force report publication-title: Value Health doi: 10.1111/j.1524-4733.2009.00603.x – volume: 53 start-page: 9 year: 2015 ident: 10.1016/j.jval.2018.01.004_bib6 article-title: Patient-focused drug development: a new direction for collaboration publication-title: Med Care doi: 10.1097/MLR.0000000000000273 – volume: 9 start-page: 335 year: 2016 ident: 10.1016/j.jval.2018.01.004_bib27 article-title: Psychometric evaluation of a new patient-reported outcome (PRO) symptom diary for varicose veins: VVSymQ(R) Instrument publication-title: Patient doi: 10.1007/s40271-015-0159-3 – volume: 3 start-page: 258 year: 2015 ident: 10.1016/j.jval.2018.01.004_bib23 article-title: Durability of treatment effect with polidocanol endovenous microfoam on varicose vein symptoms and appearance (VANISH-2) publication-title: J Vasc Surg Venous Lymphat Disord doi: 10.1016/j.jvsv.2015.03.003 – volume: 20 start-page: 838 year: 2017 ident: 10.1016/j.jval.2018.01.004_bib9 article-title: Patient-reported outcome and observer-reported outcome assessment in rare disease clinical trials: an ISPOR COA Emerging Good Practices Task Force Report publication-title: Value Health doi: 10.1016/j.jval.2017.05.015 – volume: 8 start-page: 89 year: 2010 ident: 10.1016/j.jval.2018.01.004_bib19 article-title: Patient-reported outcomes: looking beyond the label claim publication-title: Health Qual Life Outcomes doi: 10.1186/1477-7525-8-89 – ident: 10.1016/j.jval.2018.01.004_bib22 – volume: 365 start-page: 2447 year: 2011 ident: 10.1016/j.jval.2018.01.004_bib20 article-title: Copyright and open access at the bedside publication-title: N Engl J Med doi: 10.1056/NEJMp1110652 – volume: 29 start-page: 1950 year: 2007 ident: 10.1016/j.jval.2018.01.004_bib32 article-title: Pooled analysis of two clinical trials comparing the clinical outcomes of topical ciprofloxacin/dexamethasone otic suspension and polymyxin B/neomycin/hydrocortisone otic suspension for the treatment of acute otitis externa in adults and children publication-title: Clin Ther doi: 10.1016/j.clinthera.2007.09.005 – ident: 10.1016/j.jval.2018.01.004_bib4 – ident: 10.1016/j.jval.2018.01.004_bib2 – start-page: 24 year: 2012 ident: 10.1016/j.jval.2018.01.004_bib24 – volume: 5 start-page: 451 year: 2007 ident: 10.1016/j.jval.2018.01.004_bib34 article-title: Treatment of travelers’ diarrhea: randomized trial comparing rifaximin, rifaximin plus loperamide, and loperamide alone publication-title: Clin Gastroenterol Hepatol doi: 10.1016/j.cgh.2007.02.004 – volume: 31 start-page: 481 year: 2016 ident: 10.1016/j.jval.2018.01.004_bib26 article-title: The VVSymQ(R) instrument: use of a new patient-reported outcome measure for assessment of varicose vein symptoms publication-title: Phlebology doi: 10.1177/0268355515595193 – volume: 108 start-page: 84 year: 1990 ident: 10.1016/j.jval.2018.01.004_bib29 article-title: Conjunctival allergen challenge: a clinical approach to studying allergic conjunctivitis publication-title: Arch Ophthalmol doi: 10.1001/archopht.1990.01070030090035 – volume: 23 start-page: 259 year: 2008 ident: 10.1016/j.jval.2018.01.004_bib25 article-title: Venous Clinical Severity Score and quality-of-life assessment tools: application to vein practice publication-title: Phlebology doi: 10.1258/phleb.2008.008018 – volume: 44 start-page: 313 year: 2016 ident: 10.1016/j.jval.2018.01.004_bib21 article-title: Systematic review: patient-reported outcome measures in coeliac disease for regulatory submissions publication-title: Aliment Pharmacol Ther doi: 10.1111/apt.13703 – year: 2006 ident: 10.1016/j.jval.2018.01.004_bib13 article-title: Patient-reported outcome measures: use in medical product development to support labeling claims publication-title: Draft guidance. – volume: 17 start-page: A569 year: 2014 ident: 10.1016/j.jval.2018.01.004_bib17 article-title: Cdad-Daysymsâ: a new patient-reported outcome tool for Clostridium difficile-associated diarrhea publication-title: Value Health doi: 10.1016/j.jval.2014.08.1897 – volume: 14 start-page: 978 year: 2011 ident: 10.1016/j.jval.2018.01.004_bib11 article-title: Content validity—establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO Good Research Practices Task Force report: part 2—assessing respondent understanding publication-title: Value Health doi: 10.1016/j.jval.2011.06.013 – ident: 10.1016/j.jval.2018.01.004_bib16 – year: 1994 ident: 10.1016/j.jval.2018.01.004_bib8 – volume: 5 start-page: 443 issue: 15 year: 2012 ident: 10.1016/j.jval.2018.01.004_bib31 article-title: Reasons for rejection of patient-reported outcome label claims: a compilation based on a review of patient-reported outcome use among new molecular entities and biologic license applications, 2006–2010 publication-title: Value Health doi: 10.1016/j.jval.2012.01.010 – volume: 14 start-page: 967 year: 2011 ident: 10.1016/j.jval.2018.01.004_bib10 article-title: Content validity—establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO Good Research Practices Task Force report: part 1—eliciting concepts for a new PRO instrument publication-title: Value Health doi: 10.1016/j.jval.2011.06.014
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Snippet	In 2016, the Food and Drug Administration (FDA) released a Pilot Clinical Outcome Assessment Compendium (COA Compendium) intended to foster patient-focused... Background In 2016, the Food and Drug Administration (FDA) released a Pilot Clinical Outcome Assessment Compendium (COA Compendium) intended to foster...
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SubjectTerms	Clinical assessment Clinical Outcome Assessment (COA) Clinical outcomes Clinical research Clinical standards Clinical trials Cognitive ability Cognitive interviews Drug development Drugs Elicitation FDA approval Food Identification Interviews Management Measure Development Patient Engagement [Method] Patient handling Patient-focused drug development (PFDD) Patients Pedigree Quantitative psychology Transparency Validity
Title	Patient-Reported Outcome Measures in the Food and Drug Administration Pilot Compendium: Meeting Today’s Standards for Patient Engagement in Development?
URI	https://www.clinicalkey.com/#!/content/1-s2.0-S1098301518301840 https://dx.doi.org/10.1016/j.jval.2018.01.004 https://www.ncbi.nlm.nih.gov/pubmed/30098675 https://www.proquest.com/docview/2108865530 https://www.proquest.com/docview/2087993958
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