Highlights
- •A total of 323 unique patient-reported outcomes measures (PROMs) were recommended for use across 94 core outcome sets (COS) and 26 disease areas.
- •Eighty-seven percent of instruments were recommended in only one COS, and each COS included a median of 4.5 instruments.
- •Overall, global quality of life (25%) and physical functioning (22%) were the most frequently targeted health outcome domains.
- •A fragmented landscape of recommended PROMs in COS calls for better harmonization of PRO selection and measurement.
Abstract
Objective
There is no comprehensive assessment of which patient-reported outcomes (PROs) are
recommended in core outcome sets (COS), and how they should be measured. The aims
of this study are to review COS that include patient-reported outcomes measures (PROMs),
identify their target health domains, main characteristics, and their overlap within
and across different disease areas.
Study design and setting
We selected COS studies collected in a publicly available database that included at
least one recommended PROM. We gathered information on study setting, disease area,
and targeted outcome domains. Full-text of recommended instruments were obtained,
and an analysis of their characteristics and content performed. We classified targeted
domains according to a predefined 38-item taxonomy.
Results
Overall, we identified 94 COS studies that recommended 323 unique instruments, of
which: 87% were included in only one COS; 77% were disease-specific; 1.5% preference-based;
and 61% corresponded to a full questionnaire. Most of the instruments covered broad
health-related constructs, such as global quality of life (25%), physical functioning
(22%), emotional functioning and wellbeing (7%).
Conclusion
The wealth of recommended instruments observed even within disease areas does not
fit with a vision of systematic, harmonized collection of PROM data in COS within
and across disease areas.
Keywords
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References
- Patient-reported outcomes (PROS) and patient-reported outcome measures (PROMs).Health Serv Insights. 2013; 6: 61-68https://doi.org/10.4137/HSI.S11093
U.S. Food And Drug Administration. Patient-reported outcome measures: use in medical product development to support labeling claims 2009 Available at: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-reported-outcome-measures-use-medical-product-development-support-labeling-claims], 17/11/2020.
European Medicines Agency. Appendix 2 to the guideline on the evaluation of anticancer medicinal products in man - the use of patient-reported outcome (PRO) measures in oncology studies 2016 Available at: https://www.ema.europa.eu/en/appendix-2-guideline-evaluation-anticancer-medicinal-products-man-use-patient-reported-outcome-pro, 17/11/2020.
- Incorporating patient-reported outcomes into health care to engage patients and enhance care.Health Aff (Millwood). 2016; 35: 575-582https://doi.org/10.1377/hlthaff.2015.1362
- Standards for patient-reported outcome-based performance measures.JAMA. 2013; 310: 139-140https://doi.org/10.1001/jama.2013.6855
- value lies in the eye of the patients: the why, what, and how of patient-reported outcomes measures.Clin Ther. 2020; 42 (Epub 2020 Jan 10): 25-33https://doi.org/10.1016/j.clinthera.2019.11.016
- Developing core outcomes sets: methods for identifying and including patient-reported outcomes (PROs).Trials. 2014; 15: 49https://doi.org/10.1186/1745-6215-15-49
- Choosing important health outcomes for comparative effectiveness research: a systematic review.PLoS One. 2014; 9: e99111https://doi.org/10.1371/journal.pone.0099111
- A taxonomy has been developed for outcomes in medical research to help improve knowledge discovery.J Clin Epidemiol. 2018; 96 (Epub 2017 Dec 28): 84-92https://doi.org/10.1016/j.jclinepi.2017.12.020
- Methods used in the selection of instruments for outcomes included in core outcome sets have improved since the publication of the COSMIN/COMET guideline.J Clin Epidemiol. 2020; 125 (Epub 2020 May 26): 64-75https://doi.org/10.1016/j.jclinepi.2020.05.021
- Choosing important health outcomes for comparative effectiveness research: 5th annual update to a systematic review of core outcome sets for research.PLoS One. 2019; 14e0225980https://doi.org/10.1371/journal.pone.0225980
- Outcome assessments in the evaluation of treatment of spinal disorders: summary and general recommendations.Spine (Phila Pa 1976). 2000; 25: 3100-3103https://doi.org/10.1097/00007632-200012150-00003
- Development of a provisional core set of response measures for clinical trials of systemic sclerosis.Ann Rheum Dis. 2008; 67 (Epub 2007 Sep 24): 703-709https://doi.org/10.1136/ard.2007.078923
- Arthritis in systemic sclerosis: systematic review of the literature and suggestions for the performance of future clinical trials in systemic sclerosis arthritis.Semin Arthritis Rheum. 2012; 41 (Epub 2011 Dec 15): 801-814https://doi.org/10.1016/j.semarthrit.2011.10.003
- The OMERACT core set of outcome measures for use in clinical trials of ANCA-associated vasculitis.J Rheumatol. 2011; 38: 1480-1486https://doi.org/10.3899/jrheum.110276
- EULAR recommendations for conducting clinical studies and/or clinical trials in systemic vasculitis: focus on anti-neutrophil cytoplasm antibody-associated vasculitis.Ann Rheum Dis. 2007; 66 (Epub 2006 Dec 14): 605-617https://doi.org/10.1136/ard.2006.062711
- A comparative review of generic quality-of-life instruments.Pharmacoeconomics. 2000; 17: 13-35https://doi.org/10.2165/00019053-200017010-00002
- A review of generic preference-based measures for use in cost-effectiveness models.Pharmacoeconomics. 2017; 35: 21-31https://doi.org/10.1007/s40273-017-0545-x
- The role of condition-specific preference-based measures in health technology assessment.Pharmacoeconomics. 2017; 35: 33-41https://doi.org/10.1007/s40273-017-0546-9
- Consensus statement on evaluation of outcome of pharmacotherapy for substance abuse/dependence: report from a NIDA/CPDD meeting.National Institute on Drug Abuse Medications Development Division, Bethesda, MD1999 (Available at:) (17/11/2020)
- Report of the NIH Task Force on research standards for chronic low back pain.J Pain. 2014; 15 (Epub 2014 Apr 29): 569-585https://doi.org/10.1016/j.jpain.2014.03.005
- Multinational Interdisciplinary Working Group for Uveitis in Childhood. Proposed outcome measures for prospective clinical trials in juvenile idiopathic arthritis-associated uveitis: a consensus effort from the multinational interdisciplinary working group for uveitis in childhood.Arthritis Care Res (Hoboken). 2012; 64: 1365-1372https://doi.org/10.1002/acr.21674
- Reconciling healthcare professional and patient perspectives in the development of disease activity and response criteria in connective tissue disease-related interstitial lung diseases.J Rheumatol. 2014; 41 (Epub 2014 Feb 1): 792-798https://doi.org/10.3899/jrheum.131251
- Pain scoring in endometriosis: entry criteria and outcome measures for clinical trials. Report from the Art and Science of Endometriosis meeting.Fertil Steril. 2010; 93 (Epub 2008 Nov 5): 62-67https://doi.org/10.1016/j.fertnstert.2008.09.056
- Guidelines for controlled trials of drugs in cluster headache.Cephalalgia. 1995; 15: 452-462
- Guidelines for controlled trials of drugs in migraine: second edition.Cephalalgia. 2000; 20: 765-786https://doi.org/10.1046/j.1468-2982.2000.00117.x
- Guidelines for trials of behavioral treatments for recurrent headache: purpose, process, and product.Headache. 2005; 45: S87-S89https://doi.org/10.1111/j.1526-4610.2005.4502001.x
- Guidelines for controlled trials of drugs in tension-type headache: second edition.Cephalalgia. 2010; 30: 1-16https://doi.org/10.1111/j.1468-2982.2009.01948.x
- Consensus guidelines for the design and implementation of clinical trials in ALS. World Federation of Neurology committee on Research.J Neurol Sci. 1999; 169: 2-12https://doi.org/10.1016/s0022-510x(99)00209-9
- Amyotrophic lateral sclerosis: a consensus viewpoint on designing and implementing a clinical trial.Amyotroph Lateral Scler Other Motor Neuron Disord. 2004; 5: 84-98https://doi.org/10.1080/14660820410020187
- Trial design and reporting standards for intra-arterial cerebral thrombolysis for acute ischemic stroke.Stroke. 2003; 34 (Epub 2003 Jul 17): e109-e137https://doi.org/10.1161/01.STR.0000082721.62796.09
- Measuring what matters in cerebral palsy: a breadth of important domains and outcome measures.Arch Phys Med Rehabil. 2009; 90: 2089-2095https://doi.org/10.1016/j.apmr.2009.06.018
- Pharmacologic management of insomnia in children and adolescents: consensus statement.Pediatrics. 2006; 117: e1223-e1232https://doi.org/10.1542/peds.2005-1693
- 131st ENMC international workshop: selection of outcome measures for peripheral neuropathy clinical trials 10-12 December 2004, Naarden, The Netherlands.Neuromuscul Disord. 2006; 16 (Epub 2006 Jan 23): 149-156https://doi.org/10.1016/j.nmd.2005.12.003
- 136th ENMC International Workshop: Charcot-Marie-Tooth disease type 1A (CMT1A)8-10 April 2005, Naarden, The Netherlands.Neuromuscul Disord. 2006; 16 (Epub 2006 May 8): 396-402https://doi.org/10.1016/j.nmd.2006.03.008
- Recommendations for the use of common outcome measures in traumatic brain injury research.Arch Phys Med Rehabil. 2010; 91: 1650-1660.e17https://doi.org/10.1016/j.apmr.2010.06.033
- Core outcome measures for exercise studies in people with multiple sclerosis: recommendations from a multidisciplinary consensus meeting.Mult Scler. 2014; 20 (Epub 2014 Mar 17): 1641-1650https://doi.org/10.1177/1352458514526944
- A core outcome set for aphasia treatment research: the ROMA consensus statement.Int J Stroke. 2019; 14 (Epub 2018 Oct 10): 180-185https://doi.org/10.1177/1747493018806200
- Core outcome measures for interventions to prevent or slow the progress of dementia for people living with mild to moderate dementia: Systematic review and consensus recommendations.PLoS One. 2017; 12e0179521https://doi.org/10.1371/journal.pone.0179521
- Standardized measurement of sensorimotor recovery in stroke trials: Consensus-based core recommendations from the Stroke Recovery and Rehabilitation Roundtable.Int J Stroke. 2017; 12: 451-461https://doi.org/10.1177/1747493017711813
- Report from the OMERACT Hand Osteoarthritis Working Group: Set of Core Domains and Preliminary Set of Instruments for Use in Clinical Trials and Observational Studies.J Rheumatol. 2015; 42 (NovEpub 2015 Jul 1): 2190-2197https://doi.org/10.3899/jrheum.141017
- Defining appropriate outcome measures in pulmonary arterial hypertension related to systemic sclerosis: a Delphi consensus study with cluster analysis.Arthritis Rheum. 2008; 59: 867-875https://doi.org/10.1002/art.23718
- Connective tissue disease-associated interstitial lung diseases (CTD-ILD) - report from OMERACT CTD-ILD working group.J Rheumatol. 2015; 42 (Epub 2015 Mar 1): 2168-2171https://doi.org/10.3899/jrheum.141182
- Evaluation of outcomes in community-acquired pneumonia: a guide for patients, physicians, and policy-makers.Lancet Infect Dis. 2003; 3: 476-488https://doi.org/10.1016/s1473-3099(03)00721-7
- Researching breathlessness in palliative care: consensus statement of the National Cancer Research Institute Palliative Care Breathlessness Subgroup.Palliat Med. 2009; 23 (Epub 2009 Feb 27): 213-227https://doi.org/10.1177/0269216309102520
- A systematic review of outcome measures used in forensic mental health research with consensus panel opinion.Health Technol Assess. 2010; 14: 1-94https://doi.org/10.3310/hta14180
- Progress towards a core set of outcome measures in small-vessel vasculitis. Report from OMERACT 9.J Rheumatol. 2009; 36: 2362-2368https://doi.org/10.3899/jrheum.090373
- Core outcome domains and measures for pediatric acute and chronic/recurrent pain clinical trials: PedIMMPACT recommendations.J Pain. 2008; 9 (Epub 2008 Jun 17): 771-783https://doi.org/10.1016/j.jpain.2008.04.007
- Proposed preliminary core set measures for disease outcome assessment in adult and juvenile idiopathic inflammatory myopathies.Rheumatology (Oxford). 2001; 40: 1262-1273https://doi.org/10.1093/rheumatology/40.11.1262
- Recommendation for measuring clinical outcome in distal radius fractures: a core set of domains for standardized reporting in clinical practice and research.Arch Orthop Trauma Surg. 2014; 134 (Epub 2013 Jun 1): 197-205https://doi.org/10.1007/s00402-013-1767-9
- Primary outcomes for resuscitation science studies: a consensus statement from the American Heart Association.Circulation. 2011; 124 (Epub 2011 Oct 3): 2158-2177https://doi.org/10.1161/CIR.0b013e3182340239
- The promise of PROMIS: using item response theory to improve assessment of patient-reported outcomes.Clin Exp Rheumatol. 2005; 23: S53-S57
- Recommendations for a first core outcome measurement set for complex regional pain syndrome clinical studies (COMPACT).Pain. 2017; 158: 1083-1090https://doi.org/10.1097/j.pain.0000000000000866
- Core outcome measurement instruments for clinical trials in nonspecific low back pain.Pain. 2018; 159: 481-495https://doi.org/10.1097/j.pain.0000000000001117
- Core Outcome Set-STAndards for Development: The COS-STAD recommendations.PLoS Med. 2017; 14e1002447https://doi.org/10.1371/journal.pmed.1002447
- What is the evidence for the performance of generic preference-based measures? A systematic overview of reviews.Eur J Health Econ. 2018; 19 (Epub 2017 May 30): 557-570https://doi.org/10.1007/s10198-017-0902-x
- How to select outcome measurement instruments for outcomes included in a "Core Outcome Set" - a practical guideline.Trials. 2016; 17: 449https://doi.org/10.1186/s13063-016-1555-2
- A scoping review of core outcome sets and their 'mapping' onto real-world data using prostate cancer as a case study.BMC Med Res Methodol. 2020; 20: 41https://doi.org/10.1186/s12874-020-00928-w
Article info
Publication history
Published online: March 05, 2021
Accepted:
March 2,
2021
Footnotes
Conflict of interest: The authors do not declare any conflict of interest in relation to the present study.
Authors’ contributions: O.C., M.S.K., M.M. contributed to the conceptualization and methodology. O.C., M.S.K., M.M., K.S., S.G., S.D. performed the analyses. O.C., M.S.K., M.M. wrote the original draft. K.S., S.G., S.D., P.W., G.F. reviewed and edited the draft. G.F. acquired financial support for the project.
Funding: This project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 116055 (Big Data for Better Outcomes DO-IT). This Joint Undertaking receives support from the European Union's Horizon 2020 Research and Innovation programme and the European Federation of Pharmaceutical Industries and Associations (EFPIA). This work was also supported by a National Institute for Health Research (NIHR) Senior Investigator Award (NF-SI_0513-10025) and the MRC Trials Methodology Research Partnership (grant reference MR/S014357/1).
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