Founded in 2008, Patient-Reported Outcome (PRO) Consortium supports patient-focused drug development by obtaining qualification of clinical outcome assessment tools that measure how patients feel and function in their everyday lives as a result of treatment.
There are many disease areas for which “fit-for-purpose” PRO measures and other COAs re not available. The development of PRO measures and other COAs can be resource-intensive and time-consuming due to the extensive research and testing process. The development of multiple measures for the same concept of interest and context of use complicates the regulatory review process and limits the ability to compare results across clinical trials using different measures.
PRO measures assess one or more aspects of a patient’s health status based on information gathered directly from the patient, without interpretation by clinicians or others. Patients provide information concerning the impact of an intervention from their perspective. Other COAs include clinician-reported outcome (ClinRO) measures, observer-reported outcome (ObsRO) measures, and performance outcome (PerfO) measures. These tools are used in diseases or conditions where patient self-report is not possible or not sufficient, or where other assessments may measure the concept of interest more accurately. COAs offer a means for capturing clinical benefit in terms of how a patient feels or functions as a result of a therapeutic intervention.
To address the challenges of COA development by individual sponsors, a pre-competitive consortium approach brings together interested stakeholders to develop COAs jointly by sharing resources and expertise in the process. One way to ensure regulatory endorsement of COAs is to pursue qualification by regulatory agencies, such as through the U.S. Food and Drug Administration’s (FDA’s) COA Qualification Program. To accomplish these objectives, the PRO Consortium’s mission is to establish and maintain a collaborative and pre-competitive framework with appropriate stakeholders for the qualification of PRO measures and other COAs that will be publicly available for use in clinical trials to construct COA-based endpoints to support product labeling claims. The Patient-Reported Outcome (PRO) Consortium was formed in late 2008 by the Critical Path Institute (C-Path) in cooperation with FDA’s Center for Drug Evaluation and Research and the pharmaceutical industry, and formally launched in March 2009. The PRO Consortium’s membership is comprised of pharmaceutical companies along with C-Path as the managing member. Patients, clinicians, measurement consultants and representatives from FDA and the National Institutes of Health (NIH) provide critical advice and assistance to the PRO Consortium’s Coordinating Committee and working groups.
Since its launch, the following measures developed by the PRO Consortium have been qualified by FDA for use in clinical trials where COA-based endpoints can be used to support product labeling claims:
Additionally, the following measure licensed by the PRO Consortium is listed in FDA’s COA Compendium as recommended for use:
For additional information on any of the above measures or to license the measure, please visit: https://www.c-pathcoas.org.
The availability of these measures has advanced the incorporation of the patient’s voice by sponsors’ use of these licensed measures in treatment trials for drug development. As an example, clinical study results from the abdominal symptom scale of the Diary for Irritable Bowel Syndrome Symptoms-Constipation (DIBSS-C) were included in the expanded label for the drug LINZESS® (linaclotide) in 2020.
| Title | Journal | Publication Date | Authors | Link |
|---|---|---|---|---|
| Approaches to the Assessment of Clinical Benefit of Treatments for Conditions That Have Heterogeneous Symptoms and Impacts: Potential Applications in Rare Disease | Value in Health | 2023 | Murray LT, Howell TA, Matza LS, Eremenco S, Adams HR, Trundell D, Coons SJ | https://doi.org/10.1016/j.jval.2022.11.012 |
| Best Practice Recommendations for Electronic Patient-Reported Outcome (ePRO) Dataset Structure and Standardization to Support Drug Development | Value in Health | 2023 | Hudgens S, Kern S, Barsdorf AI, Cassells S, Rowe A, King-Kallimanis BL, Coon C, Low G, Eremenco S | https://doi.org/10.1016/j.jval.2023.02.011 |
| Best practice recommendations: user acceptance testing for systems designed to collect clinical outcome assessment data electronically | Therapeutic Innovation & Regulatory Science | 2022 | Gordon S, Crager J, Howry C, Barsdorf A, Cohen J, Crescioni M, Dahya B, Delong P, Knaus C, Reasner D, Vallow S, Zarzar K, Eremenco S, on behalf of the Patient-Reported Outcome (PRO) Consortium and eCOA Consortium at the Critical Path Institute | https://doi.org/10.1007/s43441-021-00363-z |
| Comparability of a provisioned device versus bring your own device for completion of patient-reported outcome measures by participants with chronic obstructive pulmonary disease: qualitative interview findings | Journal of Patient-Reported Outcomes | 2022 | Newton L, Knight-West O, Eremenco S, Hudgins S, Crescioni M, Symonds T, Reasner DS, Byrom B, O’Donohoe P, Vallow S on behalf of the Patient-Reported Outcome Consortium and the Electronic Clinical Outcome Assessment Consortium | https://doi.org/10.1186/s41687-022-00492-5 |
| Comparing patient global impression of severity and patient global impression of change to evaluate test–retest reliability of depression, non-small cell lung cancer, and asthma measures | Quality of Life Research | 2022 | Eremenco S, Chen WH, Blum SI, Bush EN, Bushnell DM, DeBusk K, Gater A, Nelsen L, Coons SJ, on behalf of the PRO Consortium’s Communication Subcommittee | https://doi.org/10.1007/s11136-022-03180-5 |
| Non-Small Cell Lung Cancer Symptom Assessment Questionnaire (NSCLC-SAQ): measurement properties and estimated clinically meaningful thresholds from a phase 3 study | JTO Clinical and Research Reports | 2022 | Williams P, Burke T, Norquist J, Daskalopoulou C, Speck R, Samkari A, Eremenco S, Coons SJ | https://doi.org/10.1016/j.jtocrr.2022.100298 |
| Recommendations for the electronic migration and implementation of clinician-reported outcome assessments in clinical trials | Value in Health | 2022 | Romero H, DeBonis D, O’Donohoe P, Wyrwich K, Arnera V, Platko J, Willgoss T, Harris K, Crescioni M, Steele S, Eremenco S, on behalf of the Electronic Patient-Reported Outcome Consortium and the Patient-Reported Outcome Consortium | https://doi.org/10.1016/j.jval.2022.02.012 |
| Asthma Daytime Symptom Diary (ADSD) and Asthma Nighttime Symptom Diary (ANSD): measurement properties of novel patient-reported symptom measures | The Journal of Allergy Clinical Immunology: In Practice | 2021 | Gater A, Nelsen L, Coon C, Eremenco S, O’Quinn S, Khan A, Eckert L, Staunton H, Bonner N, Hall R, Krishnan J, Stoloff S, Schatz M, Haughney J, Coons SJ | https://doi.org/10.1016/j.jaip.2021.11.026 |
| Non-Small Cell Lung Cancer Symptom Assessment Questionnaire (NSCLC-SAQ): psychometric performance and regulatory qualification of a novel patient-reported symptom measure | Current Therapuetic Research | 2021 | Bushnell DM, Atkinson T, McCarrier K, Liepa A, DeBusk K, Coons SJ | https://doi.org/10.1016/j.curtheres.2021.100642 |
| Considerations for development of an evidence dossier to support the use of mobile sensor technology for clinical outcome assessments in clinical trials | Contemporary Clinical Trials | 2020 | Walton MK, Cappelleri JC, Byrom B, Goldsack JC, Eremenco S, Harris D, Potero E, Patel N, Flood E, Daumer M | https://doi.org/10.1016/j.cct.2020.105962 |
| Good practices for the translation, cultural adaptation, and linguistic validation of clinician-reported outcome, observer-reported outcome, and performance outcome measures | Journal of Patient-Reported Outcomes | 2020 | McKown S, Acquadro C, Anfray C, Arnold B, Eremenco S, Giroudet C, Martin M, Weiss D | https://doi.org/10.1186/s41687-020-00248-z |
| Why reinvent the wheel? Use or modification of existing clinical outcome assessment tools in medical product development | Value in Health | 2020 | Papadopoulos E, Bush EN, Eremenco S, Coons SJ | https://doi.org/10.1016/j.jval.2019.09.2745 |
| Assessing test-retest reliability of patient-reported outcome measures using intraclass correlation coefficients: recommendations for selecting and documenting the analytical formula | Quality of Life Research | 2019 | Qin S, Nelson L, McLeod L, Eremenco S, Coons SJ | https://doi.org/10.1007/s11136-018-2076-0 |
| Symptoms of Major Depressive Disorder Scale (SMDDS): performance of a novel patient-reported symptom measure | Value in Health | 2019 | Bushnell DM, McCarrier KP, Bush EN, Abraham L, Jamieson C, McDougall F, Trivedi M, Thase M, Carpenter L, Coons SJ, on behalf of the Patient-Reported Outcome Consortium’s Depression Working Group | https://doi.org/10.1016/j.jval.2019.02.010 |
| Development of a symptom-focused patient-reported outcome measure for functional dyspepsia: the Functional Dyspepsia Symptom Diary (FDSD) | American Journal of Gastroenterology | 2018 | Taylor F, Higgins S, Carson R, Eremenco S, Foley C, Lacy B, Parkman H, Reasner D, Shields A, Tack J, Talley N | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5770596/ |
| Informing the tolerability of cancer treatments using patient-reported outcome (PRO) measures: summary of an FDA and Critical Path Institute workshop | Value in Health | 2018 | Kluetz PG, Kanapuru B, Lemery S, Johnson LL, Fiero M, Arscott K, Barbachano Y, Basch E, Campbell M, Cappelleri JC, Cella D, Cleeland C, Coens C, Daniels S, Denlinger CS, Fairclough DL, Hillard JR, Minasian L, Mitchell SA, O’Connor D, Patel S, Rubin EH, Ryden A, Soltys K, Sridhara R, Thanarajasingam G, Velikova G, Coons SJ | https://doi.org/10.1016/j.jval.2017.09.009 |
| Literature review to assemble the evidence for response scales used in patient-reported outcome measures | Journal of Patient-Reported Outcomes | 2018 | Gries K, Berry P, Harrington M, Crescioni M, Patel M, Rudell K, Safikhani S, Pease S, Vernon M | https://doi.org/10.1186/s41687-018-0056-3 |
| Literature review to characterize the empirical basis for response scale selection in pediatric populations | Journal of Patient-Reported Outcomes | 2018 | Naegeli AN, Hanlon J, Gries KS, Safikhani S, Ryden A, Patel M, Crescioni M, Vernon M | https://doi.org/10.1186/s41687-018-0051-8 |
| Patient-Reported Outcome (PRO) Consortium translation process: consensus development of updated best practices | Journal of Patient-Reported Outcomes | 2018 | Eremenco S, Pease S, Mann S, Berry P, on behalf of the PRO Consortium’s Process Subcommittee | https://doi.org/10.1186/s41687-018-0037-6 |
| Response scale selection in adult pain measures: results from a literature review | Journal of Patient-Reported Outcomes | 2018 | Safikhani S, Gries KS, Trudeau JJ, Reasner D, Rudell K, Coons SJ, Bush EN, Hanlon J, Abraham L, Vernon M | https://doi.org/10.1186/s41687-018-0053-6 |
| Clinically meaningful outcomes in early Alzheimer disease: a consortia-driven approach to identifying what matters to patients | Therapeutic Innovation & Regulatory Science | 2017 | Ropacki MT, Hannesdottir K, Hendrix S, Gordon MF, Stephenson D, Coons SJ, Stern RA, on behalf of the Critical Path Institute’s Coalition Against Major Diseases and Patient-Reported Outcome Consortium Cognition Working Group | https://doi.org/10.1177/2168479016689712 |
| Development of a harmonized patient-reported outcome questionnaire to assess myelofibrosis symptoms in clinical trials | Leukemia Research | 2017 | Gwaltney C, Paty J, Kwitkowski VE, Mesa RA, Dueck AC, Papadopoulos EJ, Wang L, Feliciano J, Coons SJ | https://doi.org/10.1016/j.leukres.2017.05.012 |
| Development of the Diary for Irritable Bowel Syndrome Symptoms (DIBSS) to assess treatment benefit in clinical trials: foundational qualitative research | Value in Health | 2017 | Fehnel SE, Ervin CM, Carson RT, Rigoni G, Lackner JM, Coons SJ, on behalf of the Critical Path Institute Patient-Reported Outcome Consortium’s Irritable Bowel Syndrome Working Group | https://doi.org/10.1016/j.jval.2016.11.001 |
| Assessing asthma symptoms in adolescents and adults: qualitative research supporting development of the Asthma Daily Symptom Diary | Value in Health | 2016 | Gater A, Nelsen L, Fleming S, Lundy JJ, Bonner N, Hall R, Marshall C, Staunton H, Krishnan JA, Stoloff S, Schatz M, Haughney J, on behalf of the Patient-Reported Outcome Consortium’s Asthma Working Group | https://doi.org/10.1016/j.jval.2016.01.007 |
| Development of a patient-reported outcome instrument to assess complex activities of daily living and interpersonal functioning in persons with mild cognitive impairment: The qualitative research phase | Alzheimer’s & Dementia | 2016 | Gordon MF, Lenderking WR, Duhig A, Chandler J, Lundy JJ, Miller DS, Piault-Louis E, Doody RS, Galasko D, Gauthier S, Frank L, on behalf of the Patient-Reported Outcome Consortium’s Cognition Working Group | https://doi.org/10.1016/j.jalz.2015.04.008 |
| Development of a symptom-based patient-reported outcome instrument for functional dyspepsia: a preliminary conceptual model and an evaluation of the adequacy of existing instruments | The Patient—Patient-Centered Outcomes Research | 2016 | Taylor F, Reasner DS, Carson RT, Deal LS, Foley C, Iovin R, Lundy JJ, Pompilus F, Shields AL, Silberg DG | https://link.springer.com/article/10.1007%2Fs40271-016-0164-1 |
| Interpretation of verbal descriptors for response options commonly used in verbal rating scales in patient-reported outcome instruments | Quality of Life Research | 2016 | Mutebi A, Slack M, Warholak TL, Hudgens S, Coons SJ | https://link.springer.com/article/10.1007%2Fs11136-016-1333-3 |
| Interpreting change in scores on patient-reported outcome instruments | Therapeutic Innovation & Regulatory Science | 2016 | Coon CD, Cappelleri JC | https://doi.org/10.1177/2168479015622667 |
| Patient-centered research to support the development of the Symptoms of Major Depressive Disorder Scale (SMDDS): initial qualitative research | The Patient—Patient-Centered Outcomes Research | 2016 | McCarrier KP, Deal LS, Abraham L, Blum SI, Bush EN, Martin M, Thase ME, Coons SJ, on behalf of the PRO Consortium’s Depression Working Group | https://link.springer.com/article/10.1007%2Fs40271-015-0132-1 |
| Practical considerations for the use of clinical outcome assessments (COAs) in pediatric clinical research: examples from pediatric gastroenterology | Therapeutic Innovation & Regulatory Science | 2016 | Kovacs SM, Turner-Bowker DM, Calarco G, Mulberg AE, Paty J | https://doi.org/10.1177/2168479015621601 |
| Qualitative development and content validity of the Non–Small Cell Lung Cancer Symptom Assessment Questionnaire (NSCLC-SAQ), a patient-reported outcome instrument | Clinical Therapeutics | 2016 | McCarrier KP, Atkinson T, DeBusk K, Liepa A, Scanlon M, Coons SJ, on behalf of the Patient-Reported Outcome Consortium’s NSCLC Working Group | https://doi.org/10.1016/j.clinthera.2016.03.012 |
| Capturing patient-reported outcome (PRO) data electronically: the past, present, and promise of ePRO measurement in clinical trials | The Patient—Patient-Centered Outcomes Research | 2015 | Coons SJ, Eremenco S, Lundy JJ, O’Donohoe P, O’Gorman H, Malizia W | https://link.springer.com/article/10.1007%2Fs40271-014-0090-z |
| Considerations for requiring subjects to provide a response to electronic patient-reported outcome instruments | Therapeutic Innovation & Regulatory Science | 2015 | O’Donohoe P, Lundy JJ, Gnanasakthy A, Greene A | https://doi.org/10.1177/2168479015609647 |
| Optimizing electronic capture of clinical outcome assessment data in clinical trials: the case of patient-reported endpoints | Therapeutic Innovation & Regulatory Science | 2015 | Fleming S, Barsdorf AI, Howry C, O’Gorman H, Coons SJ, on behalf of the Patient-Reported Outcome (PRO) Consortium and ePRO Consortium at the Critical Path Institute | https://doi.org/10.1177/2168479015609102 |
| The Patient-Reported Outcome (PRO) Consortium: lessons learned along the path to PRO instrument qualification | Therapeutic Innovation & Regulatory Science | 2015 | Hayes RP, Blum SI, Gordon MF, Piault E, Burke LB, Slagle AF, Coons SJ | https://doi.org/10.1177/2168479014549858 |
| Patient self-report for evaluating mild cognitive impairment and prodromal Alzheimer’s disease | Alzheimer Research and Therapy | 2011 | Frank L, Lenderking WR, Howard K, Cantillon M | https://doi.org/10.1186/alzrt97 |
| The Patient-Reported Outcome (PRO) Consortium: filling measurement gaps for PRO endpoints to support labeling claims | Clinical Pharmacology & Therapeutics | 2011 | Coons SJ, Kothari S, Monz BU, Burke LB | https://doi.org/10.1038/clpt.2011.203 |
| Comparability of a provisioned device versus bring your own device for completion of patient-reported outcome measures by participants with chronic obstructive pulmonary disease: quantitative study findings | Journal of Patient-Reported Outcomes | 2022 | Stacie Hudgens, Louise Newton, Sonya Eremenco, Mabel Crescioni, Tara Symonds, Philip C. G. Griffiths, David S. Reasner, Bill Byrom, Paul O’Donohoe, Susan Vallow on behalf of the Patient-Reported Outcome (PRO) Consortium and Electronic Clinical Outcome Assessment (eCOA) Consortium | https://jpro.springeropen.com/articles/10.1186/s41687-022-00521-3 |
| Updated Recommendations on Evidence Needed to Support Measurement Comparability Among Modes of Data Collection for Patient-Reported Outcome Measures: A Good Practices Report of an ISPOR Task Force | Value in Health | 2023 | O’Donohoe P, Reasner, DS, Kovacs SM, Byrom B, Eremenco S, Barsdorf AI, Arnera V, Coons SJ | https://doi.org/10.1016/j.jval.2023.01.001 |
| Recommendations on the Selection, Development, and Modification of Performance Outcome Assessments: A Good Practices Report of an ISPOR Task Force | Value in Health | 2023 | Edgar CJ, Bush E, Adams HR, Ballinger R, Byrom B, Campbell M, Eremenco S, McDougall F, Papadopoulos E, Slagle AF, Coons SJ | https://doi.org/10.1016/j.jval.2023.05.003 |
| Setting international standards in analyzing patient-reported outcomes and quality of life endpoints in cancer clinical trials-innovative medicines initiative (SISAQOL-IMI): stakeholder views, objectives, and procedures | The Lancet Oncology | 2023 | Pe M, Alanya A, Falk RS, Amdal CD, Bjordal K, Chang J, Cislo P, Coens C, Dirven L, Speck RM, Fitzgerald K, Galinsky J, Giesinger JM, Holzner B, Le Cessie S, O’Connor D, Oliver K, Pawar V, Quinten C, Schlichting M, Ren J, Roychoudhury S, Taphoorn MJB, Velikova G, Wintner LM, Griebsch I, Bottomley A, on behalf of the SISAQOL-IMI Consortium | https://doi.org/10.1016/S1470-2045(23)00157-2 |
| Assessing asthma symptoms in children: qualitative research supporting the development of the Pediatric Asthma Diary-Child (PAD-C) and Pediatric Asthma Diary-Observer (PAD-O) | Journal of Patient-Reported Outcomes | 2023 | Bradley H, Trennery C, Jones AM, Lydon A, White F, Williams-Hall R, Arbuckle R, Tomaszewski E, Shih VH, Haughney J, Eisen A, Winders T, Coons SJ, Eremenco S. on behalf of the Patient-Reported Outcome Consortium’s Pediatric Asthma Working Group | https://jpro.springeropen.com/articles/10.1186/s41687-023-00639-y |
| Flexible approaches to eCOA Administration in clinical trials: The site perspective | Contemporary Clinical Trials Communications | 2023 | Haenel E, Elash C, Garner K, Turner M, Kern S on behalf of the Electronic Clinical Outcome Assessment (eCOA) Consortium and the Patient-Reported Outcome (PRO) Consortium | https://doi.org/10.1016/j.conctc.2023.101241 |
| Best Practice Recommendations for Electronic Clinical Outcome Assessment Data Changes | Journal of the Society for Clinical Data Management | 2023 | Delong P, Humler D, Haag T, Yeomans A, Andrus J, Eremenco S, Finan A, Gable J, Gilfillan D, Howry C, Kern S, Lesniewski S, Simpliciano K, Staunton H, Turnbull J, Workman C, Raymond S. | https://doi.org/10.47912/jscdm.249 |
| Flexible Approaches to eCOA Administration in Clinical Trials: The Site Perspective | Contemporary Clinical Trials Communications | December 7, 2023 | Haenel E, Elash C, Garner K, Turner M, Kern S on behalf of the Electronic Clinical Outcome (eCOA) Consortium and the Patient-Reported Outcome (PRO) Consortium | https://doi.org/10.1016/j.conctc.2023.101241 |
eCOA: Getting Better Together Initiative This initiative is a pre-competitive collaboration among Critical Path Institute, clinical trial sponsors from the Patient-Reported Outcome (PRO) Consortium, providers of electronic data collection technologies and services from the Electronic Clinical Outcome Assessment (eCOA) Consortium, contract research organizations, and regulators. The initiative was launched in 2019 to identify and address the root cause of challenges with the implementation of clinical outcome assessments collected electronically in clinical trials, elevate eCOA improvement efforts to the clinical trial industry level, and drive positive and lasting change in the eCOA ecosystem.
Quarterly Update
Please click here to view the most recent quarterly update, which includes the status of current and future areas of focus.
| Name | Description | Links |
| eCOA Lexicon | Without a common lexicon among eCOA vendors, sponsors, and regulators, the chance for miscommunication, errors, and inefficiencies increases. The objective of this team is to review the terminology and create an aligned eCOA Lexicon for use by stakeholders across the eCOA ecosystem. | eCOA Lexicon |
| eCOA: Process/Workflow and Roles/Responsibilities | Define an eCOA process and workflow that aligns expectations for successful eCOA strategy development and deployment and clarifies roles and responsibilities. | Abbreviations Table |
| Best Practice Recommendations: User Acceptance Testing for Systems Designed to Collect Clinical Outcome Assessment Data Electronically | This article provides best practice recommendations for user acceptance testing to support a high quality eCOA system and ensure reliable and more complete data are collected during the study. | Therapeutic Innovation & Regulatory Science |
| Best Practice Recommendations for Electronic Patient-Reported Outcome (ePRO) Dataset Structure and Standardization to Support Drug Development | This article discusses best practice recommendations covering the application of CDISC standards for ePRO Datasets, involvement of key stakeholders, and strategies to manage ePRO dataset content, quality control, and validation. | Value in Health |
| Best Practice Recommendations for Electronic Clinical Outcome Assessment Data Changes | This article presents key best practices that are aligned to the latest health authority guidance. From 2018 to 2022, 45 representatives of C-Path’s Patient-Reported Outcome Consortium, eCOA Consortium, and the eClinical Forum collaborated to develop guidelines on changes to eCOA data. The resulting core principles can become the foundation upon which sponsors, investigators, and eCOA providers can evaluate and manage data change requests. | Best Practice Recommendations for Electronic Clinical Outcome Assessment Data Changes |
| Flexible approaches to eCOA administration in clinical trials: The site perspective | This article presents results of a survey of eCOA-experienced clinical trial sites to understand their challenges and gather suggestions for simplification that may result in a more flexible, participant- and site-centric approach to ePRO implementation in clinical trials. | Contemporary Clinical Trials Communications |
| Bring Your Own Device (BYOD) | This podcast discusses the use of BYOD approaches for the collection of eCOA data in clinical trials. The podcast offers a review of lessons learned from a year-long initiative that is investigating both what is needed to implement BYOD approaches as well as what can be done to accelerate the adoption of BYOD approaches in clinical trials. | Bring Your Own Device (BYOD) |
Overview
Despite a number of available safe and effective therapies to treat asthma, a high proportion of persons with asthma remain symptomatic and at risk for exacerbations. When the Asthma Working Group was formed in 2012, no standard patient-reported outcome (PRO) measure existed that was considered adequate for measuring important patient-experienced aspects of the disease. Such a measure could be used in addition to spirometric assessment of lung function in the development of drugs for the treatment of persistent asthma. The Asthma Working Group developed the Asthma Daytime Symptom Diary (ADSD) and the Asthma Nighttime Symptom Diary (ANSD) to enable the capture of the core symptoms of asthma in adolescents (12 to 17 years old) and adults (18 years and older) for the assessment of clinical benefit in asthma treatment trials. In March 2019, the ADSD and ANSD were qualified by FDA for use in drug development. The Qualification Statement can be found by searching the new CDER & CBER Drug Development Tool Qualification Project Search Database. Further information about the ADSD and ANSD and how to license them is available at: https://www.c-pathcoas.org.
Publications
Working Group Sponsors
The following firms provided financial support for Asthma Working Group:
Actelion (now part of Janssen Pharmaceutical Companies of Johnson & Johnson)
Allergan (now AbbVie, Inc.)
Amgen Inc.
AstraZeneca Pharmaceuticals LP
Boehringer Ingelheim Pharmaceuticals Inc.
Genentech, Inc.
GlaxoSmithKline plc
Ironwood Pharmaceuticals, Inc.
Janssen Pharmaceutical Companies of Johnson & Johnson
Merck & Co., Inc.
Novartis Pharmaceutical Corporation
Pfizer Inc.
Sanofi
Overview
There is an acute need for therapeutic interventions that can slow or halt the progression of Alzheimer’s disease (AD), and it is currently believed that intervention is necessary at a very early stage of the disease. Assessment of the impact of subtle cognitive changes in early AD on a patient’s ability to perform the daily activities necessary to remain independent may be a critical tool for measuring effects of potential therapeutic agents being tested in clinical trials. The Cognition Working Group is gathering evidence to support the Virtual Reality Functional Capacity Assessment Tool-Short List Mild Cognitive Impairment (VRFCAT-SL MCI) as a performance outcome (PerfO) measure for assessing the ability to perform instrumental activities of daily living in people with mild cognitive impairment (MCI) due to AD, currently defined as Stage 2 to 3 AD. This measure would be used to support labeling claims of therapies aimed at preventing or delaying the progression of cognitive impairment in persons with AD.
Publications
Working Group Sponsors
The following firms provided financial support for the Cognition Working Group:
AbbVie, Inc.
AstraZeneca Pharmaceuticals LP
Boehringer Ingelheim Pharmaceuticals, Inc.
Eli Lilly and Company
Eisai
Genentech, Inc.
Merck & Co., Inc.
Novartis Pharmaceutical Corporation
Pfizer Inc.
Regeneron Pharmaceuticals, Inc.
Sanofi
Overview
Chronic heart failure (CHF) is a condition in which the heart is unable to pump enough blood to meet the body’s needs. People with CHF experience signs and symptoms that include, but are not limited to, shortness of breath, fatigue, and swelling. Although a variety of patient-reported outcome (PRO) measures exist that are aimed at assessing symptoms, physical activity/physical capacity, and other aspects of quality of life, they were deemed to be insufficiently targeted at the most important, most proximal and, potentially, most treatable consequences of CHF. In addition, reliable and accurate measurement of specific aspects of functional capacity, such as physical activity in daily life outside of the clinic, could be enhanced by a more objective measurement approach such as by using an activity monitor. The CHF Working Group aims to obtain FDA qualification of two PRO measures—Chronic Heart Failure Symptom Scale (CHF-SS) and Chronic Heart Failure Impact Scale (CHF-IS)—and an activity monitor-based endpoint measure of physical activity for adults with CHF. The goal is to incorporate both patient-reported and activity monitor-derived data into the assessment of clinical benefit in CHF treatment trials.
Working Group Sponsors
The following firms provided financial and/or in-kind support for the CHF Working Group:
Amgen Inc.
AstraZeneca Pharmaceuticals LP
Bayer AG
Bristol Myers Squibb
Eli Lilly and Company
GlaxoSmithKline plc
Janssen Pharmaceutical Companies of Johnson & Johnson
Merck & Co., Inc.
Regeneron Pharmaceuticals, Inc.
Overview
Depression is a highly prevalent and under-treated condition in the U.S. and worldwide. Despite the availability of safe and effective medications, a high proportion of persons with depression do not achieve remission of symptoms even after switching treatments. Adequate assessment of symptom improvement from a patient’s perspective is imperative for the development of novel therapies. The Depression Working Group developed a new PRO measure — the Symptoms of Major Depressive Disorder Scale (SMDDS). In November 2017, the SMDDS was qualified by FDA for use in drug development to measure symptoms of major depressive disorder. The Qualification Statement can be found by searching the new CDER & CBER Drug Development Tool Qualification Project Search Database. Further information about the SMDDS and how to license it is available at: https://www.c-pathcoas.org.
Publications
Working Group Sponsors
The following firms provided financial support for the Depression Working Group:
AbbVie, Inc.
Allergan (now AbbVie, Inc.)
Eli Lilly and Company
Genentech, Inc.
Janssen Pharmaceutical Companies of Johnson & Johnson
Pfizer Inc.
Sunovion Pharmaceuticals Inc.
Takeda
Overview
With FDA qualification of the Symptoms of Major Depressive Disorder Scale (SMDDS), the Depression Working Group embarked on a new qualification initiative that was driven by growing recognition of the need for assessment tools that can measure antidepressant effects within shorter time frames, potentially within hours or days rather than weeks. Trials are being conducted that could benefit from well-defined and reliable PRO measures of faster onset of symptom relief in persons with major depressive disorder (MDD). Although the SMDDS, with its 7-day recall period, will continue to be a relevant outcome measure for MDD treatment trials, this initiative’s goal is to leverage what was learned in its development to create a 24-hour recall version—the Symptoms of Major Depressive Disorder Diary (SMDDD)—and an “at this moment” version—the Symptoms of Major Depressive Disorder Momentary Assessment (SMDDMA).
Working Group Sponsors
The following firms provided financial support for the Depression Working Group 2.0:
AbbVie, Inc.
Boehringer Ingelheim Pharmaceuticals, Inc.
Janssen Pharmaceutical Companies of Johnson & Johnson
Overview
Functional dyspepsia is defined by the absence of any organ, systemic, or metabolic disease likely to explain symptoms thought to originate in the gastroduodenal region. It is estimated that 3% to 40% of the general population experience this condition, which reflects the variability in diagnostic criteria used in clinical studies. The Functional Dyspepsia Working Group has developed a PRO measure, the Functional Dyspepsia Symptom Diary (FDSD), to assess symptoms related to the two functional dyspepsia subtypes: postprandial distress syndrome (PDS) and epigastric pain syndrome (EPS). The FDSD was developed for use in clinical trials to assess clinically meaningful relief of FD symptoms and to support labeling claims.
Publications
Working Group Sponsors
The following firms provided financial support for the FD Working Group:
Allergan (now AbbVie, Inc.)
Ironwood Pharmaceuticals, Inc.
Shire (now Takeda)
Overview
Development of therapies to treat irritable bowel syndrome (IBS) depends upon critical communication from the patient about symptoms such as abdominal pain and bloating that cannot be measured any other way. There is an abundance of literature on available outcome measures in IBS, however, the development and testing methods used with these measures may not meet FDA’s regulatory expectations. Hence, the IBS Working Group has developed new PRO measures for each of the three main IBS subtypes: Diary for Irritable Bowel Syndrome Symptoms – Constipation (DIBSS-C) for constipation-predominant IBS, DIBSS-D for diarrhea-predominant IBS, and DIBSS-M for mixed or alternating IBS. These measures will be used to assess primary efficacy endpoints in treatment trials for IBS.
In December 2020, the DIBSS-C was qualified by FDA to support symptom-based efficacy endpoints in clinical trials for products intended to treat constipation-predominant IBS (IBS-C) in adults. The Qualification Statement can be found by searching the new CDER & CBER Drug Development Tool Qualification Project Search Database. Further information about the DIBSS-C and how to license it is available at: https://c-pathcoas.org.
Publications
Working Group Sponsors
The following firms provided financial support for the IBS Working Group:
Allergan (now AbbVie, Inc.)
Ironwood Pharmaceuticals, Inc.
Takeda
Overview
Although clinician-reported disability and relapse are the mainstay of efficacy endpoints for multiple sclerosis (MS) trials, a measurement gap exists in the assessment of patient-reported MS-related symptoms and functional impact. Direct self-reports from persons with MS regarding symptom experience and daily functioning could provide a valuable complement to disability-and-relapse-related endpoints. In addition, incorporating MS patients’ voices into the evaluation of new medical products is consistent with the increasing emphasis on patient-centered outcomes research and FDA’s patient-focused drug development initiative. The MS Working Group aims to generate evidence to support standardized PRO measures to assess fatigue and physical function that will meet the requirements for FDA qualification. The current focus of the MS Working Group is the qualification of the PROMIS® Short Form v1.0—Fatigue-Multiple Sclerosis 8a (PROMIS FatigueMS—8a) and the PROMISnq Short Form v2.0 – Physical Function – Multiple Sclerosis 15a (PROMISnq PFMS—15a) as endpoint measures in MS clinical trials.
Working Group Sponsors
The following firms provided financial and in-kind support for the MS Working Group:
AbbVie, Inc.
Actelion (now part of Janssen Pharmaceutical Companies of Johnson & Johnson)
EMD Serono, an affiliate of Merck KGaA Germany
Genentech, Inc.
Novartis Pharmaceutical Corporation
Sanofi
Overview
Myelofibrosis is a rare bone marrow cancer that impairs the body’s ability to produce normal blood cells. As a result, excessive scarring (fibrosis) forms in the bone marrow and a cardinal sign is an enlarged spleen (splenomegaly). People with myelofibrosis may suffer from symptoms that include fatigue, abdominal discomfort, bone pain, night sweats, itching, and early satiety. Multiple versions of symptom assessment questionnaires have been used in myelofibrosis drug development programs. The Myelofibrosis Working Group created a single harmonized, consensus-defined symptom assessment questionnaire, the Myelofibrosis Symptom Assessment Form (MFSAF v4.0) based on available empirical evidence. It can be used as an endpoint measure in myelofibrosis treatment trials to assess clinical benefit. Further information about the MFSAF v4.0 and how to license it is available at: https://www.c-pathcoas.org.
Publications
Working Group Sponsors
The following firms provided financial support for the Myelofibrosis Working Group:
CTI BioPharma
Janssen Pharmaceutical Companies of Johnson & Johnson
Overview
Lung cancer is the most common cancer worldwide and non-small cell lung cancer (NSCLC) is the most common type of lung cancer. Substantial unmet medical need exists in this patient population, and the survival advantage of different treatment regimens has, historically, been small. Thus, detection of therapeutic impact that can palliate potentially debilitating symptoms is critical. The NSCLC Working Group has developed a patient-reported symptom measure— the Non-Small Cell Lung Cancer Symptom Assessment Questionnaire (NSCLC-SAQ)— for use in supporting labeling claims of clinical benefit. In April 2018, the NSCLC-SAQ was qualified by FDA for use in drug development to measure overall symptom severity in adults with advanced non-small cell lung cancer. The Qualification Statement can be found by searching the new CDER & CBER Drug Development Tool Qualification Project Search Database. Further information about the NSCLC-SAQ and how to license it is available at: https://www.c-pathcoas.org.
Publications
Working Group Sponsors
The following firms provided financial support for the NSCLC Working Group:
AbbVie, Inc.
AstraZeneca Pharmaceuticals LP
Boehringer Ingelheim Pharmaceuticals, Inc.
Bristol Myers Squibb
Eli Lilly and Company
EMD Serono, an affiliate of Merck KGaA Germany
Genentech, Inc.
GlaxoSmithKline plc
Janssen Pharmaceutical Companies of Johnson & Johnson
Merck & Co., Inc.
Novartis Pharmaceutical Corporation
Overview
Although asthma is the most common chronic disease in children, there are no standardized and generally accepted COAs for use in pediatric clinical trials of asthma therapy to support labeling claims. Since younger children may not be able to reliably self-report their symptom experience, pediatric asthma trials can involve the collection of patient-reported symptoms from older children and observer- (e.g., parent, caregiver) reported outcomes (i.e., observable asthma-related signs) for younger children. The Pediatric Asthma Working Group aims to obtain FDA qualification of the Pediatric Asthma Diary-Observer (PAD-O) for parents/caregivers of children 4 through 11 years old and the Pediatric Asthma Diary-Child (PAD-C) for self-report by children 8 through 11 years old. These measures are intended to inform efficacy endpoints in pediatric asthma treatment trials.
Publications
Working Group Sponsors
The following firms provided financial support for the Pediatric Asthma Working Group:
AstraZeneca Pharmaceuticals LP
GlaxoSmithKline plc
Novartis Pharmaceutical Corporation
Overview
Rheumatoid Arthritis (RA) affects about 1% of the population in Western countries and is characterized by painful inflammation of the joints that leads to physical disability. The objective of the RA Working Group is to generate evidence to support a PRO measure that will support the evaluation of clinical benefit in treatment trials for persons with mild to severe RA, with the intention to support claims in product labeling. Current RA clinical trial endpoints don’t reflect all concepts deemed important to persons with RA, including fatigue. The current focus of the RA Working Group is the qualification of the PROMIS® Fatigue Short Form 10a as an endpoint measure of fatigue severity in RA clinical trials.
Working Group Sponsors
The following firms provided financial support for the RA Working Group:
AbbVie, Inc.
Boehringer Ingelheim Pharmaceuticals, Inc.
Eli Lilly and Company
EMD Serono, an affiliate of Merck KGaA Germany
GlaxoSmithKline plc
Novo Nordisk
Takeda
UCB Pharma Ltd.
Overview
Lung cancer is the most common cancer worldwide, and small cell lung cancer (SCLC) is the most aggressive form. About 15% of all lung cancer cases are SCLC; the two stages of SCLC are limited (cancer that is only in the chest and can be treated with radiation therapy) and extensive (cancer that has spread outside the area that can be covered by radiation). As most current therapies for limited and extensive stage SCLC are not curative and survival rates remain low, any new therapy should demonstrate meaningful relief from distressing disease-related symptoms.
While reliable and responsive patient-reported outcome (PRO) measures exist for the assessment of lung cancer symptoms, we know of none that were developed specifically for SCLC nor that have met the current regulatory expectations for supporting an FDA-approved labeling claim. With FDA qualification of the Non-Small Cell Lung Cancer Symptom Assessment Questionnaire (NSCLC-SAQ) in April 2018, the SCLC Working Group was convened to work toward FDA qualification of a patient-reported measure of SCLC symptom severity for use in assessing clinical benefit in SCLC treatment trials among both limited and extensive stage patient populations.
Working Group Sponsors
The following firms provided financial support for the SCLC Working Group:
Amgen Inc.
AstraZeneca Pharmaceuticals LP
Boehringer Ingelheim Pharmaceuticals, Inc.
Bristol Myers Squibb
Eli Lilly and Company
EMD Serono, an affiliate of Merck KGaA Germany
Genentech, Inc.
GlaxoSmithKline plc
Janssen Pharmaceutical Companies of Johnson & Johnson
Jazz Pharmaceuticals
Merck & Co., Inc.
Novartis Pharmaceutical Corporation
Sonya Eremenco, MA
Executive Director, Patient-Reported Outcome (PRO) Assessment Consortium
Scottie Kern
Executive Director, Electronic Clinical Outcome Assessment (eCOA) Consortium
Maria Mattera, MPH
Scientific Director
Christian Noll, MBA, PMP
Associate Director, eCOA Consortium
April Hawthorne, MEd
Senior Project Manager
Tarryn Ho, MPH
Senior Project Manager
Janelle Russell
Senior Project Coordinator
Theresa “T” Griffey, MBA, PMP
Associate Director, Clinical Outcome Assessment (COA) Program
Cheryl D. Coon, PhD
Vice President, Clinical Outcome Assessment (COA) Program
Robyn T. Carson, MPH
Vice President & Head, Patient-Centered Outcomes Research, Abbvie
