Mühlbacher AC, Nübling M. Analysis of physicians’ perspectives versus patients’ preferences: direct assessment and discrete choice experiments in the therapy of multiple myeloma. Eur J Health Econ. 2011;12:193–203.
Mavris M, Furia Helms A, Bere N. Engaging patients in medicines regulation: a tale of two agencies. Nat Rev Drug Discov. 2019;18:885–6.
Smith H, Horobin A, Fackrell K, Colley V, Thacker B, Hall DA. Defining and evaluating novel procedures for involving patients in core outcome set research: creating a meaningful long list of candidate outcome domains. Res Involv Engagem. 2018;4:1–12.
Fagotto E, Burgués VA, Fung A. A Taxonomy to Engage Patients: Objectives, Design, and Patient Activation. NEJM Catalyst. Massachusetts Medical Society; 2019;5. https://catalyst.nejm.org/doi/abs/10.1056/CAT.19.0626.
Vaughn Y, Richmond A, Simpson C, Israel T, Boone L. Comparing methods to make research more patient-centered. 2017. https://doi.org/10.25302/12.2019.ME.130603342. Accessed 5 Apr 2021.
Patient-Centered Outcomes Research Institute (PCORI). Finding the keys to a longer, better life after stroke. 2017. https://www.pcori.org/research-results/pcori-stories/finding-keys-longer-better-life-after-stroke. Accessed 15 Feb 2022.
Arthritis Foundation. Externally-led juvenile idiopathic arthritis patient: focused drug development meeting report. 2019. https://www.arthritis.org/getmedia/25118249-ea68-45b5-bfe4-c20904ddc32c/FINAL-JIA-PFDD.pdf. Accessed 15 Feb 2022.
Innovation and Value Initiative. IVI methods summit: defining needs and progress toward improving methods in value assessment: convening proceedings report. 2020. https://www.thevalueinitiative.org/wp-content/uploads/2020/05/Methods-Summit-Report_FINAL_Digital.pdf. Accessed 17 Feb 2022.
Linthicum MT, dosReis S, Slejko JF, Mattingly TJ, Bright JL. The importance of collaboration in pursuit of patient-centered value assessment. Patient. 2021;14:381–4.
Facey K. Patient involvement in HTA: what added value? Pharm Policy Law. 2011;13:245–51.
Berglas S, Jutai L, MacKean G, Weeks L. Patients’ perspectives can be integrated in health technology assessments: an exploratory analysis of CADTH common drug review. Res Involv Engagem. 2016;2:1–13.
Diaby V, Ali AA, Montero AJ. Value assessment frameworks in the United States: a call for patient engagement. Pharmacoecon Open. 2019;3(1):1–3.
US Food and Drug Administration. Patient-focused drug development: collecting comprehensive and representative input guidance for industry, Food and Drug Administration staff, and other stakeholders. 2020. pp. 4–5. https://www.fda.gov/media/139088/download. Accessed 30 Apr 2022.
National Institute for Health and Care Excellence. NICE health technology evaluations: the manual. 2022. https://www.nice.org.uk/process/pmg36/resources/nice-health-technology-evaluations-the-manual-pdf-72286779244741. Accessed 30 Apr 2022.
National Institute for Health and Care Excellence. Patient and public involvement policy. 2013. https://www.nice.org.uk/media/default/About/NICE-Communities/Public-involvement/Patient-and-public-involvement-policy/Patient-and-public-involvement-policy-November-2013.pdf. Accessed 17 Feb 2022.
Patient-Centered Outcomes Research Institute (PCORI). Patient-centered outcomes research. 2014. https://www.pcori.org/research-results/about-our-research/patient-centered-outcomes-research. Accessed 3 Aug 2020.
National Health Council. The National Health Council rubric to capture the patient voice: a guide to incorporating the patient voice into the health ecosystem. 2019. https://www.nationalhealthcouncil.org/sites/default/files/NHC_Patient_Engagement_Rubric.pdf. Accessed 30 Apr 2022.
Edwards HA, Huang J, Jansky L, Mullins CD. What works when: mapping patient and stakeholder engagement methods along the ten-step continuum framework. J Comp Eff Res. 2021. https://www.futuremedicine.com/doi/full/https://doi.org/10.2217/cer-2021-0043. Accessed 28 Jun 2021.
National Health Council. Glossary of patient engagement terms. 2019. https://nationalhealthcouncil.org/glossary-of-patient-engagement-terms/. Accessed 4 Aug 2021.
Health Technology Assessment iNternational (HTAi). Resources & materials. 2022. https://htai.org/interest-groups/pcig/resources/. Accessed 17 Feb 2022.
Canadian Agency for Drugs and Technologies in Health (CADTH). Patient input and feedback. 2021. https://www.cadth.ca/patient-input-and-feedback. Accessed 17 Feb 2022.
Myeloma Patients Europe. Evidence Generation Unit (EGU). 2020. https://www.mpeurope.org/evidence-generation-unit-egu/. Accessed 15 Feb 2022.
McCorkell L, Assaf G, Davis H, Wei H, Akrami A. Patient-led research for COVID-19: embedding patients in the long COVID narrative. OSF Preprints. 2020.
Public Law No: 115-52-H.R.2430-FDA Reauthorization Act of 2017. 301. Sect. 605, 115–52 2017. pp. 102–3. https://www.congress.gov/bill/115th-congress/house-bill/2430. Accessed 30 Apr 2022.
Public Law No: 114-255-H.R.34-21st Century Cures Act. 201, 114–255 2016, pp. 1000–18001. https://www.congress.gov/bill/114th-congress/house-bill/34?q=%7B%22search%22%3A%5B%22To+accelerate+the+discovery%2C+development%2C+and+delivery+of+21st+century+cures%2C+and+for+other+purposes.%22%5D%7D&r=4. Accessed 30 Apr 2022.
Public Law No: 112-144-H.R.5651-Food and Drug Administration Reform Act of 2012. 301. Sect. 901, 112–144 2013, p. 103. https://www.congress.gov/bill/112th-congress/senate-bill/3187/text?q=%7B%22search%22%3A%5B%22S.3187%22%2C%22S.3187%22%5D%7D&r=1&s=5. Accessed 30 Apr 2022.
Innovative Medicines Initiative. Patients Active in Research and Dialogues for an Improved Generation of Medicines (PARADIGM). 2021. https://imi-paradigm.eu/. Accessed 17 Feb 2022.
Innovative Medicines Initiative. The patient preferences in benefit-risk assessments during the drug life cycle (PREFER). Uppsala University, Sweden; 2021. https://www.imi-prefer.eu/. Accessed 17 Feb 2022.
International Council for Harmonisation. ICH reflection paper: proposed ICH guideline work to advance patient focused drug development. 2021. https://admin.ich.org/sites/default/files/2021-06/ICH_ReflectionPaper_PFDD_FinalRevisedPostConsultation_2021_0602.pdf. Accessed 17 Feb 2022.
US Food and Drug Administration. The voice of the patient: a series of reports from the U.S. Food and Drug Administration’s (FDA’s) patient-focused drug development initiative: breast cancer. 2015. https://www.fda.gov/media/93924/download. Accessed 21 May 2019.
US Food and Drug Administration. Externally-led patient-focused drug development meetings. Externally-led patient-focus. Drug development meeting. FDA. 2020. https://www.fda.gov/industry/prescription-drug-user-fee-amendments/externally-led-patient-focused-drug-development-meetings. Accessed 23 Nov 2020.
Patient Focused Medicines Development. Highlighting recent trends in the fast-evolving patient engagement & patient experience data landscape. 2021. https://patientfocusedmedicine.org/docs/Trends-in-the-Fast-Evolving-PE-PED-Landscape.pdf. Accessed 21 Feb 2022.
The European Parliament and the Council of the European Union. Regulation (EU) No 536/2014 of the European Parliament and of the Council of 16 April 2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC. 2014. https://ec.europa.eu/health/system/files/2016-11/reg_2014_536_en_0.pdf. Accessed 17 Feb 2022.
Myotonic Dystrophy Foundation. Voice of the patient report myotonic dystrophy externally-led patient-focused drug development meeting. 2017. https://www.myotonic.org/sites/default/files/MDFVoicePatientReportMay2017.pdf. Accessed 16 Feb 2020.
Medicines and Healthcare Products Regulatory Agency. Patient involvement strategy 2021–25. 2021. https://assets.publishing.service.gov.uk/government/uploads/system/uploads/attachment_data/file/1022370/Patient_involvement_strategy.pdf. Accessed 21 Feb 2022.
Institute for Clinical and Economic Review. 2020–2023 value assessment framework. 2020. http://icer.org/wp-content/uploads/2020/10/ICER_2020_2023_VAF_102220.pdf. Accessed 11 Jan 2021.
Innovation and Value Initiative. Open-source value project model for major depressive disorder health economic module: draft protocol. 2021. https://www.thevalueinitiative.org/wp-content/uploads/2021/12/MDD-Model-Protocol-Draft.pdf. Accessed 24 Feb 2022.
Canadian Agency for Drugs and Technologies in Health (CADTH). Procedures for CADTH reimbursement reviews. 2021. https://www.cadth.ca/sites/default/files/Drug_Review_Process/CADTH_Drug_Reimbursement_Review_Procedures.pdf. Accessed 17 Feb 2022.
Institute for Clinical and Economic Review. Targeted immune modulators for rheumatoid arthritis: effectiveness & value. 2017. http://icerorg.wpengine.com/wp-content/uploads/2020/10/NE_CEPAC_RA_Evidence_Report_FINAL_040717.pdf. Accessed 30 Apr 2022.
Institute for Clinical and Economic Review. Modulator treatments for cystic fibrosis: effectiveness and value: final evidence report and meeting summary. 2020. https://icer.org/wp-content/uploads/2020/08/ICER_CF_Final_Report_092320.pdf. Accessed 17 Feb 2022.
Institute for Clinical and Economic Review. Disease-modifying therapies for relapsing remitting and primary-progressive multiple sclerosis: effectiveness and value. 2017. https://icer.org/wp-content/uploads/2020/10/CTAF_MS_Final_Report_030617.pdf. Accessed 4 Apr 2021.
Whittal A, Meregaglia M, Nicod E. The use of patient-reported outcome measures in rare diseases and implications for health technology assessment. Patient. 2021;14:485–503.
Single A, Facey K. Beyond guidelines: tools to support patient involvement in health technology assessment. Health Technology Assessment international. 2021. p. 17. https://g-i-n.net/wp-content/uploads/2021/06/HTA-final-for-online-publication-.pdf. Accessed 30 Apr 2022.
Sacristán JA, Aguarón A, Avendaño-Solá C, Garrido P, Carrión J, Gutiérrez A, et al. Patient involvement in clinical research: why, when, and how. Patient Prefer Adherence. 2016;10:631.
Heneghan C, Goldacre B, Mahtani KR. Why clinical trial outcomes fail to translate into benefits for patients. Trials. 2017;18:1–7.
Rahimi K, Malhotra A, Banning AP, Jenkinson C. Outcome selection and role of patient reported outcomes in contemporary cardiovascular trials: systematic review. BMJ. 2010;341: c5707.
Mercieca-Bebber R, King MT, Calvert MJ, Stockler MR, Friedlander M. The importance of patient-reported outcomes in clinical trials and strategies for future optimization. Patient Relat Outcome Meas. 2018;9:353.
Wieseler B, Wolfram N, McGauran N, Kerekes MF, Vervölgyi V, Kohlepp P, et al. Completeness of reporting of patient-relevant clinical trial outcomes: comparison of unpublished clinical study reports with publicly available data. PLoS Med. 2013;10: e1001526.
Mercieca-Bebber R, Friedlander M, Calvert M, Stockler M, Kyte D, Kok P-S, et al. A systematic evaluation of compliance and reporting of patient-reported outcome endpoints in ovarian cancer randomised controlled trials: implications for generalisability and clinical practice. J Patient-Rep Outcomes. 2017;1:5.
US Food and Drug Administration. The voice of the patient: a series of reports from FDA’s patient-focused drug development initiative. 2018. https://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm368342.htm. Accessed 30 Apr 2022.
Young B, Bagley H. Including patients in core outcome set development: issues to consider based on three workshops with around 100 international delegates. Res Involv Engagem. 2016;2:25.
Pharmacy Quality Alliance. The patient engagement in quality measurement rubric: a guide to patient partnership in the quality measure lifecycle. 2019. https://www.pqaalliance.org/assets/PQA-Patient-Engagement-Rubric.pdf. Accessed 23 Oct 2019.
O’Kane M, Agrawal S, Binder L, Dzau V, Gandhi TK, Harrington R et al. An equity agenda for the field of health care quality improvement. NAM Perspect. 2021;2021.
Armstrong MJ, Mullins CD, Gronseth GS, Gagliardi AR. Impact of patient involvement on clinical practice guideline development: a parallel group study. Implement Sci. 2018;13:55.
Addario BJ, Fadich A, Fox J, Krebs L, Maskens D, Oliver K, et al. Patient value: perspectives from the advocacy community. Health Expect. 2018;21:57–63.
Heneghan C, Goldacre B, Mahtani KR. Why clinical trial outcomes fail to translate into benefits for patients. Trials. 2017;18:122.
International Society for Pharmacoeconomics and Outcomes Research. ISPOR science strategy. 2021. https://www.ispor.org/strategic-initiatives/science-strategy. Accessed 30 Apr 2022.
Perfetto EM, Oehrlein EM, Boutin M, Reid S, Gascho E. Value to whom? The patient voice in the value discussion. Value Health. 2017;20:286–91.
Williamson PR, Altman DG, Bagley H, Barnes KL, Blazeby JM, Brookes ST, et al. The COMET handbook: version 1.0. Trials. 2017;18:280.
International Consortium for Health Outcomes Measurement (ICHOM). ICHOM standard sets: view our collection. https://www.ichom.org/standard-sets/. Accessed 28 Jul 2020.
Center for Medical Technology Policy (CMTP). coreHEM: developing comparative effectiveness outcomes for gene therapy in hemophilia. 2018. http://www.cmtpnet.org/docs/resources/coreHEM_Final_Report_21_MAY_2018.pdf. Accessed 26 Apr 2019.
Boers M, Kirwan JR, Wells G, Beaton D, Gossec L, d’Agostino M-A, et al. Developing core outcome measurement sets for clinical trials: OMERACT filter 2.0. J Clin Epidemiol. 2014;67:745–53.
Dodd S, Harman N, Taske N, Minchin M, Tan T, Williamson PR. Core outcome sets through the healthcare ecosystem: the case of type 2 diabetes mellitus. Trials. 2020;21:1–7.
Tong A, Manns B, Wang AYM, Hemmelgarn B, Wheeler DC, Gill J, et al. Implementing core outcomes in kidney disease: report of the Standardized Outcomes in Nephrology (SONG) implementation workshop. Kidney Int. 2018;94:1053–68.
Clearfield E, Tambor E, Janssen EM, Messner DA. Increasing the patient-centeredness of health economics and outcomes research through patient engagement in core outcome set development. Patient. 2021;14(4):413–20.
O’Brien N, Chi Y-L, Krause KR. Measuring health outcomes in HIV: time to bring in the patient experience. Ann Glob Health. 2021;87(1):2.
Alioum A, Dabis F, Dequae-Merchadou L, Haverkamp G, Hudgens M, Hughes J, et al. Estimating the efficacy of interventions to prevent mother-to-child transmission of HIV in breast-feeding populations: development of a consensus methodology. Stat Med. 2001;20:3539–56.
Land L, Nixon S, Ross J. Patient-derived outcome measures for HIV services in the developed world: a systematic review. Int J STD AIDS. 2011;22:430–5.
Kloppenburg M, Bøyesen P, Smeets W, Haugen I, Liu R, Visser W, et al. Report from the OMERACT Hand Osteoarthritis Special Interest Group: advances and future research priorities. J Rheumatol. 2014;41:810–8.
Jimenez-Moreno AC, Nikolenko N, Kierkegaard M, Blain AP, Newman J, Massey C, et al. Analysis of the functional capacity outcome measures for myotonic dystrophy. Ann Clin Transl Neurol. 2019;6:1487–97.
Gagnon C, Heatwole C, Hébert LJ, Hogrel J-Y, Laberge L, Leone M, et al. Report of the third outcome measures in myotonic dystrophy type 1 (OMMYD-3) international workshop Paris, France, June 8, 2015. J Neuromuscul Dis. 2018;5:523–37.
Gagnon C, Meola G, Hébert LJ, Laberge L, Leone M, Heatwole C. Report of the second outcome measures in myotonic dystrophy type 1 (OMMYD-2) international workshop San Sebastian, Spain, October 16, 2013. Neuromuscul Disord. 2015;25:603–16.
Gagnon C, Meola G, Hébert LJ, Puymirat J, Laberge L, Leone M. Report of the first outcome measures in myotonic dystrophy type 1 (OMMYD-1) international workshop: Clearwater, Florida, November 30, 2011. Neuromuscul Disord. 2013;23:1056–68.
US Food and Drug Administration. CDER pilot grant program: standard core clinical outcome assessments (COAs) and their related endpoints. FDA. 2019. https://www.fda.gov/drugs/development-approval-process-drugs/cder-pilot-grant-program-standard-core-clinical-outcome-assessments-coas-and-their-related-endpoints. Accessed 1 Sep 2020.
Walton MK, Powers JH III, Hobart J, Patrick D, Marquis P, Vamvakas S, et al. Clinical outcome assessments: conceptual foundation: report of the ISPOR clinical outcomes assessment: emerging good practices for Outcomes Research Task Force. Value Health. 2015;18:741–52.
Oehrlein EM, Perfetto EM, Love TR, Chung Y, Ghafoori P. Patient-reported outcome measures in the Food and Drug Administration pilot compendium: meeting today’s standards for patient engagement in development? Value Health. 2018;21:967–72.
Bansback N, Trenaman L, MacDonald KV, Hawker G, Johnson JA, Stacey D, et al. An individualized patient-reported outcome measure (PROM) based patient decision aid and surgeon report for patients considering total knee arthroplasty: protocol for a pragmatic randomized controlled trial. BMC Musculoskelet Disord. 2019;20:1–10.
Greenhalgh J, Gooding K, Gibbons E, Dalkin S, Wright J, Valderas J, et al. How do patient reported outcome measures (PROMs) support clinician-patient communication and patient care? A realist synthesis. J Patient Rep Outcomes. 2018;2:1–28.
Porter I, Gonçalves-Bradley D, Ricci-Cabello I, Gibbons C, Gangannagaripalli J, Fitzpatrick R, et al. Framework and guidance for implementing patient-reported outcomes in clinical practice: evidence, challenges and opportunities. J Comp Eff Res. 2016;5:507–19.
Donabedian A. The quality of care: how can it be assessed? JAMA. 1988;260:1743–8.
US Food and Drug Administration. The voice of the patient: chronic fatigue syndrome and myalgic encephalomyelitis. 2013. https://www.fda.gov/media/86879/download. Accessed 3 Aug 2020.
National Institutes of Health. FAQs about rare diseases. Genetic and Rare Diseases Information Center (GARD): an NCATS Program. https://rarediseases.info.nih.gov/diseases/pages/31/faqs-about-rare-diseases. Accessed 29 Jun 2021.
FasterCures. FasterCures mobilizes for next phase of work to save lives by improving the global medical R&D system, launches health data initiative. Milken Institute. 2018. https://milkeninstitute.org/article/fastercures-mobilizes-next-phase-work-save-lives-improving-global-medical-rd-system. Accessed 15 Feb 2022.
Morel T, Cano SJ. Measuring what matters to rare disease patients: reflections on the work by the IRDiRC taskforce on patient-centered outcome measures. Orphanet J Rare Dis. 2017;12:1–13.
Lanar S, Acquadro C, Seaton J, Savre I, Arnould B. To what degree are orphan drugs patient-centered? A review of the current state of clinical research in rare diseases. Orphanet J Rare Dis. 2020;15:1–18.
iConquerMS. The Accelerated Cure Project and the Italian Multiple Sclerosis Society collaborate to advance patient-reported outcomes in MS research, patient care and product development. 2018. https://www.iconquerms.org/accelerated-cure-project-and-italian-multiple-sclerosis-society-collaborate-advance-patient-reported. Accessed 3 Dec 2020.
Nowinski CJ, Miller DM, Cella D. Evolution of patient-reported outcomes and their role in multiple sclerosis clinical trials. Neurotherapeutics. 2017;14:934–44.
Pascrell B. H.R.1865-Further Consolidated Appropriations Act, 2020. Public Law No 116-94. Sect. 104, H.R. 1865 Dec 20, 2019. https://www.congress.gov/bill/116th-congress/house-bill/1865/text. Accessed 30 Apr 2022.