LOOKING FOR OPTIMAL STANDARDS, CRITERIA AND KPIS OF PATIENT CENTRICITY ACROSS THE BIOPHARMA INDUSTRY: AN OVERVIEW OF EXISTING FRAMEWORKS AND CONCEPTUAL MODELS (PART 1)
DOI:
https://doi.org/10.24144/2077-6594.2.2020.202481Abstract
The biopharmaceutical industry is still on the path to patient centricity, in which a key challenge is to establish new ways of working, key performance indicators and operational models. There is currently no unified approach to measuring the success of patient engagement and patient centricity across the industry.
Methods. A literature search and broader resource analysis was conducted to identify and select frameworks, conceptual models and other relevant initiatives across the biopharmaceutical industry and healthcare systems focused on performance management and success evaluation within patient centeredness, over the period of 2010-2019. The selected resources were initially assessed against their key characteristics, targeted stakeholders, medicine lifecycle stage and proposed key elements (standards, attributes, criteria, KPIs and other indicators). Following, a deep content-analysis of the key elements was conducted, in which the key elements were categorised based on being attributable or infrastructural/procedural.
Results. 192 results were found through the literature search and 13 from the broader resource analysis. 12 frameworks and conceptual models were identified and selected as relevant; 4 targeted to the industry and 8 targeted to several stakeholders, including industry. Analysis of the selected frameworks/conceptual models demonstrated their significant diversity, heterogeneity and inconsistency in terms of their presented format, purpose, key objectives, targeted medicine lifecycle stage or industry/healthcare operations and proposed key elements. Categorisation of the 30 key elements led to 11 as attributable elements and 19 as infrastructural/procedural elements. Certain key elements featured higher cross-referencing within the analysed frameworks/conceptual models: diversity and representativeness, reciprocity, transparency, capacity and capabilities for engagement, meaningfulness, roles and responsibilities (attributable elements), valued products and quality product information (infrastructural/procedural element).
Conclusions. There is a first attempt to categorise and analyse the frameworks and conceptual models of patient centricity developed over the last decade. These findings should be the subject for further research and publications, in particular with the focus on piloting, validation and broader adoption of certain patient-centric standards, impact indicators and/or KPIs across the several organisations of biopharmaceutical industry.
References
Abelson J (Editorial). Patient engagement in health technology assessment: what constitutes ‘meaningful’ and how we might get there. Journal of Health Services Research &Policy; 2018; 0(0) 1–3.
Aday LA, Andersen R. A Framework for the Study of Access to Medical Care. Health Services Research; 1974 Fall; 9(3): 208–220.
Address to the Medical College of Virginia, Richmond (1 Dec 1950). Quoted in James C. Colling and Jerry I. Porras, Built to Last (1994, 1997), 48.
AIDS info: offering information on HIV/AIDS treatment, prevention and research; Access mode: https://aidsinfo.nih.gov/understanding-hiv-aids/fact-sheets
Being Patient-Centric: An evidence-based self-evaluation toolkit for pharma…as recommended by patient groups. PatientView; November 2017.
Boutin M, Dewulf L, Hoos A, et al. Culture and process change as a priority for patient engagement in medicines development. Ther Innov Regul Sci 2017; 51:29–38.
Code of practice 2019. IFPMA; Access mode: https://www.ifpma.org/wp-content/uploads/2018/09/IFPMA_Code_of_Practice_2019.pdf.
CTTI Clinical Trial Transformation Initiative. CTTI recommendations: effective engagement with patient groups around clinical trials, October 2015. Access mode: https://www.ctti-clinicaltrials.org/files/pgctrecs.pdf.
Deane K, Delbecque L, Gorbenko O, et al. Co-creation of patient engagement quality guidance for medicines development: an international multi-stakeholder initiative. BMJ Innovation 2019; 0:1–13.
Dewulf L. Patient Engagement by Pharma – Why and How? A Framework for Compliant Patient Engagement. Therapeutic Innovation & Regulatory Science.2015; 49 (1):9–16.
Di Masi JA, Grabowski, Hansen RW. Innovation in the pharmaceutical industry: New estimates of R&D costs; J Health Econ. 2016 May; 47:20-33.
Du Plessis D, Sake J-K, Halling K, et al. Patient Centricity and Pharmaceutical Companies: Is It Feasible? Therapeutic Innovation& Regulatory Science, 2017:1-8.
Enhancing productivity in biopharmaceutical R&D. Kinapse White Paper, 2018. Access mode: https://kinapse.com/wp-content/uploads/2018/06/Kinapse-Enhancing-productivity-in-RD.pdf?aliId=eyJpIjoiMFFETHNJcTVhWUtUb1dxMSIsInQiOiJaMlBoMW1QN0dxTFYwM0Uwcng5QkJRPT0ifQ%253D%253D
FastCures, Enhancing Integration of Patient Perspective Data in the Drug Development Process, Proposal for PDUFA VI [Electronic resource]. – Access mode : http://www.nationalhealthcouncil.org/sites/default/files/FasterCures-PDUFA-Comment-Letter-FDA.pdf.
FasterCures Value Framework: Integrating the Patient Perspective into the Development of Value Frameworks, March 2016. Access mode: http://www.fastercures.org/reports/view/56.
FDA Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient’s Voice in Medical Product Development and Regulatory Decision Making; Access mode: https://www.fda.gov/drugs/developmentapproval-process-drugs/fda-patient-focused-drug-development-guidance-series-enhancing-incorporation-patients-voicemedical Assessed June 2019.
Gemeinsamer Bundesausschuss. The patient participation and the leading organizations, 2019 (https://patientenvertretung.g-ba.de/en/).
Gorbenko OV. Beyond the rhetoric and traditional advocacy: Walking the patient-centric talk to deliver meaningful change. Oral presentation at the Patient Summit Europe 2018, 16 October, London; https://eyeforpharma.com/conferencematerial/patient2018.php.
Gorbenko OV. The role of patients and community representatives in value-based assessment of health technologies and respective decision-making processes within healthcare. Economy and legislation of health care 2(4) 2016; 10-23.
Health Collaboration Guide 2017. EFPIA; Access mode: https://www.efpia.eu/media/288506/efpia-healthcollaboration-guide_2017_v13.pdf.
Health Information and Quality Authority. Guidelines for Stakeholder Engagement in Health Technology Assessment in Ireland, 2014 (https://www.hiqa.ie/system/files/HTA-Guidelines-Stakeholder-Engagement.pdf).
Hoos A, Anderson J, Boutin M, et al. Partnering With Patients in the Development and Lifecycle of Medicines: A Call for Action. Ther Innov Regul Sci. 2015 Nov;49(6):929-939.
Houses of Parliament. The Parliamentary Office of Science and Technology, POST note #487 January 2015 Value Based Assessment of Drugs [Electronic resource]. – Access mode: www.parliament.uk/post.
International Advisory Panel on HIV Care Continuum Optimization. IAPAC Guidelines for Optimizing the HIV Care Continuum for Adults and Adolescents. Journal of the International Association of Providers of AIDS Care 2015; 14(Supplement 1) S3–S34.
Lasarus JV, Safreed-Harmon K, Barton SE, et al. Beyond viral suppression of HIV – the new quality of life frontier. BMC Med. 2016 Jun 22;14(1):94.
Leading Strategies for Effective Patient Recruitment and Retention. Principles, Priorities, and Plans for Life Science Organisations. Kinapse White paper 2018; Access mode: https://info.kinapse.com/effective-patient-rentention.html.
Levitan B, Getz K, Eisenstein E, et al. Assessing the Financial Value of Patient Engagement: A Quantitative Approach from CTTI's Patient Groups and Clinical Trials Project. Ther Innov Regul Sci. 2018 Mar;52(2):220-229.
Lobban D, Arnstein L, Wadsworth AC, et al. Plain language summaries of publications: Addressing the HOW via a stakeholder survey and workshop. Poster presentation at the 15th Annual Meeting of ISMPP; April 15-17, 2019; National Harbor, MD, USA.
Lowe MM, Blaser DA, Cone L et al. Increasing Patient Involvement in Drug Development; Value in Health, 2016, V.19; 6: 869-878.
Managing Performance in Patient Centricity. Making the link between value for patients and value for the pharmaceutical industry. A Kinapse white paper; 2015: https://info.kinapse.com/patient-centricity.html.
Marcotullio S, Osorio D, Martini M, et al. Challenges in understanding the health-related quality of life of people living with HIV: a 4th 90 measure in Italy. Journal of HIV and Ageing; 2018;V.3 (3):57-61.
National Health Council (NHC) Framework Dialogue / Advancing Meaningful Patient Engagement in Research, Development, and Review of Drugs, September 22, 2015. Access mode: http://www.nationalhealthcouncil.org/sites/default/files/PatientEngagement-WhitePaper.pdf.
National Institute for Health and Care Excellence. Guide to the methods of technology appraisal, 2013 (https://www.nice.org.uk/process/pmg9/resources/guide-to-the-methods-of-technologyappraisal-2013-pdf-2007975843781)
National Standards for Public Involvement in Research. Multi-stakeholder group: INVOLVE, National Institute for Health Research, 2017; Access mode: https://sites.google.com/nihr.ac.uk/pi-standards/home.
Opportunities for efficiency in R&D; KPMG public document; 2017 (https://home.kpmg/uk/en/home/insights/2017/09/opportunities-for-efficiency-in-r-and-d.html accessed 12 Feb 2019).
Opposites attract: pairing R&D and Commercial teams. Executive Insight public doc; May 05, 2015: https://www.executiveinsight.ch/en/insights/opposites-attract-pairing-rd-and-commercial-teams.
Patient Centricity Frameworks. A practitioner’s guide; Eyeforpharma, December 2017.
Patient Input in Relative Effectiveness Assessments, The European Network for Health Technology Assessment, June 14th 2019, (https://www.eunethta.eu/wp-content/uploads/2019/06/Final_290519_Patient-Input-in-REAs.pdf).
Patient-centric pharma: Advancing the new business model; A First Word Expert Views Dossier Report; October 2016.
PCORI Funded Projects: Sample Engagement Plans From Methods Portfolio, August 6, 2014. Access mode: http://www.pcori.org/sites/default/files/PCORI-Sample-Methods-Engagement-Plans.pdf.
PCORI Patient Engagement Rubric Engagement Rubric for Applicants, Feb 4, 2014 (updated June 6, 2016). Access mode: http://www.pcori.org/sites/default/files/Engagement-Rubric.pdf.
People and Communities Board. Six principles for engaging people and communities: Definitions, evaluation and measurement. Published by the People and Communities Board, with support from National Voices, UK; June 2016; Access mode: https://www.nationalvoices.org.uk/sites/default/files/public/publications/six_principles_-_definitions_evaluation_and_measurement_-_web_high_res_0_1.pdf.
Perfetto et al. Framework: When is evidence sufficient for decision-making? A framework for understanding the pace of evidence adoption, in Journal of Comparative Effectiveness Research, July 2013, Vol. 2, No. 4, Pages 383-391. Access mode: http://www.futuremedicine.com/doi/pdfplus/10.2217/cer.13.39.
Proctor E, Silmere H, Raghavan R, et al. Outcomes for Implementation Research: Conceptual Distinctions, Measurement Challenges, and Research Agenda. Adm Policy Ment Health (2011) 38:65–76.
Pushparajah DS. Making Patient Engagement a Reality. The Patient – Patient-Centered Outcomes Research, 2017, 11(1), 1–8. doi:10.1007/s40271-017-0264-6.
Rogers, E. Diffusion of Innovations. (4th ed.); 1995. New York, NY: The Free Press.
Scott AM, Wale JL; HTAi Patient and Citizen Involvement in HTA Interest Group, Patient Involvement and Education Working Group. Patient advocate perspectives on involvement in HTA: an international snapshot. Res Involv Engagem. 2017;3:2. Published 2017 Jan 10. doi:10.1186/s40900-016-0052-9.
Scottish Medicines Consortium. Making a submission, 2017 (https://www.scottishmedicines.org.uk/media/2792/patient-group-partner-guide.pdf).
Skovlund SE, Gorbenko OV, Richards DP, et al. What are the priority patient engagement activities in medicines development? Results of a global multi-stakeholder consultation. PNS249 Research Poster J12, ISPOR, New Orleans, Louisiana, US; 19-22 May 2019.
Taylor J. Oral presentation at the Patient Summit Europe 2018, session “Discover the organisational blueprint for scaling patient centricity” / Being Patient-Centric: A National Voices Perspective; 16 October, London; https://s3.amazonaws.com/efpharma/patient2018/slides/D2-26+(b)+Jeremy.pdf.
The Canadian Agency for Drugs and Technologies in Health. Providing Input to CADTH, 2019 (https://www.cadth.ca/provide-input).
The French National Authority for Health. Develop quality in the health, social and medico-social field, 2019 (https://www.has-sante.fr/jcms/c_2891593/fr/conseil-pour-l-engagement-des-usagers; https://www.has-sante.fr/jcms/c_415958/en/mission-and-budget).
The PFMD Book of Good Practices. May 2018; Patient Focused Medicines Development www.PatientFocusedMedicine.org.
Toledo-Chávarri A, Perestelo-Pérez L, Álvarez-Pérez Y, Abt-Sacks A, Santoro Domingo P, Villalón D, et al. Participación de los pacientes en la Evaluación de Tecnologías Sanitarias: manual metodológico. Ministerio de Sanidad, Servicios Sociales e Igualdad. Servicio de Evaluación del Servicio Canario de la Salud, 2016.
University of Maryland M-CERSI Framework. Assessing meaningful patient engagement in drug development: a definition, framework, and rubric, March 2015. Access mode: http://www.pharmacy.umaryland.edu/media/SOP/wwwpharmacyumarylandedu/centers/cersievents/pfdd/mcersi-pfdd-framework-rubric.pdf.
Williams M. An analysis of the impact and barriers to patient engagement from the perspective of patient associations and pharma companies; Poster presentation at the 9th European Conference on Rare Diseases & Orphan Products, 10th-12th May 2018, Vienna; Access mode: (http://download2.eurordis.org.s3.amazonaws.com/ecrd/ECRD_2018/Poster%20PDFs/theme%208/P211.pdf).
Williams M. Working together towards a collaborative future: Patient engagement from pharma and patient associations perspectives; Oral presentation at the 27th EURORDIS Round Table of Companies Workshop; 16th October 2018, Barcelona; Access mode: (https://www.eurordis.org/publication/27th-eurordis-round-table-companies-workshop).
Woolley K, et al. Elevate Magazine. MAPS 2018; Access mode: https://maps.instantmagazine.com/publications/elevate-magazine/patient-involvement-in-publications . Accessed April 16, 2019.
Working with patients and patient organisations. A sourcebook for industry. Version 1 – June 2019. The Association of the British Pharmaceutical Industry, 2019.
Yeoman G, Furlong P, Seres M, et al. Defining patient centricity with patients for patients and caregivers: a collaborative endeavour. BMJ Innovation (published online ahead of print March 24, 2017). http://dx.doi.org/10.1136/bmjinnov-2016-000157.
Zangi HA, Ndosi M, Adams J, et al. EULAR recommendations for patient education for people with inflammatory arthritis. Ann Rheum Diseases 2015; 74:954–962.
