Abstract
Interest in patient-centric initiatives to engage patients as partners in clinical research and inform drug development strategy, planning and execution has increased exponentially during the past decade. Adoption, use, organizational approach and infrastructure supporting patient-centric initiatives, however, varies widely from company to company. The Drug Information Association (DIA) in collaboration with the Tufts Center for the Study of Drug Development (Tufts CSDD) at the Tufts University School of Medicine developed and validated an assessment tool that companies can use to evaluate their organization’s patient engagement preparedness and capabilities within the context of industry-wide practices. This paper discusses the development of the tool, the assessment experience, and implications for further refinement of the assessment process. Specifically, the team conducted an extensive literature review, compiled and analyzed case studies and gathered input from a working group of 18 biopharmaceutical companies. To validate the assessment tool and demonstrate its feasibility, the DIA-CSDD Tufts team conducted a pilot implementation involving onsite and virtual in-depth interviews among 14 biopharmaceutical companies. A subsequent paper will report on the findings from the 14 companies assessed.
This is a preview of subscription content, log in via an institution to check access.
Similar content being viewed by others
References
Anderson M, McCleary K. From passengers to co-pilots: Patient roles expand. Sci Transl Med. 2015;7:291fs25. https://doi.org/10.1126/scitranslmed.aac6023.
US Food and Drug Administration. FDA-led Patient-Focused Drug Development (PFDD) Public Meetings Information Page. https://www.fda.gov/industry/prescription-drug-user-fee-amendments/fda-led-patient-focused-drug-development-pfdd-public-meetings. Accessed 20 September, 2020.
Crossnohere N, Fischer R, Crossley E, Vroom E, Bridges J. The evolution of patient-focused drug development and Duchenne muscular dystrophy. Expert Rev Pharm Outcomes Res. 2020;1:57–68.
Yeoman FP, Seres M, Binder H, Chung H, Garzya V, et al. Defining patient centricity with patients for patients and caregivers: a collaborative endeavor. BMJ Innovat. 2017;3:76–83.
Stergiopoulos S, Michaels DL, Lopez Kunz B, Getz KA. Measuring the Impact of Patient Engagement and Patient Centricity in Clinical Research and Development. Therapeutic Innovat Regul Sci. 2020;54:103–16.
Vat LE, Finlay T, Schuitmaker-Warnaar TJ, Fahy N, Robinson P, Boudes M, et al. Evaluating the “return on patient engagement initiatives” in medicines research and development: a literature review. Health Expect. 2020;23:5–18.
Boutin M, Dewulf L, Hoos A, Geissler J, Todaro V, Schneider R, et al. Culture and process change as a priority for patient engagement in medicines development. Therapeutic Innovat Regul Sci. 2017;51:29–38.
Michaels DL, Lamberti MJ, Pena Y, Lopez Kunz B, Getz K. Assessing biopharmaceutical company experience with patient-centric initiatives. Clin Ther. 2019;41:1427–38.
Lamberti MJ, Awatin J. Mapping the landscape of patient-centric activities within clinical research. Clin Ther. 2017;39:2196–202.
Patient Focused Medicines Development. Synapse—the Patient Engagement Network Page. https://synapse.pfmd.org/.
Boudes M, Robinson P, Bertelsen N, Brooke N, Hoos A, Boutin M, et al. What do stakeholders expect from patient engagement: are these expectations being met? Health Expect. 2018;21:1035–45.
Bance L, Sethuraman V, Feldman K, and Mitchell A. Four pillars to achieve patient centricity. Appl Clin Trials. https://www.appliedclinicaltrialsonline.com/view/four-pillars-achieve-patient-centricity. Accessed October 19, 2017
Getz K. Reflections on the evolution of patient engagement in drug development. Pharm Med. 2019;33:179–85.
Kirwan JR, de Wit M, Frank L, Haywood KL, Salek S, Brace-McDonnell S, et al. Emerging guidelines for patient engagement in research. Value Health. 2017;20:481–6.
Du Plessis D, Sake JK, Halling K, Morgan J, Georgieva A, Bertelsen N. Patient centricity and pharmaceutical companies: is it feasible? Therapeutic Innovat Regul Sci. 2017;51:460–7.
Parsons S, Starling B, Mullan-Jensen C, Tham S, Warner K, Wever K. What do pharmaceutical industry professionals in Europe believe about involving patients and the public in research and development of medicines?. A qualitative interview study. BMJ Open. 2016;6: https://doi.org/10.1136/bmjopen-2015-008928.
Forsythe L, Carman K, Szydlowski V, Fayish L, Davidson L, Hickam D, et al. Patient engagement in research: early findings from the patient-centered outcomes research institute. Health Aff. 2019;38:359–67.
Tan C, McGill JM, Mallbris L. Uniting discovery and care: the role of pharmaceutical companies in research, clinical studies, and patient care. J Invest Dermatol. 2020;140:527–30.
Acknowledgements
The authors would like to thank Stella Stergiopoulos, Kinari Shah, Zachary Smith, and Emily Botto for their contributions to this manuscript.
Funding
This study was funded by unrestricted organizational grants from AbbVie Inc., Amgen Inc., Amicus Therapeutics, AstraZeneca, Bayer AG, Biogen MA Inc., Bluebird Bio, Inc., Dermira, Inc., Horizon Pharma USA, Inc., Ionis Pharmaceuticals, Inc., Johnson & Johnson, MK&A, Otsuka Pharmaceutical Development & Commercialization, Inc., Pfizer Inc., Genentech, A Member of the Roche Group, Sanofi US Services Inc., Takeda Pharmaceuticals Company Limited, and UCB Biosciences, Inc.
Author information
Authors and Affiliations
Corresponding author
Ethics declarations
Conflict if interest
No potential conflicts were declared.
Ethical Approval
This article does not contain any studies with human or animal subjects performed by any of the authors.
Rights and permissions
About this article
Cite this article
Michaels, D.L., Peña, Y., Kunz, B.L. et al. Evaluating the Feasibility and Validity of a New Tool to Assess Organizational Preparedness and Capabilities to Support Patient Engagement in Drug Development. Ther Innov Regul Sci 55, 1193–1198 (2021). https://doi.org/10.1007/s43441-021-00289-6
Received:
Accepted:
Published:
Issue Date:
DOI: https://doi.org/10.1007/s43441-021-00289-6