Reflections on the Evolution of Patient Engagement in Drug Development
This article presents the author’s opinion on the past and present state of the Patient Engagement movement and discusses ways in which the movement will need to change and evolve if it is to become viable and standard practice in drug development. For most of the past decade, drug development sponsors—both government-funded and industry-funded research—have been aspirational in their support of Patient Engagement initiatives. New frameworks and guidelines have been proposed and developed, and a wide variety of initiatives have been planned and piloted. Many factors have facilitated a tentative and experimental posture—these include internal resistance to modifying legacy practices and processes; insufficient funding and staff to implement and manage initiatives; lack of clarity from regulatory and health authorities; and uncertainty around expected impact and return on investment. Recently, research sponsors have begun restructuring their Patient Engagement capabilities and they have been seeking more concrete return on engagement measures. These developments signal that the Patient Engagement movement has entered a new phase, becoming more rigorous as it seeks to balance ethical and moral obligations with scientific and business imperatives.
The author wishes to thank Karyn Korieth for her invaluable assistance.
Compliance with Ethical Standards
The author received no funding associated with the development or publication of this manuscript.
Conflicts of interest
The author has no conflicts of interest to report.
- 2.Getz KA, Kaitin K. Why does the industry need a change? In: Schueler P, Buckley BM, editors. Re-engineering clinical trials: best practices for streamlining drug development. New York: Elsevier; 2015.Google Scholar
- 3.Hoos A, Anderson J, Boutin M, Dewulf L, Geissler J, Johnston G, Joos A, Metcalf M, Regnante J, Sargeant I, Schneider RF, Todaro V, Tougas G. Partnering with patients in the development and lifecycle of medicines: a call for action. Ther Innov Regul Sci. 2015;49(6):929–39.Google Scholar
- 5.Getz KA. Charting a course for the patient centricity movement. Clin Res. 2015;29(2):36–40.Google Scholar
- 6.Getz KA. Establishing return-on-investment expectations for patient-centric initiatives. Ther Innov Regul Sci. 2015;49(5):745–9.Google Scholar
- 8.Levitan B, Getz K, Eisenstein EL, Goldberg M, Harker M, Hesterlee S, Patrick-Lake B, Roberts J, DiMasi J. Assessing the financial value of patient engagement: a quantitative approach from CTTI’s patient groups and clinical trials project. Ther Innov Regul Sci. 2018;52(2):220–9.Google Scholar
- 11.Anderson M, Manganiello M. Back to basics: HIV/AIDS advocacy as a model for catalyzing change. Faster Cures. 2011. https://www.fastercures.org/reports/view/13. Accessed 6 May 2019.
- 14.Patient-Centered Outcomes Research Institute (PCORI). Engagement rubric for applicants. 2014. https://www.pcori.org/sites/default/files/Engagement-Rubric.pdf. Accessed 6 May 2019.
- 15.US Food and Drug Administration. The voice of the patient: a series of reports from FDA’s Patient-Focused Drug Development initiative. http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm368342.htm. Accessed 6 May 2019.
- 16.Chalasani M, Vaidya P, Mullin T. Enhancing the incorporation of the patient’s voice in drug development and evaluation. BioMed Central. 2018;4(10):1–6.Google Scholar
- 17.European Medicines Agency. European Medicines Agency’s interaction with patients, consumers, healthcare professionals and their organizations. 2016. https://www.ema.europa.eu/documents/report/european-medicines-agencys-interaction-patients-consumers-healthcare-professionals-their_en.pdf. Accessed 6 May 2019.
- 18.Clinical Trials Transformation Initiative. CTTI recommendations: effective engagement with patient groups around clinical trials. 2015. https://www.ctti-clinicaltrials.org/files/pgctrecs.pdf. Accessed 6 May 2019.
- 19.Medical Device Innovation Consortium (MDIC). Medical Device Innovation Consortium (MDIC) patient-centered benefit-risk project report: a framework for incorporating information on patient preferences regarding the benefit and risk into regulatory assessments of new medical technology. 2015. https://mdic.org/wp-content/uploads/2018/05/MDIC_PCBR_Framework_Web.pdf. Accessed 6 May 2019.
- 20.European Patients’ Academy on Therapeutic Innovation. http://www.eupati.eu. Accessed 6 May 2019.
- 21.National Institute for Health Research (NIHR). https://www.nihr.ac.uk. Accessed 6 May 2019.
- 22.Getz KA. Measuring adoption and value of patient engagement. Appl Clin Trials. 2017;26(2). http://www.appliedclinicaltrialsonline.com/measuring-adoption-and-value-patient-engagement. Accessed 6 May 2019.
- 23.Drug Information Association. Capturing the value of patient engagement: summary of results of the 2016 study of patient-centric initiatives in drug development. 2016. https://www.diaglobal.org. Accessed 6 May 2019.
- 24.TransCelerate BioPharma Inc. Our work in Clinical Development. https://www.transceleratebiopharmainc.com/what-we-do/work-clinical-development/. Accessed 29 Apr 2019.
- 25.Bloom D, Beetsch J, Harker M, Hesterlee S, Moreira P, Patrick-Lake B, Selig W, Sherman J, Smith S, Valentine J, Roberts J. The rules of engagement: CTTI recommendations for successful collaborations between sponsors and patient groups around clinical trials. Ther Innov Regul Sci. 2018;52(2):206–13.Google Scholar
- 26.National Academies of Sciences, Engineering, and Medicine. Advancing the science of patient input in medical product R&D: towards a research agenda: proceedings of a workshop in brief. 2018. http://nap.edu/25325. Accessed 6 May 2019.
- 30.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. http://ec.europa.eu/health/files/eudralex/vol-1/reg_2014_536/reg_2014_536_en.pdf. Accessed 6 May 2019.
- 31.University of Maryland Center for Excellence in Regulatory Science and Innovation (CERSI). Assessing meaningful patient engagement in drug development: a definition, framework, and rubric. 2015. https://www.pharmacy.umaryland.edu/media/SOP/wwwpharmacyumarylandedu/centers/cersievents/pfdd/mcersi-pfdd-framework-rubric.pdf. Accessed 6 May 2019.
- 32.Deane K, Delbecque L, Gorbenko O, et al. Co-creation of patient engagement quality guidance for medicines development: an international multistakeholder initiative. BMJ Innov. 2019;0:1–13.Google Scholar