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Current Reviews in Clinical and Experimental Pharmacology


ISSN (Print): 2772-4328
ISSN (Online): 2772-4336

Opinion Article

The Patient Motivation Pyramid and Patient-Centricity in Early Clinical Development

Author(s): Thijs van Iersel *, Jocelyn Courville, Cathalijne van Doorne, Remco A. Koster and Christina Fawcett

Volume 17, Issue 1, 2022

Page: [8 - 17] Pages: 10

DOI: 10.2174/1574884716666210427115820

open access plus


Background: It has been recognized that patients should be involved in the design of clinical trials. However, there is a lack of agreement on what patient-centricity means.

Methods: In this article, a Patient Motivation Pyramid based on Maslow’s theory of human motivation is introduced as a tool to identify patient needs. This pyramid is used to make a comprehensive overview of options to implement a patient-centric trial design. The Pyramid with the described options can help to identify patient-centric activities suitable for given drug development. The current article further describes the potential benefits of patient-centric trial designs with an emphasis on early clinical development. Especially in early clinical development, during which trials have many assessments per patient, and the safety and clinical efficacy are uncertain, patient-centric trial design can improve feasibility. Finally, we present three case examples on patient-centric trial design. The first example is seeking patient input on the trial design for a First-in-Human trial which includes patients with Immune Thrombocytopenic Purpura. The second example is the use of a video-link for home dosing. The final example is the use of digital medicine in a decentralized trial in heart failure patients.

Results: A comprehensive overview of patients’ needs can be accomplished by building a Patient Motivation Pyramid as a tool. Patient input can lead to improved endpoints, improved feasibility, better recruitment, less dropout, less protocol amendments, and higher patient satisfaction. The use of digital medicine can lead to a trial design with much less visits to the clinical research center in early clinical development and in a later development phase, even to a complete virtual trial.

Conclusion: We recommend using the Patient Motivation Pyramid as a structural approach for identifying elements of patient-centricity. Secondly, we recommend starting using patient-centric approaches in an early phase of the medicine’s lifecycle.

Keywords: Patient-centric, early clinical development, home-based, decentralized trial, survey, patient motivation pyramid.

Graphical Abstract
Anderson M, Manganiello M. Back to basics: HIV/AIDS advocacy as a model for catalyzing change. Report. Faster Cures and HCM Strategists. New York: Milken Institute 2011.
FDA-led Patient-Focused Drug Development (PFDD) Public Meetings. FDA Available at: [Accessed Sep 13, 2020].
Kuehn CM. A proposed framework for patient-focused policy at the U.S. food and drug administration. Biomedicines 2019; 7(3): 64.
[] [PMID: 31461892]
Stadler M. A Short History of Patient Centricity 2016. 2016. Available at: [Accessed Sep 13, 2020].
EMA. The patient’s voice in the evaluation of medicines 2013. 2013. Available at: [Accessed Sep 13, 2020].
ICH general considerations for clinical studies E8(R1) Draft Version. 2019. Available at: [Accessed Sep 13, 2020].
Fogel DB. Factors associated with clinical trials that fail and opportunities for improving the likelihood of success: A review. Contemp Clin Trials Commun 2018; 11: 156-64.
[] [PMID: 30112460]
Sully BG, Julious SA, Nicholl J. A reinvestigation of recruitment to randomised, controlled, multicenter trials: A review of trials funded by two UK funding agencies. Trials 2013; 14: 166.
[] [PMID: 23758961]
Informa UK Limited. Trialtrove Pharmaintelligence 2018. Available at: [Accessed Sep 13, 2020].
Bazell R. Her-2 The making of herceptin, a revolutionary treatment for breast cancer. New York: Random House Inc. 1998.
Levitan B, Getz K, Eisenstein EL, 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; 52(2): 220-9.
Deloitte insights. Striving to become more patient-centric in life sciences 2016. Available at: [Accessed Jan 12, 2021].
The Milken Institute. Expanding the science of patient input: The power of language 2016. Available at: [Accessed Sep 13, 2020].
FDA CDER Patient-Focused Drug Development. Available at: drugs/cder-patient-focused-drug-development [Accessed Sep 13, 2020].
Maslow AH. A theory of human motivation. Psychol Rev 1943; 50: 370-96.
Witzel M. First among marketers. London: Financial Times 2003.
Theodore L. Marketing myopia. Harv Bus Rev 1960; 38: 45-56.
Bower P, Brueton V, Gamble C, et al. Interventions to improve recruitment and retention in clinical trials: A survey and workshop to assess current practice and future priorities. Trials 2014; 15: 399.
[] [PMID: 25322807]
Levenberg K, Proctor DN, Maman SR, et al. A prospective community engagement initiative to improve clinical research participation in patients with peripheral artery disease. SAGE Open Med 2020; 8: 2050312120930915.
[] [PMID: 32587692]
Elenko E, Underwood L, Zohar D. Defining digital medicine. Nat Biotechnol 2015; 33(5): 456-61.
[] [PMID: 25965750]
Armitage LC, Kassavou A, Sutton S. Do mobile device apps designed to support medication adherence demonstrate efficacy? A systematic review of randomised controlled trials, with meta-analysis. BMJ Open 2020; 10(1): e032045.
[] [PMID: 32005778]
CTTI Feasibility studies database. Available at: [Accessed Jul 01, 2020].
Collins AB, Strike C, Guta A, et al. “We’re giving you something so we get something in return”: Perspectives on research participation and compensation among people living with HIV who use drugs. Int J Drug Policy 2017; 39: 92-8.
[] [PMID: 27780116]
NORD. Clinical trial travel and lodging support for patients participating in catalyst approved MuSK-002 clinical study 2017. Available at: [Accessed Jul 01, 2020].
Gupta R, Ordonez RM, Koenig S. Global impact of antiretroviral therapy-associated diarrhea. AIDS Patient Care STDS 2012; 26(12): 711-3.
[] [PMID: 23078549]
Avedisian J, Azzi N. The patient centered clinical trial. The Pharma Review 2015; 61-5.
Frampton GK, Shepherd J, Pickett K, Griffiths G, Wyatt JC. Digital tools for the recruitment and retention of participants in randomised controlled trials: A systematic map. Trials 2020; 21(1): 478.
[] [PMID: 32498690]
Lowit A, Egan A, Hadjivassiliou M. Feasibility and acceptability of lee silverman voice treatment in progressive ataxias. Cerebellum 2020; 19(5): 701-14.
[] [PMID: 32588316]
Willebrand M, Low A, Dyster-Aas J, et al. Pruritus, personality traits and coping in long-term follow-up of burn-injured patients. Acta Derm Venereol 2004; 84(5): 375-80.
[] [PMID: 15370704]
Progress in Mind, “Priorities and preferences of people with schizophrenia: Need for new focus,” Psychiatry & Neurology Resource Center 2017. 2017. Available at: [Accessed Jan 13, 2021].
ZonMw. Participation by patients Available at:[Accessed Jul 09, 2020].
Kost RG, Lee LM, Yessis J, Wesley RA, Henderson DK, Coller BS. Assessing participant-centered outcomes to improve clinical research. N Engl J Med 2013; 369(23): 2179-81.
[] [PMID: 24304050]
Wendler D, Krohmal B, Emanuel EJ, Grady C. ESPRIT Group. Why patients continue to participate in clinical research. Arch Intern Med 2008; 168(12): 1294-9.
[] [PMID: 18574086]
Quinn Ben. 'I felt guilty': volunteer on signing up for Oxford Covid-19 vaccine trial. The Guardian 2020. Available at: [Accessed Jul 02, 2020].
Tufts Center for the Study of Drug Development. Tufts CSDD Impact Report. Rising Protocol Complexity is Hindering Study Performance, Cost & Efficiency 2018.
Hay M, Thomas DW, Craighead JL, Economides C, Rosenthal J. Clinical development success rates for investigational drugs. Nat Biotechnol 2014; 32(1): 40-51.
[] [PMID: 24406927]
FDA. Guidance for Industry. Estimating the maximum safe starting dose in initial clinical trials for therapeutics in adult healthy volunteers CDER 2005. Available at: [Accessed Sep 13, 2020].
van Iersel MT, Greenberg HE, Westrick ML. Structured risk assessment for first-in-human studies. Ther Innov Regul Sci 2017; 51(3): 288-97.
[] [PMID: 30231711]
Cutler NR. Pharmacokinetic studies of antipsychotics in healthy volunteers versus patients. J Clin Psychiatry 2001; 62(Suppl. 5): 10-3.
[PMID: 11305842]
Tufts Center for the Study of Drug Development. Tufts CSDD Impact Report. Growth in Rare Disease R&D is challenging Development Strategy and Execution 2019.
Chu SH, Jeong SH, Kim EJ, et al. The views of patients and healthy volunteers on participation in clinical trials: An exploratory survey study. Contemp Clin Trials 2012; 33(4): 611-9.
[] [PMID: 22405971]
Iltis AS. Payments to normal healthy volunteers in phase 1 trials: avoiding undue influence while distributing fairly the burdens of research participation. J Med Philos 2009; 34(1): 68-90.
[] [PMID: 19190076]
Manton KJ, Gauld CS, White KM, Griffin PM, Elliott SL. Qualitative study investigating the underlying motivations of healthy participants in phase I clinical trials. BMJ Open 2019; 9(1): e024224.
[] [PMID: 30647042]
Segal JB, Powe NR. Prevalence of immune thrombocytopenia: Analyses of administrative data. J Thromb Haemost 2006; 4(11): 2377-83.
[] [PMID: 16869934]
Dharssi S, Wong-Rieger D, Harold M, Terry S. Review of 11 national policies for rare diseases in the context of key patient needs. Orphanet J Rare Dis 2017; 12(1): 63.
[] [PMID: 28359278]
Cohen YC, Djulbegovic B, Shamai-Lubovitz O, Mozes B. The bleeding risk and natural history of idiopathic thrombocytopenic purpura in patients with persistent low platelet counts. Arch Intern Med 2000; 160(11): 1630-8.
[] [PMID: 10847256]
Bookbinder M Janssen. PRA Launch Fully Virtual Trial. 2019. Available at: 2019/12/06/janssen-pra-launch-fully-virtual-trial[Accessed May 21, 2020].
PRA Digital Health Mobile Health Platform Fact Sheet. 2020. Available at: [Accessed Jul 01, 2020].
FDA guidance : Treatment for Heart Failure: Endpoints for Drug Development Guidance for Industry 2019. Available at: plc-expands-remote-clinical-study-support-its [Accessed Sep 13, 2020].
FDA Guidance on Conduct of Clinical Trials of Medical Products during COVID-19 Public Health Emergency Guidance for Industry, Investigators, and Institutional Review Boards. 2020. Available at: [Accessed Sep 13, 2020].
EMA. Guidance On The Management Of Clinical Trials During The Covid-19 (Coronavirus) Pandemic 2021. Available at: [Accessed Sep 13, 2020].

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