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Reviews on Recent Clinical Trials

Editor-in-Chief

ISSN (Print): 1574-8871
ISSN (Online): 1876-1038

Review Article

Patients Driving the Clinical Trial Designs – Democracy in Clinical Research

Author(s): Payal Bhardwaj*, Jeba Kumar and Raj Kumar Yadav

Volume 14, Issue 4, 2019

Page: [237 - 246] Pages: 10

DOI: 10.2174/1574887114666190808142339

Price: $65

Abstract

Background: Many of the clinical trials remain inefficient owing to the low retention rate, and an impact on the power of the study. In addition, regulatory bodies recommend including the patients’ experience, especially, patient-reported outcomes, while making clinical decisions, and approvals.

Introduction: Patient centricity has reached the stage where patients are both willing and required to participate in clinical trial designs, regulatory review and experts on other panels. Efforts are being made in the right direction and there are multiple aspects that have been or are being addressed.

Objective: The current article focuses on how to include patients in clinical trial designs, the benefits, challenges, and solutions. This means patients who were merely the participants until now, they will be the drivers of trials now, and hence the clinical trials will be more efficient and productive.

Key Findings: There is a drive to enhance patients’ participation in clinical trial designs, especially, visits, efficacy outcomes and their expectations with the treatment. Patients want to remain informed, right from before participation to the completion of the trial. Patients are now an important part of regulatory review, as apparent from recent initiatives by the FDA and EMA. This will enhance patients’ awareness, and bring ownership and transparency. Various patient organizations, advocacy groups have made some great suggestions and taken initiatives in this direction. Clinical Trials Transformation Initiative, European Patient’s Academy on Therapeutic Innovation, and Patient- Centered Outcomes Research Institute are a few key initiatives. However, there is a set of challenges emanating from the complexity of trials, associated with unique mechanism of action of drugs, their efficacy and safety profiles, which has to be dealt with properly.

Conclusion: Overall, the pharma domain is at the verge of putting the patient in the spotlight, to achieve a near-real democracy, where the clinical research is the by the patient, for the patient, and, of the patient.

Keywords: Clinical trial designs, improved retention and successful trials, patient centricity, patient experiences and expectations, retention rate, FDA and EMA.

Graphical Abstract
[1]
Davis K, Schoenbaum SC, Audet AMAA. 2020 vision of patient-centered primary care. J Gen Intern Med 2005; 20(10): 953-7.
[http://dx.doi.org/10.1111/j.1525-1497.2005.0178.x] [PMID: 16191145]
[2]
Carlisle B, Kimmelman J, Ramsay T, MacKinnon N. Unsuccessful trial accrual and human subjects protections: An empirical analysis of recently closed trials. Clin Trials 2015; 12(1): 77-83.
[http://dx.doi.org/10.1177/1740774514558307] [PMID: 25475878]
[3]
Treweek S, Lockhart P, Pitkethly M, et al. Methods to improve recruitment to randomised controlled trials: Cochrane systematic review and meta-analysis. BMJ Open 2013; 3(2)e002360
[http://dx.doi.org/10.1136/bmjopen-2012-002360] [PMID: 23396504]
[4]
Verheggen FW, Nieman FH, Reerink E, Kok GJ. Patient satisfaction with clinical trial participation. Int J Qual Health Care 1998; 10(4): 319-30.
[http://dx.doi.org/10.1093/intqhc/10.4.319] [PMID: 9835248]
[5]
Pharmaceutical Research and Manufacturers of America (PhRMA) and Battelle Technology Partnership Practice. Biopharmaceutical Industry-Sponsored Clinical Trials: Impact on State Economies 2015. Available from: http://phrma-docs.phrma.org/sites/default/files/pdf/biopharmaceutical-industry-sponsored-clinical-trials-impact-onstate-economies.pdf
[6]
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.
[http://dx.doi.org/10.1186/1745-6215-15-399] [PMID: 25322807]
[7]
US Department of Health and Human Services, Office of the Assistant Secretary for Planning and Evaluation, Examination of Clinical Trial Costs and Barriers for Drug Development. 2014. Available from: https://aspe.hhs.gov/report/examination-clinicaltrial-costs-and-barriers-drug-development
[8]
Yeoman G, Furlong P, Seres M, et al. Defining patient centricity with patients for patients and caregivers: A collaborative endeavour. BMJ Innov 2017; 3(2): 76-83.
[http://dx.doi.org/10.1136/bmjinnov-2016-000157] [PMID: 28890797]
[9]
Trial Innovation Network. Engagement, recruitment, and retention Available from https://trialinnovationnetwork.org/elements/engagement-recruitment-retention
[10]
Lloyd K, White J. Democratizing clinical research. Nature 2011; 474(7351): 277-8.
[http://dx.doi.org/10.1038/474277a] [PMID: 21677725]
[11]
Schroen AT, Petroni GR, Wang H, et al. Preliminary evaluation of factors associated with premature trial closure and feasibility of accrual benchmarks in phase III oncology trials. Clin Trials 2010; 7(4): 312-21.
[http://dx.doi.org/10.1177/1740774510374973] [PMID: 20595245]
[12]
Feller S. One in Four Cancer Trials Fails to Enroll Enough Participants 2015. Available from: https://www.upi.com/Health_News/2015/12/30/One-in-four-cancer-trials-fails-to-enroll-enough-partici pants/2611451485504/
[13]
Getz KA. Characterizing the real cost of site regulatory compliance. Appl Clin Trials 2015; 24(6): 1.
[14]
Swan M. Crowdsourced health research studies: An important emerging complement to clinical trials in the public health research ecosystem. J Med Internet Res 2012; 14(2)e46
[http://dx.doi.org/10.2196/jmir.1988] [PMID: 22397809]
[15]
Leiter A, Sablinski T, Diefenbach M, et al. Use of crowdsourcing for cancer clinical trial development. J Natl Cancer Inst 2014; 106(10)dju258
[http://dx.doi.org/10.1093/jnci/dju258] [PMID: 25217580]
[16]
Kitchen H, Cordingley L, Young H, Griffiths CE, Bundy C. Patient-reported outcome measures in psoriasis: The good, the bad and the missing. Br J Dermatol 2015; 172(5): 1210-21.
[http://dx.doi.org/10.1111/bjd.13691] [PMID: 25677764]
[17]
Kluetz PG, O’Connor DJ, Soltys K. Incorporating the patient experience into regulatory decision making in the USA, Europe, and Canada. Lancet Oncol 2018; 19(5): e267-74.
[http://dx.doi.org/10.1016/S1470-2045(18)30097-4] [PMID: 29726391]
[18]
Mullins CD, Vandigo J, Zheng Z, Wicks P. Patient-centeredness in the design of clinical trials. Value Health 2014; 17(4): 471-5.
[http://dx.doi.org/10.1016/j.jval.2014.02.012] [PMID: 24969009]
[19]
Dinan MA, Compton KL, Dhillon JK, et al. Use of patient-reported outcomes in randomized, double-blind, placebo-controlled clinical trials. Med Care 2011; 49(4): 415-9.
[http://dx.doi.org/10.1097/MLR.0b013e3182064aa2] [PMID: 21368680]
[20]
Cherny NI, Sullivan R, Dafni U, et al. A standardised, generic, validated approach to stratify the magnitude of clinical benefit that can be anticipated from anti-cancer therapies: the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS). Ann Oncol 2017; 28(11): 2901-5.
[PMID: 27604385]
[21]
Schnipper LE, Davidson NE, Wollins DS, et al. Updating the American Society of Clinical Oncology Value Framework: revisions and reflections in response to comments received. J Clin Oncol 2016; 34(24): 2925-34.
[http://dx.doi.org/10.1200/JCO.2016.68.2518] [PMID: 27247218]
[22]
Brandberg Y, Johansson H, Bergenmar M. Patients’ knowledge and perceived understanding - Associations with consenting to participate in cancer clinical trials. Contemp Clin Trials Commun 2015; 2: 6-11.
[http://dx.doi.org/10.1016/j.conctc.2015.12.001] [PMID: 29736441]
[23]
Mazouni C, Deneuve J, Arnedos M, et al. Decision-making from multidisciplinary team meetings to the bedside: factors influencing the recruitment of breast cancer patients into clinical trials. Breast 2014; 23(2): 170-4.
[http://dx.doi.org/10.1016/j.breast.2013.12.008] [PMID: 24411195]
[24]
Dias AL, Chao JH, Lee D, Wu Y, Kloecker GH. Patient perceptions concerning clinical trials in oncology patients. Contemp Clin Trials Commun 2016; 4: 179-85.
[http://dx.doi.org/10.1016/j.conctc.2016.09.005] [PMID: 29736480]
[25]
Caruso S. Increasing patient engagement is key to reducing clinical trial dropout rate 2016. Available from: http://www.wcgclinical. com/wp-content/uploads/2016/09/5-Patient-Engagement-Speak Out.pdf
[26]
Lentz J, Kennett M, Perlmutter J, Forrest A. Paving the way to a more effective informed consent process: Recommendations from the Clinical Trials Transformation Initiative. Contemp Clin Trials 2016; 49: 65-9.
[http://dx.doi.org/10.1016/j.cct.2016.06.005] [PMID: 27327780]
[27]
Lopienski K. Retention in Clinical Trials -- Keeping Patients on Protocols 2015. Available from: https://forteresearch.com/news/infographic/infographic-retention-in-clinical-trials-keeping-patients-on-protocols/
[28]
Fan W, Gordon MD. The power of social media analysis. Commun ACM 2014; 57: 74-81.
[http://dx.doi.org/10.1145/2602574]
[29]
Robinson ET, Baron D, Heise LI, et al. Communications handbook for clinical trials: Strategies, tips, and tools to manage controversy, convey your message, and disseminate results Available from: https://www.fhi360.org/resource/communications-handbook-clinical-trials-strategies-tips-and-toolsmanage-controversy-convey
[30]
Wolf SM, Evans BJ. Return of results and data to study participants. Science 2018; 362(6411): 159-60.
[http://dx.doi.org/10.1126/science.aav0005] [PMID: 30309935]
[31]
World Medical Association. World Medical Association Declaration of Helsinki: Ethical principles for medical research involving human subjects. JAMA 2013; 310(20): 2191-4.
[http://dx.doi.org/10.1001/jama.2013.281053] [PMID: 24141714]
[32]
National Institute for Health and Care Excellence (NICE). Hints and tips when preparing to be a patient expert Available from: https://www.nice.org.uk/Media/Default/About/NICE-Communities/Public-involvement/Developing-NICE-guidance/Hints-Tips-Patient-Experts.pdf
[33]
European Patients’ Academy on Therapeutic Innovation (EUPATI). Expert training course for patients and patient representatives on the medicines research & development process Available 142 from: http://www.patientsacademy.eu/index.php/en/edu
[34]
O’Neill N, Mitchell G, Twycross A. The expert patient. Evid Based Nurs 2016; 19(2): 41-2.
[http://dx.doi.org/10.1136/eb-2016-102325] [PMID: 26884510]
[35]
Qaseem A, Forland F, Macbeth F, Ollenschläger G, Phillips S, van der Wees P. Guidelines International Network: toward international standards for clinical practice guidelines. Ann Intern Med 2012; 156(7): 525-31.
[http://dx.doi.org/10.7326/0003-4819-156-7-201204030-00009] [PMID: 22473437]
[36]
Jarrett L. Patient Involvement Unit. A report on a study to evaluate patient/carer membership of the first NICE Guideline Development Groups National Institute for Clinical Excellence 2004. Available from: https://www.nice.org.uk/media/default/About/NICE-Comm unities/Public-involvement/Public-involvement-programme/PIU-GDG-evaluation-report-2004-1.pdf
[37]
Graham R, Mancher M, Wolman DM, et al. Committee on Standards for Developing Trustworthy Clinical Practice Guidelines Clinical Practice Guidelines We Can Trust 2011.http://www. nationalacademies.org/hmd/Reports/2011/Clinical-Practice-Guidelines-We-Can-Trust/Standards.aspx
[38]
Tenaerts P, Madre L, Archdeacon P, Califf RM. The Clinical Trials Transformation Initiative: innovation through collaboration. Nat Rev Drug Discov 2014; 13(11): 797-8.
[http://dx.doi.org/10.1038/nrd4442] [PMID: 25359366]
[41]
Cukor D, Cohen LM, Cope EL, et al. Patient and other stakeholder engagement in Patient-Centered Outcomes Research Institute funded studies of patients with kidney diseases. Clin J Am Soc Nephrol 2016; 11(9): 1703-12.
[http://dx.doi.org/10.2215/CJN.09780915] [PMID: 27197911]
[42]
Forsythe L, Heckert A, Margolis MK, Schrandt S, Frank L. Methods and impact of engagement in research, from theory to practice and back again: Early findings from the Patient-Centered Outcomes Research Institute. Qual Life Res 2018; 27(1): 17-31.
[http://dx.doi.org/10.1007/s11136-017-1581-x] [PMID: 28500572]
[43]
Treiman K, McCormack L, Olmsted M, et al. Engaging patient advocates and other stakeholders to design measures of patient-centered communication in cancer care. Patient 2017; 10(1): 93-103.
[http://dx.doi.org/10.1007/s40271-016-0188-6] [PMID: 27658673]
[44]
[45]
PatientPartner Available from:. www.patientpartner-europe.eu
[46]
PARADIGM Available from:. https://imi-paradigm.eu/
[47]
Patient Focused medicine Available from: https://patientfocusedmedicine.org/about-pfmd/
[48]
Patient-Focused Drug Development: Disease Area Meetings Held in Fiscal Years 2013-2017 2017. Available from: https://www.fda. gov/forindustry/userfees/prescriptiondruguserfee/ucm347317.htm
[49]
INVOLVE Available from: . https://www.invo.org.uk/
[50]
The Voice of the Patient. A Series of Reports from FDA’s Patient-Focused Drug Development Initiative Available from: https://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm368342.htm
[52]
Learn About FDA Patient Engagement Available from:. https://www.fda.gov/forpatients/patientengagement/default.htm
[53]
Plan for Issuance of Patient‐Focused Drug Development Guidance Available from:. https://www.fda.gov/downloads/ForIndustry/User Fees/PrescriptionDrugUserFee/UCM563618.pdf
[54]
EMA Patients and consumers. Available from: https://www.ema. europa.eu/en/partners-networks/patients-consumers
[55]
Ziv S. How to design a better clinical trial with the patient experience in mind 2017 Available from: http://www.newsweek.com/cancer-patients-clinical-trials-design-better-experience
[57]
Health Insurance Portability and Accountability. Standards for privacy of individually identifiable health information DHHS [45 CFR Parts 160, 162 & 164 (HIPAA)] 2016. Available from: http://www.hhs.gov/hipaa/for-professionals/privacy/
[58]
Young PD, Xie D, Schmidt H. Towards patient-centered conflicts of interest policy. Int J Health Policy Manag 2017; 7(2): 112-9.
[http://dx.doi.org/10.15171/ijhpm.2017.128] [PMID: 29524935]

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