Open Access
Subscription Access
Person-Centered Research: A novel approach to Randomized Controlled Trials
Abstract
Introduction: Integrating person-centered values with randomized controlled trials methodology is a novel idea. Person-centeredness is gaining steadily more prominence and attention in healthcare and health-related policy and research. Randomized controlled trials are considered as the gold standard in evidence-based medicine for evaluating the effects of treatment or determining the causal effect. A wide array of study designs is available, but there is a lack of designs with both strong person-centered principles and a strong position with respect to the level of evidence. In this paper we intend to introduce a novel design to fill such a gap.
Aims and objectives: The aim of this paper is to introduce a novel study design where essential values of person-centered care (PCC) are integrated with randomized controlled trial (RCT) methodology into a novel study design termed a person-centered randomized controlled trial (PC-RCT).
Methods: In this paper we discuss the importance and role of evidence in clinical research, levels of evidence, as well as the significance of study design in evidence-based medicine. Moreover, we discuss randomized controlled trials that are considered the gold standard to achieve high quality evidence. In this paper we will explain what the concept of person-centered care is and discuss the values associated with person-centeredness.
The theoretical and methodological considerations that are relevant in applying this concept will be discussed before presenting how we intend to incorporate person-centered values into a randomized controlled trial in a novel study design that is both person-centered and randomized controlled (PC-RCT). Different aspects of this proposed novel study design will be discussed, including the theory and methods underlying this new proposed design, its novelty, different stages and practical steps involved in this proposed design. Challenges, drawbacks and possible solutions for addressing challenges of this novel design will be explored, focusing on the construct, dynamics, advantages, disadvantages and novelty of PC-RCT design.
Conclusion: This paper presents how person-centered values and traditional randomised controlled trial principal values are integrated into one study design where the strengths of both concepts are merged into one. The proposed novel study design has stronger person-centered characteristics and is solid in its RCT features. This design ensures that participants have much more active participation in decision-making and gain more choice in their treatment. The proposed novel study design in this paper has clearly an important role to play in satisfying the need for a study design that can address both the need for rendering higher levels of evidence as well as simultaneously securing greater integration of person-centered values in the same study design.Keywords
References
Colditz, G.A. (2010). Overview of the epidemiology methods and applications: strengths and limitations of observational study designs. Critical Reviews in Food Science and Nutrition 50 (Supplement 1) 10-12.
Straus, S.E. & Sackett, D.L. (1998). Using research findings in clinical practice. British Medical Journal 317 (7154) 339-342.
Kuehn, B.M. (2013). Leaders in evidence-based medicine examine the path ahead. Journal of the American Medical Association 310 (16) 1663-1664.
Manchikanti, L. (2008). Evidence-based medicine, systematic reviews, and guidelines in interventional pain management, Part I: introduction and general considerations. Pain Physician 11 (2) 161-186.
Burns, P.B., Rohrich, R.J. & Chung, K.C. (2011). The levels of evidence and their role in evidence-based medicine. Plastic and Reconstructive Surgery 128 (1) 305-310.
Gopikrishna, V. (2010). A report on case reports. Journal of Conservative Dentistry 13 (4) 265-271.
Carey, J.C. (2010). The importance of case reports in advancing scientific knowledge of rare diseases. Advances in Experimental Medicine and Biology 686, 77-86.
Morris, B.A. (1989). The importance of case reports. Canadian Medical Association Journal 141 (9) 875-876.
Vandenbroucke, J.P. In defense of case reports and case series. Annals of Internal Medicine 2001;134(4):330-4.
Dekkers, O.M., Egger, M., Altman, D.G. & Vandenbroucke, J.P. (2012). Distinguishing case series from cohort studies. Annals of Internal Medicine 156 (1 Pt 1) 37-40.
Hess, D.R. (2004). Retrospective studies and chart reviews. Respiratory Care 49 (10) 1171-1174.
Song, J.W. & Chung, K.C. (2010). Observational studies: cohort and case-control studies. Plastic and Reconstructive Surgery 126 (6) 2234-2242.
Brookmeyer, R., Liang, K.Y. & Linet, M. (1986). Matched case-control designs and overmatched analyses. American Journal of Epidemiology 124 (4) 693-701.
Rothman, K.J., Greenland, S. & Lash, T.L. (2008). Modern Epidemiology. Third Edition edn. New York, USA: Lippincott Williams & Wilkins.
Everitt, B.S. & Howell, D. (2005). Encyclopedia of Statistics in Behavioral Science, p. 2352. New York: Wiley.
Sibbald, B. & Roland, M. (1998). Understanding controlled trials. Why are randomised controlled trials important? British Medical Journal 316 (7126) 201.
Miller, J.N., Colditz, G.A. & Mosteller, F. (1989). How study design affects outcomes in comparisons of therapy. II: Surgical. Statistics in Medicine 8 (4) 455-466.
Wittes, J. (2002). Sample size calculations for randomized controlled trials. Epidemiologic Reviews 24 (1) 39-53.
Spieth, P.M., Kubasch, A.S., Penzlin, A.I., Illigens, B.M., Barlinn, K. & Siepmann, T. (2016). Randomized controlled trials - a matter of design. Neuropsychiatric Disease and Treatment 12, 1341-1349.
Benson, K. & Hartz, A.J. (2000). A comparison of observational studies and randomized, controlled trials. New England Journal of Medicine 342 (25) 1878-1886.
Altman, D.G. (1991). Randomisation. British Medical Journal 302 (6791) 1481-1482.
Pocock, S.J. & Simon, R. (1975). Sequential Treatment Assignment with Balancing for Prognostic Factors in the Controlled Clinical Trial. Biometrics 31 (1) 103-115.
Suresh, K. (2011). An overview of randomization techniques: An unbiased assessment of outcome in clinical research. Journal of Human Reproductive Sciences 4 (1) 8-11.
Vickers, A.J. (2006). How to randomize. Journal of the Society for Integrative Oncology 4 (4) 194-198.
Zill, J.M., Scholl, I., Harter, M. & Dirmaier, J. (2013). Evaluation of dimensions and measurement scales in patient-centeredness. Patient Preference and Adherence 7, 345-351.
Morgan, S. & Yoder, L.H. (2012). A concept analysis of person-centered care. Journal of Holistic Nursing 30 (1) 6-15.
Balint, E., Courtenay, M., Elder, A., Hull, S. & Julian, P. (1993). The doctor, the patient and the group: Balint revisited. London: Routledge.
Mead, N. & Bower P. (2000). Patient-centeredness: a conceptual framework and review of the empirical literature. Social Science & Medicine 51 (7) 1087-1110.
Szasz, T.S. & Hollender, M.H. (1956). A contribution to the philosophy of medicine; the basic models of the doctor-patient relationship. Archives of Internal Medicine 97 (5) 585-592.
McCormack, B. (2003). Researching nursing practice: does person-centeredness matter? Nursing Philosophy 4 (3) 179-188.
McCormack, B. (2003). A conceptual framework for person-centered practice with older people. International Journal of Nursing Practice 9 (3) 202-209.
Tosh, G., Soares-Weiser, K. & Adams, C.E. (2011). Pragmatic vs explanatory trials: the pragmascope tool to help measure differences in protocols of mental health randomized controlled trials. Dialogues in Clinical Neuroscience 13 (2) 209-215.
Bergman, L.R. & Magnusson, D. (1997). A person-oriented approach in research on developmental psychopathology. Development and Psychopathology 9 (2) 291-319.
Brocklehurst, P. (1997). Partially randomised patient preference trials. British Journal of Obstetrics and Gynaecology 104 (12) 1332-1335.
Brewin, C.R. & Bradley, C. (1989). Patient preferences and randomised clinical trials. British Medical Journal 299 (6694) 313-315.
Lillie, E.O., Patay, B., Diamant, J., Issell, B., Topol, E.J. & Schork, N.J. (2011). The n-of-1 clinical trial: the ultimate strategy for individualizing medicine? Personalized Medicine 8 (2) 161-173.
Sterba, S.K. & Bauer, D.J. (2010). Matching method with theory in person-oriented developmental psychopathology research. Development and Psychopathology 22 (2) 239-254.
Bergman, L.R. & Wångby, M. (2014). The person-oriented approach: A short theoretical and practical guide. Estonian Journal of Education 2 (1) 29-49.
Molenaar, P.C.M. (2004). A Manifesto on Psychology as Idiographic Science: Bringing the Person Back Into Scientific Psychology, This Time Forever. Measurement: Interdisciplinary Research and Perspectives 2 (4) 201-218.
von Eye, A. & Bergman, L.R. (2003). Research strategies in developmental psychopathology: dimensional identity and the person-oriented approach. Development and Psychopathology 15 (3) 553-580.
Jayadevappa R. & Sumedha, C. (2011). Patient Centered Care - A Conceptual Model and Review of the State of
the Art. The Open Health Services and Policy Journal 4, 15-25.
Buetow, S. (2011). A framework for doing person-centered health research. International Journal of Person Centered Medicine 1 (2) 358-368.
Vist, G.E., Bryant, D., Somerville, L., Birminghem, T. & Oxman, A.D. (2008). Outcomes of patients who participate in randomized controlled trials compared to similar patients receiving similar interventions who do not participate. Cochrane Database of Systematic Reviews (3):MR000009.
Institute of Medicine Committee on Quality of Health Care in America. (2001). Crossing the Quality Chasm: A New Health System for the 21st Century. Washington DC: National Academies Press. Copyright 2001 by the National Academy of Sciences. All rights reserved.
Hemming, K. & Taljaard, M. (2016). Sample size calculations for stepped wedge and cluster randomised trials: a unified approach. Journal of Clinical Epidemiology 69, 137-146.
Sato, T. (2000). Sample size calculations with compliance information. Statistics in Medicine 19 (19) 2689-2697.
Dell, R.B., Holleran, S. & Ramakrishnan, R. (2002). Sample size determination. ILAR Journal 43 (4) 207-213.
Kim, J. & Seo, B.S. (2013). How to calculate sample size and why. Clinics in Orthopedic Surgery 5 (3) 235-242.
Cuzick, J., Edwards, R. & Segnan, N. (1997). Adjusting for non-compliance and contamination in randomized clinical trials. Statistics in Medicine 16 (9) 1017-1029.
Moons, K.G., Donders, R.A., Stijnen, T. & Harrell, F.E., Jr. (2006). Using the outcome for imputation of missing predictor values was preferred. Journal of Clinical Epidemiology 59 (10) 1092-1101.
Donders, A.R., van der Heijden, G.J., Stijnen, T. & Moons, K.G. (2006). Review: a gentle introduction to imputation of missing values. Journal of Clinical Epidemiology 59 (10) 1087-1091.
Gottfredson, N.C., Bauer, D.J. & Baldwin, S.A. (2014). Modeling Change in the Presence of Non-Randomly Missing Data: Evaluating A Shared Parameter Mixture Model. Structural Equation Modeling: A Multidisciplinary Journal 21 (2) 196-209.
Guyatt, G., Sackett, D., Taylor, D.W., Chong, J., Roberts, R. & Pugsley, S. (1986). Determining optimal therapy - randomized trials in individual patients. New England Journal of Medicine 314 (14) 889-892.
Schulz, K.F., Chalmers, I., Hayes, R.J. & Altman, D.G. (1995). Empirical evidence of bias. Dimensions of methodological quality associated with estimates of treatment effects in controlled trials. Journal of the American Medical Association 273 (5) 408-412.
Rivas-Ruiz, F,. Perez-Vicente, S. & Gonzalez-Ramirez, A.R. (2013). Bias in clinical epidemiological study designs. Allergologia et Immunopathologia 41 (1) 54-59.
Sackett, D.L. & Wennberg, J.E. (1997). Choosing the best research design for each question. British Medical Journal 315 (7123) 1636.
Fries, J.F. & Krishnan, E. (2004). Equipoise, design bias, and randomized controlled trials: the elusive ethics of new drug development. Arthritis Research & Therapy 6 (3) R250-255.
Lilford, R.J., Braunholtz, D.A., Greenhalgh, R. & Edwards, S.J. (2000). Trials and fast changing technologies: the case for tracker studies. British Medical Journal 320 (7226) 43-46.
Page, M.J., Higgins, J.P., Clayton, G., Sterne, J.A., Hrobjartsson, A. & Savovic, J. (2016). Empirical Evidence of Study Design Biases in Randomized Trials: Systematic Review of Meta-Epidemiological Studies. PloS One 11 (7) e0159267.
Ciolino, J.D., Martin, R.H., Zhao, W., Hill, M.D., Jauch, E.C. & Palesch, Y.Y. (2015). Measuring continuous baseline covariate imbalances in clinical trial data. Statistical Methods in Medical Research 24 (2) 255-272.
Miller, G.A. & Chapman, J.P. (2001). Misunderstanding analysis of covariance. Journal of Abnormal Psychology 110 (1) 40-48.
Overall, J.E. & Woodward, J.A. (1977). Common Misconceptions Concerning The Analysis Of Covariance. Multivariate Behavioral Research 12 (2) 171-186.
DOI: http://dx.doi.org/10.5750/ejpch.v6i2.1435
Refbacks
- There are currently no refbacks.