Skip to main content
SpringerLink
Log in

The Future of Behavioral Randomized Clinical Trials

  • Chapter
  • First Online:
Behavioral Clinical Trials for Chronic Diseases

Abstract

The chapter aims to predict the future of behavioral randomized trials in four key areas. The emergence of a meta-science for behavioral clinical trials will replace the “one-size-fits all” approach to behavioral trial design with designs that feature a progression of questions, a progression of methods, and the use of guidelines to facilitate decision-making. Behavioral treatments will produce stronger effects with the emergence of precision lifestyle medicine that targets precisely those most likely to benefit and tailors precisely to diverse persons, settings, and time, and with multilevel trials that simultaneously intervene on more than one level of ecologic risk. Behavioral trials will be easier to implement using networks that collaborate on multi-site behavioral trials, leveraging data routinely collected in the healthcare system, and enhancing accessibility to trials using digital technology. The gap between trial results and implementation in practice will be narrowed by strategic planning for dissemination featuring outcomes in trials that are meaningful to clinicians and third-party payers, commitment to refine treatments that have failed, and a push toward influencing clinical practice guidelines with Phase III efficacy trials.

“It is difficult to make predictions, especially about the future.”

Yogi Berra

This is a preview of subscription content, log in via an institution to check access.

Access this chapter

Log in via an institution
Chapter
USD 29.95
Price excludes VAT (USA)
  • Available as PDF
  • Read on any device
  • Instant download
  • Own it forever
eBook
USD 89.00
Price excludes VAT (USA)
  • Available as EPUB and PDF
  • Read on any device
  • Instant download
  • Own it forever
Hardcover Book
USD 119.99
Price excludes VAT (USA)
  • Durable hardcover edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info

Tax calculation will be finalised at checkout

Purchases are for personal use only

Institutional subscriptions

References

  1. Lillenfeld SO, McKay D, Hollan SD (2018) Why randomised controlled trials of psychological treatments are still essential. Lancet 5:536–538

    Google Scholar 

  2. Ioannidis JP, Fanelli D, Dunne DD, Goodman SN (2015) Meta-research: evaluation and improvement of research methods and practices. PLoS Biol 13:e1002264. https://doi.org/10.1371/journal.pbio.1002264

  3. ASCEND Study Collaborative Group, Bowman L, Mafham M, Wallendszus K, Stevens W, Buck G, Barton J, Murphy K, Aung T, Haynes R, Cox J, Murawska A, Young A, Lay M, Chen F, Sammons E, Waters E, Adler A, Bodansky J, Farmer A, McPherson R, Neil A, Simpson D, Peto R, Baigent C, Collins R, Parish S, Armitage J (2018) Effects of n-3 fatty acid supplements in diabetes mellitus. N Engl J Med 379:1540–1550

    Article  Google Scholar 

  4. Victor RG, Lynch K, Li N, Blyler C, Muhammad E, Handler J, Brettler J, Rashid M, Hsu B, Foxx-Drew D, Moy N, Reid AE, Elashoff RM (2018) A cluster-randomized trial of blood-pressure reduction in black barbershops. N Engl J Med 378:1291–1301

    Article  Google Scholar 

  5. Shadish WR, Cook TD, Campbell DT (2002) Experimental and quasi-experimental designs for generalized causal inference. Houghton Mifflin, Boston

    Google Scholar 

  6. Hudson KL, Lauer MS, Collins FS (2016) Toward a new era of trust and transparency in clinical trials. JAMA 316:1353–1354

    Article  Google Scholar 

  7. Choi KW, Chen CY, Stein MB, Klimentidis YC, Wang MJ, Koenen KC, Smoller JW, Major Depressive Disorder Working Group of the Psychiatric Genomics Consortium (2019) Assessment of bidirectional relationships between physical activity and depression among adults: a 2-sample mendelian randomization study. JAMA Psychiat 76:399–408

    Google Scholar 

  8. Byrne EM, Yang J, Wray NR (2017) Inference in psychiatry via 2-sample mendelian randomization–From association to causal pathway? JAMA Psychiat 74:1191–1192

    Google Scholar 

  9. Institute of Medicine (2007) The learning healthcare system: workshop summary. National Academies Press, Washington DC. https://doi.org/10.17226/11903

  10. Lauer MS, Bonds D (2014) Eliminating the “expensive” adjective for clinical trials. Am Heart J 167:419–420

    Google Scholar 

  11. Naci H, Ioannidis JPA (2015) Evaluation of wellness determinants and interventions by citizen scientists. JAMA 314:121–122

    Google Scholar 

  12. Ioannidis JPA (2005) Why most published research findings are false. PLoS Med 2:e124. https://doi.org/10.1371/journal.pmed.0020124

  13. Munafò MR, Nosek BA, Bishop DVM, Button KS, Chambers CD, du Sert NP, Simonsohn U, Wagenmakers EJ, Ware JJ, Ioannidis JPA (2017) A manifesto for reproducible science. Nat Hum Behav 1:0021. https://doi.org/10.038/s41562-016-0021

    Google Scholar 

  14. Button KS, Ioannidis JPA, Mokrysz C, Nosek BA, Flint J, Robinson ES, Munafò MR (2013) Power failure: why small sample size undermines the reliability of neuroscience. Nat Rev Neurosci 5:365–376

    Google Scholar 

  15. Fanelli D (2010) “Positive” results increase down the hierarchy of the sciences. PLoS One 5:e10068. https://doi.org/10.1371/journal.pone.0010068

  16. John LK, Loewenstein G, Prelec D (2012) Measuring the prevalence of questionable research practices with incentives for truth telling. Psychol Sci 23:524–532

    Article  Google Scholar 

  17. Makel MC, Plucker JA, Hegarty B (2012) Replications in psychology research: how often do they really occur? Perspect Psychol Sci 7:537–542

    Article  Google Scholar 

  18. Wicherts JM, Borsboom D, Kats J, Molenaar D (2006) The poor availability of psychological research data for reanalysis. Am Psychol 61:726–728

    Article  Google Scholar 

  19. Kerr NL (1998) HARKing: hypothesizing after the results are known. Pers Soc Psychol Rev 2:196–217

    Article  Google Scholar 

  20. Watts DJ (2017) Should social science be more solution-oriented? Nat Hum Behav 1:0015. https://doi.org/10.1038/s41562-016-0015

  21. Freedland KE (2017) A new era for Health Psychology. Health Psychol 36:1–4

    Article  Google Scholar 

  22. Czajkowski SM, Powell LH, Adler N, Naar-King S, Reynolds KD, Hunter CM, Laraia B, Olster DH, Perna FM, Peterson JC, Epel E, Charlson M (2015) From ideas to efficacy: the ORBIT model for developing behavioral treatments for chronic diseases. Health Psychol 34:971–982

    Article  Google Scholar 

  23. Michie S, Richardson M, Johnston M, Abraham C, Francis J, Hardeman W, Eccles MP, Cane J, Wood CE (2013) The behavior change technique taxonomy (v1) of 93 hierarchically clustered techniques: building an international consensus for the reporting of behavior change interventions. Ann Behav Med 46:81–95

    Article  Google Scholar 

  24. Thabane L, Thomas T, Ye C, Paul J (2009) Posing the research question: not so simple. Can J Anaesth 56:71–79

    Article  Google Scholar 

  25. Schulz KF, Altman DG, Moher D, CONSORT Group (2010) CONSORT 2010 statement: updated guidelines for reporting parallel group randomised trials. Ann Intern Med 152:726–732

    Google Scholar 

  26. Boutron I, Moher D, Altman DG, Schulz KF, Ravaud P, CONSORT Group (2008) Extending the CONSORT statement to randomized trials of nonpharmacologic treatment: explanation and elaboration. Ann Intern Med 148:295–309

    Article  Google Scholar 

  27. Montgomery P, Grant S, Hopewell S, Macdonald G, Moher D, Michie S, Mayo-Wilson E (2013) Protocol for CONSORT-SPI: an extension for social and psychological interventions. Implement Sci 8:99. https://doi.org/10.1186/1748-5908-88-99

  28. Chan AW, Tetzlaff JM, Gøtzsche PC, Altman DG, Mann H, Berlin JA, Dickersin K, Hróbjartsson A, Schulz KF, Parulekar WR, Krleza-Jeric K, Laupacis A, Moher D (2013) SPIRIT 2013 explanation and elaboration: guidance for protocols of clinical trials. BMJ 346:e7586. https://doi.org/10.1136/bmj.e7586

  29. Bacon SL, Lavoie KL, Ninot G, Czajkowski S, Freedland KE, Michie S, Montgomery P, Powell LH, Spring B (2015) An international perspective on improving the quality and potential of behavioral clinical trials. Curr Cardiovasc Risk Rep 9:427. https://doi.org/10.1007/s12170-014-0427-0

  30. Lavoie KL, Powell LH, Ninot G, Bacon SL, on behalf of the Faculty of the 2nd IBTN Meeting (2019) Proceedings from IBTN 2018: It’s time for a culture change in behavioral medicine. Ann Behav Med 53:296–298

    Google Scholar 

  31. Ma J, Rosas LG, Lv N (2016) Precision lifestyle medicine: a new frontier in the science of behavior change and population health. Am J Prev Med 50:395–397

    Article  Google Scholar 

  32. US National Library of Medicine (2018) What is precision medicine? https://ghr.nlm.nih.gov/primer/precisionmedicine/definition

  33. Ashley EA (2015) The precision medicine initiative: a new national effort. JAMA 313:2119–2120

    Article  Google Scholar 

  34. Khoury MJ, Evans JP (2015) A public health perspective on a national precision medicine cohort: balancing long-term knowledge generation with early health benefit. JAMA 313:2117–2118

    Article  Google Scholar 

  35. Khera AV, Emdin CA, Drake I, Natarajan P, Bick AG, Cook NR, Chasman DI, Baber U, Mehran R, Rader DJ, Fuster V, Boerwinkle E, Melander O, Orho-Melander M, Ridker PM, Kathiresan S (2016) Genetic risk, adherence to a healthy lifestyle, and coronary disease. N Engl J Med 375:2349–2358

    Article  Google Scholar 

  36. Gardner CD, Trepanowski JF, Del Gobbo LC, Hauser ME, Rigdon J, Ioannidis JPA, Desai M, King AC (2018) Effect of low-fat vs low-carbohydrate diet on 12-month weight loss in overweight adults and the association with genotype pattern or insulin secretion: the DIETFITS randomized clinical trial. JAMA 319:667–679

    Google Scholar 

  37. Goldberg JH, Kiernan M (2005) Innovative techniques to address retention in a behavioral weight-loss trial. Health Educ Res 20:439–447

    Article  Google Scholar 

  38. Kiernan M, Oppezzo MA, Resnicow K, Alexander GL (2018) Effects of a methodological infographic on research participants’ knowledge, transparency, and trust. Health Psychol 37:782–786

    Article  Google Scholar 

  39. Kong W, Langlois MF, Kamga-Ngandé C, Gagnon C, Brown C, Baillargeon JP (2010) Predictors of success to weight-loss intervention program in individuals at high risk for type 2 diabetes. Diab Res Clin Pract 90:147–153

    Google Scholar 

  40. Rosas LG, Lv N, Azar KMJ, Xiao L, Hooker SP, Lewis MA, Zavella P, Venditti EM, Ma J (2018) HOMBRE: a randomized controlled trial to compare two approaches to weight loss for overweight and obese Latino men (Hombres con Opciones para Mejorar el Bienestar y bajar el Riesgo de Enfermedades crónicas; men with choices to improve well-being and decrease chronic disease risk). Contemp Clin Trials 68:23–34

    Google Scholar 

  41. Preference Collaborative Review Group (2008) Patients’ preferences with randomised trials: systematic review and patient level meta-analysis. BMJ 337:a1864. https://doi.org/10.1136/bmj.a1864

  42. King M, Nazareth I, Lampe F, Bower P, Chandler M, Morou M, Sibbald B, Lai R (2005) Impact of participant and physician intervention preferences on randomized trials: a systematic review. JAMA 293:1089–1099

    Article  Google Scholar 

  43. Almirall D, Nahum-Shani I, Sherwood NE, Murphy SA (2014) Introduction to SMART designs for the development of adaptive interventions: with application to weight loss research. Transl Behav Med 4:260–274

    Article  Google Scholar 

  44. Williams LM, Pines A, Goldstein-Piekarski AN, Rosas LG, Kullar M, Sacchet MD, Gevaert O, Bailenson J, Lavori PW, Dagum P, Wandell B, Correa C, Greenleaf W, Suppes T, Perry LM, Smyth JM, Lewis MA, Venditti EM, Snowden M, Simmons JM, Ma J (2018) The ENGAGE study: integrating neuroimaging, virtual reality and smartphone sensing to understand self-regulation for managing depression and obesity in a precision medicine model. Behav Res Ther 101:58–70

    Article  Google Scholar 

  45. Hellhammer D, Meinlschmidt G, Pruessner JC (2018) Conceptual endophenotypes: a strategy to advance the impact of psychoneuroendocrinology in precision medicine. Psychoneuroendocrinology 89:147–160

    Article  Google Scholar 

  46. Lamont A, Lyons MD, Jaki T, Stuart E, Feaster DJ, Tharmaratnam K, Oberski D, Ishwaran H, Wilson DK, Van Horn ML (2018) Identification of predicted individual treatment effects in randomized clinical trials. Stat Methods Med Res 27:142–157

    Article  Google Scholar 

  47. Barabási A-L (2007) Network medicine--from obesity to the “diseasome”. N Engl J Med 357:404–407

    Google Scholar 

  48. Institute of Medicine Committee on Understanding Eliminating Racial and Ethnic Disparities in Health Care, Smedley BD, Stith SY, Nelson AR (eds) (2003) Unequal treatment: confronting racial and ethnic disparities in health care. National Academies Press, Washington DC

    Google Scholar 

  49. Gorin SS, Badr H, Krebs P, Prabhu Das I (2012) Multilevel interventions and racial/ethnic health disparities. J Natl Cancer Inst Monogr 44:100–111

    Google Scholar 

  50. Mangla A, Doukky R, Richardson D, Avery EF, Dawar R, Calvin JE Jr, Powell LH (2018) Design of a bilevel clinical trial targeting adherence in heart failure patients and their providers: the Congestive Heart Failure Adherence Redesign Trial (CHART). Am Heart J 195:139–150

    Article  Google Scholar 

  51. Stevens J, Pratt C, Boyington J, Nelson C, Truesdale KP, Ward DS, Lytle L, Sherwood NE, Robinson TN, Moore S, Barkin S, Cheung YK, Murray DM (2017) Multilevel interventions targeting obesity: research recommendations for vulnerable populations. Am J Prev Med 52:115–124

    Article  Google Scholar 

  52. Eapen ZJ, Lauer MS, Temple RJ (2014) The imperative of overcoming barriers to the conduct of large, simple trials. JAMA 311:1397–1398

    Article  Google Scholar 

  53. Institute of Medicine Forum on Drug Discovery, Development, and Translation (2010) Transforming clinical research in the United States. Challenges and opportunities: workshop summary. National Academies Press, Washington DC

    Google Scholar 

  54. Antman EM, Harrington RA (2012) Transforming clinical trials in cardiovascular disease: mission critical for health and economic well-being. JAMA 308:1743–1744

    Article  Google Scholar 

  55. National Research Council Committee on the Science of Team Science, Cooke NJ, Hilton ML (eds) (2015) Enhancing the effectiveness of team science, National Academies Press, Washington DC.

    Google Scholar 

  56. Fortunato S, Bergstrom CT, Börner K, Evans J, Helbing D, Milojević S, Petersen AM, Radicchi F, Sinatra R, Uzzi B, Vespignani A, Waltman L, Wang D, Barabási A-L (2018) Science of science. Science 359:eaao0185. https://doi.org/10.1126/science.aao0185

  57. Freedland KE (2019) The Behavioral Medicine Research Council: its origins, mission, and methods. Health Psychol 38:277–289

    Article  Google Scholar 

  58. Opmeer BC (2016) Electronic health records as sources of research data. JAMA 315:201–202

    Article  Google Scholar 

  59. Mandl KD, Kohane IS, McFadden D, Weber GM, Natter M, Mandel J, Schneeweiss S, Weiler S, Klann JG, Bickel J, Adams WG, Ge Y, Zhou X, Perkins J, Marsolo K, Bernstam E, Showalter J, Quarshie A, Ofili E, Hripcsak G, Murphy SN (2014) Scalable Collaborative Infrastructure for a Learning Healthcare System (SCILHS): architecture. J Am Med Inform Assoc 21:615–620

    Article  Google Scholar 

  60. Elbert NJ, van Os-Medendorp H, van Renselaar W, Ekeland AG, Hakkaart-van Roijen L, Raat H, Nijsten TE, Pasmans SG (2014) Effectiveness and cost-effectiveness of ehealth interventions in somatic diseases: a systematic review of systematic reviews and meta-analyses. J Med Internet Res 16:e110. https://doi.org/10.2196/jmir.2790

  61. Steventon A, Bardsley M, Billings J, Dixon J, Doll H, Hirani S, Cartwright M, Rixon L, Knapp M, Henderson C, Rogers A, Fitzpatrick R, Hendy J, Newman S, Whole System Demonstrator Evaluation Team (2012) Effect of telehealth on use of secondary care and mortality: findings from the Whole System Demonstrator cluster randomised trial. BMJ 344:e3874. https://doi.org/10.1136/bmj.e3874

  62. Eysenbach G, CONSORT-EHEALTH Group (2011) CONSORT-EHEALTH: improving and standardizing evaluation reports of web-based and mobile health interventions. J Med Internet Res 13:e126. https://doi.org/10.2196/jmir.1923

  63. Murdoch TB, Detsky AS (2013) The inevitable application of big data to health care. JAMA 309:1351–1352

    Article  Google Scholar 

  64. Lauer MS (2012) Time for a creative transformation of epidemiology in the United States. JAMA 308:1804–1805

    Article  Google Scholar 

  65. Nyberg K, Hedman P (2019) Swedish guidelines for registry-based randomized clinical trials. Ups J Med Sci 124:33–36

    Article  Google Scholar 

  66. Shakhnovich V (2018) It’s time to reverse our thinking: the reverse translation research paradigm. Clin Transl Sci 11:98–99

    Article  Google Scholar 

  67. Ledford H (2008) Translational research: the full cycle. Nature 453:843–845

    Article  Google Scholar 

  68. Popper KR (1959) The logic of scientific discovery. Basic Books, New York

    Google Scholar 

  69. Murphy J (2019) Reverse translational medicine: the promising future of failure. BioProcess Intl. https://bioprocessintl.com/business/pre-clinical-and-clinical-trials/elucidation-on-reverse-translational-medicine-the-promising-future-of-failure

  70. Collins FS (2011) Reengineering translational science: the time is right. Sci Transl Med 3:90cm17. https://doi.org/10.1126/scitranslmed.3002747

Download references

Author information

Authors and Affiliations

  1. Department of Preventive Medicine, Rush University Medical Center, Chicago, IL, USA

    Lynda H. Powell

  2. College of Nursing, Villanova University, Villanova, PA, USA

    Peter G. Kaufmann

  3. Department of Psychiatry, Washington University in St. Louis, St. Louis, MO, USA

    Kenneth E. Freedland

Authors
  1. Lynda H. Powell
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Peter G. Kaufmann
    View author publications

    You can also search for this author in PubMed Google Scholar

  3. Kenneth E. Freedland
    View author publications

    You can also search for this author in PubMed Google Scholar

Rights and permissions

Reprints and permissions

Copyright information

© 2021 Springer Nature Switzerland AG

About this chapter

Check for updates. Verify currency and authenticity via CrossMark

Cite this chapter

Powell, L.H., Kaufmann, P.G., Freedland, K.E. (2021). The Future of Behavioral Randomized Clinical Trials. In: Behavioral Clinical Trials for Chronic Diseases. Springer, Cham. https://doi.org/10.1007/978-3-030-39330-4_11

Download citation

Publish with us

Policies and ethics

Search

Navigation