Skip to main content

Pragmatic Trials: Solving the Dilemma of Psychiatric Nonadherence

  • Chapter
  • First Online:
Book cover Psychiatric Nonadherence

Abstract

This chapter aims to describe pragmatic clinical trials and how they differ from traditional explanatory trials in terms of validity, design, and setting. We discuss the defining principles of pragmatic clinical trials and how they are vital in moving psychiatric research forward to the development of better-informed treatment choices for patients and providers. We describe the Patient-Centered Outcomes Research Institute (PCORI), their tenets, and how they are a large funding source of pragmatic trials and clinical effectiveness research. We highlight two pragmatic clinical trials in the field of child psychiatry as a way of showcasing how pragmatic clinical trials can help in shared decision-making and solve the dilemma of psychiatric nonadherence.

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

Access this chapter

Chapter
USD 29.95
Price excludes VAT (USA)
  • Available as PDF
  • Read on any device
  • Instant download
  • Own it forever
eBook
USD 84.99
Price excludes VAT (USA)
  • Available as EPUB and PDF
  • Read on any device
  • Instant download
  • Own it forever
Softcover Book
USD 109.99
Price excludes VAT (USA)
  • Compact, lightweight 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. Sox HC, Lewis RJ. Pragmatic trials: practical answers to “real world” questions. JAMA. 2016;316:1205–6.

    Article  Google Scholar 

  2. Peto R, Collins R, Gray R. Large-scale randomized evidence: large, simple trials and overviews of trials. Ann N Y Acad Sci. 1993;703:314–40.

    Article  CAS  Google Scholar 

  3. Tunis SR, Stryer DB, Clancy CM. Practical clinical trials: increasing the value of clinical research for decision making in clinical and health policy. JAMA. 2003;290:1624–32.

    Article  CAS  Google Scholar 

  4. March JS, Silva SG, Compton S, Shapiro M, Califf R, Krishnan R. The case for practical clinical trials in psychiatry. Am J Psychiatry. 2005;162:836–46.

    Article  Google Scholar 

  5. Westfall J, Mold J, Fagnan L. Practice-based research – “blue highways” on the NIH roadmap. JAMA. 2007;297:403–6.

    Article  CAS  Google Scholar 

  6. Trochim W. Translation won’t happen without dissemination and implementation: some measurement and evaluation issues. In: 3rd annual conference on the science of dissemination and implementation. Bethesda. 2010.

    Google Scholar 

  7. Green L, Ottoson J, Garcia C, Hiatt R. Diffusion theory and knowledge dissemination, utilization, and integration in public health. Annu Rev Public Health. 2009;30:151–74.

    Article  Google Scholar 

  8. Patsopoulos N. A pragmatic view on pragmatic trials. Dialogues Clin Neurosci. 2011;13:217–24.

    PubMed  PubMed Central  Google Scholar 

  9. Thorpe KE, Zwarenstein M, Oxman AD, et al. A pragmatic-explanatory continuum indicator summary (PRECIS): a tool to help trial designers. J Clin Epidemiol. 2009;62:464–75.

    Article  Google Scholar 

  10. Tosh G, Soares-Weiser K, Adams CE. Pragmatic vs explanatory trials: the Pragmascope tool to help measure differences in protocols of mental health randomized controlled trials. Dialogues Clin Neurosci. 2011;13:209–15.

    PubMed  PubMed Central  Google Scholar 

  11. Patient-Centered Outcomes Research Institute (PCORI). (Posted: September 10, 2014; Updated: March 27, 2017) Retrieved from https://www.pcori.org/about-us/our-story. Accessed 26 Aug 2018.

  12. Star neuroscientist Tom Insel leaves the Google-spawned Verily for…a startup? Wired. https://www.wired.com/2017/05/star-neuroscientist-tom-insel-leaves-google-spawned-verily-startup/. Accessed 26 Aug 2018.

  13. Paulus MP. Evidence-based pragmatic psychiatry-a call to action. JAMA Psychiatry. 2017;74:1185–6.

    Article  Google Scholar 

  14. Eisler I, Simic M, Hodsoll J, Asen E, Berelowitz M, Connan F, et al. A pragmatic randomized multi-centre trial of multifamily and single family therapy for adolescent anorexia nervosa. BMC Psychiatry. 2016;16:422–35.

    Article  Google Scholar 

  15. Olfson M, King M, Schoenbaum M. Treatment of young people with antipsychotic medications in the United States. JAMA Psychiatry. 2015;72:867–74.

    Article  Google Scholar 

  16. Correll CU, Sheridan EM, DelBello MP. Antipsychotic and mood stabilizer efficacy and tolerability in pediatric and adult patients with bipolar I mania: a comparative analysis of acute, randomized, placebo-controlled trials. Bipolar Disord. 2010;12:116–41.

    Article  CAS  Google Scholar 

  17. Goldstein BI, Birmaher B, Axelson DA, Goldstein TR, Esposito-Smythers C, Strober MA, Hunt J, Leonard H, Gill MK, Iyengar S, Grimm C, Yang M, Ryan ND, Keller MB. Preliminary findings regarding overweight and obesity in pediatric bipolar disorder. J Clin Psychiatry. 2008;69:1953–9.

    Article  Google Scholar 

  18. Correll CU. Weight gain and metabolic effects of mood stabilizers and antipsychotics in pediatric bipolar disorder: a systematic review and pooled analysis of short-term trials. J Am Acad Child Adolesc Psychiatry. 2007;46:687–700.

    Article  Google Scholar 

  19. Correll CU, Penzner JB, Parikh UH, Mughal T, Javed T, Carbon M, Malhotra AK. Recognizing and monitoring adverse events of second-generation antipsychotics in children and adolescents. Child Adolesc Psychiatr Clin N Am. 2006;15:177–206.

    Article  Google Scholar 

  20. Patel NC, Hariparsad M, Matias-Akthar M, Sorter MT, Barzman DH, Morrison JA, Stanford KE, Strakowski SM, DelBello MP. Body mass indexes and lipid profiles in hospitalized children and adolescents exposed to atypical antipsychotics. J Child Adolesc Psychopharmacol. 2007;17:303–11.

    Article  Google Scholar 

  21. Jerrell JM, McIntyre RS, Tripathi A. Childhood treatment with psychotropic medication and development of comorbid medical conditions in adolescent-onset bipolar disorder. Hum Psychopharmacol. 2011;26:451–9.

    Article  Google Scholar 

  22. Goldstein BI, Kemp DE, Soczynska JK, McIntyre RS. Inflammation and the phenomenology, pathophysiology, comorbidity, and treatment of bipolar disorder: a systematic review of the literature. J Clin Psychiatry. 2009;70:1078–90.

    Article  Google Scholar 

  23. Fagiolini A, Frank E, Scott JA, Turkin S, Kupfer DJ. Metabolic syndrome in bipolar disorder: findings from the bipolar disorder center for Pennsylvanians. Bipolar Disord. 2005;7:424–30.

    Article  Google Scholar 

  24. Wang PW, Sachs GS, Zarate CA, Marangell LB, Calabrese JR, Goldberg JF, Sagduyu K, Miyahara S, Ketter TA. Overweight and obesity in bipolar disorders. J Psychiatr Res. 2006;40:762–4.

    Article  Google Scholar 

  25. Calkin C, van de Velde C, Ruzickova M, Slaney C, Garnham J, Hajek T, O’Donovan C, Alda M. Can body mass index help predict outcome in patients with bipolar disorder? Bipolar Disord. 2009;11:650–6.

    Article  Google Scholar 

  26. Goldstein BI, Liu SM, Schaffer A, Sala R, Blanco C. Obesity and the three-year longitudinal course of bipolar disorder. Bipolar Disord. 2013;15:284–93.

    Article  Google Scholar 

  27. Galling B, Roldán A, Nielsen RE, Nielsen J, Gerhard T, Carbon M, Stubbs B, Vancampfort D, De Hert M, Olfson M, Kahl KG, Martin A, Guo JJ, Lane HY, Sung FC, Liao CH, Arango C, Correll CU. Type 2 diabetes mellitus in youth exposed to antipsychotics: a systematic review and meta-analysis. JAMA Psychiatry. 2016;73:247–59.

    Article  Google Scholar 

  28. Christian R, Saavedra L, Gaynes BN, et al. Future research needs for first- and second-generation antipsychotics for children and young adults. [Internet]. Rockville: Agency for Healthcare Research and Quality (US); 2012. (Future Research Needs Papers, No. 13).

    Google Scholar 

  29. Maayan L, Correll CU. Management of antipsychotic-related weight gain. Expert Rev Neurother. 2010;10:1175–200.

    Article  Google Scholar 

  30. Correll CU, Sikich L, Reeves G, Riddle M. Metformin for antipsychotic-related weight gain and metabolic abnormalities: when, for whom, and for how long? Am J Psychiatry. 2013;170:947–52.

    Article  Google Scholar 

  31. Fiedorowicz JG, Miller DD, Bishop JR, Calarge CA, Ellingrod VL, Haynes WG. Systematic review meta-analysis of pharmacological interventions for weight gain from antipsychotics and mood stabilizers. Curr Psychiatr Rev. 2012;8:25–36.

    Article  CAS  Google Scholar 

  32. Ehret M, Goethe J, Lanosa M, Coleman CI. The effect of metformin on anthropometrics and insulin resistance in patients receiving atypical antipsychotic agents: a meta-analysis. J Clin Psychiatry. 2010;71:1286–92.

    Article  Google Scholar 

  33. Maayan L, Vakhrusheva J, Correll CU. Effectiveness of medications used to attenuate antipsychotic- related weight gain and metabolic abnormalities: a systematic review and meta-analysis. Neuropsychopharmacology. 2010;35:1520–30.

    Article  CAS  Google Scholar 

  34. Bjorkhem-Bergman L, Asplund AB, Lindh JD. Metformin for weight reduction in non-diabetic patients on antipsychotic drugs: a systematic review and meta-analysis. J Psychopharmacol. 2011;25:299–305.

    Article  Google Scholar 

  35. Das C, Mendez G, Jagasia S, Labbate LA. Second-generation antipsychotic use in schizophrenia and associated weight gain: a critical review and meta-analysis of behavioral and pharmacologic treatments. Ann Clin Psychiatry. 2012;24:225–39.

    PubMed  Google Scholar 

  36. Mizuno Y, Suzuki T, Nakagawa A, Yoshida K, Mimura M, Fleischhacker WW, Uchida H. Pharmacological strategies to counteract antipsychotic-induced weight gain and metabolic adverse effects in schizophrenia: a systematic review and meta-analysis. Schizophr Bull. 2014;40:1385–403.

    Article  Google Scholar 

  37. Alvarez-Jimenez M, Hetrick SE, Gonzalez-Blanch C, Gleeson JF, McGorry PD. Non-pharmacological management of antipsychotic-induced weight gain: systematic review and meta-analysis of randomised controlled trials. Br J Psychiatry. 2008;193:101–7.

    Article  Google Scholar 

  38. Alvarez-Jimenez M, Martinez-Garcia O, Perez-Iglesias R, Ramirez ML, Vazquez-Barquero JL, Crespo- Facorro B. Prevention of antipsychotic-induced weight gain with early behavioural intervention in first-episode psychosis: 2-year results of a randomized controlled trial. Schizophr Res. 2010;116:16–9.

    Article  Google Scholar 

  39. Lowe T, Lubos E. Effectiveness of weight management interventions for people with serious mental illness who receive treatment with atypical antipsychotic medications. A literature review. J Psychiatr Ment Health Nurs. 2008;15:857–63.

    Article  CAS  Google Scholar 

  40. Faulkner G, Soundy AA, Lloyd K. Schizophrenia and weight management: a systematic review of interventions to control weight. Acta Psychiatr Scand. 2003;108:324–32.

    Article  CAS  Google Scholar 

  41. Alvarez-Jimenez M, Gonzalez-Blanch C, Vazquez-Barquero JL, Perez-Iglesias R, Martinez-Garcia O, Perez-Pardal T, Ramirez-Bonilla ML, Crespo-Facorro B. Attenuation of antipsychotic-induced weight gain with early behavioral intervention in drug-naive first-episode psychosis patients: a randomized controlled trial. J Clin Psychiatry. 2006;67:1253–60.

    Article  Google Scholar 

  42. Gierisch JM, Nieuwsma JA, Bradford DW, Wilder CM, Mann-Wrobel MC, McBroom AJ, Hasselblad V, Williams JW Jr. Pharmacologic and behavioral interventions to improve cardiovascular risk factors in adults with serious mental illness: a systematic review and meta-analysis. J Clin Psychiatry. 2014;75:e424–40.

    Article  Google Scholar 

  43. American Diabetes Association, American Psychiatric Association, American Association of Clinical Endocrinologists, North American Association for the Study of Obesity. Consensus development conference on antipsychotic drugs and obesity and diabetes. Diabetes Care. 2004;27:596–601.

    Article  Google Scholar 

  44. Correll CU. Monitoring and management of antipsychotic-related metabolic and endocrine adverse events in pediatric patients. Int Rev Psychiatry. 2008;20:195–201.

    Article  Google Scholar 

Download references

Author information

Authors and Affiliations

Authors

Corresponding author

Correspondence to Claudine Higdon .

Editor information

Editors and Affiliations

Rights and permissions

Reprints and permissions

Copyright information

© 2019 Springer Nature Switzerland AG

About this chapter

Check for updates. Verify currency and authenticity via CrossMark

Cite this chapter

Higdon, C., Eichenbaum, R.M., DelBello, M.P. (2019). Pragmatic Trials: Solving the Dilemma of Psychiatric Nonadherence. In: Fornari, V., Dancyger, I. (eds) Psychiatric Nonadherence. Springer, Cham. https://doi.org/10.1007/978-3-030-12665-0_13

Download citation

  • DOI: https://doi.org/10.1007/978-3-030-12665-0_13

  • Published:

  • Publisher Name: Springer, Cham

  • Print ISBN: 978-3-030-12664-3

  • Online ISBN: 978-3-030-12665-0

  • eBook Packages: MedicineMedicine (R0)

Publish with us

Policies and ethics