The Case for Practical Clinical Trials in Psychiatry
ABSTRACT Clinical trials in psychiatry frequently fail to maximize clinical utility for practicing clinicians, or, stated differently, available evidence is not perceived by clinicians (and other decision makers) as sufficiently relevant to clinical practice, thereby diluting its impact. To attain maximum clinical relevance and acceptability, researchers must conduct clinical trials designed to meet the needs of clinicians and others who are making decisions about patients' care. The authors present the case for psychiatry's adoption of the practical clinical trials model, which is widely used in research in other areas of medicine.
The authors outline the characteristics and scope of practical clinical trials, give examples of practical clinical trials, and discuss the challenges of using the practical clinical trials model in psychiatry, including issues of funding.
Practical clinical trials, which are intended to provide generalizable answers to important clinical questions without bias, are characterized by eight key features: a straightforward clinically relevant question, a representative sample of patients and practice settings, sufficient power to identify modest clinically relevant effects, randomization to protect against bias, clinical uncertainty regarding the outcome of treatment at the patient level, assessment and treatment protocols that enact best clinical practices, simple and clinically relevant outcomes, and limited subject and investigator burden.
To implement the practical clinical trials model in psychiatry will require stable funding for network construction and maintenance plus methodological innovation in governance and trial selection, assessment, treatment, data management, site management, and data analytic procedures.
- SourceAvailable from: Sofia Brissos
[Show abstract] [Hide abstract]
- "Finally, the few studies that report tolerability data [Adams et al. 2001; Marchiaro et al. 2005; Haro et al. 2006] did not use direct measures (i.e. rating scales) but clinical observations regarding anticholinergic use. "
ABSTRACT: Despite their widespread use, long acting injectable (LAI) antipsychotics (APs), are often regarded with some negativity because of the assumption of punishment, control and insufficient evolution towards psychosocial development of patients. However, LAI APs have proved effective in schizophrenia and other severe psychotic disorders because they assure stable blood levels, leading to a reduction of the risk of relapse. Therapeutic opportunities have also arisen after introduction of newer, second-generation LAI APs in recent years. Newer LAI APs are more readily dosed optimally, may be better tolerated and are better suited to integrated rehabilitation programmes. This review outlines the older and newer LAI APs available for the treatment of schizophrenia, with considerations of past and present pharmacological and therapeutic issues. Traditional, evidence-based approaches to systematic reviews and randomized clinical trials are of limited utility in this area so this paper’s blending of experimental trials with observational research is particularly appropriate and effective.Therapeutic Advances in Psychopharmacology 07/2014; DOI:10.1177/2045125314540297 · 1.53 Impact Factor
[Show abstract] [Hide abstract]
- "Our current stage model for moving EBP from research to community settings tends to overemphasize internal validity, thus inadvertently deferring feedback until too late in the development process, particularly when the transfer of EBP relies on social processes. Certain methodological approaches could ameliorate some of the tension between internal and external validity such as practical clinical trials (e.g., March et al. 2005; Tunis et al. 2003) and hybrid efficacy-effectiveness models (Carroll and Rounsaville 2003; Curran et al. 2010). For example, Dimeff and colleagues (2009) propose a hybrid model that addresses both efficacy (e.g., randomization, assessment, intent-to-train) and effectiveness (e.g., naturalistic environment, limited exclusion criteria, sample representative of community mental health clinicians). "
ABSTRACT: This review offers practical recommendations regarding research on training in evidence-based practices for mental health and substance abuse treatment. When designing training research, we recommend: (a) aligning with the larger dissemination and implementation literature to consider contextual variables and clearly defining terminology, (b) critically examining the implicit assumptions underlying the stage model of psychotherapy development, (c) incorporating research methods from other disciplines that embrace the principles of formative evaluation and iterative review, and (d) thinking about how technology can be used to take training to scale throughout all stages of a training research project. An example demonstrates the implementation of these recommendations.Administration and Policy in Mental Health and Mental Health Services Research 03/2011; 38(4):223-37. DOI:10.1007/s10488-011-0338-z · 3.44 Impact Factor
[Show abstract] [Hide abstract]
- "" Usually an overall relative risk is applied in the decision model to the baseline rate in the specific population . Fourth, there is the problem of publication bias: negative trial results are less often published than positive ones   . Further issues are more specific for clinical trials with medical devices as opposed to those with drugs. "
ABSTRACT: In this article we investigate the possibility to account for selection bias in observational data by using econometric techniques. One-year costs of 15,237 patients who received a drug-eluting stent (DES) or a bare metal stent (BMS) in Belgium in 2004 were compared. The treatment choice between DES and BMS could be influenced by patient characteristics; therefore, cost estimates could be biased by overt and/or hidden selection bias. Overt bias was addressed by regression adjustment and propensity score matching. Hidden selection bias was dealt with by using an instrumental variable (IV) approach. Due to the higher purchase price DES patients incur higher (unadjusted) costs in the short-term; these costs are, however, compensated in the long-term due to less in-stent restenosis and hospitalizations. Analyses indicated that, for the diabetic population, the null hypothesis of similar average 1-year costs of patients receiving a BMS or DES cannot be rejected. For the non-diabetic patients a significant cost difference between BMS and DES patients was found. It cannot be ruled out that the treatment-effect model does not correct for all observable or unobservable characteristics and that the estimated treatment effect is biased, possibly due to weak instruments. It is interesting and necessary to explore the use of econometric tools in cost and cost effectiveness analysis to investigate the effect of a technology in everyday practice and to take into account patient and disease characteristics and uncertainty. Further research is however necessary to investigate how we can fully correct for selection bias when using observational data.Value in Health 01/2011; 14(1):3-14. DOI:10.1016/j.jval.2010.10.014 · 2.89 Impact Factor