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: Hyung-Jun Yoon[Show abstract] [Hide abstract]
ABSTRACT: Objective：Attention-deficit/hyperactivity disorder (ADHD) is a common, lifelong condition associated with major functional impairment, and remission is the primary goal of treatment. The purpose of this study was to identify differences in the clinical characteristics of remission and non-remission groups composed of Korean children and adolescents with ADHD. Methods：Fifty-nine children and adolescents, 6-15 years old, diagnosed with ADHD according to the Diagnostic and Statistical Manual-IV (DSM-IV) criteria were included in the study. The study design was an 8-week, open-label trial of OROS-methylphenidate (OROS-MPH) monotherapy. The subjects were assessed using the Korean ADHD Rating Scale (K-ARS), Clinical Global Impression of Severity (CGI-S), Clinical Global Impression of Improvement (CGI-I), and Barkley Side Effect Rating Scale at baseline and 1, 2, 4, and 8 weeks after starting OROS-MPH treatment. Remission was defined as both a score of ≤2 on the CGI-S and a score of ≤18 on the K-ARS at the eighth week of the trial, when we examined the differences in the clinical characteristics between the remission and non-remission groups. Results：The remission rate at the eighth week was 47.5% (n=28). No differences were observed in the age, sex, weight, severity of symptoms reported by the parent, comorbidities at baseline, or doses of OROS-MPH at each evaluation point between the remission and non-remission groups. However, the non-remission group had higher scores in the CGI-S at baseline than the remission group. A difference in symptom improvement between the two groups began at the first week of the trial, and the remission group was less likely to have side effects at the eighth week. Conclusion：The results suggest that individual biological diversity may mediate different treatment responses to OROS-MPH. Interventions other than medication are needed to achieve remission and to restore proper functioning of patients with ADHD in the nonremission group. KEY WORDS：Attention-deficit/hyperactivity disorder (ADHD); OROS-methylphenidate (OROS-MPH); Remission.Clinical Psychopharmacology and Neuroscience 01/2008; 6(1):24-30.