Sponsors of psychiatric clinical trials may face significant challenges in collecting robust, quality data to support the efficacy and safety of investigative compounds.
Identifying and mitigating study design-, subject-, and site-related factors that may influence data quality as early as possible in program development can help to drive study success. Ensuring that psychiatric clinical trial data accurately captures a compound’s safety and efficacy potential requires that sponsors account for the challenges inherent in working with a mentally ill population. Mental illness and its manifestations are almost as varied as the spectrum of human behavior. Understanding the interactions among the myriad genetic, biological, social, and environmental factors that give rise to mental illness is a complex task, which makes clinical trials involving psychiatry drugs quite a challenge.
Observing changes in the disease under study is, in part, a subjective process, and that subjectivity creates risk of undue influence that the patient and/or investigator may not recognize. To further complicate matters, not all symptomatic improvement in mental disorders can necessarily be tied to the product under investigation. Some changes may result from unrelated factors whose therapeutic effect erodes the efficacy signal of a potential treatment.
In this white paper, we explore a range of factors that, if identified and mitigated early in the development process, can maximize the potential for conclusive study results in psychiatric clinical trials.
Increased difficulty in demonstrating a treatment effect
Sponsors of psychiatric clinical trials may need to contend with a decreasing ability to demonstrate a treatment effect. In September 2007, a collaborative session between the International Society for CNS Clinical Trials and Methodology and the International Society for CNS Drug Development focused on the apparent reduction of drug-placebo differences in recent multicenter trials of antipsychotic medications for schizophrenia.1 During this joint session, presenters reported data from several recent trials that indicated higher rates of placebo response and lower rates of drug response – even to previously established comparator drugs – when compared with earlier trials. The consequent decrease in separation between treatment and control groups over time made it more difficult to obtain conclusive results.