RANDOMIZED CLINICAL-TRIALS IN SINGLE PATIENTS DURING A 2-YEAR PERIOD

Objective.-To describe the feasibility of a single patient trial (SPT) service and study the influence of formal SPTs on therapeutic precisi on. Design.-Descriptive and evaluative study of SPTs. All planned tria ls were double-blind, randomized, multiple crossover trials. Other key features of individual trials were random assignment of order and ass essment of predetermined explicit outcomes. Patients and physicians ra ted level of confidence in treatment before and after the SPT on visua l analog scales. Setting.-Two-year experience (September 1988 to Septe mber 1990) of an SPT trial referral service available to physicians in an academic medical center. Outcome Measures.-The number of planned a nd completed SPTs; proportion of completed trials yielding definitive answers; patient- and physician-rated levels of confidence in treatmen t pre- and post-SPT; time-motion studies to estimate resource consumpt ion (costs) for selected SPTs. Results.-Of 34 completed trials, 17 wer e judged to give definitive results whereas 17 showed trends only. Res ults favored active treatment in 16 trials that led to treatment being continued (nine patients) or started (seven patients). Treatment was discontinued (seven patients) or not started (11 patients) based on 18 trial results that demonstrated active treatment was ineffective (sev en), harmful (two), or apparently equivalent (nine). Most patients (65 %) reported no change in their already high level of confidence in the rapy as a result of trials, whereas physicians' confidence levels in t herapy either increased or decreased post-SPT depending on the directi on of trial results. Patients consistently rated the SPT service as ex tremely useful. Time-motion estimates indicate that 16.75 staff hours were spent per trial leading to a direct cost estimate of approximatel y $450 to $500 per trial. Conclusion.-We conclude that an SPT service is feasible, trial costs compare favorably with other conventional ser vices, and clinicians appear to gain confidence and precision from SPT s. When patients or clinicians are uncertain about the value (includin g the possibility of side effects) of treatment for symptomatic chroni c diseases, we believe an SPT can be offered to a patient and will lik ely yield results that will effect subsequent treatment.