Randomized clinical trials in single patients during a 2-year period

Area of Science:

• Clinical research methodology
• Pharmacoeconomics
• Evidence-based medicine

Background:

• Single patient trials (SPTs) offer a personalized approach to treatment evaluation.
• Assessing the feasibility and impact of a formal SPT service is crucial for optimizing patient care.

Purpose of the Study:

• To evaluate the feasibility of implementing a single patient trial (SPT) service.
• To determine the influence of formal SPTs on therapeutic precision and clinical decision-making.

Main Methods:

• A descriptive and evaluative study of SPTs conducted over two years (1988-1990).
• Trials were double-blind, randomized, multiple crossover designs with random assignment and explicit outcome assessment.
• Patient and physician confidence levels were measured using visual analog scales pre- and post-trial.

Main Results:

• Of 34 completed SPTs, 17 yielded definitive results. Treatment was continued or initiated in 16 trials based on positive outcomes.
• Treatment was discontinued or not initiated in 18 trials due to ineffectiveness, harm, or equivalence.
• Physicians' confidence increased or decreased based on trial outcomes, while most patients reported no change; the service was rated highly useful.

Conclusions:

• A single patient trial (SPT) service is feasible, with costs comparable to conventional services.
• SPTs enhance clinician confidence and therapeutic precision, particularly for uncertain treatment values in chronic diseases.
• Formal SPTs can effectively guide subsequent treatment decisions when uncertainty exists regarding treatment efficacy or safety.