All Times EDT
Keywords: Registry, long-term safety, ICH E1
In the absence of adequate information about the relationship of AEs to treatment duration, a specific number of patients are followed for at least a 1 year. The specification of the number of patients is to a large extent a judgement call based on the estimated probability of detecting a given AE frequency level and practical considerations. For products intended for long-term treatment of non-life-threatening conditions, (e.g., continuous treatment for 6 months or more, or recurrent intermittent treatment where cumulative treatment equals or exceeds 6 months), the ICH E1 guideline and FDA have generally recommended that 1500 subjects be exposed to the investigational product (with 300 to 600 exposed for 6 months, and 100 exposed for 1 year). Designs of these long-term extension trials range from the use of a catch-all, uncontrolled, open-label design, with patients coming from different originating studies, to a randomized design with active comparators. This talk will give a general survey of these studies and the implications on the assessment of long-term safety. It poses the question as to whether there are efficient ways of performing such evaluations.