Abstract: One view on why dry eye clinical trials fail and where we go from here

With the recent demise of what were once promising dry eye therapies in advanced clinical trials, this is a timely (if perhaps incomplete) summary of why we don't seem to get anywhere with dry eye drug clinical trials, by well known leader in the field Michael Lemp MD.

Clinical trials in dry eye in surgery for dry eye?
Dev Ophthalmol. 2008;41:283-97.
Lemp MA.

Purpose: To provide an overview of considerations in the design and performance of prospective clinical trials in the evaluation of new pharmaceutical and surgical treatments in dry eye disease (DED). Design: A compilation and interpretation of experiences in the challenges and pitfalls of clinical trial design based on experiences documented in the peerreviewed literature over the last 40 years.

Methods: A review of the literature in the design and performance of clinical trials in DED with an interpretative and prognostic outlook.

Results: Published results of clinical trials in DED reveal problems in the design of clinical trials which are unique to this disease. These include a discordance between the signs and symptoms of DED, variability in disease course and short-term environmental effects on ocular surface staining.

Conclusions: The development of better efficacy endpoints will be necessary to improve the outcomes of clinical trials to evaluate new pharmaceutical and surgical approaches to the management of dry eye. The most promising field is that of biomarkers which serve as surrogates for disease severity. As these markers undergo validation with clinical changes, it is likely that they will assume greater significance in future clinical trial designs.