More Efficient Ways of Assessing Treatments for Neglected Tropical Diseases Are Required: Innovative Study Designs, New Endpoints, and Markers of Effects

Abstract

Shortening trial time and reducing requirements for patients saves time and money, and spares patients from unnecessary exposure: there is therefore both an economic and an ethical motive for rationalizing trial design. Economies can be found with alternative clinical trial designs, such as adaptive trials (especially in the futility setting), though these are only partly suited for neglected tropical diseases, which have inadequate measures of treatment outcomes. Research is needed into generating better tests of treatment outcomes for neglected tropical diseases, but sizeable long-term investments are required. New, imaginative approaches should be investigated that will generate an evidence base for alternative trial designs