J Pharm Pharmacogn Res 2(Suppl. 1): S10, 2014
Special supplement with the abstract book of LATINFARMA 2013
CO 021: CLINICAL TRIAL DESIGNS THAT CAN SPEED UP NEW DRUG DEVELOPMENT
López-Saura PA, Valenzuela-Silva CM.Center for Genetic Engineering and Biotechnology; La Habana, Cuba. E-mail: firstname.lastname@example.org
The “bottle-neck” of new product development in the last years has been safety and efficacy demonstration in the clinical setting, which is the most expensive stage as well. Following the established regulatory paradigm, this evaluation must follow the sequential phase I-IV pathway, which is quite time-consuming. A new approach is required in order to optimize this process. Recently, innovations concerning clinical trials design and interpretation have been introduced. They include adaptive designs where “bridges” between subsequent phases (I – II or II – III) can be envisaged. Improvements in knowledge on the biological mechanisms involved in disease and drug actions also permit a better rationale for trials justification and evaluation. Adaptive designs permit the modification of one or more aspects in a prospective way, based on analyses of data gathered from study subjects as long as they are obtained. These modifications should be previewed in the trial protocol. Examples of such trials, sponsored by the Center for Genetic Engineering and Biotechnology where these techniques have been used for several years (with the agreement of the corresponding ethics committees and the Cuban regulatory authority) will be shown. More than 300 subjects have been “saved” to be included in those trials, including more than one third that would had received a placebo. A more flexible and rational regulatory paradigm is proposed where an exploratory phase is conceived to gain knowledge on the products’ clinical performance in order to go into a confirmatory phase to attain approval.