J Pharm Pharmacogn Res 2(Suppl. 1): S108, 2014
Special supplement with the abstract book of LATINFARMA 2013
C 037: ORPHAN DRUGS: DEFINITIONS, RESEARCH & DEVELOPMENT, MASTER PLANS, ACCESIBILITY AND BIOETHICAL ASPECTS
Orphan drugs are those in which cost of discovery and production overpass the recovery of Investments, hence there are only few available usually at a very high cost and not in all the countries. Therefore new formulas require to be managed in order to cover unmet medical needs and comply assistance to the population with equality. These formulas includes the understanding of the magnitude of the problem, the achievement of specific health and research policies, the organization of regional nets of information and research within master plans, and political decisions well harmonized with the common interest of the region. We hereby illustrate the current scenario in Latin-American and Caribbean countries with some examples and propose actions. Typically, researchers’ interested in orphan drugs are challenged by the lacks of information, poor development of diagnostic techniques, difficulties to contact patients, lack of financial support and/or academic programs, and with frequency minimal peer impact, discouraging the individual initiatives. Nevertheless, the field of orphan drugs is now envisioned as one of the main sources of discovery in the fields of biology and medicine, and one of the most productive in knowledge, goods, and human education. Therefore the current conditions should be quickly transformed and insert the capabilities of the local production within global master plans built under consensus.
Many academic centers in Latin-America and Caribbean are highly prestigious world-wide and their human resources and knowledge will be welcome to the research and development of orphan products. Some differential bioethics aspects and transparency of interest will also be discussed as the affected people should be considered within the vulnerable population from several points of view.