Category Archives: Orphan Drug

C 039: INTERNATIONAL COOPERATION AND ACTIONS FOR ORPHAN DRUGS

J Pharm Pharmacogn Res 2(Suppl. 1): S109, 2014

Special supplement with the abstract book of LATINFARMA 2013

Conference

C 039: INTERNATIONAL COOPERATION AND ACTIONS FOR ORPHAN DRUGS. INCLUDING LATIN-AMERICA AND CARIBBEAN WITHIN THE INTERNATIONAL LANDSCAPE

Llera VA.

ICORD/GEISER, Argentina.
Abstract

In the field of rare disorders the quality of epidemiological data and knowledge of the regional situation is what permits the addition of efforts for the development of strategies in the search of solutions. Due to the “rare” nature of the medical conditions the isolated plans has fail or resulted costly. There are around 7 thousands of different rare diseases that demands medical research and may account for 6% of the population. In order to expedite efficient solutions, as much as possible, the international research community if jointly working in this sense. The possibilities of introducing LA&C countries are now approaching. Therefore we will develop the conditions and chances required to fulfill this joint commitment.

C 038: ECONOMIC EVALUATION AND ORPHAN DRUGS

J Pharm Pharmacogn Res 2(Suppl. 1): S109, 2014

Special supplement with the abstract book of LATINFARMA 2013

Conference

C 038: ECONOMIC EVALUATION AND ORPHAN DRUGS

Gálvez González AM.

Escuela Nacional de Salud Pública de Cuba, La Habana, Cuba.
Abstract

Introduction: Orphan drugs financing decisions are related with different criterion. Literature reports various papers explaining the importance of economical assessment on orphan drugs designed to treat rare diseases.

Objective: The main goal of this paper is to highlight the importance of economic evaluation of orphan drugs for the decision making and its complexity in different context.

Method: A review of literature was done employing Big 6 model for the information competences management. Consults to experts in pharmacoeconomy, family doctors and decision makers in drugs area were done.

Results: The economic analysis of orphan drugs is a public health problem. Presently, it is a theme exposed in different space of discussion. Other economical items are important too, for example, to guarantee the equitative access to orphan drugs, the importance of enhance the knowledge in professionals about this subject, costs analysis , burden of rare diseases, financing and budgetary impact of orphan drugs. Finally international cooperation is identified as a key to develop future studies.

C 037: ORPHAN DRUGS: DEFINITIONS, RESEARCH & DEVELOPMENT, MASTER PLANS, ACCESIBILITY AND BIOETHICAL ASPECTS

J Pharm Pharmacogn Res 2(Suppl. 1): S108, 2014

Special supplement with the abstract book of LATINFARMA 2013

Conference

C 037: ORPHAN DRUGS: DEFINITIONS, RESEARCH & DEVELOPMENT, MASTER PLANS, ACCESIBILITY AND BIOETHICAL ASPECTS

Roldán E.

GEISER Foundation, Buenos Aires – Argentina.
Abstract

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.