Orphan drugs: drama or challenge?
Keywords:
ORPHAN DRUG PRODUCTION, RARE DISEASES, PUBLIC HEALTH.Abstract
Introduction: orphan drugs emerged for the treatment of rare diseases, characterized by their high cost, being of great importance due to their global impact on public health and society.
Objective: to describe the meaning of orphan drugs, features of the process of obtaining them, prices and challenges in their application.
Methods: A narrative literature review was carried out by means of an unstructured information search using publications in English and Spanish. PubMed, ScIELO, Scopus were the databases consulted through Google Scholar. For this purpose, the terms: orphan drugs/rare diseases were used and 15 articles were selected out of 30 detected, based on their importance in recent years in the approach to this topic. The analytical-synthetic method was used to assess and interpret the documents consulted.
Development: it was specified that the so-called orphan drugs are drugs not developed or marketed by the pharmaceutical industry, with a high cost, low profitability, low demand due to the low prevalence of rare diseases, which express the validity of the health care and social drama as a global dilemma. Conclusions: Orphan drugs are an unresolved health care need for rare diseases in today's world.
Conclusions: the high prices of orphan drugs associated with the low profitability of the pharmaceutical industry in the research-production-market process are part of the imperative development dilemma faced by health systems.
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