Abstract
Compared to a decade ago, nearly three times as many drugs for rare diseases are slated for development. This article addresses the market access issues associated with orphan drug status in Europe and the United States in contrast to the legislation in five Latin American (LA) countries that have made strides in this regard--Mexico, Brazil, Colombia, Chile and Argentina. Based on the success of orphan drug legislation in the EU and US, LA countries should strive to adopt similar strategies with regard to rare diseases and drug development. With the implementation of new targeted regulations, reimbursement strategies, and drug approvals, accessibility to treatment will be improved for people afflicted with rare diseases in these developing countries.
Highlights
Medications for the approximately 7,000 rare diseases in the world account for less than 10% of global pharmaceutical spending
This article focused largely on legislation in order to address some of the inadequacies relevant to making drugs available for orphan diseases; there are many mitigating factors surrounding the debate on market access
As indicated in a recent European review of EU orphan drug policies[18], society’s values in regards to funding these drugs, which would never be considered as cost-effective as drugs for more prevalent diseases, need to be clarified in Latin American (LA)
Summary
Medications for the approximately 7,000 rare diseases in the world account for less than 10% of global pharmaceutical spending. This takes into account the assistance required by the health care managers in regulating the access, control and evaluation of assistance for people with rare diseases in the SUS This new national policy establishes the guidelines for patients with rare diseases and the program financial funding for SUS. Specific recommendations for harmonization (based on suggestions of the international biotechnology industry organization [BIO]) The Biotechnology Industry Organization, a not-for-profit trade association representing more than 1,100 companies, academic centers and research institutions in over 30 countries globally, constructed a letter commenting on the Brazilian Ministry of Health’s proposed Standards for Enabling Specialized Care Services and Reference Centers for Rare Diseases in the SUS and Guidelines for Integral Care for People with Rare Diseases in the SUS15 These recommendations, while not universally appropriate for/specific to all LA countries, are useful as a benchmark for many developing nations.
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