A civil society view of rare disease public policy in six Latin American countries

Abstract

Patients with rare diseases across the world struggle to access timely diagnosis and state-of-the-art treatment and management of their conditions. Several recently published reviews highlight the importance of country efforts to address rare diseases and orphan drugs policy comprehensively. However, many of these reviews lack depth and detail at the local level, which we believe is necessary for rare disease advocates to identify and prioritize opportunities for strengthening each country's policy framework.We asked leading patient advocates from civil society organizations their views on rare disease public policy in Argentina, Brazil, Chile, Colombia, Mexico, and Peru with a focus on whether specific laws and regulations in these six Latin American countries have been promulgated. From December 2018 to March 2019 we supplemented their perspectives with evidence from accessible literature using key search terms. For each country, we prepared a detailed analysis on how laws or other policy initiatives took shape and the steps taken since to implement them. This allowed us to identify five broad policy categories for subsequent analysis: national laws, national regulations, health system incorporation of rare disease treatments, care delivery, and patient engagement.By describing the different approaches, challenges and timelines across six countries, our research demonstrates that strengthening rare disease policy first requires a common understanding and local consensus of each country's recent past and current situation. Subsequent analysis based on a set of common policy dimensions led us to where we believe salient opportunities lie for each of these countries to strengthen their overall policy framework for rare disease patients.

Keywords: Health system incorporation; Latin America; Legislation; National laws; Orphan drugs; Patient advocacy; Patient engagement; Policy; Rare diseases; Regulations.