ACCESS LANDSCAPE
for Rare Disease Medicines in Brazil

AHF has conducted a survey to collect relevant data from a diverse group of stakeholders involved in rare diseases to bridge the gaps in the landscape assessment for Brazil and deepen the understanding of the orphan medicines environment in the country. The data obtained has the potential to improve patient access to orphan drugs and evaluate the effectiveness of current programs or policies.

The survey reached the impressive mark of 100 responses, including the perspectives of 30 patient organization members and leaders, 30 physicians, 20 regulators, 18 payers, and 3 policymakers, all with experience in the field of access for orphan drugs in the country.

The resulting report can serve as a tool for different stakeholders to identify critical gaps that hinder patient access to rare disease drugs, informing and improving the development of innovative solutions, programs, and approaches to address and overcome identified gaps.

Thank you to Amicus Therapeutics for an unrestricted grant to support this project, which was conduced independently by AHF, avoiding perception bias.

ISPOR POSTER 1: Patient Access Landscape for Rare Disease Medicines in Brazil.

THE LANDSCAPE OF RARE DISEASE CARE IN LATIN AMERICA FROM A MULTI-STAKEHOLDER PERSPECTIVE:
focus on Pompe and Fabry diseases

Access Pathways, Challenges, and Opportunities for Rare Disease Treatments in Latin America
In 2023, the Americas Health Foundation conducted a multi-country Task Force on the access pathways, challenges, and opportunities for rare disease treatments in Brazil, Chile, Argentina, Colombia, Mexico, Uruguay, and Peru. The task force aimed to develop recommendations and innovative approaches to improve and facilitate access to rare disease treatments for patients in Latin America.

In a series of country-level meetings moderated by AHF, a total of 35 key opinion leaders including geneticists, patient organization representatives, and other relevant stakeholders convened. Findings were compiled into country-level reports containing deep insights into the rare disease market in each of the selected Latin American countries. Stay tuned for the full report.

Thank you to Amicus Therapeutics for an unrestricted grant to support this project, which was conducted independently by AHF, avoiding perception bias.

ISPOR POSTER 2: Multi-Stakeholder Approach to Rare Disease Care in Latin America.