Brazil has universal healthcare coverage and provides access to treatments for rare diseases through a special drug access program. Under this program, drugs are only obtained through public healthcare centers according to approved clinical protocols. The costs of these drugs are shared between the federal and state governments. While Brazil has provided treatments for rare diseases since the 1970s, developing its policies and reimbursement lists over time, challenges remain in providing access to all approved treatments given budget and evidence constraints.

1. Brazilian Experience with drug policy for rare diseases Fábio Mataveli, MD, PhD Novartis Oncology, Brazil Former Brazilian Drug Regulatory Agency

3. Demographic Transition 1980 2010 Costs with chronic and rare disorders jumped from US$323,8 million in 2002 to US$ 809,5 million in 2005. 250% increase in 3 years

6. Orphan drug policy started in the 70’s Special drug stock should be maintained for the treatment of patients with rare incidence diseases. 1971 Brazilian government requested University of Brasilia to develop a program to locally produce Human Growth Hormone 1971 1977 Drug dispensation according to a established Clinical Protocol for Nanism Treatment 1982 15% of the total Drug Budget For Exceptional Drugs 1986 Reimbursement List with 55 items

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