Countries across the globe are realizing the significant impact rare diseases have on their populations, and are responding by putting national policies in place to address these unmet medical needs. BIO applauds the government of Brazil for taking steps towards developing regulatory requirements to ensure patients suffering from rare diseases have access to treatment through certified reference centers, for putting forth a validated and accepted definition of rare disease, and for prioritizing access to diagnostic and therapeutic resources. At the same time, policies that help to support and advance orphan drug development, approval and access should also be considered in the formulation of a national rare disease policy.
Thus, BIO believes that significant strides in the treatment of rare diseases are achievable by leveraging and coordinating Brazil’s existing services and capabilities—improving the organization of assistance to these patient populations, providing for earlier diagnosis, and developing well-trained, assessable provider networks—as well through more efficient and broader access to appropriate medicines.
The following comments provide additional areas for consideration.