In 2017, Speyside supported a rare disease pharmaceutical company in introducing an orphan drug for Spinal Muscular Atrophy (SMA) into the Brazilian market. The project strengthened patient associations, built relationships with legislators, and facilitated meetings between advocacy groups, regulators, and the Ministry of Health. These efforts led to ANVISA approving the drug less than a year after FDA registration, sparked a public hearing that advanced reimbursement discussions, and united 10 patient advocacy groups under shared action objectives.

In 2017, a pharmaceutical company specializing in rare diseases engaged Speyside to introduce an orphan drug into the Brazilian market. At that time, the drug had not been registered with ANVISA, nor had its price been determined by CMED.

Strengthening of patient associations by training workshops on advocacy and public policy.

Mapping and relationship building with federal and state legislators to raise awareness about Spinal Muscular Atrophy (SMA).

Articulation of meetings in Anvisa between legislators and Patient Advocacy Groups.

Engagement with key stakeholders of the Ministry of Health to discuss future possibility of incorporation of the drugs.

ANVISA approved the registration of the first drug to treat SMA in Brazil, less than 1 year after the FDA registration.

Impulse to the debate on the disease generated a public hearing on the registration of people with rare diseases in the House of Representatives, which motivated the engagement for the public reimbursement of the drug.

10 Patient Advocacy Groups defined common action objectives for 2018, developing more coordinated work.