1. Associação Brasileira de Paramiloidose
Brazilian Association of TTR Amyloidosis
1st European ATTR Meeting for Patients and Doctors
Paris, France. November 2017

2. Best Practice For Patient Organization
Advocacy: drug access
The milestone for rare diseases in Brazil happened in 2014, with the institution of the National Policy of Attention to People with Rare Diseases.
There were only 37 clinical protocols and therapeutic guidelines for rare diseases in the Brazilian public health system.
These protocols define how a person with a specific disease is diagnosed, treated and accompanied.
This year we began discussions with the Ministry of Health and a priority for hATTR was given.

3. Timeline:
1) May Meeting with Ministry of Health - priority set for creation of hATTR guidelines in public health system.
2) June Meeting with Pfizer Europe. Portuguese Association helped us to set this appointment.
3) October 2014 – public consultation about rare diseases guidelines – hATTR receive 82 contributions.
4) First Half 2015 – Ministry of Health select workgroup members: 9 physicians and I representing the patient association; Doctor Márcia Waddington Cruz is workgroup coordinator.
5) June workgroup send scope of guidelines to Ministry of Health.
6) September 2015 – pharma request market authorization for Tafamidis to Medicines Agency.
7) November 2016 – Medicines Agency approve Tafamidis.

4. 8) February 2017 - Interministerial Chamber approve price for Tafamidis.
9) May 2017 – Hearing in Ministry of Health with association and national reference center representatives.
10) June meeting to assess the application for Tafamidis reimbursement at WHO headquarters in Brazil. Ministry of Health give positive opinion for reimbursement.
11) August 2017 – public consultation about Tafamidis reimbursement receive more than 1,000 contributions.
12) November 7, Tafamidis reimbursement will be approved.
13) First half 2018 – patients will have access to Tafamidis according hATTR guidelines.
The patient association led this process and cooperation with our national reference center was essential to achieve this goal!

5. Michel de Montaigne (1533 – 1592) – french philosopher
“Health is a precious thing, and the only one, in truth, meriting that a man should lay out not only his time, sweat, labor and goods, but also life itself to obtain it”.
Michel de Montaigne (1533 – 1592) – french philosopher

6. THANK YOU!