1st Brazilian Forum on Health Technology Assessment for Rare Diseases

Published Feb 13, 2023

The first edition of the Brazilian Forum on Health Technology Assessment for Rare Diseases was held on November 24th and 25th, 2022, completely online and free. Held by Unidos pela Vida - Brazilian Institute for Cystic Fibrosis Care, it was the first event of its kind held by a rare disease patient advocacy group. The event had almost 1200 subscribers - among health professionals (35.90%), students (21.42%), pharma industry professionals (14.31%), patients and family members (14.14%), patient advocacy groups (3.81%) and other interested people (10.41%) - and its transmission had more than 16.3 thousand views on the platform.

On the first day, the first roundtable called “HTA for Rare Diseases” was attended by the former Secretary of Science, Technology and Strategic Inputs of the Ministry of Health of Brazil (2019-2020), Dr. Denizar Vianna, the president of the Research Pharmaceutical Industry Association (Interfarma), Eduardo Calderari, the health lawyer and patient advocate, Dr. Tiago Farina, and the Director of the Department of Management and Health Technology Assessment of the current Ministry of Health, Vânia Canuto. The specificities of the HTA process for rare diseases, innovative therapies for rare diseases, the role of the pharmaceutical industry and health professionals in the process were addressed.

The second panel had the theme “Economic Health Assessment of Rare Diseases” and the participation of pediatrician and geneticist Dr. Salmo Raskin, health economist and international consultant, Dr. André Cezar Medici, and pharmacist and consultant Dr. Santusa Pereira. The topics discussed were: economic evaluation and the social cost of rare diseases, in addition to the challenge of clinical research on this type of pathology in Brazil.

The second day began with the first table “Reports of experiences in HTA for Rare Diseases”, which was attended by the Director of Public Policies and Advocacy of Unidos pela Vida, Cristiano Silveira, the President of the Fernando Loper Vasconcellos Institute, Adriane Loper, and the Founder of Multiple Friends for Sclerosis (AME), Gustavo San Martin. Respectively, they presented the experiences of social engagement in health technology evaluation processes of their patient associations representing cystic fibrosis, spinal muscular atrophy and multiple sclerosis respectively.

The second last table of the day had “Social Engagement” as its guiding theme and featured lectures by the Founder and Executive Director of Unidos pela Vida, Verônica Stasiak Bednarczuk de Oliveira, by the Consultant in Health Policies and Advocacy and FIPE professor, Dr. Cristina Guimarães Rodrigues, and law student Daniel Wainstock. The relationship between public policies, advocacy, social engagement, the perspective of the patient in the HTA process and international perspectives were discussed in the context of rare diseases.

Divided into two mornings and four round tables, the event can be watched in full in Brazilian Portuguese (without subtitles or dubbing in another language) on the organization's YouTube https://www.youtube.com/@institutounidospelavida from mid-January.

“Gathering researchers, scholars, government representatives, the Brazilian HTA Agency, patients, patient associations and economists in the same event was a milestone. I have been dreaming and working for this moment since I founded Unidos pela Vida in 2011, shortly after being late diagnosed with cystic fibrosis. Our focus here at the Institute, and my personal focus of research and action today, both as a master's researcher, as executive director of Unidos pela Vida and as a patient advocate, is precisely to qualify patient engagement and also to encourage the qualification of the evaluators who receive real-life reports with evidence of the use of technologies, so that they have a real impact and are considered transparently in the decision-making process. In addition, we need to shed light on and endorse the discussion on the recent approval in Brazil of the cost-effectiveness threshold of up to 3 GDP per capita (120,000 reais), which will be considered as one of the criteria for the incorporation of medications for rare diseases in the public health system. Although the HTA agency has been exhaustively reaffirmed that this is just one of the criteria and not what will define whether a medication will be made available by the health system or not, this topic is causing deep concern in the community of people with rare diseases, who fear not being able to access medicines that are already being used by patients all over the world, and saving lives of those who no longer had hope”.

One participant at the event said: "A very well organized event, with excellent technical quality from the speakers. Very appropriate topics and very clarifying questions. Congratulations to the organizers and may the next one come!"

Verônica Stasiak Bednarczuk de Oliveira: Master's student in Pharmaceutical Sciences with emphasis on health technology assessment at the Federal University of Paraná; MBA in Public Policy and Social Rights; founder and executive director of Unidos pela Vida - Brazilian Institute for Cystic Fibrosis Care; member of the Brazilian Cystic Fibrosis Study Group, Member of ISPOR. She was diagnosed with Cystic Fibrosis, a rare disease, at the age of 23. https://orcid.org/my-orcid?orcid=0000-0002-9528-9374

Marise Basso Amaral: General Director of Unidos pela Vida, Professor at the Fluminense Federal University, postdoctoral fellow at Fiocruz, Doctor and Master in Education. Mother of a boy with Cystic Fibrosis.

Gabriel da Luz Johnson: Journalist (PUCRS) and Marketologist (UNESA), has an MBA in Digital Marketing (UNESA) and Project Management (Universidade Positivo). Patient advocate for seven years, he is currently Coordinator of Projects and Institutional Relations at Instituto Unidos pela Vida and works defending the rights of patients with cystic fibrosis and rare diseases.

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