Brasília – The Brazilian Forum on Rare and Neglected Diseases, promoted by the Parliamentary Front to Support the ITEC Raras Institute, brought together legislators, experts, patients, and civil society to discuss the challenges in guaranteeing timely access to diagnosis and innovative therapies.

Lawmakers emphasized recent advances such as the Clinical Research Law (Law 14.874/2024) and the expansion of patient representation in Conitec (Law 15.120/2025), but pointed out persistent barriers including delays in technology incorporation, shortage of geneticists, and the reliance on litigation. Ongoing bills such as PL 2583/2020, which establishes the National Health Strategy, and PL 1033/2024, aimed at strengthening medical genetics training, were highlighted as key to advancing the agenda.

Patient testimonies revealed diagnostic delays of years and dependence on judicialization to obtain drugs already approved by Anvisa, underscoring systemic failures and regional inequalities. Experts reinforced that late access worsens health outcomes and increases costs for the SUS, calling for specific clinical protocols, early diagnosis, and greater professional training.

Civil society representatives, including Casa Hunter and Febrararas, defended the creation of an independent public agency to evaluate health technologies, similar to international models such as NICE in the UK, as well as modernizing pricing and financing frameworks to ensure predictability and sustainable access to advanced therapies.

Read more: NK Consultores

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