The incorporation of drugs to treat patients with rare diseases. This was the theme of the Comitê Executivo do Fórum Nacional do Poder Judiciário. The meeting happened on June 18th at the Conselho Nacional de Justiça (CNJ) in Brasilia (DF).
Otávio Clark, CEO of Evidências - Kantar Health, , attended the forum together with the advisers Arnaldo Hossepian and Gonzalo Vecina Neto, of the Comitê Executivo Nacional do Fórum da Saúde, Maria José Delgado Fagundes, director of the Associação da Indústria Farmacêutica de Pesquisa (Interfarma) and other experts and health professionals.
The definition of rare disease changes among countries. The European Union defines rare disease as one that affects fewer than 1 individual in 2000 people of the general population. In the United States, a rare disease is defined as that affecting fewer than 200,000 Americans at any given time. According to the World Health Organization (WHO), a disease is considered rare when it affects 1.3 per thousand people. The number of patients for each disease may be low, but when combined, it reaches about 6% to 8% of the world population, equivalent to approximately 420 to 560 million people, according to Interfarma.
In Brazil, is estimated that 13 million people suffer from some rare disease. 75% of these diseases appear in children aged from 0 to 5 years and contribute directly to severe, chronic or fatal cases in the first 18 years of life.
The meeting discussed the market challenges for these drugs in Brazil, since, according to Interfarma, all medicines for rare diseases are researched and produced abroad. Also, the massive tax rates in the country were the highlighted, which contributes directly to the increase in the costs of marketing these products. Thus, patients with rare diseases are often forced to resort to the so-called judicialization to obtain treatments and medicines outside the coverage of the private health insurance and plans and that are not available in the public healthcare system.
Evidências - Kantar Health has been collaborating for the scientific dissemination of rare diseases and recently contributed to the development and the pricing process of the Drug Market Regulation Chamber (CMED) of a targeted drug for a rare disease that will soon be available from National Committee for Health Technology Incorporation (CONITEC).
Besides, Evidências - Kantar Health has already produced a series of six fascicles on rare diseases. The studies address issues such as pathophysiology of the diseases, clinical picture, treatment and regulatory system. In addition to diseases, there are also fascicles discussing a general picture of rare diseases and the need for public policies.
All material is available in our publications tab and can be consulted by clicking here: