Efficacy, safety and regulatory aspects of orphan drugs for rare diseases: Zolgensma®’s case study

• Autor(a) principal: Riera, Rachel
• Data de Publicação: 2019
• Outros Autores: Bagattini, Ângela Maria, Pachito, Daniela
• Tipo de documento: Artigo
• Idioma: por
• Título da fonte: Cadernos Ibero-Americanos de Direito Sanitário (Online)
• Texto Completo: https://www.cadernos.prodisa.fiocruz.br/index.php/cadernos/article/view/538
• topic: Judicialização da saúde. Atrofia muscular espinal. Doenças raras. Terapia genética. Doenças neuromusculares.; Health's judicialization. Muscular atrophy spinal. Rare diseases. Gene therapy. Neuromuscular diseases.; Judicialización de la salud. Atrofia muscular espinal. Enfermedades raras. Terapia genética. Enfermedades neuromusculares.

Objective: to discuss the regulatory process of drugs for rare diseases in Brazil, based on the Zolgensma® case, and to critically evaluate the evidence so far available on the efficacy and safety of Zolgensma® for treating spinal muscular atrophy (SMA). Methods: descriptive study conducted at the Health Technology Assessment Center of the Sírio Libanês Hospital (NATS-HSL) in June 2019. Results: in April 2019, the use of Zolgensma® for SMA was regulated in the United States based on two open-lable (unmasked), non-comparative (and therefore non-randomized) studies. These methodological limitations increase the uncertainty related to study results. The National Agency of Sanitary Surveillance (Anvisa) has approved strategies to regulate the process and specific deadlines for completing the appraisal process of new medicines for rare diseases, such as Zolgensma®. Conclusion: it is necessary to broaden the debate about the process of regulation and incorporation of orphan drugs for rare diseases in Brazil. This debate should include evidence related to the effects (benefits and risks) of these drugs, and greater transparency of the criteria indispensable for granting registration and recommendation of incorporation into health systems.