Programa de Pós-graduação em Ciências Farmacêuticas, Universidade de Brasília, Brasília, Brasil.
Conselho Federal de Farmácia, Brasília, Brasil.
Cad Saude Publica. 2019 May 23;35(5):e00070018. doi: 10.1590/0102-311X00070018.
This study aimed to assess the level of therapeutic innovation of new drugs approved in Brazil over 13 years and whether they met public health needs. Comparative descriptive analysis of therapeutic value assessments performed by the Brazilian Chamber of Drug Market Regulation (CMED) and the French drug bulletin Prescrire for new drugs licensed in Brazil, from January 1st 2004 to December 31st 2016. The extent to which new drugs met public health needs was examined by: checking inclusions into government-funded drug lists and/or clinical guidelines; comparing Anatomical Therapeutic Chemical Classification (ATC) codes and drug indications with the list of conditions contributing the most to the national disease burden; and assessing new medicines aimed to treat neglected diseases. 253 new drugs were approved. Antineoplastics, immunossupressants, antidiabetics and antivirals were the most frequent. Thirty-three (14%) out of 236 drugs assessed by the Brazilian chamber and sixteen (8.2%) out of 195 assessed by the French bulletin Prescrire were considered innovative. Thirty-six drugs (14.2%) were selected for coverage by the Brazilian Unified National Health System (SUS), seven of which were therapeutically innovative, and none were aimed to treat neglected disease. About 1/3 of the drugs approved aimed to treat conditions among the top contributors to Brazil's disease burden. Few therapeutically innovative drugs entered the Brazilian market, from which only a small proportion was approved to be covered by the SUS. Our findings suggest a divergence between public health needs, research & development (R&D) and drug licensing procedures.
本研究旨在评估 13 年来在巴西获得批准的新药的治疗创新水平，以及它们是否满足公共卫生需求。对 2004 年 1 月 1 日至 2016 年 12 月 31 日期间在巴西获得许可的新药进行比较描述性分析，分析方法是对巴西药品市场监管局（CMED）和法国 Prescrire 药品公报进行的治疗价值评估。通过检查新纳入政府资助药品清单和/或临床指南的药品；比较解剖治疗化学分类（ATC）代码和药品适应证与导致国家疾病负担最大的疾病清单；以及评估旨在治疗被忽视疾病的新药，来评估新药是否满足公共卫生需求。共批准了 253 种新药。抗肿瘤药、免疫抑制剂、抗糖尿病药和抗病毒药最为常见。巴西药监局评估的 236 种药物中有 33 种（14%），法国 Prescrire 评估的 195 种药物中有 16 种（8.2%）被认为具有创新性。36 种药物（14.2%）被纳入巴西统一国家卫生系统（SUS）覆盖范围，其中 7 种具有治疗创新性，且没有一种旨在治疗被忽视的疾病。约有 1/3 的批准药物用于治疗巴西疾病负担最大的疾病。少数具有治疗创新性的药物进入巴西市场，其中只有一小部分获得批准纳入 SUS 覆盖范围。我们的研究结果表明，公共卫生需求、研发（R&D）和药品许可程序之间存在分歧。
