Vias alternativas de registro de medicamentos: análise de dados da ANVISA

Abstract

Since the late 1980s and early 1990s, Alternative Drug Registration Regulatory Routes (ADRR) have been regulated by the Reference Regulatory Agencies, with the aim of accelerating the evaluation of new drugs with a positive risk-benefit ratio, usually for serious illnesses, when the disease morbidity is significant or when the disease is potentially fatal or where there is an unmet medical need, causing patients and sponsors to tolerate greater risks, including not fully knowing the drug. ADRRs can increase the level of communication and commitment between the registrant and the regulatory agency, can give a greater role to surrogate drug efficacy outcomes, and shift part of the burden of proof of clinical benefit and of the generation of safety evidence from pre to post registration. For this paper we conducted a descriptive retrospective bibliographic research on the alternative regulatory pathways developed in several countries, mainly in the US, Canadian and European Reference Regulatory Agencies as well as in the Singapore Regulatory Agency. At ANVISA, an analytical study was carried out in the ANVISA database (DATAVISA), in the registration and post-registration submissions of drugs prioritized in 2018, with the purpose of preparing a newsletter with the data obtained from the research. In Brazil two alternative ways of drug registration are regulated: the prioritization of analysis, regulated by CBR (Collegiate Board Resolution) N° 204/2017 and the special procedure for treatment, diagnosis and prevention of rare diseases, regulated by CBR N° 205/2017. Of the 16 registration petitions prioritized by CBR N° 204/2017, which were granted, 7 concern drugs that are object of Productive Development Partnership (PDP), among which are antineoplastic, antipsychotic, anti-inflammatory and recombinant human growth hormone, 7 petitions concern generic unprecedented drugs and 2 petitions concern drugs for neglected, serious debilitating disease or public health emergency, being an antineoplastic and a protein kinase inhibitor. Of the post-registration petitions prioritized by CBR N° 204/2017, which were granted, 8 concern antineoplastic drug applications, among which 6 are characterized for treating serious debilitating diseases, one is a PDP drug and one is a drug with risk of shortage with an impact on Public Health. Among the 11 petitions classified for special procedure for rare diseases by CBR N° 205/2017, which were granted, there are antineoplastic drugs, a respiratory system drug, a replacement enzyme, a Duchenne muscular dystrophy drug, a protein kinase inhibitor and a drug for X-linked hypophosphatemia. The importance of Alternative Registration Routes in patients’ access to life-saving medicines is unquestionable, however proactive action by the three stakeholders is important as the Regulatory Authority works to shorten the time needed for marketing authorization, registrants expedite studies, production and marketing, and patient associations support rapid access to drugs and monitor their availability. This joint effort to make timely medications available that increase life expectancy.