Good new drug review! State Food and drug administration issues the guiding principles of real world evidence

On January 7, the State Food and Drug Administration issued the guiding principles for real world evidence supporting drug development and review (Trial) (referred to as the "guidelines"), aiming to clarify the definition of real world evidence (RWE) in drug development and regulatory decision-making, guide the collection and applicability evaluation of real world data (RWD), and clarify the position and scope of application of RWE in drug regulatory decision-making To explore the evaluation principles of RWE and provide reference for the industry and regulatory authorities to use RWE to support drug regulatory decision-making The State Food and drug administration has already approved drugs according to RWD According to GBI report, in October 2019, based on RWE approval, the State Food and Drug Administration approved the expansion of indications of the marketed product "Roche's mycophenolate ester", which is used for the induction and maintenance treatment of adult lupus nephritis of type Ⅲ - Ⅴ, becoming the only mycophenolate ester approved for this indication According to the example given in the guide, in October 2018, the State Food and drug administration expanded the indications of bevacizumab from "chemotherapy combined with carboplatin and paclitaxel" to "chemotherapy combined with platinum based drugs (including cisplatin, carboplatin + pemetrexed, gemcitabine, albumin paclitaxel, paclitaxel, vinorelbine and other common drugs)", which is also an attempt supported by RWE In addition, the US FDA broke the management of traditional drug review In April 2019, RWD approved a new indication of Pfizer's ibronce drug for the first time based on the drug's marketing Definition: RWD refers to all kinds of data related to patients' health status and / or diagnosis and treatment and health care collected daily RWD meeting the applicability is likely to produce RWE Data collection and evaluation: RWD can come from health information system, medical insurance system, disease registration system, national drug adverse reaction monitoring sentinel alliance, natural population and special disease queue database, histology related database, death registration database, patient report outcome data, mobile device data and other special data sources The applicability of RWD is mainly evaluated by data correlation and reliability Support drug regulatory decision-making: in the aspect of new drug registration and listing, safety and effectiveness data can be provided through practical clinical trials; in the aspect of drugs already on the market, RWE can be used to increase or modify indications, change dosage, scheme and route of administration, increase new applicable population, add the results of comparative study, increase safety information, etc.; in the aspect of post market re evaluation, RWE can be used Decision adjustment; research and development of traditional Chinese medicine; clinical design, etc This is consistent with the opinions of the regulatory authorities mentioned in the interview with JINGDING pharmaceutical executives by GBI in 2019, which holds that RWD and RWE have development opportunities and potential in ① rare diseases; ② guiding clinical trial design; ③ determining the target beneficiaries; ④ clinical development of traditional Chinese medicine; ⑤ post market evaluation; ⑥ indication revision and logo information change.