Breakthrough therapy label has become the wind vane of drug approval: the probability of approval is up to 45%

Author: 1 ° C On November 14, 2019, bukinsa, a global innovative drug of Baiji, was approved by FDA, indicating recurrent / refractory mantle cell lymphama Zebotinib has set many records, fast track, breakthrough therapy, priority review, accelerated approval in three months, and the approval of zebotinib in the United States has opened a new starting point for zebotinib's globalization! For details, see 3-month record: FDA accelerated approval of zebotinib for mantle cell lymphoma There has been a saying in the industry that the development of innovative drugs costs an average of US $1 billion, takes an average of 10 years, and the average success rate is less than 10% However, it is worth noting that in the case of breakthrough therapies in the United States, the probability of drug approval for those with "breakthrough therapies" has soared from < 10% to 45%!!! There is no doubt that the breakthrough therapy label has become the vane of drug approval On November 8, 2019, the drug review center of the State Drug Administration issued a number of weight policies, "work procedures for breakthrough therapeutic drugs" and "work procedures for priority review and approval", which are exciting Among them, "work procedures for breakthrough therapeutic drugs" (hereinafter referred to as "work procedures") is even more concerned The work procedure clearly points out that the overall idea is in line with the international standards, corresponding to the fast track and breakthrough therapy of foreign fast channel For the drugs with obvious clinical advantages, during the development process of drug clinical trials, technical guidance, whole process communication and other support policies are given to accelerate the drug development and marketing process The author will start with the analysis of the drugs recognized as breakthrough therapies in the United States, and briefly analyze the next Chinese version of breakthrough therapies According to the U.S FDA's break through therapy approvals [1], friends of cancer research and the author's usual accumulation, first of all, it summarizes and summarizes 336 pieces of breakthrough therapy identification information issued by FDA, of course, there must be some omissions for reference only As shown in the figure above: 1 In general, of the 336 approved breakthrough therapies, 150 have been approved for listing, with a proportion of 45%; 2 At the same time, it should be noted that there is a period of time when a certain indication of a drug is recognized as a breakthrough therapy and the indication is approved for marketing Therefore, in fact, the probability of approval for marketing of drugs recognized as breakthrough therapy is far more than 45% From 2012 to 2019, the probability is 50%, 72%, 74%, 55%, 56%, 51%, 25%, 4.6% respectively; 3 Breakthrough therapy is the touchstone of innovative drugs, which can increase the approval probability of drugs to at least 50%!!! Statistics show that in 2019, the FDA issued 47 breakthrough therapies, of which, 2 indications have been approved for listing, and multiple indications have submitted listing applications! At present, in terms of the content of the work procedure for breakthrough drugs (Draft for comments), the following points should be paid attention to in the Chinese version of breakthrough drugs: 1 Background: encourage innovation and meet the clinical urgent need, and revise the measures for the administration of drug registration; 2 General idea: fast track + breakthrough therapy; 3 Scope: it is used for the prevention and treatment of diseases seriously endangering life or seriously affecting the quality of life, or the first effective treatment scheme or obvious treatment advantage scheme; 4 Support policy: strengthen guidance + promote drug research and development process + give priority to relevant communication It is clear that the National Bureau will support and accelerate the breakthrough and innovative drug listing process that is really urgently needed in clinical practice, and release the drug review system from the imprisonment brought by the drug approval and listing process to the maximum extent! There is no doubt that in the future, more and more innovative drugs such as Pd - (L) 1 mAb, zebutinib and etaxel will appear At the same time, with the rapid marketing channels of breakthrough treatment drug procedures, conditional approval procedures, priority review and approval procedures, special approval procedures and other drugs, the Chinese version of breakthrough therapy label will also become the wind vane for drug approval and marketing, and innovative drugs are urgently needed in clinical practice Will be able to market faster and faster, improve accessibility! Reference source: [1] https://