The Food and Drug Administration’s new regulations point to clinical trials, and innovative pharmaceutical companies face a “clearance crisis”

Since April, the State Food and Drug Administration has issued two guiding principles in succession, arousing the attention of the industry.

On April 18, the CDE official website released a draft for comments on the Technical Guiding Principles for Drug Clinical Dependence Research, which aims to improve the Dependency is further checked. Just 4 days ago, the official website of the State Food and Drug Administration also released the Guidelines for Pharmacovigilance Inspections, a new round of strengthening of domestic pharmacovigilance inspections.

The details of drug safety such as pharmacovigilance and clinical dependence have not been paid enough attention to in the past by the Chinese pharmaceutical industry. In the United States, it was only after major events such as “Purdue Pharma’s new analgesic drug” occurred that it was gradually taken seriously by the FDA, required to strengthen clinical-stage drug efficacy research and post-marketing adverse reaction monitoring.

The Bureau of Health and Knowledge has noticed that although the above two documents point to different points, there is one thing in common: the key observation objects are innovative drugs.

Actually, for innovative drugs, novel targets and mechanisms only mean the starting point of the long march of research and development. Clinical trials and adverse reaction monitoring “long-distance running” are the conscience to verify drug efficacy base color.

FDA has repeatedly rejected Chinese medicines, and China is strengthening supervision

The trend at the regulatory level may be related to the frequent “going overseas” of Chinese innovative pharmaceutical companies.

On the morning of December 2, 2021, Hengrui Medicine, the “first brother” of the domestic innovative drug, issued an announcement that its investment in Dalian Wanchun’s research and development of the innovative drug plinabulin was approved. The FDA rejected it for the following reasons: Insufficient clinical data.

This drug has previously attracted much attention in China, and in the second half of 2020, it has been awarded “Breakthrough Therapy Variety” and “Breakthrough Therapy Designation” in China and the United States. In the two interim analyses of phase III clinical trials, the data of plinablin were also stunning enough. In February 2021, Huang Lan, the founder of Wanchun Pharmaceutical, said bluntly in an interview: “2021 will be the year when plinabulin shows its anti-cancer effect.”

The anti-cancer effect failed to show, but the clinical trial data was stamped with “unqualified”.

Cinda is even more embarrassing. On the evening of February 10, 2022, the FDA Oncology Drug Information Committee held a review meeting to discuss whether Innovent’s PD-1 mAb could be approved for marketing in the United States. The final review meeting rejected the marketing application of Cinda PD-1 by a vote of 14:1, and the problem still lies in the clinical trial. The FDA expert committee pointed out that the clinical trials of Innovent PD-1 did not meet the principle of diversity of the trial population.

FDA is actually not specifically targeting Chinese pharmaceutical companies, but has relatively high requirements for all clinical trials. In January this year, the clinical trial of a CD47 monoclonal antibody under Gilead Sciences was suspended by the FDA because the clinical trial may have unexpected adverse reactions.

Clinical trials are the only way to verify the efficacy of innovative drugs, and investment institutions from all walks of life are eagerly waiting for a good result. Judging from the current trend, the State Food and Drug Administration’s regulatory requirements for drug approval are becoming more and more stringent.

One of the reasons for the tightening of approvals may be the gathering of targets facing the world today. Take PD-1 as an example, when pharmaceutical giants such as Merck, BMS, and Roche have entered the market, and more than 6 models have been launched in China, the latecomers are still vigorous in this type of target. According to the clinicaltrials.gov official website, there are 762 PD-1 product projects that are “recruiting”.

Capital-intensive investment leads to wasted resources; regulatory agencies increase clinical trial requirements, promote survival of the fittest, and maintain an environment for innovation. This is the current game between capital and regulation, both in China and the US.

More than 20 documents were issued in a row, and domestic innovative drugs are under pressure

To a certain extent, FDA action has always been seen as a bellwether. Since June 2017, when it formally joined the International Association for the Registration of Pharmaceuticals for Human Use (ICH), the technical specifications related to the quality, safety and efficacy of Chinese medicines have gradually moved closer to the international ones.

One ​​of the conveniences brought by ICH is the mutual recognition of clinical research data between China and foreign countries. Therefore, joining ICH opened the prelude to the upsurge of domestic innovative drugs. Objectively speaking, China’s drug regulatory system is improving step by step with the vigorous development of innovative drugs.

The “Technical Guiding Principles for Drug Clinical Dependence Research (Draft for Comment)” released on April 18 pointed out that the guiding principles are mainly applicable to innovative drugs and improved drugs with abuse potential developed in my country. type of new drug. Previously “there is no specialized technical requirement to regulate such research in my country”, this is the first time that the technical principles of related research have been clarified in China.

It has only been three months this year, and CDE has successively issued the “Technical Guidelines for Food Impact Research in the Process of New Drug Development”, “The Guidelines for Centralized Monitoring and Statistics of Drug Clinical Trials (Trial)”, More than 20 clinical related documents including “Technical Guidelines for Clinical Pharmacology Research of Biosimilars”.

Whether it is to guide the research and development of corresponding drug categories, or to publicize the technical principles of related supervision work, CDE aims to promote the efficient advancement of drug research and development through guidance and intervention in clinical trials.

In addition to the clinical stage, the supervision of adverse reactions of post-marketing products is also an important way to be responsible for drug efficacy.

On April 15, the State Food and Drug Administration issued the “Guiding Principles for Pharmacovigilance Inspection”, which aims to further implement the main responsibility of listed pharmacovigilance and improve the management of pharmacovigilance. According to the document, drugs with large sales volume or limited alternative drugs, drugs with additional safety conditions when they are approved for marketing, innovative drugs, and improved new drugs are the main categories for pharmacovigilance inspection.

According to this standard, Vaccines, exclusive varieties, various innovative drugs and other drugs that once lived in the “outdoor” will become the key observation objects of monitoring.

Whether it is the strengthening of supervision in the clinical trial stage or the continuous monitoring of post-marketing products, it means that drug research and development will face more, more detailed and longer efficacy tests. For innovative pharmaceutical companies, pharmaceutical companies that are accustomed to rubbing concepts without real skills may face a “clearance crisis”.

Text | Nicotinamide Operation | Twenty-Three Figures|Visual China

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