Exploration of FDA's New Drug Rapid Review Path

“The high proportion of biologic agents that have been certified for breakthrough therapeutic qualifications reflects the trend that monoclonal antibodies have become the mainstream R&D drugs for large pharmaceutical companies, and biological agents are often able to obtain higher pricing and have greater potential than small molecule drugs”.

After many years of development, Chinese pharmaceutical companies have started to recommence and have gone overseas. They have learned more about the conditions and methods for priority review of new foreign drugs, which is conducive to the faster integration of local companies into the international market.

According to the US Food and Drug Administration, the evaluation agency and R&D company have disclosed that the approval rate for the first round of new drug approvals in the United States in 2013 was 60%. One of the reasons for the higher first-pass rate was that the new drug met the unmet clinical needs; the second was the closer and more effective communication between the review agency and the R&D enterprise during the drug development process.

It is well known that the FDA has several rapid review procedures for promoting and accelerating the development and review of new drugs. Accelerating the plan will help conclusions for the treatment of serious diseases as quickly as possible to confirm the efficacy and risks of the therapy. Currently, these programs include four types: Fast Track, Breakthrough, Priority Review, and Accelerated Approval. In the next article, the author will explain each of these procedures in detail.

Breakthrough Therapy Qualifications The purpose of Breakthrough Therapy Designation (BTD) is to accelerate the development and approval of drugs that can treat serious or life-threatening conditions. At the request of the applicant, the FDA may determine that a drug has a breakthrough therapy for priority review.

For example, Gazyva, the first drug approved after the breakthrough therapy qualification, is another cancer drug that Genentech has been approved by the FDA in the past three years. It is said that R & D companies have spent 20 years to develop this drug for the treatment of blood cancer, and will continue to conduct studies to assess its efficacy in other types of blood cancer. Sofosbuvir (trade name: Sovaldi), approved in 2013, is also approved as a breakthrough therapy qualification drug for the treatment of hepatitis C virus infection. In the treatment of chronic hepatitis C for specific genotypes, it is also known as "oral IFN." The market has huge demand for Sovaldi and other drugs for the treatment of hepatitis C, and sales are expected to grow rapidly.

Decryption Fast Track Groundbreaking Therapeutic Qualification encompasses all the fast-track program features. Qualified new drug development can be more closely guided, including senior FDA officials. At the same time FDA requires:

During the entire drug development process, the review team maintains communication with the applicants; provides timely advice and interacts with the applicants in the development of the drug to ensure that the R&D program and the non-clinical requirements for the collected approvals And clinical data are valid and feasible; take measures to ensure that clinical trials are designed to be effective, scientific, and appropriate (for example, to reduce the number of patients who are in a potentially ineffective treatment process).

To facilitate the review of R&D programs, the FDA review team will also arrange interdisciplinary project managers to serve as scientific contacts and internal coordination among the interdisciplinary members of the review team and communicate with the applicants.

Since FDA created a breakthrough treatment qualification in 2012, by early March 2013, FDA had received 155 BTD applications, of which 41 were awarded BTDs and 3 drugs had received marketing authorization. Most of these drugs are small molecules (68%) or orphan drugs (58%), while biopharmaceuticals constitute 32% of breakthrough treatment qualifications, and the relative proportion is higher than their sales distribution.

The author also noticed that the high proportion of biologic agents that have been identified as breakthrough therapeutic qualifications may also reflect some trends: First, monoclonal antibodies have become the mainstream R&D drug for the entire industry and even for large pharmaceutical companies; second, this qualification is one of the The main focus is on cancer-targeted drugs; again, biologics are often targeted at serious diseases and unmet clinical needs; in addition, the qualifications also imply that a new treatment model and participation in competition are less risky, while small molecules Drug R&D and manufacturing companies must face more fierce competition; finally, biologics are often given higher pricing and have greater and greater potential than small molecule drugs.