Medidata Frontier Insights – Dassault Systèmes Greater China has launched a new column to share cutting-edge insights and industry insights.
This article was written by Yan Xiaoming, Senior Manager of Dassault Systèmes' Greater China Strategic Consulting & Solutions Business Unit.
In the past year, there have been frequent good news about China's innovative drugs "going overseas". Not only have many pharmaceutical companies successfully entered the European and American markets and been approved for listing, but the scale of external licensing cooperation has also reached a new high. The huge share of the overseas cake, combined with the difference in pricing with the domestic market, has an irresistible attraction for domestic innovative pharmaceutical companies.
"Going to sea" is windy and turbulent
Global innovative drug sales are mainly concentrated in developed markets such as the United States, Europe, Japan and South Korea. In 2021, the U.S. accounted for more than half (55%) of the global market for innovative drugs. Direct listing in the U.S. is recognized as the "most difficult path" for global pharmaceutical companies to enter overseas markets. However, once you go overseas, sales may be multiplied. However, in the face of different global regulations and market cultural backgrounds, many innovative drugs have also encountered setbacks when applying for marketing overseas.
Specifically,Conducting multi-center clinical trials around the world is challenging, from the design to the execution of clinical trial protocols. For example, in terms of clinical protocol design, the standard ** protocol varies from country to country, and the same indication has very different unmet needs in China (both domestic and foreign). How to accurately locate the unmet needs of the patient population is also an important factor for the regulatory authorities of various countries to consider the approval of marketing, which is a necessary knowledge point for going overseas and one of the difficulties in going overseasWe are also pleased to see that toripalimab and fruquintinib have found the right track.
The number of participants enrolled in the trial and the different ethnic requirements of the trial were different, and how to locate the sites that performed well was also a challenge for the Chinese sponsor. In addition, during the study, the subjects were fully supported and avoided; After listing, how to quickly adapt to the requirements of different markets has become a monstrous wave before pharmaceutical companies go overseas.
Therefore, it is important to have an international partner with global experience.
Stand on the shoulders of those who came before you and draw a nautical map
Winston Churchill said, "You can see as far into the past as you can see into the future." ”
In the process of helping China's innovative drugs go overseasMedidata has supported all four FDA-approved innovative drugs in China, and continues to support the clinical development of its expanded indications. The Medidata platform supports all 23 high-value innovative drugs licensed by Chinese pharmaceutical companies, that is, transactions of more than $500 million, including almost all hot and innovative targets.
Relying on more than 20 years of clinical trial experience and expertise, Medidata has worked with Chinese pharmaceutical companies to take a good and steady path to "go global" from the R&D team, patients and research centers.
Case study: A Chinese non-small cell lung cancer drug proved to the FDA that it has similar effects in China and abroad for different ethnicities through data accumulated in previous clinical studies during the license-out process.
Case: A biotech company, by analyzing the data of 2,500 patients accumulated in previous CAR-T clinical studies, successfully achieved 80% of severe CRS in high-risk groups.
Example: A biotech company, after completing a phase 1 study, PFS showed positive results. However, the amount of data is limited, and in the absence of a control group, the company generates a synthetic control group based on data from previous clinical studies, and the efficacy is significant based on the data. The results helped the company's stock price exceed 500%** and obtain FDA fast-track review pathway, reducing the number of subjects by 20 in the subsequent Phase 2 study design.
On the way to our destination, data leads the way
Case study: A local biotech company used past clinical operation data to explore global research centers when conducting international multi-center clinical studies. Through data analysis, the company managed to scale the number of potential sites by about 9 times, successfully defining 150+ sites as "outperforming" among the newly discovered sites.
Example: A cancer study at a pharmaceutical company was conducted in a "very dangerous situation" (client). Enrollments at some of these sites lagged far behind expectations. Through Medidata's global clinical research data analysis, the client clearly understood that the center had a large number of eligible subjects, but due to the relationship with the investigator and other reasons, there was no way to enter the study. Combined with data support, the team expanded to new research centers and successfully accelerated the completion of the study in 6 months.
These examples are just a few of the areas where MedidataAI StudyFeasibility excels, i.e., selecting sites that are suitable for conducting the trial based on the characteristics and environment** of the trial, and ensuring that the trial performs well after initiation.
In the turbulent era, Medidata can help you solve the problem of "going to sea" and stabilize the "going to sea" must choose!