Medidata: Transforming Clinical Trials with AI and Cloud Platforms, Trusted by 18 of the Top 25 Global Pharma Companies

“The innovative drug industry is ushering in a golden age of comprehensive policy openness; however, with the global clinical trial success rate declining year by year, the average drug development timeline extending, and R&D costs rising sharply, this is also an era fraught with the greatest challenges.” This statement was made by Dr. Zou Jianjun, Chief Medical Officer of Hengrui Medicine, at the Medidata NEXT Shanghai Annual Conference.

Indeed, an increasing number of companies are flocking to the life sciences R&D sector, and in 2018, China surged to become the world’s second-largest country for pharmaceutical R&D.

However, on the gold rush path of life sciences, one must first traverse the "Valley of Death" known as clinical trials. Research estimates that testing new drugs in patients takes an average of 7.5 years, with costs per drug ranging from $161 million to $2 billion. According to the latest data, in 2018, the overall success rate from Phase I clinical trials to regulatory submission (measured by the percentage of drugs successfully advancing to the next stage of development) declined to 11.4%.

Compared to uncontrollable risks, a significant portion of new drug development failures stem from avoidable risks. Today, big data and AI are transforming the clinical trial process, which is characterized by long cycles, high risks, and substantial investment.

As a leader in the field of clinical trial informatics, Medidata’s solutions are trusted by 18 of the top 25 pharmaceutical companies worldwide. With over 1,300 clients globally, it serves more than 150,000 authenticated users daily.

At the Medidata 2019 NEXT China Annual Conference, VCBeat (WeChat ID: vcbeat) interviewed Jackie Kent, Senior Vice President of Product Management at Medidata; Tom Doyle, Vice President of Data Science; Huang Guiping, Senior Vice President of Operations for Asia Pacific (excluding Japan) at Medidata Solutions; and Xu Hui, General Manager for China. They shared insights on how Medidata helps life sciences companies mitigate risks, enhance efficiency, and create value through its three major solution platforms.

Throughout the multi-year clinical trial process, many stages require data-driven transformation.

Taking data collection in clinical trials as an example, many clinical studies still rely on primitive and outdated methods to collect and verify data, such as transmitting patients’ medical records via fax, manually counting remaining pills in bottles, and relying on patient diaries to assess medication adherence.

In contrast to the slow pace of digital transformation within pharmaceutical companies, regulatory authorities have been imposing increasingly stringent requirements. The 2015 “Announcement of the China Food and Drug Administration on Launching Self-Inspection and Verification of Clinical Trial Data” (Announcement No. 117 of 2015) marked the beginning of rigorous scrutiny of clinical trial data. The announcement sent shockwaves through the industry, and it was also in that year that Medidata entered the Chinese market.

In 2017, China joined the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH), signaling that its clinical trial regulatory framework would align with international standards, thereby imposing higher requirements on clinical trial management. For certain new drugs seeking simultaneous submission in both China and the United States, clinical trial data will be subject to direct scrutiny under FDA regulatory requirements.

As China has become the second-largest country in new drug research and development, the demand for high-quality clinical trial management is set to surge. Data shows that in 2018, both the number of submissions and approvals for new drug clinical trials in China grew by 20%–30%, while the growth rate of approved drugs for market launch reached a breakthrough 40%.

Pharmaceutical companies and CROs cannot reduce loopholes in clinical trials, improve efficiency, and accelerate drug launches amid fierce competition without informatized management of clinical trials. In China, the stringent regulation of drug clinical trials has directly spurred the rise of the clinical trial informatization industry.

What is the most significant barrier in the clinical trial industry? Medidata believes that data is the primary challenge. Medidata aims to address not only issues such as multi-source, complexity, and redundancy of data, but also to enable seamless end-to-end data flow.

Huang Guiping stated, “Both globally and in China, greater emphasis is being placed on data quality in clinical trials. The Medidata platform enables sponsors to seamlessly oversee regulatory compliance from Phase I through Phases II and III. In terms of data quality, the use of Rave EDC (Electronic Data Capture) and CTMS (Clinical Trial Management System) allows sponsors to identify potential issues across the entire clinical trial at an earlier stage.”

In terms of data collection, Medidata offers solutions such as Rave EDC, Rave eCOA, Rave Imaging, Rave RTSM, Rave CTMS, Rave eTMF, and Rave RBM. Data from these diverse sources are integrated into Medidata’s RAVE Clinical Cloud platform. Once sponsors enter data once, the Rave Clinical Cloud platform can synchronize it across multiple solutions.

By optimizing clinical trial operations through the Rave Clinical Cloud platform, Medidata has also reduced the burden of data entry and maintenance. The integration of various end-to-end solutions with its cloud platform has created an operating system for processes in the life sciences sector. In other words, Medidata aims to provide the “Windows” of the life sciences industry.

Jackie Kent, Senior Vice President of Product Management at Medidata Solutions (left)

Tom Doyle, Vice President of Data Science at Medidata Solutions (right)

Tom Doyle, Vice President of Data Science at Medidata, stated, “If sponsors aim to launch new products simultaneously across different markets or even globally, the cleanliness of data, its standardizability, and the ability to rapidly achieve standardized data processing and collection on a global scale are critical. This is also the core strategy of Medidata. By leveraging Medidata’s unified integrated data platform and the MEDS data platform, all data can be consolidated in one place, enabling effective tracking of the timeline for the entire clinical trial implementation.”

In Medidata’s strategy, Rave serves as the cloud-based platform for clinical trial data and is designed to create a seamless collaboration environment. The subsequent Medidata Enterprise Data Store (MEDS) platform functions as a large-scale repository of standardized, cleaned databases.

MEDS is not merely a data repository; customers can also leverage MEDS’s powerful tools to rapidly extract, expand, and utilize vast amounts of data, truly breaking down data silos and enabling large volumes of clinical trial data to flow and generate value.

While improved clinical trial efficiency directly benefits pharmaceutical companies, biotechnology firms, medical device developers, and CROs, the high costs associated with drug development are ultimately passed on to consumers. The healthcare services purchased by consumers not only cover the costs of successfully developed drugs but also bear the burden of failed clinical trials. This is why drug prices remain so high.

Improving clinical trials, reducing drug development costs, and accelerating the market launch of new drugs will ultimately benefit patients.

Enhancing the value of clinical data not only benefits patients through cost savings, but Medidata’s AI also offers a variety of patient-centric solutions. In an interview, Tom Doyle, Vice President of Data Science at Medidata, mentioned that Acorn AI, a subsidiary of Medidata, is building a synthetic control database.

In clinical patient recruitment, according to a Cognizant report on recruitment forecasting, approximately 80% of clinical trials fail to enroll suitable patients within the designated timeframe, and about one-third of Phase III clinical studies are terminated due to difficulties in patient recruitment. Domestically, public information indicates that only 45% of clinical studies conducted in China complete enrollment on schedule. As a result, the market launch of many new drugs will be delayed, postponing patients’ access to novel therapeutic treatments.

Tom Doyle stated, “By leveraging Medidata’s extensive data, we aim to eliminate the need for placebos and control groups in future clinical trials. Instead, we will conduct clinical comparisons of new drugs against real-world data and existing datasets. This approach will benefit many patients with rare diseases, where participant recruitment is typically challenging. Furthermore, it can be applied to data-rich conditions, such as common tumors or cancers. This is truly exciting.”

The database underpinning this synthetic control group is Medidata’s integrated repository of data from more than 17,000 clinical trials. It can analyze 45 billion data points from 2 million clinical trial sponsors, making it the largest structured and standardized database in the industry, with data on more than 4.8 million patients.

In addition to serving as a control group in clinical trials, AI can leverage cloud platforms to generate value by providing data insights to pharmaceutical companies or CROs, thereby facilitating more informed decision-making.

In addition to the currently offered SCA (Synthetic Control Arm) AI-generated synthetic control arm solution, Acorn AI can optimize clinical trial protocol design in multiple ways, providing data insights to sponsors and CROs.

Tom Doyle told VCBeat that by integrating data from diverse sources, Medidata can conduct more targeted and accurate analyses. For instance, it can identify geographic clusters of populations at higher risk for certain diseases and determine which research centers are best positioned to reach these patients, thereby accelerating patient recruitment for clinical trials. Medidata can even forecast trial-related scenarios, such as the workload and potential challenges faced by clinical monitors and physicians in patient recruitment, while enabling global tracking of subsequent trial progress.

Currently, Acorn AI is primarily utilized in four key areas. First, AI accelerates drug discovery; Medidata leverages the use and identification of AI-driven biomarkers to expedite this process. Second, it accelerates the clinical trial process itself. Third, it integrates medical data and medical evidence, such as site selection and research findings from Key Opinion Leaders (KOLs). Fourth, it involves connected devices, enabling Medidata to collect data outside the clinic. By continuously expanding its ecosystem, Medidata can intelligently monitor the entire clinical trial process, thereby enhancing patient experience.

Having dedicated two decades to advancing informatics in the life sciences, Medidata serves 18 of the global top 25 pharmaceutical companies. Worldwide, Medidata provides services to more than 1,300 global pharmaceutical companies, biotechnology firms, medical device developers, medical centers, and CROs, including 312 pharmaceutical companies in the Asia-Pacific region.

Jackie Kent, Senior Vice President of Product Management at Medidata, stated, “Across all solutions, Medidata adheres to three core strategies: execution, innovation, and patient-centricity. In terms of execution, our goal is to help sponsors bring their products to market rapidly by creating platforms that enable the free flow of data. Regarding innovation, we leverage AI-driven data insights to assist sponsors in making intelligent decisions and driving innovation. As for patient-centricity, for example, Medidata collects patient information through wearable devices, thereby enhancing patients’ experience in clinical trials.”

As a foreign enterprise, Medidata often raises concerns about whether its solutions are suitable for domestic Chinese companies and whether its three core principles can be effectively implemented in the local market. Xu Hui, General Manager of Medidata China, stated, “Before joining Medidata, I conducted extensive research and reflection, particularly considering whether significant localization would be required to introduce an IT product into the Chinese market. However, after joining Medidata, I gradually realized that this need was not as substantial as I had initially imagined.”

Xu Hui, General Manager of Medidata China

Xu Hui believes that the root cause of the low demand for localization stems from two aspects. The first aspect is that global regulatory control, requirements, and processes for pharmaceutical approvals are highly similar. From a product perspective, it is relatively easy to develop a solution that comprehensively covers all requirements, workflows, functionalities, and data needs. Therefore, when a product becomes mature, the need for customization naturally decreases.

“At the second level, from a product design perspective, we can see that Medidata aims to become the operating system for the life sciences sector. Much like Windows, it addresses the diverse needs of global customers by covering a comprehensive range of requirements; thus, localization needs are also met through this all-encompassing approach. From Medidata’s standpoint, is localization needed? Are local requirements necessary? The answer is yes. However, Medidata’s approach to meeting these needs does not focus on customization per se, but rather on ensuring its product encompasses all functionalities, thereby addressing all requirements—including those mandated by regulatory compliance—through its comprehensive feature set.”

Xu Hui also revealed that the majority of Medidata’s clients in the Chinese market are domestic customers, including local pharmaceutical companies, biotech firms, and startups. Medidata has maintained a very low customer churn rate, retaining 99% of its clients. This year marks Medidata’s fourth year in the Chinese market. Xu Hui stated that Medidata will continue to deepen its presence in China, supporting innovation in the country’s life sciences sector.