CDE Endorses Decentralized Clinical Trials: How DCT Is Transforming Drug Development

Prior to 2015, China’s life sciences informatics services remained in a prolonged downturn. It was not until 2015 that the industry experienced a sudden surge. The catalyst was the announcement issued by the former China Food and Drug Administration (CFDA) on July 22, 2015, regarding self-inspection of clinical trial data, which sent shockwaves throughout the pharmaceutical sector. This development brought the standardization of clinical trials, as well as the authenticity and traceability of data, to the forefront of industry attention. Consequently, digital management of clinical trials, previously overlooked, finally found a breakthrough for growth.

Since then, continuous reforms to the clinical review and approval system, along with the ongoing introduction of relevant policies, have driven steady improvements in the quality of clinical trials in China. In recent years, China’s clinical trial industry has undergone transformative change, emerging stronger through disruption.

In July 2020, the National Medical Products Administration (NMPA) and the National Health Commission (NHC) jointly issued the revised Good Clinical Practice (GCP) for Drug Clinical Trials (hereinafter referred to as the “GCP”), which officially came into effect. With fundamental industry standards now in place, what direction will the clinical trial digitalization sector take moving forward?

Decentralized Clinical Trials and Intelligent Clinical TrialsRecognized as the industry’s prevailing trend, it is poised to become the new normal.

Remote intelligent clinical trials, also known as virtual trials, hybrid trials, or decentralized clinical trials, are an emerging approach to conducting clinical trials.

The immediate catalyst for the surge of interest in decentralized clinical trials (DCTs) was the disruption to traditional trial operations caused by the COVID-19 pandemic, which necessitated the adoption of remote trial models. Fundamentally, however, DCTs have gained widespread recognition as a novel paradigm that accelerates drug approval timelines and enhances data quality, delivering greater benefits to patients, sponsors, and contract research organizations (CROs). Consequently, they are poised to become a major trend in the future of clinical development.

The draft for comments on the “Guiding Principles for Clinical Development of Antineoplastic Drugs with a Focus on Clinical Value,” released by the Center for Drug Evaluation (CDE) on July 2, also explicitly recommends that design elements of Decentralized Clinical Trials (DCTs) may be considered in the design of clinical trials for antineoplastic drugs.

The draft for public comment proposes leveraging “virtual” tools to conduct telemedicine follow-ups and monitoring, using wearable devices to collect data. It encourages applicants to explore remote visits, remote laboratory assessments, remote imaging assessments, and remote monitoring, as well as to investigate new follow-up models that combine research center-based and home-based follow-ups.

Why Has DCT Earned Strong Endorsement from the CDE? How Are Data Accuracy and Integrity Ensured in Decentralized Clinical Trials? VCBeat Interviewed Medidata, a Global Leader in Life Sciences Informatics Solutions.

Medidata is the world’s first company to offer a fully remote and intelligent solution for the entire clinical trial lifecycle, having previously facilitated the market launch of Moderna’s COVID-19 vaccine through its decentralized clinical trial approach.

Remote clinical trials can significantly reduce the burden on patients participating in clinical studies. Patients can engage in trials from home by remotely enrolling, completing informed consent questionnaires online, submitting patient-reported data via data collection tools, wearable devices, and sensors, attending visits through video conferencing, and regularly discussing adverse events with investigators.

Low participation rates in clinical trials have previously led to sluggish progress in many studies. How to minimize patient burden and optimize the clinical trial experience has long been a persistent challenge within the industry.

In China, patient participation in clinical trials has consistently remained low. The "Report on the Epidemiology of Malignant Tumors in China" indicates that in 2015, only 0.01% of patients with malignant tumors participated in clinical trials. In the United States, merely 5% of cancer patients enrolled in clinical trials.Remote clinical trials ensure study stability by reducing the burden of patient participation.

The “Guiding Principles for the Clinical Development of Antineoplastic Drugs Oriented Toward Clinical Value” also affirm that decentralized clinical trials (DCTs) help minimize barriers to participant engagement, thereby providing opportunities for participation in clinical trials to population groups that have been most underrepresented in traditional trials.The generalizability and representativeness of clinical trial results across diverse populations will be enhanced.

For clinical trial sponsors, the enhanced patient engagement brought by remote intelligent clinical trials can accelerate product launch timelines and reduce drug development costs.

Taking Moderna’s vaccine launch experience as an example, Medidata’s solutions helped Moderna enroll 30,000 patients in its mRNA vaccine clinical trials with zero on-site visits to trial centers, completing the largest remote smart clinical trial to date.

The role of decentralized clinical trials in accelerating drug market approval has been validated by multiple clinical trials.

Medidata shared the following story with VCBeat: In March 2020, TissueTech, a leader in the field of regenerative medicine in the United States, planned to initiate a pivotal Phase III clinical trial for the treatment of severe diabetic foot ulcers. The COVID-19 pandemic rendered traditional clinical trials unfeasible. However, given that diabetic foot ulcers carry high risks of amputation and mortality, significantly postponing the clinical trial was not a viable option.

TissueTech partnered with Medidata to transition toward remote intelligent trials, enabling remote patient visits, remote acquisition and review of medical imaging, and a shift to remote monitoring. The clinical trial was launched within three months.

How Does Medidata Ensure Data Integrity and Authenticity in Decentralized Clinical Trials?

Currently, the key concerns of global health regulatory authorities regarding intelligent remote clinical trials are how to ensure data quality and integrity, as well as protect participant privacy.

To achieve comprehensive digital monitoring and ensure data standardization, authenticity, and traceability, Medidata told VCBeat that its solution incorporates a full suite of virtual technologies, supporting remote patient engagement and trial quality monitoring.

In remote intelligent clinical trials, patients can use applications to record subjective experiences and wear sensors to collect precise data in real time. They can view the data generation trail, ensuring that the data recorded in documents is complete, authentic, and traceable. This eliminates the reliance on untraceable information sources that often characterized clinical trial data in the past.

Multidimensional data collection ensures the quality of source data at the patient level. In terms of data management, Medidata has introduced a centralized monitoring solution. Centralized monitoring enables sponsors and CROs to continuously monitor and analyze trial data anytime and anywhere. Digital monitoring allows for continuous remote oversight of clinical trials, empowering sponsors and CROs to innovate and optimize trial designs.

In recent years, China’s clinical trial industry has experienced rapid development, evolving from nonexistence to establishment and from scarcity to abundance, achieving a leap from quantity to quality. Looking ahead, in addition to decentralization, intelligence represents another major direction for the development of the clinical trial industry.

Intelligent clinical trials encompass all aspects of the trial process. The application of intelligent technologies across various stages can enhance clinical trial efficiency and accelerate product time-to-market.

For patients, this will reduce their burden across all aspects—including patient recruitment, data collection, and patient management—thereby enhancing clinical trial efficiency and ushering in an era of patient-centric clinical trials.

Taking the challenges of clinical trials for rare diseases as an example, patient recruitment difficulties, patient retention, insufficient data, and the alignment of clinical objectives with trial operations all pose significant challenges in clinical trials involving patients with rare diseases. Intelligent technologies can help solve previously insurmountable problems.

Medidata previously collaborated with the Castleman Disease Collaborative Network. By leveraging machine learning algorithms, Medidata Acorn AI identified biomarkers for Castleman disease that were previously difficult to detect. This AI technology significantly shortened the planned research timeline, enabling personalized precision therapy for Castleman disease and other rare disorders.

For sponsors, the greatest benefit of intelligence lies in improving efficiency and reducing costs.The complexity of clinical trials is continually increasing, making the time and cost required to bring new drugs to market increasingly burdensome, with heavy workloads and tight deadlines. The rapid market launch of pharmaceutical products largely depends on the collection and sharing of complete, timely, and high-quality data.

The advantage of an intelligent clinical trial system lies in its ability to capture data directly at the point of generation, without increasing operational complexity, enabling rapid upload to all integrated systems with a single data acquisition.

Medidata stated that a major pharmaceutical company leveraged data from Acorn AI and insights from Intelligent Trials to significantly accelerate a pivotal study that was severely lagging in enrollment.

In the high-barrier industry of life sciences digitalization, few companies are capable of providing reliable, integrated solutions. As a pioneer in the field, Medidata has consistently led the development of clinical trial digitalization, liberating clinical trials from outdated manual processes, continuously breaking through industry bottlenecks, and turning the seemingly impossible into actionable standards. Today, Medidata is bringing this globally recognized integrated solution to China, working together with Chinese sponsors, regulatory authorities, research centers, and CROs to promote the improvement of domestic clinical trial quality.