The promise and challenges of the Unified Study Definition Model (USDM)

The clinical trial industry has long recognized the importance of standardization in driving efficiency and ensuring data quality. Efforts by organizations such as TransCelerate Biopharma and CDISC have laid the groundwork for standardized data models, simplifying aspects of trial design, execution, and analysis. These initiatives have already delivered measurable benefits by improving data interoperability and harmonization across stakeholders. However, the full potential of standardization remains unrealized, as current approaches often fall short in addressing the complexities of trial operations and data integration.

Enter the Unified Study Definition Model (USDM), part of CDISC’s Digital Data Flow (DDF) initiative. The USDM promises to redefine how clinical studies are designed and executed by offering a machine-readable, standardized framework for trial definitions.

On paper, it sounds very promising, leading to more efficiency and higher quality of system design. But while the USDM presents an exciting step forward, its potential benefits and limitations merit closer examination.

The benefits of the USDM

By standardizing clinical trial definitions into a digital, machine-readable format, the USDM offers significant benefits:

• Streamlined study setup: With a standardized structure, systems like EDC can be automatically configured based on the study definition, reducing the time and effort required for manual setup.

• Improved data quality: Automation reduces the risk of errors and inconsistencies, ensuring that study data is reliable and compliant with regulatory standards.

• Enhanced collaboration: A shared data model facilitates better communication and alignment between sponsors, CROs and vendors, reducing misunderstandings and rework.

We anticipate that a protocol defined using the USDM could be directly imported into an EDC system, ensuring that case report forms align perfectly with the study design. Similarly, RTSM systems could automatically generate the dispensing visit schedule and treatment assignment per treatment arm, minimizing manual intervention and reducing risks.

Potential shortfalls and challenges

Despite its promise, we need to keep a realistic head on and think about the potential pitfalls. We want to highlight some challenges to emphasize the need for continued thoughtful implementation and widespread industry collaboration:

1. Impact on setup timelines: While the USDM aims to accelerate study setup, its reliance on an approved protocol could inadvertently delay timelines. Currently, many systems can be partially set up based on a protocol synopsis. If the USDM requires a finalized protocol for automation, sponsors may face longer lead times before setup can begin.

2. Dependency on perfect standardization: The USDM’s success hinges on strict adherence to standards. Any customization or deviation from the standardized framework could negate the benefits of automation, reintroducing inefficiencies and manual workarounds.

3. Protocol changes: Automation could streamline the process of incorporating protocol amendments, but it also introduces risks. Frequent updates to a protocol – which have become common practice over the past few years – could lead to inefficiencies or even protocol deviations if site and country re-approvals are not properly managed for example.

4. Regulatory and regional variability: Different countries and regulatory bodies may have unique requirements that challenge the universal applicability of the USDM. Ensuring global alignment will require significant effort from all stakeholders.

5. Protocols do not cover it all: Clinical protocols are focused on certain aspects of a trial execution, and do not cover everything that is required for eClinical systems to be setup. As an example, inventory management settings are crucial for the design of RTSM, but live outside of the protocol. Those details would ideally need to be standardized and digitized too.

The role of ICH M11 guidelines in supporting USDM

Standardization that is industry-led is unlikely to be widely successful by itself, as there is no strong incentive to adhere to them. However, when it aligns with regulatory guidelines, the chance of a timely and successful implementation is increased. That makes us look at the ICH M11 guidelines as the key to the success of the USDM.

The ICH M11 guidelines establish a standardized template and technical specifications for clinical study protocols. These guidelines, developed by the International Council for Harmonisation, aim to harmonize the format and content of clinical study protocols across regulatory regions. Adherence to the ICH M11 guidelines will drive sponsors to adopt uniform standards for protocol design.

This alignment between the ICH M11 and the USDM is significant. Together, these efforts create a foundation for enhanced standardization across the industry. While the ICH M11 are meant to ensure that protocols follow a globally recognized template, the USDM will translate these standardized protocols into machine-readable formats, enabling repeatable use across digital systems. By pushing sponsors toward consistency in protocol design, the ICH M11 guidelines significantly increase the likelihood of successful USDM implementation, ultimately streamlining trial operations and reducing inefficiencies.

Preparing for the future

Although we are aware of some challenges related to the USDM implementation, some of them listed above, we recognize the huge potential of the data model in delivering efficiencies to sponsors and CROs alike, helping reduce study start-up timelines. We are actively preparing our systems to ensure seamless integration with the model as soon as our clients are ready to implement it, alongside the release of ICH M11 guidelines.

Beyond the strict application of the principles of the USDM to our platform, we are considering how to leverage them to maximize automation and efficiency. We are also investing in strategies to mitigate potential challenges, thinking ahead into how our systems could benefit from the standards, whilst remaining adaptable. For example:

• Flexible setup processes: We anticipate that the transition to the USDM will be a stepped approach, requiring interim mitigations. Our platform will accommodate traditional and automated setups, ensuring flexibility moving forward.

• Change management: We are planning on implementing safeguards to handle protocol amendments efficiently while minimizing risks of non-compliance.

• Customization control: By emphasizing adherence to standardized frameworks, we aim to reduce the need for bespoke configurations that could undermine automation.

We are confident that the USDM will have a positive impact for our industry. Sponsors and CROs alike ask for increased efficiency in study start-up and protocol amendments, which can be delivered by the combination of the USDM and the ICH M11 guidelines. We look forward to implementing them to our platform!