Novartis: A Cohesive Platform for Clinical Trial DHT Regulatory Acceptance

In a highly anticipated session at Panagora Pharma’s 7th Digital Biomarkers in Clinical Trials Summit, Mireille Muller, Executive Director of Regulatory Strategy at Novartis, presented the innovative EFPIA DEEP EMA pilot. This pilot aims to streamline the development and regulatory acceptance of digital health tools (DHT), promising to revolutionize the clinical research and regulatory science landscape. Muller’s presentation highlighted the complexities of the current regulatory environment and introduced a collaborative framework designed to enhance efficiency and foster innovation.

The Complex Regulatory Landscape for Clinical Trial DHT

Muller began her presentation by outlining the intricate regulatory environment DHTs must navigate. She emphasized the diversity of regulatory guidance documents and stakeholders and the often incompatible regulations that complicate the approval process. “It’s a complex interface,” Muller noted, highlighting developers and regulators’ challenges. Digital health tools, which can be used for diagnosis, monitoring, or self-management, require adaptable regulations. For instance, a digital tool used for diagnosing a condition might need different regulatory considerations than one used for chronic disease monitoring.

Muller pointed out the lack of consensus on terminology, such as digital biomarkers and clinical outcome assessment measures, further complicating the regulatory landscape. She explained that these terms might seem similar but have different implications depending on the context, leading to various viewpoints on handling them. Moreover, Muller highlighted the fragmented nature of the regulatory environment, where other regions and countries have their own rules and guidelines. This fragmentation can lead to inefficiencies and delays in marketing DHTs.

Bridging Innovation and Regulation in Clinical Trial DHT

Despite the inherent challenges, Muller stressed that innovation and regulation coexist harmoniously. She cited the qualification process as a critical area where innovation meets regulation, enabling the development of validated, standardized measures that collect patient-relevant evidence for regulatory decision-making. This process ensures that new digital health tools are practical and safe for patients. “The ultimate goal is to provide tools that reliably and safely monitor conditions for benefit-risk assessments of drugs,” Muller explained. She emphasized that regulators need robust tools to make informed decisions about the safety and efficacy of new treatments.

However, she criticized the current model, where individual sponsors qualify their tools, as inefficient and resource-intensive. This approach often leads to duplication of efforts and wasted resources for the applicants and regulators. Muller provided an example of how the current model falls short. She described a scenario where multiple companies develop similar digital health tools for monitoring a specific condition, such as nocturnal scratching in patients with eczema. Each company must undergo the qualification process independently, resulting in redundant efforts and increased costs. This inefficiency can slow down the overall progress in the field and limit the availability of innovative patient tools.

The DEEP Platform: A Collaborative Framework

Muller introduced the DEEP platform as a solution to these inefficiencies. The platform is designed to enable the collaborative development of DHT with four key aspects. First, the platform allows for the reuse of elements throughout the lifecycle of digital health tools, enhancing efficiency. For example, once a digital biomarker is validated for a specific condition, it can be reused for other related conditions without undergoing the entire validation process. This reuse can significantly reduce the time and resources required for developing new tools.

Second, it helps define technology changes, allowing for the extension of the use context. Muller explained that digital health tools often undergo technological advancements, such as algorithm updates or hardware improvements. The DEEP platform provides a framework for managing these changes and ensuring the tools comply with regulatory requirements. Third, the platform serves as a reference catalog for developers and researchers. This catalog includes a comprehensive list of validated DHT, their regulatory status, and their use context. Developers can use this catalog to identify existing tools that meet their needs, reducing the need for redundant development efforts.

The EMA Qualification Pilot

The DEEP team conducted an EMA qualification pilot to test the platform’s effectiveness. The pilot focused on optimizing the qualification procedure, testing collaboration, standards, lifecycle management, and knowledge management. The feedback was overwhelmingly positive, with regulators enthusiastic about the ability to reuse structured evidence and extend it to new conditions. “The structured approach increased the quality of evidence submitted for qualification purposes,” Muller reported. She explained that the pilot involved a specific case study on nocturnal scratching, which affects patients with eczema. The DEEP team worked closely with the EMA and other stakeholders to develop a standardized approach for qualifying digital health tools for this condition.

The pilot demonstrated that the DEEP platform could streamline the qualification process, making it more efficient and effective. The pilot also provided valuable user feedback, which will be used to develop the platform further. Muller highlighted several key learnings from the pilot, including the importance of clear communication and stakeholder collaboration. She noted that the structured approach of the DEEP platform helped facilitate more productive discussions and provided a solid foundation for future developments. This feedback highlights the potential for the DEEP platform to bridge regulatory gaps between different regions, fostering a more unified approach to digital health tool development.

Future Prospects and Global Implications

Muller concluded by expressing optimism about the future of the DEEP platform. She believes it has the potential to accelerate the adoption of digital endpoints and position Europe as a leader in regulatory science. “Europe is well-positioned to become the center of regulatory science,” she asserted. Muller emphasized that the success of the DEEP platform could serve as a model for other regions, promoting global harmonization of regulatory processes. During the Q&A session, a question was raised about the alignment between EMA and FDA. Muller acknowledged that this is a phase two objective and will be explored in the future.

A colleague added that the EMA was more pragmatic and open to feedback than the FDA, facilitating more productive discussions. This feedback highlights the potential for the DEEP platform to bridge regulatory gaps between different regions, fostering a more unified approach to digital health tool development. Muller also mentioned that the DEEP platform could support the development of new digital health tools for various conditions, from chronic diseases to rare disorders.

Summary

Mireille Muller’s presentation on the EFPIA DEEP EMA pilot highlighted the potential for collaborative frameworks to transform the regulatory landscape for digital health tools. By enabling the reuse of elements, defining technology changes, and fostering collaboration, the DEEP platform promises to make the regulatory process more efficient and effective. As Europe positions itself as a leader in regulatory science, the DEEP platform could serve as a model for global regulatory innovation. The session ended with Muller thanking her colleagues and expressing hope for the future of regulatory science. The audience left with optimism and anticipation for the advancements the DEEP platform could bring to digital health tools.

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