Digital Innovation in Medicinal Product Regulatory Submission, Review, and Approvals to Create a Dynamic Regulatory Ecosystem—Are We Ready for a Revolution?

Abstract

The pace of scientific progress over the past several decades within the biological, drug development, and the digital realm has been remarkable. The’omics revolution has enabled a better understanding of the biological basis of disease, unlocking the possibility of new products such as gene and cell therapies which offer novel patient centric solutions. Innovative approaches to clinical trial designs promise greater efficiency, and in recent years, scientific collaborations, and consortia have been developing novel approaches to leverage new sources of evidence such as real-world data, patient experience data, and biomarker data. Alongside this there have been great strides in digital innovation. Cloud computing has become mainstream and the internet of things and blockchain technology have become a reality. These examples of transformation stand in sharp contrast to the current inefficient approach for regulatory submission, review, and approval of medicinal products. This process has not fundamentally changed since the beginning of medicine regulation in the late 1960s. Fortunately, progressive initiatives are emerging that will enrich and streamline regulatory decision making and deliver patient centric therapies, if they are successful in transforming the current transactional construct and harnessing scientific and technological advances. Such a radical transformation will not be simple for both regulatory authorities and company sponsors, nor will progress be linear. We examine the shortcomings of the current system with its entrenched and variable business processes, offer examples of progress as catalysts for change, and make the case for a new cloud based model. To optimize navigation toward this reality we identify implications and regulatory design questions which must be addressed. We conclude that a new model is possible and is slowly emerging through cumulative change initiatives that question, challenge, and redesign best practices, roles, and responsibilities, and that this must be combined with adaptation of behaviors and acquisition of new skills.

Future directions in regulatory affairs

“…Structured content and data management systems have the potential to further streamline data handling and the authoring and publication of regulatory documents. To date, companies have been developing bespoke in-house structured content management systems, particularly for clinical data, but common methodologies will be required across companies and regulatory authorities to enable rapid exchange of data and documents (29,37,38). Continued evolution of initiatives such as Transcelerate's Common Protocol Template, and the FDA's Knowledge-Aided Assessment and Structured Application (KASA), standardizing product quality/chemistrymanufacturing-controls (CMC) data and widespread adoption 10.3389/fmed.2022.1082384 of the ICH Q12: Technical and Regulatory Considerations for Pharmaceutical Product Lifecycle Management guideline will facilitate the move toward use of more structured formats for both clinical and CMC data packages (37,38).…”

“…It will allow real-time data submission to regulatory agencies via cloud-based systems such as Accumulus Synergy ( 28 ). The Accumulus Synergy platform is designed to hold company-specific spaces where companies can work on their data packages, health authority specific spaces where NRAs can work together on their reviews of product data and share their analyses of these data, and spaces where sponsors and regulators can communicate, all protected by data privacy and cyber-security ( 28 , 29 ).…”

“…This relies on an increasing trend toward structured data formats, which will enable real-time exchange of data with national regulatory authorities via cloud-based platforms such as Accumulus Synergy. So, in the future, regulatory professionals will contribute increasingly to data flows rather than document flows, requiring regulatory professionals to upskill in digital literacy ( 29 , 36 ).…”

“…Given the major impact of digital transformation, regulatory professionals should focus on their digital literacy skills such as being confident in using dashboards and cloud-based platforms for data visualization, understanding how data are acquired, processed, analyzed and used for predictive purposes, and developing statistical data analysis and data mining skills ( 29 ). Beyond this there is a need to increase competencies in complex reasoning and problem-solving, adaptive thinking, agility, communication and teamwork and leadership and initiative – all elements of what is known as 21st century skills ( 89 , 90 ) ( Figure 2 ).…”

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“…In addition, it may be beneficial to consider the use of advanced data analytics and artificial intelligence to expedite the review of clinical trial data and other information submitted in support of regulatory submissions. 29 , 30 Apart from that, capacity building within regulatory agencies in LMICs is critical to enable these agencies to conduct drug assessments efficiently, thus investments in human resources, training programs and knowledge-sharing initiatives and collaboration with stringent international regulators should be promoted.…”