Walking the talk in digital transformation of regulatory review.

Cloud-based regulatory platforms have the potential to substantially transform how regulatory submissions are developed, transmitted, and reviewed across the full life cycle of drug development. The benefits of cloud-based submission and review include accelerating critical therapies to patients in need globally and efficiency gains for both drug developers and regulators. The key challenge is turning the theoretical promise of cloud-based regulatory platforms into reality to further the application of technology in the regulatory processes. In this publication we outline regulatory policy journeys needed to effect the changes in the external environment that would allow for use of a cloud-based technology, discuss the prerequisites to successfully navigate the policy journeys, and elaborate on future possibilities when adoption of cloud-based regulatory technologies is achieved.

Medicinal Product Development and Regulatory Agilities Implemented During the Early Phases of the COVID-19 Pandemic: Experiences and Implications for the Future-An Industry View.

The COVID-19 pandemic caused considerable disruption to the development, regulatory evaluation, production, and distribution of medicinal products. Key healthcare stakeholders were under pressure to develop and review medicinal products to address the health emergency, while preserving the continuity of activities to ensure patient access to other medicinal products. In the light of this challenging situation, the National Regulatory Authorities (NRAs) and the biopharmaceutical industry applied and utilized product development and regulatory agilities to accelerate the development and authorization of safe, effective and quality COVID-19 vaccines and treatments as well as other non-COVID-19 medicinal products. On the basis of the literature review and primary research conducted, this review article gathered insights on experiences and challenges in the use of agilities related to regulatory assessment of initial marketing and post-approval change (PAC) applications, oversight of product manufacturing quality and supply chain continuity, and product development/clinical trial processes during the early phases of the COVID-19 pandemic. Agilities were thus implemented in an emergency context characterized by the lack of medicinal products to help tackle a disease that was devastating for the global public health. This review article concludes that useful lessons can be learned from these insights to improve product development practices and regulatory processes during both normal and health emergency times. Standard regulatory frameworks during normal times can be enhanced by leveraging digitalization, further simplifying and harmonizing requirements, and using reliance mechanisms which can help to increase efficiency in regulatory decision-making regarding medicinal products. During health emergencies, such as a pandemic, maximizing global coordination, collaboration, reliance, and harmonization of regulatory requirements and guidance are important to facilitate the rapid development and assessment of key medicinal products to address the health emergency.

Analysis of FDA's Accelerated Approval Program Performance December 1992-December 2021.

The accelerated approval pathway has been criticized recently for employing lower regulatory standards than traditional drug approval, undue delays in withdrawing approvals of drugs for which studies have not confirmed clinical benefit, and confirmatory trials not being pursued with due diligence. This article examines the status of confirmatory studies of drugs approved under the US Food and Drug Administration's (FDA's) accelerated approval program between December 1992 and December 2021. It includes background on the program and provides broader context about the program's performance to date over its 30-year history. Our analysis demonstrates that the accelerated approval program has been largely successful, with half of accelerated approvals converted to traditional approval in a median time of 3.2 years. Furthermore, recent FDA actions show that the agency is appropriately managing the program when a drug approved under accelerated approval fails to confirm a clinical benefit. Any proposed changes to the program should be based on cumulative experience with the program, rather than outliers.