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.

Regulatory agilities impacting review timelines for Pfizer/BioNTech's BNT162b2 mRNA COVID-19 vaccine: a retrospective study.

The appropriate use of regulatory agilities has the potential to accelerate regulatory review, utilize resources more efficiently and deliver medicines and vaccines more rapidly, all without compromising quality, safety and efficacy. This was clearly demonstrated during the COVID-19 pandemic where regulators and industry rapidly adapted to ensure continued supply of existing critical medicines and review and approve new innovative medicines. In this retrospective study, we analyze the impact of regulatory agilities on the review and approval of Pfizer/BioNTech's BNT162b2 mRNA COVID-19 Vaccine globally using regulatory approval data from 73 country/regional approvals. We report on the critical role of reliance and provide evidence that demonstrates reliance approaches and certain regulatory agilities reduced review times for the COVID-19 vaccine. These findings support the case for more widespread implementation of regulatory agilities and demonstrate the important role of such approaches to improve public health outcomes.

Impact of genetically modified organism requirements on gene therapy development in the EU, Japan, and the US.

Advanced therapies are emerging as an important class of medicinal products; among these, gene therapies are advancing at an exceptional rate. However, one of the major challenges for gene therapies relates to the additional regulatory requirements for genetically modified organisms. In this paper, we provide an overview of the regulatory requirements for genetically modified organisms in the European Union, Japan, and the United States. We share our experience in managing these requirements and their impact on the adeno-associated virus gene therapies that are under development at Pfizer. Specifically, we discuss the relative complexity of the approval process and the impact of risk assessment expectations on the clinical development of genetically modified organisms. We also compare the regulatory processes and timelines of various regions based on our experience with adeno-associated viral vectors. Finally, we propose that genetically modified organisms, for which pathogenicity and replication competency are well controlled, should be regulated solely under medicinal product regulations and be exempt from additional requirements for genetically modified organisms. Even if an exemption is not implemented, it should still be possible to significantly reduce the sponsor and agency burden by simplifying and harmonizing documentation and data requirements as well as timelines for applications for genetically modified organisms.

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

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.

Regulatory considerations in oncologic biosimilar drug development.

Biosimilar monoclonal antibodies are being developed globally for patients with different types of solid tumors and hematologic malignancies. Applications for proposed biosimilar monoclonal antibodies are being submitted to the regulatory authorities around the world and may increase patient access to key treatment options upon approval. An understanding among stakeholders (e.g., physicians, patients and their caregivers, pharmacists, payers) of the approval criteria, as well as the similarities and differences in regulatory pathways involved in biosimilar approval in different countries, as presented in this review, will facilitate identification of high-quality, safe, monoclonal antibodies that have been developed according to strict, biosimilar regulatory standards. Further guidance and resolution of the ongoing discussions on biosimilar labeling, naming, automatic substitution, and indication extrapolation may ensure, in the future, an effective and appropriate use of biosimilar monoclonal antibodies by oncologists and other stakeholders in daily clinical practice.