This article compares real-world evidence (RWE) and postmarket surveillance (PMS) data collected outside of a randomized, controlled, clinical trial. Real-world data (RWD) include individual patient data points such as complaints and anecdotal findings. The recent explosion in RWD collection has led to new challenges in RWD evaluation globally. Solutions are rapidly evolving to analyze these relatively random data points for scientifically valid purposes. This article disc...

The European Medicines Agency (EMA) this week issued a question-and-answer guidance to address sponsors’ questions on its pilot testing the review of raw clinical trial data for marketing authorization applications (MAAs) and post-authorization applications.   EMA issued an updated guidance on the pilot in October. (RELATED: EMA to explore reviewing raw data from clinical trials in applications , Regulatory Focus 12 July 2022)   The purpose of the pilot is to ...

The FDA Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) have issued draft guidance describing sponsors can leverage external controls, such as use data from registries and electronic health records, in lieu of data from randomized controlled trials (RCTs) to demonstrate the safety and effectiveness of new drugs and biologics.   The draft guidance also describes how sponsors can communicate with FDA on using dat...

Welcome to the December issue of RF Quarterly in which global regulatory experts write about the role of artificial intelligence (AI) in regulatory practice, with a focus on global change management of AI-based medical devices, AI in regulatory intelligence knowledge management, synthetic data and the innovation, assessment, and regulation of AI medical devices, and digital transformation.   We thank the authors for their generosity in sharing their knowledge and exper...

Synthetic data are artificial data that mimic the properties of, and relationships in, real data. They show promise for facilitating data access, validation, and benchmarking, addressing missing data and under-sampling, sample boosting, and the creation of control arms in clinical trials. The UK Medicines and Healthcare products Regulatory Agency (MHRA) is using its current research into the development of high-fidelity synthetic data to develop its regulatory position on ...

This article outlines the standardization work on artificial intelligence (AI) being carried out in the joint International Organization for Standardization (ISO) and International Electrotechnical Commission (IEC) committee, ISO/IEC JTC 1/SC 42. 1 The SC 42 committee develops international standards that cover both technology capability and nontechnical requirements, such as business, regulatory and policy requirements, and application domain needs, as well as ethical an...

The European Medicines Agency (EMA) is looking for industry participants for a pilot assessing whether reviewing raw clinical trial data improves its evaluation of marketing authorization applications (MAAs) for new medicines and post-authorization applications.   The pilot , announced on 12 July, stems from a joint workplan announced by EMA and the EU’s Heads of Medicines Agencies (HMA) to address issues related to big data in healthcare and medicines development. On...

This article will examine ways in which the regulatory profession can draw artificial intelligence (AI) to navigate the complexities and fluidity of the global regulatory landscape. Keywords – artificial intelligence, big data, machine learning, SaMD   Introduction Many of the processes within the regulatory landscape entail gathering data, tabulating spreadsheets, collating documents for regulatory submissions, and reviewing dossiers to ensure their quality is c...

The US Food and Drug Administration (FDA) should adopt updated data standards that accommodate the “evolving and emerging” nature of real-world data not currently reflected in the Data Standards Catalogue, according to industry comments on the agency’s draft guidance.   Overall, comments from industry generally supported the principles in FDA’s draft guidance on data standards for drug and biological products submissions containing RWD (RELATED: FDA drafts data sta...

The US Food and Drug Administration (FDA) has issued draft guidance to industry on how real-world data (RWD) could be used in clinical trials and how it could be incorporated into the design of non-interventional studies.   The draft guidance , released Thursday, outlines the applicability of FDA’s investigational new drug application (IND) regulations to clinical study designs that use RWD and clarifies the agency’s expectations on new drug applications (NDAs) or bio...

For drugs and biologics that receive accelerated approval, could information drawn from real-world data sources supplant traditional postapproval clinical trials to confirm that the therapies have benefit? Real-world data (RWD) are not yet robust enough to confirm the benefit of drugs awarded accelerated approval based on surrogate endpoints, answered the authors of a recent study.   “The findings of this cross-sectional study suggest that none of the 50 [Food and Drug...

Feature articles during October included an in-depth look at the linguistic review process in Europe and  reviews of the regulatory pathways for herbal medicinal products, medical devices, and supplements in the EU and for advanced therapies in the Asian-Pacific region. Also included was an article on the challenges of data integrity remediation in cGMP facilities.   Linguistic review and herbal product regulation The linguistic review process for centrally authori...