Purpose Many regulators offered new ways of working to help combatting the COVID-19 pandemic and the rolling review procedure is one important and successful example. In rolling reviews, data are submitted and reviewed as they become available before the full data package is available. This approach is resource intensive but faster than standard review processes and therefor of benefit to society and patient during a health emergency. In this study we analyze the European Medicines Agency (EMA) rolling review process and extract learnings, based on the vaccines and treatments that have been approved to date (November 2021), and formulate three suggestions to be carried forward. Method Data and information on rolling reviews and similar related processes were collected from Health Authority websites across the globe with a focus on the EMA. Literature searches in PubMed® and checking company websites for additional information were conducted to complement and corroborate findings as required. Findings The duration of a rolling review cycle and the number of cycles before a conditional marketing authorization (CMA) differs between different applications. Through the rolling review process, COVID-19 vaccines could be approved in record times ranging from 17 to 36 days. The rolling review process is not limited to vaccines but is applied to promising treatments as well. Implications Our paper demonstrates that rolling reviews could be successfully conducted during a health emergency to meet an unmet medical need, namely the COVID-19 pandemic. Other critical conditions or life-threatening diseases with unmet needs exist and may be suitable to be addressed by a rolling review process to accelerate patient access to life-changing treatments. Indeed, we call for an evaluation of the rolling review process, its use, and efficiency, to capture learnings with the aim to build a novel, lean, and effective expedited review procedure that could be institutionalized and added to the regulatory toolbox.

Purpose: This article provides an analysis of the various regulatory decisions available in the public domain that suggest the use of real-world data (RWD) for postmarketing surveillance activities of products that have a marketing authorization approved by the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA). The study focuses on the cases in which RWD was used for postapproval commitments or requirements (and to a lesser extent label extensions, as this has been previously published) of medicinal products comprising small molecules and biologics to support efficacy claims or confirm an acceptable safety profile.Methods: Clarivate Analytics was commissioned to collect data from cases in which RWD was used in the postapproval settings submitted to the EMA (data were found covering the last 14 years) and the FDA (data were found spanning 23 previous years). The query resulted in 165 cases in which regulatory approval was associated with RWD. The data were then categorized and expanded with supporting information gathered from public databases and company websites.Findings: The use of RWD to support regulatory decision-making in the postmarketing surveillance setting has increased in recent years. Most postmarketing surveillance activities are legally enforced requests on the marketing authorization holder to further document the product's safety profile. Data drawn from registries tend to be the most common source in this regard.Implications: RWD have increasingly been used in recent years, both for new product approvals and line extensions and, as shown in this study, in the postapproval setting. There is now a growing appreciation of the potential of RWD as a source in its own right to support regulatory decisionmaking on the benefits and risks associated with clinical interventions.

Purpose The COVID-19 pandemic has impacted the management and operation of regulatory agencies and the pharmaceutical industry around the world. It has prompted regulatory authorities to consider new ways of working and introduced, amongst others, remote inspections to validate the integrity of the regulatory data submitted by companies; evaluate the quality of production and manufacturing sites, and to ensure the conformity with Good Regulatory Practices with the overall goal of guaranteeing patient safety during the crisis. Method We summarize and discuss remote inspection guidelines and other related information made available by the Therapeutic Goods Administration (Australia), the European Medicines Agency (EMA), the Pharmaceutical and Medical Devices Agency (Japan), the Medicines and Healthcare Products Regulatory Agency (UK), and the US Food and Drug Administration (US FDA). We also analyse the impact of the pandemic on inspections conducted by the inspectorates of the EMA and the US FDA. Findings The regulatory authorities that we studied all recognized the importance of implementing regulatory policies on remote inspections in response to the COVID-19 pandemic. The remote inspection guidelines from the five selected regulatory authorities aimed at mitigating the impact of pandemic but, while providing valuable advice to the pharma companies and being similar in intent, were not always aligned in terms of approach and solutions. Implications On-site inspections are likely to continue to be the norm and preferred standard for the foreseeable future. But the health authorities will need to further adopt a risk-based inspection approach and stimulate the increased uptake of inspection reliance as proposed by the Pharmaceutical Inspection Convention and Pharmaceutical Inspection Co-operation Scheme not to overwhelm the pharma companies with repeat and redundant inspections. Remote inspections have proven to be a novel inspection tool, but it is important for health authorities to align on their approach on remote inspections in terms of methods applied and documentation requested.