Risk management, drug shortages and the EU portal for clinical trials

Feature articles during May focused on risk management and mitigation in dealing with contractors and vendors and included articles on best practices in good vendor management, use of risk management to support outsourcing activities, testing in-house versus outsourcing, and outsourcing in regulatory operations. Also included were in-depth examinations of challenges and opportunities in “bespoke” therapies, a critical appraisal of drug shortages in Germany and an update on the European Union’s planned transition from the EU Clinical Trials Register to the EU portal and database system for the submission of clinical trial information.
 
Outsourcing and risk
Many organizations are drawing on contracted or third-party services to ensure production and delivery of safe, effective products, especially as research and development costs increase and prospects for regulatory approval or market acceptance become more demanding and competitive. In Using risk management to support outsourcing activities, Jessica Schlegel, a specialist in device and digital health, and colleagues outline organizational risks and benefits of working with third-party vendors and how to form partnerships around regulatory affairs functions. They also discuss the different controls available to apply an effective risk management program. The authors argue that, with proper controls in place, risk can be effectively anticipated, managed and mitigated to ensure product quality, safety and efficacy.
 
Should testing be handled in-house or should it be outsourced? In Making the call to test in-house or outsource, strategy specialist Sandi Schaible has written a useful, hands-on guide for making that decision, right down to five important questions to ask yourself when selecting an outsourcing partner. But Schaible suggests you begin by looking at the industry, and, once you understand that context, drill down and look at the regulatory requirements, before carefully evaluating and weighing the advantages and disadvantages of your in-house and outsourcing options.
 
Quality assurance specialist Yatin Ajgaonkar, argues in his article, A guide to good vendor management, that there are no short-cuts to ensuring a risk-free environment when regulatory affairs departments work with vendors. He provides an overview of the vendor-contract giver relationship, then discusses finding an appropriate vendor, performing due diligence, vendor-related risk management, and contract building. He rounds off the article with six invaluable, realistic rules for finding the best possible vendor, gauging performance at regular intervals, meeting regularly, having a diverse team, and not shying away from micromanagement.
 
In Outsourcing in regulatory operations, Jen Multari, a global publishing specialist, meticulously documents and critically assesses a case study based on a project she initiated in 2015 to implement a large-scale, global effort to centralize technical tasks associated with dossier compilation and submission in outsourced global vendor hubs. The purpose of Project BRIDGE was ultimately to deliver improvements and minimize the regulatory compliance risk. The author describes how the project team used the framework of the 8-step Kotter model for leading change and how that helped drive the transition in a controlled, systematic way.
 
Bespoke therapies and drug shortages  
Having access to powerful genetic tools, such as RNAi and CRISPR, has allowed scientists and healthcare and regulatory professionals to forge a new therapeutic frontier in the form of personalized medicines, or gene therapies. In his article, Bespoke therapies – opportunities, challenges, and hope, Shawn Roach, an analytical chemist and regulatory affairs specialist, takes a look at the “second chapter” in personalized narrative, that of bespoke therapies, which are tailored to the specific characteristics, symptoms, and responses of one patient. The author examines the clinical and nonclinical safety perspectives for bespoke therapies in the context of the opportunities and challenges that come with their development. He argues that a recurring theme in this landscape is the importance of open communication and collaboration between the Food and Drug Administration and sponsors, as well as between academic and biotechnology company sponsors.
 
In Drug shortages in Germany ‒ A critical appraisal, quality compliance and regulatory affairs expert Ralf D. Hess and colleagues discuss drug shortages in Germany. The authors assert the root causes of the shortages ‒ production problems and compliance with good manufacturing practice, drug pricing, supply chain disruption, and the tendering and rebate processes ‒ point to a “broken marketplace” in the aftermath of the 2008 financial crisis. The article concludes with a discussion of mitigation activities, and, in an epilog, address the current COVID-19 pandemic with a warning that drugs and active pharmaceutical ingredients from China and India may be in short supply because of pandemic-related disruption.
 
Delays have beset the European Union’s planned transition from the EU Clinical Trials Register to the EU portal and database system (now known as the Clinical Trials Information System) for submitting clinical trial information. The current estimate for its launch is the second half of 2021 ‒ but that was established for the COVID-19 pandemic. In Electronic platforms for submission of clinical trial information, Federico Bonacci, a specialist in clinical pharmacology and forensic medicine, and colleagues present a follow-up to their 2017 report on the impact of the revised clinical trial regulations. In the current article, they give an overview of some of the main international portals, both available and under development, and provide an analysis of their specific aspects, including functionality and user experience, and a comparison of them, where feasible.
 
What’s coming in June?
June articles will focus on a range of topics relating to nutrition in health and disease management, including articles on recent updates on US trade policy issues in the dietary supplement industry, growth opportunities in Latin America for food supplements businesses, application of master files to the new dietary ingredient notification, an overview of class action lawsuits targeting the dietary supplement industry, and global front-of-pack nutrition labeling schemes and their impact on marketing strategies. Look for these topics and more throughout June at www.raps.org.
 
August call for articles
Regulatory Focus will address global clinical trials and clinical trial applications in August. The submission deadline is 1 July. To contribute to July or suggest a topic, contact rmatthews@raps.org.
 
Cite as: Matthews R. Risk management, drug shortages and the EU portal for clinical trials. Regulatory Focus. June 2020. Regulatory Affairs Professionals Society.