UBC has been involved in the design, development, or delivery of 82% of the active REMS in the market, with additional REMS in various stages of planning and FDA review. UBC’s partnership value is rooted in our extensive experience with regulators, our collaborative engagement model with clients, and our unwavering support of addressing patient safety while minimizing burden and barriers to access.
A number of developments occurred during 2024 that will drive considerations in the areas of risk management and REMS for 2025 and beyond. UBC is pleased to share these catalysts and how we can support.
Creating your ‘Right-Fit’ Risk Management Strategy During Initial Design
Both the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) and Heads of Medicines Agencies (HMA) released new/updated guidance in 2024 aimed to clarify methods and expectations for the development of meaningful risk management strategies.
- On May 7, 2024, the FDA issued a new draft guidance for the industry entitled “REMS Logic Model: A Framework to Link Program Design With Assessment.” The FDA hosted a webinar on November 7, 2024, to discuss the new draft guidance and the value of using the REMS Logic Model to intentionally approach the 3 phases of a REMS’ life cycle: design, implementation, and evaluation.
The REMS Logic Model offers an approach to explaining in a clear and organized way ‘why a REMS is the way it is.’ It is important that the REMS is designed and implemented in a thoughtful way with the big picture and final outcomes in mind. The more thought and logic put into the development of a REMS, the more successful the REMS will be in meeting its goals and ensuring the benefits of a product outweigh its risks.
Incorporating components of the REMS Logic Model in negotiations with the FDA will demonstrate that you are applying a thoughtful and comprehensive approach to REMS design. It can also improve efficiency in communicating with the FDA during their review of the REMS by offering a structured path for providing rationale and explaining why it designed and implemented a particular REMS element in a certain way.
- On August 6, 2024, the European Medicines Agency (EMA) and Heads of Medicines Agencies (HMA) released the final guidance on Good Pharmacovigilance Practice (GVP) Module XVI Risk Minimization Measures Revision 3 and Module XVI Addendum II – Methods for Evaluating Effectiveness or Risk Minimization Measures. These updates were key concepts discussed further as part of EMA Risk Management Information Day held on November 21, 2024.
GVP Module XVI Revision 3 and Addendum II provides more prescriptive guidance in several areas of risk minimization as compared to Revision 2 released in March 2017. Clarity on specific topics included:
- Distinguishing Risk Minimization Measures (RMM) messages and RMM tools as part of RMM
- Providing guidance on the content for different educational/safety advice tools (e.g., guides for risk minimization for patients or healthcare professionals, patient card)
- Description of different control tools that are separate from the concept of controlled distribution programs which previously caused confusion
- Addition of methods and details of approaches to assess risk minimization effectiveness, including the possibility of qualitative research (Addendum II)
The revised GVP Module XVI also acknowledges that as technology advances, the potential of supporting risk minimisation through digital applications may be considered. However, guidance on digital support to risk minimisation is not provided, but is expected to be the subject of a future EMA reflection paper.
In addition to these releases, in many forums throughout 2024, a renewed focus was expressed to create efficiencies and synergies globally for risk management planning activities. UBC understands that the areas of risk management and REMS are evolving. Our Safety and Risk Consulting Team incorporates the newest risk management approaches and tools to design, plan for, and deploy a “right-fit” strategy.
Ongoing Re-Evaluation of Risk Management Approach
Benefit-risk assessment is a dynamic process throughout the lifecycle of a product. As such, risk management strategies should be re-evaluated and evolve over time.
- In 2024, FDA continued to weigh safe use against burden on the healthcare delivery system as evidenced by continuing to bring REMS-related topics to advisory committee discussion and by approving REMS modifications.
Examples of this include the review of the Clozapine REMS by the Drug Safety and Risk Management Advisory Committee and Psychopharmacologic Drugs Advisory Committee in November 2024, and FDA’s approval of REMS modifications, such as removing REMS requirements for educational and training materials for CAR T cell immunotherapies.
- Further, the updated/new guidance from FDA, EMA, and HMA, provides considerations for initial development of risk management strategies and also identification of updates that may be beneficial post-approval.
UBC understands that continuous improvement plays a crucial role in risk management strategies. Our Safety and Risk Consulting Team partners with clients throughout the lifecycle of their product to evaluate and improve risk management strategies. UBC evaluates existing risk management or REMS strategies using existing data or designs and conducts primary data collection to propose evidence-based recommendations for improvement.
UBCs Continued Focus on Advances in Technology to Support Risk Management
Technology is the backbone of efficient and scalable risk management tactics. In 2024, UBC, FDA and Sponsors continued to look at the burden and barriers to access caused by REMS. By leveraging technology in a meaningful way, sponsors can seamlessly deploy tactics into current workflows, thus making it easier to adopt and improve compliance and, therefore, overall safety. UBC is looking to lead the REMS industry with technological advances:
- Driving an interoperable future for REMS: From January 16-18, 2024, UBC participated in the Health Level Seven (HL7 ®) International Fast Healthcare Interoperability Resources (FHIR ®) Connectathon. With multiple organizations participating, UBC was the only REMS Administrator participating to successfully demonstrate the Risk Evaluation and Mitigation Strategies (REMS) administration software, including the CDS Hooks Services and SMART App Launch as described in scenarios 1 and 2 against an Electronic Health Records (EHR) test system.
Our approach during the Connectathon successfully demonstrated and enabled the prescriber, within their EHR system workflow, to be notified of a REMS and its applicable Elements to Assure Safe Use (ETASU) at the appropriate time – in this use case, when ordering a medication for a REMS covered drug. This offers real promise, real progress, and realized efficiency by leveraging these tools and interoperability standards in a novel way. We are proud to lead the pack and will keep this moving forward in 2025.
Within the just-in-time prompt and in-workflow user interface most, if not all, of the data can be supplied directly from the EHR to the REMS form and the prescriber data entry required currently on REMS websites will be eliminated or severely reduced. “This creates enormous value for a REMS implementation by reducing the burden on prescribers, patients, and, indirectly, pharmacies, as their patients will be more likely to arrive at the pharmacy with the proper ETASU documentation in place,” said Natalie O’Donnell, SVP, Head of Safety & Risk Management. The streamlined process will improve compliance and overall safety.
UBC participated in the 2025 CodeX Connectathon for the REMS Use Case from January 14 through January 16 and performed another successful demonstration not only as a REMS Administrator but as a REMS Intermediary, passing important messages to prescribers within their EHR regardless of the responsible REMS Administrator for the product. With the introduction of the Intermediary solution, EHRs need only register for REMS interoperability once and UBC would act as a messenger between EHR systems and any respective REMS Administrator, further decreasing the burden of participating in prescribing REMS therapies.
- REMS – EHR Integration delivering on the promise of deep operational efficiencies: On October 29, 2024, UBC announced our partnership with Osmind Inc., the leading psychiatric treatment and research platform, to support clinicians and drug developers in delivering innovative treatments to patients with psychiatric conditions. This collaboration brings together UBC’s industry-leading expertise in REMS with Osmind’s purpose-built mental health EHR platform to create a first-of-its-kind EHR integration for REMS using standards from the FDA-supported HL7® CodeX FHIR® Accelerator Community and REMS Integration Use Case.
The partnership is key to advancing better patient care, reducing operational overhead, improving data quality, enhancing REMS process efficiency, and increasing REMS compliance. This initiative also sets the stage for broader EHR-REMS integration in the future, including patient status forms, enrollment forms, diagnostic testing results, and more.
- Increased participation in pharmacy interoperability standards development to advance electronic transmission of REMS data: Throughout 2024, UBC has increased participation with the National Council for Prescription Drug Programs (NCPDP), which is a non-profit organization that develops and promotes standards for electronic exchange and interoperability within healthcare systems.
UBC specifically participates in the NCPDP Standards Coordination work group, focusing on the REMS Workflow to Transaction Task Group where we help define the standards for exchanging REMS data between pharmacy systems and REMS Administrator systems. UBC provides valuable feedback and expertise as a REMS Administrator, guiding the Task Group on data elements necessary to satisfy ETASU from a dispenser perspective while keeping in mind regulatory FDA Assessment Report needs and closing the knowledge gaps between dispensers and REMS Administrators. By participating in these strategic standard-setting Task Group meetings, UBC is well prepared to implement NCPDP-recognized standard interoperability automated solutions for any REMS with a requirement for REMS Dispense Authorization.
UBC continues to leverage our participation in these standards working groups to advance the sector and our implementations. Within our REMS implementation system, we have built a solution using the NCPDP SCRIPT standard to enable pharmacy staff, within their Pharmacy Management System (PMS) workflow, to be notified of a REMS and its applicable ETASU at the appropriate time – in this use case, prior to dispensing a medication for a REMS covered drug. With this interface, data necessary to verify REMS requirements have been met to dispense the product can be supplied directly from the PMS to the REMS, and in turn the REMS will respond with a REMS Dispense Authorization or notification of any missing requirement.
This solution will dramatically reduce the burden on pharmacy staff responsible for dispensing REMS therapies, allowing them to stay within their PMS workflow and eliminating the need to use the REMS website or contact the REMS Coordinating Center for REMS Dispense Authorizations.
Utilizing UBC’s NCPDP SCRIPT compliant Pharmacy Integration Service allows manufacturers and pharmacies alike to adapt to the emerging technology innovation that aligns with industry standards and creates the next generation of REMS interoperability.
UBC is a leading voice and participant in promoting new technology advances (when and where appropriate) to align innovation, operational efficiencies, stakeholder engagement, and regulatory compliance.
UBC is a leader in risk management and REMS activities and can help Sponsors develop, implement, and assess risk management strategies. Our consultation, design, implementation, and evaluation offer a cohesive experience throughout a product’s life cycle. Our subject matter experts (SMEs) remain on the cutting edge of developments across all facets of risk management and can support customers in securing safe and appropriate use of their products.
To learn more about how UBC can help you in 2025 with all aspects of your risk management and risk minimization needs, get in touch with us HERE.
About UBC
UBC is the leading provider of evidence development solutions with expertise in uniting evidence and access. UBC helps biopharma mitigate risk, address product hurdles, and demonstrate safety, efficacy, and value under real-world conditions. Our combined scientific expertise in pharmacovigilance, risk management, and signal detection and assessment, underpinned by innovative technologies, offer our customers customized solutions generating the relevant insights necessary to make informed decisions earlier, meet stakeholder requirements, and ensure the safety of products.
Contributors
Rachel Bonfanti, MPH, is an Executive Director, Risk Management & Scientific Consulting. In this role, she collaborates with UBC safety scientists and operational teams to develop risk management strategies. She is responsible for the creation of additional risk minimization measures (aRMM), REMS, REMS Supporting Documents, REMS tools, and REMS Assessment Reports for both single product and shared system REMS. She supports sponsors in the preparation for and participation in FDA Advisory Committee meetings and other regulatory meetings, supports REMS negotiations, and drafts regulatory communications regarding risk management topics.
Dr. Janine Collins, MBBS, LLM, is Vice President, Medical, Scientific & Real-World Research at UBC. She provides leadership, direction, and guidance supporting customers in the creation of core Risk Management Plans and global and local strategy for Risk Management activities. Her experience spans a wealth of therapeutic areas and latterly has focused on several rare diseases in both adults and pediatrics including metabolic disorders, inborn errors of metabolism, neurodegenerative disorders, and pediatric and adult oncology.
Kate Macke is a Project Manager of Safety, Epidemiology, Registries, and Risk Management at UBC. In this role, she supports sponsors with the creation of REMS Documents for initial submission to the FDA, as well as supporting REMS negotiations and modifications of the REMS Documents throughout the review process.
Michelle Rice is the Principal REMS Technology Product Manager at UBC. She provides guidance and expertise to customers related to REMS technology capabilities and timelines, ensuring that each REMS technology build is set up for success from the beginning. Michelle also provides leadership and oversight to REMS Information Technology Delivery Teams as they build, implement, and maintain UBC’s proprietary and comprehensive REMS systems, bridging communications and negotiations between business units internally and externally with respective technology teams. Michelle has a wide range of experience over 18 years in healthcare technology, seven of those years in REMS. Her career focus has been in business systems analysis, program management, product management and technology innovation in regulated spaces.
References
European Medicines Agency. EMA risk management information day 2024. November 21, 2024. Accessed January 7, 2025. https://www.ema.europa.eu/en/events/ema-risk-management-information-day-2024
European Medicines Agency. Guideline on good pharmacovigilance practices (GVP)
Module XVI – Risk minimisation measures (Rev 3). July 26, 2024. Accessed January 7, 2025. https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/guideline-good-pharmacovigilance-practices-gvp-module-xvi-risk-minimisation-measures-rev-3_en.pdf
European Medicines Agency. Guideline on good pharmacovigilance practices (GVP)
Module XVI Addendum II – Methods for evaluating effectiveness of risk
minimisation measures. July 26, 2024. Accessed January 7, 2025. https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/guideline-good-pharmacovigilance-practices-gvp-module-xvi-addendum-ii-methods-evaluating-effectiveness-risk-minimisation-measures_en.pdf
U.S. Food & Drug Administration. Joint Meeting of the Drug Safety and Risk Management Advisory Committee and Psychopharmacologic Drugs Advisory Committee Meeting Announcement. November 19, 2024. Accessed January 7, 2025. https://www.fda.gov/advisory-committees/advisory-committee-calendar/updated-meeting-time-and-public-participation-information-november-19-2024-joint-meeting-drug-safety
U.S. Food & Drug Administration. Putting the Pieces Together: REMS Logic Model in REMS Design, Implementation, and Evaluation. [webinar] November 7, 2024. Accessed January 7, 2025. https://www.fda.gov/drugs/news-events-human-drugs/putting-pieces-together-rems-logic-model-rems-design-implementation-and-evaluation-11072024
U.S. Food & Drug Administration. REMS Logic Model: A Framework to Link Program Design With Assessment Guidance for Industry. Draft guidance. May 2024. Accessed January 7, 2025. https://www.fda.gov/media/178291/download
U.S. Food & Drug Administration. Risk Evaluation and Mitigation Strategies (REMS) for Autologous Chimeric Antigen Receptor (CAR) T cell Immunotherapies Modified to Minimize Burden on Healthcare Delivery System. June 26, 2024. Accessed January 7, 2025. https://www.fda.gov/vaccines-blood-biologics/safety-availability-biologics/risk-evaluation-and-mitigation-strategies-rems-autologous-chimeric-antigen-receptor-car-t-cell
