Evolving PV Landscape: Artificial Intelligence and Advanced Gene Therapy

Artificial Intelligence (AI) is rapidly reshaping pharmacovigilance (PV), offering substantial improvements in efficiency and accuracy across key activities. Tasks such as safety narrative writing or Risk Evaluation and Mitigation Strategy (REMS) assessment report writing can be streamlined by AI to reduce preparation time and mitigate the limitations associated with manual or template-based narratives.

For Development Safety Update Reports (DSURs) and Periodic Safety Update Reports (PSURs), AI can optimize the preparation of complex tabular data, presenting it clearly and systematically, and effectively extracting key safety metrics such as serious adverse events (SAEs) and serious adverse reactions (SARs). Despite these clear benefits, integrating AI into PV activities also presents challenges including data security, evolving regulatory landscapes, and the necessity for rigorous validation processes.

Human oversight remains critical to address the inherent variability of AI-generated outputs and to ensure alignment with regulatory and ethical standards. Additionally, fostering AI awareness among stakeholders is essential for successful adoption.

In parallel, the harmonization of post-marketing pharmacovigilance guidelines for next-generation biologic therapies, particularly gene therapies, is crucial. Gene therapies, defined by regulatory bodies such as the FDA and EMA, involve the insertion, modification, or removal of genetic material within a patient's cells to treat various diseases. Regulatory consistency across agencies like the FDA and EMA is essential to managing unique safety concerns, including long-term patient follow-up, ethical considerations, technical specificities, and anticipated clinical safety concerns. Key regulatory frameworks such as the EMA's Regulation (EC) No 1394/2007, the GVP modules, and the FDA's guidance on long-term follow-up and REMS plans form the foundation for effective pharmacovigilance of gene therapies.

Specialized requirements for gene therapy pharmacovigilance include enhanced lot surveillance, batch traceability, comprehensive long-term follow-up protocols and duration, tailored risk management plans (RMPs), and specialized post-authorization safety studies (PASS) designed with adapted endpoints and extended monitoring periods. Regulatory bodies, including the Committee for Advanced Therapies (CAT) from EMA and the Office of Therapeutics Products (OTP) from FDA's CBER, actively work towards developing comprehensive and adaptable post-marketing guidelines that reflect the incremental scientific knowledge and clinical experience in gene therapy.

Close international collaboration through forums such as the Cell and Gene Therapies Discussion Group (CGTDG) at the ICH is critical for achieving global regulatory harmonization. This collaborative approach aims to enhance safety reporting, improve signal detection methodologies, optimize risk management practices, and ensure a robust and cohesive pharmacovigilance system tailored specifically for gene therapy products.

In conclusion, integrating AI into pharmacovigilance and harmonizing global guidelines for advanced therapies like gene therapies are essential steps towards enhanced patient safety, improved efficiency, and optimized risk management. As the PV landscape continues to evolve, proactive adoption of these innovative approaches and ongoing international collaboration will be key to addressing emerging challenges and leveraging new opportunities.ind financial services and retailers in omnichannel experiences, but we are seeing a wide array of tactics including QR-codes in leave behinds at healthcare provider offices, brochures, TV commercials, magazine ads, internet banners, and new collaborations between patient advocacy groups to drive PSP awareness.

About UBC
United BioSource LLC (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.

About the Authors
Dobrochna Dolicka, PhD, serves as a Safety Scientist on UBC's Global Safety Writing Team. For the last 3 years, she has been responsible for authoring periodic safety reports such as DSURs, PBRERs, and PADERs, as well as conducting other signal management activities. Ms. Dolicka is also working on the development and implementation of automation and artificial intelligence across UBC's comprehensive pharmacovigilance services. She holds a PhD in Biomedical Sciences as well as a Master's in Molecular Biology.

Christopher Henry, PhD, serves as a Safety Scientist on UBC's Global Safety Writing Team. With a strong biomedical and AI background, Mr. Henry has brought his unique skillset to UBC's pharmacovigilance team. He is responsible for authoring periodic safety reports such as DSURs, PBRERs, and PADERs, as well as conducting other signal management activities for pharmaceutical products that are still in development and products that are already marketed. Mr. Henry has been leading the development and implementation of artificial intelligence across UBC's comprehensive pharmacovigilance services. He holds a PhD in Cell Physiology as well as a Master's in Health Biology, Genetics, Epigenetics, & Cell Fate Control. He has worked at UBC for the past 3 years.

Alix Garcia, PhD serves as a Safety Scientist on UBC's Global Safety Writing Team. With almost a decade of industry experience, Ms. Garcia has worked across gene therapies, vaccines, immunotherapies, and more. She works across pharmaceutical products that are still in development and products that are already marketed. Ms. Garcia features a deep background in gene therapy after performing academic research into the impact of MiRNAs on cell function. She has also been published as a peer-reviewed author. She holds a PhD in Biomedical Sciences as well as a Master's in Bio-Industrial Engineering. She has been with UBC for the past 4 years.

Irene Navarro de la Fuente, MSc, serves as a Safety Scientist on UBC's Global Safety Writing Team. Ms. Navarro has deep experience in safety activities for biologic products, including gene therapy and CAR-T cell therapy for oncology and rare disease. In addition to pharmacovigilance, she also participates in risk management planning for pharma. She has been with UBC for almost 4 years and has spent the last 9 years working in the industry.

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