Biomedical and pharmaceutical companies who intend to bring a product to market must demonstrate safety and efficacy of those products via closely monitored preclinical and clinical studies. This process encompasses years of investigation with robust scrutiny of potential safety issues, as a product is put through the various stages of regulatory review.
While this process is essential for a product to receive the necessary approvals for use, the post marketing period is just as critical for ensuring safety to end-users, namely patients with the most at stake. Unfortunately, this period is also more fraught as safety-related communications amongst stakeholders, can often be disparate, delayed, and even contradictory.
The inherited bleeding disorder (IBD) community has seen its share of poor reporting and communication of safety incidents in recent years, which compelled the National Hemophilia Foundation (NHF) and the Hemophilia Federation of Hemophilia (HFA) to hold a Safety Summit.
Held in January of 2020, the summit convened all stakeholders with a responsibility for patient safety, in some capacity, within the IBD community. The primary focus was pharmacovigilance, broadly defined as the science and activities devoted to the detection, assessment, understanding, and prevention of adverse effects related to drugs or other medical devices.
The Summit generated comprehensive recommendations for stakeholders in the IBD sphere, specifically for those that while currently contributing to pharmacovigilance, could do more to enhance their effectiveness and reach. The recommendations are targeted to regulatory agencies, patient organizations, specialty pharmacies, healthcare providers, industry, existing systems (e.g. The Patient Notification System), and the patients themselves.
The recommendations, including important background information, are detailed in a new paper published in the journal Therapeutic Advances in Drug Safety. It cites several real-world incidences of poor communication of safety issues, including those related product recalls, within the IBD community.
The availability of these recommendations come at an important time as new therapies, developed with increasingly novel and complex technologies, become available to IBD patients.
Prominent themes emerged from the Summit, such as patient-centricity, transparency, and the efficient collection/dissemination of new safety information that is clear and accessible – this includes agreed upon definitions of what constitutes an “adverse event” associated with a particular product.
The goal is to help place IBD patients in the best possible stance to make both timely and well-informed decisions about the drugs and devices they use. The authors emphasize the central role of patients as subject matter experts, centrally positioned, and ideally empowered, to drive these pharmacovigilance efforts.
“Committing to the primacy of patient well-being and establishing, and stewarding reliable, collaborative channels of communication before a crisis arises in which they are urgently needed, emerged as the two overarching principles from the bleeding disorder stakeholder discussions,” concluded the authors. “The application of these two principles to pharmacovigilance in all therapeutic areas will benefit patients, industry, and regulators alike.”
Access the full paper in Therapeutic Advances in Drug Safety to read the complete set of recommendations.
Robinson F, Wilkes S, Schaefer N, et al. Patient-centered pharmacovigilance: priority actions from the inherited bleeding disorders community. Therapeutic Advances in Drug Safety. 2023;14. doi:10.1177/20420986221146418
Disclaimer: NHF provides periodic synopses of articles published in peer reviewed journals, the purpose of which is to highlight papers that cover a wide range of topics and speak to a broad spectrum of the inherited blood disorders community. Topics include shared decision making, gene therapy, health equity, and more. NHF hopes you find this content to be informative and engaging.
Any questions about the articles featured here should be directed to the publishing journal and/or the study authors. This content is for general information only. NHF does not give medical advice or engage in the practice of medicine. NHF under no circumstances recommends particular treatment for specific individuals and in all cases recommends that you consult your physician or local treatment center before pursuing any course of treatment.