Medicines safety monitoring is a continuous and dynamic process throughout all the phases of the life cycle of a drug. During the drug development, safety is investigated in different phases. In preclinical studies, the primary goal of safety evaluation is the identification of a safe dose in humans and of safety parameters for clinical monitoring. In clinical phase, phase I studies are designed to estimate the tolerability of the dose range expected to be needed for later clinical studies in healthy volunteers; phase II studies are focused on determining appropriate range of drug doses in patients with a disease or condition of interest, while phase III clinical trials are the most important studies to refine understanding of benefit-risk profile of the drug and to identify less common adverse drug reactions. Although drug safety evaluation is very rigorous and thorough, pre-marketing clinical trials have however intrinsic limitations that do not allow to exhaustively evaluate drug safety profile. These studies are conducted on limited numbers of patients that are selected based on strict eligibility criteria and not fully representing real-world populations and have limited duration, thus preventing detection of rare and long-term adverse reactions. Therefore, the post-marketing assessment of medicines plays a key role for better defining drugs’safety profile in real-world setting and filling the evidence gap of pre-marketing studies. In the field of drug safety and regulation, a number of challenges have to be faced in the near future. First of all, COVID-19 pandemic highlighted how relevant pharmacovigilance and proper risk communication during public health emergency are. Second, the development of advanced methodologies including machine learning techniques and the availability of large amount of electronic healthcare data offer opportunity for optimizing drug benefit-risk profile evaluation in real world setting. Finally, innovative therapeutics, such as advanced therapy medicinal products, digital therapeutics, vaccines developed based on advanced technologies, requiring special pharmacovigilance monitoring have been increasingly marketed in recent years, often upon accelerated pathway approval.
A New Era of Pharmacovigilance: Future Challenges and Opportunities
Trifirò, Gianluca;Crisafulli, Salvatore
2022-01-01
Abstract
Medicines safety monitoring is a continuous and dynamic process throughout all the phases of the life cycle of a drug. During the drug development, safety is investigated in different phases. In preclinical studies, the primary goal of safety evaluation is the identification of a safe dose in humans and of safety parameters for clinical monitoring. In clinical phase, phase I studies are designed to estimate the tolerability of the dose range expected to be needed for later clinical studies in healthy volunteers; phase II studies are focused on determining appropriate range of drug doses in patients with a disease or condition of interest, while phase III clinical trials are the most important studies to refine understanding of benefit-risk profile of the drug and to identify less common adverse drug reactions. Although drug safety evaluation is very rigorous and thorough, pre-marketing clinical trials have however intrinsic limitations that do not allow to exhaustively evaluate drug safety profile. These studies are conducted on limited numbers of patients that are selected based on strict eligibility criteria and not fully representing real-world populations and have limited duration, thus preventing detection of rare and long-term adverse reactions. Therefore, the post-marketing assessment of medicines plays a key role for better defining drugs’safety profile in real-world setting and filling the evidence gap of pre-marketing studies. In the field of drug safety and regulation, a number of challenges have to be faced in the near future. First of all, COVID-19 pandemic highlighted how relevant pharmacovigilance and proper risk communication during public health emergency are. Second, the development of advanced methodologies including machine learning techniques and the availability of large amount of electronic healthcare data offer opportunity for optimizing drug benefit-risk profile evaluation in real world setting. Finally, innovative therapeutics, such as advanced therapy medicinal products, digital therapeutics, vaccines developed based on advanced technologies, requiring special pharmacovigilance monitoring have been increasingly marketed in recent years, often upon accelerated pathway approval.
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