Benefit assessment of therapeutic products: the Centers for Education and Research on Therapeutics

Abstract

The ability to manage risk depends critically on an understanding of the degree to which a known risk is balanced by the probability of a clinical benefit. Despite the massive emphasis on risk and risk management in the past few years and the long-term focus on defining benefit in the regulatory system, considerable uncertainty remains about the methods of defining benefit and how to operationalize this knowledge. In this 'think tank,' part of a larger series on risk management, issues were divided into those that can be identified before a study is initiated, those that commonly arise after a study is completed, biomarkers and surrogates, use of benefit findings in defining quality and performance indicators, implementation of findings into health systems and formularies, and methods of comparative trials. Key categories for the establishment of a research agenda to fill in gaps in our understanding of assessing benefit were developed by the group.

Additional safety risk to exceptionally approved drugs in Europe?

“…Adverse drug events, whether or not due to (in)correct use of drugs, have been estimated to be a leading cause of unplanned hospital admission [1,2]. New drugs are allowed onto the market based on relatively limited knowledge of their benefit-risk profile due to inherent and well-known limitations in pre-approval clinical trials [3]. Those trials are typically performed in carefully selected patient populations not fully representing 'real world' patients, are of relatively short duration and are primarily developed to determine efficacy [4].…”

A Review of Quantitative Risk–Benefit Methodologies for Assessing Drug Safety and Efficacy—Report of the ISPOR Risk–Benefit Management Working Group

“…The benefit associated with a medication might be its ability to reduce either an ADE or another adverse event associated with the disease itself. In this case, relative risk reduction (RRR) is useful and is defined as the ratio between the proportion of exposed individuals who experience a decline in adverse events divided by the proportion of unexposed individuals who experience such a benefit [27,28,38,40,43]. For instance, again in the case of osteoporosis, RRR rates are computed for different antiosteoporosis medications based on the number of fractures averted by the treatments.…”

“…For instance, again in the case of osteoporosis, RRR rates are computed for different antiosteoporosis medications based on the number of fractures averted by the treatments. Similarly, attributable risk reducation, otherwise known as absolute risk reduction (ARR), represents the decline in adverse events between exposed and unexposed groups [27,28,38,43]. However, "benefit" does not always mean absence of adverse events.…”

Role of the Centers for Education and Research on Therapeutics (CERTs) in Pharmacovigilance and Proper Use of Therapeutics

“…The deliberations of these think-tank meetings have been widely disseminated [5][6][7][8][9] and are credited with advancing the rapidly evolving practice of therapeutic risk management. For example, the programwide think-tank meetings bring together select groups of experts knowledgeable about a particular topic and representing a variety of perspectives important in addressing questions related to the study and use of therapeutics.…”

“…Th us far, seven meetings have been held on topics related to the assessment, communication, and management of risks associated with the use of medications, medical devices, and biological products. The deliberations of these think-tank meetings have been widely disseminated [5][6][7][8][9] and are credited with advancing the rapidly evolving practice of therapeutic risk management.…”