Assessing the benefit-risk balance of drugs. Some lessons from the COVID pandemic

ABSTRACT

Introduction

Drug efficacy and effectiveness are assessed respectively through clinical trials and pharmaco-epidemiological studies. However, relative and absolute benefits of drugs are distinct measures that must be considered in relation to the baseline risk of disease incidence, complication or progression. On the other hand, adverse drug reactions are independent of the basic risk but depend on the characteristics of the population treated. Given these prerequisites, how can we balance the benefits and risks of drugs?

Areas covered

We use the example of therapeutics evaluated during Covid to describe how assessing the benefit-risk balance of drugs is a complex process.

Expert opinion

Clinical trials are not designed to identify rare adverse events, underscoring the necessity for a pharmacovigilance system. Evaluating the balance between the benefits and risks of drugs is an ongoing process, demanding the simultaneous analysis of data from clinical trials, potential drug-drug interactions, pharmacovigilance monitoring and pharmaco-epidemiological studies, to identify potential safety concerns. In addition, pharmacologists must play a major role in educating the general public about drugs, aiding in the accurate interpretation of the benefit-risk balance and preventing misinformation.

KEYWORDS:

• Benefit-risk assessment
• Covid
• drug adverse reaction
• drug-drug interaction
• efficacy
• effectiveness
• pharmacology

Article highlights

• Drug efficacy is assessed through experimental designs such as clinical trials, while drug effectiveness is assessed in real-world scenarios, as performed in pharmaco-epidemiological studies.

• Relative and absolute benefits of drugs are distinct measures that must be considered in relation to the baseline risk of disease incidence, complication or progression.

• Clinical trials are not designed to identify rare adverse events, underscoring the necessity for a pharmacovigilance system.

• Evaluating the balance between the benefits and risks of drugs is an ongoing process, demanding the simultaneous analysis of data from clinical trials, potential drug-drug interactions, pharmacovigilance monitoring and pharmaco-epidemiological studies, to identify potential safety concerns.

• Pharmacologists must play a major role in educating the general public about drugs, aiding in the accurate interpretation of the benefit/risk balance and preventing misinformation.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Author contribution statement

JL Cracowski was the primary author who drafted the manuscript. All authors critically revised the work and gave final approval of the submitted version.

Additional information

Funding

This paper was not funded.