The Future of Population-Based Postmarket Drug Risk Assessment: A Regulator’s Perspective

Abstract

The US Food and Drug Administration emphasizes the role of regulatory science in the fulfillment of its mission to promote and protect public health and foster innovation. With respect to the evaluation of drug effects in the real world, regulatory science plays an important role in drug risk assessment and management. This article discusses opportunities and challenges with population-based drug risk assessment as well as related regulatory science knowledge gaps in the following areas: (i) population-based data sources and methods to evaluate drug safety issues; (ii) evidence-based thresholds to account for uncertainty in postmarket data; (iii) approaches to optimize the integration and interpretation of evidence from different sources; and (iv) approaches to evaluate the real-world impact of regulatory decisions. Regulators should continue the ongoing dialogue with multiple stakeholders to strengthen regulatory safety science and address these and other critical knowledge gaps.

Can social media data lead to earlier detection of drug‐related adverse events?

“…5 Leveraging social media data for pharmacovigilance requires an understanding of their strengths and weaknesses relative to conventional data sources, such as the FDA Adverse Event Reporting System (FAERS). 6 However, the existing literature comparing social media and conventional pharmacovigilance data sources is limited to the comparison of the reporting characteristics without further investigating the inter-temporal relationship of the reporting pattern between these sources. [7][8][9] The objective of this study is to shed light on the potential role of social media data in pharmacovigilance, including their ability to accelerate the detection of drug-related AEs.…”

Cardiovascular safety signals with dipeptidyl peptidase‐4 inhibitors: A disproportionality analysis among high‐risk patients

“…The FDA has acknowledged the limitations of its current signal detection methods and is actively seeking novel approaches to its surveillance activities. Two points of concern with current practices are the threshold of EB05 = 2.0 and residual confounding . Through restriction of the Bayesian prior to adverse event reports stemming from a pool of patients with related illnesses, our approach can reduce the level of residual confounding.…”

“…Two points of concern with current practices are the threshold of EB05 = 2.0 and residual confounding. 24 Through restriction of the Bayesian prior to adverse event reports stemming from a pool of patients with related illnesses, our approach can reduce the level of residual confounding. Additionally, as the EB05 = 2.0 threshold is considered a minimal threshold for further investigation of a drug safety concern, regulators can adjust this threshold based on the restricted patient population and their unique health concerns.…”

Reinventing Bioinnovation

“…Finally, bioinnovation is not limited to drug discovery and development. Innovation in postmarketing surveillance and monitoring is needed as well, as was highlighted by PCAST, and is addressed in a State of the Art contribution by Hammad et al 19 …”

“…Finally, bioinnovation is not limited to drug discovery and development. Innovation in postmarketing surveillance and monitoring is needed as well, as was highlighted by PCAST, and is addressed in a State of the Art contribution by Hammad et al 19 The bioinnovation landscape is clearly shifting. In the new environment, integrated partnerships will span the entire R&D continuum-from early research through development, postmarketing surveillance, and commercialization-and will play a vital role in ensuring that new medicines continue to reach waiting patients.…”