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Regulatory Considerations Toward Orphan Drug Designation and Orphan Drug Exclusivity in the United States and European Union: Structural Similarity, Clinical Superiority/Significant Benefit, and Case Studies
The U.S. Food and Drug Administration and European Commission have developed successful orphan drug legislation to promote the research, development,...
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Planning Benefit-Risk Assessments Using Visualizations
A Benefit Risk Assessment Plan (BRAP) describes the assessments planned to determine whether the benefits of an investigational drug outweigh the...
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Simulation for Clinical Pharmacokinetics
Simulation in clinical pharmacokinetics and therapeutic drug monitoring (TDM) is a powerful tool for optimizing drug therapy and enhancing patient...
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Impact of the US Accelerated Approval for New Anticancer Drugs on Time to Verification of Benefit and Regulatory Approval in the EU and Japan
The accelerated approval (AA) program in the USA has succeeded in expediting the regulatory approval of new cancer drugs based on surrogate endpoint...
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A Structured Benefit-Risk Assessment Operating Model for Investigational Medicinal Products in the Pharmaceutical Industry
Robust and transparent formal benefit-risk (BR) analyses for medicinal products represent a means to better understand the appropriate use of...
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Incorporating Prior Data in Quantitative Benefit–Risk Assessments: Case Study of a Bayesian Method
BackgroundMultiple criteria decision analysis (MCDA) and stochastic multi-criteria acceptability analysis (SMAA) in their current implementation...
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A Survey to Assess the Current Status of Structured Benefit-Risk Assessment in the Global Drug and Medical Device Industry
BackgroundThis industry survey was conducted to gain insight into the ways structured Benefit-Risk assessment (sBRA) of medical products is...
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Digital Health Technology (DHT) in European Clinical Trials, How to Improve the Status-Quo of the Regulatory Landscape?
Digital health technology (DHT) is increasingly used to facilitate the conduct of clinical drug trials. The European regulatory environment would...
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EU’s Medical Device Expert Panels: Analysis of Membership and Published Clinical Evaluation Consultation Procedure (CECP) Results
BackgroundThe new EU Medical Device Regulation (MDR) places greater importance on the role of clinical evidence to establish safety and performance....
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Accurate Collection of Reasons for Treatment Discontinuation to Better Define Estimands in Clinical Trials
BackgroundReasons for treatment discontinuation are important not only to understand the benefit and risk profile of experimental treatments, but...
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Decentralized Clinical Trials: Scientific Considerations Through the Lens of the Estimand Framework
While industry and regulators’ interest in decentralized clinical trials (DCTs) is long-standing, the Covid-19 pandemic accelerated and broadened the...
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Implementing Decentralized Clinical Trials in Australia through Teletrials: Where to From Here?
Implementation of decentralized approaches can improve access to clinical trials. The Australian government has focused on a teletrial model, which...
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A Modified Delphi Study to Establish Essential Clinical Pharmacology Competencies
IntroductionCompetency-based education has been commonly used to enhance the healthcare workforce for some time. A translational discipline that is...
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Good Statistical Monitoring: A Flexible Open-Source Tool to Detect Risks in Clinical Trials
BackgroundRisk-based quality management is a regulatory-recommended approach to manage risk in a clinical trial. A key element of this strategy is to...
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Digital Tools—Regulatory Considerations for Application in Clinical Trials
During the last few years, the pharmaceutical industry has adopted digital technologies/digital health technology (DHT) to improve the drug...
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Predicting Regulatory Product Approvals Using a Proposed Quantitative Version of FDA’s Benefit–Risk Framework to Calculate Net-Benefit Score and Benefit–Risk Ratio
BackgroundApproval of regulated medical products in the USA is based upon a rigorous review of the benefits and risks as performed by the US Food and...
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A Tutorial on Modern Bayesian Methods in Clinical Trials
Clinical trials continue to be the gold standard for evaluating new medical technologies. New advancements in modern computation power have led to...
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The Next Horizon of Drug Development: External Control Arms and Innovative Tools to Enrich Clinical Trial Data
Conducting clinical trials (CTs) has become increasingly costly and complex in terms of designing and operationalizing. These challenges exist in...
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The Renovation of Good Clinical Practice: A Framework for Key Components of ICH E8
The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use’s (ICH) renovation of Good Clinical Practice...
