While interventional, randomized double blind placebo-controlled studies are the gold standard used to assess efficacy of interventions [1], answering other scientific questions does not necessarily require an active intervention. My point is not to dis experimental studies in any way, as experimental studies are considered to be more reliable than observational studies because the process… Continue reading To RCT or not to RCT?
Category: Clinical trials
Transportability in Real-World Evidence (RWE) Research: Regulatory Considerations
What is Transportability in RWE?Transportability in Real-World Evidence (RWE) research refers to the ability to extend findings from one study population to a different, but related, population. This concept is critical when evaluating whether treatment effects observed in one geographical region, healthcare system, or patient cohort could be generalized to another population with different demographic,… Continue reading Transportability in Real-World Evidence (RWE) Research: Regulatory Considerations
AI-Driven Protocol Digitalization: Reshaping Clinical Research
The integration of artificial intelligence (AI) into clinical trial protocol digitalization is transforming key aspects of drug development. By applying analytical and predictive AI, there is the potential to design smarter, more efficient protocols, ensuring greater precision in endpoint definition, improved compliance, and reduced trial timelines. This article explores some of the applications AI… Continue reading AI-Driven Protocol Digitalization: Reshaping Clinical Research
EMA's Strategic Initiatives for Addressing Unmet Medical Needs
An unmet need in healthcare refers to a gap where current medical knowledge, treatments, or healthcare services do not adequately address a particular condition, disease, or health issue. These unmet needs can arise in various aspects of healthcare, such as the availability of effective treatments, access to healthcare services, or the ability to diagnose and… Continue reading EMA's Strategic Initiatives for Addressing Unmet Medical Needs
Integrated Evidence Generation Strategy for Rare Disease: A Comprehensive Approach Across Clinical Phases
Developing an integrated evidence generation strategy for a rare disease requires careful planning, cross-disciplinary collaboration, and adherence to regulatory guidelines. The strategy should be holistic, incorporating real-world evidence (RWE), clinical trials, and patient-centric data to address the unique challenges associated with rare diseases and to support regulatory approvals, market access, and ongoing patient care. … Continue reading Integrated Evidence Generation Strategy for Rare Disease: A Comprehensive Approach Across Clinical Phases