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
Category: Clinical Research
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
The Evolving Integration of NGS Data into EMRs: Transforming Clinical Research and Precision Medicine
Next-Generation Sequencing (NGS) is a technology that allows the rapid sequencing of entire genomes, targeted regions, or specific sets of genes. It represents a significant advancement that enables the parallel sequencing of millions of DNA fragments simultaneously. NGS can be used for a variety of applications, including whole-genome sequencing (WGS), whole-exome sequencing (WES), targeted gene… Continue reading The Evolving Integration of NGS Data into EMRs: Transforming Clinical Research and Precision Medicine
Leveraging Natural History of Disease Studies for Integrated Evidence Generation Plans and Regulatory Success
Natural history of disease studies are intended to track the course of a disease by identifying demographic, genetic, environmental, and other variables (e.g., treatments, concomitant medications) that correlate with the disease’s development and outcomes. Natural history of disease studies play a crucial role in integrated evidence generation plans, especially for rare diseases and conditions where… Continue reading Leveraging Natural History of Disease Studies for Integrated Evidence Generation Plans and Regulatory Success
RWD for EMA Regulatory Submissions: Differences in Use for Pre- vs. Post-Approval
Real‐world evidence (RWE) has an increasing role in both pre-, and post- approval applications to support the authorization of new medicines and indications, or to address post-marketing safety concerns. Therefore, the use of Real-World Data (RWD) in regulatory processes at the European Medicines Agency (EMA) varies depending on whether it is being used for… Continue reading RWD for EMA Regulatory Submissions: Differences in Use for Pre- vs. Post-Approval