Introduction: High-quality randomised controlled trials (RCTs) provide the most reliable
evidence on the comparative efficacy of new medicines. However, non-randomised studies …

Randomized controlled trials (RCTs) form a cornerstone of oncology research by generating
evidence about the efficacy of therapies in selected patient populations. However, their …

The emergence of the precision medicine paradigm in oncology has led to increasing
interest in the integration of real-world data (RWD) into cancer clinical research. As sources …

The precision oncology paradigm challenges the feasibility and data generalizability of
traditional clinical trials. Consequently, an unmet need exists for practical approaches to test …

Real-world data (RWD) and real-world evidence (RWE) appear now as complementary to
traditional randomized controlled trials (RCT), that remain the gold standard of the evidence …

Abstract Objectives The Joint ISPOR-ISPE Special Task Force on Real-World Evidence
included patient/stakeholder engagement as a recommended good procedural practice …

Background Gaucher disease (GD) is a rare autosomal recessive lysosomal storage
disorder. GD types 2 and 3 are known as neuronopathic Gaucher disease (nGD) because …

Real-world evidence (RWE) research helps determine whether outcomes observed in
clinical trials happen in real-life clinical practice. RWE research may help patients receive …

Simple Summary More than 50% of breast cancers may be preventable with adherence to
healthy lifestyle practices, but the influences of each single preventive/predisposing …

People with rare lysosomal storage diseases face challenges in their care that arise from
disease complexity and heterogeneity, compounded by many healthcare professionals …