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Real-world evidence is increasingly influencing trial planning and regulatory decisions in rare disease drug development. Economic pressures are prompting a strategic reassessment of efficiency and technology in this sector.
Real-world evidence complements RCTs, influencing trial planning and regulatory decisions, despite challenges like incomplete data and privacy concerns. Economic pressures in rare disease drug development lead to strategic reassessment, focusing on efficiency, technology, and alternative therapeutic areas.SHOW MORE · Generative AI adoption, regulatory scrutiny, DEI policy shifts, real-world evidence, and rare disease economics emerged as key themes across these most-read Applied Clinical ... Real-world evidence complements RCTs, influencing trial planning and regulatory decisions, despite challenges like incomplete data and privacy concerns. Economic pressures in rare disease drug development lead to strategic reassessment, focusing on efficiency, technology, and alternative therapeutic areas.SHOW MORE · Generative AI adoption, regulatory scrutiny, DEI policy shifts, real-world evidence, and rare disease economics emerged as key themes across these most-read Applied Clinical Trials articles shaping clinical research and drug development today. Rare disease drug development has expanded significantly since the Orphan Drug Act, but declining returns and rising costs are forcing sponsors to reassess their strategies. The article outlines clinical and commercial challenges, including longer and more complex trials, limited patient populations, ethical issues with control groups, and high manufacturing costs—particularly for gene therapies. While sponsors remain committed to addressing unmet needs, economic pressures are driving divestment, restructuring, and renewed focus on efficiency, technology-enabled development, and alternative therapeutic areas. Stay current in clinical research with Applied Clinical Trials, providing expert insights, regulatory updates, and practical strategies for successful clinical trial design and execution. Recent US executive orders banning DEI initiatives have created uncertainty across the clinical research ecosystem, particularly around diversity-focused recruitment and engagement efforts. The article examines potential consequences for sponsors, sites, patients, healthcare systems, and regulators, noting risks to trial representativeness, public trust, and R&D efficiency.
Original title: “Our Top 5 Most Read Articles in 2025 | Applied Clinical Trials Online”