Our Top 5 Most Read Articles in 2025

1. How Medical Writing and Regulatory Affairs Professionals Can Embrace and Deploy Generative AI at Scale

Inger Ødum Nielsen, Vanessa de Langsdorff, and John April

Generative AI has become a strategic priority across life sciences, with growing use in clinical documentation and regulatory submissions, particularly clinical study reports (CSRs). The article explains that successful automation depends on data and content readiness, including standardized data formats, streamlined content ecosystems, and alignment with regulatory guidance. It details how eliminating subjective and redundant content, adopting objective data descriptions, and referring to validated tables and protocols can reduce review burden and improve consistency. The authors also outline how TransCelerate’s CSR template and a clearly defined CTD content ecosystem can support scalable AI adoption while meeting regulatory expectations.

2. The Impact of DEI Ban on Clinical Research Ecosystem

Rebecca Johnson, PhD

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. It highlights ongoing requirements under FDORA for diversity action plans, while outlining how ambiguity in federal policy may affect academic research funding, workforce diversity, and community engagement. Despite shifting language and compliance pressures, many sponsors and stakeholders continue pursuing population-representative trials to meet scientific and regulatory expectations.

3. FDA Crackdown on Trial Design: What July’s CRLs to Replimune and Capricor Mean for Sponsors

Andy Studna

Complete Response Letters issued to Replimune and Capricor in July signal heightened FDA scrutiny of trial design, even for programs that previously appeared aligned with agency guidance. In both cases, the FDA cited concerns related to study control and the need for additional or more robust data. The article details how these actions, alongside broader FDA transparency efforts and new policy positions, underscore the importance of documenting regulatory communications and planning for shifting expectations early. Sponsors are advised to prioritize rigorous control arms, adaptive designs, geographic diversity, and clear written confirmation of FDA feedback to reduce regulatory risk.

4. The Importance of Real-World Evidence in Medical Research and Drug Development

Manfred Stapff, MD, PhD

Real-world evidence (RWE) is increasingly complementing randomized controlled trials by providing insight into how treatments perform in routine clinical practice. Enabled by the 21st Century Cures Act, RWE has influenced trial planning, site selection, regulatory decisions, and medical guidelines. The article reviews FDA approvals that relied on real-world data, including expanded indications granted without traditional RCTs. It also discusses challenges such as incomplete data and privacy concerns, while emphasizing RWE’s role in improving generalizability, informing treatment decisions, and supporting personalized medicine.

5. The Hard Truth About Rare Disease and Gene Therapy Drug Development

Ken Getz, MBA

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. Data from Tufts CSDD and Deloitte show extended timelines and sharply reduced ROI for recent launches. 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.