As cell and gene therapies continue to reshape treatment for complex and rare diseases, clinical research teams are navigating new operational realities. As these therapies advance, development strategies are also evolving to address new operational, regulatory, and evidence-generation challenges. Unlike many traditional therapies, cell and gene therapies may...
In recognition of Women’s History Month, we spoke with a leader who brings complementary perspectives to women’s health development: Stacey Ayres, PhD, Vice President, Regulatory Strategy. She reflected on how women’s health research has evolved — and what it will...
In rare disease research, every patient counts. Recruitment is challenging, retention even harder, and each data point is invaluable. Yet rigid trial protocols often create unnecessary barriers for patients, families, and sites alike. For rare conditions where participants are geographically...
Each year on September 17th, the World Health Organization recognizes World Patient Safety Day to raise awareness and promote action to reduce patient harm. This year’s theme, “Ensuring safe care for every newborn and child,” highlights the importance of protecting...
Long-term follow-up (LTFU) is a defining feature of oncology and cell and gene therapy (CGT) trials, where post-treatment monitoring often spans 2 to 15 years. These extended timelines allow regulators to assess long-term safety and efficacy from detecting delayed adverse...
The 2025 American Society of Clinical Oncology (ASCO) Annual Meeting offered a compelling look into the future of cancer care—one that is increasingly shaped by innovation, data, and a deeper commitment to the patient experience. From artificial intelligence (AI) transforming...
In the complex and costly world of new drug development, pharmacokinetic (PK) modeling has emerged as a promising method for driving more efficient and cost-effective clinical trials. This scientific approach leverages mathematical models to describe the relationship between drug dose,...
In the realm of clinical trials, patient involvement is not just a nicety—it's a necessity. The patient voice has become central to the success of clinical trials, shifting the focus from traditional stakeholders like healthcare providers and regulatory bodies to...
The traditional entry point for clinical research organization (CRO) engagement is delivery of the first clinical research study. However, companies with limited internal resources may benefit from engaging with a CRO as early as possible to leverage the full breadth...
The journey of developing effective treatments for rare diseases, particularly in a pediatric population, has its own unique challenges, including (but not limited to), a small population size, disease heterogeneity, and the absence of established endpoints to support regulatory decisionmaking....