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...
Premier Research
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...
Oncology & Hematology
Harnessing Innovation in Oncology: Insights from ASCO 2025
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,...
Clinical Development
Amplifying Patient Voices: The Impact of Advocacy Groups in Clinical Trials
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 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....
The FDA’s Oncology Center of Excellence (OCE) plays a pivotal role in fostering innovation, collaboration, and efficiency in the development and evaluation of oncology products. Established in 2017 under the 21st Century Cures Act, the OCE brings together multidisciplinary scientific...
Clinical Development
Sponsor Responsibilities Under ICH GCP E6: The Non-Negotiables for CRO Oversight
In the rapidly evolving landscape of clinical research, sponsors are under increasing pressure to conduct efficient, high-quality trials that meet stringent regulatory standards. The International Council for Harmonisation's Good Clinical Practice (ICH GCP) E6 (R2) guidelines and the forthcoming E6...
New drug development is a long and expensive process that can be fraught with obstacles, including unexpected delays, poor efficacy results, safety issues, or regulatory challenges. Pharmaceutical companies may, at times, find themselves at an impasse during development, facing weighty...
Bringing a new therapeutic to market is a complex and multifaceted process culminating in submission and approval of a new drug application (NDA). Within the NDA, the chemistry, manufacturing, and controls (CMC) section plays a pivotal role in demonstrating drug...