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, concentration in bodily fluids, and
On April 10, 2025, the US FDA announced that it has a long-term plan to eliminate conventional animal testing in drug development, starting with monoclonal antibodies (mAbs).[1] Instead, within 3-5 years, the expectation will be to leverage an integrated safety assessment approach, combining information from
Alzheimer’s Disease (AD) is a progressive, irreversible brain disorder that affects memory, thinking, and language skills. Age is the best-known risk factor for AD, with symptoms usually appearing after 60 years of age. According to the Alzheimer’s Association and the Center for Diseases Control and
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. While regulatory authorities have special
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 expertise to accelerate the review
Barry Mangum, Senior Vice President, Pediatrics at Premier Research, talks about the importance of pediatric research, its evolution over the years, and the obstacles and opportunities the industry faces. Q: How long have you been in pediatric clinical research, and how did you enter the
While the COVID-19 pandemic has accelerated innovation in many areas, including clinical trial design, many recent advances were already in motion before 2020 as sponsors and CROs sought to improve the patient journey and encourage trial participation. Traditional trials, especially those for pediatric populations, often
Conducting a clinical study in a pediatric patient population requires careful planning, as providing treatment to a child without causing harm is a weighty responsibility. The first tasks in striking this balance are dose selection and dosing interval determination, and this blog post describes how PK modeling
This is the third installment in a four-part blog series highlighting FDA designations for products with rare disease indications: orphan drug designation (ODD), rare pediatric disease designation (RPDD), and humanitarian use device (HUD) designation. Part 2 examined orphan drugs, and in Part 3, we take a detailed
Between 2010 and 2018, 33.8 percent of the 402 orphan indications approved by the FDA were approved for children only or targeted pediatric diseases.1 The rare pediatric disease priority review voucher program is one incentive that’s helped to spur research and development in this area
