Diversity in clinical trials: Optimizing drug development strategy, leveraging data and increasing efficiency with modeling and simulation.
By Lisa Benincosa and Katy Moore Clinical trials have evolved over the years with inclusion of broader patient populations; yet, additional efforts are needed to better understand the safety and efficacy across the diversity of patients who will be using the medicine. Factors such as race, gender, preexisting conditions, and age can all impact how a […]
How to Overcome Challenges in Combination Immunotherapy Clinical Trials
Immunotherapy has quickly become a major focus in the world of cancer research and treatment. In recent years, there has been a surge in the number of biotech companies conducting clinical trials for combination immunotherapies. What are Combination Immunotherapies? The combination immunotherapy approach involves the use of two or more different types of immunotherapies in […]
Developing CNS Neurotherapeutics with Cerebrospinal PK/PD Clinical Studies
Neurological diseases affect the brain, spinal cord, and peripheral nervous system. They range from diseases that affect large populations such as Alzheimer’s, epilepsy, stroke, Parkinson’s, and multiple sclerosis to rare and genetically inherited diseases like Huntington’s, lysosomal storage diseases, and Amyotrophic Lateral Sclerosis. With more than 2 billion people affected worldwide (and rising), neurological diseases […]
Accelerating Drug Development of Innovative Oncologic Agents: Master Protocols
By Marites T. Woon, Associate Director, Nonclinical, and Jessica Lee, VP, Regulatory Strategy, Head, Cell / Gene Therapy Exponential advancements in oncology continue to drive personalized medicine and biomarker-driven therapeutics into reality. The development of innovative therapies to treat serious diseases remains a frontrunner across drug and cell and gene therapy (CGT) pipelines, with oncology […]
Long-Term Follow-Up for CGT Trials: Best Practices to Operationalize
By Desmond Cabrera, Project Director, Cell and Gene Therapy With today’s cutting-edge technology, we are on the cusp of developing truly magnificent therapies using cell and gene science. However, as with any new therapy, we must always consider the patient first. Guidelines to Ensure Patient Safety As far back as 2001, as new cell and […]
What is Pharmacodynamics?
By Rachel Rozakis, PharmD, Clinical Pharmacologist II and Ben Kennard, MS, CPMS Intern Pharmacodynamics (PD) is the study of how drugs affect the human body given their mechanism of action. In contrast, pharmacokinetics (PK) is the study of what the body does to the drug and describes information about ADME: absorption, distribution, metabolism, and excretion. PK […]
The Role of Statisticians in Study Design and Why to Engage them Early
By Melanie Buitendyk, Director, Biostatistical Consulting A Sponsor has a promising therapy and is in the process of planning their clinical development strategy. Key components of this planning include regulatory and corporate objectives, go-no-go decisions, and on a study level, includes study design, study objectives, and endpoints. Well-planned, well-designed, and well-written protocols are essential in […]
Risk Assessment: Traditional and Novel Approaches
Risk assessment and management are complex processes intended to protect human health by identifying product-related toxicities (i.e., hazards); determining dose-response relations for a product within relevant biological systems; and assessing exposure levels to characterize potential risks. These assessments may be conducted via traditional frameworks using data from in vitro and in vivo studies, as well as novel frameworks using pharmacokinetic […]
The Role of Clinical Pharmacology in New Drug Development
Bringing a new drug to market is a cost and time-intensive process requiring a diverse range of professionals to come together and drive the program to the desired outcome. Clinical pharmacologists play an important role in this multidisciplinary team via the appropriate use of pharmacological principles and techniques during the clinical development cycle. This blog […]
Gap Analysis: The Benefit of Conducting Technical and Regulatory Due Diligence
By Marites T. Woon (Associate Director, Nonclinical), Nick Sanich (Director, Business Development), Richard Stewart (VP, Nonclinical), William Lee (VP, Regulatory Affairs), and Marcus S. Delatte (VP, Regulatory Strategy) Regulatory strategies are different for each product and should be designed specifically with the end objectives in mind. Regulatory due diligence helps to plan for regulatory requirements […]