Benefits of Bayesian Dose Escalation Designs for Oncology Studies
As acceptance of flexible trial designs continues to advance, many sponsors are increasingly exploring alternate methods for dose escalation to support identification of a maximum tolerated dose (MTD) and recommended Phase II dose (RP2D) in open-label Early Phase or First in Human studies. Bayesian dose escalation designs have also coincided with advances in oncology therapy […]
Risk Assessment: Traditional and Novel Approaches to Assess and Manage Risks
Risk assessment is a complex process intended to protect human health by identifying drug-related 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 and pharmacodynamic model-based […]
Inspection Readiness: What is it, why it matters, and what you need to know to be prepared
Organizations must have inspection readiness since inspections can be unannounced or come at any time. Good Clinical Practice (GCP) inspectors use different approaches depending on the regulatory authorities for which they represent; however, all of them will verify the compliance status on GCP and other applicable regulations during the inspection. Planning ahead to anticipate all processes are compliant means an organization […]
Navigating Regulatory Strategies for Cellular & Gene Therapies
While any drug development program can face regulatory hurdles, developing cellular and gene therapies often presents unique challenges. Small and midsize biotech and pharma companies developing cellular and gene therapies should begin to understand critical nuances for successful regulatory meetings and submissions and how they impact strategic decisions during the product development lifecycle. At the […]
Roadmap To Marketing Authorisation in Europe: Taking All the Right Turns
Approval of your Marketing Authorisation Application (MAA) is the final hurdle to pharmaceutical product launch in Europe. Adequate and timely navigation of this roadmap is essential to achieve a successful review by the EMA. Navigation of the drug development pathways towards MAA approval starts much earlier than you may know till date and is essential […]
Webinar | Pharmacogenomics and Pediatric Drug Development: When Are Children Not Little Adults and Why?
This webinar covers an introduction of the intersection of ontogeny and pharmacogenomics as it relates to differences in drug biotransformation and pharmacokinetics in pediatrics. At the end of this webinar, attendees should have a better understanding of: The concept of “developmental trajectory” in the context of drug biotransformation How ontogeny and pharmacogenomics contribute to variability […]
Next Generation Strategies for Orphan Drug Development
Original Air Date: Monday, October 19, 2020 Time: 10:00 AM EDT/4:00 PM CET The past decade has witnessed a remarkable surge in rare disease research leading to an impressive pipeline of promising investigational products. Novel approaches, including genomics, data mining and computational repurposing of drugs will continue to fuel discovery and consequent clinical development of medicines. In […]
The Challenges of Cancer Immunotherapy Development and Ensuring Durability of Response
Original Air Date: Wednesday, September 2, 2020 Time: 10:00 AM EDT/4:00 PM CET Although cancer immunotherapy has entered the treatment mainstream and holds the potential for long-term durable responses, only a minority of patients currently respond to these treatments. The translation of preclinical findings to human cancer has been limiting and preclinical researchers are being challenged […]
The Value of Orphan Drug Designation in your Drug Development Program
Original Air Date: Tuesday, July 28, 2020 Time: 11:00 AM EST/4:00 PM London As a drug developer you may wonder if you have a portfolio product or development candidate that qualifies as an orphan drug and thus provides options unique to orphan drug development. There are approximately 7000 known rare diseases and more expected to be […]
Future Proofing Inflammatory Bowel Disease Diagnosis & Clinical Trials
Original Air Date: Tuesday, June 23, 2020 Time: 12:00 PM EST Inflammatory Bowel Disease (IBD) encompasses a cluster of disorders that cause chronic inflammation of the gastrointestinal tract. Crohn’s Disease (CD) and Ulcerative Colitis (UC) are the most common forms of this prototypically complex disorder that presents multiple diagnostic and therapeutic challenges. The advent of machine learning, […]