Study of rare cancers poses special challenges for drug developers, who often must draw on their experience in both oncology and rare disease. Current strategies and processes for general oncology drug development don’t always apply to rare oncology, a field that today accounts for up to 20 percent of new cancer diagnoses.
To optimize drug development, study designs should maximize the percentage of patients on effective treatment and minimize overall sample size to limit patient exposure to drugs or doses that have no effect. This can be achieved through use of adaptive design techniques and more rigorous oversight of patient eligibility to define the optimal trial population.
Researchers should consider alternative statistical principles — for example, estimating survival when further lines of therapy are anticipated — along with interim analyses and proper futility management.
Biomarkers and surrogate endpoints of efficacy, such as response duration and progression-free survival, also need special consideration and may be required as part of the regulatory review process. Collecting information beyond survival is becoming more important, with payers increasingly requiring quality survival be demonstrated. Development plans should encompass expanded access to bridge the gap between clinical trials and market approval.
New regulatory pathways and processes are expediting the translation of novel therapies to the bedside, and understanding these options helps ensure availability of more new treatments while minimizing delays.
In this webinar, we will:
- Discuss important aspects of rare oncology, including trial design and execution, market exclusivity, and today’s regulatory environment
- Examine trends in trial design, including use of the continual reassessment method in Phase 1, use of adaptive design Phases II and III, statistical implications, planning for expanded access, and capturing market access outcomes
- Review regulatory pathways in rare oncology: breakthrough, accelerated/conditional approvals, and orphan designation
Peter Larson, Senior Medical Director, Hematology-Oncology, Premier Research
Peter Larson, M.D., is Senior Medical Director for Hematology-Oncology. Dr. Larson supports the drug development work of the innovative biotech companies that comprise most of Premier Research’s customer base, bringing to the role extensive clinical and medical affairs experience.
With a background spanning large pharma, startup biotech, and contract research, Dr. Larson was Senior Director of Clinical Research at pharmaceutical start-up Chimerix Inc. before joining Premier Research in 2016. Prior to that, he was Senior Director of Oncology Global Medical Affairs at Novartis Pharmaceutical Corporation.
Dr. Larson also held senior positions at F. Hoffmann-La Roche AG, where he led the company’s global virology franchise, and Bayer Corporation, where he was head of global clinical strategy. He also was Assistant Professor of Pediatrics at the University of Pennsylvania. Dr. Larson’s therapeutic expertise includes hematology, oncology, hemophilia, mastocytosis, AML, ALL, multiple myeloma, GIST, and virology.
He holds a Doctor of Medicine and a bachelor’s degree in biology from the University of North Carolina and is a fellow in transfusion medicine, blood banking, and hematology at University of North Carolina Hospitals.
Colin Hayward, Chief Medical Officer, Premier Research
Dr. Colin Hayward is Premier Research’s top medical expert, providing global leadership for patient safety, scientific and ethical governance, and delivery of exemplary customer service.
Possessing a rare combination of business and scientific expertise, Dr. Hayward entered the pharmaceutical world with a focus on anesthesia and intensive care after a career in hospital medicine. Using his expertise in local medical affairs and pharmacovigilance, Dr. Hayward became International Medical Leader at Roche, developing innovative medical marketing strategies for products in supportive care, anemia, and oncology.
In 2007 he joined the board of Prism Ideas Ltd. After helping grow Prism and earning a Queen’s Award for business, he came to Premier Research in 2012 as Vice President of Medical Affairs, providing round-the-clock medical support to ensure that customers’ multimillion-euro drug development projects met their goals and adhered to stringent safety rules. He left the company to serve as European Medical Director for Myriad Genetics and returned in 2015 to assume the newly created Chief Medical Officer position.
Dr. Hayward holds a degree in medicine from Guys and St. Thomas’s Hospitals, London.
Who Should Attend?
VPs, Directors, Managers, Department Heads, Scientists and Researchers working within:
- Clinical Affairs
- Clinical R&D
- Clinical Research
- Clinical Pharmacology
- Clinical Operations
- Project Management
- Regulatory Affairs
- Medical Affairs
from pharmaceutical, biotechnology and medical device companies.
Premier Research is a leading CRO serving highly innovative biotech, pharmaceutical and medical device companies. The company has a wealth of experience in rare disease and pediatric research having managed about 100 projects in each area in the last five years alone. Its services include clinical research and regulatory outsourcing in the areas of analgesia; neurology; infectious, cardiovascular, and respiratory disease; dermatology; oncology; and medical devices.
Premier Research and operates in 84 countries. It employs 1,000+ clinical professionals dedicated first and foremost to fulfilling each customer’s requirements in a timely, accurate, and cost-effective manner. This includes a strong international network of monitors and project management professionals combined with regulatory, data management, statistical, scientific, and medical experts, and staff at its well-established network of dedicated clinical sites.
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