Study of rare cancers tends to look more like rare disease research than standard oncology research. This webinar will highlight some of the challenges in designing and conducting trials for rare cancers and explore positions adopted by various authors and research consortia on how to best design and conduct these trials, including a review of pending legislation — the 21st Century Cures Act — and how it might affect rare oncology research if passed.
Considerations include recruiting patients with rare cancers, maximizing the value of data points in clinical trials involving small populations, potential use of adaptive design in these trials, and the appetite for risk among researchers and patients.
Typically in oncology trials, and similarly in rare disease trials, the investigational product is tested only on affected subjects rather than on healthy volunteers. Testing new drugs on this vulnerable population requires consideration of the study design so the fewest possible subjects are exposed to the investigational product, and so the study yields information that supports viable research decisions.
As the cost of clinical trials rises along with the cost of bringing new drugs to market, research should include alternative study design approaches and emphasize early indicators such as biomarkers, natural history data, and preclinical data. This approach also reduces the patient burden and may accelerate drug development timelines.
Sachin Kulkarni, Executive Director, Strategic Development, Oncology & General Medicine, Premier Research
Sachin Kulkarni has 14 years of experience in clinical research and project management. He is responsible for strategic planning, coordination, and providing subject-matter expertise to drive process improvement and introduce new tools that benefit internal teams and sponsors. Mr. Kulkarni oversees projects in a broad therapeutic spectrum that includes oncology, rare disease, analgesia, and gastrointestinal disorders.
Prior to joining Premier Research, Mr. Kulkarni worked at PPD Inc. as a Senior Project Manager with a focus on oncology trials. He has simultaneously led multiple Phase I studies as a project manager. Being involved with hematology and oncology trials ranging from a single institution and single geographic region to multi-institution, global studies, he has a keen understanding of trial design and operational oversight.
Mr. Kulkarni also worked as a Project Manager at Fox Chase Cancer Center in Philadelphia. He holds an MBA from the University of North Carolina at Chapel Hill and a master’s degree in pharmacy administration from the University of the Sciences in Philadelphia.
Juliet M. Moritz, Executive Director, Strategic Development, Rare Diseases, Premier Research
Juliet Moritz has worked in clinical research for more than 25 years, and her extensive background covers the spectrum from single-site studies to large, multinational trials. She joined Premier Research in 2016 to specialize in rare diseases, supporting the strategic development of products that address unmet medical needs associated with rare and orphan afflictions.
Prior to joining Premier Research, Ms. Moritz was Associate Director of Global Project Management at PPD Inc., overseeing infectious and respiratory disease research, and prior to that was Associate Director of Clinical Research at Knopp Biosciences. She held senior positions at Wyeth, Theravance, and PPD and began her career as a clinical research associate.
Ms. Moritz holds a Master of Public Health degree from the Drexel University School of Public Health and a bachelor’s degree in biology from the University of Pennsylvania. She is a member of the National Organization for Rare Disorders and the Drug Information Association.
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
for 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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