Basket and umbrella trial designs are becoming more prevalent in rare disease clinical trials because they can offer significant benefits in the development process; however, they also introduce challenges necessitating strategic planning and careful execution.
The diverse presentation of rare diseases complicates the standardization of endpoints, and the subjective nature of some clinical outcomes may lead to varied interpretations by different investigators. In this webinar, we explored strategic advantages and statistical considerations or benefits of choosing either a basket or an umbrella trial design, including increased research efficiencies and the ability to explore multiple therapies or rare diseases within a single trial structure.
Our team also examined adaptive medical monitoring strategies that are crucial in supporting master protocol frameworks while ensuring the trial remains flexible and responsive to emerging data, enhancing the potential for success across various treatment arms.
Furthermore, effective management practices for multi-arm clinical trials were discussed, from logistical considerations to ethical implications, with a focus on optimizing patient recruitment and efficient data collection methods. We highlighted how each trial phase could be conducted with precision and in accordance with regulatory standards.
Watch this webinar recording you will get a deeper understanding of how these advanced trial designs can be utilized not only to accelerate the development of therapies for rare diseases but also to address challenges and pave the way for improved patient outcomes.
About the Presenters
Maria-Cruz Morillo, Global Head, Allucent Rare Disease Center of Expertise
Maria-Cruz Morillo, MS, Pharmacy, has deep knowledge in drug development after 27 years of experience in Project Management and Strategy and IMP Supply in both biotech and CROs. Her strategic mindset and demonstrated ability to design and implement effective study protocols has significantly contributed to the achievement of market authorizations for numerous rare endocrinology and hematology autoimmune programs.
Milan Marinkov, Executive Medical Director, Rare Disease Medical Lead, Allucent
Milan Marinkov is an Executive Medical Director at Allucent with over 15 years of experience in the fields of clinical research and drug development coming from the industry and CROs. Focusing on rare and neuroscience diseases, Milan has played a pivotal role in the design, conduct and refinement of study protocols to optimize clinical research methodologies.
Genevieve Wills, Associate Director, Biostatistical Consulting, Allucent
Genevieve Wills has more than 11 years’ professional experience in clinical trial research. As Associate Director, Statistical Consulting, at Allucent, she is responsible for statistical consulting activities for Phases I-IV studies globally. This involves assisting sponsors to support clinical program development, protocol development, sample size and endpoint selection, regulatory interactions, exploratory analyses, scientific meetings and executive decisions.
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