Maximizing Therapeutic Success in Rare Diseases through Basket & Umbrella Trial Designs

Basket and umbrella protocol 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. This webinar will examine the 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.

The team will also explore 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 will be explained, from logistical considerations to ethical implications, with an emphasis on optimizing patient recruitment and efficient data collection methods, ensuring that each trial phase is conducted with precision and in accordance with regulatory standards.

Attendees will gain 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.

Register for this webinar to discover how basket and umbrella trial designs are transforming rare disease clinical research.

Speakers

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.

She is currently heading Allucent Rare Disease Center of Expertise and integrating cross-functional expertise to provide strategic solutions that foster successful outcomes in rare disease clinical trials and ensure excellence in delivery.

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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.

He serves as a medical team leader, overseeing and presiding over steering committees, as well as evaluating risks and safety signals in numerous clinical trials. Currently, Milan is leading a team of medics dedicated to advancing research in rare diseases. Prior to venturing into the realm of clinical research, Milan pursued a career as a clinician and holds a medical degree and specializes in neurology and clinical pharmacology from the University of Belgrade.

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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.

Prior to her work at Allucent, Genevieve was a senior research fellow (assistant professor) at the Medical Research Council at University College London with a focus on infectious disease clinical trials.

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