Assessing the Competition in Rare Disease Clinical Trials: The Need for a Thorough Feasibility Strategy

When planning a rare disease clinical trial, drug developers can expect to face several challenges. In many cases, developers approach the study believing that patient recruitment will be their biggest obstacle. They are correct in making this assumption, but it is not always for the reasons they assumed.

Finding patients is, in fact, the most common obstacle experienced in rare disease clinical trials. Nevertheless, it is not always due to lack of patient presence, rather it is the culmination of several key challenges. This includes but is not limited to understanding the diagnostic odyssey, competitive study and treatment landscape, ability to operationalize at the global, regional and site level. This is where a robust, data-driven feasibility analysis becomes a necessary, key component.

This webinar will provide an in-depth perspective from the experts developing the most innovative feasibility strategies being applied to the rare diseases of today.

Speakers

Lisa Dilworth, BS, MS, Vice President Rare, Orphan and Pediatric Diseases, Synteract

Lisa Dilworth has had the honor of being named on the list of 100 Most Inspiring People by PharmaVoice.  Her breadth of experience encompasses more than 20 years of clinical research in all phases of drug development. Dilworth is a passionate collaborator committed to working directly with patients and their families, physicians, sponsors, and other key stakeholders in the rare and orphan disease space. Dilworth has extensive experience in study design, eligibility criteria, feasibility, endpoint selection, operations/project management and overall project/program strategy. She combines her previous work, as a study coordinator, neurophysiology technician with a patient-focused mindset and strong relationships with advocacy groups, to ensure that the appropriate strategies are employed to successfully execute trials.

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Rachael Young, Associate Director, Feasibility, Synteract

Rachael Young is an experienced feasibility strategist with over eight years of industry experience. She has focused on the development of complex, data-driven feasibility strategies to support robust clinical trial planning. She works in a cross-therapeutic team supporting all stages of feasibility from early pre-award study planning through to site identification, selection and initiation. With expertise across multiple therapeutic areas Young can analyse wide data sets to inform evidence-based and data-driven decision making, which ensures effective strategies are implemented bespoke to the clinical program. Young’s passion lies in rare and orphan drug development, by which understanding a multitude of unique factors is critical to overcoming study obstacles and ultimately ensuring patients gain access to these life-changing trials.

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