Rare disease drug development is evolving, driven by innovation and regulatory advancements. With many devastating rare diseases still lacking approved treatments, regulatory agencies are taking significant steps to support innovators, advance research and improve patient outcomes. Through updated and newly developed guidances, they aim to provide a roadmap for navigating the complexities of this specialized field.
In this webinar, they will highlight key regulatory guidances published within the past year, addressing artificial intelligence (AI), accelerated approval pathways and fostering diversity, equity and inclusion (DEI) in clinical research. Attendees will gain insights into the challenges these guidances aim to address, their potential to transform the rare disease landscape, and strategies for incorporating any necessary changes into drug development programs.
Register for this webinar to explore how the rare disease landscape is changing with guidance from AI, accelerated approval pathways and fostering DEI in clinical research.
Speakers
Kenneth Ndugga-Kabuye, MD, FACMG, Vice President, Cell and Gene Therapy , Premier Research
Dr. Ken Ndugga-Kabuye is Vice President, Cell & Gene Therapy at Premier Research. He brings direct experience supporting drug development candidates for clinical studies leveraging innovative approaches in the treatment of disease, inclusive of cell and gene therapies, synthetic biotics, RNA therapies, and enzyme replacement therapies.
In previous roles, Dr. Ndugga-Kabuye has supported CRO and biotechnology clients in the ATMP space. Dr. Ndugga-Kabuye is a clinical geneticist with residency and fellowship training in medical genetics and genomics from the University of Washington in Seattle, WA, US. He is board certified by the American Board of Medical Genetics and Genomics in Clinical Genetics and Genomics. He holds a medical license in Washington State and California. He is a Diplomate of the American Board of Medical Genetics and Genomics. He is a member of the American Society of Human Genetics (ASHG), the European Society of Human Genetics (ESHG), the American College of Medical Genetics (ACMG), the African Society of Human Genetics (AfSHG), the American Medical Association (AMA) and the American Academy of Pediatrics (AAP).
Message Presenter
Paulla Dennis, Executive Director, Program Strategy, Rare Disease, Pediatrics, Cell and Gene Therapy, Premier Research
Paulla Dennis has been dedicated to rare disease and pediatric clinical research for more than 25 years, specializing in gene therapy for the last 10+ years. She is highly experienced in operational delivery after spending 17 years at a pediatric hospital running the Clinical Research Services group, where she delivered pivotal studies in several rare pediatric disorders inclusive of gene therapies. Paulla has an additional 8 years’ experience in the CRO setting in startup project management as well as in program strategy, delivering rare, pediatric disease and advanced therapies studies.
As Executive Director, Program Strategy, Paulla advises, consults, plans and directs the design and strategy for prospective and ongoing rare, pediatric and gene and cell therapy studies. In her current role, she provides strategies and solutions for operational excellence in this complex clinical research setting with a focus on patient and family pathway planning and comprehensive site assessment and educational support.
Paulla has extensive experience in phase I-IV studies in a global setting, including autosomal, neurological, metabolic and endocrine diseases. Her key experience areas include rare/ultra-rare disease, pediatrics, gene therapies and neurological (neuropsychological, neurodegenerative and neuromuscular) indications.
Message PresenterWho Should Attend?
This webinar will benefit Managers and above at biotech and specialty pharma companies with job functions including, but not limited, to:
- Drug development
- Clinical operations
- Medical affairs
- Regulatory affairs
- Project management
What You Will Learn
Attendees will gain insights into:
- The rare disease landscape, including 2024 approval trends
- New guidance and their impact on rare disease clinical research
- Study design strategies to ensure efficiency and compliance
- A medic’s perspective on what the changes will mean for patients and investigators
Xtalks Partner
Premier Research
Premier Research, a global clinical research, product development, and consulting company, is dedicated to helping innovators transform life-changing ideas and breakthrough science into new medical treatments. We offer strategic solutions across the entire development lifecycle, from pre-clinical through commercialization, specializing in smart study design and full-service clinical trial management.
Leveraging technology and therapeutic expertise, we deliver clean, conclusive data with a focus on reducing development timelines, securing access to the right patients, and effectively navigating global regulations to ensure submission-ready results.
As an organization that puts patients first, we pride ourselves on helping customers answer the unmet needs of patients across a broad range of medical conditions. Visit premier-research.com.
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