Our expertise in rare disease R&D allows us to diversify our pipeline and portfolio to make an impact across disease areas with significant unmet need, using an array of innovative modalities.
With a diverse late-stage development portfolio and a robust pipeline spanning multiple therapeutic areas and modalities, we continue to do more and better for patients.
Science is complex and can be hard to translate into effective medicines. This is especially true in rare disease where many sources of information that would typically be available for more common diseases do not exist.
Developing transformative therapies for rare diseases means overcoming unique challenges and reimagining foundational elements of research and development.
Our R&D philosophy listen, understand and respond to the communities we serve. We have proven what's possible in a variety of rare diseases and we continue to advance our science to help patients and families fully live their best lives. Rooted in deep rare disease subject matter expertise, our unique R&D capabilities and approach include:
Designing research frameworks to ensure trials with small samples are both scientifically rigorous and clinically meaningful.
Building systems to collect, interpret and test disease-specific data to improve and shorten the rare disease diagnostic journey.
Designing and implementing rare disease clinical trials, including recruitment and retention within small, dispersed patient populations.
Infusing patient insights into every stage of the R&D process to ensure trials and medicines represent patients unique needs.
Leveraging long-standing relationships with academics, physicians and global health authorities to support development and approval of innovative medicines.
Establishing regulatory pathways for rare disease therapies through advanced use of real-world evidence, patient registries and natural history data.
Clinical Trials for Rare Diseases Understanding The Challenges
Why is conducting clinical trials for rare diseases difficult? Because studying a rare disease poses unique challenges and requires thinking differently.
Overcoming the Challenges: Rare Disease R&D
Fearlessly pursuing today's most pressing rare diseases requires researchers to think differently about R&D (research and development).
Learn more about our clinical trials programs
Veeva ID: GL/ALL/0055