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A Data-Driven Approach to Rare Disease Drug Development
Are you facing the unique challenges of clinical trial design for rare diseases? Disease complexity, exacting eligibility criteria, limited patient access, and accelerated timelines all create a perfect storm for drug development that requires a holistic trial support approach and dedicated expertise to successfully navigate.
End-to-End Solutions for Rare Disease Trial Challenges
Cytel is at the forefront of rare disease drug development. Our end-to-end solutions and proven expertise can guide you through the chaos of early phase orphan drug development and accelerate the delivery of life-changing drugs to market. Our global experience in regulatory frameworks helps novel and complex trials anticipate obstacles in the path to market and help you proactively overcome them.
Unique diseases present specific challenges. Our professionals are experts at compassionately and effectively structuring and executing trials by skillfully handling key obstacles:
- Small populations from which clinical trial participants can be recruited, which may be exacerbated by a reluctance of some to enroll in the control arm of the trial
- Limited knowledge of the disease’s natural history and the difficulty of defining appropriate endpoints that are feasibly measured during a clinical trial
- Minimal access to accurate information on trial characteristics and patient outcomes that can be used to estimate a trial’s success rate.
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Our Complex Trial Solutions
Traditional approaches to drug development don’t always meet the needs of those who study rare diseases. Our consultants can help you assemble the best team and methods available to fully unlock the power and promise in new therapies. Our toolkit includes:
Customer Testimonial
In biotechnology, the pursuit of treatments and cures for rare diseases stands as both a moral imperative and a scientific frontier. Each individual affected by a rare condition represents a unique challenge, yet also an unparalleled opportunity for innovation, impact, and healing. We must recognize the profound significance of our efforts, and together, we can unlock the potential to transform lives and pioneer breakthrough therapies that offer hope to those who need it most.
John Crowley
President and CEO, Biotechnology Innovation Organization
James Matcham
Vice President, Innovative Statistics
Sadiqa Mahmood
Senior Vice President, Customer Transformation
Boaz Adler
Senior Director, Global Product Engagement
Evie Merinopoulou
Senior Director, Real-World Evidence
Natalia Mühlemann
Vice President, Clinical Development
Krishna Padmanabhan
Vice President, Innovative Statistical Consulting
Patti Arsenault
Vice President, Quality Assurance
White Paper
Discover innovative solutions in clinical trial design with Cytel’s white paper, “Adaptive Design Solutions for Rare Diseases Study Challenges.” Essential for clinical researchers and biotech firms, this document addresses the unique challenges of conducting studies on rare diseases, where traditional approaches often fall short due to small patient populations.
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Claim your free 30-minute strategy session
Book a free, no-obligation strategy session with a Cytel expert to get advice on how to improve your drug’s probability of success and plot a clearer route to market.