Rare & Orphan Disease Clinical Development Expertise

Veristat is passionate about helping clients accelerate their rare disease therapies for unmet medical needs through the clinical development process. With over 350 million rare disease sufferers worldwide today, the urgency for more treatments is paramount.  Our teams are passionate and work tirelessly to learn and become experts in each unique and rare disease indication we work in. Veristat is proud to have worked on over 300 rare disease treatments and prepared more than 35 regulatory submissions so far.

Rare Disease Expertise from Consultation to Submission

Rare Disease trials present additional unique challenges and opportunities. We help determine if your study qualifies for an accelerated regulatory approval pathway – including Orphan Drug Designation, Fast Track Status or Priority Review – and we’ll represent you at US Food & Drug Administration (FDA) and European Medicines Agency (EMA) meetings.


Veristat ensures that your clinical trial or program design supports your regulatory strategy, disease progression analytical models or previous adult trials. Our experts can help you explore and simulate adaptive trial designs. Then, we find, recruit and engage the patients and sites through to study completion. Lastly, we analyze the data and prepare your submission for regulatory agency review.

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Explore  Our Recent Rare Disease Experience

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Regulatory Submission Support for First-in-Class Approval

Long-term collaboration leads to a drug approval for hATTR amyloidosis, a rare disorder.

"I am greateful for the dedication, excitement, and clinical trial expertise that the Veristat team has given Alnylam since the start of this program. Veristat accepted our challenge and their collaboration has been critical to the successful clinical trials that led to the U.S. FDA approval of ONPATTRO."

Akshay Vaishnaw, MD, PhD, President of R&D at Alnylam

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Accelerated Pathways for Rare Disease Submissions

Making informed decisions for selection of the right regulatory pathway at the start of the orphan drug development process helps mitigate risk and ensure the fastest path to approval. We specialize in rare disease submissions - more than 55% of the submission projects we've worked on were for rare disease indications.

Of the total disease rare disease submission projects that Veristat has supported, the following have an accelerated pathway:

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Contact Veristat to Learn More

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Veristat can help you overcome the most complex challenges of conducting rare and ultra-rare disease research across a breadth of therapeutic areas - Contact us or explore more of our rare disease resources:

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