Rare Disease Clinical Development Expertise

The urgency for developing rare and ultra-rare disease treatments or cures is paramount

With over 400 million rare disease sufferers worldwide today, accelerating therapies for unmet medical needs through the clinical development process is monumental. We understand that nothing is standard when developing a therapy for a rare disease, so we have assembled a scientific-minded team of experts who have supported nearly 600 clinical programs and prepared >70 marketing applications for rare disease treatments and cures.

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.

Worried that you won’t select the right regulatory pathway, study design, or patient population? Trust the Veristat experts who have supported more than 580 clinical projects for rare disease therapies of which: 

  • Over 60% of the marketing applications we prepared are for rare diseases 
  • Over 250 oncology programs we supported are in rare cancers
  • More than 200 rare genetic disease studies 

Explore  Our Recent Rare Disease Experience


Regulatory Submission Support for First-in-Class Approval

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

"I am grateful 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


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:


Learn More with These Resources

The Time for Virtual Clinical Trials is Now

Download the infographic to learn how virtual trials are defined, the tool kits required to run a virtual trial, examples of what the patient experience can look ...

Case Study
Complex Trial for a Rare Pediatric Gene Therapy

A clinical-stage biotechnology start-up asked Veristat to run a new European trial of their complex gene therapy. Veristat experts complimented the client’s team ...

The Benefits of a Central Site Model

A centralized site model allows patients to be treated at one or two central sites, which minimizes the travel burden on patients and caregivers. A combination of ...

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