Nothing is standard when developing a therapy for a rare or ultra-rare disease. From regulatory pathway selection to patient recruitment challenges to navigating the volume of data to collect and clean, a rare disease trial requires extraordinary coordination.
Veristat's scientific-minded experts excel at supporting the development of therapies to treat rare and ultra-rare diseases, accounting for 35% of the work we do. In the past five years, our team members have supported more than 350 clinical trials and consulting projects for rare diseases, and we've celebrated 30 regulatory approvals in the past 5 years for rare disease therapies where we supported the marketing application preparation.
35% of the Work We Do Supports Rare and Ultra-Rare Diseases
Why Veristat
Rare disease trials come with unique challenges and opportunities. Veristat helps you navigate every stage, from regulatory strategy to execution:
Accelerated pathway strategy
We help determine if your study qualifies for Orphan Drug Designation, Fast Track designation, or Priority Review, and represent you at meetings with the FDA, MHRA, and EMA.
Program design
We ensure your clinical trial or program design supports your regulatory strategy, disease progression analytical models, or previous adult trials.
Regulatory execution
Our experienced teams are poised to swiftly plan and execute these efforts, with regulatory consulting advice, marketing application support, and regulatory publishing.
Cross-disciplinary support
Gain trusted strategic advice from scientific experts across all disciplines along the clinical development and commercialization pathway.
Post-market safety
We ensure ongoing safety for marketed products with post-market pharmacovigilance.
Our Offering
Clinical Trial and Regulatory Consulting Expertise for Complex and Rare Diseases
Scientific Excellence Across Rare Disease Therapy Development
Our team brings hands-on clinical development experience across six major categories of rare and ultra-rare disease.
Rare disease trials come with unique challenges and opportunities. We help determine if your study qualifies for an accelerated regulatory approval pathway, such as Orphan Drug Designation, Fast Track designation, or Priority Review, and we will represent you at meetings with the FDA, MHRA, and EMA.
Veristat ensures that your clinical trial or program design supports your regulatory strategy, disease progression analytical models, or previous adult trials. Our experienced teams are poised to swiftly plan and execute these efforts.
Reliably achieve regulatory success with regulatory consulting advice, marketing application support, and regulatory publishing. Gain trusted strategic advice from scientific experts across all disciplines along the clinical development and commercialization pathway, and ensure ongoing safety for marketed products with post-market pharmacovigilance.
Strategies for Success in Rare Disease Clinical Trials
Given the challenges of finding, recruiting, and retaining patients for rare disease trials, there are countless considerations to improve clinical program success:
Consider using a central site model to ease patient participation and travel burden.
Add a virtual site or an at-home visit model to reach a broader patient pool and accelerate your study enrollment timelines.
Select the right regulatory strategies, study design, and expedited pathway(s)
Understand the specific disease progression through a natural history study to determine how that might affect study design, data capture, and safety reporting
Fortunately, patients with rare diseases and their families are among the most involved and proactive patient populations in the world. They actively participate in trial design, help recruit other patients, and contribute to building registries.
Above all, patients' primary goal is to advocate for effective therapies for rare diseases, urging sponsors to expand their research on an international scale. Veristat sees great promise in this collaborative and encouraging approach.
of Veristat's work supports rare and ultra-rare disease.
350+
clinical trials and consulting projects.
30
regulatory approvals supported through marketing application preparation.
“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
PROOF & RESOURCES
Learn More with These Resources
Flexibility Drives Progress During a Rare Disease Gene Therapy Clinical Trial
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Data-Driven Strategies to Advance Development and Approval of Rare Disease Therapies
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Supporting Global Approval of a First-in-Class Gene Therapy
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Clinical Development and Regulatory Considerations for Developing Rare Disease Treatments
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Rare Doesn't Mean Routine. Your Trial Shouldn't Be Treated Like One.
From expedited pathway strategy to natural history study design, our team brings genuine depth across rare and ultra-rare disease. Share where your program stands, and one of our rare disease experts will follow up.
