Rare Disease Expertise

Veristat’s scientific-minded experts excel at supporting the development of therapies to treat rare and ultra-rare diseases, accounting for >30% of the work we do. Our trusted expertise comes from more than 850 rare/ultra-rare disease projects, 50% of these in the last 5 years, and from the preparation of  85+ marketing applications for rare disease treatments and cures.

• +300 Rare Cancer Projects
• +350 Rare Genetic Disease Projects
• +250 Rare Endo/Metabolic Projects

Planning Around Trial Complexities

Rare disease trials present additional unique challenges and opportunities. We help determine if your study qualifies for an expedited regulatory approval pathway – such as Orphan Drug Designation, Fast Track designation, or Priority Review – and we will represent you at the U.S. 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 experienced teams are poised to plan and implement these efforts quickly, providing: 

• Development of regulatory strategy, expedited pathways, and regulatory agency interactions
• Clinical program planning, inclusive of statistical planning and analysis 
• Agile patient recruitment strategies to support virtual trial and hybrid approaches
• Writing of clinical trial, safety, and regulatory documents
• Preparation and defense of Marketing Applications – NDAs, BLAs, NDSs, MAAs, jNDAs, etc.
• Collection of real-world evidence with prospective and retrospective natural history studies
• Rapid deployment of clinical trial databases
• Virtual and remote clinical and medical site monitoring
• Data analysis and migration into CDISC formats
• Post-Marketing Pharmacovigilance and Safety Monitoring to ensure patient safety beyond regulatory approval

Strategies for Success

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.
• By adding a virtual site or an at-home visit model, you can 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
• Listen to our podcast on Considerations for Developing Rare Disease Treatments to hear Veristat experts share important clinical development and regulatory planning considerations.

Fortunately, patients with rare diseases and their families are among the most involved and proactive patient populations in the world. They participate in trial design and help recruit other patients as well as build registries. Most of all, they want rare disease therapies that work, and they are encouraging sponsors to take their research to the international level.  At Veristat, we think this is a promising approach.

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

US & EU approval for an ultra-rare hematologic malignancy

Regulatory Submission Strategy and Novel Efficacy Endpoint for Treatment of an Ultra-Rare and Aggressive Hematologic Malignancy lead to approval from the FDA and EMA

Download the Case Study.

Rare Disease Indication Highlights

Our deep rare disease expertise spans across every therapy area, below is a select listing of key indications:

Find out how our clinical research expertise can help with your next study or program. 

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