A Focus on Therapeutics for Rare and Ultra-Rare Diseases

At Veristat - Rare Diseases are the focus of our work every day

When developing a treatment for patients suffering from a rare or ultra-rare disease, every step of the clinical development and regulatory process is unique and complicated. From regulatory pathway selection to patient recruitment and site access challenges to navigating the volume of data to collect and clean - a rare disease therapy requires an extraordinary amount of care and coordination.

That is why Veristat assembled a scientific-minded team of experts who excel at supporting the development of therapies to treat rare and ultra-rare diseases across a broad range of therapeutic areas. Our trusted expertise comes from more than 760 rare/ultra-rare disease projects, 50% of these in the last 5 years, and from the preparation of 85+ marketing applications for novel treatments and cell/gene therapy cures.

Bridging the Gap from Trial to Approval for Rare and Ultra-Rare Diseases Requires Focus At Every Development Stage

We never assume we can address or solve your problem in the same way it's been done before. Our approach - marrying strategic consulting with regulatory insights and operational expertise - brings you informed decision-making and execution throughout the entire drug development journey.

Supporting Sponsors to Make the Impossible Challenges in Rare Disease Development, Possible

Get the Right Regulatory Guidance

Having a solid regulatory roadmap is critical to bringing life-saving and life-extending therapies to patients with limited or no treatment options.  Here are some thoughts to consider:

Challenge The Operational Norms - Rare Diseases Are Truly Unique

Plan, Plan, Plan Your Clinical Trial Data 

We understand the operational complexities in accessing the patients, lacking comparator/control data, and navigating the limited disease-progression knowledge.   


Trust Your Partner To Drive Your Success

Clinical trial data collection is more complex for rare and ultra-rare disease trials, especially when COVID-19 limits in-person contact with patients.

The positive impact of our bold thinking and scientific-minded approach is seen in these success stories:  


Rare Diseases Require a Bold Approach

When successfully meeting trial milestones on budget and on time can seem like almost impossible challenges for your therapy, it pays to have a trusted partner by your side.

Put Veristat’s global expertise in rare diseases to work for you.