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.
- Overcoming Challenges in Orphan Drug Development
- Considerations for Natural History Studies – How They Might Help in Rare Diseases
- Benefits of a Central Site Model For Rare Disease Trials
- Consider Adopting a Virtual or Decentralized Trial Model to Reduce Patient Burden
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.
- Virtual Or Decentralized Trials – Lessons learned for Planning and Data Collection
- Flexibility is the Key to Data Acquisition for Rare Disease Trials
- Overcoming Data Analysis Hurdles
The positive impact of our bold thinking and scientific-minded approach is seen in these success stories:
- Complex Trial for A Rare Pediatric Gene Therapy
- Supporting the First Gene Therapy Ever Approved in Europe
- Full-Service Support of a Gene Therapy Trial in North America and Europe
- Preparing Data for Rare Disease Marketing Authorization Application (MAA) on A Short Timeline
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.
