Developing a therapy for a rare or ultra-rare disease is life-changing work with unique challenges. One of the cornerstones of developing therapies for rare diseases is the use of natural history (or non-interventional) data to help inform the clinical development process, and natural history studies can also function as comparators or control datasets in marketing applications.
Veristat has completed 30+ natural history and observational trials, and specializes in the rare, complex, and novel therapies that require thoughtful planning and conduct for successful clinical development.
Our Offering
Overcoming Rare Disease Development Challenges with Natural History Trials
Rare disease development involves complex clinical program planning and complicated regulatory approval processes. Veristat addresses patient recruitment challenges, data collection and cleaning, and coordination requirements through:
Regulatory strategy development, including special designations
Disease-specific efficacy endpoint identification
Medical expert identification and global site establishment
Full-service clinical trial planning and project management
Agile patient recruitment strategies
Retrospective and prospective data collection operational solutions
Marketing application preparation
Amplify Rare Disease Research with a Natural History Trial
Data and information from a natural history study can provide an untreated, external control group for use as the comparator to the treatment group(s) in an investigational drug trial.
Why Veristat
Veristat specializes in the rare, complex, and novel therapies that demand thoughtful planning and conduct, applying that same depth of experience to natural history and observational study design. Our teams combine regulatory strategy, disease-specific endpoint expertise, and agile patient recruitment to help sponsors generate the real-world evidence rare disease programs depend on.
Natural History and Observational trials completed.
350+
rare and ultra-rare disease projects in the past 5 years.
40+
Rare Disease Marketing Applications Prepared
30+
Rare Disease Marketing Applications Approved
PROOF & RESOURCES
Learn More with These Resources
Data-Driven Strategies to Advance Development and Approval of Rare Disease Therapies
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Four Pillars of Success in Neurology Trials
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Understanding the Impact of Real-World Evidence on Clinical
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The Role of Natural History Studies in Ultra-Rare Disease Trials
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Let's Talk. Contact Our Experts Today.
We know there are always unknown challenges when bringing a novel therapeutic to market, so we've assembled an extraordinary team of scientific-minded experts who have mastered the complexities of therapeutic development, enabling sponsors to succeed in extending and saving lives.
A natural history study collects prospective or retrospective real-world evidence on how a disease progresses without intervention. For rare and ultra-rare diseases, this data helps inform clinical development and can serve as a comparator or control dataset in a marketing application.
In some cases, yes. Data from a natural history study can provide an untreated, external control group for use as the comparator to the treatment group(s) in an investigational drug trial, which is especially valuable when patient populations are too small or geographically dispersed to support a traditional control arm.
Natural history data may uncover important predictors of disease progression and help identify which patient subgroups may benefit from a particular drug trial. It can also be used to evaluate a new or existing clinical outcome assessment's ability to detect disease change.
Veristat has completed 30+ natural history and observational trials and supported 380+ rare and ultra-rare disease projects over the past 5 years.
