Therapeutic Areas

The Science-First Full Service CRO and Consultancy for Rare Diseases

Advance Therapies for Rare and Ultra-Rare Diseases to Approval and Commercial Success.

We understand that 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 have worked on more than 40+ US and European regulatory marketing applications – 30 of which have been approved so far.

Developing a therapy for a rare/ultra-rare disease? Let’s talk.

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Clinical Trials & Consulting projects in the past 5 years

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For Rare Endocrine/Metabolic Diseases

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For Rare Cancers

Clinical Trial and Regulatory Consulting Expertise for Complex and Rare Diseases

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 FDA 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.

 

Your Global Partner in Rare Disease Therapy Development

Podcast: Clinical Development and Regulatory Considerations for Developing Rare Disease Treatments

In this episode of Advancing Revolutionary Therapies, Veristat Regulatory experts discuss the challenges of developing rare disease treatments and key considerations for planning studies for rare diseases in the U.S. and globally. Check out the full episode to learn more.

LISTEN TO PODCAST

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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: 
 
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.

Scientific Excellence Across Rare Disease Therapy Development

Rare Autoimmune 

ADA-SCID
Familial Mediterranean Fever
Graft vs. Host Disease (GVHD)
Hereditary Angiodema (HAE)
Primary Biliary Cholangitis
And More...

Neurological Disorders

Rare Endo/Metabolic 

Achondroplasia 
Adrenoleukodystrophy 
Gaucher Disease 
Lysosomal Storage Disorders 
Sanfilippo Syndrome 
And more… 

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Rare Hematology 

Beta-Thalassemia 
Hemophilia B 
Sickle Cell Anemia 
Thalassemia 
Thrombocytopenia 
And more… 

Pain Management

Rare Neurology  

Canavan Disease 
Menkes Disease 
Huntington's Disease 
Tardive Dyskinesia 
Tourette's Syndrome 
And more… 

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Rare Oncology

Adrenoid Cystic Carcinoma 
Cholangiocarcinoma 
Glioma 
Ovarian Cancer 
Thyroid  
And more… 

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Rare Opthamology 

Achromatopsia 
Choroidal Neovascularization 
Leber's Congenital Amaurosis 
Retinal Degeneration 
Retinitis Pigmentosa 
And more… 

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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

Find out how our clinical trial and regulatory experts can help with your next study or program. 

LET'S TALK

Learn More with These Resources

Newsletter

Recognize Rare Disease Month with Veristat

Case Study

Strategic Clinical Development Consulting

Fact Sheet

Adaptive Design Fact Sheet