Rare Disease Clinical Trial Expertise

Getting Your Novel Therapies to Patients Everywhere

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 therapy requires an extraordinary amount of coordination. 

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.


Rare Cancer Projects


Rare Genetic Disease Projects


Rare Endo/Metabolic Projects

Our Focus on Complex and Rare Diseases

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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 US 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: 

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:

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


Rare Disease Indication Highlights

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

Sampling of Rare Disease Indications
Acute Myeloid Leukemia Adrenoleukodystrophy (ALD) Amyloidosis
beta-Thalassemia Cold Agglutinin Disease (CAD) CTCL/PTCL
Cystic Fibrosis Duchenne muscular dystrophy (DMD) Ebola
Follicular Lymphoma Friedreich's Ataxia Gaucher Disease
Glioblastoma Haemophagocytic Lymphohistiocytosis (HLH) Hemophilia
Homozygous Familial Hypercholesterolemia (HoFH) Idiopathic Pulmonary Fibrosis Idiopathic Thrombocytopenic Purpura
Limb Girdle Muscular Dystrophy Type 2i (LGMD2i) Lipodystrophy Lysosomal Acid Lipase (LAL) Deficiency
Mesothelioma Multiple Myeloma Ovarian Cancer
Pancreatic Cancer Renal Cell Carcinoma Sickle Cell Disease
Sjogren-Larsson Syndrome (SLS) Tardive Dyskinesia Tourette's Syndrome
Transthyretin (TTR)-Mediated Amyloidosis West Nile Virus Yellow Fever

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


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