Rare Disease Clinical Trials Are Complex

Bold thinking accelerates therapies for rare and ultra-rare diseases through the development and approval process

We understand that nothing is standard when developing a therapy for a rare or ultra-rare disease. Every step of the clinical development process is unique and more complicated. From regulatory pathway selection to patient recruitment challenges to navigating the astronomical 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 >35% of the work we do.

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 rare disease treatments and cures.

Rare Disease Trials are Challenging

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:     


270+ Rare Cancer Projects

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320+ Rare Genetic Disease Projects


250+ Rare Endocrine/Metabolic Projects

Strategies for Success

Given the challenges with 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.

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

Explore Our Recent Rare Disease Experience

Experience From Rare Diseases Across All Therapy Areas
Allergies Blood Cancers
Cardiovascular Disease Dermatology
Endocrine/Metabolic Disorders Gastroenterology
Genetic Disorders Hepatology
Immunology Infectious Diseases
Leukemias/Lymphomas Neurological/CNS Disorders
Nephrology Ophthalmology
Respiratory/Pulmonology Solid Tumors

First-in-Class Approval

Long-term collaboration leads to a drug approval for hATTR amyloidosis, a rare disorder.

quote-check-40-oI 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


Expedited Pathways for Rare Disease Submissions

Making informed decisions for selection of the right regulatory pathway at the start of the orphan drug development process helps mitigate risk and ensure the fastest path to approval. We specialize in rare disease submissions - more than 55% of the submission projects we've worked on were for rare disease indications.

Of the total disease rare disease submission projects that Veristat has supported, the following have an accelerated pathway:



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