Rare Disease Clinical Trials Are Extraordinarily Complex

Expertise that accelerates therapies for rare and ultra-rare disease through the clinical development 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.  At Veristat, we have assembled a scientific-minded team of experts who have supported more than 600 studies and prepared over 70 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:     

Rare Disease Studies

> 250

Rare Cancer Projects

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Rare Genetic Disease Projects



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    Musculoskeletal 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.

"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


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:


Learn More with These Resources

Published Article

Overcoming Challenges in Orphan Drug Development

While there is no ‘out-of-the-box’ roadmap for orphan drug development, through a toolkit of innovative solutions, we can address the unique aspects of clinical development ...



The Benefits of a Central Site Model

A centralized site model allows patients to be treated at one or two central sites, which minimizes the travel burden on patients and caregivers. A combination of ...


Case Study

Keeping Up with an Over-Performing Site

Find out how Veristat was able to expertly rescue the site management and monitoring activities for a  sponsor seven months into patient enrollment at the site by ...

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