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 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 nearly 600 clinical programs and prepared >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 Trials

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Rare Cancer Trials

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Rare Genetic Disease Trials



Rare Endocrine/Metabolic trials

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)
Cold Agglutinin Disease (CAD)
Cystic Fibrosis
Duchenne muscular dystrophy (DMD)
Follicular Lymphoma
Friedreich's Ataxia
Gaucher Disease
Haemophagocytic Lymphohistiocytosis (HLH)
Homozygous Familial Hypercholesterolemia (HoFH)
Idiopathic Pulmonary Fibrosis
Idiopathic Thrombocytopenic Purpura
Limb Girdle Muscular Dystrophy Type 2i (LGMD2i)
Lysosomal Acid Lipase (LAL) Deficiency
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
Blood Cancers
Infectious Diseases
Cardiovascular Disease
Musculoskeletal Disorders
Endocrine/Metabolic Disorders
Genetic Disorders
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

Webinar Presentation
Webinar: Virtual Trial Planning Lessons Learned

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Case Study
Writing Multiple Marketing Applications At The Same Time

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The Time for Virtual Clinical Trials is Now

Download the infographic to learn how virtual trials are defined, the tool kits required to run a virtual trial, examples of what the patient experience can look ...

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