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


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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)
  
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
  
Immunology
  
Blood Cancers
  
Infectious Diseases
  
Cardiovascular Disease
  
Leukemias/Lymphomas
  
Dermatology
  
Musculoskeletal Disorders
  
Endocrine/Metabolic Disorders
  
Nephrology
  
Gastroenterology
  
Ophthalmology
  
Genetic Disorders
  
Respiratory/Pulmonology
  
Hepatology
  
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

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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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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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Infographic
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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