Solutions

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.

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:

Development of regulatory strategy, expedited pathways, and regulatory agency interactions
Clinical program planning, inclusive of statistical planning and analysis
Agile patient recruitment strategies to support virtual trial and hybrid approaches
Writing of clinical trial, safety, and regulatory documents
Preparation and defense of Marketing Applications – NDAs, BLAs, NDSs, MAAs, jNDAs, etc.

Collection of real world evidence with prospective and retrospective natural history studies
Rapid deployment of clinical trial databases
Virtual and remote clinical and medical site monitoring
Data analysis and migration into CDISC formats
Post-Marketing Pharmacovigilance and Safety Monitoring to ensure patient safety beyond regulatory approval

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:

Consider using a central site model to ease patient participation and travel burden.
By adding a virtual site or an at-home visit model, you can reach a broader patient pool and accelerate your study enrollment timelines.
Select the right regulatory strategies, study design, and expedited pathway(s)
Understand the specific disease progression through a natural history study to determine how that might affect study design, data capture, and safety reporting
Listen to our podcast on Considerations for Developing Rare Disease Treatments to hear Veristat experts share important clinical development and regulatory planning considerations.

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

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