Rare disease CRO services, deep lab experience and a passion to help patients in need

Rare disease research is our passion and complexity, our playground. 

From ultra-rare and pediatrics to cell and gene therapies, our extensive experience running rare disease clinical trials requires a personalized approach to each study, bringing your project from research to realization. 

Whether it's a child's first chance or a family's last resort, patient outcomes are the heartbeat of our passion. Across each operational stage, we are an extension of your team, delivering the right skill set to the right role for your study's unique needs.

CRO Trial Services >

Generating rich biomarker data to maximize rare disease insights. 

Every clinical sample in a rare disease trial is precious. Precision's expertise in developing complex assays for any tissue type—and the global footprint of labs to ship and process samples from any clinical trial location—are ideally suited to support the needs of rare disease trials.  

Our comprehensive approach starts with a foundation of expertise in key biomarker discovery and analysis methods, such as immune monitoring, genomics, and bioanalysis. We then specialize in developing new technologies and customized approaches for biomarker assay to generate rich, multi-omic biomarker data. 

Specialty Labs >

Rare populations face uncommon challenges.  

95% of rare diseases have no available treatments. Finding and supporting rare patients requires dedication and persistence, especially when researching the depths of rare and ultra-rare indications. Collaboration and creativity are not optional; they're required.  

50% of rare disease impacts pediatric populations. Fielding patient and caregiver-centric solutions to maximize awareness and participation is a must. Finding and communicating with social and advocacy groups, building study portals, leveraging decentralized clinical trial (DCT) strategies, and coordinating with concierge travel services are often the essential elements required in rare research. We have a long and successful history of crafting rarefied solutions to make rare research a possibility for the patients and sponsors we serve.   

Rarefied solutions for sites studying rare diseases.

For investigators, participation in rare disease clinical trials can increase daily demands, staff expectations and create logistical challenges.

Providing solutions and tools to ease study participation, support patient education, enrollment, and patient retention is critical to your study’s success. Rare clinical trials require specialized tools and solutions, including:

• Proven decentralized strategies tested in rare environments
• Genetic counseling support and mining of database networks
• Crafting custom patient engagement campaigns for rare, orphan, and ultra-rare diseases

Simplify rare clinical sample management and gain richer insights.

Every clinical sample in a rare disease trial is precious. Ensuring proper collection, processing, logistics, and storage of these samples requires a central lab services approach. One tailored to meet all sponsor needs, including custom kit production, rapid global sample processing, and more. Learn about Precision’s harmonized approach, featuring our virtual Sample Inventory Management solution for monitoring samples and derivatives across multiple sites, labs, courier services, and repositories with 24/7 access to shipment tracking and sample status.

Central Lab Services >

Navigate regulatory pathways with rare efficiency.

The rare disease regulatory environment is nuanced and often competitive, with peers also targeting your rare indication. We understand how to optimize your clinical development plan strategy in coordination with your planned regulatory pathway.

With the right combination of regulatory, medical, biometric, and translational consultancy, we leave nothing open to interpretation.

Regulatory Services >

Efficient and effective data collection solutions to support rare research.

Despite small patient populations, rare clinical trials generate large and complex datasets.

From unique endpoint support to incorporating intricate data streams from numerous qualified vendors, Precision’s rarefied experts craft custom clinical data management solutions that are creative, efficient, and effective, meeting the needs of patients, sites, and sponsors.

Clinical Data Management >

Extracting rich insights from rare study data.

Put your project on the pathway to approval. From designing statistical solutions to analyzing the results of studies with small sample sizes and unique endpoints, rare data sets are our specialty.

Across rare, orphan, and ultra-rare indications, our biostatistical experts can help to define rare endpoints, assess the inclusion of specialized biostatistical techniques, identify surrogate endpoints, and more.

Biostatistics Services >

Advancing biomanufacturing strategy & execution in Rare Disease

Precision manufacturing consulting is focused on providing bespoke biomanufacturing strategy and execution plans for pharma and biotechs, advanced therapy organizations, financial institutions, academia, and others pursuing the development of advanced therapies.

Services include:

• turnkey capital projects tech transfers, and facility builds
• owner’s representation
• program/project management
• validation
• quality, regulatory, and compliance
• engineering and automation
• serialization track and trace
• reliability and maintenance
• asset management and more

Precision has pioneered the industrialization of the first-ever approved rare diseases and led 60+ facility builds with a total spend of over $6B. 

Manufacturing Solutions >

Establish evidence for rare development and commercialization.

Precision’s HEOR team has a long history of success supporting value demonstration for rare disease investigational products that are often first to market. In rare diseases, it’s even more critical to generate strategic, innovative, credible, and relevant supporting evidence for 5 successful commercialization.

When pharma or biotech Sponsors are pioneering new therapies, starting HEOR planning in early phase is vital. Precision provides strategic planning support along with specific research services including systematic literature reviews, network meta-analyses, economic models and microsimulations, and analysis and generation of real-world evidence.

HEOR Services >