Rare Disease
CRO Services

Rare patient 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.

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.

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.

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.

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.

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 a pharma or biotech is pioneering a new therapy, starting your HEOR planning in the early phase of your program is vital. Our HEOR team 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.

Break through traditional data silos in rare research.

Precision’s QuartzBio® Data Platform centralizes data across file types and storage solutions, revealing insights. Seamlessly link clinical data (EDC) to biomarker and pharmacokinetic (PK) data to provide your team with the ability to explore trends across subject IDs, time points, response data, and treatment groups.

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.

Advancing biomanufacturing strategy & execution in Rare Disease

Precision’s Project Farma (PF) team is a consulting firm focused on providing bespoke biomanufacturing strategy and execution plans for pharma and biotechs, advanced therapy organizations, financial institutions, academia, and more, pursuing the development of advanced therapies. Their 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 and asset management; and more.

The PF team has pioneered the industrialization of the first-ever approved rare diseases and led 50+ facility builds with a total spend of over $5B. If your team is trying to plot your biomanufacturing needs, speak with our experts at Project Farma.