Rare and Orphan Solutions 2017-09-21T12:13:27+00:00
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Rare Diseases

Specialized expertise addresses regulatory hurdles and the challenges of small, geographically dispersed patient populations.

FPO

Rare diseases create novel regulatory and operational challenges. Precision provides an end-to-end solution. We combine highly specialized regulatory and clinical development experts in rare diseases with integrated biomarker and specialty labs and a global footprint to more rapidly find physicians and patients. The acquisition of Agility Clinical in combination with Precision’s worldwide footprint, has extended our abilities to deliver sophisticated global studies and help innovative life sciences companies achieve their clinical development goals faster. Overall, Agility has supported over 100 clients in the last 5 years.

A flexible, personalized approach that adjusts to client and patient needs. Our dedicated staff has an average of 11 years of experience. We are skilled at providing the unique support small and virtual biopharma groups need while remaining equally focused on helping patients and their families.

A pioneering technique that addresses the complex challenges of rare disease trials. By working with patient advocacy groups and key opinion leaders, we ensure that trial protocols work within the context and limitations of the patient population; define prevalence at country and site levels; and apply our specialized knowledge to identify, recruit, screen, and monitor patients across a wide geography.

A regulatory strategy that maximizes the likelihood of an orphan designation from both FDA and EMA. Our seasoned team develops a project-specific regulatory strategy by working closely with you and therapeutic area experts. We coordinate every aspect of the regulatory communications and draw on years of experience to ensure the strategy is sound and the submission is easily reviewed and and structured by regulators.

The expertise to design studies for small, highly targeted populations. Our team of biostatisticians use advanced biostatistical approaches to address the realities of rare diseases. Our statistical designs ensure proper powering to reduce regulatory risk.

Proficiency and resources that streamline all your specialty lab needs. Our broad team of geneticists, biologists, and statisticians can define your assay needs and rapidly deliver your assays and the answers needed, helping to enroll and treat patients as quickly as possible.

Education of healthcare providers, patients, and caregivers to get the right medicine to the right person. We efficiently build awareness and understanding of a rare disease, supporting short-term patient recruitment and developing long-term product demand.

For more details showcasing our rare and orphan disease solutions, visit the Agility Clinical website.

Precision Medicine Group is an integrated team of experts in fields from advanced lab sciences to translational informatics and regulatory affairs, payer insights to marketing communications. Together, we help our pharmaceutical and life-sciences clients conquer product development and commercialization challenges in a rapidly evolving environment.