Rare disease clinical research is complicated. Running studies on small patient populations that are distributed across a wide geographic area raises regulatory hurdles, coordination challenges, and cultural barriers many other trials don’t face. At Precision, we understand precisely what it takes to run a rare disease trial because we’ve had a team whose strong focus for the past 25 years has been to execute these types of studies.
Our experience conducting 150+ orphan disease projects covering 80+ rare diseases gives us the ability to anticipate and address logistical and regulatory obstacles, while our scientific acumen generates bold and innovative solutions to rare disease technical challenges. That’s rarefied thinking, and it’s what makes us a one-of-a-kind rare disease CRO that can drive your clinical development success.
Rare disease clinical research is complicated. Running studies on small patient populations that are distributed across a wide geographic area raises regulatory hurdles, coordination challenges, and cultural barriers many other trials don’t face. At Precision, we understand precisely what it takes to run a rare disease trial because we’ve had a team whose strong focus for the past 25 years has been to execute these types of studies.
Our experience conducting 150+ orphan disease projects covering 80+ rare diseases gives us the ability to anticipate and address logistical and regulatory obstacles, while our scientific acumen generates bold and innovative solutions to rare disease technical challenges. That’s rarefied thinking, and it’s what makes us a one-of-a-kind rare disease CRO that can drive your clinical development success.
When your study is focused on a small, highly targeted population, every patient is critical. We use our deep understanding of the patient and family mindset to address each individual need and concern-reducing the burden on the patient-so patients stay in your trial and follow your protocols.
It takes a deep knowledge of the precise proof points regulators require-and how to establish clinical verification of those parameters-to streamline the ODD process. Our track record of 100% data acceptance across all orphan projects speaks for itself.
By working with patient advocacy groups and key opinion leaders, we
Maybe that’s why we’ve won the ROAR Awards “Best Orphan Drug CRO” for 2 consecutive years.
With far smaller population sizes, rare disease clinical trials produce limited evidence relative to traditional clinical trials, affecting both trial design and statistical approaches. Superlative biostatistical approaches and analytic expertise enables us to support your protocol review and study design, sample size determination, endpoint selection, randomization schedules, outcomes measures and other critical study aspects-serving as your biostatistical analysis partner.
We efficiently build awareness and understanding of your rare disease among the full spectrum of interested parties, supporting short-term patient recruitment and developing long-term product demand.
Our patient-focused approach helps boost trial enrollment and retention, which are critical to success. Ellen Morgan explains.
Our unparalleled rare and orphan disease experience spans 80+ indications covering all phases of clinical development. We pair this experience with in-house biomarker and specialty lab services that refine trial targets and speed results, as well as sophisticated expertise in adaptive clinical trials, basket studies, and early phase rare disease trials. Because every rare disease trial is unique, we deliver a customized approach from trial design to commercialization.
We specialize in developing workflows that maximize what you can learn from each sample, including:
In rare inherited metabolic disorders, predefining the best endpoint to demonstrate efficacy of a new treatment can be challenging. We worked with one sponsor who was developing such a treatment. When results from the placebo-controlled clinical trial proved unimpressive, the contract research organization (CRO) that conducted the trial, satisfied it had followed the established statistical analysis plan, concluded there was nothing further to be done.
The sponsor then turned to Precision to see what could be salvaged from the data. We recognized immediately that the study’s established primary endpoint simply was not
the best metric by which to judge success. Our task was to secure FDA approval for the drug based on the new proposed efficacy endpoint.
Find out how Precision convinced the FDA to consider a different efficacy endpoint for approval by reading the full case study.