Your Rare Disease CRO Partner
ARG has the agile mindset and the network of stakeholders to effectively operationalize rare disease clinical trials
We Understand Rare Disease Study Challenges:
- Does your CRO understand how to succeed in studies with limited and thinly dispersed populations?
- Is your CRO team driven by curiosity and the capacity to think disruptively to drive innovation?
- Does your CRO use tools and technologies that can be used both out of the box and individually geared towards specialized orphan projects?
- Does your CRO think about each clinical trial as its own individual project rather than a commoditized study?
- Does your CRO create and leverage a vast network of rare disease stakeholders that allow them to get things done when the next steps are unclear?
We Can Put the Pieces Together
Rare disease clinical trials require innovative thinking and savvy adjustments along the way. There is often no standard trial design or formula for rare disease study success. Rare and orphan disease trials may be rife with challenges, but they are also the most rewarding projects.
Working with a CRO with extensive experience in and an understanding of rare disease drug and device development can efficiently alleviate many of the barriers that can stand between patients and much-needed therapies. Success is determined by a group of partner experts collaborating to ask the right questions that anticipate unique problems and generate appropriate solutions both before trials start–and as they are being executed.
“I enjoy the diversity of projects we manage as a rare disease CRO. It is interesting and challenging to be on the cusp of new medical developments in treatment modalities, such as gene therapy. Our commitment to excellence, regardless of the size or scope of the trial, is what makes us successful; our desire and ability to listen is what makes us special. We listen to sponsors, research site teams, advocacy groups, and the patients themselves. We want to make a difference in the lives of these patients, but we know that first we have to understand them.”
ARG Best Practices in Rare Disease Trials
Flexibility Drives Success
- Adjusts based on unique study conditions
- Addresses challenges via anticipation and adaptation
- Collaborates to further and reshape best practices
Partnership Fuels Value
- Addresses one of the biggest challenges in clinical trials: retention<
- Provides real-time experience with patients and products
- Facilitates faster clinical development from trial to market
Tools Foster Visibility
- Integrates products and platforms seamlessly and efficiently
- Unifies best-available technologies for each functional area
- Provides project visibility with near real-time reporting
ARG Innovates and
In an ongoing ARG study of a drug for a rare eye disease in which blinding fibrous membranes grow under children’s eyelids, a small number of patients were treated at two large referral centers in the US and Europe. The first interim analysis revealed an issue: measuring membranes manually on squirming children was producing inconsistent readings between sites, and even from the same patients at different visits.