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 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.

“As a pediatrician, children are my particular passion. Being part of a process that brings hope (and hopefully successful treatments) to children who previously had no viable options is incredibly rewarding.”

“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.”

Jennifer, Rare Disease Medical Monitor
We have the Rare Disease expertise
15+ YEARS OF EXPERIENCE AS A RARE DISEASE CRO
3 MARKET AUTHORIZATIONS RESULTING IN OVER $1B OF SALES
RARE EXPERIENCE RANGE FROM OCULAR TO ONCOLOGY TO BLEEDING DISORDERS
OVER HALF OF OUR STUDIES INVOLVE RARE DISEASE
ESTABLISHED RELATIONSHIPS WITH ADVOCACY AND THOUGHT LEADERS
DEEP COMPREHENSION OF UNIQUE ASPECTS OF PEDIATRIC STUDIES

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

Flexibility Is Critical ›

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

The Importance Of Partners ›

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

Visibility Builds Trust ›

Case Study
Rare Disease
ARG Innovates and
Achieves Results

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.