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.

“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 at ARG. 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
Jennifer, Rare Disease Medical Monitor
We Have The Rare Disease Expertise







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

Case Study
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.

The Atlantic Research Group Difference
Technology and Customer Service

ARG is able to offer optimal value because we combine the best of all offerings from all sizes of CROs: a full suite of services including the latest technologies featuring integration, automation, and data visualization — along with the same individual attentiveness we have offered since 2004. We achieve all of this via efficiency, operating at a very high level of sophistication through the use of cutting-edge technologies.

We specialize in a total team approach to meeting and exceeding the requirements of clients and have been remarkably effective in consistently delivering on deadlines for clinical trials. Our high-performance rates have resulted in receiving multiple repeat-business opportunities.

The Role of CROs in Rare Disease Trials

We know that a CRO is an essential tool in bringing a new drug to market in today’s demanding world of orphan drug development. While the global patient pool is small for each investigational product, the impact of its potential use is immeasurable.

It is impossible for large pharmaceutical companies to dedicate valuable in-house lab space and staff for every phase of the drug and device development process, so using ARG can lead to remarkable reductions in the time required to achieve final regulatory approval. This is because we assign high visibility and importance to each and every one of our projects, regardless of the size of the Sponsor.

Global Reach, Global Perspective

As rare diseases span the globe, so must the research that results in successful treatment for them. From North America and South America to Europe and Asia, ARG has continued to extend its reach. In Europe, we have partnered with other CROs in order to maximize and consolidate our clinical trial opportunities. As a result, we have access to a wide network of sponsors, partners, clinicians, lab professionals, and most importantly patients.

Industry-Leading Tools and Applications

The rare disease clinical trial space demands total command over project platforms, and we continually demonstrate the capacity to not only build and utilize individual pieces but bring them together in ARG EAI (Enterprise Application Integration). We integrate a suite of platforms and services that best fits the needs of our sponsors, from eTMF to EDC to Safety to Quality Assurance. Beyond these, we use SAS to analyze data and Spotfire to create easy-to-use sponsor dashboards.

A Strong Legacy of Orphan Drug Success

ARG’s primary goal is to improve and prolong the lives of the people affected by rare diseases through the clinical trials we manage. One notable example of the many ARG clinical trial success stories is a C1 inhibitor developed to treat hereditary angioedema (HAE). HAE symptoms include edema (swelling) in the hands, feet, face, and throat; in addition, patients often have abdominal pain, nausea, and vomiting caused by swelling in the intestinal wall. Before this therapy became available in late 2008, the mortality rate caused by throat swelling was believed to be as high as 30%.

ARG managed the pivotal clinical trials that led to the FDA and subsequent international regulatory approvals as well as the global market release of this C1 inhibitor. We were grateful for the opportunity to make an impact on lives.

Over time we continue to realize our patient-centric approach by carefully cultivating relationships, fostering meaningful communication, and anticipating and addressing all of the challenges in our studies. ARG is rare for a reason: we successfully operationalize orphan development projects because we believe everyone deserves to be well.