Rare Disease Papers & Case Studies

Since 2004, ARG Has Been on the Leading Edge of Orphan Drug Clinical Trials

As a mid-size rare disease CRO, ARG has spent more than 16 years managing and optimizing rare disease and orphan drug trials. Below you will find some of our thought leadership and experience in this therapeutic area.


White Paper:
Critical Characteristics in
Identifying a Rare Disease CRO Partner

This paper is a guide to critical questions to ask and best practices to employ when selecting a CRO to operationalize rare disease clinical trials. These questions identify an ideal set of CRO characteristics that will mitigate the unique challenges of rare disease studies.

White Paper: Patient Centricity in a Consumer Model

Better-informed patient-consumers are developing and realizing their own engagement expectations on par with the other decisions and transactions in their lives: fuller individual participation in outcomes, and richer customer service experiences. 

Case Study: Factor X Deficiency

Factor X (FX) deficiency is a rare bleeding disorder where affected individuals are not able to produce normal amounts of FX, a crucial enzyme during blood coagulation that helps form stable blood clots following blood vessel damage in the body. 

Case Study: Droxidopa

As NOH 301 recruitment lagged, the FDA asked for additional data to ensure that droxidopa didn’t have a similar adverse effect as midodrine. That meant starting a new supportive trial — NOH 305 — and hiring a new CRO to launch it. ARG was selected to lead this new safety study. 

Case Study: Cinryze

For nearly three decades, people born with the rare disease hereditary angioedema, or HAE, on either side of the Atlantic were fated to very different lives. A potentially life-saving medication, Cinryze, was approved only in Europe. This left families in the U.S. with few treatment options.