- Leverages an ever-expanding network of relationships
- Demonstrates the capacity to be effective when no blueprint exists
- Adapts to unique study circumstances
ARG has “been there and done that” when it comes to operationalizing rare disease clinical trials. We have experience in several therapeutic areas, which of course is very valuable. But even more importantly, we know how to think about each project individually, and plan each study for optimization. These qualities have allowed us to take on and deliver more diverse and sophisticated projects over time.
ARG’s evolution is much more profound than the fact that our company size has dramatically grown since our 2004 founding. Our maturity can be seen in creating deep relationships with a vast network of rare disease stakeholders, which allow us to get things done with no preexisting roadmap and adapt to unique study circumstances.
The ARG SAB (Strategic Advisory Board) regularly convenes to discuss emerging trends and issues in rare diseases, adding to the spirit of innovation that is already present in all of our functional areas.
Our partnerships extend through the rare disease space all the way out to patient advocacy groups. ARG drove the success of now FDA-approved Cinryze® because of an exclusive relationship with the U.S. Hereditary Angioedema Association (HAEA), using their patient registry to augment our internal search. Faster enrollment equated to first-to-market and over $1B in sales.
ARG also knows how to navigate environments where there is no established regulatory pathway. We were able to succeed with a program in neurogenic orthostatic hypotension, an orphan indication with a minimally effective standard of care, to get FDA-approval of NORTHERA®. We used our experienced team members and our collaborative approach to get things done.
We also adapt by thinking disruptively– and at times, turning the study start-up around. In one recent trial, we felt that traditional patient recruitment would work best in reverse and employed a “patient-hunter” model. Instead of the traditional site-then-patients approach, we identified patients initially via specialty pharmacy and other channels, then opening qualified investigative sites around them. The result: patients were recruited in nine months versus the planned 24.
At ARG, our big-picture experience has led to consistent operational success in managing individualized rare disease studies.
(Photo by Edward Cisneros)