California-based CRO Agility Clinical and EU-based PSR Orphan Experts have partnered to allow orphan drug companies to utilize their safety and regulatory knowledge in both markets.
“Each company has expertise in its particular geographic region -- the US for Agility and Europe for PSR -- and there are differences in the regulatory requirements in North America and Europe,” Dr. Marilyn Carlson, vice president of medical, regulatory and scientific affairs, told Outsourcing-Pharma.com. “The partnership will provide biopharma and device companies developing orphan products with access to this combined knowledge base, and allow them to leverage our combined experiences.”
Orphan drugs are used to treat rare disease, which in the US affects fewer than 200,000 Americans, or less than one in 2,000 Europeans.
“With few CROs focused on rare diseases, partnering with another like company to share knowledge and experiences strengthens our services to clients, and our reach,” Ellen Morgan, Agility CEO said.
The partnership also will benefit clients “who wish to run international clinical studies, and more importantly, it will benefit patients in need of treatments for rare diseases,” Dr. Carlson said. The US NIH (National Institutes of Health) estimates there are about 6,800 rare diseases for which no treatment is currently available. Nearly 30 million Americans, almost 1 in 10, are affected by a rare disease.
“Given the current challenges in orphan drug development, it is of crucial importance to provide innovative and solid infrastructure and expert services,” said Roger Legtenberg, CEO of PSR. The company has previously worked on projects to develop a treatment for the rare genetic disorder MELAS (mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes).
Agility, meanwhile, explicitly serves small and virtual biotech and medical device companies through consulting and various other research-related developments. The firm’s senior staff also volunteers with its partner, the Public Health Alliance for Clinical Trials (PHACT), a non-profit CRO conducting clinical trials for products to treat diseases that affect the poorest people in developing countries.