Orphan drug development presents unique challenges for any life sciences company. Defined as a rare disease or condition affecting less than 200,000 persons in the United States, this presents an immediate enrollment challenge due to the limited number of affected patients, compounded with at times incomplete understanding of the disease and very limited prior data available to design effective trials. Despite the smaller pools of affected patients, these drugs still require safety and efficacy regulatory approval.
As an emerging and innovative biotech company, PhaseBio has evolved in recent years with advanced pipeline growth opportunities, developing therapies with a focus on orphan-cardiopulmonary diseases. With growing clinical programs, the need for a robust eClinical platform became a priority for them; promoting better data management, and greater efficiency through all phases of their clinical studies. PhaseBio identified the need for a robust data management platform solution and outsourcing relationships that could scale up to a large, global program.