The FDA Office of Orphan Products Development (OOPD) offers incentives for developing drugs for rare diseases. The market has experienced a massive boom between 1983 and today more than 400 drugs and biologics treating rare diseases have hit the market, which compares with fewer than 10 between 1973 and 1983.
Due to small patient populations, however, it can be difficult to recruit sufficient numbers of participants for clinical trials in one geographic area, and even more difficult to deliver trial materials to patients. With multiple sites at different locations and few patients enrolled at each, it becomes increasingly constraining to get patients involved without spending exorbitant amounts of money to run a trial.
But as supply chain executives seem to understand these difficulties and are expressing interest in increasing their spending on logistics , these investments may make the orphan market easier to navigate.
Logistics companies are also beginning to offer ways to serve isolated patients.
For instance, Marken’s direct to patient home delivery service provides delivery of clinical trial materials directly to a patient’s home, and can include full temperature monitoring from pick up to delivery, which is key for the orphan market as a number of these treatments are biologics.
Leslie Chaney, global director of preclinical and direct to patient services, told us that as the orphan market grows, the use of biologics is increasing consistently.
“Within the scope of the clinical trial setting, more companies are looking at solutions to increase enrolment rates and ease of use for participants. We’ve developed an innovative approach with direct to patient, and can work within the protocol to deliver treatments to patients directly to their home,” Chaney said.
Direct to patient services can decrease the number of times a patient has to travel to a site and increase the convenience factor for the patient, she added, noting that not a lot of these direct to patient services have been offered in clinical trials so far.
But these services will also increase clinical trial costs, Chaney said.
“Each company needs to consider the value of some of these intangibles versus the added costs of delivery fees,” she said. There are other providers that are looking at this potentially as it “makes a lot of sense in orphan drug market,” particularly for oncology patients or others who may have difficulties traveling, she said.