Clinical trials for patients with rare, debilitating diseases who have limited mobility and greater caregiving requirements can be challenging, particularly for clinic visits and medication adherence. But, we like a challenge. In a recent clinical trial, we collaborated with the sponsor to successfully ship the study drugs directly from the depot to a sample of US-based patients with a rare, progressive debilitating disease characterized by motor, cognitive and behavioral symptoms.
In this pivotal 12-week, randomized, double-blind, placebo-controlled phase 3 trial, an updated version of existing therapy for this disease was being evaluated. The 12 weeks consisted of an 8-week titration phase with regular visits and a 4-week maintenance phase, during which the dose could be reduced at any time.
The sponsor identified a depot with experience shipping directly to the patient; the in-depot pharmacist prescribed, dispensed and shipped the appropriate medication for each patient, according to the study protocol. The initial visit was conducted at the site, and the patient and/or caregiver received instructions on how and when to take their prescription (AM and PM doses). The site entered the visit information into Bioclinica's Trident IRT system, which was an essential component of the management of this process. All subsequent visits were conducted by phone, and data entry completion triggered the dispensing process at the depot.
We successfully implemented direct-to-patient shipping for this trial, and the drug was approved by the Food and Drug Administration after trial completion. An open-label, long-term safety trial of the same drug is now ongoing. However, as with any new process, there were a number of important lessons. This process is probably better suited to smaller studies with a relatively small study population, for logistical reasons. In addition, the process should be evaluated on a country-by-country basis, including the availability and selection of an appropriate depot with a pharmacist and previous experience.
Based on this experience, we feel that depot-to-patient dispensing is a novel approach to the distribution of study drug that can respond to the subject's clinical needs regardless of whether the visit is conducted at the clinic or via telephone. However, successful implementation requires commitment from all parties, strong partnerships, clear communication and the flexibility and agility to respond to non-routine events.
For more information on Bioclinica's Trident IRT technology, please contact me at Laurel.Ferenchick@bioclinica.com.