In 2018, Pfizer initiated a Phase Ib clinical trial of its gene therapy treatment for Duchenne muscular dystrophy (DMD). The AAV9 gene therapy, PF-06939926, is designed to provide DMD patients with a form of the dystrophin gene at the root of the condition, thereby countering the progressive muscle degeneration and weakness that characterizes the disease.
Pfizer planned to dose 15 patients with the gene therapy, which must be stored at -70°C. In light of those factors, Pfizer opted for a patient-led supply model that would only ship product once a subject was enrolled and ready for treatment.
The model made the lag between a site requesting and receiving product critical, as during that time patients with a progressive disease would be waiting for a potentially beneficial treatment. Pfizer’s goal was to package and ship the gene therapy to patients within two weeks.
That goal created challenges. Typically, the lag between the ordering and shipment of trial materials is six to eight weeks. Almac had already reduced that lead time by applying LEAN packaging and labelling principles but needed to shave a further two weeks off to meet Pfizer’s demands.
To do so, Almac’s clinical services unit developed a dedicated packaging and labelling process. The process, which Almac executed at its US facility in Souderton, Pennsylvania, resulted in a 12-day lead time for the first patient enrolled in the trial.
Almac provided the packaging specification within two business days. Pfizer granted approval in one working day. Packaging and labelling took place four to five days after receipt of the initial request.
The case study presented by Almac covers the shipment of a gene therapy to a single patient. Yet, the process it describes has broader relevance for drug developers and contract packagers in an era defined by therapies targeting small patient populations.
In the four years preceding late 2019, the number of clinical trials of advanced therapy medicinal products (ATMP), such as cell and gene therapies, increased by two thirds, according to data tracked by the Alliance for Regenerative Medicine. Many ATMPs place new pressures on supply chains, which are adapting to quickly get medicines to patients.