Pfizer has made a significant effort to extend the life expectancy and improve the quality of life for patients with Duchenne Muscular Dystrophy (DMD), one of nine types of muscular dystrophy. In 2017, Pfizer’s solution— a new mini-dystrophin gene therapy drug (PF-06939926)— was granted Orphan Drug and Pediatric Rare Disease Designations by the FDA and Orphan Medical Product Designation by the European Medicines Agency. The following year, Pfizer embarked upon a Phase Ib, US-based clinical study to assess the safety, tolerability and efficacy of the product.
When the first patient enrolled in the study at a clinical site in North Carolina, Pfizer had no time to waste in administering the potentially transformational drug. But the necessity of using a patient demand-led drug supply model presented several behind-the-scenes, packaging-based challenges.
Here’s how, with help from Almac Clinical Services, Pfizer successfully overcame these challenges and kept a child in urgent need of life changing treatment at the heart of its operation.