Objective: The high cost of orphan drugs limits their access by many patients, especially in
low- and middle-income countries. Many orphan drugs are off-patent without alternative generic or biosimilar
versions available. Production of these drugs at the point-of-care, when feasible, could be a
Methods: The financial feasibility of this approach was estimated by setting up a small-scale production
of recombinant human acid alpha-glucosidase (rhGAA). The commercial version of rhGAA is
Myozyme™, and Lumizyme™ in the United States, which is used to treat Pompe disease. The rhGAA
was produced in CHO-K1 mammalian cells and purified using multiple purification steps to obtain a
protein profile comparable to Myozyme™.
Results: The established small-scale production of rhGAA was used to obtain a realistic cost estimation
for the magistral production of this biological drug. The treatment cost of rhGAA using bedside
production was estimated at $3,484/gram, which is 71% lower than the commercial price of Myozyme
Conclusion: This study shows that bedside production might be a cost-effective approach to increase
the access of patients to particular life-saving drugs.