Plasma-Derived Augmentation Therapy for AATD: Products and Access

In "Plasma-Derived Augmentation Therapy for AATD: Products and Access," the panel turns to the current standard of care and how clinicians explain it to patients and payers.

Dr. Haumschild introduces the current standard of care, noting four FDA-approved plasma-derived alpha-1 proteinase inhibitors given as weekly IV infusions at 60 mg/kg. He asks Dr. Barjaktarevic how he explains this class to patients and payers and which differences matter.

Dr. Barjaktarevic calls augmentation the gold standard and a lifesaving measure used for almost 40 years. He stresses that it is not conventional COPD therapy but true replacement, and it depends on plasma donation. Four products from three manufacturers are available in the United States. All contain purified alpha-1 antitrypsin at the same dose, so physicians should expect the same effect and the same ability to correct the deficiency.

The differences lie elsewhere. Products vary in manufacturing and purification, formulation concentration, and whether they arrive as ready-to-use liquid or require reconstitution. Manufacturers also recommend different infusion rates and administration times, and storage and handling requirements differ. Some features may suit home infusion better than an infusion center. He sees no efficacy or safety difference between products.

Clinical interchangeability does not mean administrative interchangeability, he explains. A patient's insurance and benefit design often decide the product, because managed care organizations have preferred products, specialty pharmacies, and site-of-care rules. These vary among private insurance, Medicare, and Medicaid, and access hoops are common.

Even so, he does not see a significant national problem with patients failing to obtain therapy. AlphaNet coordinators guide newly prescribed patients through the process and direct them to financial assistance. Manufacturers also run separate access programs. In the hands of an experienced prescriber, he says, these steps are usually handled without major delay.

In "Is the 11 Micromolar Threshold Right for AATD Augmentation?," the experts examine Dr. Kuhn's account of where the 11 micromolar target came from and whether a higher threshold might work better.