Leveraging the Benefits of Biologic Medicines

PATIENTS DIAGNOSED with some of the most perplexing diseases — autoimmune disorders, cancer, immunodeficiencies — have few treatment options that work as well as biologics, the genetically engineered medications made from living organisms and their products, such as human and animal proteins. Typically injected or administered intravenously, biologics are by far some of the most expensive medications, costing patients upwards of $45,000 a year. According to World Biological Drugs Market 2013-2023, published in May 2013, the world biological drugs market is estimated to top $178 billion in 2017.

It’s no wonder, then, why there is such a push to develop biosimilars, the cost-effective alternatives to biologics. After all, they are predicted to cost 20 percent to 30 percent less than their reference biologic products. What’s even more important, with growing worldwide demand for biologics, biosimilars promise greater availability and increased treatment options. But, their introduction is not without challenges. In our cover feature “Future Biosimilars: Pros and Cons,” we delve into key issues surrounding the U.S. Food and Drug Administration (FDA) policymaking decisions that are at the heart of the biosimilars debate. As FDA publishes its rules regarding biosimilars, at stake is their efficacy and safety that will be determined by the stringency of testing and dispensing requirements.

Also in this issue, we highlight a few disease states that are treated with specialty plasma products. The first, primary immunodeficiency disease (PI), comprises at least 176 hereditary disorders. According to the Immune Deficiency Foundation, some 50,000 individuals in the U.S. have been diagnosed with a PI. In our Patient and Physician Focus columns, we profile Brandon Dillon who was diagnosed in 2010 with the most common form of PI, common variable immunodeficiency. Brandon discusses how he was ultimately diagnosed and how, with immune globulin therapy (a human plasma protein biologic) and great determination, he has gone from suffering recurrent illnesses to running marathons. On the flip side of Brandon’s story, Dr. Terry Harville, immunologist and leading expert in the treatment of PI, discusses why he chose this specialty and how biologic therapies have evolved over the years to allow PI patients to lead relatively normal and active lives.

PIs typically are diagnosed when individuals are age 40 and under; however, it isn’t uncommon for older adults to also be diagnosed with PIs, as well as secondary immunodeficiencies, many of which are also treated with biologics. In “Immunodeficiency in Older Adults,” Dr. E Richard Stiehm, immunologist and professor of pediatrics, outlines case studies of the diagnosis of immunodeficiencies in seven older adults and how, in many of them, biologic therapies resulted in a positive prognosis.

Lastly, our article “BabyBIG: Definitive Early Immunotherapy for Infant Botulism,” describes the 15-year effort to develop a hyperimmune globulin that spares infants long hospital stays and lengthy recovery periods complicated by serious adverse events. Discovered in 1976 as an entirely new disease pathway, infant botulism was found to infect infants lacking the adult bacterial flora to fight the disease, after they swallowed a few spores of Clostridium botulinum. Today, a single infusion of BabyBIG given to infants with laboratory-confirmed infant botulism neutralizes the neurotoxin.

As always, we hope you enjoy this issue of BioSupply Trends Quarterly and find it both relevant and helpful to your practice.