Is 'High Dollar' About to Become the New Normal in Specialty Pharmacy?

In specialty pharmacy, we are used to handling high-cost medications. In fact, a high price point is typically included in the general definition of what constitutes a specialty therapy.

However, we are rapidly approaching new heights in terms of what the term “high dollar” could mean in the current and future health care space. As some specialty therapies have cleared the 6-figure mark, manufacturers, payers, specialty pharmacies, and health systems are being forced to reinvent the wheel.

The next wave of pharmaceutical products being developed are focused on targeted gene therapies for rare disease states that affect small populations. As the therapies within this pipeline come to market, they will invariably alter the current infrastructure and could be replaced by a system that supports the dispensing, billing, and distribution of these new drugs.

Approaching the Glass Ceiling

In December 2017, Wall Street was anticipating the release of US drug maker Spark Therapeutics’ novel gene therapy Luxturna. Although the price tag was not divulged right away, initial speculation by Wall Street analysts was that the treatment could top in excess of $1 million.

The drug was approved by the FDA a month ahead of schedule and would be the first directly administered gene therapy product brought to market. Although it was hard to imagine that any drug could possibly be worth that much, it was equally hard to believe the benefits this therapy could potentially deliver. In clinical trials, Luxturna demonstrated that it was capable of reversing a specific type of hereditary blindness.

In early January 2018, Spark Therapeutics surprised the masses when the drug came in under the million-dollar mark at $850,000 to treat both eyes. Even though the actual price tag was lower than initially anticipated, it was still the highest priced therapy in the United States to date.

A first of its kind treatment, Luxturna is a one-time pharmacologic gene therapy designed to treat patients with a rare progressive form of blindness called inherited retinal disease, which is caused by mutations in both copies of the RPE65 gene. Administered by sub-retinal injection, the mechanism of action of the drug involves using a modified adenovirus to replace the defective copy of the RPE65 gene with a functional copy, thus reversing the inherent progression of blindness associated with this disease.

The therapy sounded as if it were straight out of a work of science fiction, but it was real and it was finally here. Although the launch of gene therapies provides a wave of hope, excitement, and new possibilities that were once only dreamed of, the potential of prescription drugs now shattering the $1 million glass ceiling simultaneously creates a mixed bag of conundrums.

How will the current health system sustain these exorbitant drug costs? Who should pay for such therapies? Are we treating or are we curing? What changes does this mean for the current infrastructure of our pharmacy and payer systems?

The Rundown on Gene Therapy

There are approximately 150 gene therapies currently in development. Both present-day and future applications of gene manipulation include 3 main approaches: gene replacement, gene editing, and antisense oligonucleotides or small interfering RNAs.

Luxturna is an example of gene replacement technology in which a functional copy of a gene is delivered exogenously and replaces the mutated copy. Nearly two-thirds of the gene therapy pipeline are comprised of gene replacement products.

Antisense oligonucleotides and small interfering RNAs work at the level of translation by silencing gene expression, accounting for approximately 30% of programs in development. The smallest segments of gene therapy projects are those targeting gene editing and are still in the earliest stages of development.

Gene editing involves the use of engineered nucleuses to actually correct a dysfunctional gene in situ. Seventy percent of emerging gene therapies are being tested in rare disease states with ophthalmologic, metabolic, and hematologic disorders accounting for more than half of the disease targets in the gene therapy pipeline.  

In specialty pharmacy, we should all be preparing for what that could possibly mean for our business, our systems, and our patients. With a pipeline that is bursting with novel gene therapies, one can only assume that as these entities come to market, they will boast a hefty price tag as well. Are we ready?


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