A recent list of the top 10 most expensive drugs marketed in the United States shows each of them has a list price of more than half a million dollars annually.
As the 2020 presidential election fires up, the cost of prescription medications is already becoming a central issue, particularly after Novartis announced the list price of its one-time gene therapy treatment for spinal muscular atrophy at $2.125 million.
The cost of the treatment seems exorbitant at first glance. Current 10-year costs of SMA treatment are in excess of $4 million. At one time, it was anticipated that Zolgensma, the gene therapy treatment approved in May, could have a cost of double the $2.125 million amount announced at the time of its approval. The only other SMA drug approved, Biogen’s Spinraza, has a price of $750,000 for the first year of treatment and then $375,000 per year afterward for the remainder of the patients’ lives.
High prices are the norm for orphan disease drugs. A recent list of the top 10 most expensive drugs marketed in the United States shows each of them has a list price of more than half a million dollars annually. The list, released by GoodRx, is a comprehensive list of the most expensive drugs in the U.S. The list includes drugs that are available at pharmacies and those only administered by healthcare practitioners. The majority of the drugs on the list are used to treat rare and complicated conditions, such as different types of cancers and blood disorders. GoodRx regularly compiles a list of the most expensive drugs. This list ranks the drugs by their annual cost for a typical course of therapy, as many of the drugs are taken for an extended period of time.
The top 10 drugs and their costs are:
| Drug | Annual cost based on length of therapy |
| Zolgensma | $2,125,000 |
| Luxturna | $850,000 |
| Myalept | $778,314 |
| Folotyn | $745,785 |
| Soliris | $678,392 |
| Blincyto | $641,533 |
| Ravicti | $633,072 |
| Lumizyme | $630,630 |
| Actimmune | $575,540 |
| Takhzyro | $573,820 |
GoodRx noted that unlike gene therapies, which are, as of now, viewed as one-and-done treatments, many drugs are taken over an extended period of time, which can rack up the costs. In its analysis, GoodRx ranked the drugs based on their annual cost for a typical course of therapy. The prices are based on the list price, which is not typically the price that a consumer pays at a pharmacy or through a drug support program.
In its report, GoodRx noted that the same drugs seem to remain on the list, although new ones do appear from time to time, such as Zolgensma. An additional newcomer to the list is another gene therapy recently approved by the FDA. In December 2017, Spark Therapeutics was given the green light for Luxturna, a gene therapy for a rare, genetic form of blindness. The approval of Luxturna marked the first time the FDA approved a directly administered gene therapy that targets a disease caused by mutations in a specific gene. Luxturna was priced at $850,000 for the one-time treatment and is now the cornerstone of Roche’s $4.8 billion bid for Spark that has hit a few snags from regulators.
Coming in at number three on the list is Myalept, which has an annual price tag of $778,314. Aegerion’s Myalept, which is used to treat leptin deficiency in patients with generalized lipodystrophy, is the most expensive medication on the list that is dispensed in a pharmacy, GoodRx noted. Cancer drug Folotin comes in at number four with a price of $745,785 and Paroxysmal nocturnal hemoglobinuria treatment Soliris takes the fifth spot at $678,392.
Of the 10 drugs on the list, the cheapest has an annual list price of $573,820. Takeda’s Takhzyro (lanadelumab-flyo) was approved last year by the FDA. Takhzyro is an inhibitor of kallikrein, an enzyme which is chronically uncontrolled in people with hereditary angioedema.
The drugs that made this list differ slightly from other GoodRx lists regarding expensive treatments. Previous lists hone in on the most expensive drugs, based on list price that patients can acquire at pharmacies and self-administer. That list will not include drugs that must be administered via infusion. The top drugs on that list have typically included Horizon Pharma’s osteoporosis treatment, Actimmune and its monthly list price of $52,321. The drug that typically takes the second spot on the list was made infamous by pharma bro Martin Shkreli. Darparim, a treatment for toxoplasmosis, has a monthly list price of $45,000, GoodRx notes. That was after Shkreli jacked up the price from $13.50 per pill to $750 per pill, a 5,000% increase.
In 2017, four treatments for hepatitis C topped GoodRx’s list, Gilead’s Harvoni, Sovaldi and Epclusa, as well as Merck’s Zepatier were the top four drugs for a 30 day supply. Those drugs ranged from $87,800 for Harvoni to $52,600 for Merck’s Zepatier.
As the election season moves forward, candidates from both political parties are likely to hone in on the list prices for these different treatments to bolster arguments for various programs that could address a consumer’s concerns. While the drugs on these top 10 lists are taken by a small patient pool, GoodRx noted that the “outsized costs translate into higher premiums and more restrictive formularies for everyone.”
