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A week rarely goes by without the issue of drug prices making headlines. Despite the fact that about three-quarters of Americans reported in 2016 that they found it “very easy” to “somewhat easy” to afford their prescription drugs, several high-profile cases of alleged price gouging have kept the pressure on the entire industry to bring prices down.
Now, in the midst of this heightened scrutiny, personalized medicines and gene therapies are hitting the market — often with historically high price tags. The eye-popping costs are typically associated with the labor involved in making gene therapies, which requires that a patient’s stem cells are removed, mingled with the genes needed to address the illness, and then introduced back into the patient. Gene therapies are also being used to treat the rarest conditions, and the smaller the patient pool, the more it costs drugmakers to produce a cure. But analysts expect that as drug companies master the technology involved with gene therapies and automate some of the processes, the prices will likely come down.
It’s important to note that list prices for drugs rarely represent the actual amount paid by patients. Because of rebates, discounts and insurance involvement, the true cost is not necessarily in the price listed here.
Nevertheless, gene therapies have added a new dimension to the conversation about drug pricing, raising questions about who should pay and when it’s worth it to cure rare conditions. At the same time, the typical one-time costs of gene therapies are still no match for some of the highest-priced monthly medicines. Here’s a current look at some of the costliest, most revolutionary and in some cases, controversial, therapies on the market.
When it was being developed, investors predicted that Luxturna, which treats a rare and genetic form of blindness, could be the one of the first treatments to cost $1 million. But after being approved in December, Spark Therapeutics listed the price as $850,000. The therapy is designed to be a one-time injection into each eye. Spark also announced that it will offer rebates to health insurers if patients fail to meet certain thresholds in the months following the treatment. Only 10-20 people are born in the U.S. each year with the retinal disease Luxturna treats.
There aren’t many patients in the world who need this medication. According to its manufacturer, Horizon Pharma, fewer than 500 patients take Actimmune to treat its two intended targets: malignant osteopetrosis and chronic granulomatous disease. But for those patients, the drug comes with a price so high — $52,322 per month, or a whopping $627,852 a year — Actimmune is considered the most expensive drug in America. Horizon maintains, however, that insurance covers part of the cost and that the company assists patients who are unable to pay.
Approved in 2016, Strimvelis was the 2nd gene therapy to be available commercially. Used to target a rare immune deficiency and administered at just a single healthcare facility in Italy, the therapy was released with a listed price of $648,000. In April, GlaxoSmithKline divested its rare disease portfolio, which includes Strimvelis, to Orchard Therapeutics. So far, only a handful of patients have received the treatment.
If there’s one medication that thrust the issue of drug prices into the spotlight, it’s Daraprim. Approved in 1953, the drug, which is used to prevent infections in HIV or transplant patients and treat toxoplasmosis, did cost $13.50 per pill. Then the “Pharma Bro,” Martin Shkreli, came along and jacked the price up to $750 a pill (or $45,000 per month). As the CEO of the company that owns Daraprim, Turing Pharmaceuticals, Shkreli originally defended his decision. But after facing backlash, the company gave unspecified plans to lower the price. Yet, in many pharmacies, Daraprim still reportedly sells for nearly $800 a pill.
Like many treatments developed for rare conditions, Cinryze is the only option for patients with hereditary angioedema. To prevent attacks associated with the disease, which can be fatal, patients face a monthly $44,141 price tag for Cinryze. The drug’s manufacturer, Shire, has stated that the company prices its medications to commensurate with its value, which allows the company to continue investing in life-saving medications for rare conditions.
Continue at: https://www.pharmamanufacturing.com/articles/2018/5-of-the-priciest-drugs-on-the-market/
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