Many people who live with “orphan diseases” must pay thousands or even hundreds of thousands of dollars on their medications to survive. Many of their medications have outrageous prices and currently many of the pharmaceutical companies are allowed to do this even under the Affordable Care Act (Obamacare).

One of those drugs is Procysbi. What is Procysbi? What is it used to treat? How much does it cost? Should it cost that much? These are the questions that I will answer in this post. They are also questions that are continually being asked because I cannot fully answer them.

Procysbi was created to treat nephropathic cystinosis, the “orphan disease” that I have spoken about many times in this blog. Nephropathic cystinosis is a rare genetic disorder in which the amino acid cystine rises to toxic levels, which results in irreversible tissue and organ damage if left untreated even for a short time. Cystinosis commonly affects many parts of the body including the kidneys, eyes, mouth and throat, liver, thyroid, and other organs (Orphan Disease).

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Procysbi has the same active ingredient as Cystagon (cysteamine bitartrate), which is the immediate-release from of the main treatment for nephropathic cystinosis. Procysbi has an enteric coating over the active ingredient. The active ingredient cysteamine bitartrate forces a metabolic transformation of cystine into cysteine and cysteine-cysteamine mixed disulfide compound. This creates a way for both cysteine and cysteine-cysteamine disulfide to exit the lysosomes in cystinosis patients (Morrow)

This new form of cysteamine bitartrate was only recently approved by the Food and Drug Administration (FDA) in 2013. Before Procysbi, cystinosis patients only had the choice of taking the immediate-release form which was approved by the FDA in 1994.

The new drug Procysbi, was studied in six clinical research trials. However, it was the third trial that claimed,

Procysbi administered every 12 hours was non-inferior to immediate-release cysteamine bitartrate administered every six hours (Morrow).”

The researchers found that the most common side effects of this drug were vomiting, abdominal pain/discomfort, headaches, breath odor, fatigue, dizziness, skin odor, and rash. Personally, I experience several of these side effects on a daily basis (Morrow).

On average, the price of Procysbi is $250,000 a year or a 2,400% increase, compared to the original drug, Cystagon. The average cost of Cystagon is $10,000 a year (Carroll). However, my health insurance pays $50,000 per month for Procysbi. This is probably because I take a larger dose compared to the average patient. Thus, my health insurance pays about $600,000 a year just for that one medication (Myself from my Medicare bills).

Therefore, debate has risen around the cost of this medication and whether it is ethical to charge so much. According to Christopher Starr, co-founder and chief executive of Raptor said that the price reflects that value of the drug and the need to recoup Raptor’s development costs (Pollack). However, if you look at the company’s regulatory filings, the company spent $37.4 million on research and development of Procysbi, and their total expenses in that period were $110 million (Pollack). Nevertheless, within the first year after approval experts said that they could expect sales to exceed $100 million annually (Pollack).  That has already happened and they even make more, especially from myself and my health insurance.

Unfortunately, because of the cost being so high for Procysbi, insurance companies might being to stop covering it. Even a recent survey of 50 insurers and pharmacy benefit managers by J.P. Morgan found that drugs for rare diseases are increasingly subject to scrutiny and possible restrictions on use (Pollack).

You health insurance might be paying for drugs that cost up to $50,000 if you do live with an “orphan disease.” However, it is becoming more difficult to prove the ethics behind increasing the price of a drug by 2,000%, especially when health insurance companies are beginning to look at restricting the amounts of those drugs or just not covering them. Procysbi is used to treat cystinosis, and it is very expensive. The debate continues on whether it is ethical for it to cost so much.

Works Cited

Carroll, John. “Raptor’s orphan drug-win at FDA overshadowed by debate over steep price,” fiercebiotech.com, Biotech Industry’s Daily Monitor, 1 May 2013. Web. 2 Nov. 2015.

Morrow, Thomas MD. “Do comparable Efficacy & Convenient Dosing Justify Procysbi’s Extremely High Price,” managedcaremag.com. Managed Care, July 2013. Web 2 Nov. 2015.

Pollack, Andrew. “Parental Quest Bears Fruit in a Kidney Disease Treatment,” The New York Times (2013): B1 Print.