One of my medications, Procysbi costs $63,000 for a 30-day supply.

I live with cystinosis, a very rare orphan disease that causes the amino acid cystine to accumulate in the cells. It slowly damages all my organs including the kidneys, liver, thyroid, eyes, lungs, muscles, and brain.

The primary medication to treat cystinosis is Procysbi. It has the same active ingredient as Cystagon (cysteamine bitartrate), which is the immediate-release form of the primary treatment for cystinosis. The difference is that Procysbi has an enteric coating over the active ingredient. This new form of the drug was only recently approved by the Food and Drug Administration in 2013.

In 2015, the average price was $250,000 a year which is a 2,400% increase compared to the original drug, Cystagon (1).

Unfortunately, because of the high cost of this medicine, insurance companies fight not to cover the drug, and I am left without the drug sometimes for weeks. A recent survey of 50 insurers and pharmacy benefit managers by J.P. Morgan which found that medicines for the rare disease are increasingly subject to scrutiny and possible restrictions on use, as mine are (2).

The Affordable Care Act does not come close to combating this problem of skyrocketing medication prices and the prices of medical care. It does, however, provide that everyone is guaranteed health insurance.

Let’s demand that Congress enact regulations that control the costs of prescription drugs and even lower the cost of these drugs.

Sign this petition calling on Congress to act: Sign here

1.  Carrol, 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

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