A Cystinosis Story: No Patient Should Have to Fight for Access to Their Medication


Cystinosis is a rare disease that causes cystine, an amino acid, to build up in the body’s organs. However, there is treatment that can prevent this accumulation. Normally, patients take medication to prevent the buildup in their body in addition to eye drops to specifically stop the buildup in their eyes. If the eyes accumulate too much cystine, patients can become blind.

Approximately 100 Canadians have cystinosis and 1 in every 12 Canadians has some sort of rare diagnosis.

A New Problem

Health Canada has recently approved a new formulation of eye drops (Cystadrops) which costs 103,272 dollars each year. This new formulation means patients will only have to use the drops a maximum of four times a day which could improve their quality of life. As this is now regarded as the official version, pharmacies are no longer supposed to create their own eye drops. However, these drops made in-house cost only a few thousand dollars. The price difference is far too much for the vast majority of patients and families to even fathom.

What’s truly shocking is that this has happened twice in just a two-year period for cystinosis. Some pharma company has tweaked a version of their medication and began selling it at an exorbitant price point. This new version made the old, cheaper versions either difficult to obtain or completely impossible for patients. It makes no logical sense. We would think that the approval of a new medication would always benefit patients. What is the reason for this discrepancy?

Olivia Little is one of the children affected by this change. Her family can’t afford the new version of the drops and they are at a loss for what to do. Already, her pharmacy has stopped making the drops she had been consistently using.

The Canadian Agency for Drugs and Technologies in Health has announced that they believe the Cystadrops should be covered for patients. However, this coverage is contingent on a price reduction of at least 80% by the company who created them. The company, called Recordati Rare Diseases Inc., declined to comment on why they chose the price they did. But currently the organization is in conversation with the pan-Canadian Pharmaceutical Alliance (pCPA) to discuss the price tag.

The Waiting Game 

Currently, it’s just a waiting game. Some provinces are covering the Cystadrops until a price reduction deal is made and some pharmacies are continuing to make their own unofficial drops. However, for the patients like Olivia Little who aren’t living in areas where coverage is insured, they’re at a loss for where to turn.  

“We need to provide patients with adequate medication and we should be grateful that there are companies taking that up. But it will make health care, sooner or later, unaffordable.”

You can read more about this issue here.

What are your thoughts on this story? Have you struggled to pay for your medications? Share your stories, thoughts, and hopes with the Patient Worthy community!

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