At the age of three, John Rae was diagnosed with a rare neuromuscular disorder called congenital myasthenic syndrome -- a condition that made moving hands, feet and legs almost impossible.
But a medication called 3,4 DAP changed his life.
“If I don’t have it I can’t move. I can’t walk, I can’t keep my eyes open. Just to get out of bed I need to have these pills,” explained John, who is now 21. The drug improves signals between nerves and muscles.
For decades, Jacobus Pharmaceutical, a small family-owned American company, supplied 3,4-diaminopyride (3,4 DAP) for just $100 a month in Canada. In the United States, it is free. The drug was never formally approved for sale but was provided through a compassionate care program. Because “it was the right thing to do,” the company said in a statement.
Now a rival company Catalyst Pharmaceutical applied to the FDA to sell their new, unrefrigerated version of the drug called Firdapse. If successful the company will get exclusive rights to sell the drug for seven years. Jacobus will be shut out.
There are fears that the company will hike the price to over (according to some reports) US$60,000 a year, putting it out of reach for some North American patients living with this condition. There are an estimated 3,000 patients.
In “Muscle & Nerve,” 80 concerned doctors wrote about the “potentially harmful price increase” and asked for “ethical and just pricing.”
“The main thing we can do is to ask companies to consider fair pricing and that is what my colleagues in the United States have done through an editorial,” said Dr. Hans Katzberg, John’s neurologist at University Health Network in Toronto.
In Europe, where the drug is already for sale, the price shot up to US$60,000 a year when it had been previously only US$1,600.
“Knowing what has happened in Europe with the drug costs or other drugs where prices have been raised exorbitantly,” Katzberg said, “we worry about that for drugs that are used for rare diseases.”
“If the price did go up for this particular medication and there were no other options, then (John) would not likely be able to afford his medications,” he adds.
John says paying for the low-cost version is a struggle. A higher price would be out of the question.
“I definitely would not be able to afford the drug at all -- people have been throwing around $60,000 to $80,000. That alone is more than I will be able to make in a year and that is what it will cost for my medication for a year. I won’t be able to have any life outside of trying to pay that off,” said John.
“I am angry at the company that is going to raise the price on this drug. If they do that, they are ruining lives just to make more money and it should not be about that, drugs should be about helping the people that need them and help them get access to that drug,” he said.
In latest development, Jacobus began the process of getting their drug approved for sale by the FDA. If they get approval, they say their company committed to fair pricing.
It is news John welcomes. “My hope is that the drug is affordable. That is all I needed -- for that drug to be available to me so I can lead my life as I need to and want to.”
