Families Hope That Provinces in Canada Will Cover Expensive Therapies for Rare Diseases

British Columbia faces excruciating decisions about how and whether to cover drug treatment for rare diseases.

One particular drug is Spinraza.  It is a drug that is a part of a clinical trial that was used as an experimental treatment for three year old Emmett. At five months old, Emmett’s family was told he had a very severe form of spinal muscular atrophy.

He was not expected to live past one year. His disease is one that affects his nervous system -especially muscle movement.

For most of Emmett’s life, he had trouble choking on his own saliva, but after receiving the Spinraza, he was able to sit up. His family, who was initially skeptical and unsure about entering him into the trial, are overjoyed and filled with hope at the possibility of what this drug can do.

The Catch?

Spinraza cost about $750,000 just for the first year of treatment and then about $350,000 every year after.

To treat less than 30 patients with spinal muscular atrophy will cost several million dollars.

Who will pay for this medicine?


The Challenge

In the past, the pharmaceutical industry has not been particularly concerned about rare disease drugs because of the low number of patients.

However, in recent years the pharma industry has developed a strategy for the market. Most of the rare disease drugs have extremely high prices.

Obviously most families cannot pay for these medicines and sometimes there are no alternative treatment options.

Spinraza, for example, is still being reviewed to see how effective it is for people with milder cases of spinal muscular atrophy.

Potential Bankruptcy

Alan Cassels, a University researcher of drug policy, estimates that there are 16 expensive drugs on the market for rare disease that currently range from $100,000 to 3 million dollars.

Together, they could all cost the British Columbia health care system 75 million dollars per year to treat about 250 patients.

Alan Cassels says that if all the treatments were approved, it is possible that the province would go completely bankrupt.

So the vital questions is: should the medicines be publicly covered?

For families like Emmett’s, the medicine is priceless and provincial coverage may be the only way they could ever hope to afford such expensive treatment.

Emmett’s family is so grateful for Spinraza and the positive changes it has already had in their son’s health.

We are just so excited “we can have more time with him [Emmett],” says Emmett’s dad Josh.

Read the source article here.

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