CYSTIC FIBROSIS: ORKAMBI - Winners & Losers. . . .

CYSTIC FIBROSIS: ORKAMBI - Winners & Losers. . . .

Postby Oscar » Fri Oct 12, 2018 7:33 pm

Picking winners & losers among CF patients

[ ... wZc.mailto ]

Ian Thompson Published: Oct 09, 2018

Twenty-five years ago, Nova Scotia’s then health minister, George Moody, became a national hero. He broke ranks with other provincial health ministers and agreed to compensate Nova Scotians affected by tainted blood. Other provinces were forced to follow.

Inspired by the advocacy of, in particular, Janet and Randy Conners, Mr. Moody said in announcing his decision: “It’s the right thing to do.”

Mr. Conners, by then dying of AIDS as a result of having received tainted blood, was finally, with his family, fairly compensated.

It’s now time for our current health minister, Randy Delorey, to be a hero. It’s time for him to do the right thing.

Cystic fibrosis is the most common fatal genetic disease affecting young Canadians. There is no cure, but in the last five years three new drugs have been brought to market that, for the first time, address, for certain sub-sets of persons with CF, the cause of CF, not just the symptoms.

These drugs represent a breakthrough of historic proportions in the fight against CF, building on the last huge breakthrough — the discovery of the cystic fibrosis gene, in Toronto, in 1989.

The first of these new drugs, Kalydeco, suitable only for persons with specific mutations of the CF gene, is already in use around the world, including Nova Scotia. A third medicine has now been approved from a safety and efficacy perspective but has yet to be submitted for provincial drug coverage.

The second drug, Orkambi, is intended for a different CF population than Kalydeco. Orkambi is intended for those with the most common mutations of the CF gene and this month the Canadian Agency for Drugs and Technologies in Health (CADTH) recommended that provincial and federal drug plans not provide coverage for Orkambi.

The CADTH decision has stunned Canada’s CF community. It flies in the face of the real-life experiences of persons with CF who are already on the drug by virtue of their private insurance or as a result of some other special access. CADTH’s ruling makes it impractical for CF clinicians to prescribe this $250,000-a-year drug to a patient who can benefit, but who does not have private insurance.

Canadians living with CF and by their care givers have told their stories to the agency but were not heard. Yet CF patients have benefited from this drug in the United States, Australia, Germany, Sweden, Austria, Luxembourg, Scotland, France, Italy, the Netherlands, Ireland and Denmark.

Canadians have reason to be proud of our health system. We’re proud to live in a country where one’s bank balance never dictates the level of care provided.

That’s no longer the case if you have cystic fibrosis in Canada.

Our governments agree to pay for those with one particular CF gene mutation — the mutation for which Kalydeco is the ideal drug. But, if you carry a different mutation of the same gene, a mutation best addressed by Orkambi, you’re out of luck.

Our governments won’t pay; in fact, they won’t even help to negotiate a better price from the manufacturer. As a CF family, you either pay the $250,000 a year or you do without, eviscerating any hope of slowing the relentless progression of this horrible disease.

Our ministers of health and their bureaucracies, as represented by CADTH, should be ashamed of themselves for effectively denying access to this medication. We say to Mr. Delorey: “Remember George Moody and do the right thing.”

The CADTH decision is not binding on Nova Scotia or any other province. Do the right thing for those with CF who could benefit from this medication. Failing that, Mr. Delorey and his counterparts will carry the burden of knowing that some Canadians are needlessly suffering, and even prematurely dying, because of this decision.

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Ian and Donna Thompson are the parents of two children with cystic fibrosis. Rob died in 1998 at 23 and Jane died in 2003 at the age of 26. Ian serves on the board of Cystic Fibrosis Canada.
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