Parents are Worried That Only One of Their Two Children Will Get Access to Cystic Fibrosis Drug


A recent article in CBC News describes the heartbreak and struggle of a Canadian couple appealing to the Canadian government to fund medication for their younger son.

Both their children, Andre age 8 and Joshua age 6, have cystic fibrosis. Andre was accepted into a clinical trial investigating the drug, Symdeko. Andre has been treated with Symdeko for one year. There is a noticeable improvement in his breathing.

In that same time frame, his brother Joshua’s condition has noticeably worsened. His parents are aware that early treatment can lengthen a patient’s life span and are desperately trying to get help for Joshua. There is no cure for cystic fibrosis.

About Cystic Fibrosis

The disease is progressive and eventually fatal. It currently affects 4,200 Canadians.

The lungs are most often damaged in cases of cystic fibrosis. It is a genetic disorder affecting a protein in the body. The resulting inflammation can lead to impairment of the pancreas and lungs. Once lung function is affected, it cannot be restored. Cystic fibrosis also causes an accumulation of mucus that may lead to blockage or infections of glands, tissues, and cells.

Cystic fibrosis patients are often deprived of adequate nutrients due to malabsorption. This condition can impair growth. Additional information about cystic fibrosis is available here.

Efforts to Save Cystic Fibrosis Patients

Most of the available treatments for cystic fibrosis in Canada can only minimize symptoms. However, there are three approved drugs known to ameliorate damage to a patient’s lungs. One is Symdeko. It is being studied in the clinical trial that accepted Andre as one of the subjects. The other two drugs are Orkambi and Kalydeco.

All three drugs are not readily available to the vast majority of cystic fibrosis patients because the Ontario government does not allocate funds for the drugs. The cost of these drugs is out of reach for most patients.

Another problem is that the government of Canada does not have an efficient method to evaluate drugs that treat rare diseases. It is difficult to find a sufficient number of participants to form a well-established trial.

Therefore the burden of proof is on the investigators to demonstrate the effectiveness of the newly-developed drugs. This paradigm continues when researchers, armed with very little evidence, are trying to persuade private insurers and provincial governments to fund the new drugs.

It appears that in Canada, many quality medications are not approved for coverage because most tests are designed to evaluate drugs that treat common disorders. It is much easier to structure large clinical trials for these drugs.

Exceptional Cases Criteria (the Rules)

Funding is available for children between six and seventeen years of age who have dealt with cystic fibrosis over an extended period of time. The name for this criteria is “exceptional cases”.

The qualifications for “exceptional cases” includes a loss of twenty percent of lung function during a six-week time frame over a six month period.

According to Cystic Fibrosis Canada, about 2% of children living in Ontario who are affected with cystic fibrosis qualify for Orkambi coverage.

Three provinces require compliance with the rules. All other provinces do not provide any coverage for the drug.

The Case for Orkambi

Joshua’s doctor believes that Orkambi would slow the progression of his disease. In fact, it is believed that over one-half of cystic fibrosis patients would benefit from Orkambi. However, even if they were able to gain access to the drug they would be unable to afford its annual cost of $250,000. Most private plans will not provide coverage.

CADTH, consisting of members of the health-care and educational systems as well as the general public, determines public funding for various medications.

CADTH determined that there was a lack of sufficient evidence of Orkambi’s therapeutic value. Therefore it did not recommend that governments cover the drug.

If you enroll only a few patients with a rare disease the results will be similar. The evidence will fall short of the CADTH requirements and it will not be accepted by the government.

According to one of the directors in community relations, Canada’s system is not set up to give a fair assessment to drugs that have been developed to treat rare diseases. The director also points out that the Canadian government has not made an effort to negotiate with pharmaceutical companies. He referenced other countries where the same drugs are listed for fifty percent less than the $250,000 price of Orkambi.

One answer may be that the provinces should negotiate nationally and purchase in bulk.

The Future of Cystic Fibrosis Funding

Kalydeco was approved for coverage in Ontario just recently, but few patients qualify. Orkambi is currently under consideration in “exceptional” cases that demonstrate strong clinical evidence.

There is a slim chance that the government will add a rare disease section to its 2019 budget. This would have a major impact on the lives of cystic fibrosis patients.


Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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