Kalydeco: What is the whole story?

The other night I caught a little foreign film in which the woman protagonist was one difficult lady to love. She had a young niece who was also damn difficult. Thrown into the mix was a boyfriend who had his own little, and so little, quirks. The movie ended on an upbeat note but with such a convoluted story it was difficult to know how all would play out in the end.

Sun Media is using the story to attack Ontario Liberals.

Now take the news. Everyday we read, hear and watch stories reported in the media. They are usually good tales with strong, neat story lines. Maybe too neat. I often have the feeling there is more to these stories than we are being told. Life is depicted by the media more as bad fiction than rich reality.

Take the story of the young Ontario girl taking the very expensive Kalydeco (ivacaftor) to treat a rare form of cystic fibrosis (CF). Sun Media reports it costs $348,000 annually in Canada for this unique drug. This is completely out of the financial reach of most people. At this point, the Ontario government is refusing to assist this young girl and her family as they struggle to cope with this massive expense.

The government has stated that it would like to help and to this end it and a number of other Canadian provinces are in negotiations with Vertex, the maker of the drug. At this point, the company and the provincial drug consortium in Ontario are at loggerheads over the price of the drug which is perceived by many to be outrageously expensive.

The kicker in this is that the genetic variation of CF the drug treats is rare. It is so rare that only about 20 people in the province would be helped by taking Kalydeco. The entire cost to the province would be less than $8 million.

Read the story at news.com.au.

What has the mainstream media left unsaid? Well to start, it is not just bureaucrats in Ontario balking when confronting the high cost of the Vertex drug. Health care bureaucrats around the world have choked when confronting the high cost.

A story similar to the one being played out in Ontario made headlines across Australia. Ellie Haikalis has the type of cystic fibrosis controlled by Kalydeco. Unfortunately, her family cannot afford the treatment and the Australian government refused to assist them.

Her mother, Rachelle, told the media  she and her husband had discussed selling their home but realized that even taking that drastic action would not raise the money demanded by the drug maker.

Almost thirty concerned American doctors banned together to pen a letter to Vertex. They wrote:

It is at best unseemly for Vertex to charge our patient's insurance plans (including strapped state medical assistance plans), $294,000 annually for two pills a day . . . This action could appear to be leveraging pain and suffering into huge financial gain for speculators, some of whom were your top executives who reportedly made millions of dollars in a single day (Boston Globe, May 29).

The dispute over the cost of Kalydeco is just one skirmish in a larger, ongoing war being fought in numerous countries scattered across the globe. But the Kalydeco story on its own is a good one. Many see the price charged for this drug as "unconscionable."

Read the fine Milwaukee-Wisconsin Journal Sentinel story.

A story in Medpage Today, Cystic Fibrosis: Charity and Industry Partner for Profit, points out Vertex developed ivacaftor (Kalydeco) "with the help of a $75 million investment from the Cystic Fibrosis Foundation -- as well as a hefty investment from taxpayers through grants from the National Institute of Health which underwrote the cost of early research."

It isn't just Yanks who have some skin in this game. There are Canadians at the table too, such as Lap-Chee Tsui and John R. Riordan of the Hospital for Sick Children in Toronto. The two research scientists, along with Francis Collins of the University of Michigan, led a team isolating the gene responsible for cystic fibrosis. Medical breakthroughs do not occur in a research vacuum.

On the upside, the drug cost isn't out of sight for everyone. Ian Smith, Vertex executive vice president and chief financial officer, exercised stock options to gross more than $60 million in one day, according to the Milwaukee-Wisconsin Journal Sentinel.

If Smith had the type of CF in question, he could live to be a hundred and never experience any difficulty in paying for his supply of Kalydeco thanks to his after tax profits from just that one day.

Of course, Smith does not have CF. The millionaire has no reason to worry. He can breath easy.