This Quebec ideology – a matter of principle, clearly, so don’t look for a rational explanation – also includes the astonishing fact that this is now the only Canadian province that has not instituted testing of all newborns for the presence of CF. And that is baffling in the sense that there is  clear and unequivocal evidence from countries that have implemented NB testing for CF  many years ago that this actually saves the healthcare system money in the long run,  in addition to making a significant difference to the quality of life and life expectancy of folks with CF. But I digress …

Looking at how a pan-Canadian deal for Kalydeco was eventually brought about – I can’t do much better than quoting the following from a Toronto Star article written by Rob Ferguson on June 2nd, when it referred to the Kalydeco situation as   “a political football in recent months, with the cystic fibrosis community pressing the Ontario government for help and Liberal Leader Kathleen Wynne — now in the middle of an election — saying the province couldn’t afford it without a lower price from Vertex Pharma, the Boston-based maker of the drug.” As well, the article states that: “Provincial health ministers took the issue into their own hands in April, pledging to hold a meeting with Vertex instead of leaving the negotiations in the hands of bureaucrats. That meeting was held four weeks ago in Toronto.”

Clearly, this meeting proved to be the game changer, but it would be quite unfair to blame the civil servants tasked with reaching an agreement on price with Vertex for the more than two years it took to get to this point. As we stated here earlier, in a post titled Negotiating a Price for Kalydeco, on December 21, 2013: “… they are doing so with one arm tied behind their backs.” In the absence of any kind of political will to make this happen, they were simply not a position to move their bottom line unless they were told to do so from above.

With the federal refusal to renew the Health Canada Accord and the dismissal of the Canada Health Council by cutting off its funding in 2014, the pan-Canadian Pricing Alliance notwithstanding, there simply wasn’t the organizational clout in terms of a national mandate to make things happen. What was needed, clearly, would be some leadership at the political level to break the impasse in the negotiating process and bring a pricing agreement about.

We saw one effort to provide that kind of leadership in December last year, when Nova Scotia Health Minister Leo Glavine stood up for CF patients in his province to suggest their province would go at it alone in funding Kalydeco. We can only imagine the pressure he faced from the inside when he was forced to retreat from that position on behalf of the pan-Canadian Pricing Alliance.

In the end, it was Alberta’s Health Minister Fred Horne – representing the lead province in the pan-Canadian Pricing Alliance – who pushed the deal through with Vertex after he personally invited and met with Vertex representatives and some of his own colleagues in Toronto to get the matter settled for all of Canada.

Finally, we would be severely remiss if we didn’t recognize and congratulate the Vanstone family from Beeton, ON – Beth Vanstone and her 12-year old daughter Madi with CF – for their brave and persistent fight to keep Kalydeco in the media, and for confronting and keeping the pressure on local politicians to make the life-saving Kalydeco deal happen, and not only for them, but for all Canadians!

“While we had a lengthy discussion, we have not yet reached an agreement with the
provincial and territorial governments, despite the fact that our proposal is as good as or
better than the agreements in place in 15 countries around the world where people with
CF are receiving the medicine through public reimbursement,” said Stuart Arbuckle,
Executive Vice President and Chief Commercial Officer for Vertex. “We are extremely
disappointed that today’s meeting did not result in an agreement to allow the eligible
children and adults with CF in Canada to receive this medicine through public
reimbursement. People with CF are still waiting for access to KALYDECO, and this
process is taking much longer than they expect and deserve.”

Based on the meeting, both parties have committed to further discussions this week.

In Canada, we continue to provide KALYDECO at no charge to people with CF who are
very sick as part of a compassionate use or expanded access program while we wait for
provincial and territorial governments to make a decision regarding public
reimbursement. Vertex will continue to provide the medicine to these patients while
reimbursement discussions are ongoing. Further, for the patients receiving KALYDECO
under private insurance in Canada, Vertex will continue to provide patient support
services, including financial assistance to eligible patients who need it.

KALYDECO was approved by Health Canada in November 2012.

First of all, Vertex seems to hold all the cards: they alone have the medication that – for some – can make the difference between life and death, or at least restore their quality of life to a point that they are able to live normal lives again. Mind you, a recent announcement by Galapagos NV in Belgium stated that they are about to conduct trials on a CF potentiator that is claimed to be more effective than Kalydeco. This might eventually bring some badly needed competition into the marketplace and result in more reasonable pricing for this type of medication, and CF patients in need of it might feel less like victims in a hostage taking. I know that is putting it very bluntly, but that is how many are feeling in the current situation.

As well, Vertex is not hurting for money at the moment – nor for the foreseeable future – as they have pricing agreements in place in a number countries that will guarantee them millions of dollars in revenue for some time to come, or at least for as long as it takes for the competition to enter the marketplace, but that will likely still be a number of years away. In addition, the key players at Vertex have already cashed in substantially on behalf of Kalydeco’s success, when on April 19 Vertex’ market value rose by 6 billion after positive trial data.  At least one of its executives sold shares worth more than $60 million, nearly doubling the purchase price of his share options.

Secondly – and as we have pointed this out in an earlier blog – there is the lack of political will to make this happen for Canada as a nation. A nation, I thought, that wants to be known foremost as a leader in how it looks after its citizens. With due respect to the Albertan negotiators trying to work out a deal with Vertex, they are doing so with one arm tied behind their backs.  This is so because the Harper government has all but abandoned Health Care in this country – this is not a priority for them, as clearly evidenced by a number of things, such as the refusal to renew the Health Canada Accord due for renewal in 2014, as well as the dismissal of the Canada Health Council by cutting off its funding.

It is important to note the Canada Health Accord and the Health Council of Canada were Pan-Canadian health strategies, designed to provide Canadians with equal access to the highest standards of healthcare, regardless of where they lived. As well, the Council was supposed to monitor and nurture the development and implementation of the 2004 National Pharmaceuticals Strategy that would, amongst other things:

• Develop, assess and cost options for catastrophic pharmaceutical coverage;
• Establish a common National Drug Formula for participating jurisdictions based on safety and cost effectiveness;
• Accelerate access to breakthrough drugs for unmet health needs through improvements to the drug approval process;

With the disbandment of the National Health Council,  this Strategy is now truly dead. Instead, we have what appears to be an ad-hoc agreement by the provinces, the Pan-Canadian Purchasing Alliance, and that is the body now sitting down with Vertex to decide on a price for Kalydeco.  Essentially, the provinces have been left on their own because – Mr. Harper seems to say – “Health Care is a provincial responsibility, so there – go for it, and see if you can sort these things out between yourselves. And don’t come to me for more money, as there isn’t any.”

Well, it isn’t so much that there isn’t any more money in the Tory-managed coffers, as the money they are spending elsewhere – such as in commercial development abroad – appears to flow freely: $25-million for an institute on mining and development based at the University of British Columbia; $16-million to improve environmental practices of Peru’s mining sector, and $17.4-million to “promote economic competitiveness” in mining regions to help build the businesses of small rural farmers in three areas of Peru where Canadian companies operate mines.

Not to say that these projects aren’t of any value to Canada, but you can draw your own conclusions as to the priority our federal government places on the overall Health and Well-being of Canadians.

Cystic Fibrosis Australia chief David Jack welcomed the decision but said there was still work to do before patients could use the medicine. He urged Vertex, the pharmaceutical company that makes Kalydeco, to put patients ahead of profits in its price negotiations with the government. And he said the government also needed to do the same and recognise that this was an important breakthrough treatment that should be funded.

“A positive PBAC recommendation now allows Vertex and the Department of Health to negotiate a price agreement. We have every confidence that Vertex will continue to act in the best interests of the CF community and expedite negotiations required for a timely listing,” he said. “To use a sporting analogy, while we have triumphed during the first half there is still another half to play before victory is celebrated,” he said.

Around 200 Australians who have cystic fibrosis have the G551D gene mutation that can be treated by Kalydeco.The government’s Pharmaceutical Benefits Advisory Committee which decides which medicines deserve a subsidy said yesterday it agreed the 10 per cent improvement in lung function of patients using the drug was “clinically significant and important”. It recommended Kalydeco be subsidised under the government’s Highly Specialised Drugs Program for the treatment of cystic fibrosis in patients aged six years and older.

It is one of the most expensive medicines ever approved for government subsidy and the PBAC wants a better deal on the price. Kalydeco was developed with funding from a cystic fibrosis charity in the United States and much of the basic research for the drug was performed in publicly funded institutions in the United States.

Many prominent medicos have been critical of the high price the company is charging for the medicine because much of the research behind it was funded by charities or the public.
Australia is one of the last developed nations to fund the drug.

Excerpted from http://www.news.com.au/lifestyle/health/subsidy-for-cystic-fibrosis-kalydeco-approved-by-pbac-with-conditions/story-fneuzlbd-1226787687401