Lawyer with CF pushing for greater access to life changing drug

Chris MacLeod, founding partner at Cambridge LLP, calls Kalydeco “a game changer” and he should know — he has CF and has been on the drug since it became available in 2012. Before starting the medication, he was in the hospital for four months. He hasn’t been back since he started taking the drug.

One pill in the morning and one at night, and the drug essentially halts the progression of CF, he explains. 

“It’s a huge leg-up in the fight,” MacLeod says, who was diagnosed with the disease at age two.

People with CF have one of a few types of genetic defects, and Kalydeco works to normalize certain ones. But the drug has only been approved, and therefore is only covered under provincial health insurance plans, for one specific genetic defect despite the fact there are other mutations it is equally as effective on. It was a battle to get the pan-Canadian Pharmaceutical Alliance, the body that negotiates prices for approved medications, to move on it in the first place.

“Ultimately we had to launch a huge political campaign that got quite negative in the last political election,” MacLeod says. “You can’t say you’re negotiating but effectively be not negotiating — you need to do so in good faith and you need to get a deal done.”

Political pressure was applied, and Kalydeco was funded publically — but only for one gene pool. In 2014, the government said it would keep negotiating for access for the other genetic mutations, but then backtracked on that commitment. In November, the government announced it was halting price negotiations with Vertex Pharmaceuticals, a company based in the United States that focuses specifically on developing important medication for CF patients, without reaching a deal for CF patients with other versions of genetic defects.

“Aside from being morally offensive, there’s a constitutional argument to be made that it’s discrimination on an analogous ground,” MacLeod says, noting there is a large number of CF patients with genetic defects that would respond well to the drug, but can’t access Kalydeco because they can’t afford it without coverage.

One of the main issues is that how drugs are approved for coverage in Canada is “an opaque process,” he says.

Once Health Canada approves a drug, the company gets a drug information number and private insurers can immediately pick it up. If you want to be listed on the public formulary and reimbursed by the provincial health plan, the company needs to apply to the Canadian Agency for Drugs and Technologies in Health’s common drug review which is a panel of economists and methodologists with a few health care professionals. That panel decides whether or not to recommend the drug to the next level, to the pCPA, where that body can negotiate with the drug company on price. 

Once the pCPA negotiates the price, there’s a three-year window where provinces are free to decide whether or not they enter the agreement.

CADTH, which, according to its website, is “an independent not-for-profit agency funded by Canadian federal, provincial and territorial governments” that “supports informed decisions about the uptake and use of drugs and other health technologies in Canada,” gets “their money, their staff and their executive board of directors” from the ministries of health, MacLeod points out.

“The pCPA follows it, ministers of health defer to it and it’s an unaccountable body” — you don’t get access to information requests because it’s a not-for-profit, independent organization — but government relies on it “almost exclusively” when it comes to which drugs to fund.

“Now that’s a conflict, which they don’t see,” he says. “They view it as very independent and transparent.”

Compounding an already complicated process, despite being the most common fatal genetic disease affecting children and young adults in Canada, CF is still a rare disease in the general population and like with any other rare disease, it can be expensive to bring in medications.

The system for how Canada bargains with drug companies to get the best prices is an economic one based on large-scale purchases, such as for Tylenol or saline solution, not smaller batches of a medication that’s formulated to manage a genetic issue.

“Significant medication to address a condition — particularly a biologic which are the new precision drugs — are going to be expensive,” MacLeod says. “When you’ve got a rare disease drug, this system breaks down and we haven’t addressed that fundamental problem yet. As a result, we’re seeing discrimination between different categories of patients.”

MacLeod, a commercial litigator, says he’s fortunate his private insurance covers Kalydeco, but “that’s why I’m so committed to this. I’ve got access but we can’t leave everybody else behind.” He estimates he spends a few hundred hours a year, pro bono, engaged in “boardroom advocacy, not courtroom advocacy.”

He meets with insurance companies about coverage and advocates with pharmaceuticals to keep prices within range, develop compassionate care programs and cover the co-pay if possible. Through the CF Treatment Society, he helps “any patient that is struggling with maneuvering in the system,” such as recourse available to them after a denial of drug coverage.

MacLeod also engages in “more high-level, broad-based” advocacy for the development of more helpful policies.

“Is there a legal avenue that we could take, is there a potential Charter challenge or a constitutional argument that could be levelled? That’s one option we’re looking at,” he says. “But the big win isn’t going to be in the courtroom on this.”

Improvement will come from political pressure on government to change the way it deals with drug funding for rare diseases, MacLeod believes.

“A court action is convoluted and not ideal — but when I get Kalydeco and someone else doesn’t . . . then I think we have a problem and we need to look to legal remedies to address it.”