Picture of Nigel RawsonNigel Rawson is President of Eastlake Research Group

Picture of Donna Lawrence


Donna Lawrence is Director, HTA and Health Economics, with PDCI Market Access Inc.


You’ve been diagnosed with a rare disorder. It’s probably taken several referrals to many different medical specialists, countless tests and some misdiagnoses for you to get here because it frequently takes years to reach a correct diagnosis. Now what? Is there a medicine available to reduce your suffering or extend your life?

The odds are against you because fewer than one tenth of the estimated 7,000 rare disorders have an effective treatment. However, more and more drugs for these disorders are becoming available. You’re lucky – your physician tells you that a safe and effective drug has been approved by Health Canada. But, accessing it will likely mean travelling the Beatles’ long and winding road, surmounting obstacles along the way.

You may be fortunate and have private health insurance that covers some of the cost of treatment. However, the drug is expensive and your policy likely has a lifetime cap, so coverage will only be provided for a limited time. After that, you have to pay, which may be impossible on your income.

If you don’t have private insurance, you will probably want to find out if your provincial drug plan covers the medicine and, if so, whether you can access it.

Pharmaceutical manufacturers must overcome several hurdles to have drugs listed in Canada’s public plans. First, the medication should have a positive recommendation from health technology assessment agencies, which means it has clinical benefit. In recent years, rare disorder medicines approved by Health Canada commonly receive a positive recommendation, unless an agency has doubts about its efficacy. However, the health technology assessment report often states that the manufacturer should make a substantial price reduction. This is intended to be the set-up position for the federal, provincial and territorial governments’ collective price negotiating process.

The manufacturer must next seek the drug’s admittance to the price negotiation process. The managers of each public drug plan decide at the beginning of a negotiation whether it will be included. If the negotiation is successfully completed, the cost and criteria under which the plans will pay for the medicine are agreed. However, this doesn’t guarantee that all public plans will cover the drug because each plan must reach an individual agreement with the manufacturer. If a negotiated drug is not listed, it is unclear as to whether the province opted out of the negotiation or if it opted in but failed to complete an agreement with the manufacturer – because the process is confidential.

Luckily, the price negotiation for the drug you need has been successful and the drug is listed in your provincial plan. Can you obtain coverage? It depends…

A patient’s disease stage is often required to match restrictive clinical criteria, which sometimes make little clinical sense. For example, coverage might be limited to patients who are sicker than you are. Although your health could improve substantially if you received the drug now, you can only obtain coverage when your health has declined, at which point the drug might not be as beneficial.

Or perhaps your provincial drug plan has less restrictive criteria, but coverage is limited to seniors, social assistance recipients, children or some special group of patients. Even if you fit into one of these categories, you might find a co-payment and/or deductible must be paid before receiving coverage, which you may not be able to afford. Every province has schemes to assist low-income residents, but you and your doctor will require endurance to work through the red tape to apply for the benefits, and you might still have to pay some of the cost.

If your public drug plan doesn’t list the medicine or it refuses to cover you because you don’t match the coverage criteria, what can you do? You could petition your provincial government for compassionate access. Provinces sometimes provide such coverage on a case-by-case basis, but the probability of success is low.

Alternatively, you could appeal to the manufacturer. Manufacturers’ compassionate access has always been limited but will decrease further with the federal government increasing the powers of the tribunal that sets Canadian price controls on new medicines. The government intends to reduce the prices of new innovative drugs by 40% on average which, in practice, may require medicine developers to drop prices by what some consider to be unsustainable amounts. This may result in manufacturers cutting back on clinical studies in Canada and launching novel medicines here long after doing so in other countries, or not bringing them to Canada at all.

You could take your situation to the media, especially if you learn that patients in other provinces with your disorder have access to the drug through their public plans. However, you should be aware that most media regard pharmaceutical companies to be as untrustworthy as banks, insurance providers and oil companies. Consequently, even though the manufacturer completed the necessary assessments and price negotiations, the media’s story may focus more on the drug’s high cost than the plan not covering the medicine. You might even be portrayed as a mouthpiece for the manufacturer in its effort to convince the plan to list its drug.

Canadians like you, who need rare disorder medicines essential to extend their life or improve its quality, are commonly faced with many impediments in accessing them. While these expensive drugs often raise questions about affordability, many are for conditions for which there has been no previous effective treatment. The cost of these new drugs must be balanced against both the suffering endured and the cost of hospitalizations and other care needed to treat the symptoms of the disorder.

The federal government has promised national pharmacare so that Canadians have the drug coverage they need and to establish a national strategy for high-cost drugs for rare diseases to improve access to these medicines. Unless the road to accessing high-cost specialized therapies for rare disorders is shortened, obstructions removed by reversing the punitive price controls to begin in July 2020, and a fair national system for delivering medicines based on patients’ needs is implemented, Canadians like you will continue to suffer and even die waiting for access to new innovative therapies. In the words of Lennon and McCartney, “why leave me standing here?