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Cheryl Rockman-Greenberg, Alex MacKenzie and Sandra Sirrs are clinician investigators and metabolic specialists caring for patients and their families with rare genetic disorders at tertiary-care centres in Winnipeg, Ottawa and Vancouver, respectively.

On Aug. 9, the Patented Medicine Prices Review Board of Canada announced new drug-pricing regulations for new patented medicines that will go into effect in 2020. And that has raised worries among those with rare diseases, as well as their advocates.

Some patients and advocacy groups are concerned that these changes will greatly disadvantage access of Canadian children and adults with rare and ultrarare genetic disorders to new effective treatments. They are concerned that pharmaceutical companies, who see the new pricing system as likely reducing the costs of their drugs and thus diminishing Canada’s already small market, may be less interested both in conducting expensive clinical trials as well as commercializing new treatments for the few patients who have rare diseases here. Some believe that these new pricing reforms will offset and compromise any new commitment proposed in the 2019 federal budget to increase funding for drugs for such diseases.

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But as Canadian physicians with extensive experience in industry-sponsored clinical trials in rare diseases, and who prescribe new approved drugs for patients with rare genetic disorders, we believe that these new regulations will not be as troubling as they fear.

By definition, rare diseases are rare. So in order to conduct clinical trials, pharmaceutical companies must engage such patients and their physicians from all over the world, including Canada, as there are simply not enough patients with a particular rare disease in a single country to complete a trial. From a strictly business perspective, then, ending the recruitment of Canadian children and adults with rare diseases in clinical trials here or internationally seems counterintuitive. After all, patients with such rare diseases provide the scientific evidence needed to confirm that a new medication is safe and effective, such that pharmaceutical companies can apply for licences for the drugs internationally, and recoup their investment.

The profit margins for pharmaceutical companies for drugs for rare diseases are currently close to double those for drugs for common diseases, so manufacturers are highly motivated to conduct the trials needed for drug licensure and to involve as many patients as they can. Physicians such as ourselves will continue to advocate for our Canadian patients with rare diseases, for their participation in clinical trials and for access to new life-sustaining medications at affordable pricing.

In Canada, the process of listing new approved drugs for rare diseases on provincial formularies, a list of medications that can then be prescribed, is complicated and arduous, lacks transparency and too often is met with rejection. But to us, one of the main threats to access to drugs for rare diseases in Canada is not the current regulations or the proposed requirements, but the price. When the first drug for Gaucher disease, a rare genetic metabolic disorder, came to market, it was priced at around $250,000 a year for each patient. But this jaw-dropping cost was accepted, because regulators believed this was a one-off success against a rare disease.

Now, with advances in technology, it is increasingly straightforward to develop drugs for rare genetic diseases that were previously untreatable – and yet prices have skyrocketed. It is now common for drugs for rare diseases to come to market with a suggested list price of anywhere from $750,000 to more than $2-million a year for each patient, who often requires lifelong treatment. Pricing on these drugs is not subject to market forces in that even as competition comes into the market, the drug prices do not fall.

Ultimately, it will be necessary to collect data over time to ensure that there is no negative impact from the changes proposed by the review board on pricing for rare-disease drugs. But we strongly believe that these changes need not and will not hamper the ability of Canadian patients to participate in well-designed clinical trials. We hope that patients, advocacy groups and physicians continue to collaborate with manufacturers and regulatory agencies on any strategies to improve access to rare-disease drugs, including, but not limited to, strategies that attempt to bring more transparency and value for health-related outcomes into drug-pricing models.

We believe that this would be the right thing to do – placing the interests of patients and their families first.

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