Controlling Drug Prices Shouldn’t Harm Rare Disease Patient Access to Treatment

According to a story from The Globe and Mail, Canada’s Patented Medicine Prices Review Board announced last month that drug prices would be subject to new regulations. These new rules are slated to go into effect next year. Despite the fact that these new rules should help keep down the cost of drugs, some rare disease patient groups have raised concerns that these regulations could compromise the development of new rare disease therapies. However, responsible price controls won’t necessarily have such a negative effect.

Responsible Regulation Should Be Beneficial

Such concerns sound like a talking point fed to advocates from corporate donors. Price regulations alone aren’t enough to make a successful drug entirely unprofitable. Fears that drug developers are going to stop conducting trials and recruiting patients are most likely unfounded. Ultimately, rare disease trials often require the recruitment of patients from outside the country if a disease is exceptionally rare, and a developer is going to need a participant group of significant size if it is going to gather meaningful information about a drug. It’s the only way to get a drug approved.

We should also take into account the fact that drug companies can make very hefty profits from rare disease therapies, even after the cost of research and development. In fact, margins are often nearly double that of drugs for more common diseases. Even with price controls, developers should still have plenty of motivation to make new rare disease drugs as long as profits are being made.

Lower Prices = Better Access

Another factor that these concerned rare disease advocates should consider is the benefits of better pricing regulations for drugs. Ultimately, extreme price has always been a greater barrier to treatment access than regulations that control prices. Take, for example, the first drug for Gaucher disease, which was priced at $250,000 annually. This was considered acceptable because it was the first therapy for the illness, but prices have only continued to climb since then, with some drugs climbing into the millions for a year of treatment.

Obviously diligent monitoring will be necessary to understand what impact the new drug pricing rules will have, but rare disease advocates should be hopeful that these rules will help and not harm their access to critical medications.


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