This week, Health Canada updated their website to include information about the approval process for orphan drugs to treat rare diseases. There are several intriguing aspects regarding this newly posted information. But being intriguing is not necessarily a good thing.

Suffice it to say, the new information does not answer some of the most basic questions that the rare disease community are asking.

What is the Definition of a Rare Disease?

Health Canada still does not have a definition of a rare disease. They state that about 1 in 12 Canadians have a rare disease but later note that there is no common definition of an orphan drug to treat a rare disease. The agency cites 2 possible definitions – 1 from Europe (prevalence no more than 5 in 10,000 people) and 1 from the U.S. (less than 200,000 Americans) but Health Canada added a little twist to both of those definitions.

For the European definition, Health Canada specifically wrote “prevalence of no more than 5/10,000 and a life threatening, chronically debilitating condition for which no alternative treatment is available (Europe)”.

Actually, that is not how the European Union (EU) defines an orphan drug. Their definition of an orphan drug is:

  • it must be intended for the treatment, prevention or diagnosis of a disease that is life-threateningor chronically debilitating;
  • the prevalenceof the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;
  • no satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorised, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.

For the American definition, Health Canada wrote “a presumption of unprofitability when a disease or condition affects no more than 200,000 people in the United States (USA).” That is not how the U.S. government defines an orphan drug.

According to the U.S. Food and Drug Administration (FDA), an orphan drug is a medicine to treat a rare disease and they define a rare disease as follows:

  • affects less than 200,000 persons in the United States, or 
  • affects more than 200,000 in the United States and for which there is no reasonable expectation that the cost of developing and making available in the United States a drug for such disease or condition will recovered from sales in the United States of such drug. Determinations under the preceding sentence with respect to any drug shall be made on the basis of the facts and circumstances as of the date the request for designation of the drug under this subsection is made.

So, the ‘profitability’ factor only applies to those drugs that affect more than 200,000 and not less, as Health Canada implied.

All of this leads to 2 questions.

First, what is Canada’s definition of a rare disease or orphan drug? That is still not clear but an educated guess would be that they are simply going to let the U.S. and EU define what is and is not an orphan drug/rare disease and leave it at that.

Second, why has Health Canada suddenly added new text about alternative treatments and/or profitability to the definition of an orphan drug? This question is more concerning. It implies that if a person with a rare disease has a treatment available, there is no reason to approve a second treatment option.? Using that logic, there should only be 1 medicine for treating high blood pressure and only 1 medicine for treating a headache. Why would a person with Fabry disease or cystic fibrosis or muscular dystrophy need more than 1 treatment option?  Also, the phrase profitability is only relevant to the U.S. Orphan Drug Act in that the U.S. government provides tax breaks and other financial incentives for a company to develop an orphan drug. The Canadian government provides no such tax breaks or incentives.

Will the addition of ‘alternative treatments’ and ‘profitability’ change the approval of orphan drugs by Health Canada? Probably not. Health Canada is largely focused on safety and efficacy of a drug and it is not concerned about less effective treatments or profitability.

My main concern with the addition of ‘alternative therapies’ and ‘profitability’ is that those phrases can later be used by policy makers, politicians, ministry offices, payors and other regulatory agencies to deny or delay the accessibility of orphan drugs to patients with rare conditions. Those people are already really good at delaying things. They do not need more verbal weapons.

Health Canada needs to define rare diseases properly. To rely in other countries to define a rare disease and then include additional misleading text to their definitions is a concern and they should be called out on it.

How will the Review Process Change?

The second intriguing feature found on Heath Canada’s new page about rare diseases is that all the links about assisting companies and patients for this newly formed rare disease initiative go to links that are older and that do not specifically mention rare diseases or to links that do not really state anything new and then refer back to the original rare disease page by Health Canada. It’s a bit of a shell game. One sentence that did stand out however was “Current work is ongoing to assess and develop a National Formulary through the Advisory Council on the Implementation of National Pharmacare.” It will be interesting to see if that Advisory Council will come up with a definition of an orphan drug. We will have to wait and see.

Is There Anything Positive about Health Canada’s Rare Disease Page?

Yes.

Health Canada does acknowledge that they understand the particular needs and challenges that rare disease patients face. The agency also stated they are committed to improving the access to medications to treat rare conditions. To that end, Health Canada listed 6 changes they are currently taking to improve access to orphan drugs and they are:

  • making greater use of the reviews and decisions of trusted foreign regulators
  • working with health technology assessment organizations to reduce the time between Health Canada approvals and reimbursement recommendations
  • building a program to provide early parallel scientific advice with health technology assessment organizations to assist new drug development
  • expanding the priority review policy to decrease review time for products needed by the health care system
  • using existing and new real world evidence to support regulatory decision-making across a drug’s life cycle
  • renewing the Special Access Programme (SAP) to improve access to products that are not currently authorized for sale in Canada.

We will just have to wait and see.