Legal & Regulatory > Canada > Orphan Drugs and Rare Diseases: Canada

All legal aspects surrounding orphan drugs and rare diseases in Canadian Pharma. Prepared in association with Fasken, a leading global law firm, this is an extract from The Pharma Legal Handbook: Canada, available to purchase here for USD 99.

1. What is the definition of Rare Diseases in your country?

In Canada, “Rare Diseases” are defined as “life-threatening, debilitating or serious, and chronic conditions affecting a small number of patients, many of which predominantly affect children, as these diseases are often genetically based and appear at birth or in early childhood”.

2. Does the designation of ‘Orphan Drug’ exist in your country? (Does it correspond with the definition of Rare Diseases?)

The designation “Orphan Drug” does not currently exist in Canada.

3. What is the regulatory framework for the authorization of an Orphan Drug? (Is this regulatory framework based on Rare Disease status or can it alternatively be based on Orphan Drug foreign status?)

There is currently no regulatory framework for the authorization of an Orphan Drug.

Health Canada proposed an Orphan Drug Regulatory Framework in 2012, but it was not enacted and, in October 2017, the federal government deleted all references to the framework from the Ministry of Health’s website without notice or consultation.

Orphan Drugs therefore follow the same regulatory approval process as other drugs in Canada. This standard pathway includes a “New Drug Submission”, which requires manufacturers to submit evidence of a drug’s safety, efficacy, and quality to Health Canada. If a submission is acceptable, Health Canada will issue a Notice of Compliance (“NOC”), which allows a manufacturer to market the drug in Canada.

4. Does your country have provisions for relaxed clinical trial/scientific evidence requirements in respect of Orphan Drugs as compared to other drugs?

No, Canada does not currently have provisions for relaxed clinical trial/scientific evidence requirements in respect of Orphan Drugs as compared to other drugs. However, in some cases, a Notice of Compliance with Conditions (“NOC/c”) may be issued.

In particular, market authorization through a NOC/c allows Health Canada to provide earlier market access to potentially life-saving drugs but requires that the manufacturer undertake additional studies before a full NOC can be issued. This undertaking allows Health Canada to facilitate access based on promising evidence of clinical significance while monitoring the drug through enhanced post-market surveillance.

5. Is there an expedited pathway for Orphan Drugs?

There is no expedited pathway specifically for Orphan Drugs, however, drug applications may generally be fast-tracked under one of two pathways: the Priority Review pathway or the NOC/c pathway described above.

Most new drugs have a review target of 300 days; however, the Priority Review pathway allows for the quickest review target of 180 calendar days; whereas the NOC/c pathway allows for an expedited 200-day target.

In both cases, the pathways are available to manufacturers if the drug is intended for the treatment, prevention or diagnosis of serious, life-threatening or severely debilitating diseases or conditions for which: (i) there is no alternative therapy available on the Canadian market or, (ii) where the new product represents a significant improvement in the benefit/risk profile over existing products.

However, the Priority Review pathway applies to drugs that show substantial evidence of clinical effectiveness, i.e. at least two adequate and well controlled clinical studies, each convincing on its own to establish effectiveness of the drug involved.

In contrast, the NOC/c pathway applies to drugs with promising evidence
of clinical effectiveness throughout the clinical trial phases. For example, the
data to support authorization under the NOC/c policy is often:

• limited due to a small number of patients eligible for clinical trial participation;
• based on surrogate marker data predictive of clinical benefit; or
• based on larger trials in which final outcomes of morbidity and mortality
  are lacking.

The data required for a NOC/c submission is therefore unlikely to meet the evidentiary requirements of the Priority Review Pathway.

In addition to these two pathways for drug expedited approval, the Special Access Program allows patients to access a drug which is not authorized in Canada under a shortened twenty-four hour application process for treatment, diagnosis, or prevention of serious or life-threatening conditions when conventional therapies have been considered and ruled out, have failed, are unsuitable or unavailable.

6. Are foreign marketing authorizations recognized in your jurisdiction for Orphan Drugs? If yes, marketing authorizations from which countries are recognized?

There are no foreign marketing authorizations recognized in Canada for Orphan Drugs.

7. Can Orphan Drugs be reimbursed? If so, is there a specific reimbursement procedure for Orphan Drugs?

There is currently no specific reimbursement procedure for Orphan Drugs. In general, drugs may be reimbursed if a public or private insurer deems it cost-effective based on a health technology assessment.

In exceptional cases, including, for example if a drug may be (i) a last resort (ii) for a chronic and (iii) serious medical condition, then a public or private plan may provide coverage for a medication that would otherwise not be reimbursed by the plan.

8. How are the prices of Orphan Drugs regulated?

The prices of Orphan Drugs are regulated in the same manner as other drugs in Canada. In the cases of patented drug, they are regulated by the Patented Medicines Review Board (“PMPRB”), which sets the maximum price for which a patented drug may be sold in Canada.

The prices of drugs are also negotiated by the pan-Canadian Pharmaceutical Alliance which represents a group of provinces, territories and other institutions, who, by combining their bargaining power, seek to reduce the cost of pharmaceuticals.

9. In case of reference price based on a basket of countries, what countries are included?

The PMPRB uses reference pricing to determine the maximum price for which a patented drug may be sold in Canada. The current basket of countries the countries considered includes France, Germany, Italy, Sweden, Switzerland, the United Kingdom, and the United States.

10. Have there been any significant legal/judicial developments in relation to Orphan Drugs in your country?

No, there have not been any significant legal/judicial developments in relation to Orphan Drugs in Canada.

11. Are there proposals for reform or significant change to the regulation of Orphan Drugs? If yes, when are they likely to come into force?

Yes, there are proposals for significant changes to the regulation of Orphan Drugs in Canada. The 2019 federal budget proposal stated that a distinct process and funding for specialized drugs should come into effect by January 1, 2022. The proposed reformation is stated to focus on five key elements:

1. The development of a national strategy to ensure uniform patient access
   to Orphan Drugs across the country;
2. The establishment of a unique drug approval pathway to improve patient
   access to Orphan Drugs;
3. The development of a structured evidence collection strategy to study
   the impact of Orphan Drugs on patients;
4. The negotiation of performance-based funding agreements with manufacturers of Orphan Drugs whereby the amount paid for an Orphan Drug will
   depend on the efficacy of the drug; and
5. Ensuring an approval process for Orphan Drugs that is both transparent
   and clearly communicated.