Rare Disease Commercial Opportunities in Canada

The Canadian pharmaceutical market represents 2% of the global market. Canada does not have an explicit definition or regulatory pathway for rare disease drugs. At first glance, these aspects may appear to hinder companies from researching, investing, and launching their rare disease products in Canada. In reality, however, Canada offers a terrific opportunity for these companies.

Canada offers intellectual property rights on par with the US, Europe, and other jurisdictions, as well as eight-year data protection legislation for newly launched products. The average regulatory approval time for a Notice of Compliance (NOC) following submission is generally twelve months and stable. There are two mechanisms currently in place for orphan drugs: Priority Review and Notice of Compliance with Conditions (NOC/c). Many molecules developed to treat rare diseases meet the criteria for these mechanisms and have faster approval times. If the sponsor has yet to obtain approval for the product, drugs can also be made available to patients through a Special Access Program (SAP). The conditions for this include:

• The disease is serious or life- threatening
• Available evidence confirms that the new drug is effective
• Other available therapies have been attempted

In addition to these options, the rare diseases community has proposed the implementation of a specific regulatory approval program. They are urging the government to support the development of a regulation framework, versus proceeding with piece-meal guidance.

One of the key concerns for any company is obtaining product reimbursement from payers within a reasonable time frame. Canada has a robust private payer market that represents 40% (and even higher in some therapeutic areas) of the overall drug reimbursement. Private payers have established cost- saving strategies to ensure that the funding of orphan (and other) drugs remains afford- able. Canada’s ten provinces and three territories determine the public reimbursement of pharmaceuticals, which may appear daunting. The good news is that four provinces (Quebec, Ontario, Alberta, and British Columbia) represent 80% of the Canadian pharmaceutical market. All provinces have exceptional approval status, with qualification conducted on a one-by-one basis. It is important to under- stand the public and private payer landscape when entering this market to ensure that new drugs are accessible to those suffering from rare disorders.

Market size, prevalence, and incidence of most diseases generally constitute 10% of the US market. Given the low incidence of rare diseases, relatively low number of health care providers/Key Opinion Leaders (KOLs) active in these areas, and the fact that rare disease communities are often global, disease management guidelines tend to be similar across Canadian, US, and EU markets. Canadian research centres, known for their world-class reputations and capabilities, often take part in the industry’s Phase II and Phase III global clinical trials. Canadian KOLs are experienced in operating at an inter- national level and integrate well with their US/EU counterparts. They may either influence or participate in establishing treatment regimens for the disease areas in which they are experts.

The Canadian Organization for Rare Disorders (CORD) is a national network for organizations that represent people with rare disorders. CORD works with governments, researchers, clinicians, and industry to pro- mote research, diagnosis, treatment, and services for all rare disorders in Canada. It provides an existing network for all players to facilitate entry into the market.

An additional benefit of commercialization in Canada is the extremely small corporate foot- print required to resource pre- launch, launch, and post- launch. Most companies can use internal shared services or outsource locally to qualified service providers. The Canadian team could be as small as GM, Medical, MSL, Marketer, and three to six Specialty Representatives.

In conclusion, Canada represents a significant opportunity for companies producing rare disease drugs. Exclusivity is on par with or better than many major markets, reimbursement potential is possible through different payer types, and successful commercialization requires a very small investment footprint.

Originally published in BTC Insights Spring 2016 and Canadian Pharmaceutical Marketing Feb/ Mar 2017

For further information, please contact Joe Knott, Managing Director, The Pangaea Group or Helen Kalra, Associate Managing Director, The Pangaea Group