{"id":10090,"date":"2022-01-13T21:34:07","date_gmt":"2022-01-14T02:34:07","guid":{"rendered":"https:\/\/rqmo.org\/treatments\/"},"modified":"2026-02-06T20:02:20","modified_gmt":"2026-02-07T01:02:20","slug":"treatments","status":"publish","type":"page","link":"https:\/\/rqmo.org\/en\/treatments\/","title":{"rendered":"Treatments"},"content":{"rendered":"\t\t
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Treatments<\/h1>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t
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There are different types of treatments for the multitude of rare diseases: medication medical devices, specialty foods, surgery, physiotherapy, etc.<\/p>

Medication may be drugs used for common conditions or may be specific to a rare disease. The latter are called “orphan drugs <\/strong>“. <\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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What is an orphan drug?<\/h2>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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An \u00ab orphan<\/strong> \u00bb drug is intended for the treatment of a rare disease. In some countries (e.g. United States, European Union, etc.), a pharmaceutical company can apply for its experimental drug to obtain an \u00ab “orphan designation” <\/strong> \u00bb and thus benefit from certain advantages (financial and others) to continue the research and development of this therapy. If, following clinical trials, the drug is found to be safe and effective and it is authorized for sale, it is then called an \u201corphan drug.\u201d <\/p>

In Canada, there is no special program for the development of orphan drugs like the Orphan Drug Act<\/a> in the United States or the \u00ab <\/strong>Orphan Drug Products<\/a> \u00bb <\/strong>program in the European Union. <\/p>

Did you know that approximately 40% of orphan drugs authorized elsewhere in the world are not available in Canada?<\/em><\/p>

To learn more about the drug evaluation process in Canada, listen to our webinar: <\/strong>M\u00e9dicaments orphelins : comment on d\u00e9cide s\u2019ils sont mis en march\u00e9 au Canada, combien ils co\u00fbtent et si les gouvernements provinciaux les paient ?<\/strong> \u00bb<\/a><\/em> <\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t

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For information on orphan drugs and medications in general, visit our Drug Resources – RQMO<\/a> page.<\/strong><\/h4>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t
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Surveillance \nfor orphan drugs<\/h2>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t
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Did you know that in Canada we do not use the terms \u201corphan drug\u201d or \u201corphan designation\u201d?<\/em><\/p>

This makes it difficult to follow a drug intended for a rare disease through the authorization and evaluation process in Canada. Orphan drugs are buried among the many other drugs in the various databases of government organizations (Health Canada, Canadian Agency for Drugs and Technologies in Health (at Canadian Drug Agency), National Institute of Excellence in Health and Social Services of Quebec (INESSS).<\/p>

We monitor orphan drugs that pharmaceutical companies want to market in Canada. We follow the drug through the entire authorization and evaluation process to find out if it will be authorized by Health Canada and whether or not it will be recommended for reimbursement by public drug insurance plans.<\/p>

We notify patients, caregivers, associations and health professionals when a drug for a rare disease is being evaluated at INESSS. These notices are made through our various platforms (social media, newsletter, email to the people in our contact registry, etc.). We want, among other things, to encourage and help patients\/caregivers to participate in INESSS consultations. Your opinion matters! See more details below.<\/em><\/strong><\/p>

We also collaborate with Canadian associations for evaluations carried out by the Canadian Agency for Drugs and Technologies in Health<\/span> <\/span>(CADTH).<\/span><\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t

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Do you want to know more about treatments for your rare disease? Do you want to know if there is an orphan drug in development or on the market? Contact us.<\/span><\/h4>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t
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\n\n\t\t\t\t\t\tNote to drug manufacturers<\/span>\n\t\t\t\n\t\t\t\t\t\tIf you have a drug about to be introduced into Canada, let us know!<\/span>\n\t\t\t\n\t\t\t\t\t\t