Accessing Treatments

New treatments for porphyria are being developed and becoming available in Canada. Accessing treatments for rare diseases like porphyria in Canada can be challenging: they go through a long series of regulatory processes, and once treatments are approved,  they can be expensive or have limits on who can use them. The Canadian process can be extra frustrating because other countries often have access to the treatments before us and we hear about the treatments’  life-changing impacts.

Advocacy efforts from patient organizations and patients is essential to bringing new treatments to Canada, contact us to find out how you can help.

  • For more information on what the the status of different treatments mean, see Canadian Drug Approval and Access (below).

    PANHEMATIN (HEMIN)

    • Status: Available Canada-wide

    • Manufacturer: Recordati Rare Diseases

    Panhematin is available through Canadian Blood Services and Hema-Quebec (in Quebec) at no cost to patients.

    Information for ordering the product from Canadian Blood services is available here.

    • If you are having an attack, the option “For urgent use” should be selected. Current lab results aren’t needed before ordering if you have a diagnosis and history of attacks.

    • If you have recurring attacks, Panhematin may be used prophylactically (preventatively). Your doctor will need to apply for you to get Panhematin for this purpose and there may be criteria that limit who can receive it.

    GIVLAARI (GIVOSIRAN)

    • Status: Available, requires special approval

    • Manufacturer: Alnylam Pharmaceuticals

    Access to givosiran requires special approval. Please have your doctor contact AlnylamAssist Program at 1.833.831.2065 for support to access this treament.

    NORMOSANG (HEMIN)

    • Status: Approved in other countries, not available in Canada

    • Manufacturer: Recordati Rare Diseases

    Normosang is not generally available in Canada, Panhematin is used instead. In special circumstances, Normosang has been made available to Canadians, but this requires a specific reason and advocacy to access.

  • We are currently advocating for EPP/XLP treatments to come to Canada. You can help us make this happen! Please contact us if you’d like to get involved.

    For more information on what the the status of different treatments mean, see Canadian Drug Approval and Access (below).

    SCENESSE (AFAMELANOTIDE)

    • Status: Being submitted to Health Canada Review (May 1, 2023)

    • Manufacturer: Clinuvel

    Access may be available through the Special Access Program if your private insurance is willing to cover the cost. Please contact us for more information.

    DERSIMELAGON (MT-7117)

    • Status: Phase III clinical trials

    • Manufacturer: Mitsubishi-Tanabe

    At this time, this treatment is only available to clinical trial participants. The trials are not currently recruiting in Canada.

    BITOPERTIN- RECRUITING

    • Status: Phase II clinical trials, fully enrolled

    • Manufacturer: Disc Medicine

    This treatment is currently undergoing Phase II clinical trials in the United States. Patients who participate in the trial will have the option to receive bitopertin for up to 5 years after the trial period.

    Canadians were eligible to participate in the Phase II trials and we anticipate they will be eligible to participate in future trials when they are announced.

  • For more information on what the the status of different treatments mean, see the section below on Canadian Drug Approval and Access.

    BITOPERTIN

    • Status: Under development

    • Manufacturer: Disc Medicine

    This treatment is currently undergoing Phase II clinical trials for EPP. It may be a potential treatment for CEP.

Canadian Drug Approval and Access

It is helpful to understand the different stages that a treatment goes through before it becomes available in Canada and how to access it at each stage. For more information on accessing specific treatments, see the above sections.

  • Clinical trials are used to determine the safety and effectiveness of a treatment. In phase II and III trials, patients may either be given the treatment or a placebo to determine its effects.

    HOW CAN I ACCESS A TREATMENT IN THIS STAGE

    At this stage, the treatment will only be accessible by participating in the trial. You will need to meet eligibility criteria to participate. For current opportunities see Participate in Research or sign up for updates.

  • Once the clinical trials are completed, the pharmaceutical company will submit to the regulatory agencies in different countries to approve the drug as a treatment in that country.

    Often, companies will get approval for the treatment in other countries like the US or Europe before it is approved in Canada.

    HOW CAN I ACCESS A TREATMENT ?

    You may be able to access the treatment through Canada’s Special Access Program (SAP).

    This program is to allow access to treatments for serious or life threatening conditions that are not approved in Canada. Your doctor will need to fill out the paperwork for the program.

    While SAP may allow you access to the treatment, it doesn’t always pay for it. Some pharmaceutical companies offer a compassionate access program so patients don’t have to pay out of pocket. Some patients are also able to get financial support through provincial access programs or private insurance.

  • If a treatment has gone through clinical trials and is safe and effective, the drug manufacturer submits a request to Health Canada to make the drug available in Canada. The approval time for these requests can take a few months to more than a year.

    Often, companies will get approval for the treatment in other countries like the US or Europe before it is approved in Canada.

    HOW CAN I ACCESS A TREATMENT IN THIS STAGE

    You may be able to access the treatment through Canada’s Special Access Program (SAP).

    This program is to allow access to treatments for serious or life threatening conditions that are not approved in Canada. Your doctor will need to fill out the paperwork for the program.

    While SAP may allow you access to the treatment, it doesn’t always pay for it. Some pharmaceutical companies offer a compassionate access program so patients don’t have to pay out of pocket. Some patients are also able to get financial support through provincial access programs or private insurance.

  • Once a treatment is approved by Health Canada, it is reviewed by CADTH. CADTH looks at the economic and medical impact of the treatment and recommends whether or not a treatment should be reimbursed (paid for) by health care systems. This review usually takes about 9 months.

    HOW CAN I ACCESS A TREATMENT IN THIS STAGE?

    If the treatment has been approved by Health Canada, it can be prescribed, however you may need to pay for the treatment out of pocket.

    Options for coverage may include private insurance companies or the pharmaceutical company’s compassionate access program.

  • Once CADTH issues a recommendation, the pharmaceutical company will enter into negotiations with the pCPA (pan-Canadian Pharmaceutical Alliance). These negotiations are to determine what price the provincial health care systems will pay for the treatment. This can take 3 months to more than a year.

    HOW CAN I ACCESS A TREATMENT IN THIS STAGE?

    The treatment is available to be prescribed, but will not be paid for by the public health system.

    Some private insurers may provide coverage for the treatment at this point.

  • Once the pCPA negotiations are completed, each province will make an agreement with the pharmaceutical company about whether they will cover the treatment and who will be available.

    There may be criteria for who can be prescribed a treatment and it can be covered by either the health care system or private insurers.