Compassionate Access Programs: Getting Drugs Not Yet Approved

When Standard Options Are Not Enough

Sometimes the drug you need is not yet approved for sale in Canada. It might be available in the United States or Europe, or it might still be in clinical trials. In these situations, compassionate access programs can provide a pathway to treatment.

Canada has both government-run and manufacturer-run compassionate access pathways. Understanding how they work could make a critical difference in your care.

Health Canada's Special Access Programme (SAP)

The Special Access Programme allows physicians to request access to drugs that are not currently authorized for sale in Canada. The SAP exists for patients with serious or life-threatening conditions where conventional therapies have failed, are unsuitable, or are unavailable.

How the SAP Works

Your doctor identifies a need for an unapproved drug

They submit a SAP request to Health Canada, including:

- Your diagnosis and medical history - Why approved alternatives are not suitable - Evidence supporting the use of the requested drug - The manufacturer and product details

Health Canada reviews the request (typically within 24 hours for urgent cases)

If approved, the manufacturer is authorized to sell the drug directly to the treating physician

Your doctor oversees your treatment and reports outcomes to Health Canada

Key Facts About the SAP

Who Pays?

This is often the most challenging part. Health Canada does not cover the cost of drugs accessed through the SAP. Payment responsibility falls to:

• The patient (out of pocket)
• Private insurance (some plans cover SAP drugs on a case-by-case basis)
• Provincial drug plans (some provinces have mechanisms to cover SAP drugs in exceptional circumstances)
• The manufacturer (some provide the drug at no charge)

Manufacturer Compassionate Use Programs

Many pharmaceutical companies run their own compassionate use or expanded access programs, separate from the SAP. These programs typically provide the drug free of charge to qualifying patients.

Common Eligibility Criteria

• Serious or life-threatening condition with no adequate alternatives
• The drug has shown promising clinical data (usually in Phase II or III trials)
• The patient is not eligible for an active clinical trial
• A licensed physician manages the treatment

How to Access Manufacturer Programs

Ask your specialist — they often know about available programs

Contact the manufacturer directly — look for "patient access" or "compassionate use" on the company's website

Check clinical trial registries — many trials have companion compassionate access programs

Contact patient advocacy groups — disease-specific organizations often maintain lists of access programs

Other Pathways to Unapproved Drugs

Emergency Drug Release

For life-threatening emergencies, Health Canada can authorize emergency release of an unapproved drug within hours. The treating physician contacts the SAP emergency line, and Health Canada expedites review.

Provisional Market Authorization

Health Canada has introduced provisional authorization pathways for drugs that address unmet medical needs, allowing some drugs to reach the Canadian market faster while post-market studies continue.

Clinical Trial Participation

If a drug is currently in clinical trials in Canada, participating in the trial gives you access at no cost. See our guide on clinical trials for details on how to find and join trials.

Practical Steps If You Need an Unapproved Drug

Step 1: Talk to Your Specialist

Your doctor is the only person who can initiate a SAP request or contact a manufacturer program. Be direct: "Is there a drug available elsewhere that could help me, even if it is not approved in Canada yet?"

Step 2: Research the Drug

Understand the evidence base for the drug you are seeking:

• Has it been approved in other countries (US, EU, UK)?
• What do the clinical trial results show?
• What are the known risks and side effects?

Step 3: Address the Cost Question Early

If the SAP is approved but the manufacturer charges for the drug:

• Ask the manufacturer about compassionate pricing or free supply
• Contact your private insurer to request an exception review
• Apply to your provincial drug plan for exceptional coverage
• Explore patient advocacy organizations that may provide financial assistance

Step 4: Document Everything

Keep detailed records of:

• Your medical history and treatment failures
• All communications with Health Canada and manufacturers
• Costs incurred (for potential insurance reimbursement or tax claims)

When the Drug Eventually Gets Approved

Once a drug receives full Health Canada approval, it enters the standard pricing and coverage pathway:

PMPRB ensures the price is not excessive

pCPA negotiates a listing price

Provincial formularies decide whether to cover it

It becomes available at pharmacies — and you can compare prices using TransparentMedz

This transition can take 12 to 24 months after approval, during which you may continue on the compassionate access program.

Do Not Assume the Answer Is No

Compassionate access programs exist because the system recognizes that patients cannot always wait for the standard approval process. If you or a loved one needs a drug that is not yet available in Canada, ask your doctor about the SAP and manufacturer programs. The process is not simple, but it has provided life-changing treatment to thousands of Canadians.