Special Access and

Advanced Access Programmes Marion Law

Canadian Expert Patients in Health Technology Conference Toronto

November 8, 2016

Access to drugs

•  Market authorization •  Clinical Trials •  Special Access Programme

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Market Authorization •  Most common, most robust and most desirable access

pathway •  Information on safety, efficacy and drug quality has been

thoroughly reviewed (New Drug Submission: 300 day review)

•  Drugs for serious, life-threatening or severely debilitating diseases that have substantial evidence of efficacy may be eligible for a priority review (180 day review)

•  Promising new drugs for serious, life-threatening or severely debilitating diseases may be eligible for a Notice of Compliance with conditions, allowing market access until efficacy is confirmed

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Market Authorization (cont’d) •  Provides the most oversight: Inspection program and post-

market safety surveillance (Canada Vigilance) in place •  HC has the authority to take action in the event of a safety,

efficacy or quality issue •  Cost-recovered activity

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Clinical Trials •  The study of a new drug, or of a marketed drug for a new

indication, is subject to Health Canada and Research Ethics Board approval

•  Health Canada review focussed on safety of participants (includes a review of the quality of the drug)

•  Patients are closely monitored and must sign an Informed Consent Form

•  Inspection program and safety surveillance in place •  HC has the authority to take action in the event of a safety,

efficacy or quality issue

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Special Access Programme: Mandate

•  SAP considers requests for access to non-marketed drugs from medical practitioners treating patients with serious or life-threatening conditions when conventional therapies have failed, are unsuitable or unavailable.

•  SAP authorized sales are exempt from all aspects of the Food and Drugs Act & Regulations.

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Programme profile

•  24hr service, 365 days of year •  ~16,000 requests processed in 2015 •  ~100 requests handled each work day •  ~75,000 patients •  1500-2000 telephone calls per month •  ~500 drugs with active status •  2-3 requests for ‘new drugs’ /week

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Scope

•  Unapproved drugs

•  Drugs for treatment, diagnosis or preventative purposes: •  Pharmaceuticals, radiopharmaceuticals, biologics, NHPs •  Includes drugs for rare diseases e.g. orphan drugs

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Special Access Programme requests

•  Section C.08.010 of the Food and Drug Regulations allows the SAP to consider requests for access to drugs that are not authorized on the Canadian market.

•  The request must be made by a practitioner for a patient.

•  The practitioner must supply the following information: –  The condition for which the drug is requested (must be serious or life-

threatening) –  Other therapies that have been tried and failed, or considered and deemed

unsuitable –  Data supporting the use, safety and efficacy of the drug for this condition

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Special Access Programme requests

•  A SAP authorization allows a manufacturer to sell a quantity of a drug to be used for that patient

•  The manufacturer must be willing to provide the drug and may impose additional conditions for access

•  The manufacturer will sometimes provide the drug free of charge

•  The physician must report the outcome of the use of the drug, including any adverse events, to Health Canada and to the manufacturer

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Processing of requests

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Special Access Programme requests: Decision Making

Consideration of requests involves: •  determining whether the patient suffers from a serious or life-

threatening condition; •  confirming that other therapies have been tried or at least

considered, or are unavailable; •  reviewing data with respect to the use, safety and efficacy of the

drug for the condition being treated.

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Request Activity: 2015

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Types of drugs authorized under the Special Access Programme

•  New drugs under development •  Older drugs that never sought market authorization in

Canada •  Drugs for rare cancers •  Drugs for rare infections (including tropical diseases) •  Drugs for rare accidental situations (snake bites, various

types of poisoning) •  Drugs to treat rare disorders: About 50 such drugs are

currently being accessed through the SAP –  E.g. Buphenyl, Calcort, Ceprotin, Cholbam, Cystagon, Factor

X, Factor XI, Hemophil M, Increlex, L-citruline, Normosang, Panhematin, sacrosidase, Syprine, Thiola

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Limitations of the Special Access Programme

•  Limited safety and efficacy information is available for review

•  No drug quality (chemistry and manufacturing) review

•  No authority over the manufacturer when dealing with unapproved drugs

•  Limited data collection to increase benefit/harm knowledge and inform future decisions

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Challenges of the Special Access Programme •  Balance between role of Health Canada versus practice of

medicine •  Balance between intent of the programme and the

expectations of patients and physicians •  Using the Special Access Programme for purposes not

intended by the regulations, e.g. for managing drug shortages

•  # of drugs that should be on the market rather than on the Special Access Programme

•  Transitioning drugs off the Special Access Programme •  Communications: emotionally charged, urgent •  Process: not fully electronic, cumbersome database

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Going forward

•  Special Access Programme renewal –  Policy work –  Concerted effort to get some drugs off the programme and

into the market –  Process improvements/efficiencies

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Summary

•  While SAP is not intended to promote or encourage the early use of drugs, to conduct research or to circumvent the clinical trial or drug review process, it has allowed patients with rare diseases to access drugs that were otherwise unavailable

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