Consultation Health Canada Orphan Drug Regulatory Framework and Beyond

Consultation Health Canada Orphan Drug Regulatory Framework and Beyond

Goal: Improve Canadian access to orphan drugs

There are several key components to the proposed Canadian Orphan Drug Regulatory Framework.

1. The orphan drug designation is based on criteria and processes that are aligned with those of the US and EU so collaboration is possible. The proposed drug is intended for use in a life threatening, seriously debilitating, or serious and chronic disease or condition affecting not more than five in 10 thousand persons in Canada (1 in 2,000). There is no drug currently authorized for the same use, or the drug will be of significant benefit.

2. Health Canada provides advice for clinical trials and in conjunction with international regulators. The designated drug receives priority review and waiver/remission of filing fees. There are specific requirements for benefit-risk assessment (as part of post-market plan) and 8 years of market exclusivity.

3. Transparency and information sharing are provided throughout the lifecycle of the drug; information is available to key decision makers (including healthcare providers, health technology assessors (HTAs) and patients) at the most appropriate time.

4. Life-cycle approach takes into account a wide body of evidence before and after a drug is marketed.
1. Are the criteria for “orphan drug” designation appropriate?
YesMaybeNoDon't know
Is prevalence of “no more than 5 in 10,000 (1 in 2,000) persons affected” for orphan designation appropriate?
Should the orphan drug policy be restricted to conditions that are “life-threatening, seriously debilitating, or serious and chronic?”
Is it important that the Canadian orphan drug designation [rare disease] criteria be similar to those of the European Union and USA?
2. Patient Input
YesMaybeNoDon't know
Is it important that patients can provide input throughout the process, including application for designation, developing protocol for clinical trials, and application for market authorization?
Who should be allowed to provide input: patients, caregivers, and/or patient groups?
3. How should input be provided?
YesMaybeNoDon't know
Individual written perspective,
Summarized group submission,
Patient representative oral testimony,
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