Draft guidance on human and veterinary drug submissions based on promising evidence and terms and conditions: Template

Request for advance consideration of promising evidence status template for human drug submissions

The advance consideration of promising evidence (ACPE) status request should be no longer than 25 pages (excluding references) and should be submitted along with the pre-submission meeting package. Sample text is provided as guidance for content and should be removed from the completed document before filing along with this introductory text. The headings should be kept in bold text.

Date of request for advance consideration of promising evidence:

Sponsor:

Contact information:

Product information:

• Dose / route / strength:
• Is this a combination product? Y / N
• Drug class:
• Is this a first-in-class drug? Y / N

Proposed brand name (if known) (Proper or common name of product):

Specific indication(s) sought:

In many instances, a single drug may be authorized for many indications. ACPE status, however, will only be granted on the basis of an applicable indication(s). The sponsor is requested to present only the most compelling rationale for Advance Consideration. List only the indication(s) for which ACPE status is being sought. If ACPE status is granted, only the indication(s) for which Advance Consideration has been granted should be included within the submission.

Regulatory decision or application status in foreign jurisdictions:

In a tabular format, indicate if the drug is authorized in other jurisdictions, including date of authorizations, and if any accelerated approval or conditional authorizations have been granted. Include details of authorized indications.

Indicate if the drug was denied authorization in other jurisdictions, including date of denial. Include details of the proposed indication and reasons for denial.

Indicate whether the product is currently under review for the proposed indication in other jurisdictions and the anticipated decision date, if known.

If meetings were held in other jurisdictions, provide a summary of the advice or discussion as an appendix.

Proposed patient population:

Eligibility criteria:

Complete criterion A and at least 1 of criterion Bi or Bii.

To avoid duplication, hyperlinking to relevant sections of the pre-submission package is recommended.

Criterion A: The drug submission being filed for review is for a drug intended to diagnose, treat, mitigate or prevent a serious or severely debilitating disease or condition.

Provide an overview of the disease or condition and therapeutic options available in Canada. Discuss the clinical context within which the drug will be used to support the request. Describe how the drug will contribute to the clinical management of the disease or condition.

Criterion B(i): The available data demonstrate the drug has the potential to provide effective treatment, prevention or diagnosis of a disease or condition for which no other drug has been assigned a Drug Identification Number (DIN) that has not been cancelled.

Describe how the drug fulfills an unmet medical need for treatment, prevention, or diagnosis of the disease or condition. Clearly indicate that no other drug in Canada which has been assigned a DIN that has not been cancelled is approved for the same conditions of use.

Criterion B(ii): The available data demonstrate the drug has the potential to provide a clinically meaningful improvement in benefit and/or reduction in risk, such that the overall benefit-risk profile is improved over that of existing drugs that have been assigned a DIN that has not been cancelled in Canada, that are currently available to manage the serious or severely debilitating disease or condition.

Describe how the drug provides a clinically meaningful improvement in efficacy or safety, such that the overall benefit-risk profile is more favourable than other drugs for which a DIN has been assigned but not cancelled in Canada.

Clinical evidence:

Include the following:

1. Description of the study to be submitted including, but not limited to: design, enrolled population, primary and key secondary endpoints, key safety findings, and the number of patients withdrawn due to safety concerns or lack of efficacy. If evidence is derived from a surrogate endpoint(s), provide justification and validation as to how it predicts a meaningful clinical outcome. Where applicable, it is recommended that references be included to support the validation of surrogate endpoints and their relationship to clinical outcomes.
2. Description of findings demonstrating statistically significant and/or clinically meaningful results to support the claim for efficacy and/or safety. If results are based on interim analyses, provide anticipated dates for availability of the final results.

Confirmatory studies:

Include the following:

• The status of any relevant ongoing and planned studies, and indicate if any of these studies are intended to verify the benefit-risk profile of the drug for the proposed indication.
• Provide justification on why these studies are considered confirmatory for the proposed indication.
• For each such study, include a brief outline of the design and anticipated timing of study completion.

References:

Generally, up to 12 supporting key references may be included as part of this ACPE request to facilitate the review. However, this will be assessed on a case-by-case basis. All references should be provided as an appendix to the ACPE request.

Additional Information:

• Anticipated timeline for drug submission and then marketing in Canada if authorized:

Note: An aligned review process with health technology assessment (HTA) agencies (for example, Canada's Drug Agency [formerly CADTH] and Institut national d'excellence en santé et services sociaux [INESSS]) is available as a tool to reduce the time between authorization and HTA reimbursement recommendations.

Aligned reviews between Health Canada and health technology assessment organizations