Draft guidance on advanced therapeutic products framework: Overview

This guidance document provides an overview of how Health Canada designates and regulates advanced therapeutic products (ATPs) under sections 21.9 to 21.96 of the Food and Drugs Act.

On this page

• Introduction
• Scope and application
• Framework highlights
• Enhanced support for innovators
• Collaboration with interested and affected parties
• Decision-making
• Definitions

Introduction

Scientific and technological advances are accelerating the pace of change in health care. This acceleration is leading to the development of a range of innovative therapeutic products that are:

• personalized
• developed at point-of-care
• manufactured, distributed and used in ways that differ significantly from traditional health products

Health Canada administers regulations and has well-established protocols for the pre- and post-market oversight of drugs and medical devices. However, existing regulatory frameworks may not adequately address the characteristics inherent to certain innovative therapeutic products.

The absence of suitable or appropriate regulatory frameworks for such products:

• creates a barrier in bringing them to market
• limits access to new, potentially life-changing treatments

In 2019, the Food and Drugs Act (act) was amended to enable the regulation of advanced therapeutic products (ATPs).

An ATP is a therapeutic product or class of therapeutic products that represents an emerging or innovative technological, scientific or medical development. The Minister, by order, adds these types of products to Schedule G of the act.

ATPs are drugs, medical devices or any combination of drugs and medical devices that are so unique, complex and distinct that they fundamentally challenge:

• the existing regulations and
• Health Canada's ability to protect people's health

Most therapeutic products will continue to be regulated under existing regulatory frameworks.

Some therapeutic products are so unique or complex that they need a different regulatory approach. The ATP framework provides the agility to create, implement and adjust the requirements to ensure the regulations:

• are sufficiently tailored to the product
• protect the health and safety of people

Scope and application

This guidance document applies to:

• all ATPs that are or may be set out in Schedule G of the act
• the following activities related to ATPs:
  • manufacture
  • prepare
  • preserve
  • package
  • label
  • test
  • store
  • import
  • advertise
  • sell

Framework highlights

The ATP framework can be used for ATPs where:

• there is a regulatory barrier
• there is enough evidence to support the conclusion that a product's benefits outweigh its risks
• the risks associated with the product and the activity will be adequately managed and controlled following its authorization

It takes time to develop a tailored ATP pathway. An early and collaborative approach with those who make or use these therapeutic products is required when developing a tailored ATP pathway.

Health Canada will:

• work with interested and affected parties to understand the barriers in bringing these products to market
• consider what has worked well in other jurisdictions (how they have addressed innovations)

Once an ATP is described in Schedule G of the act, the tailored ATP pathway becomes an optional pathway:

• To obtain an ATP licence, submit an application to Health Canada.
  • The licence may have terms and conditions associated with it. These ensure appropriate oversight of the product's safety, efficacy and/or quality and to manage uncertainties or mitigate risks.
• Health Canada may make an order of permission that allows a class of persons to conduct an activity provided that certain terms and conditions, if any, are met. The order would authorize members of the class to undertake the authorized activities related to the ATP, provided they meet the terms and conditions.

We recognize the importance of harmonizing requirements with other key jurisdictions to reduce barriers to entry in Canada.

We are also able to make changes to requirements, if needed, while learning how to best regulate different types of products.

Enhanced support for innovators

Health Canada will give enhanced support ('concierge service') to those navigating the ATP framework. This means:

• assigning a main contact to each applicant
  • to ensure continuity and consistency on an application file
• providing support throughout the licence authorization process

Collaboration with interested and affected parties

The success of the ATP framework hinges on interested and affected parties working together with Health Canada.

Health care delivery is a shared responsibility in Canada. Health Canada intends to regularly engage with innovators and health care system partners, such as provinces, territories, health technology assessment bodies and international regulators.

Working together, we will:

• identify ATP candidates
• consult with experts
• develop a tailored pathway
  • one that addresses the complexity and unique characteristics of ATP candidates

Decision-making

The decision to add an ATP to Schedule G of the act lies with the Minister. Health Canada will consult with interested and affected parties in this scheduling process. This process will be clear and transparent so that those in Canada who want to be meaningfully engaged can be heard.

Decisions to issue an ATP licence will be based upon science and driven by evidence that is well-analyzed and well-documented.

As a regulator, Health Canada plays an important role in protecting the health and safety of people in Canada. We are committed to transparency and openness to further strengthen trust in our regulatory decisions. We will apply these principles to ATP licensing as appropriate.

Definitions

Advanced therapeutic product: A therapeutic product that is described in Schedule G or that belongs to a class of therapeutic products that is described in that Schedule (produit thérapeutique de pointe)

Advanced therapeutic product framework: The legislative provisions, guidance documents, decisions, directives, regulatory policies, guidelines, recommendations and procedures made to:

• identify advanced therapeutic product candidates
• develop tailored advanced therapeutic product pathways and
• regulate advanced therapeutic products

(cadre réglementaire pour les produits thérapeutiques de pointe)

Device: An instrument, apparatus, contrivance or other similar article, or an in vitro reagent, including a component, part or accessory of any of them, that is manufactured, sold or represented for use in:

1. diagnosing, treating, mitigating or preventing a disease, disorder or abnormal physical state, or any of their symptoms, in human beings or animals
2. restoring, modifying or correcting the body structure of human beings or animals or the functioning of any part of the bodies of human beings or animals
3. diagnosing pregnancy in human beings or animals
4. caring for human beings or animals during pregnancy or at or after the birth of the offspring, including caring for the offspring, or
5. preventing conception in human beings or animals

It does not include such an instrument, apparatus, contrivance or article, or a component, part or accessory of any of them, that does any of the actions referred to in paragraphs a) to e) solely by pharmacological, immunological or metabolic means or solely by chemical means in or on the body of a human being or animal (instrument)

Drug: Includes any substance or mixture of substances manufactured, sold or represented for use in:

1. the diagnosis, treatment, mitigation or prevention of a disease, disorder or abnormal physical state, or its symptoms, in human beings or animals,
2. restoring, correcting or modifying organic functions in human beings or animals, or
3. disinfection in premises in which food is manufactured, prepared or kept

(drogue)

Person: An individual or an organization as defined in section 2 of the Criminal Code (personne)

Tailored advanced therapeutic product pathway: The tailored regulatory requirements to import, sell, advertise, manufacture, prepare, preserve, package, label, store or test an advanced therapeutic product. Also the tailored oversight framework that would be used to monitor the advanced therapeutic product (voie réglementaire sur mesure d'un produit thérapeutique de pointe)

Therapeutic product: A drug or device or any combination of drugs and devices, but does not include a natural health product within the meaning of the Natural Health Products Regulations (produit thérapeutique)

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