What we heard: Agile regulations for advanced therapeutic products and clinical trials
On this page
- Feedback on the advanced therapeutic product (ATP) pathway
- Feedback on concierge service to support the ATP pathway
- Feedback on clinical trial modernization
- Staying connected and learning more about regulatory innovation
We have undertaken extensive consultations and environmental scanning. The Health and Biosciences Sector Regulatory Review gave us an opportunity to further understand regulatory barriers to innovation in Canada. This allowed us to define a plan for more agile regulations that can keep pace with new:
- emerging technologies
The Health and Biosciences Sector Regulatory Review Roadmap outlined a number of regulatory modernization commitments. These included plans to modernize the regulations for clinical trials and advanced therapeutic products (ATPs).
We asked for feedback on these modernization efforts by posting a discussion paper online from June to September 2019.
We asked stakeholders 3 questions:
- What products would you want us to put forward for consideration under the ATP pathway?
- Can you suggest any good models of an enhanced client “concierge” service that would help companies wishing to be considered for the ATP pathway?
- What recommendations do you have for creating clinical trials regulations that would attract clinical trials to Canada and be an effective model in the Canadian context?
Feedback on the Advanced Therapeutic Product (ATP) Pathway
Health product innovations – including new methods of design, manufacture and use – are challenging our existing regulations. These include ATPs, drugs, devices, or a combination of both so novel, complex, and distinct that the current regulations aren’t equipped to handle them. Many blur the lines between traditional product classifications.
In addition, the growing number of personalized medicines can present regulatory challenges. For example, cell therapies prepared at the bedside and given to a specific patient for whom they are designed. These products fall outside the standard health care model, where drugs are:
- manufactured in a factory environment
- distributed for broad use by health care professionals
As part of our regulatory innovation plan, we established a new pathway for ATPs. This follows the addition of new legislative provisions to the Food and Drugs Act (FDA) in June 2019. The new pathway will permit authorizing ATPs in a flexible and risk-based manner, with requirements tailored to ATPs’ unique characteristics.
We will only use the pathway when existing regulatory frameworks under the FDA cannot accommodate a product appropriately. Health Canada will consider potential candidates on a case-by-case basis, guided by criteria in the new legislative provisions. We will continue to regulate most drugs and devices under existing frameworks.
To help develop this new pathway, we asked stakeholders what products they would put forward for consideration. The most common product suggestions were related to:
- gene and cell therapies, such as those manufactured at point of care
- 3D printing, such as bio-printed products or 3D printing at point of care
- artificial intelligence, such as products using machine learning algorithms
Additional suggested products included:
- tissue engineered products
- personalized cancer vaccines
- various digital health products
- products using nanotechnology
- antimicrobials based on human microbiota and phage therapies
Stakeholders gave additional advice on how we might prioritize products for the ATP pathway. Their recommendations included prioritizing products:
- with promising evidence of efficacy in other jurisdictions
- that may not have a large financial incentive to commercialize
- that possess the potential to significantly improve the health of Canadians
Feedback on concierge service to support the ATP Pathway
Innovators and industry will need guidance to navigate the new ATP pathway. We are planning to develop a specialized “concierge” service to better serve this need. In responding to the discussion paper, stakeholders discussed what they would like to see in this service.
Stakeholders recommended an equitable approach between small and large innovators. Small innovators may need more time and resources to understand and meet regulatory information and submission requirements. Crucially, the concierge should guide innovators through the entire ATP process and provide assistance in gaining market access.
Stakeholders further recommended a concierge with intimate knowledge of the ATP pathway and its criteria. The concierge should be able to:
- leverage this knowledge to achieve regulatory agility
- educate innovators about the flexibilities available through existing regulatory pathways
- clearly communicate intersections between the ATP pathway and Health Canada’s traditional regulatory framework
Stakeholders suggested that the concierge operate as a free and accessible single window. Contact should be through human interaction, like a phone line or central online portal.
Engagement from stakeholders
The concierge should liaise with stakeholders who support or are part of the development and market access of ATPs. This includes engaging with others in the Canadian health and innovation ecosystem, such as:
- research ethics boards
- International counterparts
- health technology assessment bodies
The concierge would also liaise with the various pre- and post-market areas of Health Canada, including those responsible for:
- establishment licensing
- post-market compliance
- pre-clinical and clinical trials advice
- science and statistical methods advice
Other concierge models
Stakeholders submitted models of concierge services from Canada and other jurisdictions for review. These included:
- European Medicines Agency: Innovation Task Force
- National Research Council Canada: Industrial Research Assistance Program
- Japanese External Trade Organization: One-Stop Business Concierge Service
- U.S. Food and Drug Administration: Small Business and Industry Assistance Program
- Innovation, Science, and Economic Development Canada: Accelerated Growth Service
- U.S. Food and Drug Administration: Regenerative Medicine Advanced Therapy Designation
As we develop the concierge service for the ATP pathway, we will:
- learn from the diverse programs submitted
- leverage relevant information where applicable
- review and evaluate each of the recommendations
Feedback on clinical trial modernization
Clinical trials are an important first step in health product development. Due to evolving product complexity, there is a need for more flexible approaches to clinical trial types and designs. Studies increasingly focus on individual treatments rather than groups, with new technologies and data changing how studies are conducted. We are working to modernize our regulations to increase flexibility and risk-based oversight to:
- better address the evolving clinical trial environment
- ensure continued protection for participant safety
We asked stakeholders for recommendations on how to create regulations that would:
- attract clinical trials to Canada
- be an effective model in the Canadian context
Stakeholder input centred on several key themes.
Stakeholders recommended greater flexibility to accommodate innovations in how trials are conducted. This flexibility would also better meet patient needs to help them to participate in trials. Examples of how to achieve this included:
- greater trial transparency
- adaptive trial designs, such as:
- bandit trials
- basket trials
- platform trials
- umbrella trials
- improving the consent process to reduce patient drop out
- better leveraging of technology, like platforms that enable remote trial participation
Stakeholders highlighted risk-based oversight as an approach that could foster more clinical trials in Canada. This oversight would be based on less stringent regulatory requirements and reduced administrative barriers. For example, a risk-based approach to audit activities would:
- support patient safety
- support high-quality clinical trials
- help to avoid imposing unintentional barriers to entry into the Canadian clinical trials market
Stakeholders further discussed how investigator-initiated trials with marketed therapeutics for off-label uses might be considered lower risk. They also discussed not being subject to the same application and reporting requirements as a new trial.
Assisting clinical trial conductors
Stakeholders suggested enhancing the assistance Health Canada provides to innovators who conduct clinical trials. This could take the form of education on trial processes and requirements, as well as guidance on:
- trial design
- review timelines
- statistical methods
- manufacturing, including:
- site licensing
- temperature control
- closed versus open manufacturing
Stakeholders also recommended increased international alignment and harmonization. This would:
- reduce duplication
- bolster the consistency of trials across borders
- prevent delays and barriers to trials designed for global reach
Stakeholders asked Health Canada to continue to enhance its work as a central coordinator in the Canadian health innovation ecosystem. This will continue to foster connections between stakeholders and further collaboration.
Staying connected and learning more about regulatory innovation
The feedback we received from stakeholders throughout consultations will continue to inform Health Canada’s work as:
- we implement these modernization efforts
- new regulatory approaches are designed and developed
Stay connected and learn more by visiting Health Canada’s Regulatory Innovation Agenda webpage.
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