A strategy to optimize the use of real-world evidence across the medical device life cycle in Canada

Health Canada and the Pan-Canadian health technology assessment collaborative^{Footnote 1}

1. Executive summary
2. Introduction
3. Activities to optimize the use of RWE across the medical device life cycle
   1. Create team
   2. Develop framework
   3. Prepare guidance
   4. Define implementation strategies
4. Conclusions
5. References

A. Executive summary

Through the optimization of real-world evidence (RWE)^{Footnote 2} use across the medical device life cycle, Health Canada and the Pan-Canadian Health Technology Assessment (HTA) Collaborative (the Collaborative)^{Footnote 3} aim to improve the accessibility, affordability, and appropriate use of medical devices.

Health Canada and the Collaborative already accept and consider RWE in their decision-making process. This strategy document outlines the steps to be taken in order to optimize the use of RWE to improve the safety and effectiveness of medical devices across the product life cycle. As well, the plan describes the activities and the outputs required to achieve the objectives. 

Key activities include: 

1. Create Team:  Create a Medical Device RWE Core Action Team (CAT) for Collaboration and Discussion: Improvements in the use of RWE across the medical device life cycle requires the perspectives and partnerships of multiple stakeholders. Health Canada and the Collaborative will establish a team to carry out activities, two to four described in the plan, and to enable a forum which will guide the development of the RWE framework (i.e., activity 2) and accompanying guidance documents. The Medical Device RWE CAT will also facilitate ongoing dialogues, so continued progress can occur.

   Final Output: Formation of the Medical Device RWE CAT

   Start Date: Summer 2019

   Co-leads: Health Canada and Canadian Agency for Drugs and Technologies in Health (CADTH)

2. Develop Framework:  Develop a RWE Framework^{Footnote 4} that Defines Points across the Medical Device Life Cycle where RWE can be Useful: The purpose of the framework is to specify the type of regulatory decisions or HTA^{Footnote 5} recommendations for which RWE is a reasonable or complementary data source.

   Final Output: RWE framework for medical devices

   Start Date: Fall 2019

   Co-leads: Health Canada and CADTH

3. Prepare Guidance:  Prepare Guidance^{Footnote 6} Documents on the Generation of RWE for Medical Devices: The quantity and type of evidence required to make decisions across the medical device life cycle vary depending on the context. The guidance documents will help to clarify the expectations related to optimizing real-world data collection, accessibility, sharing, analytic approach, and use to support regulatory decisions and HTA recommendations.

   Final Output: Guidance documents on the generation of RWE for medical devices - Fall 2019 and ongoing.

   Start Date: Fall 2019

   Co-leads: Health Canada and CADTH

4. Define Implementation Strategies:  Define Implementation Strategies to Support the Use of RWE across the Medical Device Life Cycle Based on the RWE Framework and Guidance Documents: To build on Canada’s current experience in the collection, interpretation, and use of RWE, HEALTH CANADA and the Collaborative will operationalize the principles of RWE use for medical devices as described in the framework and guidance documents.

   Final Output: Implementation strategies to operationalize RWE use for medical devices

   Start Date: Spring 2019 and ongoing

   Co-leads: Health Canada and CADTH

B. Introduction

Health Canada and the Pan-Canadian Health Technology Assessment (HTA) Collaborative (the Collaborative)^{Footnote 7} aim to improve the accessibility, affordability, and appropriate use of medical devices in Canada through optimization of the use of real-world evidence (RWE)^{Footnote 8} across the medical device life cycle. This project was launched as part of Health Canada’s Regulatory Review of Drugs and Devices (R2D2) initiative.

In December 2018, HEALTH CANADA released the Action Plan on Medical Devices entitled, Continuously Improving Safety, Effectiveness, and Quality. The plan briefly outlines a three-part strategy to continue to improve the safety, effectiveness, and quality of medical device use in Canada. The three parts were as follows: to improve how medical devices are introduced to the market; to strengthen post-market monitoring and follow-up of devices on the market; and to provide more information to Canadians about clinical studies, inspections, incident reports, and device approvals. The objectives of the 2018 Action Plan align with the goals of the current project to optimize the use of RWE for medical device decision-making.

Real-world data (RWD)^{Footnote 9} sources are increasing in quantity and quality, and it is also recognized that these data sources can provide useful and complementary information for the enhancement of decision-making with respect to safety, effectiveness, utilization, and treatment patterns. Furthermore, data gathered in real-world settings has the potential to be a powerful tool within the HTA^{Footnote 10} process and when making decisions about the use of medical devices.

To optimize the use of RWE, there is a pressing need for validity in its generation, appraisal, interpretation, and application. Alignment among stakeholders, including industry, regulatory bodies, HTA agencies, payers, as well as health care providers and patients, is required on the decision points across the medical device life cycle for which RWE is a valuable and an acceptable source of evidence. Data gathered in real-world settings can help to provide insights into health product development and utilization. In addition, data quality measurements, such as accuracy, validity, and completeness, as well as the context in which RWD were collected, can shed some light on the appropriateness of RWE for regulatory decisions and HTA recommendations. To help ensure the validity of the evidence generation process, it is important to pose relevant scientific questions that are characterized by a well-defined estimand^{Footnote 11}, develop and register a detailed protocol, and conduct rigorous statistical analyses.

Despite the potential benefits of RWE, it is not without its drawbacks. Using data gathered in real-world settings presents a number of issues including:

• increased risks for bias, confounding, and variability;
• increased risks regarding issues of privacy and data security;
• challenges related to data accessibility, data sharing, and data standards;
• ability to collect appropriate and valid outcomes on safety and effectiveness; and
• challenges related to tracking devices in use in Canada.

It is imperative that Canada characterizes the use of RWE with an explicit understanding of these challenges. Factors that affect the quality of the evidence should be comprehensively evaluated to consider if the collection of RWD and if the generated evidence will be useful to inform regulatory decisions or HTA recommendations at various points in a product life cycle.

Health Canada, the Canadian Agency for Drugs and Technologies in Health (CADTH), and l’Institut national d'excellence en santé et services sociaux are also working together to optimize the use of RWE across the drug life cycle. Information sharing and exchanges, therefore, will occur on a regular basis.

This strategy document aims to outline the activities that HEALTH CANADA and the Collaborative will undertake to optimize the use of RWE across the medical device life cycle to support regulatory decisions and HTA recommendations. HEALTH CANADA and CADTH will lead or co-lead the activities described in the plan, but the Medical Device RWE Core Action Team (CAT) will be consulted periodically to provide support and guidance. To contextualize the optimization of RWE use, the strategy presents the “who”, “what”, “when”, “why”, and “how”.  More specifically, Medical Device RWE CAT will be the “who”, the framework will discuss the “what, when and why”, and finally, the guidance documents and implementation strategies will focus on the “how”. The outcomes of the activities described are intended to both maximize the benefits and minimize the risks for Canadians and provide further support for our health care system.

C. Activities to optimize the use of RWE across the medical device life cycle

1. Create team

Create a medical device RWE core action team for collaboration and discussion

Improvements in the use of RWE across the medical device life cycle require the perspectives of and partnerships with different stakeholders. Health Canada and the Collaborative will create a Medical Device RWE CAT to carry out the activities described in the strategy and to facilitate continued progress through ongoing dialogues with stakeholders.

By creating a Medical Device RWE CAT, the collective efforts of many partners across Canada will be harnessed towards the overall aim of improving the accessibility, affordability, and appropriate use of medical devices. The Medical Device RWE CAT will be supported by working groups that will be formed to address key challenges that limit the optimal use of RWE across the medical device life cycle in Canada. Details on the membership are still to be determined, but it can include regulators, payers, the clinical community, health care facilities, industry, patient groups, research institutions, and academia.

Final Output: Formation and creation of the Medical Device RWE CAT

Start Date: Summer 2019

Co-leads: Health Canada and CADTH

2. Develop framework

Develop a framework^{Footnote 12} that defines points across the medical device life cycle where RWE can be useful

To optimize the use of RWE across the medical device life cycle, the context in which RWE is considered acceptable as a supportive source of information must be characterized. Through the Medical Device RWE CAT, Health Canada, the Collaborative, and all relevant stakeholders will work together to provide greater transparency across the product life cycle on the types of regulatory and HTA issues for which RWE may be applied.

Health Canada and the Collaborative will prepare a framework that will direct the development of guidance documents on the processes and methods required to optimize the use of RWE for medical devices to support regulatory decisions and HTA recommendations.

This activity will include a number of tasks. Health Canada and the Collaborative will conduct a review that attempts to identify how RWE is currently used across the medical device life cycle. This review will be based on the environmental scan completed by Health Canada in March 2019 to understand medical device RWD sources and stakeholder perspectives on the use of RWD and RWE for regulatory decisions and HTA recommendations on medical devices. The environmental scan highlights are scheduled to be posted on the Health Canada website in fall 2019. With this information, a framework will be drafted that establishes the decision points and contexts  across the medical device life cycle for which RWE would be an acceptable, or a potentially preferred, data source to support regulatory decisions and HTA recommendations.

Final Output: RWE framework for medical devices

Start Date: Fall 2019

Co-leads: Health Canada and CADTH

Milestones:

• Environmental scan to understand RWD sources for medical devices in Canada, as well as stakeholder perspectives on the use of RWD and RWE for regulatory decisions and HTA recommendations
  • Completion Date: March 2019
  • Lead: HEALTH CANADA
• Review on the current uses of RWE to assess the safety, clinical effectiveness, and cost-effectiveness of medical devices
  • Start Date: Fall 2019
  • Lead: CADTH

3. Prepare guidance

Prepare RWE guidance1^{Footnote 3} documents on the generation of RWE for medical devices

In addition to the overarching principles that will be described in the framework, there is a need for the standardization of methods and processes to guide the actual generation, analysis, and appraisal of RWE. The quantity and type of evidence required to support informed decision making across the device life cycle will vary depending on the product of interest, the population impacted, other types of existing evidence, and the nature of the decision to be made. The guidance documents are intended to provide some advice on RWD collection, accessibility, and sharing, and proposed analytic approaches to produce RWE that is of sufficient quality to support regulatory decisions and HTA recommendations. This work will build on the current guidance documents available on RWE as applied to both drugs and medical devices with a lens on what is applicable to the Canadian context.

Final Output: RWE guidance for medical devices - Fall 2019 and ongoing. There would be separate documents reflecting regulatory and HTA perspectives.

Milestones:

• Quality of RWD and RWE document that provides overarching principles to guide the generation of RWE that is applicable to both drugs and medical devices and that would be consistent with the evidence standards in place in Canada and internationally
  • Completion Date: Spring 2019
  • Lead: Health Canada
• Draft document1^{Footnote 4} on the pre-market evidence, including RWE, requirements for regulatory approval of medical devices
  • Start Date: Fall 2019
  • Lead: Health Canada

4. Define implementation strategies

Define implementation strategies to support the use of RWE across the medical device life cycle based on the RWE framework and guidance documents

To build on Canada’s current experience with the use of RWE, Health Canada and the Collaborative will work together to define implementation strategies to operationalize the principles described in the framework and guidance documents on the generation of RWE for medical devices. These strategies will include education and outreach activities among stakeholders, including regulators, industry, the clinical and HTA communities, patient groups, research institutions, and academia. Two proposed strategies will involve the expansion of the Canadian Medical Devices Sentinel Network to include additional health care settings outside of hospitals, such as long-term care facilities and private clinics, and the implementation of mandatory reporting of medical device incidents to Health Canada by hospitals. For HTA, examples include operationalizing reassessments of health technologies with RWE.

Final Output: Implementation strategies to support the use of RWE for medical devices

Start Date: Spring 2019 and ongoing

Co-leads: Health Canada and CADTH

Milestones:

• Expand the Canadian Medical Devices Sentinel Network to include additional health care settings outside of hospitals
  • Start Date: Spring 2019
  • Lead: Health Canada
• Implement mandatory reporting of medical device incidents to HEALTH CANADA by hospitals
  • Start Date: End of 2019
  • Lead: Health Canada
• Establish ability to compel information on medical device safety and effectiveness
  • Start Date: Fall 2020
  • Lead: Health Canada

D. Conclusions

Health Canada and the Collaborative are working closely together to improve the accessibility, affordability, and appropriate use of medical devices through the better use of RWE across the product life cycle. The activities described in this strategy document are as follows: i) the creation of the Medical Device RWE CAT; ii) development of a RWE framework for medical devices; iii) preparation of guidance documents on the generation of RWE for medical devices; and iv) definition of implementation strategies to support the use of RWE across the medical device life cycle. In addition to regulatory decisions and HTA recommendations, these activities will help to increase support for our health care system and will lead to improved patient care for Canadians.

E. References