A guide to the new disaggregated data questionnaire for drug submissions
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Health Canada uses disaggregated data to analyze the safety and efficacy of health products. This data allows us to:
- verify that clinical trial participants reflect the diversity of the types of people who will use the product
- analyze the safety and efficacy of health products by subpopulation, where feasible
- indicate where post-market monitoring might be needed to verify safety and efficacy within certain populations
Lack of diversity in clinical trials can result in situations where the benefits and risks of certain health products are unclear for under-represented populations. These population groups often include women, gender-diverse people, racialized and ethnic subpopulations, and specific age groups.
To help us determine which regulatory submissions include disaggregated data, we have created a list of questions for sponsors. You will find these questions in the product information template that is part of the regulatory enrolment process. If sponsors indicate they have clinical evidence supporting their submission, they will then be asked 4 extra questions on sex, age and race or ethnicity (please refer to Annex I).
The questionnaire is the first step in a multi-phase approach to collecting disaggregated data from drug submissions. Other phases will include amending regulations and working internationally to promote the need for greater diversity when a drug is in development.
This work is part of Health Canada’s Sex- and Gender-Based Analysis Plus Action Plan. Our vision is for people in Canada to have access to information so they can make the best decisions about their treatment options based on the safety and efficacy profiles of people like them.
The purpose of this guide is to help sponsors complete the submission questions on disaggregated data.
Explaining the questionnaire
The purpose of these questions
The collection and analysis of disaggregated data in clinical trials is key to scientific rigour. This data:
- confirms that the diversity of clinical trial participants reflects the population groups that will use the product
- is used by sponsors and Health Canada to look at a drug’s safety and efficacy by subpopulation, where feasible
The data may also indicate if we need to verify a product’s safety or efficacy in certain populations through post-market monitoring.
Health Canada is asking sponsors to answer 5 questions on disaggregated data in clinical trials. The questions relate to all of the clinical evidence in a submission, not to individual clinical trials. We want to establish a baseline for the percentage of applications that include disaggregated data. This is an important first step in validating that health products have been tested on diverse clinical trial participants.
Responses to the 5 questions will not be used in the official review of the submission.
When questions should be completed
Sponsors should answer the questions on whether data is disaggregated by sex, age and race or ethnicity when submitting an application with clinical evidence for market approval of a drug. In other words, they should answer the questions if clinical trials were undertaken in product development and testing.
What is disaggregated data
Disaggregated data reflects smaller subpopulations. It can be used to analyze the effect of a product between populations.
The standards for disaggregated data are often specific to the country and topic.
For example, the U.S. Food and Drug Administration’s race and ethnicity categories represent those populations that are relevant to the United States:
- Black or African American
- Native Hawaiian or Pacific Islander
- American Indian or Alaskan Native
However, it would make sense to consider additional population groups in other parts of the world.
Statistics Canada uses the following data categories to report on groups in Canada:
- Latin American
- West Asian (for example, Iranian, Afghan)
- South Asian (for example, East Indian, Pakistani, Sri Lankan)
- Southeast Asian (or example, Vietnamese, Cambodian, Laotian, Thai)
Statistics Canada also asks about Indigenous identity (First Nations, Inuit and Métis).
Application to rolling submissions
Sponsors should answer questions based on the planned enrolment and data collection for submissions filed in accordance with the following guidance for COVID-19:
If the disaggregated data for safety and effectiveness is not statistically significant
Many clinical trials are not powered to detect differences in safety or efficacy of a drug between subpopulations.
However, it’s important to undertake a comparative analysis and to validate that there is no clinically meaningful evidence on the safety or efficacy of a health product in different subpopulations. This type of comparative analysis may identify areas where more testing or collecting evidence once a product is on the market is warranted.
There’s no need to pool data in order to undertake any analyses on safety or efficacy in order to complete this survey.
Why we don’t ask about gender
'Sex' and 'gender' are often used in place of each other. However, they have different meanings. The Canadian Institutes of Health Research defines them as follows:
- Sex refers to a set of biological attributes in humans and animals. It’s mainly associated with physical and physiological features, including chromosomes, gene expression, hormone levels and function, and reproductive or sexual anatomy. While ‘sex’ is usually categorized as female or male, the biological attributes that comprise sex and how those attributes are expressed vary.
- Gender refers to the socially constructed roles, behaviours, expressions and identities of girls, women, boys, men and gender-diverse people. It influences how people perceive themselves and each other, how they act and interact, and the distribution of power and resources in society. Gender identity is not confined to a binary (girl/woman, boy/man) nor is it static. It exists along a continuum and can change over time. There is considerable diversity in how individuals and groups understand, experience and express gender through the roles they take on, the expectations placed on them, relations with others and the complex ways that gender is institutionalized in society.
The current practice in clinical trials is often to ask about the biological variable of sex (male or female). This is why our question only refers to sex.
If the categories for race/ethnicity are different from the Canadian context or if the submission includes multi-site clinical trials with multiple categories for race/ethnicity
At this time, Health Canada is only asking if race or ethnicity data has been collected in a disaggregated manner. What’s important is that the clinical trial participants reflect the diverse populations that will use the product. The race or ethnicity categories that are used will depend on:
- the country in which the trial takes place
- if there are specific populations that are more likely to use the product
Access to medicines for children in Canada has been a challenge for some time. Many drugs prescribed to children in Canada are used off-label. This means there is no data on the product’s safety or effectiveness in the pediatric population.
Health Canada has developed a Pediatric Drug Action Plan to ensure children and youth in Canada have access to the medicines they need in age-appropriate formulations. The collection of this data will help support the implementation of this action plan by providing a baseline for the number of submissions that include pediatric populations.
If only some of the trials include disaggregated data
If only some of the clinical evidence in your submission is in a disaggregated form, sponsors should still select “yes” to the relevant question.
Our overall goal is to have all clinical evidence disaggregated by subpopulations, especially in pivotal clinical trials. This may not always be possible for some studies or submissions.
If we do not provide responses to the questions
For an initial two-month period, starting from the implementation of the questions on October 3, 2022, sponsors will be encouraged to provide responses. As of December 1, 2022, sponsors must answer all the questions when submitting their application. Following December 1, if an application is submitted without responses, the submission will be screened out. Note: The first question asks whether there is clinical evidence to support the application. If the answer is no, the remaining 4 questions will not appear.
If our clinical trial data is not disaggregated
The collection and analysis of disaggregated data in clinical trials is an important component. The data confirms that the diversity of clinical trial participants reflects the populations that will use the product. It also makes it possible for sponsors and Health Canada to assess a product’s safety and efficacy by subpopulation, where feasible, and may point to a need to monitor the product after it’s on the market.
As with all health product submissions, Health Canada will apply standard review practices. Reviewers who have concerns about the data will contact the sponsor.
Explanation for answers
You will not be able to provide text for the yes/no checkboxes. However, you may include information in your submission, which our scientific evaluators will review as part of their standard practice in the usual manner.
Questions will be standard
Given its importance in health product development, we will continue to ask about disaggregated data. The questionnaire is the first step in a multi-phase approach to collecting disaggregated data from submissions.
Health Canada expects a regulatory amendment to ensure international alignment for disaggregated data requirements by mid-2023. Additional phases may bring other requirements.
If you have any questions, please contact the Centre for Policy, Pediatrics and International Collaboration by email at email@example.com.
Annex 1: Data disaggregation questions in product information forms
|Does this submission include clinical trial data?
|Are the clinical trial data in your submission disaggregated by sex? (male/female)?
|Are the clinical trial data in your submission disaggregated by age?
|Are the clinical trial data in your submission disaggregated by race/ethnicity?
|Does this submission include clinical trial data from pediatric populations (those under 18 years of age)?
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