Question Period Note: Regulating pediatric medicines

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• Access to medicines for children in Canada has been a long-standing issue, and is a global challenge. Health Canada is working with national and international partners to improve both access to and the regulation of medicines for children.
Access to Drugs for Children
• Health Canada prioritizes the review of drugs addressing unmet needs, including drugs for children, with a target review time of 180 days compared to the regular timeframe of 300 days.
• In 2020 and 2021, Health Canada authorized 17 new drugs for use in children in 2020 and 10 additional new drugs in 2021. Health Canada recognizes that more needs to be done to increase access to safe and effective medicines for children, and is working with key stakeholders to make this happen.
• In June 2021, Health Canada launched a Pediatric Drug Action Plan, with the ultimate vision that children in Canada have access to the medicines they need in age-appropriate formulations. A number of policy and regulatory actions are currently being implemented.
Rare Diseases and Children
• Rare diseases are life-threatening, seriously debilitating and sometimes chronic in nature. Approximately 50% of people affected by rare diseases are children, and 70% of rare diseases with a genetic basis start in childhood.
• Canada’s national strategy for drugs for rare diseases, led by Health Canada, which will be launched later this year, would help to ensure patients have improved access to effective rare disease drugs, leading to better health outcomes.

IF PRESSED ON ACCESS TO MEDICINES FOR CHILDREN …
• Health Canada is committed to increasing access to drugs for children by implementing regulatory measures similar to those that exist in other jurisdictions. For example, Health Canada is looking to compel pharmaceutical companies to conduct pediatric studies on medicines that are expected to be used in pediatric populations, and to submit those data to Health Canada for approval. A pilot to implement the pediatric regulation in late 2023 is currently under development.
• Health Canada is also working closely with the Canadian pediatric medical community to develop a draft list of priority medicines to include in Canada’s first National Priority List of Pediatric Drugs. , Once finalized, this list will help to notify industry stakeholders of the areas of urgent need for pediatric patients in Canada and will help to encourage increased drug submissions and development.

Health Canada Initiatives to Increase Overall Access to Drugs for Children:

Access to medicines for children in Canada has been a long-standing challenge. Up to 80% of drugs prescribed to children in Canada is done off-label (i.e. without safety or efficacy data in the pediatric population) because pharmaceutical developers tend not to focus their research on children.

To address this gap, the Department has developed a Pediatric Drug Action Plan (PDAP) with the ultimate vision that children and youth (0-17 years) in Canada have access to the medicines they need in age appropriate formulations. There are a number of specific actions within the plan that are tied to the its 3 main goals:
• Increasing the development of pediatric medicines and formulations;
• Improving access to pediatric medicines and formulations; and
• Providing more information to Canadians.

Pediatric stakeholders across the country (i.e., academics, clinicians, patient organizations, Health Technology Assessment organizations and industry) were consulted regularly throughout the development of the PDAP, and continue to be engaged through early implementation. The pediatric stakeholder community are broadly supportive of the goals and focus areas of the PDAP.

Implementation of various activities within the PDAP has begun, and will continue over the next 5 or more years. Current priorities include:

• Developing a “pediatric regulation” (pediatric provisions within the Food and Drug Regulations) that will bring Health Canada into alignment with other jurisdictions in requiring pediatric studies to be completed and submitted when it is expected that a drug will be used in the pediatric population;
• Assessing the impact of our current pediatric incentive (see below), and exploring other incentive models to bring more pediatric products to Canada; and
• Working with pediatricians across Canada to develop a National Priority List of Pediatric Drugs that are needed for the pediatric population. Health Canada will identify the regulatory pathways and flexibilities that can be offered to bring these products to Canada.

Health Canada is looking to include the new “pediatric regulation” in the Food and Drug Regulations as part of its Agile Licensing Framework expected to move forward in 2024-2025. These new pediatric provisions will be tested via a policy pilot scheduled for launch in 2023.

Current Pediatric Incentive: Under the current regulations, all manufacturers of innovative drugs are provided with a guaranteed minimum period of eight years of market exclusivity. If a sponsor provides clinical trial data that increases the knowledge about the use of the drug in pediatric populations, regardless of whether the data results in a pediatric indication, an additional six months may be added to this period of market exclusivity if the data is found to be acceptable by Health Canada. The sponsor may submit this information at any point during the first five years of the eight year period of market exclusivity in order to obtain the six month extension.

National Priority List of Pediatric Drugs (NPLPD): The purpose of the list will be to highlight the pediatric therapies most urgently needed in Canada, and to work with industry stakeholders to support the submission and approval of these products. The NPLPD will be developed by Health Canada based on the expert advice from the pediatric medical community.
Because improving access to medicines for pediatric populations is a global and multi-faceted issue, in order to address the challenges and barriers, Health Canada will work across the health and other government departments, and with our external national and global partners, on key initiatives. This collaborative approach will take time, but is already underway through, for example:
• Regulatory Cooperation Forum: the European Medicines Agency and Health Canada are working together on a pediatric workplan that aims to align regulatory approaches to pediatric medicines in both jurisdictions
• World Health Organization – Paediatric Regulatory Network: brings together regulators from across the world to address global issues of access to medicines for children. Health Canada is the co-chair of this network.

DRUGS FOR RARE DISEASES
Patients with rare diseases generally have few treatment options, resulting in unmet clinical need. Accordingly, treatments are in high demand and can command high prices. The pharmaceutical treatments for rare diseases are often referred to as orphan drugs, or expensive drugs for rare diseases, or drugs for rare diseases.

High prices are often attributed to factors such as the high cost of research, limited number of patients, small market size, and lack of competitors.

The Government of Canada is working with provinces and territories as an active member of the pan-Canadian Pharmaceutical Alliance (pCPA) to combine the governments’ collective buying power to negotiate lower prices on brand name drugs for all public plans, including drugs for rare diseases.

Budget 2019 proposed to invest up to $1 billion over two years, starting in 2022-2023, with up to $500 million per year ongoing, to help Canadians with rare diseases access the drugs they need. We are working with provinces, territories and other partners willing to move forward to develop a national strategy for drugs for rare diseases to be launched in 2022.

SPECIFIC CHALLENGES FOR CHILDREN WITH RARE DISEASES
The four main challenges that are exacerbated for Canadian children with rare diseases and their families are: (1) the inadequate receipt of pediatric data, particularly in comparison to counterparts in the US and Europe; (2) barriers to pediatric clinical trials; (3) economic challenges associated with developing, marketing, and maintaining a stable pediatric drug supply; and (4) current widespread use of off-label medications in children.

Economic, ethical, regulatory, and infrastructure considerations associated with the conduct of child health research have limited the numbers of clinical trials conducted with children. There are several challenges specific to conducting clinical trials with a pediatric population with rare conditions that have contributed to current gaps in the evidence base on medicines for children. These challenges include the difficulty to obtain a sufficient sample size to design and execute a well-powered clinical trial, and the lack of special methodological and analytic expertise required to conduct innovative and adaptive study types.

Despite these challenges, there is growing recognition of the undesired consequences of the vast gap in knowledge, such as a lack of information to guide dosing, inappropriate formulations, less than optimal clinical and patient-oriented outcomes, and issues related to transitioning into adulthood.

• Health Canada is responsible for regulating all therapeutic products, including medicines for children.
• Health Canada has mechanisms in place to prioritize the review of medicines addressing unmet needs for children.
• During the pandemic, Health Canada expedited the review of several vaccines and treatments for COVID-19, some of them for use in children.
• Providing access to drugs for children remains a priority for Health Canada. Currently, the majority of drugs prescribed to children are used off-label, meaning outside of the approved use of the drug.
• Health Canada has an action plan in place to increase access to needed drugs, including appropriate formulations that were specifically developed and studied in children.