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Thursday, January 18 2018
Press release

Commentary to Fee Proposal for Drugs and Medical Devices

OTTAWA, January 3, 2018 – The Goodman Pediatric Formulations Centre (GPFC) and the KidsCAN Clinical Trials have partnered to provide a commentary regarding the proposed Health Canada cost-recovery fee proposal.

The GPFC has the goal of improving access to child-friendly medicines. We are the only Centre in Canada whose mandate is to facilitate the development of safe and effective age-appropriate formulations for children. The GPFC operates as a not-for-profit organization, whose exclusive goal is to support the well-being of children by facilitating the availability of formulations adapted to their needs. Even though the GPFC works closely with hospitals, health care providers and industry, our positions and actions are completely independent of these third parties.

KidsCAN Trials is a national hub to coordinate research, training and knowledge transfer in the safe therapeutic use of medicines for children. By federating all academic child health centres and their affiliated research institutes, KidsCAN Trials will encourage new processes and practices, new policy approaches and new ways of organizing care for children. A video is provided to outline the importance of clinical trials from a patient perspective.

The relative lack of availability of pediatric dosage forms can lead to treatment failure or adverse events for children. Approved adult forms often need to be modified in some manner to administer the desired dose to children, and as such, are used off-label. Compounding is the process by which an adult form is manipulated by a health care provider, or others, to adapt the adult form to be used in children. Compounding is regulated by provincial pharmacy standards, based on Guidelines published by the National Pharmacy Regulatory Authorities (NAPRA) on behalf of pharmacy colleges. NAPRA guidelines admittedly falls short of Health Canada’s Good Manufacturing Practices (GMPs) in multiple ways and, even though compounding may be acceptable in some circumstances, it should not be considered an equivalent surrogate for a Health Canada approved formulation that has been assessed for use in children. Given that compounded medicines are not approved in full compliance with Health Canada’s rigorous requirements, the medication’s characteristics and specifications are not always known, well established or controlled: such as the stability, potency, content uniformity, purity (chemical and microbial) or the bioavailability of the compounded product in children. More importantly, that the appropriate dose is being administered is not always known. Moreover, if the compounded medicine has an unpleasant taste, then compliance with the child can be challenging. Although the exact percentage of compounding used in children is unknown, a common understanding is that 75% of all compounded prescription medications may fall outside of regulatory approval1. Research and clinical trials can elucidate and increase the understanding in these areas and are a vital part of improving child-friendly formulations to Canadian children. A video is linked to illustrate the challenges from a patient and caregiver perspective.

The ideal is to have commercial pediatric formulations available for Canadian patients. In many cases, commercialized pediatric formulations exist in other jurisdictions, such as in the United States and in Europe. However, oftentimes, these commercial pediatric formulations are not marketed in Canada, leaving an already vulnerable and unaddressed population without access to the commercially available pediatric formulations.

There are several reasons why existing commercial pediatric formulations have not been introduced into the Canadian market. Firstly, from a commercial perspective, the approximately six million children in Canada (of which only a small percentage are ill) results in a market size that is too small to build a sustainable business case.

Second, the regulatory approval pathway is perceived as unclear and there is significant concern that it will include costly additional clinical studies targeting very restricted patient populations. In the case where there is no need for further clinical studies, the regulatory costs for the preparation of a Canadian New Drug Submission utilizing an NDA or an MAA can involve considerable costs (>$100,000 to $250,000), not including the fees related to that review.

Third, once approved by Health Canada, reimbursement groups may not list these medications, as the health technology assessments (HTA) is based on criteria applicable to adult forms, which may not apply, or be possible, in children. Therefore, reimbursement for a pediatric formulation may be rejected.

Fourth, unlike in the United States and Europe, there are no commercial incentives or regulatory protection granted to bring commercialized pediatric formulations to Canada, resulting in American and European children having access to pediatric formulations whereas our Canadian children can only have the compounded equivalent, with the significant limitations and risks that this may bear.

Finally, the Orphan Drug Framework, which was an area in which some pediatric medicines could have worked, has now been stopped, leaving no options for special accommodations for pediatric medicines.

The GPFC and KidsCAN have read Health Canada’s “Fee Proposal for Drugs and Medical Devices” and is responding to this proposal by submitting this letter. The GPFC and KidsCAN understand that other regulatory jurisdictions have a full cost recovery fee structures for the various services offered to review, or allow the sale of medications. Given the inaccessibility of commercialized pediatric formulations in Canada that already exists, we can only expect that this new proposed fee structure would further exacerbate the significant existing problems for pediatric formulations. As cited in the Fee Proposal, costs associated with a New Drug Submission will change from $173,106 to $312,562, or an 80% increase. In pediatrics, this will augment the barrier to provide Canadian children access to commercially available pediatric formulations that are available elsewhere.

The GPFC is a non-profit organization that is working as a facilitator between industry, regulatory and reimbursement agencies to bring commercialized pediatric formulations into Canada. It is already a difficult and challenging situation for the reasons cited above. KidsCAN is also a non-profit organization whose aim is to facilitate pediatric clinical trials and research by centralizing these activities in Canada. Building on an existing mitigation strategy, we would like to submit for your consideration that fees associated with the submission of pediatric formulations could be granted a waiver when the fee exceeds a certain percentage of the anticipated Canadian pediatric market. Alternatively, perhaps the pediatric formulations approvals could use the already proposed medical urgent need framework outlined in the Proposal. Such a waiver would not be expected to impact significantly on Health Canada’s cost recovery needs, while providing drug developers with an incentive to consider introducing a pediatric formulation where a need exists. This measure would also stand as an acknowledgement by Health Canada of the need for more pediatric formulations and better drugs for children.

The challenge facing pediatrics in Canada is real and unique, and the GPFC and KidsCAN would therefore respectfully recommend using a waiver or specialized fee remission schedule specific for pediatric medications (both for new indications and new formulations). The GPFC could offer its expertise to help design a fee structure that would approach being acceptable to industry while trying to meet the cost recovery needs of Health Canada.

Thank you for consideration in this matter. We encourage you to contact the Goodman Pediatric Formulation Centre, who is taking the lead on this initiative, at any time to discuss this multifaceted issue. It would be our pleasure to work with Health Canada to find a solution that takes into consideration this complex area of regulatory approval of pediatric medications.

Best regards,
 

Andrea Gilpin, Ph.D, MBA 
General Manager, GPFC
3175 chemin de la Côte Sainte-Catherine, Office 8229 
Montreal, Quebec, H3T 1C5 
514 465-2114
agilpin.cfpg@gmail.com
Thierry Lacaze, MD, Ph.D., FRCPC
Neonatologist, Professor of Paediatrics
Cummings School of Medicine, University of Calgary
KidsCAN Trials Lead
kidscantrials.ca
Catherine Litalien, MD, FRCPC
Pediatrician
Associate Clinical Professor
Executive Director, GPFC
goodmanpedformulations.ca
 

 

1 Improving Medicines for Children in Canada, 2014, Council of Canadian Academies, pg 22.

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