The Rosalind & Morris Goodman Family Pediatric Formulations Centre of the CHU Sainte-Justine, also known as the Goodman Pediatric Formulations Centre (GPFC), was opened on February 22, 2016. The overall goal of the Centre is to improve access to child-friendly medicines. We are the only Centre in Canada whose mandate is to facilitate the development of commercially available age-appropriate formulations for children. Promoting practices to increase safety of medicines administered to children is part of our mission.
Many medicines used in children are not available in pharmaceutical forms adapted to their needs. Indeed, most oral medications are produced for adults as tablets and capsules, which lack dosing flexibility to meet dosage requirements of the spectrum of ages and sizes, ranging from neonates to adolescents. Furthermore, most children under the age of 8 are unable to swallow pills.
Approved adults forms often need to be modified in some manner to administer the desired dose to children, and as such are used off-label. When this happens, the adult form is manipulated by either a health care provider, such as pharmacist, or at home by the parents or caregivers (e.g., grinding a tablet and sprinkling it into fluid or a food vehicle such as apple sauce). This process is called compounding and is commonplace for those medicines that lack pediatric formulations. Click here to watch a short video.
Given that compounded medicines are not approved by Health Canada’s rigorous process, we do not always know the medication’s characteristics. Therefore, it is sometimes difficult to know the exact quantity of the medicine that is absorbed. Furthermore, food vehicle (e.g., apple sauce, juice or yogurt) used to administer these compounded medicines, or to mask the bad taste of some formulations, can alter drug absorption.
We strive to contribute to making available convenient and high-quality pediatric products adapted to children’s needs that can spell the difference between successful treatment and therapeutic failure and prevent adverse drug events.
The Goodman Pediatric Formulations Centre is a non-for-profit organization dedicated to working with all stakeholders that could be part of the solution to improving access to child-friendly medicines:
The Centre’s vision is to improve access to child-friendly medicines by increasing the number of medicines formulated for children. As a result of this initiative, we will facilitate increasing the efficacy, safety, and compliance of medicines for children. As a non-for-profit, we endeavour to shape the environment to encourage drug development and commercialization of pediatric formulations, in Canada, and elsewhere, by being a trusted partner for all parties. We are the voice of Canadian children to support efforts so that age-appropriate medicines are available.
- To facilitate the development and commercialization of safe and effective pediatric drug formulations by:
- Promoting a research-based approach to pediatric formulation development
- Advocating for a clinical and regulatory environment that fosters pediatric formulation development
- Advocating for incentives that will stimulate and attract development of pediatric formulations
- Identifying hurdles and challenges in the drug development process and in the regulatory landscape and act as a change agent
- To promote practices to increase safety of medicines administered to children
These goals are in line with a recent report of the Council of Canadian Academies, Improving Medicines for Children in Canada, and a second report from the Canadian Senate, Prescription Pharmaceuticals in Canada.
The Centre provides to the pharmaceutical industry, or other Parties, the following services in relation with the development and regulatory approval of pediatric formulations:
- Clinical, pharmacological and regulatory expertise
- Identification and prioritization of children’s needs
- Development of Pediatric Formulation Development Plan (PERFORM)
- Clinical research services and expertise
- Research capacity with clinical investigation unit and trained personnel in pediatrics
- Expertise in the design and conduct of pediatric pharmacokinetic, efficacy and safety studies
- Supporting advocacy efforts at Provincial, Federal, and International Levels
Additional services may be provided, as needed.
About CHU Sainte-Justine
Affiliated with the Université de Montréal, CHU Sainte-Justine is the largest pediatric teaching centre in Quebec and amongst the top in Canada.
ABOUT THE TEAM
Catherine Litalien, M.D., F.R.C.P.C.
Catherine Litalien is the Executive Director and one of the founders of the Goodman Pediatric Formulations Centre. Using her experience as a pediatric critical care specialist for 16 years, and now, as an academic pediatrician, she understands the need for pediatric formulations and the challenges that compounding brings. With a vision to bringing more child-friendly medicines for children, Dr. Litalien brought together a team and found the initial financing for the Goodman Pediatric Formulations Centre, which opened in February 2016. Dr. Litalien is also an Associate Professor in the Department of Pediatrics and an Accredited Member in the Department of Pharmacology at the Université de Montréal, and the Medical Director of the Clinical Pharmacology Unit at the CHU Sainte-Justine. More recently, she had the opportunity to act as an expert for the Council of Canadian Academies on the Expert Panel on Therapeutic Products for Infants, Children, and Youth that published a report in 2014 entitled Improving Medicines for Children in Canada.
Andrea Gilpin, Ph.D., M.B.A.
Andrea Gilpin is the General Manager of the Goodman Pediatric Formulations Centre. As the main point of contact and spokesperson, she leads the Centre’s team, provides the strategic direction, and oversees the operations of the Centre. Dr. Gilpin began her career as a scientist and completed her Doctorate in Molecular Biology and Biochemistry from the University of Toronto. Since that time, her career has brought her through several different roles including working 12 years in small biotechnology companies in investor relations and communications. In 2011, she took a position working at Pfizer and later spent 3 years at Novartis, where she was Head of Corporate Communications for Novartis Canada and in 2015 she moved to a marketing oncology position in the Lung and Melanoma franchise at Novartis.
Julie Autmizguine, M.D., F.R.C.P.C., M.Sc.
Julie Autmizguine is the Scientific Director of the Goodman Pediatric Formulations Centre, is responsible of its research activities. Since 2014, she is a pediatrician specialized in infectious diseases at the CHU Sainte-Justine. After her clinical training in pediatrics and infectious diseases at the University of Montreal in 2012, she completed an additional two-year training in clinical pharmacology at the Duke Clinical Research Institute within the American Pediatric Trials Network. During her research training, she also obtained a master degree in the Clinical Research Training Program at Duke University, and didactic training at the Eshelman School of pharmacy, The University of Chapel Hill. Her research activity focuses on pediatric clinical pharmacology. She is interested in reducing off-label prescription and increasing safety and effectiveness of drug therapies for children of all ages. More specifically, she studies the pharmacokinetics and pharmacodynamics of antibiotics used to treat infections caused by bacteria resistant to first-line antibiotics in children.
Jean-Marie Leclerc, M.D., F.R.C.P.C.
Jean-Marie Leclerc sits on the Internal Advisory Committee for the Goodman Pediatric Formulations Centre and is one of the founders of the Centre. Dr. Leclerc is a trained hematologist-oncologist with an interest in clinical research and drug metabolism in oncology. After his medical training, Dr. Leclerc completed two post-doctoral fellowships, first at CHU Sainte-Justine and the second at the University of North Carolina in Chapel Hill. Upon his return to CHU Sainte-Justine, Dr. Leclerc opened his research laboratory and initiated and lead the local oncology clinical research group to participate to the Pediatric Oncology Group studies (POG, now Children Oncology Group) for patients with solid tumors as well as in the Dana Farber Cancer Institute Group (Boston protocol) for patients with acute lymphoblastic leukemia. From 1996-2012, he worked in different leadership roles in the pharmaceutical industry and was in charge of the clinical research, regulatory and medical affairs groups at Pfizer (1996-97), Schering-Plough (1997-2002) and Novartis Canada (2002-12) while keeping part-time clinical activities in hematology-oncology. Upon his return full time at CHU Sainte-Justine in 2012, Dr. Leclerc became the principal investigator of the Boston studies and protocols in the rare tumors program. He also developed and launched a Phase II study in neurofibromatosis. In addition, he developed a new partnership with the DFCI leading to CHU Sainte-Justine becoming a core lab for the group new leukemia study (asparaginase monitoring, under Dr. Y. Theoret, and new generation sequencing for Ph-Like evaluation, under Dr. D. Sinnett).
Yves Théorêt, Ph.D, Pharm.D.
Yves Théorêt sits on the Internal Advisory Committee of the Goodman Pediatric Formulations Centre. Dr. Théorêt completed his PhD in neuropharmacology at McGill University, followed by a post-doctoral fellowship in neurotoxicology at the Université de Montréal and the University of North Carolina. He joined the CHU Sainte-Justine Research Centre in 1990 as a Primary Investigator, focusing on pharmacotheraphy for brain tumours. Dr. Théorêt is one of the founders of the Clinical Pharmacology Unit of CHU Sainte-Justine whose mandate is to perform “real-time” pharmacotherapy to personalize drug treatment for children and mothers. Dr. Théorêt is the head of the laboratory of this Unit and uses innovative approaches to develop new tests to improve therapeutic drug management. He has several patients to his name.
Denis Lebel, B.pharm. M.Sc., F.C.S.H.P.
Denis Lebel sits on the Internal Advisory Committee of the Goodman Pediatric Formulations Centre. Mr. Lebel has been working as a pharmacist at CHU Sainte-Justine since 1992. In this role he is responsible in patient care, teaching, and research. Mr. Lebel has an interest in using technology for process improvement to reduce risks associated with preparation and administration of medicines in children. He is part of the Unité de Recherche en Pratiques Pharmaceutiques. Mr. Lebel understands first-hand the challenges that patients and their families have when they leave the hospital with a prescription that requires a compounded medicine.
Identify and Prioritize Children’s Needs Using a Patient-Driven Approach
Many medicines that are currently used for children are compounded either by a health care provider or at home by their parents. Although today’s way of compounding provides access to many medications for the pediatric population, this is not without any challenges. As such, we aim to increase the availability of pediatric formulations with highest pharmaceutical grade standards, avoiding manipulation of adult formulations.
By increasing the number of medicines formulated for children, we aim to improve safety, efficacy and compliance of medicines used in children.
One of our priorities is to determine the medicines most in-need and find ways to make these medicines available commercially. As a first step, we are developing a national list of the most commonly compounded medicines in collaboration with pharmacists associated with Canadian pediatric academic health centres. This list is dynamic and will be updated periodically.
Partner with Pharmaceutical Companies to Develop Pediatric Formulations for Children
We are approaching pharmaceutical companies to seek partnership to facilitate the regulatory approval and commercialization of pediatric formulations for the currently compounded medicines most in need in Canada.
For the pharmaceutical partner, who will be the sponsor of the regulatory application, this will mean:
- Bringing to the Canadian market already existing pediatric formulations that are approved and available in other countries
- Developing pediatric formulations when they do not exist
Our business model is that we will partner with pharmaceutical companies to build a dossier to bring to regulatory agencies (e.g., Health Canada). The Centre will build a development plan, called a “PERFORM Plan”, for each drug of interest. The PERFORM covers the following aspects:
- Outline of clinical uses of the drug in pediatrics and formulation needs according to age groups
- Summary of published pediatric pharmacokinetic, efficacy, and safety data
- Systematic review and meta-analysis in the case of a submission relying on Third Party Data to Health Canada
- Description of existing pediatric formulation(s), if any, and indications for which they have been approved in other jurisdictions
- Identification of knowledge gaps, if present
If needed, the PERFORM plan may include the design of pediatric studies (including pharmacokinetic, bioequivalence, efficacy, and safety studies) needed in order to fulfill regulatory requirements for approval. The Goodman Centre can also take a leadership role in the undertaking of pediatric studies.
Our experienced team is available to attend meetings with regulators to support any regulatory applications.
STAKEHOLDERS AND PARTNERS
A Team Effort
Stakeholders and partners are a key to success to improve access to child-friendly medicines. One of the Centre’s mission is to drive collaboration between stakeholders that extends across disciplines and geographical boundaries. It is vital to bring all partners to work together, regardless of the regulatory landscape, to solve the challenges that we are collectively facing to make available pediatric formulations. It is a shared responsibility to ensure that children’s needs are addressed.
Interested parties who would like to join this initiative are invited to reach out to Andrea Gilpin, General Manager.
The Pharmaceutical Partner
One of the key partnerships for the Centre is to work with Pharmaceutical companies to gain regulatory approval and commercialize child-friendly formulations of medicines. The pharmaceutical partner has the objective to provide access to pediatric formulations that are effective, safe and easy to administer, have flexible dosage increments and minimal excipients, be palatable, and stable. In general, we are looking to partner with companies who are interested in:
- Bringing to the Canadian market an already existing pediatric formulation that is approved and available in other countries
- Developing a pediatric formulation in an area of core competency or bringing
- Committing to manufacture the pediatric formulation for the Canadian market and a willingness to share outside of Canada
- Using our PERFORM service to prepare a new drug submission
- Using our research expertise to assist in the conduct of pediatric studies as part of the regulatory approval process
BUILDING ADVOCACY FOR THE CAUSE
Support Advocacy Efforts for a Favourable Environment for Pediatric Drug Development in Canada
Working with all stakeholders, the Centre would like to use its expertise to increase awareness of the issues concerning pediatric formulations and contribute to creating an environment that would favour the development of medicines specifically formulated for children. One of the key aspects in this regard is to understand what strategies might encourage pharmaceutical companies as well as other stakeholders to invest in developing pediatric formulations.
Recognizing the importance of developing safe and effective medicines specifically for children the Minister of Health, on behalf of Health Canada, asked the Council of Canadian Academies to provide an evidence-based and authoritative assessment of the state of research and regulations leading to the approval of medicines for children, in Canada and abroad. In this report, the challenges with the existing system are outlined and one of the conclusions is that we need to strive to develop medicines that are available in a formulation that suits the needs of children. Hence the areas for improvement have been well outlined, and the Centre will build on this assessment to develop some concrete plans to improve access to child-friendly medicines for children.
Building networks is pivotal for the Centre to succeed and it can take many forms. The Centre needs to build relationships with regulatory authorities, pediatricians, hospital pharmacists, politicians, other pediatric hospitals in Canada (and abroad), pharmaceutical industry and patients and their families. These networks allow us to establish priority medicines and identify medicines that are most in need for a pediatric formulation. Moreover, we can use these networks to gain feedback on our strategy and initiatives. The information gained can be shared with our stakeholders who can help us to improve the environment to encourage pharmaceutical companies to invest in pediatric formulations.
There is an important role for the Centre to educate stakeholders whenever possible, as many do not fully understand the current situation. Therefore, the Centre will aim to participate at major pediatric scientific conferences, lead workshops, organize symposia all with the goal to contribute to increasing awareness and understanding of the challenges that exist today with pediatric compounding, and the urgent need for commercially available age-appropriate pediatric formulations.
NEWS & EVENTS
Your Support is Crucial
The Goodman Pediatric Formulations Centre is a non-for-profit organization dedicated to improving access to child-friendly medicines to Canadian children and abroad. We are forever grateful to our founding partners the Morris & Rosalind Goodman Family Foundation and the CHU Sainte-Justine. To allow the Centre to meet this objective, your support is essential. By choosing to donate to the Centre, you are enabling a team of dedicated healthcare professionals the ability to provide age-appropriate medicines for children.
For more information about how to donate, please contact Andrea Gilpin, General Manager.
Want to Get Involved?
Whether you are an individual or a company – if you are interested in helping our cause, please contact us. We are a non-for-profit and always looking ways to bring stakeholders and interested parties together for this important cause. Please contact Andrea Gilpin, General Manager.
DefinitionsAdverse drug events or side effects are unintended side-effects from a medicine. Compounding is the process where two or more ingredients are mixed (one has to be a medicine) to create a final product in an appropriate form for dosing. It can involve the alteration of the form and strength of the commercially available product and can include reformulation for allow for a new drug delivery. In this website we do not make the distinction between compounding and extemporaneous formulations. Excipients are substances that are formulated alongside the active ingredient of a medication. They can be included for example, to stabilize the medicine or to aid in the manufacturing process. Forms and Formulations: Formulations is the term denoting the combination of an active pharmaceutical ingredient (API) with other non-active constituents – termed excipients - to create a medication. Form refers to the overall physical configuration of a medication (e.g., tablet, powder, capsule, or liquid). For the purposes of simplicity, both are used interchangeably in the website and refer to both definitions.
What is Off-Label Use?
Drug approval is specific to the indication(s), age groups, route of administration, formulations, method of manufacturing, and package labelling.
Off-label refers to a use that differs from that approved by regulatory authorities and can refer to any departure from than authorization. As such, using a medication in a different formulation than approved is considered off-label use. The lack of pediatric formulations often leaves health care providers caring for children no alternative, but to use adult medicines in a manner that has not been approved by Health Canada (manipulation of adult pharmaceutical forms).
Off-label use is a fairly common practice amongst health care provider and is not specific to Canada but widespread around the globe. It is estimated that the prevalence of off-label use in the pediatric population is approximately 45 to 60% of all medicines given to children, and up to 90% in vulnerable populations such as neonates, critically ill children, and children with cancer.
Off-label use does not necessary imply off-evidence. It is the responsibility of the prescribing physician to evaluate whether or not the available clinical and scientific evidence supports the use of the medication in a specific patient, that beneficial patient outcomes outweigh potential risks. Patient safety should always remain the top priority of health care providers.
Off-label use is different from use of an unauthorized medicine. It is legal for physicians to prescribe drugs as off-label medications.
What is Compounding?
Compounding is the process where two or more ingredients are mixed (one has to be a medicine) to create a final product in an appropriate form for dosing. It can involve the alteration of the form and strength of the commercially available product and can include reformulation to allow for a new route of administration.
This manipulation of dosage forms designed for adults by health care providers and parents may cause some concerns. Our goal is to increase the number of approved pediatric formulations thereby decreasing the need to compound medicines. Some of the concerns that may arise with compounding include:
- Variation existing in the preparation of compounded medicines. As such, compounding practices may vary among hospitals and compounding pharmacies in the community, and across Canada, which can lead to significant variations in drug exposure when the child changes from one place to another, or even when a different pharmacist compounds the medication.
- Medicines that are compounded often have a limited stability and therefore need to be prepared frequently which can translate into multiple visits for parents to the pharmacy.
- Since compounded medicines are not approved by Health Canada’s rigorous process, we do not always know the medication’s characteristics. Therefore, it is sometimes difficult to know the exact quantity of the medicine that is absorbed.
- Sometimes safety issues for the family may arise, in cases of toxic drugs that parents need to manipulate at home. In such examples, parents need to protect themselves, and their family members from involuntary exposure to the toxic medications, such as anti-cancer medications.
- Pharmaceutical manufacturing practices are regulated and great care is taken to make the requested formulation; however, since these compounded materials are not made in a dedicated manufacturing facility, the standards cannot be at the same level as commercially manufactured medicines.
Other Organizations Sharing Common Goals with the GPFC
Pediatric Formulations – Global research in Pediatrics (GRIP)