July 12, 2017

The Canadian Association of Paediatric Nephrology

Imagine that you are sitting in a doctor’s office and you have just been told that your child has a rare disease. There is a medicine to combat the disease, but the medicine costs over $10,000 per year. Without treatment, you child would slowly become sicker, and his quality of life would be poor. Your doctor is aware of studies that have been done showing that this medication helps adult patients with the same disease. However, the medicine is not approved for use in children in Canada, and thus is not paid for by your provincial drug plan, or personal drug plan if you are lucky enough to have one.

Now imagine that a similar scenario is unfolding in a different province in Canada, except this time, the provincial health system will pay for the medication based on the recommendation of a doctor, if the doctor can show that it effectively treats the disease by showing improvement in lab tests. That child’s home province will guarantee access to the medicine.

These scenarios play out in doctors’ offices across Canada every day. Children diagnosed with rare diseases do not have equal access to medical treatment in Canada. For children with kidney disease, the Canadian Association of Paediatric Nephrology surveyed paediatric physicians and determined that medicines used to treat rare but life threatening causes of kidney failure are not available to all patients because they are not approved or funded the same way in each region. For example, rituximab, a medicine to treat a variety of kidney diseases in children, can be fully paid for in Alberta and Quebec, but not in Manitoba. In fact, treatment in Manitoba would still cost a family living at the poverty line over $1000/year. (Welch, 2015, Government of Manitoba, 2016).

So why does this happen? The approval process for bringing medicines to the marketplace is regulated by Health Canada. This process uses the best medical evidence to ensure that medications in the marketplace are safe when used appropriately. However, most of this evidence has only been studied in adults. If the medication has not been studied in children, Health Canada may allow the use of a medicine in children as “off label” – meaning the physician is using the medicine outside of an approved population or health condition. It may come as a surprise that most medicines used in children are used “off label” due to lack of paediatric data. This leaves each province and territory to analyse the safety, affordability and effectiveness of a medicine, allowing each province or territory to reach a different decision on funding the medicine.

However, the system gets more complicated; it is not just where you live, but who you are that determines whether you receive funding for medicines. A doctor must apply on a patient’s behalf to obtain special funding for medicines in children; applications can be made to a provincial drug funding program, or if the patient is First Nations or Inuit, to the NIHB for First Nations and Inuit. The funding decisions between NIHB and provincial drug programs are also different.

What about asking drug companies for help? Many drug companies have in place a compassionate release program that allows the medicine to be released at a substantially reduced cost, or no cost to the patient with a prescription based on demonstrated financial need. However, this requires that the drug is currently marketed in Canada. In many cases, medications for rare diseases are not marketed in Canada, because the market is perceived to be too small. In this circumstance, compassionate programs cannot provide the drug.

So what do other countries do to address the problem of accessibility to those medications? Europe and the United States of America approach this issue differently. Both regions have legislation that requires studies in children to be included in drug approval applications. While neither system is perfect, they have both increased the number of applications for medications to be approved in children. Many of the medicines used to treat rare diseases that are not available in Canada are approved in the United States, Europe or both.

Health Canada does not mandate that pediatric data be included in drug approval applications, unless a medicine is specifically marketed for a specific pediatric condition (Health Canada: Drugs and Health Products, 2003). Of the more than 16,000 medicines approved in the Health Canada drug database, the number approved for use in children is unknown.

As stewards of the health care system, doctors are intimately aware of the limited resources and expanding healthcare budgets that stretch the system. We are also aware, as kidney health professionals, that limiting access to these medications to treat children with kidney disease that have been proven to reduce kidney disease burden based on where they live and who they are is unacceptable. It is not a cost savings solution; it is the more expensive option since those patients may progress more rapidly to end stage renal disease with tremendous cost to their quality of life and cost to society. It is time for a harmonized program for drug approval in Canada for children to insure equal access to evidence-based therapies. Health Canada is encouraged to follow the lead of regulatory authorities in Europe and the USA to mandate the inclusion of children in approving medicines in the Canadian marketplace. A national strategy for approval, funding and release of medications is necessary to remove barriers to healthcare that have been constructed on the foundations of regional resources and government silos. We are reminded that the inequities we see in pediatric access to therapies define us as a community. In the words of Mahatma Ghandi "A nation's greatness is measured by how it treats its weakest members."

Cited resources

Adriana Ceci, et al., June 3, 2015. Chapter 12: Clinical Trials in Pediatrics - Regulatory and Methodological Aspects. In: Drug Discovery and Development - From Molecules to Medicine. s.l.:InTech, pp. 271-297.

Anon., 2003. Health Canada: Drugs and Health Products. [Online]
Available at: http://www.hc-sc.gc.ca/dhp-mps/prodpharma/applic-demande/guide-ld/clini/e11_addendum-eng.php
[Accessed 14 November 2016].

Anon., 2016. Manitoba Drug Benefits Interchanagebility Formulary. [Online]
Available at: http://www.gov.mb.ca/health/mdbif/
[Accessed 14 November 2016].

Group, I. E. W., 2000. International Conference on Harmonization of Technical Requirements for the Registration of Pharmaceuticals for Human Use, article E11 (Clinical Investigation of Medicinal Products in the Pediatric Population). [Online]
Available at: http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/E11/Step4/E11_Guideline.pdf
[Accessed 14 November 2016].

Manitoba, Governement of, 2016. Manitoba Pharmacare Deductible Calculator. [Online]
Available at: http://web2.gov.mb.ca/health/pharmacare/summary.php
[Accessed 14 November 2016].

Welch, M. A., 2015. Nearly 1 in 3 children below poverty line in Manitoba: report. [Online]
Available at: http://www.winnipegfreepress.com/local/Nearly-1-in-3-children-below-poverty-line-in-Manitoba-353166861.html
[Accessed 14 November 2016].