Interview with Dr Jean-Pierre Chanoine, MDPhD, FRCPC (Academic), Clinical Professor, Endocrinology and Diabetes Unit British Columbia Children’s Hospital, President Global Pediatric Endocrinology and Diabetes (GPED), Vancouver, Canada

Who are you and what is your relationship to OI?

I am a paediatric endocrinologist with a longstanding interest in Osteogenesis Imperfecta (OI). I provide clinical care for children with this condition in Canada, where they benefit from access to bisphosphonate therapy and high-quality orthopaedic support.

Tell us about your role in the GPED and what they are doing for OI in Kenya and other places!

The Global Pediatric Endocrinology and Diabetes (GPED) is a Canadian non-profit organization and registered charity founded in 2010. Its mission is to improve the clinical care of children with endocrine disorders and diabetes in low- and middle-income countries (LMICs). GPED achieves this through advocacy, training of local healthcare providers, support for families and patients, clinical collaborations, and research initiatives.

I serve as president of GPED, and one of our core areas of focus is improving access to essential medicines, including treatments for OI. In low and middle income countries (LMICs), we work with local partners to enhance the capacity of healthcare systems to diagnose and manage OI effectively.

For instance, in Kenya, at the Machakos clinic, Drs Specky Mbula (paediatric anesthetists) and Dr Levis Nguku (orthopaedic surgeon) achieve wonderful results which you can read more about here. However, in the absence of bisphosphonates and orthopaedic equipment, they are confronted with severe deformities that are rarely seen in high-income countries.

You have done some work to have bisphosphonates included in the World Health Organization List of Essential Medicines (WHO EML). Please tell us about this!

Our goal was to have bisphosphonates—specifically zoledronic acid—included in the WHO EML specifically for the treatment of OI. This would be a significant step toward increasing access to this therapy in countries where it remains unavailable or unaffordable. Although zoledronic acid is already included in the EML for cancer-related bone pain, it has not been listed for use in OI, which limits its availability for this important indication. Unfortunately, our application was not approved by the Expert Committee.
The Committee expressed concerns about the quality of existing evidence, noting that current studies do not conclusively demonstrate a reduction in fracture frequency or bone pain. This contrast with the clinical experience of healthcare professionals who care for children and adolescents with OI and highlight the need for rigorous, well-designed clinical trials that clearly quantify the benefits of bisphosphonates in OI.

As you know, there are new drugs on the horizon – but in many countries, even off-label use of relatively affordable bisphosphonates is unavailable. What seems to be the main obstacles?

Access challenges are multifactorial. In many LMICs, zoledronic acid – although affordable in high-income countries – is still considered expensive. Additional barriers include the absence of national guidelines or treatment protocols, limited awareness among healthcare providers, and a lack of interest from pharmaceutical companies due to low market incentives. As a result, even generic, off-patent medications such as zoledronic acid remain out of reach for many children with OI.

What does it mean that bisphosphonates are off patent? Is this part of the problem?

Once a drug is off patent, any pharmaceutical company can manufacture and sell generic versions, which are typically much more affordable than the brand-name product. Zoledronic acid falls into this category. In theory, this should increase access. However, in practice, the production and distribution of these generics may be limited in LMICs, especially when there’s little commercial interest.

The result is a paradox: an affordable, effective drug exists but remains inaccessible to many of those who need it most. Other barriers include the time-consuming and costly registration process in many countries, which could be streamlined, as well as the markup on medicines resulting from a multi-step distribution system, which could be reduced through centralized distribution.

What can we do to make new and more effective therapies available?

Current bisphosphonates like zoledronic acid provide significant clinical benefits for most children with OI, including reduced pain, fewer fractures, and improved bone strength. Their safety profile is well established. Emerging treatments – such as monoclonal antibodies targeting sclerostin, as well as gene therapy or stem cell-based approaches – hold promise for the future. However, these treatments are likely to be prohibitively expensive for patients in resource-limited settings. Ensuring equitable access will require global advocacy, early engagement with manufacturers, and innovative funding mechanisms.

How can we work to improve the knowledge of OI in low-income countries?

Raising awareness and building clinical capacity are essential. This includes organizing targeted educational sessions for healthcare providers and families, developing locally relevant guidelines, and supporting the creation of parent advocacy groups. Initiatives like GPED’s global webinars help disseminate knowledge widely. Increased awareness leads to better diagnosis, improved school and community support, and greater pressure on health authorities to prioritize treatment access.

What do you do when you don’t work as a doctor and advocate?

I love to travel, especially when I can spend extended periods working in the same community – it allows me to feel more integrated and connected. I also enjoy outdoor activities such as hiking and playing golf, which is a very popular pastime here in Canada.

This article has first been published in OIFE Magazine 2025-03.