Jennifer DiRaimo, MS, CCGC
Director of Research & Canadian Advocacy,
Platelet Disorder Support Association (PDSA)
Chronic disease can be managed with treatments, but they remain out of reach for many.
In a world where emergency rooms are bursting, families are often without access to primary care, and healthcare services are at a premium, one would think that governments and insurers would do everything they could to avoid hospitalizations and unnecessary doctor visits.
For many patients in Canada’s immune thrombocytopenia (ITP) community, that’s not the case.
A rare but serious blood disorder
ITP is a rare, autoimmune platelet disorder that causes a low platelet count in the blood. Since platelets are important in the body to help stop bleeding, a low platelet count results in an increased propensity to bleed internally, even spontaneously. ITP is a potentially life-threatening disorder. Symptoms can vary – from minor symptoms like easy bruising to more critical symptoms like internal bleeding, including in the brain. For some, ITP is not chronic or life threatening; for others, it can be a chronic disorder that individuals live with for their entire lives.
Jennifer DiRaimo, MS, CCGC is the Director of Research and Canadian Advocacy at the Platelet Disorder Support Association (PDSA). She understands first-hand the nature and potentially devastating consequences of ITP – she lost her 10-year-old son to complications from ITP in 2018.
Challenges in the ITP care pathway
“ITP can take shape in many forms. For some, it can be easily treated and relatively minor; for others, it can be serious and potentially devastating. As a disorder that can be wide-ranging, it is important to have care that can address that wide range of cases as well.”
The care pathway for Canadian ITP patients is long, consulted, and restrictive – especially for chronic patients. Patients are typically treated first with corticosteroids and plasma-derived therapies like intravenous immunoglobulin (IVIG), which have issues with both long-term efficacy and, in the case of IVIG, sustainability of supply issues. It’s also very expensive, requiring frequent use due to its short-acting nature.
Following that treatment, most provinces consider splenectomy – a significant procedure that involves the removal of the spleen. This procedure, while effective for some, has serious, sometimes life-limiting, drawbacks: it is irreversible, not guaranteed to resolve ITP, and confers a lifelong risk of serious infection often requiring treatment.
“We’ve been advocating for some time to streamline the care pathways for ITP. The one element that stands out in discussions with patients and clinicians is the potential inclusion of splenectomy as a second-line treatment”, DiRaimo noted. “In a time where we hear about healthcare shortages and surgical backlogs, why wouldn’t we look to other alternatives if they are available to us that are more or just as effective, and less burdensome on both the patient and the healthcare industry?”
The need for innovative therapies
Those alternatives do exist. Within the last decade, several new, innovative therapies have come to light for the treatment of ITP, including a new class of treatments called thrombopoietin receptor agonists (TPO-RAs.) These therapies are proven to effectively treat ITP in chronic patients and provide treatment with significantly less risk and side effects than surgery or conventional therapies.
TPO-RAs are used across the world. Their use in ITP treatment is supported by the Canadian Hematology Society and is recommended in both Canadian and international medical guidelines for the treatment of ITP – but remain out of reach for many Canadian patients.
Barriers ITP patients face
In recent years, drug reviews from Canada’s Drug Agency for TPO-RAs have largely prevented patients from being able to easily and equitably access these treatments under provincial drug programs. While these reviews highlight the potential role for TPO-RAs, they also flag some uncertainty due to a lack of robust evidence – which is difficult to collect in a rare disease – which has been enough for provinces to shut the door on providing access for most eligible patients.
“It’s frustrating for many in the ITP community in Canada. Patients dependent on public drug plans are being prevented the opportunity to utilize medicines that have been around for a while, are available in other places like the US and the UK, leaving them with limited treatment routes”, said DiRaimo. “Our fear is that the gap in care is growing, and our community will continue to face unnecessary hardships in accessing the care they need.”
Advocating for a better future
DiRaimo isn’t giving up. Under her watch, PDSA continues to advocate to provincial governments across Canada about better access to care for ITP patients. “We have such an active and supportive community of ITP patients, families, and clinicians across the country, and they’re ready to have their voice heard.”
For more information about PDSA and their activities in Canada, visit pdsa.org/canada.
This article was made possible by the support of Sobi Canada Inc.
