Study cautions against switching Gaucher treatments to cut costs
Changing ERTs can bring on new side effects, researchers say
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Switching patients who are doing well on their current Gaucher disease treatment to a different medication comes with risks, and it may be “inappropriate” for health insurers to require a change to cut costs, according to a study from Quebec.
Gaucher disease patients who switched their enzyme replacement therapy (ERT) from Cerezyme (imiglucerase)Â to Elelyso (taliglucerase alfa) after the province’s single-payer health system revised its policy to prioritize Elelyso found their disease remained controlled, but “more than a quarter experienced significant side effects,” the researchers wrote.
“Our results highlight that [Elelyso] is an effective ERT therapy for [Gaucher disease], however certain patients may experience side-effects,” researchers wrote. “Thus, among patients who are stable and tolerating ERT well, our experience suggests that switching them (especially for financial reasons only) may be inappropriate.”
The study, “Real-world experience of switching to taliglucerase among patients with Gaucher disease in Québec: A case series,” was published in Molecular Genetics and Metabolism Reports.Â
Gaucher disease is caused by mutations in the gene that encodes glucocerebrosidase (GCase), an enzyme necessary for breaking down certain fatty molecules. In people with Gaucher disease, GCase deficiency causes these fatty molecules to accumulate to toxic levels in cells, ultimately leading to Gaucher symptoms such as blood problems and enlarged organs.
Scientists aim to shed light on ramifications of switching
Cerezyme and Elelyso are both approved ERTs for Gaucher disease. The medications, administered by infusion into the bloodstream under the supervision of a healthcare professional, work to deliver a working version of the GCase enzyme into the body.
Cerezyme is one of the oldest ERTs for Gaucher, having been in use since the 1990s. Elelyso is a newer therapy that was approved in the mid-2010s.
Quebec’s healthcare system revised its policy for coverage of Gaucher ERTs in 2016. The new policy, which was based on cost and efficacy comparisons, prioritized coverage of Elelyso over other ERT products.
Quebec’s provincial Gaucher Disease program covers 62 people with the genetic disorder, 42 of whom are receiving treatment. Following the 2016 policy update, 19 of these patients, who had been on Cerezyme for an average of 13.6 years, were switched to Elelyso. The study detailed outcomes from these 19 patients.
“We wish to raise awareness of the range of symptoms that patients may experience while undergoing this type of switch in ERT,” the researchers wrote.
Results indicated that switching from Cerezyme to Elelyso didn’t lead to substantial changes in how well Gaucher disease was controlled. Average scores on disease-related measures, including blood cell counts and organ sizes, were not significantly different after patients switched treatments.
However, six of the 19 patients experienced side effects on Elelyso that they had not experienced while on Cerezyme.
The reported side effects were mostly reactions that occurred during or after the infusion, even after premedication with antihistamines. Symptoms included rash and hives, digestive upset, pain, nasal congestion, and fatigue.
In five of the six cases, these side effects persisted with continued Elelyso, sometimes for years, until patients ultimately switched back to Cerezyme. In the sixth patient, side effects persisted for a few months but then resolved; that patient is still on Elelyso.
“This experience highlights the risks of switching therapy for cost-based considerations – here, mandated by our single-payer system – for patients already responding well to one type of ERT,” the scientists wrote. “Rather, patients whose [Gaucher disease] is stable and are tolerating ERT well, there is no need to switch formulations, and risk new adverse events. Indications to switch ERT formulations should be based on efficacy and tolerance, not cost.”
Cerezyme is sold by Sanofi, and Elelyso by Pfizer. Neither company was involved with the study.
