Switching patients with Gaucher disease type 1 (GD1) from enzyme replacement therapy (ERT) to Cerdelga (eliglustat), an oral substrate reduction therapy (SRT), may strongly reduce treatment-associated costs, a recent study suggests.
The study, “Budget Impact Analysis of Eliglustat for the Treatment of Gaucher Disease Type 1 in the United States,” was published in the Journal of Managed Care & Specialty Pharmacy.
Current treatment options for GD1 — which accounts for 94% of all Gaucher disease patients — include three intravenous ERTs — Cerezyme (imiglucerase), VPRIV (velaglucerase alfa), and Elelyso (taliglucerase alfa) — and two oral SRTs — Cerdelga and Zavesca (miglustat).
ERT consists of the delivery of copies of the glucocerebrosidase — the enzyme deficient in Gaucher patients and responsible for the breakdown of specific fat molecules — preventing the accumulation of these fat molecules, while SRT partially blocks the production of the fat molecules themselves, reducing their accumulation.
ERT Cerezyme (developed by Sanofi Genzyme) is the standard therapy for GD1, but ERT infusions are expensive, with highly variable costs depending on the place where patients receive treatment and on the patient’s weight.
The SRT Cerdelga (developed by Genzyme), was the first oral approved therapy for the treatment of adult GD1 patients, and has been shown to be well-tolerated and to have comparable effectiveness to Cerezyme.
Since Cerdelga has no infusion-associated costs and a less variable price than ERTs, it has the potential to reduce treatment-associated costs for third-party payers.
Evidera, a company that provides consulting and other research services to biopharmaceutical companies, evaluated the budget impact of shifting GD1 patients already receiving ERTs to Cerdelga in the United States. The study was sponsored by Genzyme.
Annual ERT costs were calculated assuming a biweekly infusion (24 infusions per year) of the average dose and adjusted for the proportion of patients receiving infusions in each site of care (hospital outpatient, infusion center, or home). Annual costs of Cerdelga were calculated assuming the standard dose administered twice a day.
The company evaluated the potential economic costs of switching treatment in an estimation of five million members in a health plan, with an estimated 25 GD1 patients with an average weight of 72 kg (or about 158 pounds) eligible for treatment with Cerdelga.
In this model, increasing the use of orally administered Cerdelga resulted in an annual savings of $1,526,710 and a three-year savings of $4,580,130.
Considering that administering ERTs in the hospital accounts for the largest percentage of the total costs, and it is in the hospital that most patients receive infusions, the company also analyzed what would happen to the costs if 50% of patients receiving infusions in the hospital were shifted to Cerdelga.
The results showed that, in this scenario, the cost saving would be even higher, with a three-year cumulative savings of $5,162,841.
These findings suggest that “third-party payers can potentially save money if patients switch from ERT infusion to orally administered eliglustat [Cerdelga],” the study’s authors noted.
They added that starting treatment of GD1 patients with Cerdelga instead of ERTs may also lead to savings in treatment-associated costs.
