Could a drug that improves patients’ quality of life also be beneficial to the healthcare system? In the case of efmoroctocog alfa (Elocta *), the first recombinant factor VIII with a prolonged half-life for haemophilia A, the answer is yes according to a recent study published in the ‘Italian Journal of Public Health’ and conducted by Altems, the High School of Economics and Management of Health Systems of the Catholic University of the Sacred Heart, realized with the unconditional contribution of Sobi. The researchers simulated what would happen if the use of efmoroctocog alfa progressively replaced the administration of the therapeutic alternatives available today. calculating a saving of over 18 million euros in 3 years.
Hemophilia A is an inherited disease caused by the absence or low levels of factor VIII which plays a key role in blood clotting. Those who suffer from it undergo hemorrhages that can manifest themselves in a very varied way: from small bruises and hematomas to very painful and disabling joint bleeding (haemarthroses), up to major bleeding that can be life-threatening. Prophylaxis therapy with factor VIII substitutes is the ‘gold standard’ for haemophilia patients and allows, if taken constantly, to prevent bleeding episodes, haemophilic arthropathy and preserve normal musculoskeletal function. For patients, this means having infusions every 1-2 days in the case of standard half-life products, or every 3-5 days in the case of efmoroctocog alfa, which has a prolonged half-life.
“Efmoroctocog alfa – says Raimondo De Cristofaro, hematologist at the ‘Agostino Gemelli’ University Polyclinic, Catholic University of the Sacred Heart – has proven to be very effective in the prevention of arthropathies and haemarthroses. The fact that it remains in circulation for a prolonged time allows infusions to be scheduled less frequently, with an undoubted advantage for patients and their caregivers. Furthermore, thanks to its formulation, the possible development of antibodies (IgG) against the exogenous coagulation factor, the most serious complication associated with replacement therapy, is reduced “.
A solution of great advantage for patients and their families, but also for the health system, underlines a note: in fact, haemophilia centers see a reduction in the impact both on the organizational front and on that of costs.
“This is one of the rare cases – he explains Americo Cicchetti, director of Altems – where the introduction of an innovative solution does not have an impact on expenditure, but rather reduces it. Our study shows that the reduction can reach over 18 million euros in 3 years, to which must be added the indirect costs: patients with a better quality of life are more likely to continue working and lead a satisfying life “.
As he also points out Cristina Cassone, FedEmo president: “The availability of innovative long-lasting products, effective and at the same time reducing the number of infusions, represents an undoubted advantage for patients, making it easier for them to adhere to therapy in the prophylaxis regimen and significantly contributing to improve their quality of life “.
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