The company offers this “pay over-time” model, which it says is part of the U.S. health care system and has already entered into results-based agreements with up to 15 payers. MHE: Explain the AveXis results-based approach. “Zolgensma is radically changing the lives of families affected by this devastating disease, and given the new efficacy data for the pre-ptomatic population, the price announced today is within the value-based PRICE reference range of ICER. All the noise set aside, Zolgensma is in its core an effective drug. It showed a significant improvement in motor functions, such as the ability to sit unseemly, as well as event-free survival in patients with presymptomatic and infant ADM, even beyond five years after treatment. Compared to its rival, Biogenspinraza – which requires chronic dosing – Zolgensma has the potential to cure the disease with a single injection of an adeno-associated treatment of the virus that contains a functional copy of the human sméno gene. Novartis hopes that Zolgensma`s unique gene therapy (onasemnogene abeparvovec-xioi) will not only provide permanent healing for infants with spinal muscular atrophy (ADM), but also provide a new product to the blockbuster through a brand new payment model based on results. Italy has long been at the forefront of managed access, thanks in large part to its network of online registries.
It is interesting to note, however, that the use of “results payment” has declined sharply in recent times, as has the savings that the health sector derives from these agreements. Instead, Italy has increasingly focused on appropriate prescription agreements and spending limits. With the introduction of the “Day One” patient access program for Zolgensma, a new high-level gene therapy for spinal muscular atrophy distributed by AveXis (a subsidiary of Novartis), the manager`s entry has taken a fascinating new turn in Europe. The company is reported to have met with more than 100 interest groups across Europe to ensure that the programme – which offers retroactive discounts, staggered payments, results-based discounts and a global product register – is compatible with pay payers` pricing and reimbursement frameworks and can be tailored to different markets. This strategy is particularly interesting when viewed in the context of high-level commissioning in the context of the current COVID 19 pandemic, which is causing significant delays in HTA negotiations and prices and refunds. #managedentry #MEA #Zolgensma #Novartis #AveXis #SMA #genetherapy access program “Day One” was designed to harmonize patient treatment costs, as national price and reimbursement agreements were negotiated on a price basis. The program contains a number of customizable options: these agreements are not routine at Medicaid. Massachusetts is one of the few states authorized by the Centers for Medicare and Medicaid Services to track these cases.