With more than $ 2 million, Zolgensma is the most expensive therapy in the world, but relatively profitable

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Last month, the Food and Drug Administration (FDA) approved gene therapy for a rare childhood disorder; spinal muscular atrophy (SMA). The name of the therapy is Zolgensma. It is indicated for the treatment of patients less than two years of age with spinal muscular atrophy and bi-allelic mutations of the survival motor neuron gene 1. At approximately $ 2.1 million per patient, this is the most expensive treatment on the market. Zolgensma can be a& nbsp;Unique treatment that saves lives and allows children with AS to function in ways unimaginable just a few years ago.

The therapy developer, Novartis, set the price at about $ 2.1 million, but offered insurers the opportunity to pay annual payments of $ 425,000 over five years.& nbsp;

As with any new treatment, there are invariably uncertainties about its effectiveness, safety and sustainability in the real world. This is especially true for orphan treatments, such as Zolgensma, which, during the various stages of drug development, often include only a relatively small number of patients. Therefore, it is necessary to generate evidence after marketing.

In the absence of post-marketing data at the present time, badysts at the Institute for Economic and Economic Review (ICER) have evaluated the Phase III evidence to calculate the profitability of Zolgensma. Zolgensma's value-based price has been estimated at between $ 1.1 and $ 1.9 million per treatment, for a break-even point of $ 100,000 to $ 150,000 per quality-adjusted life year. And one price based on value for Zolgensma has been estimated at between $ 1.2 million and $ 2.1 million, taking into account the alternative threshold of $ 100,000 to $ 150,000 per year of life earned.

Obviously, the price chosen by Novartis is the upper limit in ICER's calculations of what constitutes a profitable price.

Too often, the discussion of a price of the medicine focuses on numbers that are obvious, not value. It is commendable that ICER has redirected us to the value discussion. In some cases, high prices may be justified by value; in others, no. At the same time, cheap products can sometimes produce great value, and in other cases, no value.

The key thing to remember is that the value of a new technology is related to its opportunity cost, or what could have been purchased with the resources used for a given technology. In this case, spending money on Zolgensma implies the need to evaluate the value of the best possible alternative; in that case, Spinraza. & nbsp; Spinraza is not a single treatment, it must be administered regularly. It does not restore the same functioning as Zolgensma. Over a 10-year period, Spinraza's costs would total more than $ 4 million.

It is important to note that the evaluation of the differences between Zolgensma and Spinraza is not necessarily a direct comparison between apples. Indeed, Spinraza was approved by the FDA in December 2016 to treat all types of spinal muscular atrophy, regardless of the age of the patient. Therefore, for many people with ADS, Zolgensma will not be an option and Spinraza will be the best alternative available.

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Last month, the Food and Drug Administration (FDA) approved gene therapy for a rare childhood disorder; spinal muscular atrophy (SMA). The name of the therapy is Zolgensma. It is indicated for the treatment of patients less than two years of age with spinal muscular atrophy and bi-allelic mutations of the survival motor neuron gene 1. At approximately $ 2.1 million per patient, it is the most expensive treatment on the market. Zolgensma can be a Unique treatment that saves lives and allows children with AS to function in ways unimaginable just a few years ago.

The therapy developer, Novartis, set the price at about $ 2.1 million, but offered insurers the opportunity to pay annual payments of $ 425,000 over five years.

As with any new treatment, there are invariably uncertainties about its effectiveness, safety and sustainability in the real world. This is especially true for orphan treatments, such as Zolgensma, which, during the various stages of drug development, often include only a relatively small number of patients. Therefore, it is necessary to generate evidence after marketing.

In the absence of post-marketing data at the present time, badysts at the Institute for Economic and Economic Review (ICER) have evaluated the Phase III evidence to calculate the profitability of Zolgensma. Zolgensma's value-based price has been estimated at between $ 1.1 and $ 1.9 million per treatment, for a break-even point of $ 100,000 to $ 150,000 per quality-adjusted life year. In addition, Zolgensma's value-based price was estimated to be between $ 1.2 million and $ 2.1 million, taking into account the alternative threshold of $ 100,000 to $ 150,000 per year of life earned.

Obviously, the price chosen by Novartis is the upper limit in ICER's calculations of what constitutes a profitable price.

Too often, the discussion about the price of a drug is focused on striking numbers and not on value. It is commendable that ICER has redirected us to the value discussion. In some cases, high prices may be justified by value; in others, no. At the same time, cheap products can sometimes produce great value, and in other cases, no value.

The key thing to remember is that the value of a new technology is related to its opportunity cost, or what could have been purchased with the resources used for a given technology. In this case, spending money on Zolgensma implies the need to evaluate the value of the best possible alternative; in this case, Spinraza. Spinraza is not a single treatment, it must be administered regularly. It does not restore the same functioning as Zolgensma. Over a 10-year period, Spinraza's costs would total more than $ 4 million.

It is important to note that the evaluation of the differences between Zolgensma and Spinraza is not necessarily a direct comparison between apples. Indeed, Spinraza was approved by the FDA in December 2016 to treat all types of spinal muscular atrophy, regardless of the age of the patient. Therefore, for many people with ADS, Zolgensma will not be an option and Spinraza will be the best alternative available.