Novartis has completely decoupled the price of the drug from the cost of its development, ensuring a 50% reduction on average current expenditure for treatment. The Zolgensma case born from the Telethon France offers and sold for 2.125 million dollars.
Altre Notizie – 29 settembre 2019
There is certainly one very important thing that the first Conte government did, concluded a month ago, which was hardly talked about at all. A very courageous initiative, which the international press has covered a lot. With a concerted action by the Ministry of Health, in particular by the competent director 
Access to medicines: no longer just a problem for the poor. Il mancato accesso ai farmaci essenziali è storicamente una questione che colpisce I Paesi a basso reddito. Una battaglia per il diritto alla salute che compie venti anni, se vogliamo fissare per convenienza il suo debutto internazionale con la mobilitazione della società civile – inclusi medici e pazienti – alla prima conferenza interministeriale dell’World Trade Organization (WTO) in Seattle in November 1999. Ironically, but as was already foreseeable then, the problem has become global. Even rich countries have for some time been facing increasingly insurmountable obstacles, in terms of health budgets, to guarantee their citizens essential care.
How much does research really cost? And yes: how much does research really cost to develop an innovative drug? Each case is unique, and recent genetic science has radically changed the scenarios of research in the pharmaceutical field, so it is not possible to give figures lightly. Since the battle over essential medicines has broken out, starting with antiretrovirals for millions of people affected by HIV/Aids in the southern hemisphere, the figures that estimate the cost of research have been wasted. They have grown from year to year. Often, blown up to become best-selling book titles (The $800 Million Pill, by Merrill Goozner) that revealed the captious narrative of the pharmaceutical industries. The most recent estimate is $2.6 billion.
The logic of profit is not in question. No one questions the corporate need to make profits, but medicines are goods of public utility that should not only be subject to commercial rules applied without discounts on a global scale, without even making the difference between essential medicines and therapies that are not essential. Vice versa, the rules established by the agreements on intellectual property of the WTO, which treat medicines like any other industrial product, give pharmaceutical companies an increasingly dominant position, because they operate in a regime of twenty-year patent monopolies.
What Novartis doesn't say. E’ che Zolgensma, whose sale it expects to make a profit of $2.4 billion a year, is the result of research initially funded by the marathon of Telethon in France. In this case, from a laboratory no profit created on purpose, Genethon, who has worked for years in the field of spinal muscular atrophy which paralyzes the muscles and respiratory system of children, with an investment of between 12 and 15 million Euros raised with the television marathon. The team of scientists had discovered that injecting a certain “viral vector” could correct the faulty gene. In March 2018, Genethon sold its patent to startups American AveXis, which already had Zolgensma in its research portfolio, for 15 million 
Philosophy shapes the price of drugs. As Gilead Sciences had already successfully experimented, in 2013, with the innovative drug Sofosbuvir against hepatitis C (discovered by the biotech Pharmasset then acquired), launched in the USA at the prohibitive cost of 82,000 dollars for a 12-week therapy, and in Italy at a cost of 68,000 Euros, Novartis has completely separated the price of the drug from the cost of its development. If governments and international agencies such as the WHO accept the philosophy of basing the price of medicines on their intrinsic value, this will mean that life-saving medicines will end up costing more than others. And then yes, public health budgets will really be in danger.
Written by Nicoletta Dentico, an expert journalist on global health, he led the campaign to ban anti-personnel landmines and followed the campaign to cancel the debt of impoverished countries. Former director of Doctors Without Borders Italy
Note: Zolgensma is designed to address the genetic cause of SMA by delivering a functional copy of the human SMN gene to arrest disease progression through sustained SMN protein expression with a single intravenous (IV) infusion. Zolgensma is the first and only gene therapy approved by the FDA for the treatment of SMA, including those that are pre-symptomatic at diagnosis.
laria Giancaleoni Bartoli, Director of the Rare Disease Observatory (OMaR), wrote: Holder '2.1 million per dose' distorts the reader's opinion, because the dose is always and only one. Not to mention who called it 'the million dollar pill' by mistaking the type of drug – because it's not a pill but an infusion – and the price. Worse still, 'A single pill costs 1.2 million euros', even making the coin wrong, but thus insinuating the doubt that this price is paid in Europe, perhaps in Italy. All the Italian press headlined the price, the only element that does not apply to Italy: the therapy will be the same, the needs of the children the same, the price will not, that will be established by Aifa, our regulatory body, which knows how to do his bargains are very good. But flaunting the American price, owner of 'the most expensive drug', is accessed on social networks, and never mind if it doesn't do a service to readers.
Related news: Zolgensma for SMA: the most expensive drug in the world, 2.15 million dollars
Why can a drug cost $2.1 million?
SMA, how RNA targeted therapies have changed the history of the disease
Spinal muscular atrophy, the revolution underway: gene therapy is also coming
Zolgensma for SMA: the most expensive drug in the world, 2.15 million dollars
Le critiche riguardano innanzitutto il modo con il quale viene determinato un tale prezzo e gli alti margini di guadagno delle aziende farmaceutiche, tenendo anche conto del fatto che si sa ben poco sui costi di sviluppo di tali medicinali. Novartis non ha sviluppato Zolgensma, ma è diventata proprietaria del farmaco attraverso l’acquisto della società americana AveXis, costata 8,7 miliardi di dollari.
Molte aziende sostengono che i prezzi sono calcolati utilizzando un modello basato sul valore, il che solleva una serie di questioni spinose su quanto vale una vita. Mancano inoltre delle prove sull’efficacia di questi trattamenti e sui rischi potenziali a lungo termine. Alcune terapie giunte sul mercato, come Imlygic, non sono state all’altezza delle aspettative. Sorgono pure questioni etiche legate alle manipolazioni dei geni.
In definitiva, però, la principale fonte di controversie concerne l’onere per la collettività di questi costosi trattamenti. La maggior parte dei sistemi di assicurazione sanitaria non sono previsti per assumere trattamenti a tali prezzi. Novartis ha già detto che sta negoziando con gli assicuratori per consentire pagamenti in cinque anni, a 425’000 dollari all’anno, e per offrire sconti parziali se il trattamento non funziona. Ma non è chiaro come questo verrà messo in pratica in ogni paese. (swissinfo)