On December 23rd 2016, the US Food and Drug Administration (FDA) issued Biogen with an authorization for Spinraza (Nusinersen). The drug is approved for use for all American patients affected with spinal muscular atrophy (SMA), regardless of disease type. AFM-Telethon welcomes this landmark development which opens the way for treating an incurable, devastating and extremely life-threatening condition for its most severe forms.
This major achievement should now benefit European patients rapidly. The authorization file is under examination by the European Medicines Agency (EMA). AFM-Telethon strongly hopes that the EMA follows in the FDA’s footsteps in terms of short timelines.
Without waiting for the EMA’s decision which is likely to be made in a few months’ time, AFM-Telethon calls upon Biogen to make the broadest possible use of France’s regulatory framework in the field of early access to innovative drugs, i.e temporary use authorisations (TuA), deemed to be one of the best in the world. AFM-Telethon therefore wishes that Biogen files, as quickly as possible, a TuA by cohort application with the French Medicines Agency, for all patients involved in the pending drug authorization application with the EMA, and not only for some forms of the disease.
For patients with SMA, expectations are extremely high. Unfortunately, the announcement made by Biogen of the price claimed for the drug in the United States was truly shocking. The extremely high price level claimed for Spinraza came as a surprise to all analysts, as used as they are to the staggering drug price escalation witnessed in the last few years. With a 750,000 $ price tag for the first year of treatment, and then a yearly price of 375,000 $, healthcare systems are faced with an exorbitant bill that is practically impossible to sustain. In France alone, public health authorities would have to foot a bill of over a billion euros in order to finance the initial year of treatment for 1,500 patients!
As an active drug promoter, we are fully aware of the high risks and costs involved in the development of innovative drugs for treating rare conditions with no alternative therapy options. It is therefore only normal that costs be redeemed and innovation rewarded. However, it is unacceptable that such efforts generate inflated margins that will prevent many patients from accessing therapy.
Research and production costs as well as the large number of patients concerned (tens of thousands in the United States and Europe) cannot justify such high price levels. Besides being the first drug against spinal muscular atrophy, Spinraza would become the costliest medicine in human history! In a tight budgetary context, such a pricing policy would put into question the very access to innovative drugs, far beyond Spinraza alone. As a patients’ organization, our responsibility is to stand for access for treatment for all. That is why AFM-Telethon urges Biogen to disclose in full transparency all necessary information that will account for the price levels claimed for Spinraza.