The availability of Zolgensma in the USA has also fuelled the price discussion for new drugs in Germany. Zolgensma is approved in the USA for the treatment of children with spinal muscular atrophy (SMA) and can (potentially) cure the disease. The drug has not yet been approved in Germany – European approval is expected next year.
Now there are two big discussions in Germany: On the one hand the handling of drugs that have not yet been approved in Germany and on the other hand the pricing of these drugs.
The handling of drugs prior to approval is clearly regulated in the Drug Hardness Ordinance (AMHV) on the so-called Compassionate Use Programme and provides for a cohort programme care of German patients. The drug is made available free of charge! The individual case regulation, which is now also criticized by the health insurance companies, is also clearly regulated by the so-called “Nikolaus” judgment from the year 2005 and is especially made for life-threatening diseases.
As far as the price is concerned, it should be noted that 1) SMA only affects 500 patients throughout Europe, i.e. approx. 80 patients in Germany (!) – so with 100 insurances on average 1 patient per insurance – and 2) without an expectation for high profit in such ultra orphan diseases, no new therapies and medicines will be available in the future either, as the research and investment incentives are then no more given.
Press article on the issue available in German, e.g. in the Badische Zeitung as of November 20 2019: Hoffnung, Leid und viel Geld as well as a comment.
MArS supports you with the optimal reimbursement strategy, pricing for the D-A-CH market and, of course, in a possible early contact and discussion with the statutory health insurance funds.