Last week, EMA approved Strimvelis for the treatment of severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). It is the second genetic therapy approved by EMA after Glybera was cleared in 2012.
The usual discussion goes around rarity, investment and pricing, which have been set at very high levels with a few unknowns on efficacy and long term safety.
Let alone the price and the efficiency, the social need and the safety concerns, I would like to raise a parallel question, from the industrial and economic perspective. If the EU has invested so much in genetic research, is still investing in transferring technologies that will enable more gene therapy, shouldn’t we have some more clarity on how we’re going to pay for this?
The contradiction is obvious: why accumulate technologies for which we are never going to pay for? Or alternatively, if the strategy is to generate competition to break monopolies, will these companies resist the desert valley? If there is no industrial reward, how are we going to raise money to fund RnD?
I guess it is time to discuss about how to proceed with this. The uncertainties are great, the clinical risks unknown, the future developments an unrevealed promise, but yet we should discuss how to look after this emerging industry, or have some kind of mid term strategy.