The recent announcement of the European authority’s pricing strategy for Strimvelis, the world’s second approved gene therapy, is in line with the predictions groupH made a year ago in “Nature Biotechnology” – but does Strimvelis prove our point or is it a one-off?

In our article, “The Payers’ perspective on gene therapies”, we suggested that setting the price of gene therapy on the basis of a high multiple of the cost of yearly enzyme replacement therapy was going to be difficult to achieve. We indicated that a realistic target was a multiplier of around 2.5 times the cost of annual treatment.

Strimvelis was priced in line with this multiplier at €594k, 2 times the annual cost of the current treatment of enzyme replacement therapy injections.  Other conditions were also attached to the reimbursement, including a money back guarantee and a payment-by-result mechanism. The payment-by-result could result in treatment being reimbursed by GSK in 1 patient out of 6. So, taking that into account, the effective price per patient could be lower at around €500K.

Given the high cost of the alternatives (bone marrow transplants and long-term enzyme injections), and the expectations that were set by Glybera’s price tag of €1.1m back in 2014, Strimvelis’s price may come in under what companies developing new gene therapies would hope for.

The price set now begs the question “does the EU price of Strimvelis create a new ‘glass ceiling’?”

For upcoming gene therapies, will payers in Italy and in other countries – especially the US – accept the circumstances surrounding Strimvelis’s price, and view it less as a reference point and more as an anomaly?

Strimvelis treats an ultra-rare disease, Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency, estimated to affect just 15 patients per year in Europe. It was developed initially through charity money, and furthermore GSK may always have anticipated that it would be a loss maker given the extremely small patient population. This rarity is another reason for questioning its relevance in price setting going forward.

It is hoped that payers will take into account the special circumstances surrounding Strimvelis, and future gene therapies will be fairly recognized for the value created in their indication. Good data for each particular therapy will be required to establish its exact benefits, including longevity of efficacy.

We believe that groupH’s philosophy of needing to inform, engage and listen to all stakeholders early in the drug development process will continue to play a useful role in realizing the full potential value of these life-changing therapies.