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Spark’s bright idea on $850,000 blindness gene therapy

Published on 03/01/18 at 03:43pm

Spark Therapeutics knew that it had to think very carefully about where it pitched its price for its inarguably ground-breaking gene therapy to treat a specific kind of blindness.

With rumours and rumbles about a potential price tag potentially in excess of $1 million and with its CEO suggesting the treatment held such a value to the wider society, there was huge interest in where the biotech would pitch the price of Luxturna, as the therapy is known.

It revealed that it had finally pegged the price at $850,000 for a one-off treatment, a figure that just about hit the middle-ground of expectations – with estimates ranging between $600,000 and beyond $1 million.

Perhaps of greater interest than the eye-popping price tag (middle-ground or not, make no bones about it, $850,000 is an expensive treatment) is the way in which the company has tried to hedge its bets by offering various methods of making the cost more palatable.

For instance, Spark will offer rebates based on short term efficacy, pitched as between 30 and 90 days after treatment, and longer-term durability, after 30 months. The amount of discount was not released as part of the announcement.

There are also discussions ongoing whereby insurers would pay for the treatment over a period of time, rather than have to pay the full whack of the treatment upfront.

“To help ensure eligible patients have access to Luxturna, we are striving to bring the same level of innovation applied in development to the delivery of, and access to, this product,” said Jeffrey D. Marrazzo, Chief Executive Officer of Spark Therapeutics. “We believe that access to therapy is a shared responsibility among Spark Therapeutics, payers, health benefit providers, physicians and treatment centres. We have been working with stakeholders across the health care sector to help ensure that appropriate patients have access to a product that challenges all of the current conventions of how patients are treated, how products are delivered and how payments are handled.”

The question of price is one that Spark cannot afford to get wrong; one wrong move in the area by, for instance, pricing patients out of a treatment would be disaster for everyone involved – meaning that patients would miss out on treatment and Spark would fail to reap back development costs.

As it is, analysts predict that the treatment could see peak sales of around $300 million. More importantly than this for the biotech, if the payment options are accepted all round then it would lay the groundwork for future gene therapy treatments, including from Spark itself.

It announced in the first half of 2017 that its treatment for haemophilia B, SPK-9001, had achieved consistent and sustained levels of Factor IX activity following gene therapy treatment.

The potential for this treatment to reach a far larger market means that the pricing question is likely to arise again, but will be less openly questioned if Luxturna’s pricing plan unfolds without a hitch.

Ben Hargreaves

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