No winner in drug-price balancing act

Cantillon: Patients have one shot at treatment but what if it is deemed too expensive?

Jordan Perez (11) has a rare muscle-wasting and life-limiting disease. He and his mother, Sinead, like many others, place their hope in access to Spinraza. Photograph: Ciara Wilkinson

There are no winners when it comes to a decision to withhold payment for a new drug.

First there are the patients, often desperate, especially those with a rare disease for which no other treatment is available. Then there is the Health Service Executive, cast in the role of Scrooge but obliged to make decisions based in large part on the available budget. Finally, there are the pharma companies, an easy target, which must try to make a return on costly investment in research and development – including in those many other aspiring drugs that never make it to market.

And amid all this emotion is the National Centre for Pharmacoeconomics (NCPE), charged with making level-headed decisions on cost effectiveness of new drugs against a necessarily subjective set of parameters – most importantly the value one places on the quality of human life.

That is precisely where 26 Irish patients, often small children, with the generally fatal muscle-wasting condition spinal muscular atrophy, now find themselves – alongside Biogen, the company whose drug, Spinraza, has been rejected, health service chiefs and Minister for Health Simon Harris.


Value for money

Is the drug value for money?

The NCPE says no. The price must be cut tenfold to find value.

Despite the clamour in Ireland, the agency’s findings are supported by a new study funded by Biogen and published this month in Sweden where they have actually approved the drug.

And in the US, a report last week by the independent drug-pricing watchdog, the Institute for Clinical and Economic Review, found that the "current pricing of Spinraza would require a substantial discount to meet traditional cost-effectiveness ranges".

Crucially, it added that, “as with all treatments for ultra-rare conditions, judgments of overall value require consideration of contextual issues and broader benefits for patients and families”. Pricing that in a world of tightening budgets is the real challenge.