- British doctors will be able to prescribe Biogen's spinal muscular atrophy treatment Spinraza following an agreement between the biotech and the UK's cost-effectiveness agency NICE for a time-limited "managed access agreement" that will allow health officials to assess the drug's effectiveness.
- NICE had earlier recommended against use of Spinraza — which carries a list price of 75,000 pounds ($96,300) a vial — judging it to cost at least 400,000 pounds per quality adjusted life year (QALY) gained.
- The agreement follows closed-door meetings in which the two sides had "commercial discussions" on an agreement that would be "consistent with NICE’s framework for determining cost effectiveness." NICE typically recommends new technologies if their cost is below 20,000 to 30,000 pounds per QALY gained.
The public price is never the price actually paid for pharmaceuticals. This truth is even more pronounced when it comes to new rare disease drugs for children where the long-term health implications have yet to be fully measured and remain a sticking point between payers and manufacturers.
In the case of SMA, the most severe form of the disease afflicts infants who usually die before age 2 if untreated. Spinraza (nusinersen) often prevents death, but doesn't always prevent permanent disability like the need for ventilation. Payers are therefore eager to see longer-term data before accepting anything close to the prices biopharma companies are seeking.
NICE, or the National Institute for Health and Care Excellence, had preliminarily rejected Spinraza on the basis of cost last year, but continued discussions with Biogen. An April 30 update posted on the NICE website stated that the appraisal committee had asked NHS England and Biogen to begin private commercial discussions ahead of a final appraisal to be published May 31.
The announcement from the two sides today signifies a positive outcome from those talks. Neither NICE nor Biogen would say whether a discount was offered when asked by BioPharma Dive, citing the confidential nature of the negotiation.
Nonetheless, the difference between NICE's usual cost-effectiveness thresholds and the assessment that Spinraza cost at least 400,000 pounds per QALY suggests Biogen could have offered a lower price.
By comparison, NICE's U.S.-based counterpart, the Institute for Clinical and Economic Review, had assessed Spinraza as costing $1.1 million per QALY. A big difference is the list price, which in the U.S. is $125,000 per vial.
NICE spokesman Phil Ranson said the duration of the limited-time agreement will be revealed when the agency's final appraisal of Spinraza is published.
The conclusion of the Biogen-NICE talks also hints at what Novartis will face should its SMA gene therapy Zolgensma (onasemnogene abeparvovec) eventually receive approval by European or British health regulators.
Novartis has argued for a multi-million-dollar price tag for the one-time treatment based on health benefit, but NICE will likely want to give it the same type of scrutiny it's giving Spinraza.
Payers and pharma companies alike have suggested paying for gene therapies in installments based on continuing positive patient outcomes.