The National Institute for Health and Care Excellence (NICE), the United Kingdom’s watchdog on drug value, has recommended Spinraza as the first treatment for the rare and deadly muscle-wasting disease spinal muscular atrophy.

The positive recommendation is for the treatment of infants, children and adults with 5q spinal muscular atrophy (SMA), including pre-symptomatic and symptomatic SMA Types 1, 2 and 3. The recommendation follows the reaching of a deal between the Biogen, the maker of Spinraza, and National Health Service (NHS) England, the publicly funded national healthcare system.

“This promising treatment has the potential to be life changing for children and their families,” stated Simon Stevens, NHS England chief executive. “The NHS has now reached one of the most comprehensive deals in the world, which allows us to assess real-world evidence of its long-term benefits.”

The decision ends an impasse between NICE and Biogen and could lead to similar resolutions with other drugmakers over high-priced products for rare diseases. It is estimated there are between 600 and 1,200 children and adults in the United Kingdom living with SMA.

NICE had previously refused to recommend Spinraza for routine use because of uncertainties over its long-term effectiveness and its high cost. In August 2016, NICE rejected Spinraza at a cost of $578,000 (£450,000) for the first year and $289,000 (£225,000) for following years. In the United States, Spinraza has a price tag of $750,000 for the first year and $375,000 for subsequent years.

Though NHS and Biogen did not release detailed terms of their agreement, the NHS said it will fund treatment with Spinraza for a time-limited period to collect additional data on its effectiveness. Biogen will immediately make the treatment available to patients with SMA type 1, the youngest and most severely affected SMA patients. NHS England will offer funding on NICE’s publication of final guidance next month.

For older babies, children and young adults with SMA types 2 and 3, the NHS will begin to roll out Spinraza shortly after NICE’s guidance is published.

NICE has been the target of criticism by both industry and patient groups for having too restrictive a framework for determining the value of rare disease drugs. In February, MAP BioPharma, a biopharmaceutical consultancy, issued a report critical of NICE’s approach, which it said blocked access to innovative new medicines. It called on NICE to adapt more flexible approaches to determining the value of rare disease medicines.

“The committee has recognized that [Spinraza] is a promising treatment that has been shown to improve a range of outcomes important to patients. But it also recognized that there are significant uncertainties, particularly around its long-term benefits,” said Meindert Boysen, director of the Centre for Health Technology Evaluation at NICE. “All along we have felt it important to give all parties every opportunity to try to find a way to mitigate these uncertainties in order to make [Spinraza] available to patients in England.”

No matter how good any drug is, patients can’t benefit from if they are unable to get access to it. Boysen said the agreement shows that when companies demonstrates appropriate flexibility, it is possible to find a way to provide important treatments to patients in a way that is cost effective for the NHS and taxpayers.

It’s unclear how much of the agreement reflects flexibility on the part of the NHS or Biogen, but hopefully it will point a way forward for other drugmakers to find workable solutions to resolve pricing matters with NICE and open the door for greater access for rare disease patients to needed therapies.

Photo: Simon Stevens, NHS England chief executive

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