RARE Daily

After NICE Rejection of NPC Therapy, Sanofi Calls Process “Fundamentally Flawed”

March 6, 2024

Rare Daily Staff

The National Institute for Health and Care Excellence, England’s drug price watchdog, issued a final draft guidance that it would not recommend Sanofi’s olipudase alfa, a treatment for the ultra-rare lysosomal storage disorder acid sphingomyelinase deficiency or Nieman Pick disease.

NICE made the decision despite acknowledging that olipudase alfa is the first therapy for the condition and demonstrated evidence of improvement in several clinical outcomes. NICE considers a most plausible incremental cost effectiveness ratio below $128,000 (£100,000) per quality-adjusted life year. It said that both the company’s and an external group’s assessment were in excess of $383,000 (£300,000) per quality-adjusted life year gained.

“The committee was not presented with a plausibly cost-effective estimate after taking into account all of its preferred assumptions and other considerations,” NICE said in its final draft guidance.

An estimated 40 to 50 people in England are living with the lysosomal storage disorder.

Rippon Ubhi, country lead, Sanofi UK and Ireland, noted that the NICE decision stands in stark contrast to Scotland, where the Scottish Medicines Consortium has made olipudase alfa available via its ultra-orphan pathway. The pathway, created to assess medicines for very rare conditions, allows eligible patients to access olipudase alfa whilst additional data is collected for three years.

“We believe this decision demonstrates that the framework for assessing and funding innovative treatments for ultra-rare diseases in England and Wales is fundamentally flawed and is failing to keep pace with innovation,” she said. “In our view, patients with ultra-rare diseases in England and Wales are being let down by an inflexible appraisal system which fails to recognize the benefits and full value of treatments such as olipudase alfa for patients, carers, families and wider society.”

Olipudase alfa is available to patients in a number of other countries, including Germany, Austria, Italy, Cyprus, and Hungary. In England only 59 percent of non-oncology orphan medicines with a marketing authorization are reimbursed, compared to 86 percent in Germany and 77 percent in France, she noted.

Ubhi said the company is seeking to engage with government, NICE, and NHS England to discuss and adapt the way that innovative treatments for ultra-rare conditions are assessed.

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