The NICE appraisal (ID737) was initiated in 2015 to assess the clinical and cost-effectiveness of sebelipase alfa (Kanuma®) for the treatment of all patients with LAL-D. Despite the best efforts of all parties in the appraisal process, it was not possible to agree on a pathway for patient access, and as a result NICE issued a negative recommendation in February 2017.
Since that time, Alexion, NICE, NHS England, the MPS Society and clinical experts, have worked collaboratively to try to find a route to enable patient access to sebelipase alfa. These efforts culminated in the initiation of a new appraisal in 2022 (ID3995) for the treatment of patients with the rapidly progressive, infantile form of LAL-D (Wolman Disease). In November 2023, NICE was able to issue a positive recommendation for use of Kanuma® in the Wolman patient population (infants who are two years or younger).
However, despite best efforts, NICE was unable to make a recommendation for the use of sebelipase alfa in LAL-D patients with non-Wolman disease, as Alexion was not able to present evidence of cost-effectiveness for this patient population.
On 28 March 2024; NICE discontinued the appraisal issuing the following statement:
“NICE is unable to make a recommendation on sebelipase alfa (Kanuma®) for treating lysosomal lipase deficiency that is not Wolman disease in adults. The Wolman disease population was evaluated separately in NICE highly specialised technology guidance on sebelipase alfa (HST30). The cost effectiveness of the remaining non-Wolman population has not been demonstrated at this stage. We will review this decision if Alexion Pharmaceuticals makes a submission in the remaining non-Wolman disease population”.
Alexion has confirmed that it “remains confident in the clinical benefit that sebelipase alfa offers to all people with LAL-D and are committed to finding a sustainable way forward for access to Kanuma® in non-Wolman patients in the UK”.
Assessment of rare disease medicines through the Health Technology Appraisal process, presents significant challenges to demonstrate the value a product delivers in the treatment of ultra rare conditions. Despite this the MPS Society along with our clinical colleagues remain committed to supporting Alexion if they make a resubmission in the future.
Alexion has confirmed that the termination of the appraisal has no impact for patients with late onset LAL-D who already have access to sebelipase alfa treatment.
