We are writing to inform you that the re-evaluation of elosulfase alfa for the treatment of IVa type mucopolysaccharidosis [ID1643] has now been suspended. This is because the company has not provided adequate evidence for the committee`s decision and will not accept the terms of the highly specialized technology pricing process. The terms of the Elosulfase alfa (MAA) access agreement are not changed. Access to alpha elosulfase is continued for new and existing patients if they meet treatment requirements. The re-evaluation of elosulfase alfa for the treatment of type IVa mucopolysaccharidosis (re-evaluation of HST2) [ID1643] was initially planned to announce the result before the MAA expiry date in December 2020. NICE follows the company with its forward submissions and options. We`ll be writing more updates when the time comes. The first MAA was agreed in November 2015 for the use of BioMarin Simizim (Elosulfase alfa) in the treatment of Morquio A syndrome (MPSIVA), a very rare hereditary lysosomal storage disease that affects only 105 children and young adults throughout Britain, including 88 in England. The MAA was signed by NHSE, NICE, BioMarin, a clinical expert and a patient association. Observers hope that these patients, along with other patients with rarer diseases, will not only provide access to effective treatments much earlier, but also that the cost element will have a broader positive effect on the NHS as a whole, where patients who will be denied long years of treatment for generalized diseases for cost reasons. , will have a broader positive effect.
The more confident we are of the number of patients, the more confident the payers will be about our budget impact forecasts; an AMA may define the number of patients who can access treatment over the life of AMA, but appropriate patients need to be identified, i.e. it is necessary to identify the patients most likely to match or benefit most from the technology. But one analyst was not so sure. “The UK`s HTA processes are world-class; They set the scale and, as they are examined after Brexit, if they do not go bankrupt, why should we want to fix them? “,” he asked. And with the previous access process, an early yes shows confidence in the data. Early access means discounts that break the overall price policy – some companies will accept it because an early NICE agreement is so important,” he stressed. The committee concluded that the company, clinical and patient experts and NHS England, as part of an administered access agreement, should take steps to establish evidence of the use of elosulfase alfa in the treatment of MPS IVa by researching and collecting “real” data directly relevant to patients in the UK, including ongoing support of the MPS IVa registry. I will put my trust in MPS Society UK to fight on our behalf to have the disputes between NICE and BioMarin Pharmaceutical resolved before December 2020. It should not be differences between organizations, because we are faced with the real differences that want and need continuous access to Vimizim. “The drug is very expensive – the average cost per year of alfa elosulfase is $394,680 per patient based on the list price; although the company offered a confidential discount as part of a patient access system. Although the committee was satisfied that there was sufficient evidence that some patients were working well with alfa elosulfase, further studies of the benefits and costs in routine clinical practices were required.