Vimizim Managed Access Agreement

December 20, 2020 | Category: Uncategorized

However, other experts have warned that the proposals could exacerbate the serious problems of access to treatment that already exist in people with rare diseases. According to one industry, it was highly unlikely that the cost per QALY of rare disease treatments would be less than or equal to $10,000, making them inoperative with the proposed expedited assessment route. In addition, there is evidence to suggest that none of the products that went through NICE`s HST program would result in a cost per QALY of $100,000 or less. As part of its highly specialized technology programme, NICE Vimizim (Elosulfase alfa) recommended the treatment of type IVa mukopolysaccharidosis (also known as DEP IVa and Morquio A SYNDROMES), which affects only 88 people in England, as part of an access to management agreement between the drug manufacturer and the NHS. People in England who suffer from extremely rare Morquio A syndrome should now have access to the only drug for their condition, in accordance with a groundbreaking agreement between NHS England and the manufacturer. 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. Emma Harvey is an independent medical advisor specializing in rare diseases and biotechnology. She participated in two aperitifs of special technology DE NICE (HST) and represented Alexion as clinical director of Strensiq™ (asfotase alfa) and kanuma™ (sebelipase alfa).

She represented Alexion on the first appeal against a determination of the final evaluation of NICE (FED) for an HST, for sebelipase alfa. For both products, she led the creation of Managed Access Agreements and worked closely with specialist physicians, patient groups and NHS England. Since her independence, Emma has advised other companies on their NICE HST clinical records and whether a Hand Access Agreement (MAA) can help answer unanswered questions. The correct application of this mechanism, put in place three years ago, should mean that no further budgetary control measures are necessary to limit the state`s risk. But, he said, the PPRS cap is still not being used to improve access – while it would improve the health of the population at no cost to the government. A MAA is a hybrid document. To begin the development of an MAA, you must assume that it is a clinical document, but you must have a commercial agreement for the evaluation points collected. It is also very unlikely that all patients will meet the marketing authorisation criteria, so which subpopulations are ready to act? 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.

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