Now that the MAA has been agreed, the extended access program for people with type 1 ADM is closed and it is no longer possible to access treatment with the drug through this route. Although there are now official guidelines on MAas on their website, the guidelines are limited and it is therefore essential to use precedents. A MAA can be used to restrict access to patients and reduce the number of patients eligible for treatment, or the scope may be narrower than your marketing authorization. The National Institute for Health and Care Excellence (NICE) makes recommendations on new drugs by verifying clinical evidence and cost-effectiveness. If a drug has promising potential, but there are gaps in clinical data, it may be recommended for temporary access to the NHS in England as part of an AMA. In this way, physicians and the NHS can assess the long-term benefits of a new drug by collecting the results of agreed tests over a period of time in patients with certain symptoms of a disease. At the end of the MAA period, NICE will review the new evidence to make a definitive recommendation as to whether the drug will be available over the long term via the NHS. The following groups/individuals have committed to the agreement: What, if any, role of the Managed Access Agreement (MAA)? 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. NHS England has announced a new example of its negotiations with companies and stressed its willingness to use access to management agreements to ensure the cost-effectiveness of a product.
