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. 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? 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. 4.
Who can access the treatment of nusinersen through this MAA? This is an important milestone for ADM patients, as Spinraza 2018 was rejected in the Appraisal Consultation Document (CDA) phase due to concerns about the high price. NHS England`s participation broke the blockade of the negotiations, with both sides showing flexibility and willingness to compromise to ensure patient access, and NICE announced that they had completed their orientation development and would recommend the product. 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. This research identified the collection of observational data as a requirement in all MAAS, primarily through existing registries (with the exception of the Ataluren, which required the development of a custom registry), while the collection of ongoing test data was limited to CDFs. The relatively low cost of using existing registries to meet data requirements, with the ability to obtain a refund while collecting data from current RCTs, makes MAAS an attractive offer for manufacturers. According to reports, NICE plans to strengthen the use of MAA, the ongoing NICE consultation on changes to the evaluation process that may allow for enlargement to include all indications, which would enhance the possibility of exploring innovative MAASs to support future access.