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? 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. The following groups/individuals have committed to the agreement: Now that the MAA has been agreed, the extended access program has been concluded for people with type 1 ADM and it is no longer possible to access treatment with the drug through this route. 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. 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. 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. 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. 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.