Subscribe and access all BMJ articles and more. The following groups/individuals have signed the agreement: For an MAA to meet its objective, it is very important that patients participate in follow-up appointments and that all necessary data be collected (see data collection section), as this is used to prove whether there will be future access to treatment once the MAA is complete. 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? 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. So what is the 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.
The National Institute for Health and Care Excellence (NICE) has recommended that the first drug be made available through the cancer prevention fund`s new access to management system. According to interim guidelines published in September, osimertinib should be made available to patients with a particularly aggressive form of lung cancer for the next two years, while other efficacy and cost-effectiveness data have been collected and analyzed.1 This is an important step for ADM patients, as Spinraza was rejected in 2018 due to concerns about the high price of the Appraisal Consultation DocumentCD (ACD) phase.