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? 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 expressed a growing willingness to reimburse innovative products with great uncertainty under the Access to Employment Agreements (ASA), while additional data are being collected on the new Cancer Drug Fund (CDF) or Highly Specialized Technology Channels (HST). The purpose of this research was to verify the data collection rules of the current MAAS. “We have worked hard and constructively with NICE, NHS England, lawyers and doctors, and we are very pleased to have reached an agreement to make available paediatric PPH patients in England who need care the most,” said Ludwig Hantson, Managing Director of Alexion.
“The decision to grant access to Strensiq is an important step for patients and their families.” Since August 2017, children and adults with a rare hereditary bone disease called hypophosphatasia (PPH) have had access to a potentially life-enhancing drug called asfotase alfa (Strensiq). NEW OASE, Conn.–(BUSINESS WIRE)– Alexion Pharmaceuticals, Inc (NASDAQ: ALXN) announced today that it has entered into a national funding agreement with the National Institute for Health and Care Excellence (NICE) and the National Health Service (NHS) England on the basis of an Access to Management Agreement (MAA) that allows patients in England to access Strensiq® (asfotase alfa) with pediatic hypoasiphosphates (PPH). The funding agreement was announced today as part of a positive final assessment (FED) published by NICE Special Technology Assessment COMMITTEE (HST) to recommend Strensiq in accordance with the MAA. With contributions from NICE, NHS England, Alexion and Soft Bones UK, we are pleased to present our information to patients on the Asfotase alfa Managed Access Agreement for the treatment of hypophosphatasia. We would like to thank all those who took the time to comment on the drug and the manacle access agreement to Metabolic Support UK and Soft Bones UK. We will continue to work together to ensure that we continue to represent the voices of those we support. MaA was developed in collaboration between medical thinkers, patient groups, NHS England and Alexion. The MAA guarantees access to Strensiq for infants, children and adults with pediatric PPH who have the most symptoms of disability and who will benefit most from treatment. So far, the drug asfotase alfa has been recommended only for use in babies of NICE in design guidelines showing evidence that it could save lives.