Managed Access Agreement Nice

It`s also highly unlikely that all patients will meet the admission criteria, so which subpopulations are you willing to compromise? The National Institute for Health and Care Excellence (NICE) has expressed increasing willingness to reimburse innovative products with high uncertainty under Managed Access Agreements (MSUs), while additional data is being collected on the new Cancer Drugs Fund (CDF) or Highly Specialized Technologies (HST). This research aimed to review the data collection rules of current MMA. Emma Harvey is an independent medical advisor specializing in rare diseases and biotechnology. She participated in two NICE Highly Specialized Technology (HST) appraisals after representing Alexion as clinical manager for Strensiq™ (Asfotase alfa) and Kanuma™ (Sebelipase alfa). She represented Alexion in the first appeal of a final assessment decision of NICE (EDF) for an HST, for sebelipase alfa. For both products, she led the creation of managed access agreements and worked closely with experienced physicians, patient groups and NHS England. Since independence, Emma has advised other companies on her HST nice clinical files and whether a Managed Access Agreement (MAA) could help answer outstanding questions. The National Institute for Health and Care Excellence (NICE) makes recommendations on new drugs by checking clinical and inexpensive evidence. If a drug has promising potential, but there are gaps in the clinical evidence, it may be recommended for temporary access to the NHS in England as part of an MAA.

In this way, doctors and the NHS can assess the long-term benefits of a new medicine by collecting the results of agreed tests over a period of time from patients with certain symptoms of a disease. At the end of the MAA period, NICE will review the new knowledge in order to make a definitive recommendation as to whether the medicine will be accessible in the long term via the NHS. This research identified the collection of observational data as a requirement in all MAAS, which is done mainly through existing registries (with the exception of atalurens requiring the development of a custom registry), while ongoing trial data collection was limited to CDFs. . . .