Asfotase Alfa Managed Access Agreement

This research identified the collection of observational data as a requirement in all MAAS, mainly through existing registries (with the exception of atalurens requiring the development of a custom registry), while ongoing trial data collection was limited to SFDs. The relatively low cost of using existing registries to meet data requirements, with the ability to obtain reimbursement while collecting data from ongoing RCTs, makes MAAS an attractive offering for manufacturers. According to some information, NICE plans to increase the use of AAMs, nice`s ongoing consultation on changes in the evaluation process could allow it to be extended to all indications, which would mean more opportunities to explore innovative MMA in order to support access in the future. The MAA was developed in collaboration with doctors` thinkers, patient groups, NHS England and Alexion. The MAA provides access to Strensiq for infants, children and adult patients with pediatric PPH, who have the most disabling symptoms and are expected to benefit most from treatment. Twenty-two MMA were identified (19 by CDF; three by HST). All MAAS included a data observation component. Existing NHS databases (19/22 MMA: 86.5%), existing independent registries (one MAA: 4.5 per cent [ataluren]) were the source of the collection of observational data; Custom-made MAA register maintained by the manufacturer (1/22 MAA: 4.5 % [asfotase alfa]) and register developed under the administrative authorisation and maintained by the manufacturer (1/22 MAA: 4.5 per cent [elosulfase alfa]). Only eight MAAs (asfotase alfa, ataluren, elosulfase alfa, brentuximab vedotin, venetoclax, ibrutinib, daratumumab and pembrolizumab) had observational data collection as their primary data collection method. In addition, 17/22 MMA (77 percent, all from the CDF) also required ongoing collection of data from clinical trials as a key component of the data collection agreement. So far, the drug asfotase alfa has only been recommended by NICE in the draft guidelines for use in babies, as it has shown that it can save lives. The managed access agreement, along with the new 2017 guidelines, meant that access to the drug has been extended to infants, children and adult patients with pediatric PPH who have the most disabling symptoms and are expected to benefit the most from treatment.

The agreement between NICE, NHS England and Alexion has a duration of 5 years and allows for the collection of additional information and data. . . .