Lavery C, Jones SA, Hughes D, Murphy E, Jovanovic A, Hendriksz CJ, et al. Poster presentation: Impact of elosulfase alfa treatment on patient-reported outcomes in Morquio A Syndrome: results from the first year of an English managed access agreement. International Congress of Congenital Metabolic Disorders (2017). 2017. MAs undoubtedly play a role in providing patients with life-changing treatments in therapeutic areas where there are currently few or no treatment options and where the rarity of the disease makes it very difficult to conduct clinical trials. Many of the conditions treated with highly specialized technologies limit life and therapies have the potential to significantly improve patients` quality of life. However, NICE still has a responsibility to ensure that the level of supporting data is sufficient to justify NHS spending on treatments that are generally very expensive. Of the maAs currently in force, only one has completed data collection (elosulfase alpha for the treatment of mucopolysaccharidosis type IVa), and final NICE guidelines are expected by the end of 2021. So it`s too early to draw conclusions about how effectively MAAs are filling the data gap, but it`s certainly an area to watch. For a MA to achieve its purpose, it is very important that patients attend follow-up appointments and that all necessary data is collected (see Data Collection section), as it is used to prove whether or not there will be future access to treatment after the end of the MA. Highly Specialised Technology Assessments (HST) are carried out by the National Institute for Health and Care Excellence (NICE) to make recommendations on the use of medicines and treatments within the National Health Service (NHS) in England for very rare diseases [1]. The HST process was introduced in recognition of the fact that orphan treatments are rarely cost-effective at the thresholds applied to single and multiple technology assessments (ATT and ATM) [2, 3]. Small patient populations associated with rare diseases can often lead to great uncertainty due to a lack of data on natural history, resource consumption and quality of life [4].
As a result, insufficient evidence and high unit costs often prevent orphan drugs from meeting the cost-effective requirements of health technology assessment (HTA), reducing access to life-changing treatments for patients with rare diseases [5]. If, at the end of the 5-year MA, elosulfase alfa is not recommended by NICE, funding for elosulfase alfa by NHS England will no longer be available to all patients and treatment will be discontinued (the setting will be managed by BioMarin and NHS England to ensure it is controlled). On the other hand, if a treatment is recommended, additional funding from NHS England will be subject to the agreement of commercial terms between NHS England and BioMarin. These potential contingencies were identified to patients before participating in the study to ensure they could make an informed decision. Despite the lack of evidence, past experience with performance-based agreements highlights a number of good practices. These include four main themes: keywords: elosulfase alfa; Managed Access Agreement; Morquio A; Mucopolysaccharidosis type IVA. It is difficult to assess the extent to which performance-based MEAs have been successful to date. Few countries have formally evaluated their experience. Confidentiality of agreements remains a barrier to independent evaluation, and there is little public evidence. However, available information from expert interviews and previous studies suggests that coverage of evidence-based development agreements (ITNs) has so far had a poor record in reducing uncertainty about drug efficacy. Pay-as-you-go (PbR) agreements are still used quite frequently, but they don`t always generate evidence of product performance because the data used to trigger payments isn`t always aggregated and analyzed. The administrative burden of collecting and analysing data on the effectiveness of medicines can also make their execution costly.
Managed Entry Agreements (MEAs) are agreements between companies and healthcare payers that cover new drugs while managing uncertainty about their financial impact or performance. Financial agreements are used in at least two-thirds of OECD countries and EU Member States. Many of these countries also use performance-based agreements that perform hedging, payments to companies, or discounts paid by companies based on product performance, but these MEAs are less common. With the support of the European Commission, the OECD reviewed countries` experience with performance-based multilateral environmental agreements to identify best practices and possible ways to improve the use of these agreements in the future. Managed access agreements provide an essential mechanism for the systematic collection of real-world data to reduce uncertainty about available clinical and economic data, while providing the opportunity to identify patient subpopulations most likely to benefit from a new treatment. This manuscript aims to share the insights of the first managed access agreement initiated following positive conditional approval in 2015 by the National Institute for Health and Care Excellence (NICE) for elosulfase alfa, an enzyme replacement therapy for the treatment of mucopolysaccharidosis type IVA (MPS IVA). This managed access agreement enabled the collection of comprehensive real-world data for patients with MPS IVA, with results showing that patients who started treatment with elosulfase alfa showed similar gains to those in the pivotal study for outcomes such as endurance, respiratory and heart function, pain, quality of life and urinary levels of keratan sulfate….