Oversight of the Institutional Review Board and the Ethics Committee did not apply; Patients who received elosulfase alfa under the managed access agreement were cared for by their attending physician in accordance with all local standards of care. Participating patients were required to sign a Managed Access Patient Agreement in which they accepted the terms of the Managed Access Agreement and consented to the collection and sharing of data with the manufacturer, NHS England and NICE. However, despite the additional criteria considered in the HST assessment process, additional data may be required to minimize uncertainty in the rebate process. Subsequently, it may be necessary for products that receive conditional approval under the HST program to provide additional evidence through a Managed Access Agreement (MAID) in order for reimbursement to be considered in the long term . Once the MA has been approved, the Expanded Access Program for Patients with SMA Type 1 is closed and it is no longer possible to access treatment with the drug through this route. As the first MA initiated in response to NICE HST`s comments, it is important to think critically about elosulfase alfa MAA and share relevant results. Despite the recent development of formal nice guidelines on the use of MAs , best practice approaches for MAs in rare diseases are still under discussion and, therefore, the use of precedents and shared evidence could prove crucial for future agreements of this type . By sharing our ideas on this process, we hope to educate the community about the strengths and challenges associated with the practical application of this methodology and, ultimately, guide future agreements of this type. It is also important to recognize that performing longer MAs does not necessarily have to be the solution to improving patient access to new treatments. Ultimately, reducing uncertainty can be both time-consuming and resource-intensive for patients, physicians and manufacturers, and can also delay access to treatment for those who are not eligible to participate in MAID. Therefore, it is important to consider costs and benefits when planning for the generation of actual data after the HTA assessment. In the case of this MA, the original pivotal studies had a placebo-controlled study duration of 24 weeks , so it was assumed that a 5-year MA would significantly reduce uncertainty for elosulfase alfa. However, it may be desirable for manufacturers to prepare before initiating the authorization process, i.e.: as part of the HST process, conduct a full analysis of the information to understand the value of producing additional data before making this commitment .
Manufacturers should also consider the logistical and ethical implications in the event that a treatment does not receive reimbursement, while highlighting the potential risks to patients when setting up the system as described above. Highly Specialised Technology Assessments (HST) are carried out by the National Institute for Health and Care Excellence (NICE) to provide recommendations on the use of medicines and treatments within the National Health Service (NHS) in England for very rare diseases . The HST procedure was introduced in recognition of the fact that orphan treatment is rarely cost-effective when thresholds for single and multiple technology assessments (ATT and ATM) apply [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 . As a result, insufficient evidence and high unit costs prevent orphan drugs from meeting cost-effective health technology assessment (HTA) requirements, reducing access to life-changing treatments for patients with rare diseases . Here we have reported the results of the first MA, which was initiated in response to comments from NICE HST, from the perspective of the patient organisation, the contract research organisation and the manufacturer involved in the initiation. This MA aimed to reduce uncertainty about the short- and long-term efficacy of elosulfase alfa in patients with MPS-IVA while providing much-needed access to treatment. The evaluation process found that patients who started treatment under MAID had similar gains to those in pivotal studies , and that these former patients in the study continued to see benefits in both clinical assessment and quality of life/activities of daily living after nine years . In assessing the strengths and limitations of this process, it is hoped that the results of this MA can be used to inform future agreements. Keywords: elosulfase alfa; Managed Access Agreement; Morquio A; Mucopolysaccharidosis type IVA. Confidentiality of performance-based MEA information relative to interest in such informationNumber of countries where the information is not confidential and where there is interest in such information from other countries, based on interviews with experts from 12 OECD countries(1) using performance-based MEA notes: MEA.
Managed Entry Agreement.- Published: Information is publicly available (e.B. . . . .