Italy and Spain have a national web platform for which data collection requirements are created for each medicinal product/therapeutic indication. This not only enforces eligibility and maintenance criteria, but also promotes timely data entry for access to processing. However, in Italy, the national platform is not linked to the reimbursement system of hospitals, so prescribing doctors require double data entry. In Spain, the platform is under construction and was not fully functional for tisagenlecleucel. Our case studies also show that there is an opportunity for HTA/payers to work together across jurisdictions to harmonise not only their assessments in order to reach a common agreement on decision-making uncertainties, but also a core data set for HTA data collection. This could significantly improve the timeliness, cost and efficiency of data collection [3] and optimise re-evaluation. In practice, however, this is difficult to achieve. The joint evaluation of NUSINERSEN INNELUXA resulted in an agreement on the need for an international register of ADMs, but each country in this partnership has nevertheless developed its own OBMEA, as shown in Tables 1 and 2. The challenges were also recognised by EUnetHTA, which noted that in the case of rare diseases, “cooperation between Member States to set the minimum data set and collect evidence from different registries and databases is crucial for HTA” [18]. In Austria, tisagenlecleucel had a national agreement on the reimbursed indication agreed by the National Section of the Hematologist Footnote 4, and outcome data are documented in the European Society for Blood and Marrow Transplantation. Reimbursement decisions (including any OBMEA) have been agreed in the regions and are confidential. The review, which involved Biogen, patient groups, clinicians, SMA REACH UK and NHS England, and NHS Improvement, assessed whether the new available evidence would support a change in the eligibility criteria for maa treatments. Specifically, it was examined whether people with type III SMA who cannot walk can benefit from Nusinersen and should therefore be included in the MA.
Bouvy JC, Sapede C, Garner S. Management of entry agreements for pharmaceuticals in the context of adaptive pathways in Europe. Frontal pharmacology. 2018;9:280. Several countries have introduced OBMEAs with a duration of 2-3 years for these treatments, while in England, data collection is at least 3 years to allow re-evaluation within 4.5-5 years or earlier. In the Netherlands, a legal OBMEA with Nusinersen is planned for 7 years, which goes beyond the usual period of 4 years given the small number of patients expected. We are pleased that the review concluded that it is appropriate to expand the clinical approval criteria to allow access to Nusinersen for patients with type III SMA who cannot leave the national online OBMEA systems and automatic summary reports are created that update the level of data collection. When a variety of data sources are used and multiple processing centres are involved, careful monitoring processes are needed to collect enough data, but not all countries have organized it for the case studies. This is essential not only to ensure data quality, but also to monitor patient recruitment and data completeness from all required assessments.
NICE uses a multi-stakeholder managed access oversight committee for the nusinersen. This committee meets every 6 months to discuss data collection issues that arise in clinical practice and monitors the completeness and quality of the data. A difficult issue to address in 2020 was the high level of missing data due to the cancellation of clinic visits due to the COVID-19 pandemic. As a result, additional treatment protocols have been developed and data analysis plans have been modified. Xoxi E, Facey K, Americo C. The development of AIFA registries to support controlled entry into orphan drugs in Italy. Frontal pharmacology. 2021 (Adopted june 2021) “Risdiplam should be used as monotherapy and not in combination with Nusinersen or Onasemnogen. If the clinician treating a patient believes that there has been a deterioration in motor and/or respiratory function after treatment with onasemnogen, he or she may exceptionally refer the case to the NHS England clinical panel. The panel should advise the treating clinician on whether it is clinically appropriate to initiate (or resume) treatment with Nusinersen or Risdiplam. Although the data to be collected in OBMEA were similar in different countries, the duration of the agreements differed and the justification for the duration was unclear. It may have been associated with decision-making milestones such as price and refund negotiations.
Only the Netherlands clearly indicated the expected recruitment period and the duration of the follow-up. . . .
