Patient population and cost assumptions for SMA -related health care and drugs and management costs
Patient group
The average SMA type 1 incidence rate of 5.4 patients (Dutch population, 2015-2019) and expert opinions (personal communication, L. van der pol and F. Asselman) estimated 5.1 patients in 94%of the patients in 94%of the patients. I was able to receive SMA treatment to receive treatment. 2015–2019) (14). Therefore, the patient is an onasemnogene Abeparvovec treatment (ie, anti-adeno-related serin serin serum type serum type serum type serum-type serum type serum type serum type serum type serum type serum type serum type serum type serum type serum type serum type serum type 1:50 In the case, only the newly diagnosed patient was excluded from the diagnosis.17,,,1819). Eligible patients were classified as two cohorts: Onasemnogene Abeparvovec or NusinersenNDivision per group = 5.1). The data comes from not only published sources but also health technology evaluation and local experts.
SMA -related health care costs
The annual SMA -related health care cost and survival rate for SMA type 1 patients came from the Netherlands Nusinersen repayment report, which is openly available and assumed that it is the same per cohort. SMA -related health care costs were calculated by the treatment category (ie, respiratory, gastrointestinal tract, nutrition, and orthopedic surgeon) (Table 1, Figure S1A, Figure S1B) (6, 20). Side effects are assumed to be the same between processing; Therefore, the cost related to side effects was not included. Similarly, due to the characteristics of SMA, it was difficult to separate the side effects of treatment from SMA -related complications, which is already described in the cost of health status (2, 3). Nusinersen therapy included actual treatment costs to estimate the health care costs of patients inappropriate (NUSINSEN repayment reports (20)). Patients who have stopped nusinersen (ie, non -metaphor patients) are not included in the analysis of health care costs.
Drug and management cost
The cost of drugs for each treatment was derived from G-Standard Databank; Annual drug costs were calculated according to the label administration guidelines (Figure S1A and S1B)6,,,78). Each price of OnASEMNOGENE Abeparvovec and Nusinersen was € 1,945,000 and € 85,733, respectively, and € 3,800 of € 3,800 per person, respectively. OnASEMNOGENE Abeparvovec is managed only once, so there is no annual treatment cost. Nusinersen treatment, when four load doses start (within the first 63 days), the maintenance capacity (e.g. 8, 9) (e.g., 6 times in the first year after the start of treatment) (€ 514,395); 22,800 € 11, 257, 3 dosage in the drug;6,,,78).
In order to integrate compliance measurements into the model, we included an average dose 2.499 for the patient treated after loading capacity (4). All patients treated with nusinersen were assumed to comply with the first four loading capacity. Nusinersen’s price discounts are subject to commercial trust contracts. Therefore, there is no data in the open area. Our analysis did not include price discounts or price epidemiology.
Model structure and specifications
The cost comparison model was built using Microsoft Excel 2016 © Microsoft Inc (Microsoft Inc, Richmond, VA, USA) (Figure 1). The primary model output was a difference between the total cost per treatment and the cost between the OnASEMNONENE Abeparvovec- and the nusinersen-treated patients. Separate drug costs, management costs, and SMA -related health care costs per patient are offered outside the total cost per treatment.
Cost comparison analysis model structure. The reason why you are not qualified to be treated with OnASEMNOGENE Abeparvovec is the hesitation of parents to start high anti -AAV9 -AB and gene alternative therapy. AAV9-AB, adeno-related virus serum type 9 antibodies; SMA, spinal muscle atrophy
Basic case analysis
The basic case analysis was used for 20 years of time horizon according to the long -term investment time horizon and chose to capture the relevant costs of the decision makers within a limited period of time to reduce uncertainty. Background mortality was not included. The average age of clinical diagnosis was 4.8 months, as observed in the Dutch clinical practice (personal communication, utecht utrecht; SMA patient data in the Netherlands; 2015-2019). Only drug and health care costs were evaluated according to the perspective of payment. Social costs such as travel, informal treatment and productivity loss were not included (21). All cost results were discounted at a 4%rate every year as required by the Dutch Economic Guidelines and were indexed on the value of 2023 euros (21 and 22).
Interest in the breakthrough
In order to assess the time that the patient must treat Nusinersen to generate the same cost as the prepaid cost of onasemnoene Abeparvovec, the gains and breaks are analyzed.
Determination of sensitivity analysis
Determined sensitivity analysis (DSA) was performed to analyze the impact of changes in major parameters on cost differences. Major parameters included a percentage and management cost of patients with discount rate, drug costs, health care costs, incidence, time horizons, and onASEMNOGENE Abeparvov. The parameter value has only changed ± 20%.
Probabular
A probabilistic sensitivity analysis (PSA) with 1,000 repetitions was performed for economic and epidemiological parameters (Table S1). Economic parameters included SMA -related health care costs, as well as costs associated with the treatment and administration of Nusinersen or OnASEMNOGENE Abeparvovec, and epidemiological parameters included SMA type 1 incidence.
Scenario analysis
Scenario analysis was obtained by the literature -based variation of model input families, drug costs and alternative assumptions of time horizons. Analysis was analyzed by changing the percentage of patients in the time horizontal, OnASEMNOGENE Abeparvovec, and changing the incidence, drug cost, health care costs, management costs, and discounts for patients with SMA type 1 (Table 2). Scenario analysis was also performed to evaluate the cumulative effects of the combined households of cost differences. Finally, over time, sustainable scenario analysis was performed to explain the changes in the number of patients remaining in the treatment of nusinersen. Using persistent input, we evaluated the meaning of patients who stop nusinersen treatment for the first two years after the start of treatment.
