Budget impact analysis: comparing the costs of nusinersen and risdiplam in the treatment of type 3 spinal muscular atrophy patients.

OBJECTIVES: The aim of the budget impact analysis (BIA) was to determine the economic impact of introducing risdiplam in the treatment of type 3 spinal muscular atrophy (SMA) patients in a rare diseases reference centre on a 3-year time horizon, as compared with nusinersen. METHODS: Public databases were used to estimate the target population. Two market scenarios were assessed over a 3-year time horizon: with nusinersen and with the introduction of risdiplam. Drug acquisition and administration costs were considered. BIA is calculated as the difference between scenarios with nusinersen - scenario with risdiplam. RESULTS: The introduction of risdiplam would generate a 3-year saving of €3411.50. There could be a saving in the administration of risdiplam in the treatment of patients under the age of 2 with a weight of 5 kg (€26 382.08). CONCLUSION: The BIA shows the overlapping costs of these therapies, although the oral administration of risdiplam could be a decisive factor for the therapeutical switch from nusinersen.

Changes in first-line injectable disease-modifying therapy for multiple sclerosis: predictors of non-adherence, switching, discontinuation, and interruption of drugs.

This study was aimed to describe changes of Disease-Modifying Treatments (DMT) in an Italian cohort of patients with multiple sclerosis (MS) and to identify predictors of therapeutic modifications. Patients with MS and treated with the first-line injectable DMT (interferons-IFNs or glatiramer) between 1/7/2009 and 31/10/2012 were selected from administrative databases of the MS Center of Cagliari (Sardinia, Italy). Socio-demographic, therapeutic, and clinical information was collected in the 6 months preceding the index date. All patients were followed for 36 months to evaluate therapeutic changes in terms of non-adherence, switch, temporary discontinuation, and permanent interruption. Predictors of changes were estimated by multivariable regression models. Data on 1698 patients were collected: glatiramer was prescribed in 27% of cases, IFNß-1b in 22%, IFNß-1a-im in 20%, IFNß-1a-sc-44mcg in 19%, and IFNß-1a-sc-22mcg in 12%. Non-adherence was observed in 25% of cases, therapeutic switch in 30%, discontinuation in 37%, and permanent interruption in 28%. The risk of non-adherence was higher for IFNß-1b, compared with IFNß-1a-im (adjOR = 1.73). Therapeutic switch occurred especially in patients recently diagnosed (each year from diagnosis causes a decrease of this risk adjHR = 0.97); the risk of discontinuation was higher with EDSS = 4-6 and 7-9 (adjHR = 1.52 and 4.42, respectively). The risk of permanent interruption increased with the augmentation of disability (adjHR = 1.67 and 5.43 for EDSS 4-6 and 7-9). This study mirrored a detailed framework of DMT prescription and identified factors related to changes in the MS therapy. These findings could support healthcare providers in the evaluation and maximization of benefits associated with a long-term DMT.