Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy (SMA) Receiving Disease Modifying Therapies (DMT) - Real-world Analysis Using the Komodo Claims Data
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 4,114
- 试验地点
- 1
- 主要终点
- Number of Patients by DMT During Each Year of DMT Treatment
研究概览
简要总结
The aim of this study was to assess real-world dosing patterns, long-term healthcare costs, and characteristics of SMA patients who received treatment with DMTs. DMTs included onasemnogene abeparvovec, nusinersen, and risdiplam. This study was conducted using both open and closed claims data from the Komodo Health Research Database (KRD+) between 01 January 2016 and 31 October 2024.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Nusinersen and Risdiplam Cohorts:
- •Inclusion criteria
- •Patients with ≥1 SMA diagnosis (International Classification of Diseases, Tenth Revision, Clinical Modification [ICD-10-CM] codes: G12.0, G12.1, G12.9) at any time
- •Patients with ≥1 record of nusinersen or risdiplam in the data based on relevant Healthcare Common Procedure Coding System (HCPCS) codes and National Drug Code (NDC) on or after December 23, 2016
- •Patients aged ≥ 2 at the index date
- •Patients with ≥ 1 quarter of clinical activities within 1 year prior to the index quarter (i.e., the quarter containing the index date)
- •Patients with ≥ 2 quarters of clinical activities in the first year of the follow-up
排除标准
- •Onasemnogene Abeparvovec Intravenous Infusion (OAV) Monotherapy Cohort:
- •Inclusion criteria
- •Patients with ≥1 SMA diagnosis (ICD-10-CM codes: G12.0, G12.1, G12.9) at any time
- •Patients with ≥1 record of OAV in the data based on relevant HCPCS codes and NDC on or after May 26, 2019
- •Patients under 2 years old at the index date
- •Patients with ≥ 2 quarters of clinical activities in their first year of follow-up
- •Exclusion criteria
- •Treatment with nusinersen or risdiplam at any time
研究组 & 干预措施
Nusinersen Cohort
Patients with SMA aged two years or older who received nusinersen.
Risdiplam Cohort
Patients with SMA aged two years or older who received risdiplam.
Onasemnogene Abeparvovec Intravenous Infusion (OAV) Monotherapy Cohort
Patients with SMA who were under two years of age and received OAV monotherapy.
结局指标
主要结局
Number of Patients by DMT During Each Year of DMT Treatment
时间窗: Years 1, 2, 3, 4, and 5
Number of Patients Using Nusinersin During the Entire Treatment Follow-up Period
时间窗: Up to 5 years
Annualized Drug and Drug Administration Costs During Each Year of Nusinersen Treatment
时间窗: Years 1, 2, 3, 4, and 5
Annualized Drug and Drug Administration Costs for Nusinersen During the Entire Treatment Follow-up Period
时间窗: Up to 5 years
Number of Nusinersen Injections During Each Year of Treatment
时间窗: Years 1, 2, 3, 4, and 5
Number of Patients Using Risdiplam During the Entire Treatment Follow-up Period
时间窗: Up to 4 years
Number of Nusinersen Injections During the Entire Treatment Follow-up Period
时间窗: Up to 5 years
Annualized Drug Costs During Each Year of Risdiplam Treatment
时间窗: Years 1, 2, 3, and 4
Annualized Drug Costs for Risdiplam During the Entire Treatment Follow-up Period
时间窗: Up to 4 years
Annualized Risdiplam Costs Among Patients With More Than 90 Days of Supply in Year 1
时间窗: Year 1
Mean Prescribed Risdiplam Dosage During Each Year of Treatment
时间窗: Year 1, 2, 3, and 4
Mean Number of Days of Supply of Risdiplam During Each Year of Treatment
时间窗: Year 1, 2, 3, and 4
Mean Daily Dosage of Risdiplam During Each Year of Treatment
时间窗: Year 1, 2, 3, and 4
Mean Prescribed Risdiplam Dosage During the Entire Treatment Follow-up Period
时间窗: Up to 4 years
Mean Number of Days of Supply of Risdiplam During the Entire Treatment Follow-up Period
时间窗: Up to 4 years
Mean Daily Dosage of Risdiplam During the Entire Treatment Follow-up Period
时间窗: Up to 4 years
次要结局
- Annualized All-cause Healthcare Costs During Each Year of OAV Follow-up(Years 1, 2, 3, and 4)
- Annualized All-cause Healthcare Costs During the Entire OAV Follow-up Period(Up to 4 years)
- Annualized SMA-related Healthcare Costs During Each Year of OAV Follow-up(Years 1, 2, 3, and 4)
- Annualized SMA-related Healthcare Costs During the Entire OAV Follow-up Period(Up to 4 years)