Novartis Pharmaceuticals Australia Pty Ltd.

This analysis evaluated the cost-effectiveness of newborn screening (NBS) for spinal muscular atrophy (SMA) from the perspective of Australian state hospital payers.

A cost-utility analysis consisting of a decision tree and Markov cohort designed to calculate the difference in costs and health outcomes between two scenarios: (1) disease-modifying treatment (DMT) for SMA after diagnosis through NBS, and (2) DMT for SMA after diagnosis as symptoms appear. A population of 295,906 newborns was modeled, based on the total number of live births in Australia in 2023. Inputs included screening parameters, epidemiology inputs, SMA natural history data and DMT parameters (nusinersen and onasemnogene abeparvovec), costs, and health-related quality of life parameters. Assumed participation in NBS was 100%. A one-way sensitivity analysis and probabilistic sensitivity analysis were conducted to examine the impact of parameter uncertainty.

There were 30 patients identified with SMA, of whom 25 patients would be eligible for presymptomatic treatment. NBS for SMA was dominant compared with no NBS for SMA. On a population level, NBS demonstrated a lifetime gain of 267 quality-adjusted life years (QALY) and incremental costs of -AUD$3,983,263 (i.e., cost savings). Every dollar invested in NBS would save hospitals $3.69. Deterministic and probabilistic sensitivity analyses demonstrated the robustness of the base-case results.

NBS for SMA was dominant compared with no NBS for SMA in Australia from a state and territory payer perspective. Universal implementation of NBS for SMA would support access equity, as well as early diagnosis and treatment in infants with SMA, potentially leading to improved outcomes.

Siponimod is approved for use in people with secondary progressive multiple sclerosis (pwSPMS). An integrated digital platform, MSGo, was developed for pwSPMS and clinicians to help navigate the multiple steps of the pre-siponimod work-up.

To explore real-world onboarding experiences of siponimod amongst pwSPMS in Australia.

Retrospective, non-interventional, longitudinal, secondary analysis of data extracted from MSGo (20 April 2022). The primary endpoint was the average time for siponimod onboarding; secondary endpoints were adherence and sub-group analyses of variables influencing onboarding.

Mixed-cure modelling estimated that 58% of participants ( N = 368, females 71%, median age of 59 years) registered in MSGo would ever initiate siponimod. The median time to initiation was 56 days (95% CI [47–59] days). Half of the participants cited ‘waiting for vaccination’ as the reason for initiation delay. Cox regression analyses found participants with a nominated care partner had faster onboarding (HR 2.1, 95% CI [1.5–3.0]) and were more likely to continue self-reporting daily siponimod dosing than were those without a care partner (HR 2.2, 95% CI [1.3–3.7]).

Despite the limitations of self-reported data and the challenges of the COVID-19 pandemic, this study provides insights into siponimod onboarding in Australia and demonstrates the positive impact of care partner support.

Evidence from network meta-analyses (NMAs) and real-world propensity score (PS) analyses suggest monoclonal antibodies (mAbs) offer a therapeutic advantage over currently available oral therapies and, therefore, warrant consideration as a distinct group of high-efficacy disease-modifying therapies (DMTs) for patients with relapsing multiple sclerosis (RMS). This is counter to the current perception of these therapies by some stakeholders, including payers.

A multifaceted indirect treatment comparison (ITC) approach was undertaken to clarify the relative efficacy of mAbs and oral therapies.

Two ITC methods that use individual patient data (IPD) to adjust for between-trial differences, PS analyses and simulated treatment comparisons (STCs), were used to compare the mAb ofatumumab versus the oral therapies cladribine, fingolimod, and ozanimod.

As IPD were available for trials of ofatumumab and fingolimod, PS analyses were conducted. Given summary-level data were available for cladribine, fingolimod, and ozanimod trials, STCs were conducted between ofatumumab and each of these oral therapies. Three efficacy outcomes were compared: annualized relapse rate (ARR), 3-month confirmed disability progression (3mCDP), and 6-month CDP (6mCDP).

The PS analyses demonstrated ofatumumab was statistically superior to fingolimod for ARR and time to 3mCDP but not time to 6mCDP. In STCs, ofatumumab was statistically superior in reducing ARR and decreasing the proportion of patients with 3mCDP compared with cladribine, fingolimod, and ozanimod and in decreasing the proportion with 6mCP compared with fingolimod and ozanimod. These findings were largely consistent with recently published NMAs that identified mAb therapies as the most efficacious DMTs for RMS.

Complementary ITC methods showed ofatumumab was superior to cladribine, fingolimod, and ozanimod in lowering relapse rates and delaying disability progression among patients with RMS. Our study supports the therapeutic superiority of mAbs over currently available oral DMTs for RMS and the delineation of mAbs as high-efficacy therapies.