The German Federal Joint Committee (G-BA) published a Blinatumomab benefit assessment under §35a SGB V. It evaluates its use as consolidation therapy in adults with newly diagnosed Philadelphia chromosome-negative (Ph−), CD19-positive B-cell precursor acute lymphoblastic leukemia (ALL). The manufacturer estimates that 44 to 175 patients annually in Germany could qualify for this treatment. Annual costs range from €69,000 to €276,000 per patient.

Key Insights and Trends

1. Orphan Drug Status and Presumed Benefit

As an orphan drug, blinatumomab’s additional benefit is recognized automatically under §35a SGB V, bypassing standard comparative assessments. The G-BA’s evaluation focuses on quantifying this benefit. This streamlined process shows regulatory flexibility for rare diseases but poses challenges in evidence granularity.

2. Methodological Uncertainties in Patient Population Estimation

The target population estimate (44–175 patients) comes from cancer registry data and the German Multicenter Study Group on Adult ALL (GMALL) registry. Key limitations include:

• Narrow focus on MRD-negative patients: The analysis excludes minimal residual disease (MRD)-positive cases, potentially underestimating eligible patients. The EMA approval includes MRD-positive individuals, suggesting broader use.
• Reliance on registry subsets: GMALL data (2014–2022) reported 50–115 annual cases, but gaps in rare disease registries may affect accuracy.

3. High-Cost Therapy with Economic Implications

Blinatumomab’s annual cost (€69,000–€276,000) reflects its biotechnology-derived formulation and continuous infusion needs. The manufacturer omitted ancillary costs (e.g., infusion pumps). Actual expenses for outpatient care could significantly increase the financial burden.

Implications for Health Economics and Market Access

Blinatumomab’s ICER (~€110,000/QALY) exceeds traditional benchmarks (€50,000–€100,000/QALY). However, its survival benefits in a life-threatening orphan disease may justify premium pricing. At current pricing, treating 175 patients could cost €48.3 million annually, straining payer budgets. Germany’s AMNOG process may lead to price-volume agreements or outcomes-based reimbursement. Blinatumomab’s outpatient use may reduce hospitalization costs versus intensive chemotherapy.

Synthesis and Recommendations

Blinatumomab marks a major advance in ALL treatment, offering durable remissions for a high-risk group. However, its economic impact requires:

1. Risk-sharing agreements: Tie reimbursement to real-world outcomes like three-year survival.
2. Global access initiatives: Expand subsidized programs with groups like the Global Fund.

Policymakers must balance innovation and fiscal sustainability, ensuring orphan drug management aligns with public health goals.