Case Study – Zolgensma (Novartis): World’s most expensive drug → debates on affordability of gene therapy

Case Study: Zolgensma (Novartis) – The World’s Most Expensive Drug

Drug: Zolgensma (onasemnogene abeparvovec)
Company: Novartis Gene Therapies (AveXis subsidiary)
Indication: Spinal Muscular Atrophy (SMA) – a rare, often fatal genetic disorder in infants.
Approval: FDA (2019), EMA (2020), other global markets followed.
Price: Approx. $2.1 million per patient (single dose) → making it the world’s most expensive medicine.

Affordability: The high one-time cost sparked debates worldwide on drug pricing, equity, and access.
Reimbursement: Many healthcare systems struggled to decide whether and how to cover Zolgensma.
Access Inequality: Patients in low- and middle-income countries faced challenges due to lack of insurance and reimbursement policies.
Regulatory Scrutiny: Concerns about data manipulation in preclinical studies submitted to the FDA.

Novartis Pricing Justification:
- Claimed that the one-time therapy is more cost-effective than lifelong SMA treatments (Spinraza, Risdiplam), which may exceed several million dollars over a patient’s lifetime.
- Highlighted that Zolgensma offers potential cure, not just management.
Reimbursement Models:
- Introduced installment payment plans (spread over 5 years) and outcomes-based agreements with insurers.
- Some countries negotiated lower prices through health technology assessments (HTA).
Regulatory Oversight:
- FDA investigated data manipulation but allowed Zolgensma to remain on the market due to its proven clinical benefit.

Drug Pricing & Ethics: Raised global debates on fairness in pricing life-saving medicines.
Regulatory Vigilance: Highlighted the importance of data integrity in advanced therapy approvals.
Healthcare Policy: Pushed innovation in value-based pricing and payment models.
Patient Advocacy: Sparked stronger advocacy for rare disease patients to ensure faster approvals and access.