Renting a lifetime therapy vs. Buying a ‘one-off’ – Has the high price tag historically hindered reimbursement?


Only recently did FDA approve Zolgensma® (by AveXis, a Novartis company), a gene therapy costing 2.1$ million, making it the most expensive medicine in the history of pharma.

Zolgensma®’s most compelling feature is that it is a ‘one-off’ treatment. It utilises a viral vector to inject genetic code into the malfunctioning neurons directly; as viruses innately inject genetic code into their target cells. The result is a potential cure from a one-time treatment, where defective genes are effectively fixed. In terms of patient applicability and safety, Zolgensma® can cover approximately 80% of Spinal Muscular Atrophy (SMA) patients and comes with a Black Box Warning for acute liver injury. Additionally, the long-term treatment cost is eventually less than that of the competitive Spinraza®.


On the other hand, Spinraza® (by Biogen) has the potential to cover all SMA patients and is, contrastingly, a lifetime maintenance treatment. For the first year, Spinraza® occurs an upfront cost of 750,000$ and for the following years 375,000$ for maintenance.

Thus, despite Zolgensma®’s big high price tag, a few years of Spinraza® can eventually incur the same spend for payers. The main concerns around long-term efficacy and impact on cash flow, however, remain on the horizon; leaving room for innovative risk-sharing or even cost-spreading agreements to come into play in response. Almost more than a year ago, Spinraza® achieved reimbursement by the Spanish government under a confidential agreement incurring a substantial discount.


A recent GPI study presented at the World Orphan Drug Congress USA 2019, showed that, despite its high cost, Biogen’s Spinraza® had achieved considerable global coverage and patient access. In a sample of 83 countries, Spinraza® achieved access:

  • via formal reimbursement (statutory, national level) in 30 countries (36.1%),
  • via individual reimbursement (alternative route, i.e. regional/free samples) in 8 countries (9.6%)
  • via a Named-Patient Program (NPP) in 6 countries (7.2%)

The market is under the microscope as reimbursement and access in health systems across the globe will heavily depend on payers’ willingness to pay for Zolgensma® as well as Novartis’ willingness to negotiate price. ‘’


By Dimitra Sotou – P&MA Consultant & Shivani Shah – Business Analyst