Novartis has negotiated Zolgensma access for half of the European SMA population, exec says. How'd it get there?

When Novartis reported first-half Zolgensma sales this summer, the company said its spinal muscular atrophy (SMA) gene therapy was growing thanks to "expanding access" in Europe. This week at the Fierce Biotech Cell & Gene Therapy event, an exec outlined what's driving the uptake.

Mike Fraser (Novartis)

So far, Novartis has negotiated Zolgensma access for half of the SMA patient population in Europe, including for patients in England, Scotland, France, Germany, Italy, Belgium and other countries, Mike Fraser, general manager of Europe, Middle East and Africa at Novartis Gene Therapies, said in an interview. Novartis also has deals in place with six countries in the Middle East and Africa, he added.

Zolgensma scored its FDA approval in 2017 and its EU nod in 2020. Even ahead of the EU approval, Novartis was working behind the scenes to ensure a quick rollout in the region.

In some countries, it can take months or years for access negotiations to play out, Fraser said. Babies with SMA “don’t have time to wait,” he added. 

To hit the ground running, the company implemented a “day one" access program. In government access negotiations, Novartis offered outcomes-based contracting, annuity payments or deferred payments, in which the company would cover the cost of treatment upfront and collect payments after access talks play out.

RELATED: New Zolgensma 'inflection point' is here as Novartis snags EU nod for SMA gene therapy

While Novartis has seen progress, Fraser still sees limitations with current healthcare systems. As he sees it, healthcare systems "are still not set up to recognize the value of these one-time therapies." That's despite the fact that expensive gene therapies can bring savings over a patient's life compared with conventional medicines. 

By 2030, Fraser said he expects more than 20 gene therapies to be on the market. While Novartis is on the “cutting edge of interacting with payers,” he urged other companies to start getting involved in their own access talks.

One lesson for Novartis? Because healthcare and financial situations vary from country to country, the pharmaceutical industry is “going to have to get used to offering" a range of access options, Fraser said.

“With these new and innovative treatments, bespoke and customized solutions are going to be the future,” Fraser said.  

Aside from access talks, Novartis is also participating in an effort to screen babies for SMA. Through a partnership with SMA Europe, the pharmaceutical industry aims to screen 100% of newborns across Europe by 2025. 

RELATED: Novartis back with bid for 'multibillion-dollar' Zolgensma expansion in older SMA patients as FDA lifts clinical hold

The European approval of Zolgensma last year was one "inflection point" that Novartis had been anticipating for the gene therapy since its original FDA nod. Meanwhile, the company is advancing a spinal injection formulation for patients between two and 18 years. A potential approval for that use represents a "multibillion-dollar" opportunity, CEO Vas Narasimhan has said.

Zolgensma generated $634 million during the first half of the year, a 69% increase compared with 2020. The medicine is approved in more than 40 countries.