Novartis ‘Urgently’ Working To Improve Zolgensma Access Program

Criticism of Novartis/Avexis’s controversial lottery style managed access program for Zolgensma has come largely from Europe, while other countries in Latin America and Asia have been far more receptive.

Novartis is continuing with its Zolgensma managed access program

Amid fierce criticism of the lottery style managed access program for the gene therapy Zolgensma, Novartis AG/AveXis Inc. say they are working “with a sense of urgency” with clinicians and patients to improve the program. Avexis also warned at the Expanded Access Summit in Washington, DC that bad press could persuade companies with gene therapies to avoid managed access programs altogether.

Novartis/Avexis came under attack from EU member states, patient groups and legal experts for its managed access program for Zolgensma, a one-off gene therapy for treating spinal muscular atrophy. (Also see "Zolgensma ‘Lottery’ Attacked From All Sides" - Pink Sheet, 6 Feb, 2020.) 

Under the program, potential recipients are chosen through a blinded selection process every 14 days and patients who are not selected are re-entered into the pool for the next selection as long as they are still eligible. Up to 100 doses would be made available in 2020. Critics have accused the company of behaving unethically by avoiding hard decisions about who should receive treatment and have expressed concern over the lack of input into the program’s design from patients and neuromuscular experts.

“Frankly it’s impossible to prioritize equitably the need of one dying baby over another” – Douglas Sproule

Novartis told the Pink Sheet that despite the criticism, it has not stopped the program. It said it was “open to making adjustments” but that no decisions had yet been made and details were still under discussion. The comments came amid social media reports that Novartis had halted the program.

The company said it was confident it can find a solution in the near future. “We are working collaboratively, with a sense of urgency, with patient advocates and clinicians to identify ways in which we could strengthen and enhance the managed access program to more fully meet the needs of the SMA community while still upholding the program principles of fairness, clinical needs and global accessibility.”

Challenges

At the Expanded Access Summit, held on 29 January, Avexis’s Douglas Sproule defended the company’s approach to the program and explained some of the rationale behind the lottery style approach. The approach was decided on the basis of guidance from an external ethics organization, said Sproule, who is the Avexis’s vice-president, SMA therapeutic area head.

According to Sproule, the promise to provide 100 doses was a “major commitment from the standpoint of the burden on supply, manufacturing, throughput and personnel.” Nevertheless, in terms of global unmet need, the program was “only a drop in the bucket,” he said. The important question the company faced was therefore how to prioritize patients for treatment.

“Frankly it’s impossible to prioritize equitably the need of one dying baby over another,” said Sproule. “Do you prioritize the children you think would benefit the most? So a newborn who is just picked up because of a prior sibling or through some newborn screening program, versus the child who is critically ill and dying.”

He asked if it should be more important to maximize benefit or access. “Should you prioritize the ability to really maximize, have all the supportive care? Because that then can take away the option for a child who, because of circumstances, doesn’t have access to all that wonderful ancillary care that one would expect in a tertiary medical setting in a Western country.”

Bad Press

Sproule was unsurprised by the criticism, particularly from European countries where he said the program “has been received like a lead brick.” The idea that every single eligible patient would not receive access has been “extremely poorly received,” he said.

However, he warned that criticism and bad press about imperfect access programs could serve to support the idea that companies should not engage at all in such early access schemes.  While criticism was welcome, he said, there must also be active support of “the premise and importance” of early access programs.

Unlike in Europe, the program has been welcomed in parts of the world such as Latin America and Asia. “When you talk to people in Brazil or in Asia, they’re thrilled because their expectation frankly is that we’re just going to be bypassed, and so the notion that there’s an opportunity and access for children affected by this disease in their country, even if it’s imperfect, is celebrated.”