Launching Zolgensma® in Belgium: Lessons Learned

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In late 2019 Novartis hit the headlines in Belgium after it refused to grant access to its one-off gene therapy Zolgensma® for the ultra-rare muscle-wasting disease spinal muscular atrophy (SMA) to Pia Boehnke, a Belgian child.

 

With Zolgensma® at that point having not yet been authorised by the European Medicines Agency (EMA), Baby Pia’s family raised EUR 1.9 million via a crowd-funding campaign to pay for the cost of the treatment, often cited as the world’s most expensive. This was done in the hope that Novartis would make the drug available under the EMA compassionate use program (CUP), which allows national governments to request drug makers to offer their treatments to a suffering patient prior to market approval. After a media backlash, Novartis relented and Baby Pia was able to receive treatment with Zolgensma® in Belgium.

 

Around 550-600 babies are born with SMA in Europe each year, which causes rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement. With the most severe form of SMA, patients may not live past their second birthday.

 

Zolgensma®, which Novartis acquired via the USD 8.9B acquisition of AveXis in 2018, transfects additional copies of the SMN1 gene into motor neuron cells using an adenovirus vector. In two remarkable single arm clinical trials, infants who had already developed symptomatic type 1 SMA and then received Zolgensma® not only survived well past their predicted date of death, but experienced improvement in neuromuscular function. At around EUR two million, the cost of the treatment has proved controversial, but Novartis claim that its pricing rivals the existing treatment for SMA over a five-year period.

 

PharmaBoardroom recently spoke to Philippe de Pougnadoresse, country president and general manager pharma of Novartis Belgium & Luxembourg, who reflected on the journey of Zolgensma® – which was finally approved in the EU in May 2020 – and the lessons that can be learned in terms of access and pricing solutions for advanced cell and gene therapies.

 

He explained, “We understand that reactions in Belgium regarding access to Zolgensma® pre-reimbursement have been strong and emotional, which is normal as it concerns the lives of children and the impact it has on their families. I am father to an 18-month old child, so I completely empathise with the distress it can generate for parents. The longer a child with SMA waits for treatment, the more damage will be caused. Finding an early access solution is, therefore, our number one priority.”

 

The development of our managed access program (MAP) for Zolgensma® has been an excruciating ethical dilemma. We had significant production capacity limitations, yet our goal was to help patients across all markets, including where there is no access

Philippe de Pougnadoresse, Novartis Belux

 

“Reflecting on this one year on, the development of our managed access program (MAP) for Zolgensma® has been an excruciating ethical dilemma. We had significant production capacity limitations, yet our goal was to help patients across all markets, including where there is no access. We wanted to give an equal chance to any child in the world to have access to our revolutionary treatment. We did not want to be a company that dictates which children do or do not get treated. We sought guidance from an external panel of independent international bioethical experts to design this “first of its class” Zolgensma® managed access program (MAP) because we did not know how to do that; there were no reference points or precedents.”

 

“They advised that the medical criteria alone was not enough as there were still too many potential children in the pool of patients, so the best thing we came up with based on their guidance was to apply the same randomisation methodology that they use in clinical trials. We realise that this solution may not be perfect; we have always kept an open mind and invited our stakeholders to a constructive dialogue.”

 

de Pougnadoresse continued, “With Zolgensma® we are entering unchartered territories. This therapy has generated profound societal questions for which we did not have all the answers immediately. It has been a time of trial and error and we have learned a lot, at times, the hard way. That is the price to pay for being a pioneer, but again we have constantly kept an open mind and challenged ourselves in the first place. We have been able, from Belgium, to elevate this topic and talk about it with our global CEO, who was genuinely open to the challenge and helped us try to find better solutions.”

 

Looking to the future, de Pougnadoresse emphasises the need for greater stakeholder engagement at a much earlier stage. “Moving forward, I trust that together with stakeholders we can find sustainable access solutions. This will demand more work as these revolutionary one-time treatments do not fit in the current boxes,” he stated.

 

“It has been disruptive for all of us. If I could do it again, to avoid this tsunami of emotions that did a lot of damage, I would engage with the government, patient associations, healthcare providers, hospitals, and the media much earlier to explain and start co-creating solutions as a company and as an industry.”

 

de Pougnadoresse concluded, “That would have hopefully spared a significant emotional impact on the impacted families, like the one from baby Pia, in the first place, in the general public and finally, on our employees who were appalled by how Novartis was portrayed in the media. There was such a gap between what we live every day together as a community of co-workers, serving a better life for patients, and what was being said in the media.”

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