How much to cure the kid?

7 July 2021

It’s now been seven months since the PBAC sat down for what was a revolutionary moment in Australian health technology assessment – its first consideration of a gene therapy.

The moment passed without fanfare and has since been virtually overlooked by everyone other than the company trying to get this therapy to patients, and the families of the babies that need it.

It is indeed a sad reflection on Australia’s reimbursement system that, despite meeting three times since November when Novartis’ gene therapy ZOLGENSMA (onasemnogene abeparvovec) was first considered and a fourth time this week, the PBAC has still not come to a decision even though the therapy offers to ‘cure’ babies born with Spinal Muscular Atrophy (SMA) – a debilitating condition that sees their motor neurones progressively die, leaving them condemned to a wheelchair.

The Public Summary Document for the meeting shows Novartis has already drastically cut its price to try and get an outcome. The PBAC has now moved the goalposts; it wants Novartis to show how its gene therapy compares with Roche’s EVRYSDI (risdiplam) despite Zolgensma being considered before Evrysdi was either registered in Australia or submitted for PBAC consideration.

The new requirement would be laughable if it wasn’t so utterly tragic and desperately short-sighted. How do you compare the benefits of a single, life-changing infusion with ongoing lifetime treatment? Novartis must have been horrified, to say the least. For parents of babies with SMA, it is a living nightmare from which they will never awaken.

The PBAC had no qualms in recommending Evrysdi when it first considered the oral medication in March. No doubt, the drug will be added to the PBS on 1 August with great fanfare from Health Minister Greg Hunt, extolling its benefits while ignoring the fact the babies who will take the medicine for the rest of their lives could have been cured, had he given them the chance.

Try this quiz Minister. If it was your child born with SMA, would you prefer: a) they have a lumbar puncture every four months for the rest of their life; b) take a strawberry-flavoured medication for the rest of their life; or c) have a one-off infusion that means you and they never think about it again. Not a difficult choice, is it?

Both Biogen’s SPINRAZA (nusinersen) and Roche’s Evrysdi are a great option for SMA patients who previously had nothing. Evrysdi, recommended by the PBAC cost-minimised to Spinraza, offers the convenience of an oral medication over a needle in the spine, but in outcomes, the two are roughly comparable. When it comes to Zolgensma, the gene therapy is not just in a different league, it’s from a different planet.

The UK shows how it’s done

The Parliamentary Inquiry into Approval Processes for New Drugs and Novel Medical Technologies (aka the Zimmerman Inquiry) will tonight (Wednesday) hold one last hearing. After more than 50 hours of evidence, the committee will finish with the UK’s National Institute for Health And Care Excellence (NICE) deputy CEO and director of the Centre for Health Technology Evaluation Meindert Boysen.

Once lagging in HTA when compared with other countries, the UK has leapfrogged forward, becoming the virtual pin-up country for others eager to reform their ageing HTA systems. The numerous references to the UK in other evidence given before the inquiry is why Boysen will be the only international witness called to give evidence.

If the parliamentary committee has done its homework, it will unpack NICE’s most recent and exciting initiative; new guideline recommendations unveiled in April that have the specific aim of accelerating patient access to new treatments and innovations such as cell and gene therapies.

The five-year strategy allows the HTA body to engage with industry earlier in the innovation pathway to enable faster access, saying Covid taught it “that we can be flexible and fleet of foot without losing the rigour of our work”.

Calculating the cost of delay

On average, 30 babies are born in Australia each year with SMA. Zolgensma was registered in Australia in March, meaning 10 babies born since then must now face a lifetime of treatment as they were not given the same option as babies in many other developed countries with access to Zolgensma.

Instead, the Government will pay for these children to access Spinraza or potentially Evrysdi for the rest of their lives, along with the associated hospital costs. The published cost of these therapies is just under $400,000 a year.

The PBAC noted in its deferral of Zolgensma that Novartis needed to take into account the annual five per cent discount applied to Spinraza in costing its own one-off treatment. A rough back-of-the-envelope calculation finds that if the lifetime therapies did cost $400,000 in Year 1 and were discounted by five per cent annually, over 20 years the cost is more than $5 million per patient*.

The published price of Zolgensma is US$2 million, or $2.6 million* in Australian money, and Novartis says it has cut this price drastically to get a decision, so the actual price is likely to be closer to $1.5 million. If the child lives a normal life and dies aged 80, the cost works out at $18,750 a year.

The future is here. Gene therapies are here. Cell therapies are here. Yet Australia is trying to pretend they are just like every other therapy as our HTA system tries to push and shove them through the same tiny hole as it has managed to squeeze every other drug that came before.

The time for procrastination and discussion is over. The time for action has come. As it waits for the outcome of the Zimmerman Inquiry and the start of the National Medicines Policy Review, Australia needs to ask itself, how much a life is worth.

Our country just paid a very high price in Covid to protect our elderly and vulnerable. Now we need to ask, how much to cure the kid?

*The published price is not the actual price, with local pricing agreements likely to cut this by around 40 per cent.

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