Time for an update to this thread.
Anaxagoras wrote: ↑
Sun Dec 31, 2017 8:08 am
I think we're about to enter a new era.
So I just came across this article:
When a lottery 'wins' sick babies life-saving drugs
Eva was diagnosed with spinal muscular atrophy, a motor neuron disease, just a few weeks after her birth in August. Her parents plan to enter her in a controversial "drug lottery" in the hopes of getting access for a promising new treatment that costs $2.1m (£1.6m) per patient.
The focus of the article is on how expensive it is (over $2 million/patient), and the fact that there's a lottery for people who don't have $2 million to pay for the drug but need it. But I believe the cost will eventually come down. This is a brand new kind of gene therapy. It took a lot of money of course to develop it and to test it, and it's for a pretty rare genetic condition. So I don't look at it as "greedy corporation preys on desperate people" because the amazing thing though is that this kind of therapy exists at all, for any price. And they are in fact, giving it away for below cost to those who cannot afford it.
The Batistas' priority was getting the best care for their newly diagnosed child.
In Canada, where the couple live, they have access to Spinraza, a prescription drug taken during the patient's entire lifetime that can increase survival and motor function. It's been on the market since 2016 and is the first approved therapy to treat all types of SMA.
There is also a brand new drug - Zolgensma. It's produced by Swiss pharmaceutical giant Novartis and was recently approved by the US Food and Drug Administration (FDA) for children under the age of two.
It's a one-time prescription gene therapy touted as a potentially life-changing treatment. It's also currently the world's priciest single-dose treatment, at $2.1m per patient.
Gene therapy, at the cutting edge of health science, involves introducing genetic material into cells to compensate for abnormal genes - in the case of SMA, the faulty SMN1 gene.
In consultations with Eva's doctors, the Batista's have begun treatment with Spinraza - but they see gene therapy as their daughter's best hope.
There is third option for the Batistas - one that depends on the luck of the draw.
When demand from families for Zolgensma spiked following its approval in the US last May, Novartis subsidiary AveXis announced a programme to provide the therapy free to some eligible SMA patients.
Drug becomes 'coveted prize'
It would give out up to 100 doses to patients who meet clinical criteria using a "blinded selection" - a lottery - every two weeks through 2020, starting on 3 February.
The firm said in December that the managed access programme was "anchored in principles of fairness, clinical need and global accessibility...that doesn't favour one child or country over another".
Novartis says the programme was developed with input from bioethicists - but it has been criticised by patient groups around the world.
Kacper Rucinski, co-founder of the UK's TreatSMA and a board Member with SMA Europe, say they will be asking the pharmaceutical firm to remove "fully or partially" the lottery element of the programme.
Is a lottery an unfair way to distribute a limited resource? Anyway, let's hope the day arrives soon when every patient who needs this therapy can have it.
Here's an article about the drug itself:
And another one about gene therapy, a very new field.
A fool thinks himself to be wise, but a wise man knows himself to be a fool.