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Gene Therapy Was Hailed as a Revolution – Then Came the Bill

bloomberg.com

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Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#2
What this article misses is that even with a price tag of more than $1M, many of these therapies will save the system money.

They kind of gloss over it when talking about Zolgensma, but the current treatment, Spinraza, costs ~$750K in the first year and $375K every year after. So even by year 2, you've already spent one million and you pay that indefinitely.

So if Zolgensma shows 5 years of efficacy and Zolgensma costs $2M, it's already 10% cheaper than current costs.

Yes of course, paying it in a lump sum is challenging, but these companies are all exploring alternative payment methods; either paying installments ($500K per year for 4 years) or performance based models (you pay me $500K, every year, until the drug stops working).

Gene therapy will certainly require a new payment plan, but it's not something the system can't handle.

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#3
post #2

What this article misses is that even with a price tag of more than $1M, many of these therapies will save the system money . They kind of gloss over it when talking about Zolgensma, but the current treatment, Spinraza, costs ~$750K in the first year and $375K every year after. So even by year 2, you've already spent one million and you pay that indefinitely . So if Zolgensma shows 5 years of efficacy and Zolgensma c…

At that point it's probably just cheaper to get a plane ticket to a country that does not patent or enforce a patent for the treatment.

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#4
post #3
post #2

What this article misses is that even with a price tag of more than $1M, many of these therapies will save the system money . They kind of gloss over it when talking about Zolgensma, but the current treatment, Spinraza, costs ~$750K in the first year and $375K every year after. So even by year 2, you've already spent one million and you pay that indefinitely . So if Zolgensma shows 5 years of efficacy and Zolgensma c…

At that point it's probably just cheaper to get a plane ticket to a country that does not patent or enforce a patent for the treatment.

Unlike small molecules, gene therapy isn't something that somebody can just whip up in a lab. Similar to biologics, there are often some trade secrets involved in the manufacturing that require a lot of time and money to figure out.

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#5
I think advancements in DNA sequencing (ie nano pore) are going to change the landscape. Once it costs $50 to do a proper sequencing (not the messy bullshit 23andMe does), along with the analytical tools needed to isolate problematic sequences, patients and caregivers are going to be clamoring for targeted treatments. Big pharma will see the money and deliver.

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#6

I think advancements in DNA sequencing (ie nano pore) are going to change the landscape. Once it costs $50 to do a proper sequencing (not the messy bullshit 23andMe does), along with the analytical tools needed to isolate problematic sequences, patients and caregivers are going to be clamoring for targeted treatments. Big pharma will see the money and deliver.

I totally disagree. I don't think the cost of sequencing is the bottleneck here, and it doesn't need to come down to $50. Even at $1k for a full genome sequence (which I think is in the range of a good high-quality sequence these days), it's totally feasible for anyone with a disease to get themselves sequenced.

The hard part is interpreting which part of the sequence is causing the problem, and developing targeted therapies to treat it. Sequencing any patient's genome has already been totally feasible for a few years now, but we still don't understand how to make the information actionable once we have it.

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#7

I think advancements in DNA sequencing (ie nano pore) are going to change the landscape. Once it costs $50 to do a proper sequencing (not the messy bullshit 23andMe does), along with the analytical tools needed to isolate problematic sequences, patients and caregivers are going to be clamoring for targeted treatments. Big pharma will see the money and deliver.

Reliable whole genome sequencing runs about $1000 these days. Reducing it to $50 would shave $950 off the cost of identifying genomic disorders.

It wouldn't affect much the cost of producing therapies that are tailored to individual genomes.

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#8
post #4
post #3

Earlier quoted context omitted.

At that point it's probably just cheaper to get a plane ticket to a country that does not patent or enforce a patent for the treatment.

Unlike small molecules, gene therapy isn't something that somebody can just whip up in a lab. Similar to biologics, there are often some trade secrets involved in the manufacturing that require a lot of time and money to figure out.

How can anyone think this is ethical for lifesaving medical care?

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#9
post #2

What this article misses is that even with a price tag of more than $1M, many of these therapies will save the system money . They kind of gloss over it when talking about Zolgensma, but the current treatment, Spinraza, costs ~$750K in the first year and $375K every year after. So even by year 2, you've already spent one million and you pay that indefinitely . So if Zolgensma shows 5 years of efficacy and Zolgensma c…

I don't think if the question is whether large bills can be handled by distributing $1M payments over N years. The question is, should this really be costing $1M? I understand if there is a billion dollar investment by big pharma that benefits 1000 people and so there is no other way but to charge $1M. However, it appears that fundamental techniques are all same and it gets reused for hundreds or thousands different treatments. So with cost spread, the cost to patient shouldn't be $1M. If big pharma has life saving treatment and they thought they got people by their balls to pay up any ransom amount then its just wrong.
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