Europe approves high-price gene therapy(reuters.com)
reuters.com
Europe approves high-price gene therapy
http://www.reuters.com/article/2012/11/02/us-genetherapy-europe-idUSBRE8A10K520121102
6 comments
Whatever happens - we'll learn an enormous amount about gene therapies. The uncertainties you mention increase the importance of this therapy. There's only one way we'll really find the answers.
A little bit of background in the form of an Editorial from Molecular Therapy, a well-regarded gene therapy journal (pay-walled, I assume):
http://www.nature.com/mt/journal/v20/n10/full/mt2012194a.htm...
An interesting paragraph from the editorial, indicating that the regulatory process for this sort of gene therapy is still quite difficult:
"This lengthy and tortuous approval process raises several questions. Frequent reapplications are very time-consuming and expensive, which in this case led to the demise of Amsterdam Molecular Therapeutics, although thankfully private investors were able to continue the process. Nevertheless, investors will shy away from supporting biotech companies unless greater clarity and predictability can be achieved in the process of regulatory evaluation and approval. The Glybera saga also highlights problems specific to ultra-orphan drugs—because obtaining large-scale phase III data with a very limited number of patients is virtually impossible, procedures to handle these indications must be further developed."
http://www.nature.com/mt/journal/v20/n10/full/mt2012194a.htm...
An interesting paragraph from the editorial, indicating that the regulatory process for this sort of gene therapy is still quite difficult:
"This lengthy and tortuous approval process raises several questions. Frequent reapplications are very time-consuming and expensive, which in this case led to the demise of Amsterdam Molecular Therapeutics, although thankfully private investors were able to continue the process. Nevertheless, investors will shy away from supporting biotech companies unless greater clarity and predictability can be achieved in the process of regulatory evaluation and approval. The Glybera saga also highlights problems specific to ultra-orphan drugs—because obtaining large-scale phase III data with a very limited number of patients is virtually impossible, procedures to handle these indications must be further developed."
This is fantastic news. As with all thing in life, the price will come down.
It's great that they're using Adenovirus for this. One of my favorite. I'm really excited to see viral therapies starting to nudge toward the mainstream.
Could something like this help someone with Down's syndrome?
I think that's in the (very) early stages of research, but who knows what might come of it.
http://www.ncbi.nlm.nih.gov/pubmed/22541296
http://www.ncbi.nlm.nih.gov/pubmed/22541296
While I'm excited by the possibilities and future improvements of this sort of research and therapy, the price strikes me as egregiously exorbitant. I don't reject the notion that they should be duly compensated, but 250K/yr for 5 yrs is just insane. There is so much wrong with that.
Genetic improvement shouldn't be built from the ground up as the territory of the extremely wealthy alone.
Genetic improvement shouldn't be built from the ground up as the territory of the extremely wealthy alone.
It's price based on what the market will bear.
A number of factors determine that:(1) the therapy helps patients a great deal (no other way to treat and the disease is fatal) (2) the patient population is small, so even at $250k per year, the total cost is low since so few patients exist
Keep in mind that development costs are still in the 100s of millions of dollars. If the total world population of these patients is only a few hundred, how much do you need to charge to cover your developments costs (even if you assume $0 profit)?
A number of factors determine that:(1) the therapy helps patients a great deal (no other way to treat and the disease is fatal) (2) the patient population is small, so even at $250k per year, the total cost is low since so few patients exist
Keep in mind that development costs are still in the 100s of millions of dollars. If the total world population of these patients is only a few hundred, how much do you need to charge to cover your developments costs (even if you assume $0 profit)?
In the beginning, using a plane was for the rich (jet set), using a mobile phone was for the rich, using the internet was expensive and for specialists, using a car was available only for few people as was using electric light and having a warm, comfortable home. Now it's the same with space tourism and gene therapy. So don't worry, just wait (or help making it cheaper).
On the other, and I speak from experience, we really don't fully understand what goes on in terms of gene expression regulation. We have ideas which generally work in vitro, mostly in vivo with model organisms (drosophila, yeast, etc) but relatively little basic research looking at fundamental mechanisms of regulation is done in higher eukaryotes. The upshot is that we may have ideas of what's happening, but those ideas may be one of several possible explanations which lead to similar outcomes. Moreoever, the impact of gene therapy in terms of a long term cellular (and immune) response is poorly understood.
I'd be intrigued to see what longitudinal data have been generated to show Glybera's safety over the kinds of timeframe relevant here.