

Europe approves high-price gene therapy - tokenadult
http://www.reuters.com/article/2012/11/02/us-genetherapy-europe-idUSBRE8A10K520121102

======
alexholehouse
I'm torn here. On the one hand, this is a great development, and could
represent an effective way to treat a whole range of diseases.

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.

~~~
paulsutter
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.

------
shrivats
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...](http://www.nature.com/mt/journal/v20/n10/full/mt2012194a.html)

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."

------
coob
This is fantastic news. As with all thing in life, the price will come down.

------
nachteilig
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.

------
trustfundbaby
Could something like this help someone with Down's syndrome?

~~~
EwanToo
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>

------
bobwaycott
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.

~~~
refurb
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)?

