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For many, many years we've been hearing about gene therapy – the chance that we can get into people's DNA and fix it to resolve problems and fix disease. In a recent piece in Science, Stuart Orkin and Philip Reilly discuss what finally achieving success might mean. Let's talk about that. This is Healthcare Triage News.

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For many, many years, we've been hearing about gene therapy, the chance that we can get into peoples' DNA to fix and resolve problems and diseases.  In a recent piece in Science, (?~0:10) discuss what finally achieving success might mean economically.  Let's talk about that.  This is Healthcare Triage News.


As the authors report, gene therapy has been 44 years in the making, but gene therapy, which has cost billions of dollars in research and development, is different from traditional pharmaceutical markets.  For one thing, most diseases which are the focus of gene therapy research are relatively rare.  Most of the conditions affect children.  In addition, gene therapy is more like a procedure than a drug.  You perform it once, and potentially achieve a lifetime cure.  There's no way that, like many pharmaceutical products, profits can be made on volume.  

It's important to note that at this time, we still don't have a lot of promising results in human subjects, but we're getting close, close enough that it's best we consider how we might pay for it now rather than wait until it's here and we all start fighting about it.  We may expect the prices for gene therapy might approach previously unseen amounts.  The only gene therapy currently approved for use in Europe is (?~1:10).  It treats lipoprotein lipase deficiency, a rare illness.  It's now priced at more than $1 million per patient, even though, as the authors point out, its efficacy is not without doubt.  

This may seem ridiculously high, but it's not.  Organ transplants can run that high and they sometimes offer less of a cure than gene therapy might.  Even bone marrow transplant can cost more than $500,000 and we do that all the time, but when it comes to therapies that aren't procedures, we often balk.

The fights over (?~1:40), which was still arguably cost effective compared to other treatments, might give us an inkling of what's to come.  The authors present some ideas on where to start.  First they talk about estimates and the current cost of managing genetic disorders.  Cystic fibrosis costs almost $6 million per patient over a lifetime.  Gaucher disease about $5 million.  Sickle cell disease about $1 million, and hemophilia A can cost between $5 and $10 million.  

They also suggest we consider the modality of the specific gene therapy.  If it's as intensive as bone marrow transplant, it may be priced similarly.  Development cost must be considered, which are likely similar regardless of the prevalence of the disease being treated.  The prevalence is its own factor, however.  The price would likely have to be higher when the disease is rarer.  Further considerations involve how much it costs to produce the therapy and of course, what outcome is expected.  A full definitive cure may be worth quite a bit.

Different countries will make these determinations in varying ways.  The United States, more than any other, lets the market decide what it will pay.  That doesn't always turn out well.  The authors suggest the following instead, "First, very expensive gene therapies with large, up-front payments should require that the burden of re-treatment be borne by the drug-maker or its successors.  Second, some reasonable portion of the economic benefits that under the Orphan Drug Act flow to companies that develop therapies for "orphan" disorders might be redirected to reducing the price of the drug, perhaps by greatly reducing the pricing of copays.  Third, the US National Academy of Medicine (or a similar body) should commission a study to explore new methods to streamline the regulatory process for developing genetic and perhaps other therapies for ultra-rare disorders."

These aren't definitive solutions, but they're a good place to start.  It's better that we have this discussion before decisions have to be made rather than after.  Go read the article.  It's a good one.  

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