The Cost of Approving an Old Generic Drug as a New Autism Drug


Some readers of this blog are commenting how hard it is to obtain prescription only drugs for “off-label” use in autism.

None of the drugs mentioned in this blog are actually approved for use in autism.  There is some science showing that they might be effective, but there is no mention of autism on the “label” approved by the regulator.

This means that your doctor will not know how to prescribe it and your insurer will not want to pay for it.

So how do I access these drugs?

This is a frequent question.  In theory you do not need to wait for the drug to be approved, you can apply to the national drug agency in your home country for permission to use a drug based on the experimental use that showed it might be effective.

Better still, in many countries like the USA, doctors are not banned from prescribing “off-label” drugs.  If the doctor follows the new research, he is permitted to apply it on his own patients.  If he does it recklessly, he might eventually lose his license.

In cancer therapy, many drugs are used off-label.


Why not just approve a new use for an old drug?

This would seem an obvious question and this is what is being done with bumetanide, one of drugs described in this blog.

The problem is the cost and the time taken: EUR 4 million  (USD 5 million) and four years.

As you can see below, in the case of Bumetanide, the French Government will contribute EUR 1 million and it appears the Simons Foundation another EUR 1.5 million.


RECHERCHES PARTENARIALES ET INNOVATION BIOMEDICALE (RPIB) 2012 : projet CURE AUTISM
Curing autism with the diuretic bumetanide 


Since Bumetanide is available today as a cheap generic drug, they cannot really ever get their money back.  Only if they modified the molecule slightly, patented it, and got that new drug approved could they recoup their USD 5 million, which would then be even more, since they would have even higher costs.

The last I heard, Bumetanide will only be approved for autism in Europe, not the USA, due to cost issues.

So with this kind of financial logic, you can see why off-label uses of old generic drugs are likely to stay off-label.  

Best find yourself an off-label doctor.







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