[Ip-health] FDA enforcement discretion to allow unregistered generic Daraprim to be imported and sold at lower prices

Biotech. Info. Inst. biotech at biopharma.com
Fri Oct 9 09:42:35 PDT 2015

There would surely be significant legal problems (and political problems too) using this approach.  

FDA allowing parallel importation of presumed functionally generic equivalents of a U.S.-approved product experiencing a shortage, such as with Fabrazyme, with laws/regulations in place for products in shortage, is much different than allowing parallel importation of products because the prices for the U.S. products are too high.  One would presume, if high prices were sufficient for FDA to allow foreign imports, this would already be common.  Also, it’s not as if with daraprime, as with Fabrazyme, there was NIH/federal R&D support and even patents (as I recall), that might provide some rationale for the federal government imposition of “reasonable pricing."

Also, the nature of the market for these products is different, and this may impact such proposals, particularly their political acceptabilty.  Fabrazyme is very much an orphan product, with a very small number of users receiving life-long repeated therapy, already paying high prices, with no alternatives and with this product (or equivalent) needed by many Fabry patients to stay alive or avoid extensive hospitalization.   With daraprime, the product is presumably used by patients for only 1 course of treatment, not for years or the rest of their lives (is my presumption correct?); and with alternatives available, patients not receiving daraprime are unlikely to die or require extensive hospitalization.  

But this is a good way to keep the issue churning, to try to draw FDA and NIH into the current controversies, for activists to score points with consumers, etc.

Perhaps, there could be daraprime [and also hepatitis C drugs] “buyers clubs” distributing parallel imported daraprime, much as there were buyers clubs for HIV/AIDS therapeutics.  It would be easy for FDA to do nothing (simply ignore enforcement actions against the clubs), much easier than having to do something proactive, such as explicitly allow foreign daraprime imports.

Thank you.

Ronald A. Rader
Biotechnology Information Institute
1700 Rockville Pike, Suite 400
Rockville, MD 20852
Phone:  301-424-0255
E-mail:  biotech at biopharma.com

On Oct 9, 2015, at 8:39 AM, Jamie Love <james.love at keionline.org> wrote:

> http://www.keionline.org/node/2332
> ​In 2011, the FDA told the NIH it could use enforcement discretion to
> permit importation and sale of unregistered drugs
> 8. October 2015
> In 2011, in the context of a shortage of Fabrazyme, the FDA told the NIH if
> they could "in theory" allow Shire to import and sell Replagal, a drug
> approved for sale in Europe, but not in the United States, as an
> unregistered biologic substitute. The remedy explored for the Fabrazyme
> shortage may be the best way to address a different problem, the price
> gouging associated with the drug Daraprim (generic name: pyrimethamine).
> Fabrazyme is the Genzyme/Sanofi brand for an enzyme replacement therapy to
> treat Fabry disease, an inherited disorder caused by deficiency of the
> alpha-galactosidase A enzyme. Ultimately, Sanofi, Shire and the Icahn
> School of Medicine at Mount Sinai entered into a series of agreements that
> left the United States with only one supplier for an enzyme replacement
> therapy to treat Fabry disease (http://keionline.org/node/2055).
> Daraprime has been on the market in the United States since 1953, and there
> are no patents for the drug listed in the FDA Orange Book. Outside the
> United States, there are plenty of generic versions of this drug with
> prices as low as 2 cents per pill. Turing Pharmaceuticals has increased the
> price in the United States to $750 per pill, an aggressive action that has
> attracted considerable public interest and outrage.
> The notion that the FDA could use its enforcement discretion to allow the
> importation and sale of an unregistered drug is a possible and appealing
> way for the Obama Administration to address Turing's aggressive price
> gouging. The generic alternatives sold outside of the United States include
> products that have received marketing approval from high standard
> regulatory agencies, so there is no reason to think they present a health
> risk.
> Below is the August 5, 2011 email sent by Kathy Hudson, the Deputy Director
> for Science, Outreach, and Policy at the National Institutes of Health
> (NIH), to Francis Collins, the Director of the NIH. (Note also that Hudson
> recommended the NIH Director grant the Fabrazyme March-In request).
> -------------------
> From: Hudson, Kathy (NIH/OD) [E]
> Sent: Friday, August 05, 2011 2:31 PM
> To: Collins, Francis (NIH/OD) [E]
> Cc: Tabak, Lawrence (NIH/OD) [E]; McGarey, Barbara (NIH/OD) [E]; Devaney,
> Stephanie (NIH/OD) [E];
> Hudson, Kathy (NIH/OD) [E]
> Subject: Fabry's - it is time to act
> Hi,
> Last week we spoke to FDA who said that “in theory” they could use
> enforcement discretion to permit Shire to market replagal in the US.
> Yesterday Genzyme reported a new problem with fabryzyme supply that some
> are reporting as NO enzyme for month of august (gulp…)
> http://www.pharmalot.com/2011/08/genzymepatients-angry-over-fabrazyme-supplies/
> and pasted below
> I talked to Shire today about whether, in a hypothetical scenario, they
> would be able to supply replagal to cover drug shortage in the US. I was
> told that they would be able to provide HRT to hundreds of patients
> immediately growing to full supply to end shortage in 4 months though she
> was doing back of envelope and would need to get better data if in fact we
> had a magic wand to wave.
> I would like to discuss this with you but I would propose that we:
> 1. Ask FDA to give replagal temporary okay to sell in US. We might ask KGS
> to back us….
> 2. Set up call with mt sinai, genzyme, and sanofi and say that we are
> prepared to march in unless they give Shire a license for US and Europe at
> reasonable costs and that the deal be done immediately.
> Patients will only be helped in the short term if (1) happens.
> Patients will only be helped in the long term if both 1 and 2 happen.
> I think we should act right away… This is heart breaking.
> Attachment
> FOIA_42737_Packet 5_Redacted-pages32-35.pdf
> -- 
> James Love.  Knowledge Ecology International
> http://www.keionline.org/donate.html
> KEI DC tel: +1.202.332.2670, US Mobile: +1.202.361.3040, Geneva Mobile:
> +41.76.413.6584, twitter.com/jamie_love
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