Paul's Christmas Epistles

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  • 12/21/2011

Once upon a time, Paul Kim worked for both Congressman Henry Waxman (on the Energy & Commerce Committee) and Senator Ted Kenendy – giving him the honor of negotiating with FDA on the original PDUFA package and then, subsequently on the reauthorizations both on the House and Senate side.

As a panelist at CMPI’s PDUFA without the Politics conference, he had a lot to say.  (A video of his full comments can be found here.) Specifically, when it came to ornaments to hang on the PDUFA Christmas Tree he had two – both aimed at enhancing and incentivizing innovation.

There are bills that even in a very partisan environment find a lot of bipartisan support and interest in a bicameral way. And there were two I just felt I had to call out. They go to the innovation gap -- an issue and a set of concerns that are so much more resonate now in this economy and the role that the agency plays in promoting innovation. There are two proposals. The first is called the Gain Act. It’s sponsored by Congressman Gingrey - Dr. Gingrey, here in the house – and Senator Blumenthal in the Senate. Wonderful bipartisanship, bicameral support there. It would enhance incentives in terms of exclusivity for sponsors that are trying to generate and create and innovate on microbials. And given the enormous challenge that microbial resistance presents to public health, this is an area where it seems like there’s a classic market failure. There aren’t enough players in the field. The marketing incentives are not strong enough to draw interest, to draw investment. So, that’s a proposal that already has bipartisan support in both chambers. I think it’s the subject of a lot of discussions in the committees of jurisdiction. So, do look out for the GAIN act as a possible ornament somewhere in the upper branches of the PDUFA Christmas tree.

The second proposal is one that has also received bipartisan support, sponsorship and interest on both sides, Senate and House. And that’s to reform the humanitarian device exemption or HDE process.

Little known, it’s kind of the lesser known sibling to the Orphan Drug Act, which has promoted and really generated tremendous innovation for diseases, rare diseases and disorders affecting 30 some million Americans. When it was originally enacted in 1990, there was a profit cap. It said if you develop a device for a very, very small population, you can only charge for cost. It was intended as a safeguard, but instead what’s happened is it’s blocked and chilled interest in using that pathway to market.  The consequence is that we’ve had some 54 HDEs or Humanitarian Devices approved since 1990. In contrast, literally hundreds of orphan drugs have been approved since the enactment of that act. Again, it’s an issue that has bipartisan interest and support, a very small change to existing laws. It’s not overturning the apple cart, but I think you’ll see some significant changes in the marketplace among sponsors, investors, investigators, and scientists as a result of any federal change like that.


Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

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