Darrow, Avorn, and Kesselheim on the new FDA category of “Breakthrough” Drugs.

This weeks New England Journal of Medicine includes a terrific article by Darrow, Avorn, and Kesselheim: New FDA Breakthrough-Drug Category- Implications for Patients, 370 New Eng. J. of Med. 1252-1258 (March 27, 2014).  (the full text is available free online at http://www.nejm.org/doi/full/10.1056/NEJMhle1311493).  The authors point to the relatively high rate of post-market safety issues as well as non-efficacy issues that have surfaced in recent years with respect to drugs approved on the basis of accelerated approval using surrogate endpoints.  There is an important balance to be maintained between expediting access to life-saving new drugs and the hasty approval of new drugs that pose serious risks that outweigh their benefits.  The authors make a strong case that this balance has been lost, and that we may well be doing more harm than good for some of the most vulnerable members of our society– patients affected with life-threatening diseases for which there are few if any therapeutic options. As the article points out, in the case of accelerated approval with commitments for post-market studies (referred to as Phase IV studies), NDA sponsors have generally been very slow in conducting those studies and gathering data. Continue reading

Tu, Holman, Mossoff et al. Make a Valuable Contribution to the Often Wildly Inaccurate Discussion of Gene Patents.

There has been a great deal of hyperbole and plain old inaccuracy in public discussion of gene patents. One would have hoped that the Supreme Court’s decision in the Myriad Genetics case, Association For Molecular Pathology et al. v. Myriad Genetics, Inc., et al.) (12-398, Decided June 13, 2013) would have put an end to some of the most inaccurate discussions, which wax on about how the human genome is largely covered by patents and science has been stymied. However, the media still repeats those assertions and, remarkably, they even continue to be made in peer-reviewed journals. GENOME MEDICINE recently published another one of those “the end is near” articles and fortunately, just published a well-written and thorough response by a group of law professors who actually know what they are talking about: Tu et al.: Response to ‘pervasive sequence patents cover the entire human genome’. 6 Genome Med 14 (2014). It has been my opinion that whole genome sequencing largely rendered the problem moot, at least for genetic testing, and that other uses of genetic material, for therapeutic purposes in gene therapy or for protein production for biological therapy should continue to be patentable. Truly workable gene therapy methods are undoubtedly patentable because they are still to be invented and clearly non-obvious. However the patentability of the use of genetic material for protein production for biological therapy has been cast into some doubt by the Supreme Court’s regrettable resurrection of the product of nature doctrine in the last two years. I believe that in the context of recombinant protein production this is somewhat less troubling than it might otherwise seem, as there just do not seem to be very many naturally occurring, non-immunoglobin proteins left that can be used therapeutically. Other than in the case of very rare Orphan diseases, there have been no recombinant proteins approved for therapeutic use in the U.S. in a number of years. It appears that the low hanging fruit that was brought to market in the days of Epogen, Neupogen, Humulin, etc., may have been the only fruit worth harvesting at all. From a pharmaceutical policy perspective, the message is clear. Gene patents simply are not an issue of importance.