In our August 5, 2013 article entitled, Big Pharma Says Jump And The FDA Says How High?, we detailed the potential problems with the then new FDA Safety and Innovation Act (FDASIA). This Act streamlined approval for new "breakthrough therapy drugs" for serious and life threatening conditions. While the purpose of the Act is commendable, the existence of the potential for rushing unproven drugs to market in the name of greed is seemingly undeniable.
Case and point is the FDA's recent approval of Eteplirsen for the treatment of a common form of muscular dystrophy. Eteplirsen was designed to increase dystrophin, which is a muscle protein lacking in many muscular dystrophy patients. In support of the drug's approval, the manufacturer Sarepta Therapeutics reportedly submitted data from clinical trials with a total of only 25 patients. The FDA is stated to have found the trials to have major flaws, that they demonstrated only a minute increase in dystrophin levels and that they did not show any meaningful improvement of physical performance. In addition, FDA scientific experts recommended against approval in part due to a lack of substantial evidence of the drug's efficacy.
Notwithstanding all the foregoing, the Director of the FDA's Center For Drug Evaluation and Research (CDER) approved Eteplirsen. Many say that the approval was a combined result of pressure from Big Pharma, politics and greed in that the drug is estimated to cost $300,000 per year for treatment. Opponents also argue that if unproven drugs are rushed to market, they are selling desperate patients false hope. Further, that it may even accelerate their disease if they abandon more proven treatment methods in favor of the new "miracle drug du jour." Proponents submit that anyone with a loved one suffering from a terrible disease, would want any drug with a chance of cure approved as fast as possible.
Perhaps the problem is not with the FDASIA but with the FDA itself and the way it operates. The FDA is a governmental agency designed to protect the American people, but in order to do so it must be free of all outside influence, conflicts of interest and pressure, political and otherwise. This begs the question as to how can a governmental agency operate on that basis, if the one agency is entrusted to handle both food and drugs in our country? This alone would appear to create an inherent conflict of interest, now left to our next president to either remedy or continue to ignore like the those before him or her.
Long Island Lawyer
Paul A. Lauto, Esq.