Standing Up to the Paralysis of Care for the Paralytic Diseases of the Digestive Tract

Gastroparesis is a disease that doesn't get much attention--except of course, from those who suffer from it, and from their family and friends. Which is to say, it is one of a thousand diseases that would benefit from a Serious Medicine Strategy, in which the political class took medical security as it did economic security or national security.

This is part two part two of a guest piece from Michael Smith, a lawyer in New York City and a board member of G-PACT, the Gastroparesis Patient Association for Cures and Treatment. Part one of Mike's piece, published on September 23, is here.

The issues Mike raises here are long and complicated, and yet the issues are vital, not only for gastroparesis, but for serious illness overall. Those who suffer from every other kind of serious illness, and who take the time to study the issues in detail, will quickly see common elements in their struggle, too. We all have an interest in a Serious Medicine Strategy.

Standing Up to the Paralysis of Care for the Paralytic Diseases of the Digestive Tract

by Michael Smith

In Part I, we discussed the key issue for patients suffering from paralysis of the digestive tract; namely, not the inability of medical science to offer treatments, but the inability of the bureaucracy responsible for approving and maintaining the effectiveness of treatment to use and decipher the best information available to make appropriate choices. The following is a series of solutions, both short-term and long-term meant to reform the current drug approval bureaucracy in a manner that both responds to what should be the central goal of this bureaucracy, namely getting patients better with the fewest side effects possible, while also properly respecting the scientific process that drugs must and should go through to get approved in the United States.

1) Appropriate and Speedy Appeal of Drug Withdrawal: When Zelnorm was initially removed from the US market on March 30, 2007, the FDA promised in a press release the following:

“FDA has also indicated to Novartis the possibility of considering limited re-introduction of Zelnorm at a later date if a population of patients can be identified in which the benefits of the drug outweigh the risks. Any such proposal would be the subject of a public advisory committee meeting before an FDA decision.” (Source: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/2007/ucm108879.htm)

Unfortunately, the promise of the public advisory committee has never been fulfilled by the FDA; what instead has been gained is a nebulous and ever-changing emergency directive process which lacks the transparency that Jim Pinkerton has alluded to in other pieces.

The one foundation that FDA must be built on prior to any other is a constitutional foundation. When FDA pulls an approved drug from the US market, it is not just pulling a simple economic good from the US market; it is a pulling both an economic right of patients, in essence their license to be able to take a prescription drug which has been granted through both the doctor-patient relationship, when a doctor determines that a drug is best suited for a patient’s well-being, as well as in some case a right to life and liberty by the denial of right to medicine that in some cases will simply allow patients to maintain quantity of life and in others will allow patients to maintain quantity of life.

A solution to this problem should be that when FDA pulls any drug from the US market that a right to a public advisory committee meeting or hearing should automatically be granted upon a drug’s withdrawal from the US market. The taking of a property right must from a procedural due process standpoint be granted when government revokes a right that cannot be easily substituted. To those who would argue that FDA has a larger public health and safety consideration to deal with in making such decisions, I would argue that the right to a hearing is different from the right to the medication itself; the hearing represents an open forum is which all interested stakeholders should have the right to properly present valid scientific information which may not have been available to the FDA at the time of its original decision. To those who would argue that patients are either overly emotional or overly vested in a single drug, I would contend that most patients managing serious chronic illnesses have been managing them for several years, have had to work out difficult treatment plans themselves which require the recognition of medical knowledge that perhaps only doctors themselves or forced to convey and that we harbor no particular loyalty to any single drug company whose support for a given condition may last only as long as a given drug remains on patent.

2) Standard of Review for FDA’s Decision Making Process: In situations where it appears such a hearing process as I allude to here has either been denied or improperly administered, we must give careful look at the credence given to FDA’s review and drug approval or rejection process and standard under which such decisions are reviewed in a courtroom or by Congress. Generally, FDA is perhaps given the fullest credence of any administrative agency in the land when decisions are provided with regard to the approval or rejection of drugs. As seen in the recent Abigail Alliance case, or in other FDA-related drug litigation or alternatively in the setting of legislatively-induced FDA reform, courts or Congress contend since most judges and legislators are not scientists or doctors, they are not in the position to review the merits of a decision made by FDA under any circumstances. In the world of corporate law, we call this situation, a “futile demand” meaning that there is really no one we can properly appeal to with regard to merits of a decision since no arbiter feels that they have the standing to listen. I would contend that there must be a standard below that of absolute discretion granted to the FDA. Such a standard should ensure that no decisions made by the FDA stand on the ground of being “arbitrary and capricious”; in other words, these should not be decisions which are solely based upon past FDA practice, lacking in scientific validity or lacking insight after careful examination of the fullest information base available. Patients, doctors and other stakeholders can have faith in a system that even when it makes a decision that goes against their interests is a system that works in a manner where transparency is not simply a watchword and that such stakeholders are viewed not as hysterics with no standing, but as human beings with a right to stand on their two feet.

3) A More Compassionate Clearance Process: Currently, all compassionate use indications for medications under clinical trial or clearance to proceed with individual directives are cleared through the FDA. Many patients caught in this process will tell you that it takes months or years until applications for such use are cleared by the FDA. This often is insufficient for patients whose need for medication is based upon imminently emergent medical conditions where the need to treat is predicated upon the speed in which treatment can be administered. Those who claim understanding of FDA’s difficulty in moving through with the process have often contended that FDA is burdened with the constraints of a budget equal to that of the Montgomery County school district. Unfortunately, as we have seen in the private sector bailouts of the last two years, there are moments where improvements upon decision making processes can and will prevail over large sums of money deployed to correct given situations. I would contend that potentially ceding FDA authority over such compassionate clearance protocols over to the NIH which controls a series of hospital-based Institutional review boards or to an independent agency fully resourced and geared to making such decisions might be more appropriate. When looking at the structure of FDA, one is struck by the fact that no matter that size of their budget, FDA is tasked with making “en mass” decisions regarding the effect of medications on substantially populations of individuals; the difficulty of reshifting bureaucratic thought and focus for the good of one may not be a fair request of the limited resources and manpower that FDA purports to have.

Even more essential than what I noted above, there needs to be a shift in the focus of the general public. Members of the public, regardless of whether they have been touched by chronic illness or not need to be more properly engaged in this debate. The decisions made by the FDA on a daily basis more greatly affect any decision made by the President on health reform; regardless of how pays for your health insurance, if a treatment is not available for your condition, the payment system is immaterial. Additionally, if the FDA does not hear your voice and Congress does not your voice, the few trees in the forest who are rustling make no difference. Myself and other members of my organization do not feel that FDA harbors bad intent in any way; these are good people who have families and often join up with FDA because their families face similar problems to ours. But a discussion needs to be had where FDA does not assume that they are working in the patient’s best interest, but where the FDA can know that they are working in the patient’s best interest.

And it needs to start now.