Stephen L. DeFelice, M.D.
Chairman, FIM, the Foundation for Innovation in Medicine
The ideal purpose of our national health policy should be to accelerate the discovery of medical breakthroughs that will cure or much better treat diseases which would a) reduce patient suffering and premature death and b) dramatically and quickly reduce health care costs. One way to do this is to enact the Doctornaut Act which deals with medical discovery only. Cost reduction would then follow due to natural market forces.
To appreciate the rationale of this proposal one must understand the system which brings patients new medical therapies. We, unfortunately, are profoundly ignorant of it. Generally speaking, medical discovery involves two processes- the identification of potential therapies discovered in scientific laboratory studies or based on theory and the clinical testing of them in patients. If, for example, something like aspirin is shown to have pain killing-inflammation reduction potential in animals, it can only be proven to work in clinical studies in patients with headache and arthritis among others. The extremely good news is that, there are, and will be, enormous numbers of potential medical breakthroughs. The very bad news is that our system is almost perversely designed to block the clinical testing of the vast majority of them. The costs and risks are simply prohibitive particularly with natural substances and combination therapies. And who pays the price? Defenseless patients!
In addition to the tomes of data that characterize the barriers to clinical studies, I have personally experienced practically all of them. In 1965, as a young doctor at St. Vincentâ€™s Medical Center in Greenwich Village, I conducted the first clinical study in the United States on carnitine. It took me almost 20 years utilizing all of my know-how, including experimenting on myself with a dubious intravenous solution of carnitine, before FDA approved it for Carnitine Deficiency, a rare and often fatal disease in children. And, unlike many therapies for rare diseases, it is inexpensive!
While FDA is thought by many to be the official culprit obsessed by anti-innovative safety issues that permeate our culture particularly regarding clinical research coupled with an unbudgeable cultural blindspot to its essential importance, there is another extremely influential player- the mass media. The death of one patient volunteer during a clinical study evaluating a new gene therapy was trumpeted by the media causing a national uproar causing the FDA to create additional barriers. Yet thousands of Americans are mutilated and murdered each year unnoticed.
The solution is not to reform FDA- have we not learned that it is not re-formable? We must maneuver around it. Given our cultural mindset, the most doable way, in my opinion, is for Congress to enact the Doctornaut Act. â€œDoctornautsâ€ are physician patients who volunteer to participate in clinical studies conducted by physician researchers under the supervision of their colleagues with minimal FDA involvement as well as waving their right to sue. Letâ€™s not forget that doctors are the most qualified to judge the benefits and risks of clinical studies, not laymen laden committees. If enacted, an array of highly promising candidates would be promptly clinically evaluated and, inevitably, breakthrough hits would soon follow. Who can, with studied reason and altruism, object to doctors taking such chances to help their patients in their battle against disease? Patients would surely be happy, but would physicians accept the challenge?
FIM, the Foundation for Innovation in Medicine, has a particular interest in encouraging clinical research on natural substances because they offer the greatest promise to combat disease but lack financial backing because of the difficulty in obtaining strong patents. FIM sent a questionnaire to physicians asking â€œWould you as a physician-patient want the privilege to volunteer for clinical research of natural substances under the supervision of a physician-clinical researcher without FDA, institutional or other restraints?â€ Over 50 percent agreed. Today there are 800,000 physicians in the United States. If only 20 percent volunteered that would immediately make available 160,000 doctornauts!
You may be wondering how the Doctornaut Act would decrease health care costs. In fact, my colleagues on Capitol Hill believe it would do the opposite! They believe that it would accelerate the testing of new, expensive technologies, raising such costs. Not necessarily so- and hereâ€™s why. Low- cost therapy candidates will also be tested and will compete with high- costs ones in the medical marketplace. For example, the annual cost of the renal dialysis sector is well over $30 billion and growing with our aging population. The largest percentage are diabetic patients. Diabetes is a metabolic disease where the natural biologic processes go haywire. It is not an unreasonable hypothesis to assume that the administration of an inexpensive nutraceutical mixture of logically designed dietary substances along with standard pharmaceutical therapy might normalize kidney function markedly diminishing the need for dialysis and saving billions of dollars. Another example involves cancer chemotherapy. For reasons not clear, anti- cancer pharmaceuticals often have a strong initial effect on killing tumor cells but the latter becomes resistant to them during therapy. Something happens inside those damnable cells. If we can prevent these changes from happening with a combination of an inexpensive non- patented natural substance along with chemotherapeutic therapy, and such a possibility already exists, then patients would benefit soon followed by substantial cost reduction â€” the best of possible worlds!
It is a reflection of our cultural blindspot that Congress has never had a Hearing on what patients want â€” to create a system that will generally accelerate medical discovery for all diseases and other conditions that ail us. Paradoxically, there have been numerous Hearings on health care costs and ways to cut them.
Congress has gotten it backwards!
We are currently addressing major health issues. Doctornauts should be somewhere on the top of the list.