Prominent Physican Proposes A Can-Do Way To Reduce Health Care Costs – By Finding Cures


There is general agreement that the US health care system is in crisis with no doable solution in sight. The ever-increasing cost of health care is the biggest concern of policymakers, politicians and the American people. Access to health care is also of great concern to millions of Americans who have health insurance that, ironically, they cannot afford to use. The controversial Affordable Care Act has already substantially raised health care costs with no end in sight.

Into this chaotic situation steps “A Man with a Plan,” Stephen L. DeFelice, the founder of the Foundation for Innovation in Medicine (FIM), a physician with a long, creative career in medicine. He approaches the crisis in health care in a radically different manner. He is not introducing new policies or bureaucratic programs, but instead, a practical program of action, to be carried out by brave women and men he calls “Doctornauts.”

What is a Doctornaut? Simply put, it is a physician-patient who will volunteer for clinical research of pharmaceuticals, natural substances or new medical devices under the supervision of a physician-clinical researcher with minimal FDA, institutional or other restraints.

Dr. DeFelice has outlined this approach in “The Doctornaut Act,” a discussion draft of which was circulated by Senator Bill Frist and available on the FIM website (

What will most effectively bring down health care costs? The answer, according to Dr. DeFelice, is finding cures. Who can argue? Cure diabetes and there will be no costs.

What is needed to find cures? Clinical trials of promising new treatments, which now face extraordinary obstacles: New therapies, however, can only be discovered in clinical trials.

What is his definition of cures? A cure is any therapy that either prevents or eliminates disease or disabilities by treatment.

What then can overcome the obstacles in the way to the vitally necessary clinical trials? The Doctornaut Act, which will allow the clinical testing of promising new drugs, natural remedies and medical devices quickly and bring about the new cures that are needed.

The ancient Greeks had their Argonauts who sailed unknown seas on dangerous journeys. The Russians had Cosmonauts and the Americans had Astronauts who sailed space craft on dangerous journeys into the cosmic ocean. All of them took great risks to advance knowledge and improve the life of mankind. Some of them suffered – even died – in this effort. They are considered heroes for their bravery. Dr. DeFelice suggests we need a new breed of heroes, this time in medicine – Doctornauts who will bravely and altruistically head into uncharted medical waters in search of cures in the short rather than the long term.

While the term Doctornaut may be new, the concept is part of a long tradition in medicine in which physicians have practiced self-experimentation, trying out new and risky treatments on themselves first. A century ago, Werner Forssmann, a German physician, inserted a catheter in his vein and guided it to his heart. This risky act revolutionized the field of cardiology and he was awarded a Nobel Prize. More recently, Australian physician Barry Marshall swallowed a concentrated solution of H. pylori to prove his theory that this bacterium causes gastrointestinal ulcers and gastritis. His brave act was a major medical breakthrough for which he also was awarded a Nobel Prize. History is replete with self-experimenting courageous doctors in the search for cures. In this process, error and harm are unavoidable, even the possibility of death.

Dr. DeFelice himself is in this tradition. As a young doctor, working with physicians and nurses in Yugoslavia, he acted as a true Doctornaut to move one of his studies forward. He injected himself with two different carnitine solutions in separate arm veins to test for safety. The results permitted him to proceed with other foreign and U.S. clinical studies which were instrumental in obtaining FDA approval for carnitine that saves the lives of thousands of children, both in our country and abroad.

Today, our risk-averse culture is not willing to take such chances. That is why Dr. DeFelice is working so hard to make the Doctornaut Act a reality. He is convinced that history and current trends indicate that physicians will step up to the plate and take the risks others fear to take or which our cultural rules prohibit. In order to avoid the misconception that we are dealing with doctors gone wild, Dr. DeFelice emphasizes that the vast majority of such clinical studies will not be life-threatening because physicians understand, better than others, what the benefits/ risks are.

Dr. DeFelice is doing his best to see that this actually occurs through Doctornauts and the approach he calls “Cure Care vs. Health Care” and how they are related.

If President Obama can undertake an ambitious $1 billion “Cancer Moonshot” to eliminate cancer in his last year in office, perhaps the next President can start out by supporting the Doctornaut Act which will deal with all diseases. This will increase innovation in medicine and accelerate the discovery of cures for the costly major diseases that plague humanity, including diseases and disabilities in children. “Cure Care” will deliver those treatments to the American people – soon.

I first met Dr. DeFelice in the 1980s at a FIM conference. I was intrigued by his straightforward, no–nonsense message: the best way to reduce health care costs is by curing disabilities and disease by prevention and treatment.

But what intrigued me even more was how he proposed to discover these cures. Years ago, he proposed that Congress pass the Doctornaut Act. He had support from Senator Bill Frist, a physician and then Senate Majority Leader. It’s based on the premise that the only way to discover new therapies is to test them in clinical studies in patients. For example, penicillin could not be discovered until tested in patients with bacterial infections, and insulin in diabetic ones. There is indisputable, published evidence of the enormous obstacles to clinical testing of new therapies. Dr. DeFelice calls this the Barrier System in which large numbers of promising therapies have not been and never will be tested. For this reason, the discovery of cures is a rarity despite our exploding technology of which our culture doesn’t get the connection.

Few appreciate the enormous sums of money – billions upon billions of dollars – spent on research on diseases, such as cancer, cardiovascular, mental, neurological, arthritic and pulmonary and many others, without the discovery of cures. The NIH annual budget for medical research is approximately 32 billion dollars. Over the past decade the NIH, apart from the pharmaceutical industry, has funded close to 50 billion dollars on cancer research without the discovery of major cures. There are close to three million patients with the primary diagnosis of epilepsy, with 32 drugs available as treatment, none of which is a cure. Patients over the age of 65 take a daily average of five drugs, none of which is a cure.

Oftentimes, less prevalent conditions, including disabilities such as Down’s syndrome, escape sufficient national attention and how our aging population is changing the status quo of the disabled and their families. In the past, these children left us in their twenties, but due to modern therapies they can now live up to the age of 60, when their parents, however, are much older and afflicted with the costly chronic diseases of aging. So we are dealing with the long-term suffering of two very costly and suffering patient populations in a single household without the availability of cures – an unacceptable outcome, if there ever was one.

The no-cure list is long. Dr. DeFelice has unsuccessfully attempted to have our country ask the challenging question, “Why are there so few cures?”

The Doctornaut Act will rapidly overcome the barriers which block the discovery of cures as well as more effective therapies. It will permit physician volunteers to freely volunteer for early clinical trials, some risky and, importantly, waive their right to sue. If enacted, the base of medical innovators would immediately broaden; more promising therapies would be tested; more medical discoveries would reach patients, curing many. Because of his experience, he also believes doctornauts would immensely benefit children. And these benefits would occur in the short-term.

Despite decades trying to convince Congress to pass the Doctornaut Act, he has, with the exception of Senator Frist, repeatedly run into a stone wall. But he’s betting that the current presidential race will produce an opening for his innovative ideas.

Dr. DeFelice believes the next president could seize the moment and help accelerate the discovery not only of cures but also of low cost medical breakthroughs through the Doctornaut Act. He plans to deliver his message of Cure Care versus Health Care to the candidates during the presidential race.

When I asked what sparked his passion to pursue the passage of the Doctornaut Act, DeFelice attributed it to three personal experiences: his grandmother’s diabetic coma; a child with leukemia; and his discovery and pursuit of the natural substance, carnitine – an interesting triad, to say the least.

When he was 12, his grandmother, or “nonna,” was in diabetic coma lying on a bed in the dining room without hope of recovery. There was a 24-hour vigil by family and friends. He couldn’t accept the fact that she would die and he talked to her, trying to elicit some type of response, which failed. He then went to the local Catholic Church and made a deal with God promising to do good things if He saved her life. He was convinced he had made a deal. But she died that night.

He unexpectedly felt two powerful emotions: an intense hatred of disease and a strong conviction that disease must and can be conquered. He met only one person, ‘Doc’ Druckenmiller, a country doctor who he made rounds with when he was a medical student – $3 an office visit and $5 a house call –who proclaimed hatred for disease. About 15 years later, as a third-year medical student covering the pediatric ward, Dr. DeFelice cared for a nine year-old child with terminal leukemia. The mother and father were kneeling by her bed silently praying. He said, “The scene of Christ and the manger came to mind. The first scene dealt with life; the one before me with death. About an hour later, when I was alone with her, she expired. It hit me hard. One moment she was alive, the next gone forever. Incomprehensible!”

Only a handful of people know that it was Dr. DeFelice who brought carnitine to America in 1965. He conducted the first successful clinical studies on it. After repeated failures, he found funding for development through his friend, the late Claudio Cavazza, proprietor of Sigma-Tau Pharmaceuticals.

Together, they guided its way to FDA approval for the treatment of the fatal disease in children, Carnitine Deficiency, and also for patients on renal dialysis. It’s also given to premature babies who fail to thrive and other conditions. His unparalleled experience in all sectors of clinical research qualifies him to be considered one of the world’s top experts.

As we discussed his third experience, with carnitine, his adrenalin production skyrocketed. He began, “Carnitine taught me about the entire Barrier System which begins with the identification of the drug itself to FDA approval and beyond. If you understood the entire Barrier System, you would conclude that it was devised by a sadist who finds happiness by creating obstacles to keep promising medical therapy from being clinically tested and reaching physicians and patients.”

In his first book, Drug Discovery, the Pending Crisis, published in 1972, Dr. DeFelice predicted, “Our present system of drug discovery is almost designed not to cure the great diseases that confront us. There is no doubt that many will be cured in the distant future, but it is unfortunate that many must wait.” In this book, he first proposed physician volunteers or doctornauts for clinical studies as the solution.

According to Dr. DeFelice, the complicated Barrier System includes the nature of the drug, patents, funding, patient availability, doctors, universities, hospital Institutional Review Boards (IRBs), the FDA, the National Institutes of Health (NIH), the pharmaceutical and medical device industries and many other factors. But the cultural mindset is the governor of the other components of the aforementioned. Interestingly enough, he knows of no one who has traveled through the entire system.

“How would you describe this cultural mindset?” I asked.

“It’s a syndrome characterized principally by fear combined with ignorance, apathy and the absence of knowledgeable leaders who represent the patient. It’s simply too difficult and costly to conduct clinical studies. Since the thalidomide tragedy and the rise of safety–obsessed consumerism, we view clinical research as a necessary evil and something to fear. An over-emphasis on safety permeates all aspects of the Barrier System.”

Dr. DeFelice continued, “Often, the media labels clinical research as ‘human experimentation. ’ This connotes an evil act. If an astronaut dies, he’s considered a hero. If, however, a patient in a gene study dies, all hell breaks loose. The doctor and hospital are somehow considered as baddies. The FDA and IRBs, responding to pressure, create further regulations and rules that profoundly inhibit clinical research and medical discovery which, ironically, are welcomed in the name of safety. What is ignored is the primary concern of patients – to be cured!”

I asked Dr. DeFelice to give us a simple example what best demonstrates our cultural blind spot to the critical importance of clinical research. Without hesitation, he replied, “Rock Hudson,” the famous movie star who died of AIDS in the early phases of the epidemic. “He was a man who was well–liked and well–known to most Americans. Inaccurate media coverage had produced a pervasive national fear of an AIDS epidemic. There were no effective therapies back then.

“An anti–viral drug was in the research phase in France which might have helped Rock Hudson. But the FDA ruled that it didn’t meet their requirements and could not be given to Mr. Hudson in the United States. He had to fly to France to be treated! He should have been able to be treated with this drug in the United States.”

The popular TV show, Good Morning America, learned about Dr. DeFelice’s position and invited him and the head of the FDA to a debate. “I sincerely believed that this was the golden opportunity to finally pierce our cultural blind spot about clinical research,” Dr. DeFelice said. “I stressed that Mr. Hudson should, for example, be able to receive the therapy at Memorial SloanKettering where the experts are. The FDA policy on clinical research is a huge barrier and should have no role in this early medical discovery phase.”

The FDA official was evasive, not addressing Dr. DeFelice’s point. “I was confident I made the point clearly,” he said, “and fully expected that I had started a national discussion on the urgent need to reduce the barriers to early clinical research. Good Morning America has millions of viewers and the AIDS phenomenon was of great national concern bordering on near hysteria as if it were another bubonic plague. It seemed to me to be a perfect media storm.

“I alerted Patricia Park, my indispensable sidekick for over 40 years, to ‘man’ the foundation telephone. The response? Zero! And I mean zero! Not one call from the media, the foundations, the medical community or individuals. If that’s not a cultural blind spot, what is? And who pays the price? The defenseless patient!”

Rock Hudson’s diagnosis with AIDS was a huge story. The thousands of others who were ill and dying was a big story. What, then, could account for the lack of attention to the need for clinical trials?

“Joe, I wish I knew, but I have a theory. Our society is simply not interested in the general issue of why we don’t have cures. Over the years, I’ve asked hundreds of men and women in different walks of life, many with serious and fatal diseases, ‘When was the last cure?’ The overwhelming response has been silence, coupled with blank faces. The few who did respond mostly mentioned the polio vaccine which happened in the fifties!

“When I informed them that, despite our booming technology, there are few cures, the almost unanimous lack of curiosity and concern regarding the reasons why was and remains striking. When I explain the role of clinical research in medical discovery, blank faces and lack of curiosity still prevailed. Many, influenced by persistent media coverage, mentioned concerns about the dangers of clinical studies. Many more inquired whether there are new therapies on the horizon for what specifically ails them or their family and friends. These experiences bespeak of a blind cultural mindset which is unbudgeable.

“Even Christopher Reeve, the then extremely popular actor who played the role of Superman, couldn’t make a dent regarding the importance of clinical research. In the mid-nineties he fell off his horse, partially severed his spinal cord in his neck and became a quadriplegic – paralyzed from the neck down. He later formed the Christopher and Dana Reeve Foundation which, to this day, is dedicated to funding research for cures for spinal cord injury.

“He observed that, although there was much promising research in laboratory studies, particularly with rodents, few were being tested in clinical studies. His emotionally moving declaration, ‘If I were only a rat’, which basic on-target message is the difficulty of conducting clinical research went virtually unnoticed and unheeded.” •

Part II of this article will appear in EP’s August 2016 issue, as well as on

Stephen L. DeFelice, M.D., is the founder and Chairman of FIM, the Foundation for Innovation in Medicine whose mission is to speed up the discovery of breakthrough medical therapies, including cures. He has proposed the Doctornaut Act as the way to discover such cures as well as substantially reduce health care costs. Visit He brought carnitine into the United States and guided it through our entire system to obtain FDA approval which now saves the lives of children with the previously fatal disease, Primary Carnitine Deficiency.

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