Health care is an immoral battlefield on which to wage political warfare.
People dedicated to the preservation of endangered wildlife understand perfectly that certain animals are more glamorous than others. The haunting eyes of a young male Siberian tiger or the incomparable personality of an elephant matriarch will generate more public support than a lemur, frog or eel species.
Can the same be said of ordering human tragedies? The agony of the peoples of Syria, Sudan, Haiti and many others generate very different levels of sympathy. The enigmatic rules of this phenomenon are, in my view, largely political and result in a whole lot of suffering populations that are almost entirely forgotten. It is heartbreaking!
In research and health care, scientists and activists experience the same hierarchy among illnesses
For years, my mother experienced cruel symptoms that relentlessly worsened. The condition was so misunderstood that it was only after her death that I learned its name: scleroderma.
What makes an illness worthy of more attention than others involves many factors among which are the number of patients affected by it, political support or a celebrity face associated with it that captures popular imagination. Other relevant elements are arbitrary, but consequential, like the relative effectiveness of lobbyists.
ALS is a rare illness, a medical orphan. It is a degenerative nervous system disease that dramatically reduces physical activity. It affects nerve cells in both the brain and the spinal cord and progressively and irreversibly weakens the muscles. It has currently no cure but certain treatment can slow down its progression with some patients.
In spite of the merciless nature of the scourge that can disable and kill very young people, it has to date remained unable to inspire a sense of urgency at both the FDA and the funding agencies for medical research. On average death comes approximately two years after diagnosis usually through the complications of pneumonia. Others can live decades with the illness.
Matt Bellina is a Pennsylvania resident. He is 32 and a former navy pilot. He was diagnosed with ALS in 2014 but remembers problems with coordination as early as 2006. His short hair is dark and his kind smile has an infectious quality. He is married with two young sons and a new baby born today.
Retired Lieutenant Commander and Ms Bellina with their two sons
Matt has become an unlikely but highly effective and articulate crusader to find a cure for ALS and persuade the FDA to authorize, more freely, experimental treatments. He captures perfectly the absurdity of the current situation by observing that he could, more easily, find a U.S. state that allows physician assisted suicide than an experimental drug to prolong his life.
Caitlin, his beautiful and determined wife, rightly calls the clear heartlessness of the options offered: “The dumbest thing I ever heard!”
But stupidity alone cannot explain this cynical reality. Our implicit message to these patients is transparent and sordid: “Why don’t you just go and find a place to die quietly.” After they die, neglected disease sufferers cease to disturb our conscience.
To compound the challenge, some drugs approved by FDA for treatment have become dramatically more expensive. It follows a nationwide pattern that includes Epipen, Daraprim, etc. A New York Times article describes the situation: “Although some prices have been caused by shortages, others resulted from a business strategy of buying old neglected drugs and turning them into high-priced specialty drugs.”
Another scandalous example of this is the very recent announcement by Marathon Pharmaceuticals of the new U.S. pricing of $89,000 for the old medication Emflaza. It is only $1,200 in Europe.
For years, Mary Kay, a Montana native and currently in Arizona, worked in government affairs in one the world’s largest pharmaceutical companies. She became a friend of Matt and his family while focusing on ALS. She is the one who brought to my attention his heroic battle and the extreme vulnerability of ALS patients.
She explains the economic reality of drug research and pricing: “The cost of drug development is astronomical. It costs $2.6 billions to bring one drug to market in the United States. Less than 10 percent of the compounds that American pharmaceutical companies research come to market. It’s extremely risky.”
But some medical and financial triumphs make the risk entirely justified. She continues: “I used to work in HIV/AIDS and saw it go from a death sentence to a chronic disease. I stopped going to the funerals of all my positive friends.” She then lists other equally stunning successes with Taxol for breast cancer victims and Opdivo for lung cancer.
Sadly, there has been no new approved drug for ALS in 20 years.
The message of Matt and May Kay is simple: Ethics matters supremely when considering the pricing of certain drugs and the swifter process of authorization of experimental treatments for so called “incurable diseases.”
Special family time for Matt and his two sons Kipp and Jp. He often wonders: “ How will they remember me?”
Somewhere along the way our society has begun to value compassion a lot less. Our superficial celebrity/social media culture has created a moral numbness that makes it almost comfortable to ignore vulnerable populations.
Matt, Mary Kay and others are dedicated to reminding us that behind every rare illness is a lonely human drama of intense suffering. They represent the conscience that we, as a society, are at great risk of losing.
Note: In 1983 congress passed the Orphan Drug Act to create economic incentives to develop new treatments for rare diseases.