Bureaucrats, like many of us, are enamored with their perceived intellect. Intellectual pursuits morph into a dependence on procedures and self imposed protocol. This dependence is comparable to our modern day dependence on navigation devices.
On Monday I was walking from a subway station to a conference center at a hotel. I had looked for the place on a map many days before but on that day I chose to be dependent on the navigator app on my phone. It served me well until just a few minutes before the estimated time of arrival. I came to a complicated intersection where streets and a highway converged. The navigation map directed me to make a left turn across the highway. There were no crosswalks that would lead me there. I was flummoxed for a good ten, sweaty, minutes. Finally, I looked up the location of the event I was attending and read the conference center was at a Marriott Hotel. I was looking for a Sheraton. I looked up from my phone to read the large letters on the side of the building across from me to the right (not left) and rolled my eyes as I read “Marriott”.
My use of the navigation tool had overruled my use of common sense.
Little did I know that the death of common sense was going to be the theme for the day. I spent the rest of the day at the Food and Drug Administration’s Advisory Committee Meeting for the proposed accelerated approval of a life changing drug. And there I saw intellectual bureaucrats exhibit a tragic tenacity to hold protocol up on an altar while the blood of common sense was spilled from it.
Eteplirsen is a drug being considered for approval by the FDA. This drug is a treatment for Duchenne muscular dystrophy, a fatal genetic mutation that damages muscles and confines boys to wheelchairs by the time they are in their teens and ultimately causes their heart or lungs to fail. There is no cure or effective treatment for this horrendous condition and this is the first drug to slow the progression and contribute to a better quality of life without side effects.
This advisory committee concerned themselves with the methodology of the trial done by the drug’s company, Sarepta. This advisory committee chose to vote on questions that addressed proper protocol versus the drugs effectiveness testified to by experts on Duchenne. Some on the committee realized they were being maneuvered by the FDA scientists. Yet the negative vote was still the majority.
The flexibility necessary for a rare disease was disregarded as the FDA scientists worshipped procedure and dismissed the observations and experience of geneticists, doctors, scientists and many other experts on Duchenne! And with egregious condescending attitudes they dismissed the real experts – the boys, young men and family members in the Duchenne community who know the disease and the startling effectiveness of eteplirsen. The Duchenne community spoke with expected passion but not ignorance or desperation. They spoke as experts.
If, when the FDA makes their final decision in May they choose to side with this advisory committee they too will be leaving common sense on death’s doorstep. They will be showing a startling lack of ethics. How can this government agency in a country that stands for freedom, withhold an effective treatment that has no side effects? There are grave consequences to a decision to not approve: those with this particular strain of Duchenne being denied a drug that can improve their quality of life, Sarepta likely losing the funds to continue their breakthrough research, and the entire Duchenne community losing the momentum of breakthrough research.
Breakthroughs that they don’t have the time to wait for. The death of common sense means the death of boys and young men. The FDA’s decision is one of life and death. No, eteplirsen is not a cure. It is hope and relief for some. And it is the beginning of a hope for a cure for all. Common sense testifies to that fact.