Courage is a word you hear frequently in the world of rare diseases.
Courageous kids battling a horrible conditions. Courageous parents fighting for treatments and, in some cases like John Crowley
, Karen Aiach
and others, starting their own biotechnology companies to develop treatments for their kids. But with those exceptions, you don't often hear courage associated with the pharmaceutical companies who serve rare disease patients, or the FDA who approves rare disease drugs.
It does take courage to develop a drug for a very tiny population of patients, especially if the current high pricing bubble deflates.
It takes courage to have an expanded access program to save kids lives when it may pose a small risk to a company's research program.
It takes courage to truly advocate on behalf of patients within an industry that is often fraught with greed and excess.
But in reality, the pharmaceutical industry, like any other industry, is not a monolith. It is filled with actual people, and those people may themselves be courageous and, if powerful enough, may push their company to courage.
I have met and walked alongside some of those in rare disease and big pharma who are personally courageous people and who do good things on behalf of patients. I wish there were more of them.
But not unlike the pharmaceutical industry, you don't often hear the word "courage" associated with the FDA either. You hear bureaucracy, intransigence, impediments. Courage? Not so much.
But this week I have witnessed the courage of one person at the FDA - Dr. Janet Woodcock.
On Monday, she made the decision
to overrule the advisory committee's recommendation to deny approval for eteplirsen (now Exondys 51), in order to grant accelerated approval to the first real treatment for Duchenne muscular dystrophy.
In so doing, Dr. Woodcock, at professional risk, was courageous. She was courageous to do the right thing on behalf of patients, on behalf of their families, and on behalf of the entire rare disease research community.
That courage was manifested in 126 pages of dispute brewing within the FDA
. Conflicting scientific opinions, disparagements, insinuations, appeals, and finally, the decision by FDA Commissioner himself, Dr. Robert Califf, to defer to Dr. Woodcock's decision.
But in between the lines of that 126 pages, I couldn't help but bristle. There was the implication that meeting with patients, understanding their disease and their burden better, somehow interfered with scientific independence and integrity. It is a battle not unlike the ones patients and that families have faced for decades - the suggestion that "emotional" and involved families can't possibly have keen and valid insights into drug development and evaluation.
In fact, I've also personally met with Dr. Woodcock regarding my son's rare disease, Hunter Syndrome, and our insights on his expermental drug. I've met with the review team as well. Such insinuations are not only incorrect, but they have the potential to chill the possbility of obtaining true insight throughout the drug development process. It is only those wed to bureaucracy and systems, at the expense of truth and innovation, who disparage such meetings.
Dr. Woodcock's courage was not because, in the face of patient pressure, she approved a drug that "might or might not work," as some articles suggest. Her courage was not to think, "Well, the risks are low, so why not give patients some hope even if it turns out not to work."
Her courage was to listen and trust the true experts in a disease, even if they were not within her own bureaucracy. It was not only patients and families in the clinical trial who saw that the drug worked, it was other patients in the DMD community, it was other rare disease advocates like myself who saw it, but even more so, it was actual experts in the disease itself - Duchenne muscular dystrophy - 36 of which signed onto a letter to FDA urging approval
based on the data.
It shouldn't take courage for the FDA to get it right. That should be part of its DNA. But we're still in a transitional phase of how to use the flexibility required by PDUFA on behalf of patients who are truly dying before our eyes, intersected with approving only safe and effective drugs.
Courage goes a long way toward saving lives and saving the image of the FDA as a stumbling block to innovation and medical miracles.
So thank you, Dr. Janet Woodcock, for your courage.