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When the Life and Death of My Child May Be Determined by a Drug Company

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When news that a drug company first became interested in a tiny disease like Canavan, it seemed like a dream come true. It was the exact thing we had all been hoping for. The research and technology I have been tirelessly advocating for and funding since my child was diagnosed in 1998, had been sold for millions of dollars. The company that purchased it vowed to pursue a cure for our children and we believed them.

Well, I really wanted to believe them, but 19 years of fighting for the life of my son has taught me time and again to hold off on the celebration until after we reach the clinic and are allowed to actually use the medicine to help our children.

This was almost two years ago, and there has been no progress towards a clinical trial. The company that promised to help our children was recently acquired by one of the biggest drug companies in the world, a pharmaceutical giant with unlimited resources to finally discover the cure for Canavan.

But will they move forward quickly enough to save more children from dying? Will they move forward towards development of the new drug we have worked so hard on for almost two decades? Will they move forward at all?

This is one of the most helpless and desperate feelings I have ever experienced.

When I was told over and over again that no one was working on a cure for Canavan, at least I had the option to start working, raising money and do something proactive towards saving my 5-month-old baby. When the FDA said “no” to our clinical trials, at least I had the option to enlist the aid of every elected official who would listen to my story.

Our government officials are supposed to help the people who voted for them, and even the people who didn’t. Legislators and the FDA are publicly accountable for their actions and inactions. In hindsight, I realize that asking my government to save a few dozen cute babies who didn’t “look sick” yet was not so bad after all. It feels like nothing now, compared to dealing with a drug company.

I have been working with a patient advocacy representative at the drug company trying to figure out what I can do to help move this life-saving technology forward more quickly. The answer I dreaded most was the one I received.

I was told I needed to show a leading pharmaceutical company how they could make a profit by pursuing the cure for Canavan disease.

I took frantic notes during my first phone call. I was granted a meeting, and advised to create a presentation of my business model and cost analysis showing how they could make money from developing a cure for Canavan. The list of what I needed to produce made my head spin. I have access to everything they want, but this is a hard sell. I have to show a multibillion dollar company how they can make money by curing my child and the hundreds of other children around the world whose families are desperate to save their babies.

From my experience working with them, it feels like these companies are not expected to answer to anyone, and seem to have little public accountability.

I have learned that the squeaky wheel gets the oil, but can someone flying in a private jet even hear the small squeak of a tiny wheel down here? I’m going to make as much noise as possible and find out.

Originally published: September 30, 2016
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