Right to Try?

Remember her as the badass she was.’ The first bit of the title caught my attention, as I expect that it was intended to do. I had a look. I like to stir the pot from time to time, this one delivered.

Expanded Access always seemed like a good idea to me, I mean if there are no other options, from where I sit, I might ask why not? I expect that was the thinking behind the passage of many states’ Right to Try legislation, and the federal law passed in May, 2018.

While that clears the deck from a legal perspective, as before, the final decision on who gets to try belongs to the drug sponsor. And as described in this article, it is a tough call.

I was at my first job at FDA in the mid 1990s when the Parklawn Building was seemingly surrounded by protestors clamoring for access to unapproved HIV drugs. At the time, the argument that I often heard was that expanded access would complicate enrollment in pivotal, placebo-controlled trials. The ultimate outcome, then, would be societal loss when development and approval were delayed.

That made sense to me then.

Shortly thereafter, I began my own struggle with expanded access. I desperately wanted to get my hands on Lorenzo’s Oil, at the time, the only potential hope for my disease, adrenomyeloneuropathy. FDA position was clear: the only way that you could get the Oil was to be enrolled in a trial. I was not. It was trickier here; I had no crew of angry protesters to help me make my case with FDA. Besides, I was relatively stable, nowhere near my deathbed. I found another way around, and that worked for me. If I was terminally ill and was short on time, it could have played differently.

There are thousands of people dying of rare diseases. In those one-off cases where there is a therapeutic that might help, the government has cleared a path to it. Still, getting it done is a complex thing for so many reasons, whys and wherefores that I cannot begin to understand.

From my angle, it is, and always will be a battle. No matter what wonder cure the industry develops, I fear that they will always be perceived as evil profiteers. And that makes sense to me, though I do not agree with the sentiment. Maybe if it were me crying for help, I would feel different about it. I know that the Mauriello’s do, and they should.

Published by bradleygillespie

I am just a guy with a disease called adrenomyeloneuropathy. I want other guys with the disease to see the good parts of disability. Not the gloom. Not the doom. Make sense?

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