A Day Without the FDA

The recent government shutdown makes me consider the ramifications on the FDA.  As the mother of Hunter, a terminally ill two-year-old with Spinal Muscular Atrophy Type I (SMA), I can't help but wonder.  What will this mean to my baby?  What will this mean the others with SMA?  What will this mean to the tens of millions with orphan diseases?  We have had a day without the FDA (technically a few days with a limited FDA).

When Hunter was diagnosed with SMA in 2011, we were told there was no cure.  We were told there were no treatments.  A more accurate statement would have been that there were no FDA approved treatments or cure.

I want to pose the question:  What if there were no FDA?

 The FDA was created for the protection of consumers.  I believe the FDA was created to provide a give and take relationship in this protection.  Today, the gives significantly outweigh the takes.  Drug costs are at an all time high, and those in need are left to wait.

I believe the FDA's protection has gone too far to the consumers' detriment.  Over regulation makes many orphan disease trials cost prohibitive.  Moreover, the lengthy application and trials prevent medications from reaching patients for years.  This directly affects the more than 25 million Americans with rare diseases.  More than half of these are children.

Many orphan disease treatments are in the pipeline.  However, the timeframe for new drug approvals is over a decade, and costs over a billion dollars.  This is a hard pill to swallow for those with terminal illnesses and their loved ones.  I know this.  I live this.

Without the FDA, researchers, pharmaceutical companies, and doctors would be permitted to provide life saving medications to terminally ill patients in need, as the need arises.  Perhaps there is an argument for a resulting abuse, and harm to consumers.

I believe the ethical committees researchers go before, the research and medical institutions where they work, and the leading doctors in their research fields are the proper regulatory bodies.  I do not believe a rush of freelance researchers providing medications to the detriment of consumers would result in the FDA's absence.

I would not think twice about opting out of the FDA's "protection" to obtain potentially life saving medications for Hunter here in the United States.

Consider this: Physician assisted suicide is legal in Oregon, Washington, and Vermont.  In regard to non FDA approved medication, it is illegal for physicians to prescribe or provide the terminally ill with potentially life saving medication in every state. 

For now, we do have the FDA's overregulation.  The terminally ill must travel to other countries to obtain non FDA approved life saving medication.  This requires the ability to travel, and the funds necessary to pay for the travel and medication.  Sadly, the majority of the terminally ill lack one or both of these requirements.  They are essentially denied a right to life, because the medication is not FDA approved.  On the other hand, they are afforded the right to die in Oregon, Washington, and Vermont, so long as the medication to assist in death is FDA approved.