The FDA Is Blocking gene therapy Treatment for terminally ill Children

Recommendable! 

A family raised a over $240,000 by donations to have their terminally ill daughter undergo a gene therapy treatment in Milan Italy, because the treatment was not available in the U.S. for bureaucratic reasons!

"... To that end, the family is advocating for legislation in Arizona (also backed by the Goldwater Institute) known as “Right to Try for Individualized Treatments.” The legislation aims to protect a patient’s right to access potentially lifesaving treatments, and it’s a bill other states could replicate as well. ..."

"That’s because the Food and Drug Administration (FDA) prohibits gene therapy in its antiquated clinical trial system. As a result, the family had to raise half a million dollars to relocate to Italy (during a pandemic) to even attempt to save their daughter. ...

This is not the first time the practices of the federal government and specifically the FDA have gotten Americans killed. In recent memory, the FDA has also blocked the development of COVID tests, initially restricted TESLA from selling ventilators, and stalled access to treatments for AIDS.

This agency not only prevents people from accessing many treatments altogether, they also prevent medical innovations through their lengthy and expensive regulatory apparatus.

In their [1980] book, Free to Choose, economist Milton Friedman and his wife called attention to the ways FDA regulation did more to slow the introduction of new medicines than to prevent the spread of harmful ones. They wrote, “The effect on the rate of innovation of new drugs is dramatic: the number of ‘new chemical entities’ introduced each year has fallen by more than 50 percent since 1962. Equally important, it now takes much longer for a new drug to be approved and, partly as a result, the cost of developing a new drug has been multiplied manyfold. According to one estimate for the 1950s and early 1960s, it then cost about half a million dollars and took about twenty-five months to develop a new drug and bring it to market. Allowing for inflation since then would raise the cost to a little over $1 million.” By 1978, that cost was $54 million. Today, conservative estimates put that number at $985 million. ..."

The FDA Is Blocking Treatment for Children with Special Needs. Here’s How - Foundation for Economic Education The general public fails to see the hidden costs imposed by the FDA's red tape, in this case, the real loss of life when people cannot access experimental medicines.

How the FDA made me move to Italy to save my daughter’s life (behind paywall and very annoying ads)