It seems that some form of the drama of Evolve is actually playing out: the FDA has outlined a way for a Duchenne muscular dystrophy drug to gain accelerated approval.

There is a lot to say about this – an entire novel, in fact – so I won’t go into most of my commentary here. Here is one of the key points (from the article):

How much weight should the agency give to the voice of parents and patients, who often are willing to shoulder outsize risks?

Really, the way to look at this is: how many DEATHS does the FDA regulatory process cause?

We tend to focus on how many lives a drug saves because it is safe and effective (those are the FDA mandates, to ensure that drugs are safe and effective. How well do they do given that there are frequently recalls and revisions of their initial findings?). What always gets lost – in part, because it is essentially impossible to quantify – is how many people die because they cannot access drugs. See, for example, Dallas Buyers Club.

As you think about this and form judgement, remember, we’re almost always talking about adults and we’re usually always talking about life-threatening diseases; and think of all the things that you have in your house that COULD kill you or cause you harm, but which don’t (because they work the way they are supposed to) and which aren’t as heavily regulated as drugs.

-JD Cross

 

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