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Remarks Delivered at Public Meeting on FDA's Rare Disease Innovation Hub

October 16, 2024


Attorney Mary Kohler was invited to comment during a public meeting on FDA's plan to launch a Rare Disease Innovation Hub. The Directors of CBER and CDER outlined their bold vision for the hub to embrace collaboration through communication, coordination, cooperation and community. Ms. Kohler's remarks as prepared for delivery are below.


 

Microphone with audience in background

What’s good enough? That’s a question I never thought I’d ask FDA. Yet here we are.

I watched last week’s advisory committee meeting.

  • A room of experts spent an entire day weighing compelling community evidence against a tiny patient population and tough data questions.

  • I heard advisors struggle to wrap their minds around your efficacy ask. We’re all so deeply conditioned. And that magic word comes from the agency alone.

  • That’s when it hit me – creating this hub is going to be really hard.


I have some ideas on priorities. I’ll submit them to your docket. But for now, let’s click up.

 

The real trick is to avoid getting lost in the details.

 

When the question becomes “what’s good enough,” some of those Cs you mentioned will be big challenges. This question sparked real debate in the context of a file. I can only imagine how hard it will be to get people collaborating on this idea in the abstract.  


But I can see how easily this hub could be derailed.


Yet, the patients want this. And they made their point loud and clear last month – those who don’t have the luxury of time can see things a little differently.


You have a tall order.

 

FDA is undergoing massive change. And you’re challenging some deeply held views. That may feel threatening to some. Your new director may soon have detractors. Especially if they start making inroads on that “good enough” question.

 

I launched healthcare compliance into a reticent biotech at the dawn of the PhRMA Code.  I know how hard it is to move a big organization.

 

May I suggest:

  1. Help your new director find their champions. Leaders who share the vision can help so much.

  2. Find some high-impact quick wins. They’re easier. You’ll get traction. Then dig into the hard stuff.

  3. Give this person real authority. Then get out of their way. You have an impressive steering committee. That’s fine. But consensus can stall progress. I’ve been there.


Trust your director. Encourage them to act. And support them when they do. Let them surprise you!


Finally, don’t let perfect be the enemy of good. There’s only one “bad” outcome here. And that’s if we’re still just talking about this in three years.

 

So, onward! And best wishes.


Thank you.  

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