How can precision medicine help small biotech companies?

In our biotech company, we oftentimes are dealing with, again, patients or with programs that are focused on, on rare diseases.

Um, and again, we can't always be thinking about standard or conventional trial designs.

Oftentimes, when you're in early drug development or early phase drug development, you don't necessarily have to think about, will this endpoint that we're evaluating in the early phase setting be supportive of an approval in a later disease setting or in a later line of, of investigation rather? Um, that being said, I think it's really important to think from the very beginning what the registrational pathway is going to look like.

The ARCC program does have some initiatives to encourage increased engagement between sponsors and the FDA.

For example, there was this START pilot program, and again, this is when you have more complex design questions, and this allows for additional engagements between sponsors and FDA.

I will say in this pilot program for CDER programs, this was primarily limited to neurodegenerative disease and metabolic disorders.

What happens if natural history data is limited?