Example of how you respond to FDA across the negotiating table – be careful about how and when you respond to an FDA suggestion. Many times FDA is not saying no, they are warning you to watch out for things, to not make assumptions, but not necessarily to change what you are doing.
“forced” is an appropriate word – by new science and current public perceptions about what is “reasonable” Give FDA background beyond your product and disease area To do something innovative, you and your consultants have an advantage if you can show FDA how it fits with recent science, other similar products, other not so similar products, new FDA laws such as 21st Century Cures examples – biomarkers that are “reasonably” likely to predict clinical benefit in support of accelerated approval: patients demand faster approval of promising drugs and they help define what is “reasonable” evidence; the wide breadth of evidence supports taking a risk, and your product coming forward forces the FDA decision. Another good example is rare diseases – FDA understands that innovative thinking is necessary to develop drugs for the many thousands or rare, and sometimes ultra-rare conditions that currently have no available treatments.
Good example – what agreements can you reach about phase 3 study design at the pre-IND meeting? usually some – but often it’s premature to talk, for example, about the size of efficacy studies before you have some human data to predict effect size. Sometimes FDA says “the issue is premature” but often they say “no” when that’s what they really mean, and then trying to disentangle what they meant by “no” can take important time away from more productive discussions.
To clarify the first bullet – internally you need to think early across every step – e.g. create the product’s label (target product profile). but while FDA does recommend you do this, it doesn’t typically want and is not able to talk about the bigger picture
Starts with how you write the questions – each question should basically contain one concept
Good examples – rare disease with not enough patients. if there are only 50 patients, it’s on-face not possible to have a safety database larger than 50 patients.
Sitting at the meeting table, FDA suggests an idea you think won’t work – often see sponsors say “np, that won’t work”. Right away. Instead adding, “we’ll take that under consideration after the meeting” is not committing but promising to give serious thought to the suggestion
“please” – what do you think that means?
*witness chosen on purpose – they don’t need to say much. Their impact is that they hear what you are saying, and what the sponsor is saying