Cancer is the reference, but it should also apply for other diseases as well.
What do we do when we have a targeted drug, introduced in a properly selected group of patients, which has an outsized effect on overall survival in a rare or deadly cancer, but where that benefit is seen in a small trial, where we would still need more evidence to fully understand how to best use the drug in clinical practice?
We might want to approve such a product earlier, and require a post-market confirmatory study to validate the finding – similar to an accelerated approval approach.