In my mind, there is no doubt that we need to make sure that people who stand to gain the most from a medication get access to it. However, you're right: in the real world, things often start to spread out, such that other patient groups or patients who are maybe not the focal point of the treatment benefit will get these drugs.
I think that's where I would say that investing in rare disease registries will be very informative and is a systematic way to approach this, so that we can follow patients over time who are getting access to treatments and who are perhaps using off-label treatments. It's a little bit different from drug-based registries. I think investing in these kinds of infrastructure is really critical.
If you don't mind my making a short comment on Dr. Coyle's initial suggestion, I think using cumulative figures around describing rare disease is really an effort to do two things.
One is to demonstrate that when you put together all rare disease, in fact it's not that uncommon. We spend a lot of health care resources—extraordinary health care resources, sometimes—on some very common things like heart disease, stroke, and cancers. I think that in the rare disease space we want to get some skin in the game here, so describing it in that terminology helps people to recognize that it's a big problem.
The second thing it does is show that solutions that crosscut all rare diseases are needed. Yes, you may have an ultra-rare disease versus something that's one in 20,000, but there are deficits in our way of approaching that in the health care system and in the evidence review. If we actually develop effective paradigms, it would be useful across all these rare diseases.