Life's full of lovely loops.
For so long, the (sometimes sniffy) response to n of 1 'case studies' has been 'ho ho, of course, evidence is not the plural of anecdote.'
There are so many interesting questions around our 'gold standard' of the randomised clinical trial, but that's not what I'm here for (or in any way qualified for...). I am interested in the n of 1 application of personalised AND precise medicine: gene therapy, cell therapy, approaches like the Notable Labs take on treating your cancer, rather than the cancer studied en masse.
One of the wonderful things about the discipline of path-to-market design is that it is design thinking in action - designing for eventual use (and commercial application). So when it comes to technologies that no-one has launched before, there are a thousand and one questions: how to study, how to measure, how to standardise, how to approve, how to commercialise.
All of these questions require imagination, rather than steadfast adherence to golden rules or comfort zones. The interesting thing seems to be that the rule makers have a lot more imagination in these areas than the folks who should be looking to work within new rules.
If you even take non-cancer applications, approaches like fecal transplant for C diff infection are transformational (the change wrought by that approach is so far off the charts of 'value' as calculated next to pharma that it would be a challenging benchmark). Fortunately, the people behind that idea are not a pharma company, so it is likely to spread quickly in practice.
There are very real and important questions, ethical as well as commercial, but they're now. Imagination, deep understanding of what is possible, what is desirable and passion will all be needed. Just like path-to-market design then, but on a macro scale.