Rubik’s Cube as Construct in the Rare Disease Journey

In my less cynical moments, I like to think that the organizing principle for much of healthcare today is this: how can we do the most for the most people for the least amount of money?

The cynical version might be: what can I create for the most people so I can create the greatest amount of revenue for me?  Another version of that: what can I minimize doing so I can make sure I make more money on the revenue I have?

I was thinking about this set of mental models recently as I set out to moderate a really fascinating meeting of some of the great minds in rare disease.  Sponsored by Alexion Pharmaceuticals, the meeting brought together people from the entire spectrum of healthcare roles – physicians, startup leaders, insurance executives, patient representatives, researchers, pharma, diagnostics, etc.—to ask the question that rarely gets asked: how can we reasonably do the most for the very few people who have specific rare diseases and figure out how to best pay for it and scale it on a national basis without regard to the usual cross-incentives.

I often joke that if it were not for the perverse incentives in healthcare, I wouldn’t have employment.  In my venture role I’m always trying to bet on companies to work around those mixed-up incentives.  In my consulting role I’m always trying to align the elaborate array of Rubik’s cube sides so the colors face the right direction. Either way, it’s no picnic.  Everyone starts their day by donning their most self-interested positions (they go well even with post-pandemic sweatpants).  But for those with one of over 7,000 rare diseases recognized by NIH, and especially for those who remain undiagnosed, their core self-interest of staying alive stands out among all others.

The conversation among those who live on all sides of that Rubik’s cube was fascinating.  Everyone came to the discussion acknowledging their own incentives and self-interests, but open minded to try to bridge the gaps.  The charge for the meeting was to look at the issues around the patient’s rare disease diagnostic journey and think of ways to scale solutions that could be supported by a broad array of stakeholders.  Starting with patient-centricity was, of course, key to the discussion.  But equally important to the dialog, as it turned out, was starting with a framework for looking at the problem through an agreed upon set of lenses that had neither a dark nor rosy tint.

The team that put the meeting together, and especially Alexion, together with Avalere, had the foresight to group issues into a set of 7 domains to help guide the conversation.  The domains created an organizing construct that allowed us to rapidly break through the disparate areas of expertise, enabling what one participant called “human interoperability.”  What that means is that, rather than coming to the meeting thinking about one’s own role in the system, we asked people to come to the meeting to contribute to accomplishable goals that could move the needle in each of these domain areas forward within the next 3 years. It sounds simple, but it was actually kind of magical because it moved the focus to problem-solving instead of leaving it on the spin-cycle of why nothing works.  By adding in just a little competitive spirit, the 15 attendees, who had mostly never met each other before, dove into talking about solutions rather than talking about either the problems or giving their own pre-conditioned response.  They were trying to get the Rubik’s cube aligned on 6 (well, actually 7) sides by thinking in smaller chunks than “cure disease” or “fix diagnostics.”

Conventional wisdom is that it is always better to give people 1 or, at most, 2 choices so they aren’t too paralyzed by options to make a decision.  This meeting worked on a similar principal.  By asking people to work on one slice of the problem, rather than try to take on the whole pie, targeted, solution-oriented thinking could take place.

Our healthcare system is so siloed that most people don’t even stop to think about what’s happening on the green side of the cube when they live firmly on the yellow side.  But when you mix up the colors and set them on a defined task, they engage a whole different part of their brain – the part that makes people want to collaborate on the task at hand.  As many of us know, science is often not the problem in healthcare, humans are (that may actually be the biggest understatement of pandemic times).

In rare disease, the problem is actually the opposite.   People genuinely want to help kids (and adults) who are suffering, but they are sometimes paralyzed by the magnitude of the task and often by the challenge of scaling the solution to the whole country, much less beyond.  Science isn’t always where it needs to be and knowledge-sharing is complicated. When it comes to speeding up the process of diagnosing and treating rare disease, collaborative problem-solving could not be more important. But trying to boil the ocean can stand in the way of taking a sip.

Vincent Van Gogh once said, “Great things are done by a series of small things brought together.”  Our rare disease convening made the truth of that statement clear.   By applying this principle broadly and among people who are not accustomed to working together, it’s possible to build a solution that is greater than the sum of its parts.