I’ve been spending a lot of time courtside lately. Basketball court, that is. And although I once played competitively myself (no surprise at 6’2”), these days I’m mostly watching my son Ellis from the safety of the sidelines.
I love basketball. It’s a complex team game and for a team to succeed they really have to know how to play together, how to move as one, playing to each other’s strengths while staying united and focused on the goal. That’s a big ask for anyone and, particularly in Ellis’ case, for a group of independently-minded 14-year-old boys with varying skills and roles.
Lately, while watching him play, I have reflected on similarities in our CF world. The introduction of two new drugs—Kalydeco and Orkambi—has turned things a little topsy turvy. On the one hand, we have found ourselves seeing possibility and hope where there had been darkness. But, unfortunately, it’s not a total slam-dunk.
Firstly, because we know these drugs aren’t going to win the game for everyone. At best, it will be those with the right mutations and, even then, the drugs seem to work to varying degrees for different people. Many of us have heard of patients whose sweat chloride has gone back into the normal range on Kalydeco, they have gained significant weight and seemed to halt the progression of their illness. Wow! Most clinicians working in the field are saying this is a remarkable accomplishment. The extreme success stories on Orkambi are less common, but there have been some.
For those who haven’t experienced this turnaround—or who have the ‘wrong’ mutation—it’s a tricky time. Anyone who has lived with this illness and knows its horrors and heartaches wants everyone in the game to win, to be cured. Yet as we see things coming down the pipeline for others but not us, or our kids, it’s scary. And we can begin to feel a little more alone.
The thing is that there are a lot of players on this team and we all have slightly different ideas of how the game might best be won. There are the patients and families who, until the introduction of mutation-specific drugs, all had a fairly similar idea of how to play. Then there are the clinical teams, the researchers, the support organisations (CFV and CFA), the government (who funds our healthcare and drugs), and the pharmaceutical companies. Lately, many voices have been speaking up about how to win. And I have found myself at times lately wondering if we’re all playing to win the same game.
So, what is the goal? It’s healthier and longer lives for people with CF. That has to be our ‘true north’, the goal we focus on when things get messy and complicated.
While we all feel panicky about needing treatments and changes today, we must focus on our goal and take the long view. We’re going to need drug funding and friends in government for a long time to come, so it’s critical we maintain those strong relationships so we have a sympathetic ear when the next wave of drugs arrives. And there will be another wave. We also need to remember that sometimes the other players, like the pharmaceutical companies, have competing goals and priorities besides our ‘true north’.
As patients and families, we certainly can be single-minded—and we need to be. We also need to be smart and united, to speak as one. Our stories will sway hearts, but the evidence must back us up. Our whole team must be onside through strong, well-supported arguments. Because when the game is over, we want everyone with CF to win.