Why it’s hard to be in the minority of the minority

Last week was an historic and monumental one for the Cystic Fibrosis Community – after over 10 months of relentless campaigning, a deal on the breakthrough drug Orkambi was finally reached.

The deal means that around 600 people with the ‘double delta’ version of this life-limiting condition will get access to a drug that has been shown to reduce the time spent in hospital, reduce the number of exacerbations, improve weight and lung function and generally offer a much better quality of life. A life with more promise. A life with more hope. A life more normal. A life!

I could not be happier for the patients and families who can now put the uncertainty behind them and look to the future without being crippled with fear. I campaigned heartily alongside them and I’m bursting with pride at what the community has achieved. We may be small in numbers but we’re massive in heart and determination. We are the definition of small but mighty.

However, I also have to acknowledge the anti-climax I’m feeling on a personal level. I put so much time into running #YesOrkambi online that I guess it was a distraction from the reality of my own daughter not being eligible for the drug. So despite myself, my heart feels devastated. A devastation that I don’t want the success to be tainted with but that it’s hard to shake off. Generally I’m still devastated that my daughter is unlucky enough to be in the minority of people born with Cystic Fibrosis. But specifically I’m devastated that she’s unlucky enough to be in the minority of CF patients that neither wonder drug (Orkambi or Kalydeco) will treat.

There is still 30% of the CF Community without a drug available to treat the underlying cause of the illness and I worry that they will be forgotten about in the hype.

PLEASE don’t get me wrong, this deal is as good as it could be at this point in time as it also includes any pipeline drugs that might come to market from Vertex (the pharmaceutical company) over the next ten years. This is so important as it means that breakthroughs in that time won’t have to be fought for in a soul-destroying circus like the one we’ve just lived through. This means we’ve won a bigger fight and a more important one. We won the fight that is the PRINCIPLE of access to life-changing, high tech medication. This is landmark. This is crucial for more than this one patient group. This is everything.

Orkambi long press 3

And without this step, there would be no next step.
There would be no next drugs.

BUT it’s still hard to be in the minority. To be in the group that’s still fiercely watching clinical trials, waiting for a breakthrough for their own child’s rare CF genetics.

Am I jealous? No.
Am I bitter? No.
But I am a little sad.

The heart will feel what it feels no matter what the head tells it. So rather than put a front on and pretend those mixed emotions don’t exist, I’m acknowledging them in the hope of dealing with them and moving on, as this is a good news story for everyone, not just those who will immediately benefit.

I’ve cried so much in the last week and most of the time I can’t even tell if the tears are happy or sad. In truth, they’re a mix of every feeling imaginable. I sense that we are living through a massive moment in history, the beginning of what will hopefully be a golden era in medicine. But I’ve been listening to my daughter cough for 6 months solid, so it’s hard to not feel impatient when the day-to-day is sometimes so challenging.

Yet I also feel I have no right to be sad. What about those families who have already lost children or other family members to this unforgiving disease? For them it is the ultimate bittersweet and there is no ‘maybe our time will come’. Science didn’t come in time for them and that must be impossible to live with emotionally. I admire their strength and positivity in fighting for this next generation. We still have a maybe, and a very real and large maybe at that. So in the words of a certain songstress, I need to shake it off. I need to stop thinking about our miracle that feels painfully close yet still so painfully out of reach. I need to focus on keeping my daughter as well as she can be, so that WHEN her breakthrough comes, she is ready, able and well enough to embrace it with both arms. I need to channel that feeling of hope. Just hurry the f*** up already!!

Illustration courtesy of the lovely Olivia Golden



5 thoughts on “Why it’s hard to be in the minority of the minority

  1. Bernie, if you didn’t feel like you have done this past week, there would be something wrong.

    The energy and passion you have shared for the community are so admirable, I know that we are indebted to you.

    We will also stand side by side with you until everyone in the community has a drug of hope.

  2. Bernie beautiful written piece that we all identify with and you have explained so well how the 30% must be feeling and can’t quite put words to it. The one word you do say is HOPE and that is so important. I know soon that hope will be reality for you and as Keith said we will be there with you. Thank you for all the energy passion and enthusiasm you invested in the yes Orkambi campaign 💜xxx

Leave a Reply

Fill in your details below or click an icon to log in:

WordPress.com Logo

You are commenting using your WordPress.com account. Log Out /  Change )

Facebook photo

You are commenting using your Facebook account. Log Out /  Change )

Connecting to %s

This site uses Akismet to reduce spam. Learn how your comment data is processed.