Wednesday, June 25, 2014

Proceed with Caution - Vertex's Big News

For those of you who haven't heard, Vertex announced the results of it's big Phase III combo drug that may cover roughly 50% of the community. You can read the press release here.

I'm so pumped to see the joy and hope exhibited by so many in the CF community today. I hate to "be that guy", but I did want to throw out a bit of context and history to be sure that we are all managing our expectations:

1. The Phase 3 study showed an average increase in lung function of 3% Context: Cayston showed a 2% increase in it's Phase 3 head-to-head study against TIS.

2. The results have not been completely vetted by those outside of Vertex. Context: This is the same company that is now being sued for "grossly overstated" results in the Phase 2 trial of the combo drugs -

3. About 46 people dropped out of the Phase 3 study because of complications. Context: Extrapolate this to the potential users of this combo, and you'd have around 1400 people with DDF508 who still wouldn't benefit from this drug. You'd also have to include those who saw no benefit but elected to stay in the trial. (NOTE: There are also people who blew the doors off of old PFT results!!!!!)

I could go on, but I'm feeling like Captain Buzzkill. I just see a lot of celebrating without facts (or misinformation) and want peeps out there to understand that we have a long way to go for the next generation of CFers. With that said, if this drug has the potential to in essence halt the decline in lung function at an early age, I'd cut off my left arm if that meant that everyone had access to it.


Added after reading feedback: Yes, celebrate!! Any move towards a better life experience while managing cystic fibrosis is a win. I'm not saying that this is not good news. Selfishly, anything that could possibly extend my life even a minute with my wife and daughter, is something worth being excited about. My biggest goal, and frankly why I got so involved with the CF community, is to make the life experience of the next generation of CFers, and the generation after that, better than my own. I don't want to see the CF patients of the 90's, 00's and 10's experience the same struggles as I did, a patient of the 80's. Progress doesn't only come in the form of a pill, but I'll sure as heck take one if/when it does!

And I've always been consistent in saying that we do not need to see positive numbers for it to be a success. Remaining steady would be a BIG WIN for the CF community. Shoot, not having to work so hard to maintain what I currently have is something I'd pay good money for :)

(I took this from my FB page as I know many of you are not friends of mine on there yet you read this blog. I'd love to hear your thoughts)