Monday, May 21, 2012


Hi everyone~
I have some crazy news that I think we all need to be aware of as we go forward in our agreement to take the vertex drugs.  I had become increasingly concerned about CFers getting sick and experiencing more exacerbations as the vertex drugs were being discontinued after Phase II trials.  While the vertex scientists are not all doctors, some of them are and I feel  and felt like they were completely falling away from one of the principal precepts of medical ethics taught to doctors: First, Do no harm.  Yes, the drugs are life-changing in a positive way (a cure even) and, for this reason, are harmful when discontinued.
Because of this I contacted a top 25 Lobbyist in Washington to just see if there was anything he could do to help us atleast get the patients who are volunteering a pass to continue the drugs when the phase II trials end (seeing as there is no glaring safety concern).  While he had never heard of this issue with CF patients, he was very aware with all going on with Vertex and CF.  He graciously contacted the FDA commisioner and addressed our concern....
According to the FDA commisoner:  there is no FDA law keeping these patients from receiving these drugs following Phase II trials!!!!  WHAT???  VERTEX has complete control and authority to allow these patients access to these drugs.  Now, keep in mind, that is not all of us, but can we not agree that our community should stand behind these patients who are agreeing to take these drugs!?
According to the LOBBYIST, he said we must all rally together and agree to NOT SIGN up for trials, unless it is written in the contract that the trial patient is able to remain on the drugs after Phase II ends.  We have the power.  Vertex needs us just like we need them....  They can't get paid unless we agree to be a part of this... it is ok to stand up for ourselves in this way....sometimes the boat needs to be rocked.
Along those same lines, there is a bill going through Congress right now with approval (they think) by October that will allow drugs that are life changing to have a very different course of review and trial period.  It is called the Advancing Breakthrough Therapies for Patients Act.... Basically, this allows companies to expidite clinical trials so that patients can get access to drugs possibly after Phase II trials.  The company will have to conduct phase III trials even while patients have access to the drugs.  GUYS, IF THERE WAS EVER A TIME TO CALL YOUR CONGRESSMAN IT IS NOW.  The ExPert Act is also important, but the other will allow drugs to reach us much more quickly.

And, Finally....  Do you know how powerful our words are??  One of the reasons that Vertex released interim results from the 770/809 Phase II trials was due to the fact patienst were blogging and Vertex could no longer control what information was being released.  From the CFO of Vertex at the Deutesche Bank Securities Annual Healthcare Conference last week:
"The safety finding was very strong and we want to accelerate the program into a Phase III setting. For us to do that we have to talk to regulators both in the US and Europe. We have to talk to trial investigators, patients are now blogging regarding the results and there was a corporate concern here about information getting out of the company and not being in a controlled environment. That is what drove us to the announcement"
It is time to make our voices heard...  They need us as much as we need them.

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