The new PI trials are kinda rigged

[Upbeat]

After dealing with the people running the vx950-vx-222 trial in Colorado I learned these trials don't reflect the general response people will have after approval..  A true trial would include everyone not the hand picked subjects they expect to show good results with. I had no Idea that in all previous vertex trials the min requirement was at least 150 platelets. It explains to me why the FDA is so tough on new drugs being approved.

[IAmTheWalrus]

Well put, Hector. That is exactly why transplant patients can't get into any of the studies. Nevertheless, well said. It is some comfort knowing it is for the cause of scientific advancement (me, only a little bit sarcastic)! :-)

[spectda]

The fda approval system obviously needs to change. when you have a disease where the response to treatment is determined by mathematical/biological values why does their need to be a placebo.

Of course side effects can often be subjective and trials are also largely about the sides.When the choice is to start saving lives and let people who are getting very sick and close to death use these new drugs, it is a crime to extend the red tape for so many years and bureaucratic garbage.

Of course new drugs need to be tested for safety and effectiveness, I don't have the answer. but someone more intelligent then I should have some new and more efficient way of doing this.

My understanding is that the fda has much to do with the terms of the trial. They may not come up with the presentation, but the are very involved with the details before approval.

[eureka254]

It's always about fighting exclusions, isn't it?

The way I see it, it's a constant exercise to try and make all things equal -- and accessible -- in every playing field for anyone who want to play (or pay, or treat, as the case may be).  Everywhere we go, whatever we do, it all comes down to working toward achieving access for all and equal freedom for everyone to choose, whatever your condition, whichever your circumstance.

I used to wonder if everything was rigged, but I've simply resigned myself to the fact that the world is absurd.  Medicine should be a right, not a privilege, but we're not quite there yet.  Fight the power.

~eureka

[robo229]

As mentioned in the FDA expanded use hearing, additional study arms that are more reflective of a real patient population should be created and investigators can opt to include the data in their findings or at least report it as a different ttype of study.  Let's get these new promising drugs to those who need them most.  Stop the cherry picking, it's absurd.  

Just my .02

Robo

[RobertBeWell]

For better or worse, they want the best chance of showing their drug "works". If you're within a standard deviation of the middle of the bell curve, as far as severity of disease goes, this is a good thing, because it makes it more likely that the drug will get approved and into the hands of the general population, where, hopefully, it can do the good we all hope it will.

What might be reasonable is to perhaps conduct two "parallel" trials for the real sick, and the not-so-sick. Not sure if it will ever happen, but at least it would give everyone a reasonable shot at the new stuff.

Me best wishes to all who are excluded for a treatment soon.

[Upbeat]

You have never struck me as being the mean type.   But what do I know :)   I have a good feeling about this new combo of vx-950 and xv-222 as a stand alone.  After talking to the trial people so do they.  They believe that this combo will make you RVR in just 2 weeks and SVR in 12.  But you never know. I have been disappointed before.  I really thought that the first PI was the answer untill the Monkey studys came out.  But still this may be for you!!

[nygirl7]

Sorry that was an el typo should read Dear Trinity 4 sweety pie :)

[nygirl7]

I think you are a liar - you are wonderful and sweet (and can certainly be my friend forever)  ♥

[Trinity4]

I think Hepc is rigged.  The meaner you are, the more virulent the virus.  That being said, I'll never get rid of it!!  :)

[HectorSF]

All trials have requirements that must be met. The trials studies responses of particular groups of patients not all patients with the disease. For a trial to be statically accurate the trial members must be limited. You couldn't allow people with other major disease to join a trial as you couldn't be sure if the other disease affected the results. It would skew the results, making them unless. That is why different trials are performed for different populations. For example, there are separate Telaprevir trials for treatment naive patients and patients that failed previous treatment.  
By limiting the vx950-vx-222  trial in CO I would imagine they don't want patients having thrombocytopenia and cirrhosis in the trial.
The FDA doesn't control the trials, the drug companies do. The drug companies present trial evidence that the drug is safe and effective. The FDA decides if the trials conclusions are accurate based on the documented evidence presented to them. Limiting trial members has nothing to do with the FDA being tough on approving new drugs.

[jmjm530]

Hey Upbeat, how ya doing! Still long on Vertex?

To be fair, trials must have inclusion and exclusion data in order to produce results that can be used in a meaningful way down the road in the clinical setting. Hard enough to get meaningful data when testing a few variables, but you simply cannot open the flood gate and let everyone in on a particular trial.  The trick of course is to be informed and to evaluate all this data before making any treatment decisions. Unfortunately, this step often gets skipped. Like "Walrus" says, the devil is indeed in the details.

All the best,

Jim

[IAmTheWalrus]

The devil is in the details of any statistical study. The difficulty is in knowing what was "included" and what was "excluded".

"There are three kinds of lies: lies, damned lies and statistics."
- Benjamin Disraeli