Important Legislation. Contact Your Senators- Right To Try

Hi Rob,

Thank you so much for sharing this, it's such a huge issue.  I'm a huge advocate of compassionate use and right to try (100% for both compassionate use and right to try).  I actually currently work in clinical research and with the statisticians and investigators who develop protocols and analytical plans to reduce bias, confounding etc. and I completely understand the importance of sound scientific study design and not introducing bias, but I don't understand why they can't have two separate data pools. Double-blind randomized (that's key) trials remain the gold standard for research but when you are facing an outcome that is permanent, who wouldn't want to try?  I remember when I was involved in dialysis research and Isaw some of the first dialysis machines (they looked like torture machines) but when your only outcome is death, you do it, that is your only hope at survival.   Also, you hit the nail on the head, they are worried about the numbers, but I'm sure they could figure out a way to deal with this and you are absolutely right , they treat the sickest patients at the end and then the numbers looked skewed.  There was a case of a melanoma patient that made the news several years ago, him and his wife really fought until he passed before PD1 was approved:  http://www.savelockysdad.com/   

So sorry for your loss of Adriana, I can only imagine how much you miss her, but I'm so moved by your support of Adriana and she was an inspiration to us all on the board.   Many hugs. I'm sure she is proud of you.

I agree with you both.  Problems with clinical trial as they currently exist and "right to try" legislation has been a huge topic in my house for years.  Here is a post we put together with some of our thoughts:

http://chaoticallypreciselifeloveandmelanoma.blogspot.com/2017/01/the-trial-of-trials.html  

I am 100% behind the "right to try".  Who am I to tell someone with nothing left to lose what it is they can and cannot do.  I do have a huge concern that the "rigth to try" will not mean much if insurance payers are simultaneously given the "right to fail to pay" while Big Pharm has the "right to charge whatever they like"!!!  If that piece of the "right to try" legislation is not addressed, even if passed, it will still leave most patients in need without care.

I also feel that the knowledge gained from ratties like myself is critical.  While not as essential as the removal of ALL obstacles from treatment options…that is a big piece of how we bring better treatments to more folks down the line.  Again, something that could and should be addressed in "right to try" legislation.

And finally, here is a piece on just how much you can attain if you know the right people AND how much good "experimental" treatments can do from a piece I wrote back in 2014:

For those who buy into the… 'lack of control Pharma, the FDA, and researchers have – I beg to differ.  Exceptions can be made any time [those agencies] choose to make them.  They just don't often choose to do so.  For instance:  Take the case of Ralph M. Steinman.  Don't know Ralph?  Well, he was an amazing researcher in the area of the immune system and cancer….very smart….was awarded the Nobel Prize just three days after his death in 2011…and apparently, a really great guy.  Well.  Ralph got pancreatic cancer.  Perhaps the only cancer along with renal cell carcinoma that gives melanoma a good name.  So what happened?  His research buddies, folks at the FDA and various review boards pulled every string in the book.  "In addition to standard treatment, Steinman ended up enrolled – under a special patent provision – in four ongoing clinical trials of various dendritic cell-based cancer treatments, most of which were not even being tested for pancreatic cancer, along with several other experimental immunotherapy and chemo treatments.  Schlesinger, a member of the Rockefeller Institutional Review Board, steered his treatment through all the necessary IRB and FDA channels…"  [The Patient Scientist, Scientific American,  Harmon, Jan 2012]  Hmmm….  So, did Ralph deserve that special treatment?  Treatment that kept him alive years longer than most folks with his disease…and without said special treatment?  Sure!!!  I think so!  But!  Don't we all????  Must we all be brilliant, well connected scientists in order to be granted such care?'

Sorry for the long rant!  But, Rob is right!  If this stuff is important to you…contact your representatives.  We have more power, and more at stake, than we sometimes realize.

Hang tough everybody.  Ratties rock! – celeste

Quite simply I am alive today because I was accepted into the MDX-010 (Ipi at 10mg/KG) trial back in late 2008 when they opened it briefly for compassionate use – and the experimental drug worked.  NED since early 2009 and no other treatments since.  Under the trial the drug was free to me.  Insurance picked up the tab for the frequent bloodwork and scans – which I would have had as a normal course of treatment at Stage IV.  

Rocco, Stage IV in 2005, IPI responder