Dear Vice President Pence,
Two weeks ago, you met with several families and encouraged them to lobby Congress for a federal “right-to-try” law, which would allow people with fatal illnesses to gain access to experimental medicines, even though they are not enrolled in a clinical trial.
Your goal is admirable. Unfortunately, though, you’re peddling false hopes.
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These responses are covering lots of territory, from political cynicism to thalidomide to charlatans.
I may be oversimplifying but can the comments focus on three criteria?
– Currently, the patient has zero hope. So “false hope” is better than no hope.
– The patient has received a death sentence. So “dangerous” is not a consideration.
– More permission is better than no permission, even if the law could ideally be more comprehensive. If pharmaceutical companies refuse to give out the experimental drug, then that is a new problem to be dealt with. At least the Pharma companies can’t point to the lack of a federal law allowing it as their excuse.
I also believe the experimental drugs should be protected by non-disclosure of results. Otherwise, pharmaceutical companies will not take chances with people who might have co-morbidities.
I have worked with big PharmaCos since my career started some 35++ years ago. From an advisor to a independent consultant to a full fledged project director and I have so far seen one common thread with all of them. They are all risk averse.
This new program won’t make one bit if difference in them freeing up their drugs. The ability to claim no liability is shear fiction. As long as there are lawyers there will be lawsuits. Without the final FDA approval to withstand the basic jury scrutiny the risk is FAR greater then the PharmaCos are going to tolerate on anything but a VERY small basis
In my own case I have a VERY rare stage 4 cancer. I know the PharmaCo who has the potential drug I want/need and I know the VP of R&D personally and yet even with the FDA’s approval the PharmaCo is still not willing to allow me access to the drug. Why you might ask? SIMPLE they are right now in their final phase of approval and if they give it to me and somehow something goes wrong even with this new bill stating that the FDA can’t use the data surely they are in jeopardy. Do we really want drugs approved that have been found to have outside spectrum effects that have to be ignored by the FDA?
Lets say they give it to me and 30 others with my cancer and lets say half of us get some horrific unseen before effect? Is it really proper to force the FDA from using that as a criteria to review the drug? Who wins in that situation? I lose because I got the effect the population loses because the effect is now known but can’t be used and even noted to physicians as a possibility and the company loses because it now knows it is going to get a deluge of complaints that will have to be dealt with post approval that might get them a black box warning.
What about the stock holders? They are expecting smooth sailing and now are not aware that Dr Dave and 20 others are having effects that could be a financial issue down the road
Forcing anyone to do anything is not a good thing, PERIOD! The process we currently have is a real PITA the FDA needs to make it much easier to leap thru the hoops but to force the PharmaCos to provide their drugs one for free (they can’t charge since the drugs are unapproved) and two in an uncontrolled manner that only has a negative spin for them is insanity defined. There is NO win for the drug companies if the drug works great they gave it away for free. If the drug in ANY way loses they take the hit and might even lose the drug in its entirety
We too often forget Laetrile and Thalidomide and yet the FDA is based on the Thalidomide debacle of the 1950’s and will never forget it as neither should we.
Yes I wish I could get the drug and I might not be here when it finally arrives but if the drug helps all the others with this instead then I am thankful. Atleast I didn’t get the process messed up for my own sake
Dr Dave
Dr Dave, personal relationships aside I think you’re getting a line of BS from the company. In the industry 34 years I know how the baloney gets made. In reality, if the drug has been submitted for approval to FDA it will win or lose based on the Phase III studies that were performed PRIOR to NDA submission and NOT based your putative experience were you to be given the drug in Phase IIIB. Furthermore, it is unusual for a brand new side effect heretofore unseen to pop up de novo in Phase IIIB if the drug has been properly studied up through NDA submission. Even if there were some de novo side effect, unless it was on a large scale, serious, life threatening or cause a multiplicity of deaths, those events would not influence FDA approval but would be added to the package insert. Even if the drug company wanted to have a “clean” package insert, such adverse events would ultimately come out post approval since the company must periodically update the labeling after launch with new side effect information.
I have ALS (Lou Gehrig’s Disease) and I see desperate patients make poor choices every day. RTT is a state solution to a federal issue. A state may pass an RTT law but if the drug crosses to another state for delivery to the patient, that automatically trips the Interstate Commerce law and makes it a federal matter. Passing a federal version of RTT is still meaningless because giving patients the “right to try” means nothing without a pharmaceutical company willing to provide their product. And even if one does, the patient very rarely has sufficient funding to purchase the drug, and nothing in any of the RTT bills compels the companies to provide their product for free. Then there is the matter of how the drug is administered. Many drugs require injection or transfusion, and almost no patient has the credentials or training to obtain and use the required equipment. That means trying to enlist qualified medical personnel, none of whom would risk their license to practice or being summarily dropped by their malpractice insurance.
Basically, Republicans are using us, the patients, as mere tools to further their objectives of eroding all government regulation of industry. The “jobs” and “economy” excuses weren’t working fast enough so they found this new strategy. FDA, along other regulating agencies, have very important jobs to do to protect consumers from unscrupulous businesses. Read up on the history of the early 20th Century. A great book demonstrating the predations rampant during the time is “The Jungle” by Upton Sinclair.
The current process of drug approval was created in 1962 and definitely requires updating, taking into account 21st Century technology and techniques. But anarchy is never the answer. Refining the process is required, not removing it. I co-founded an advocacy group dedicated to updating the old system using new 21st Century techniques (Hope Now For ALS – http://www.hopenowforals.org). These techniques can remove the need for the placebo group in trials, and analyze data better so that smaller trial patient populations are required. This makes trials more humane. It also makes trials shorter and less-expensive, meaning drugs are delivered to patients more quickly or futile drugs rejected more quickly so that the search for truly effective ones can be continued.
THAT is useful change and a strategy in which FDA has already expressed willingness to use as a secondary endpoint in order to test it. HNFA has had multiple meetings with FDA on this subject. Fix what’s broken, don’t throw the baby out with the bath water.
Correction, there are no issues with interstate commerce laws. It would only apply if the every component of the drug, from active ingredient, to final product were made, packaged and sold in the same state. In reality drugs are sourced from many places and ingredients cross state lines on a regular basis, so there is no violation of Interstate commerce law.
Miracles happen everyday. Who are you to sentence someone to death?
Laertril was advertised as a “miracle” by unscrupulous clinics in Mexico. And only by the resistance of FDA “bureaucrats” who were placed under enormous pressure by pharmaceutical companies, was Thalidomide kept out of the US.
“Right to Try” is an ethically thorny issue, and one that may not have a clean and simple resolution. I work with chronic pain patients every day in social media. Many of them would try ANYTHING to get their pain under control. And many of them are victimized by their own desperation every day, manipulated by quacks and charlatans in the so-called “alternative medicine” movement.
The FDA drug approval process likely does need to be shortened and made more efficient. But the devil is in the details.
Dear Jeanne,
Thanks for the note. I’m not sentencing anyone to death. I’m simply trying to point out that the bills – and the effort – are misguided. Yes, the FDA must approve requests, but does so nearly all of the time and has attempted to simplify the process. Companies remain ultimate arbiters. You can read the bills if you wish; the links are there. This is a complicated issue and patients do need consideration, but blaming the FDA appears to be a straw man.
Regards
ed at pharmalot