The story me cringe. I disagree with the company's choice. There has to be a way to get full from the FDA for immunity from the results of the treatment if it's negative. I just can't believe how the company and the FDA can just sit there and not allow some bending of rules to give this kid a chance to live. Your thoughts?
As a staunch believer in the non aggression principle one of the fundamentals of libertarian belief, I find it to be morally repugnant that we would expect a company to do anything that they do not wish to so. Property rights, being the most sacrosanct of rights, must be protected at all points even if that protection comes at the cost of a rivers and rivers of blood vomited from the mouths of a thousands of 7 year olds.
Whoa. Dunno if sarcastic but I agree to the core idea. I find ironic humor in forcing people to donate. And consider the circumstances: they're a reasonably small company and the drug costs a lot. It reminds me of the LOFs asking Les for a Lin buyout. I think they'd go farther if they'd crowdfund the drug.
Wow there, don't compare a midget Asian guy who speaks with a heavy communist accent to a freedom born American drug that could save millions of true patriots. The lives of all the Asians in the world wouldn't worth this drug. Amirite dude? High five.
most people that claim to oppose drug prohibition are perfectly ok with the FDA performing that same function the FDA should at best be relegated to an advisory agency with no approval power <iframe width="560" height="315" src="//www.youtube.com/embed/Qu4C1yi_FKw?start=63" frameborder="0" allowfullscreen></iframe>
It's an experimental drug, I don't believe the hypothetical chance of saving an individual life obligates the company legally or even morally; and I don't like that their work up to this point to try and save multiple future lives somehow opens them up to additional charges of cold-bloodedness. There are probably multiple people dying of this condition, and multiple companies working on treatments that they either haven't disclose or that the father simply hasn't heard of or been ale o contact. Whatever anger or disgust about the company's testing protocols should probably be apportioned across all those other actors and scenarios.
Of course the kid's parents are desperate, who wouldn't be. but I understand the company's position. 1. This kid is mostly going to die with our without the drug. If the company give him the drugs and he ends up getting kidney & heart failure as expected, than the company is required report to the FDA that a patient suffered a fatal kidney & heart failure while taking this drug,, this will obviously not help with their FDA approval process. 2. You can't just expect a company to shell out $50K + much more in man hours just to save your kid. This is not some big pharma company, it's a 50 employee company. 3. People like to say you can't put a price on a human life. That's complete BS. There's a price tag associated with everyone's life, probably billions of people have died because they didn't have access to $50K. Millions of people will die this year alone because they didn't have access to $100 let alone $50K (3.4 million people die every year because of drinking from dirty water, 3 million die every year because they can't afford cheap & common immunizations like polio, measles etc)
Thanks Obama.... I side with the company. If it was a no-lose proposition, then I'd form the opinion that the company was cold and heartless (yet still perfectly justified), but there's a lot of risks that could torpedo years of research. The president can't just say outright, "I know we built this thing up as some magic bullet, but your kid could still die". Just hearing the kid's story though... If he were 80, I'd wonder if the family was ruining the patient's quality of life just for a few more moments, but the way he fights for a life ahead of him...What have I done lately that would have that resolve?
I'm surprised. I was expecting a lot more "THOSE COLD-BLOODED ASSHOLES WON'T HELP A KID" but everybody's pleasantly rational.
I guess I shouldn't be surprised that so many want to blame the FDA when it says in the very article that the FDA allows for compassionate care exemption. Anyway as another poster said if you want to go back to the days of snake oil salesmen then do away with the FDA regulatory power.
I side with the company but I think the "it's for all or for none" isn't necessary. There can be a way for it to be for some (lottery for example with limited # of available per year).
This is where I find the company's argument rather problematic. There are many ways they could address this issue without giving out their drug to everyone. In this case given the possibility of a still long and productive life for a 7 year old they could give the drug to someone in their 80's. I understand the company's reasons for not giving the drug but I am not going to side with them. This is a difficult issue but it seems to me there are ways to still give this drug while addressing the company's concerns.
So if it's about not being able to afford the philanthropy, if the family paid the $50K, would they get it? Maybe? Tough for the kid...he's a fighter, but definitely dealt 2-7 off in life.
If the burden is the organizational distraction of setting up a system to evaluate cases for compassionate use, then I think a lottery might not really fix it. If they take the time to set up the organization, they may as well run everyone through it. And, they might have to run everyone through it just to see who qualifies for this lottery. And then, after all that, they'll still get blasted by allies of patients who lost in the lottery. So, I'm not sure I believe this is a viable solution. Here's my suggestion: Ask a Big Pharma company to run all the paperwork for your compassionate use cases. You may or may not have to pay them for the work. Ask a charity to provide the funds to help pay for the process and to pay for the drug. Keep your own company focused on getting to market asap. I'm not going in for the property is sacrosanct bs, but I do see the logic that the greater good can be served by staying focused on getting the product to market. But, you don't want to see these people die in the interim. It doesn't all have to be on these 50 people to solve the problem; there are a lot of people who are in a position to be a part of the solution. There might be a space here where a nonprofit can be focused on identifying and funding compassionate use cases (maybe funded by member pharmaceutical companies in proportion to their size).
If there is an issue with companies afraid of the FDA derailing the approval of the drug due to compassionate cases where the drug didn't help, then the FDA is at fault here. There should be a general clause that any negative results from a "compassionate" case cannot be used against the company or the drug. It's a win-win. Insurance should cover this, PERIOD. There is no way in hell an experimental drug would cost more than the amount being spent on the treatments this poor kid is receiving just to prolong the inevitable. But unfortunately since insurance won't cover it, if the costs for treatment ($50,000 up-front cost + $100,000 salary for a year's worth of manpower) are payable by the family, the company should try to accommodate if FDA changed their rule. No, the article says that FDA allows use of the experimental drugs but still will hold negative results against the drug when evaluating it for final approval. FDA needs to change their stance on using "compassionate case" data against the drug.
Exactly. I'm not blaming the company but disagree with their choice. It's not their fault that they have hurdles to jump that forces them to say 'no'. There should be a change to the process and the FDA should be there to help make it happen.
Anybody remember Vioxx - how everybody was blasting the FDA and Merck for not doing enough to make sure it was safe? Everybody was incensed that there wasn't more stringent regulation. This is the other side of that coin. Damned if you do, damned if you don't.