I recently came upon a petition, Its a petition asking compassionate access to a new drug manufactured Bristol-Myers Squibb I can't truly explain it with justice so please

Visit the video appeal:http://www.youtube.com/watch?v=olwDT7NPSsM&feature=share

Please sign the petition

http://www.change.org/petitions/bristol-myers-squibb-help-save-our-dad

And please pass it on.

Thank you.

Really no one? Sigh.

While certainly an emotional appeal, I'm more inclined to trust the judgment of the doctors involved with the clinical trials of BMS-936558. I looked up a bit of information on the drug, and it seems that it's only just finished an extended phase 1 trial (initial trial data was only released earlier this month), which is still quite early in the development process (data at this point is primarily safety data, along with a small amount of extremely preliminary efficacy data). Depending on the specifics of the patient in question, the prognosis for giving him the drug could be anything from "we can't make any accurate predictions on whether it would help or hurt" (which would make giving him the drug ethically questionable), to possibly an extreme of "given the adverse event profile of the drug and complications the patient is already suffering, there's a good chance giving him the drug would actually shorten his life" (in which case giving him the drug would be downright unethical).

So while I can understand that a terminal cancer patient and his family may feel ready to try absolutely anything, and will latch onto even the slimmest of hopes, I would ultimately defer to the judgment of the people who have been working on the drug's clinical trial for years.

(Disclosure: I work in the pharmaceutical industry in drug development, so take from that what you will).

That's more or less my thought. I do think that they should have a compassion program, however, there is a significant amount of red tape. Sometimes it makes sense other times it makes me want to roll my eyes. I remember back in college that just doing a cheek swab to look at the cells required the human experimentation panel to sign off on the idea. Granted it was mostly a rubber stamp deal, but there is a fair amount of bureaucracy.

Unfortunately, there's always going to be the trade off between releasing medication too soon and waiting too long. I definitely feel for the folks that need it now, but there have been some notable cases of medications getting to market and having to be yanked because they weren't safe.

And yes, Phase 1, is quite early in the process. IIRC there are usually 3 phases and at this stage I would be surprised if they even know if it works as intended, ignoring side effects and risks.

But what if the patient is informed of the risks and still willing to get the drugs?

It's tough to make a call on whether BMS should have a compassionate care program without knowing the specifics of what's required for such a program, as well as how the specifics of such a program apply to different therapeutic areas. And while all the bureaucracy can seem excessive at times (and trust me, I have plenty of criticism for the FDA and other regulatory bodies), the vast majority of such requirements are there in response to the numerous people (the scum of the earth) all too ready to peddle snake oil to desperate people like the ones behind this petition.

And you're correct about there typically being three phases to clinical trials. Phase 1 typically tests the safety of the drug in healthy volunteers. Phase 2a tests the efficacy of the drug on sick patients to determine optimum dosing regiments. Phase 2b tests the efficacy of the drug on a medium-sized group of sick patients at the dosages arrived at in phase 2a. And finally phase 3 tests the drug on a large group of patients (typically in a double-blind study with either a placebo control or against the current gold standard in therapy), to both definitively determine efficacy as well as to detect any rare adverse events and any notable difference between different patient populations. Oncology trials can be a bit different, though, as most oncology drugs are highly toxic so giving them to healthy volunteers would be highly unethical. In such cases you can see a phase 1 and phase 2 trial basically rolled together into an extended phase 1 trial (which basically tests safety, dosing, and preliminary efficacy), and after that the drug moves directly to a phase 3 trial. Informed consent is a critical part of patients taking part in any kind of drug trial. However, at times a lack of data or a patient's emotional state make informed consent pretty much impossible. Ultimately the doctors have to do what they consider ethical, even if a patient wants them to do otherwise.

I tend to agree that it really depends on a number of factors, but it seems a bit risky to allow something in Phase 1 or barely out of Phase 1 trials to be used in such a way. I was more thinking of medications that were closer to being released being a part of the program rather than things that are still basically Phase 1.

Cancer patients and other people that are terminally ill are more or less the last ones that ought to be making those sorts of decisions even without corporate misdeeds there is a certain amount of duress inherent in the situation. It's unfortunate, but for many of these conditions if they started to open them up for compassionate care could lead to undermining the whole point of doing the drug trials in the first place as you could just do the analysis after the fact.

Sucks, if you're in the position to need those drugs, but things can and do get pulled even after being approved. I had one of the medications I was taking pulled, which was a shame because I still haven't found anything that worked as well and I personally didn't have any problems with it.