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The Pills of Last Resort
November 1, 2013 11:44 AM   Subscribe

How Dying Patients Get Access to Experimental Drugs
posted by Blazecock Pileon (16 comments total) 16 users marked this as a favorite

 
Broom, too, survived for years beyond what doctors first told her she could expect. She wasn’t supposed to see Amelia’s first birthday, and instead she was able to celebrate her fourth.

Fuck.
posted by 256 at 12:27 PM on November 1, 2013 [1 favorite]


Fascinating article. I will say without having read it all that when my mom was dying from breast cancer her doctors put her on some newer experimental drugs. I don't know if they helped or hurt her, but I do know she was proud to be able to help other people not suffer as she did. Certainly helped her keep her pride as things went bad.
posted by Carillon at 12:32 PM on November 1, 2013 [3 favorites]


This blog post is from a friend of mine who won the Crixivan "compassionate use" lottery in 1996. He was weeks, if not days away from death. And uh, he wrote this blog post last weekend.
posted by roomthreeseventeen at 12:41 PM on November 1, 2013 [14 favorites]


Doesn't look like the article mentions it but the FDA still has to authorize a patient's access arrangements to an investigational drug even if the pharma approves.
"In order for a patient to gain access to an investigational drug outside of a clinical trial, the patient must have a serious or immediately life-threatening disease or condition and no comparable or satisfactory therapeutic alternatives. Additionally, the drug manufacturer and the patient’s doctor must make special arrangements to obtain the drug for the patient. These arrangements must be authorized by the FDA. These safeguards are in place to avoid exposing patients to unnecessary risks."
The feds have a site set up for patients and ClinicalTrials.gov has a list of expanded access studies.
posted by qi at 12:41 PM on November 1, 2013 [1 favorite]


Broom had advanced-stage lung cancer. “I was told nothing could be done to cure the cancer, but that various treatments could give me time,” she recalled. Less than 1 percent of patients live more than five years.

They told my wife something very similar. By the time she was ready to try the experimental treatments (which was only a matter of weeks later), she was "too sick". Nobody wanted lost cause patients in their study because when they died, they'd make their new drugs look less effective. They were probably right, I don't think there was anything that could have been done for my wife at that point, honestly, but it still sort of sucks.

You read a lot of stories about the cancer patient who against all odds made it five years or more, or is in complete remission. You don't hear so many about the cancer patient who was diagnosed in November, given 3-5 years, with that estimate revised to 6-12 months in November, who then dies on the 4th of February. Nobody tells that story.
posted by tylerkaraszewski at 12:42 PM on November 1, 2013 [22 favorites]


At the risk of being a heartless scrooge, I have a problem with the messiness of the article. The first story is indeed a classic case for a Treatment IND under the FDA's Expanded Access program, but the next story, about the author's father, is a completely different issue. It was an approved drug, which means the father's physician was within his rights and responsibilities as a physician to prescribe it if he thought it would be of benefit. The issue was whether the insurance company would cover the cost. The question of charging for investigational drugs, including expanded access programs, is already covered in the FDA regs. The article then jumps to the approval of AZT, treating Parallel Track (for HIV/AIDS) and Group C (for cancer) expedited approvals of new drugs as something miraculous, rather than decades old programs.

I get that this is supposed to be a piece that pulls at the heartstrings, but I'm not sure what point it's trying to make. Better patient advocacy and education of physicians that these options exist, I guess? I don't think anyone is against that, but bringing up "the Abigail Alliance, which sued the F.D.A., arguing for a constitutional right to early-phase drugs" and telling scare stories about patients denied access to experimental treatments turning to quacks gives me a strong whiff of therapeutic misconception. Clinical trials are designed to find out whether a certain treatment works for a certain condition, and until they've been completed, that answer is not known. Getting seriously ill people access to investigational drugs may seem like getting them access to the newest and most cutting edge treatments, but they are really just getting the latest unproven attempts at treatments, not a panacea.
posted by Panjandrum at 12:56 PM on November 1, 2013 [10 favorites]


These safeguards are in place to avoid exposing patients to unnecessary risks.

This is the knife edge between thalidomide and AZT. Thalidomide is a decent antiemetic and anxiolytic sedative medication, that (in general) had few side effects. It seemed perfect.

Right up until pregnant women started taking it. A few months later, that's when everyone realized it was also a fairly potent tertatogenic agent.

The backlash against thalidomide, esp. in the US and Canada, was huge. And that's when the FDA basically drew a bright line on testing. You will test, and you will test carefully, before we're going to let a drug get anywhere near general release, never mind over the counter.

Time passes. HIV skips into humans, gets over the US, and the AIDS epidemic is raging. In the mid 1980s, AIDS is a death sentence. In 1984, research discovered that azidothymidine (AZT) is effective in at least slowing HIV in the lab. Trials commenced.

But people were dying. The normal trials process took years. That was too long. AIDS Advocacy organization swung into action, and the FDA approved a trial of a fast track trial process. (Yes, this was a trial of a trial.) AZT was in general approval for the treatment of AIDS in 25 months. The FDA Fast Track Process is formally adopted in 1998. So, yes, the first Fast Track trial was a double success -- it resulted in AZT and the Fast Track Process.

So, we try to find the balance. We don't want another thalidomide, where a drug with spectacularly bad side effects against a group of individuals slips out without us knowing that, but we don't want drugs that save lives bottled up in years long approval processes.

Aside: Years later, we find that thalidomide is effective in the treatment of erythema nodosum leprosum, a painful skin condition associated with leprosy. It clearly lowered TNF Alpha factors, which implied it could be useful in cancer and sure enough, it's very good at attacking multiple myeloma. It may be useful agains AIDS. It's received FDA approval for use again ENL….

…but. If you're female, you *must not* become pregnant while taking it.

Sometimes, there are no easy answers.
posted by eriko at 1:22 PM on November 1, 2013 [27 favorites]


I get that this is supposed to be a piece that pulls at the heartstrings

This is not what I took away from this, but I appreciate the other parts of your comment.
posted by Blazecock Pileon at 1:28 PM on November 1, 2013


Thanks Eriko; just thanks
posted by NiteMayr at 1:39 PM on November 1, 2013


If doctors simply let people take untested medicines without going through all the clinical trials, drug companies would most likely never get anyone to enroll in them, never get the data on safety and efficacy for F.D.A. approval and never pass the gateway to big sales.

So this is a real dilemma.

On the one hand, an individual's life - a child's fourth birthday celebrated instead of none. On the other hand, everyone has a sob story and with no rigorous trials, we lose some of the biggest advantages of modern medicine over folk remedies. It's like a perverse tragedy of the commons, with human lives instead of grazing cows, and I don't have an answer.

But the current system is basically "who do you know that is well connected?" which boils down to money and social class. Yet again. Might as well just farm out drug trials to the third world at that point. (Oh wait, they already did.)
posted by RedOrGreen at 1:42 PM on November 1, 2013


Interesting read. My dear mother passed on Labour Day this year at the age of 67 after being diagnosed with Stage IV pancreatic cancer on July 17th. I knew nothing of how brutal this cancer is until it entered my life in such a personal way. I was with her everyday but one from diagnosis to passing. I'd visit with her each day and then go back in the evening and hang out until she fell asleep. Then I'd come home and research every god damn clinical trial available worldwide. I had a close Lebanese internal medicine friend of mine at a major hospital in the US school me on how to read these things. I tried and I tried and I tried; it didn't matter. My mum's cancer had spread from her pancreas to her liver to her ovary. She had such bad Fibromyalgia (a crinkle in her sheets would be painful) that there was no way she could handle the catho-port required for FOLFIRINOX, which is the approved drug with the best success rate, and even then we wouldn't be talking years. She knew her body well and knew that this four drug 48 hour infusion per week cocktail would have killed her immediately. Our plan was for her to try an IV of Gemcitabine, which is much weaker and only half an hour a week. We intended to see how that went and then introduce nab-Paclitaxel, which if I'm remembering correctly, was a way to break the cocoons down that the cancer cells form, so the chemo could actually get where it needed. Everything went fuckin' south the day after her first chemo on August the 8th. She was never the same physically after that. We had to take her to Emergency because of a fever. She was discharged about nine hours later, only to be admitted again the very next day for a week. After that, I took her home, where she wanted to be so so badly, and she died two and a half weeks later with me in bed beside her; no tubes, no wires, no nothing. Fuck, I'm crying. Anyway, my mother dealt with this whole experience with grace that I can only hope to have when my time comes. She apologized to me for what she called "giving up" because she didn't want to continue with chemo after the first one. I just told her that she was the bravest woman I knew and she was right to make that decision after her recent experience.

She wanted to remain as with it as possible for as long as possible, so we made various batches of THC tincture and they really helped her with sleep, nausea, and eating (until she couldn't anymore). She managed to stay off the opiates until the final two weeks, and even then, she was taking small doses.

My mother never once asked "Why me?". She held court daily with the two or three friends and family members I'd schedule in each day, aside from the closest circle her were around all the time. She made us laugh our fuckin' asses off; any filter that she once had no longer existed, and she said to me, "Anything I think, I now say". It was absolutely beautiful and nothing was left unsaid. Nothing. If it's somehow possible to push the pain and the awfulness aside, it was an absolutely amazing experience, and I learned more about my mother in that month and a half than I could ever have imagined.

My mum was a tutor for learning disabled children and the woman refused to give it up. It provided a sense of normalcy for her, even though she knew she was not long for this world. She tutored until four weeks before her passing, even if it was on her bed. Some of these kids were with her for seven or eight years, and it's them I feel for most - I can't tell you how lost they are without her. I get emails from their parents all the time. I'm amazed at how much apart of their lives she was, but it fuckin' kills me that they now don't have her.

My mother also left six figures for me to build a girls' school in Ethiopia. It's been an amazing focus for me these past couple months (I got on it immediately). Everything is pretty much all set, I'm just waiting for probate to finish. There will be an ECE class, grades 4 through 8 classrooms, latrines, teacher training, all supplies, and salaries for three years, before the Ministry of Education takes over my mum's school in 2017. What an amazing legacy this woman left.

I think I'll just leave you with this painting of my mum that I'd never seen until she died. My girlfriend found it in her locker; it's from her 1964 graduation photo at the age of 18, and I had it framed the other day. I don't mind sharing this experience, so if you're interested, here are three albums that I photographed from diagnosis to passing and then her funeral and subsequent celebration of her life. Two of the albums have more than one page.

I totally appreciate that chemo and various other drugs work really well for a lot of cancers, but in my mum's case, I am positive the right decision was made and I'm so grateful her suffering was minimal.
posted by gman at 2:06 PM on November 1, 2013 [31 favorites]


Dallas Buyers Club is scheduled to be released today, and deals directly with this issue.
posted by Brian B. at 3:17 PM on November 1, 2013


Getting seriously ill people access to investigational drugs may seem like getting them access to the newest and most cutting edge treatments, but they are really just getting the latest unproven attempts at treatments, not a panacea.

There have been several studies of investigational therapies entering phase III clinical trials. About 55 percent turn out to be harmful or ineffective. An example is an early trial of prophylaxis for PCP in HIV patients that showed higher mortality in the active treatment compared to placebo. Unfortunately, many MDs were pushing the prophylaxis prior to the trial, and many people died unnecessarily as a result. Further, most effective drugs have relatively small effects. The chances that an early investigational drug for a late stage fatal disease will provide some major benefit are vanishingly small. A few anecdotal stories of "miracle cures" from such drugs are just as misleading as the smarmy pitches of the quacks who sell any other untested product.
posted by Mental Wimp at 3:19 PM on November 1, 2013 [2 favorites]



If doctors simply let people take untested medicines without going through all the clinical trials, drug companies would most likely never get anyone to enroll in them, never get the data on safety and efficacy for F.D.A. approval and never pass the gateway to big sales.


I've made experimental cancer treatments that are so far removed from the start of clinical trials that we were still testing them on purified enzymes. We still had people asking if they could come in (to a chemistry lab not a hospital) to get treatment because they had no options left. People with nothing to lose will risk anything for a chance.
posted by koolkat at 3:57 PM on November 1, 2013 [2 favorites]


This is a bit of a tangent, but I think it's worth pointing out the startling (to me) fact that up to 60-80% of people with advanced (i.e. metastatic) cancer don't always realize that chemotherapy is not intended as a cure in their case, that it's meant to keep the disease at bay and let them have a few more [weeks/months/years] to live, all the while wondering about the possibility of the cancer coming back when they least expect it. (This article from the New England Journal of Medicine is where I get the numbers from.)

Please, folks, if you or your loved ones are going to even consider chemotherapy, make sure to take the time to discuss the ultimate goal of it with your oncologist. There are certainly lots of people who go through chemo with flying colours and benefit a lot, and the medications that we have now to control the nasty side effects are excellent, but the process is not for everyone.
posted by greatgefilte at 4:02 PM on November 1, 2013 [2 favorites]


People just do not accept death in this country. It's un-American. It's a huge problem, both in terms of patients or their families unwilling to accept "99% fatality within six months" as a realistic threat to their continued existence, and an even bigger one in terms of doctors dancing around the issue. I have hundreds of stories about how a colleague delayed or flat out refused to sit down with a patient and even just say the c word, much less metastatic, terminal, fatal...or God forbid, I suggest you get your affairs in order.

Generally we all try to have that conversation once, late at night in the middle of the intern year, with the best of intentions, and you would think from the reaction someone had suggested we cart grandma down to the glue factory. Or, after saying the odds are pretty bad, 100 to 1, but you can't say for sure....everyone thinks they're the 1%

Luckily at my hospital we have death panels. I mean, dementors. I mean, mortality liason intervention committees. I don't remember their real name. But after extensive specialized training that likely involved some truly excruciatingly bad VHS tapes from renowned self help gurus, they get to have that conversation instead of me.

I was going to respond to this when it first came up, but I'm glad I waited. My initial reaction was that public policy should never be decided because of the chances of a miracle happening. But if you told someone, as in the story above, that since they have stage IV pancreatic cancer we're just going to send you home and give you enough medication to make you comfortable, there would be riots in the street. So that's what we're left with, doctors giving chemotherapy regimes that could not possibly extend lifespan, performing surgeries that don't have a chance in hell of making that back pain go away, ordering labs and radiological imaging that won't alter the treatment plan....because, well, it's easier than thinking; and certainly is less likely to lead to a malpractice suit. Despite, y'know, literally constituting malpractice.
posted by hobo gitano de queretaro at 11:58 PM on November 1, 2013 [4 favorites]


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