Minnesota family sues company for muscular dystrophy drug

A Minnesota family has filed a lawsuit seeking to force a drug company to let their son have access to an experimental drug for a terminal form of muscular dystrophy.

[Libbie-Lou]

This article says absolutely NOTHING. Why wasn't the child a candidate for the trial? Trials are very specific for a reason. Does the family expect requirements to be waived for everyone, or just their son? What's the point in the researching if you don't have good control in it?

[miesl]

Well, thank you very much for causing the rest of us problems.

If this puts that company out of business, what are all the people who might have benefitted from PTC124 going to do... oh wait... die.

I'm very sad that PTC124 isn't going to help my husband (not the right mutation), but I'm not suing PTC.

This is incredibly irresponsible. Clinical trials have exclusion criteria for a reason. There are a number of clinical trials that *could* help certain people, but they are not eligible for one reason or another. It's for two extremely important reasons. 1) To protect the patient. 2) To protect the company. If less than ideal participants are recruited it will NOT help the results of the trial. Unsucessful trials lead to dropped drugs that help no one.

While it is incredibly sad that this boy will not be helped - it is not that company's fault. The company wants this drug to succeed - and so did this family, up until it wouldn't benefit them personally.

[mom of boys with BMD]

As a mother of boys with MD (who also does alot of promoting awareness and fundraising ). I wish them all the luck. That is just horrible to hear he was excluded from trials

[Small Business owner]

miesl wrote:

Well, thank you very much for causing the rest of us problems.
If this puts that company out of business, what are all the people who might have benefitted from PTC124 going to do... oh wait... die.
I'm very sad that PTC124 isn't going to help my husband (not the right mutation), but I'm not suing PTC.
This is incredibly irresponsible. Clinical trials have exclusion criteria for a reason. There are a number of clinical trials that *could* help certain people, but they are not eligible for one reason or another. It's for two extremely important reasons. 1) To protect the patient. 2) To protect the company. If less than ideal participants are recruited it will NOT help the results of the trial. Unsucessful trials lead to dropped drugs that help no one.
While it is incredibly sad that this boy will not be helped - it is not that company's fault. The company wants this drug to succeed - and so did this family, up until it wouldn't benefit them personally.

She is not asking for her son to be part of the trial, just to have access to the drug. If the drug company is excluding him solely on the basis of defining a group for a favorable outcome(as they usually do) and they had an agreement that they would get the drug regardless of the trial acceptance or not, shame on them.

[Andy]

And people wonder why healthcare is so costly?

[Kacy]

Andy wrote:

And people wonder why healthcare is so costly?

Healthcare is costly because insurance companies and drug companies and lobbyists and politicians are all in bed together, NOT because this family filed what very well might be a legitimate law suit.

[Paulie]

Small Business owner wrote:

<quoted text>She is not asking for her son to be part of the trial, just to have access to the drug. If the drug company is excluding him solely on the basis of defining a group for a favorable outcome(as they usually do) and they had an agreement that they would get the drug regardless of the trial acceptance or not, shame on them.

According to the article, the family was told they would have access to the drug. I can only imagine that means they were told they WOULD be included in the trial.

FTA:
"He and his parents sued in federal court in New Jersey on Wednesday, alleging that the company had told them earlier that the he would get access to the drug."

[miesl]

The primary study outcome is:

To determine the effect of PTC124 on ambulation in subjects with nonsense-mutation-mediated DMD/BMD (as assessed by changes in the distance walked during a 6-minute walk test)

The boy in the article is confined to a wheelchair and cannot walk. Therefore, he is excluded. If at some point in the past he could walk and was told he was eligible... so be it. That does not change that he is no longer eligible. Anyone with ANY exposure to clinical trials knows that these things happen. My husband has been eligible for trials at some point that he is no longer eligible for - that happens in progressive illnesses.

It is also in Phase 2. EAP (expanded access to those who are not in the current study) doesn't usually happen until Phase 3. Phase 2 trials are SAFETY and effectiveness.

This isn't a legitimate lawsuit. It's the way pharma HAS to work get get drugs to market. This drug is already fast tracked due to it being a orphan drug.

[ottermann]

Small Business owner wrote:

<quoted text>She is not asking for her son to be part of the trial, just to have access to the drug. If the drug company is excluding him solely on the basis of defining a group for a favorable outcome(as they usually do) and they had an agreement that they would get the drug regardless of the trial acceptance or not, shame on them.

If the drug has not been approved by the FDA, the company can not, by law, give her son access to the drug without violating several federal laws.
The only way to gain access to experimental drugs is through a clinical trial.
There may be mitigating factors as to why he isn't being admitted to the trial. Maybe the drug is know to be especially harmful to people with a weak heart, and the son has a weak heart. If something like that is the case, then the drug may shorten his life rather than extend it.
And, you just know, deep down, that if the company is forced to let the son into the trail and the drug ends up killing him, his mother will sue for wrongful death.

[miesl]

This is the study:

http://www.clinicaltrials.gov/ct2/show/NCT005...

There are a LOT of exclusionary criteria - many of which are things that can change in a mattter of a few days of the Day 1 study visit.

[Andy]

Kacy wrote:

<quoted text>
Healthcare is costly because insurance companies and drug companies and lobbyists and politicians are all in bed together, NOT because this family filed what very well might be a legitimate law suit.

Shhhh...don't turn around. Big Brother is watching you.

[Joey]

Kacy wrote:

<quoted text>
Healthcare is costly because insurance companies and drug companies and lobbyists and politicians are all in bed together, NOT because this family filed what very well might be a legitimate law suit.

Wow Kacy, that's an intelligent answer. Quit giving a rubber stamp to people with "Everyone With More Money Than Me Is Out To Get Me Syndrome". I think if you were a doctor paying malpractice insurance, you'd have a different opinion about the costs of health care.

[Kacy]

Joey wrote:

<quoted text>
Wow Kacy, that's an intelligent answer. Quit giving a rubber stamp to people with "Everyone With More Money Than Me Is Out To Get Me Syndrome". I think if you were a doctor paying malpractice insurance, you'd have a different opinion about the costs of health care.

Wow, Joey, you aren't as smart as you think. I worked in health law for many years and actually defended malpractice suits, so I know what I'm talking about. I understand that there are patients out there who don't understand the difference between malpractice and a bad outcome. But that is NOT why our healthcare costs so much in this country. Contrary to what President Bush might say, it's just not the case.

[fishinmusician]

Kacy,which lawsuits were you involved in and where did your salary come from.I'm ignorant and cant understand how lawyer fees do not effect the price of any service/product that is constantly involved in needless lawsuits.Since you know what your talking about by your own admission I'd appreciate any info you can share with us

[Pragmatist]

ottermann wrote:

<quoted text>
If the drug has not been approved by the FDA, the company can not, by law, give her son access to the drug without violating several federal laws.
The only way to gain access to experimental drugs is through a clinical trial.
There may be mitigating factors as to why he isn't being admitted to the trial. Maybe the drug is know to be especially harmful to people with a weak heart, and the son has a weak heart. If something like that is the case, then the drug may shorten his life rather than extend it.
And, you just know, deep down, that if the company is forced to let the son into the trail and the drug ends up killing him, his mother will sue for wrongful death.

Yeah, because the AMA and CDC have been so ethical in the past.(see http://en.wikipedia.org/wiki/Tuskegee_Study_o... ) AIDS patients are/were allowed access to drugs that had not gone through studies because their disease was terminal. Is it any different for this boy? Bio-medical ethics is a term much like military intelligence, or jumbo shrimp, all are oxy-morons.

[Kacy]

fishinmusician wrote:

Kacy,which lawsuits were you involved in and where did your salary come from.I'm ignorant and cant understand how lawyer fees do not effect the price of any service/product that is constantly involved in needless lawsuits.Since you know what your talking about by your own admission I'd appreciate any info you can share with us

I'm not a lawyer, first of all, but a legal professional who has worked in health law. Yes, it costs a fair amount for a hospital or health plan to have a good legal team defend their lawsuits. This is a cost they include in their budget.

President Bush and a lot of Republicans have gone out of their way to accuse those who file suits against providers or hospitals or health plans as being the sole reason health care costs are what they are, all the while failing to mention the drug companies, insurance companies, and the fact that many Americans have no insurance and therefore use the ER as their clinic with no ability or intention of paying for the services they receive there.

Yes, there certainly are and always have been people who file frivilous suits, many of which are dismissed or settled for minimal amounts. Generally if a case goes to trial, there is some merit. Not always, there will always be a few that slip through, but generally speaking, yes.