Why would a cancer drug not work in children?

For more see 'list of neglected diseases' here; http://www.who.int/neglected_diseases/diseases/en/ These are neglected mainly because there is no financial incentive to invest money into new drugs.

It is not at all 'highly unethical' to trial drugs on children. It is the only way you know they work! I think it unethical to treat children with drugs without trials! The problem is the drug companies are unlikely to trial a drug if there is little financial benefit. 7% of childhood cancer is only 100 children a year - how many of this will let their kids have an experimental drug ? There are very many other problems in recruiting children for trial drug studies. Also there are tolerability concerns and impact to the company should the trials go wrong

I will give you one example :

Gefitinib is a new cancer drug licenced for lung cancer in adults. Lung cancer you dont get in children. If you are the one in a million get cancer lung cancer as a child it is not the same type of cancer in adults therefore drugs like gefitinb do not work for it.

The problem is that gefitinib, for example, may be beneficial in other childhood cancer. Children get a cancer called neuroblastoma (7% of childhood cancer) which it may work for because it also has the same EGFR mutation gefitinib targets. Under the waivered law an oncologist cannot use for children until the pharmaceutical industry trials. The phamaceutical giants will not do this unless there is a good business case, the thoughts and whims of a paediatric oncologist is not enough to justify the excessive price needed for clinical trials! This often means that the pharmaceutical industry will not trial new cancer drugs in children and under the law they do not have to. Unless it is a good drug (which many of the new cancer drugs are not!) they have no financial incentive to do it. When they have a drug that is poor or mediocre they know they will not get NICE approval for excessive price that would justify the investment they must make.

It is not at all 'highly unethical' to trial drugs on children. It is the only way you know they work! I think it unethical to treat children with drugs without trials! The problem is the drug companies are unlikely to trial a drug if there is little financial benefit. 7% of childhood cancer is only 100 children a year - how many of this will let their kids have an experimental drug ? There are very many other problems in recruiting children for trial drug studies. Also there are tolerability concerns and impact to the company should the trials go wrong.

I have attached the list to show you the different sorts of cancer you get in children which is completely different from adults.

http://www.cancer.net/cancer-types/childhood-cancer

What exactly is your proposed solution ?

As kyalimers mentioned above childhood cancers are very different to those seen in adults. Genetically adults tend to suffer from multi mutation caused cancers where as childhood tends to involve other models. If you want to understand this more look into genetic models such as the two hit hypothesis.
Also for example, when looking at treatment for brain tumours in childhood (these have highest mortality) the treatment can stop neural development often leaving the child disabled. Therefore this is ethically an area drug companies don't want to get involved in.

Thank you. That really helped. Do you have a source for that discussion about gefitinib? I'd like to read more about it and perhaps use it as an example in my essay, if you don't mind.

As for my proposed solution, I'm using this BBC article as a starting point (http://www.bbc.co.uk/news/health-26118914) which explains the ICR's proposal that the EU waiver should be granted to drug companies only when it can be said that the drug would not work in children, but not where the cancer itself does not occur in children (because, as you said, the gene mutations may be the same in different cancers, so the drug might still actually be effective). What do you think about that? How can a drug company know that its drug won't work on children if it hasn't tested it on them (surely the point of a clinical trial is to determine whether the drug will work!)?

Sorry I can't find the link, I think it was a guardian article.

It is a difficult topic. I don't think you can legislate to force trials for childhood cancer. It may cause the industry not to release the treatment for adults if the law will make them test for all childhood cancers containing similar mutations.

The bit I don't follow is why it seems like a remotely good idea to almost double the amount of testing required (and hence almost double the costs of development), when pharmaceutical profit margins are already sodding tight. Prices will have to rise significantly to cover the costs of licensing for a very small number of extra prescriptions. Its madness.

From personal experience parents who have children with currently incurable cancers will travel all over the world in order to get their children on drugs trials for solutions. Wouldn't you? If the options were see your child suffer for a few years and then die, or take a risk and trial a new drug, what would you do?

That's a good point. Where has that requirement come from?

That's a good point. Where has that requirement come from?

1. The main reason is ethics. Several other points come off this and contribute to it but in essence, it is not ethical to trial unknown drugs on children (similar with pregnant women).

2. Paediatric pathology and physiology is often different to that in adults as well so you can not directly translate results from adults to children.

3. Add to this the fact that anti-cancer drugs are highly damaging to the body. They quite literally kill cells. For this reason, you wouldn't want to put children through the unknown side-effects of various drugs.

4. Clinical trials involve using placebo drugs. It is thought to be increasingly negligent to not offer treatment to children if it is available.;

5. Cost.

Your actually not allowed to do placebo trials if it seriously harms/ threatens someone's life and instead have to trial against the best known/standardised treatment protocol which drug companies don't like as it's harder to get mediocre drugs approved.

Prescribing off licence is different from prescribe any drug for children. Most drugs are not licenced for children. For depression only fluoxetine is licenced for under 18 for example. Oncologists cannot just give any drug, it needs to undergo clinical trials. Otherwise how would they know what is a safe dose ? Who is going to pay for the treatment ? Etc.

That is still considered to be a placebo group. Instead of receiving sugar pills (it's not actually, duh!), they receive standard care.