In the endless war on pharmaceutical companies, there is a consistent refrain; the US Food and Drug Administration is too slow to approve new drugs unless it approved drugs too quickly and the product hurt someone. Mainstream media highlight the complaints of doctors and patients that some drug or another is available in Europe or Asia but not here and then on another page delights in a lawsuit about how evil the company was for making a drug which carried risks.
Safety is important or it isn't and we often default to the precautionary principle which says it is. Thus it feels odd to have cancer experts in Europe criticizing drug companies because they don't rush to use untested pharmaceuticals on kids with cancer and enroll desperate families who lack the ability to make rational informed consent into a trial. These experts say drug companies are avoiding testing on kids and the EU is letting them get away with it. The same drug companies that are criticized for just throwing treatments at the public is now under fire for for denying kids "new, potentially life-saving drugs", says the Institute of Cancer Research, London.
And they want changes made or else, they warn, evil corporations won't sell their expensive products to governments and insurance companies that deal with sick children.
It's hard to defend pharmaceutical companies - until we consider the alternative. Currently drug discovery is one of the last areas of American research that isn't controlled by the government. It often takes a decade or more, it costs billions, it fails 95 percent of the time, it then takes years to get approved and, if it gets approved, companies have only a tiny window to sell it during which time they are called profiteers exploiting sick people, including by the government that puts new regulations and hurdles in place.
Yet the alternative to drug companies is...the government. If politicians control drug research, each drug would cost $20 billion and take 30 years to complete and, if recent history holds, a new president who didn't like the old one would wipe out entire programs because they had a predecessor's name on them (President Clinton with the Superconducting Super Collider and President Obama with the Constellation program are two giant examples) and then they would start a new "Manhattan Project" For X with their own name attached to it.
The Institute of Cancer Research instead wants to force companies to test their drugs on kids. Now, it reads ridiculous because it is, and I am not against kids in clinical trials, when it makes sense. Anyone who knows anything about the industry knows that by the time a product gets to a Phase III clinical trial the ethical and safety issues should be settled. These trials cost hundreds of millions of dollars, no company is wasting that kind of money frivolously unless they truly believe it works. But the perception among the the public is there are "approved" drugs and everything else. We've taught them to distrust medicine, the anti-vaccine and anti-medicine community says they are not anti-science, they are anti-corporation and we let them get away with it. So if the company does not want to test on kids, I think they should have a super vote.
But they will err on the side of cautions because we have demonized drug companies to such an extent that they don't want to test anything that could get them in the newspapers. Most parents won't enroll their children in clinical trials, which means even drugs designed for kids have a hard time getting a large enough study. A parent who does enroll a child in a clinical trial even uses that term "guinea pig" about their child because they have been trained to self- excoriate for embracing new treatments. It's so against the grain that enrolling a child in a trial makes it into the Wall Street Journal.
The Institute of Cancer Research not only wants exemptions for companies to not test on kids removed, they want to force them to test on kids. Their concern is that some new drugs may have pediatric uses even if they aren't indicated, but companies are unwilling to spend the money and take the risks to conduct the trials to find out; as evidence, they say 14 out of 28 recent cancer drug approvals (since 2007) might be used for kids. So they want the rules amended; currently a company doesn't have to test a drug on kids if the adult cancer targeted does not occur in children. It seems cut and dried if it doesn't occur in children, right? Not really, almost any genetic link to cancer can be found by someone somewhere to also have a potential genetic instance in a child also.
The EU offers longer exclusivity in return for Paediatric Investigation Plans that involve children and critics say they are not taking advantage of it. Imagine a for-profit company turning down the chance to sell its product at a profit for longer, if they would just agree to test on kids - they still don't want to do it.
In reality, these are only potential instances where a certain drug may have a benefit in pediatric cases. The authors highlight drugs that treat cancers caused by mutations in the ALK or EGFR genes and say ALK and EGFR mutations have been implicated in some childhood cancers. Given that, they contend companies should be forced to stretch out approval for a really small population that has no indication it will be helped.
Professor Alan Ashworth, Chief Executive of The Institute of Cancer Research, London, wrote in their statement, "It's essential that ground-breaking cancer treatments are tested not only in adults but also in children, whenever the mechanism of action of the drug suggests they could be effective. That requires a change to EU rules, since the current system is failing to provide children with access to new treatments that could add years to their lives."
Yes, drug companies are not being greedy enough. We need to force them to care more about the bottom line.