This topic has been popular so far and I have decided to expand on this area here and discuss some other problems with the system while I have an audience. The problem I would like to discuss is that of the ‘Clinical Trial‘ process of anti-cancer drugs. This is a very VERY expensive component of the drug companies expense when trying to get a drug to the market. The FDA and other agencies like it do require that the drugs are tested in clinical settings with humans. This is actually a good idea and should be done before a drug is released to the market as Humans will be using it in the clinical settings (not mice in cages…as early studies are performed with). However, this process has perhaps gotten out of hand these days and drug companies are struggling with unimpressive drugs that cost WAY too much to develop.
What is the problem? 1) The cost of clinical trials in the US and Europe is absolutely huge and is driving away drug development by smaller companies who actually have innovative and potentially effective ideas. 2) Most anti cancer drugs are required to be tested on the most sick cancer patients and not on newly diagnosed cancer patients (the latter only happens when the drug has been around for many years and lots of data is available, but not for new drugs being tested). Now, this sounds like a great idea, but as you might see in a minute it’s not. The sickest of patients who have tried other therapies and are still unresponsive and their cancer is progressing are usually eligible for clinical trials with new agents (as long as they can tolerate the drugs, eg. they can not be too old or have other underlying conditions like heart problems, etc.). So you might think that is a drug works in the sickest of patients it is likely to work on patients who are less sick and whose cancers are less advanced. But, this is not the case. You see, late stage cancer patients look nothing like early stage cancer patients….especially late stage cancer patients who have failed previous therapies. As cancers progress and get more and more lethal, they get larger of course and they also move to other locations…and the cancers have added extra mutations etc. So, although the cancer may shrink due to the new cancer drug and things look good in the more advanced cancer patients, the drugs don’t always work and produce different results in patients who are newly diagnosed or not as advanced. 3) Another problem is that since these patients are so sick even if their lives are extended for an additional month and even if only 10-15% of patients respond well, this may be enough for the trial to be considered a success. In fact, those increases are not very big for the average patient and rarely translate to patients who are not in the earlier stages of their cancer. The 12-14 years is takes for the company to develop a drug that frequently leads to 10-15% survival benefits of a few extra months is not very good indeed and is often too typical. Some drugs do shrink tumors but end up having no or little survival benefits.
These new drugs usually cost thousands of dollars a month, typically provide only limited survival benefits, in usually patients with the most advanced cancers, and have taken well over ten years to develop. I am not sure about you, but this does not sound like a resounding success to me. It seems like a bit of a failure. But, wait…let’s not go that far. Le’s examine why this system works this say and leads to these kinds of problems.
First the science. The drugs are usually targeted at very specific molecular pathways in the cancer cell. To be fair to the scientists (as I have been for many years) it’s only natural to take this reductionist approach of developing something that is very specific and maybe even sensitive. So, if you make something so specific you might not be surprised to know that it may only work on a small subset of cancers with that particular issue. The granting agencies are only giving money usually to those who focus on very manageable problems that can be tightly controlled (unfortunately cancer is not like this at all in the real world), so the scientist need to follow the money. Testing the scientific principle like we are taught requires very controlled and specific pathways etc. Drug companies base their work on what thousands of scientist have done and thus are strongly influenced by this as well.
Now the FDA. The FDA is going to allow drugs that are safe. Unfortunately, to be safe sometimes means that the amount used has to be reduced so that the toxic effects of the drugs are reduced. This also reduced the beneficial effects of the drug on the cancer. Furthermore, the FDA likes to award marketing status to drugs that have a lot of previous data on them and are hesitant to do so with completely new classes of drugs or drugs which come from undefined sources (such as plants, etc.). So, companies must constantly look for drugs that fit these parameters and frequently are stuck with less than blockbuster drugs.
Legal/Regulatory; The regulatory/legal framework is set up such that safety is first and that the unknown is unacceptable. This is why new drugs can only be tested on patients with advanced forms of the cancer, ethical guidelines say that only very sick patients can be experimented on, when in fact there are a lot more patients who are recently getting the disease. That is why advanced cancer patients can be tested but not newly diagnosed patients. This may sound like a good thing, but in the end its keeping a lot of those who might benefit from new drugs from not getting them. The idea is that people in the early stages of cancer are healthy and do not need to be treated…this is a very silly idea and I don’t really know where it came from. But, that is what clinicians are taught. So, again don’t do anything until the cancer is more advanced.
Anyhow, I hope that you now have a better understanding of why anti cancer drugs cost so much, are frequently not very effective, and the limitations that exist for their development. I will talk more about this issue from time to time. Do send me some comment if you have further questions and do visit my Cancer made simple website for more information.