Whenever we get sick, we hope to be prescribed the leading approved medicine for whatever ailment we’re facing. But for some diseases, especially more rare cases like mesothelioma, there may be fewer options available that have been approved by the Food and Drug Administration. Fortunately, doctors have the option of turning to off-label or unapproved uses of drugs to treat these patients.
What Are Off-Label Drugs?
While the term “off-label drugs” may seem a bit scandalous or dangerous, it is actually perfectly legal. To start from the beginning, a drug or treatment can first become approved by the FDA after going through a series of clinical trials to prove that the drug is safe, works the way researchers expected, and is effective in treating whatever condition it is being tested for.
The FDA also must approve a report, called the drug label, on how the treatment must be used, including the proper or recommended dose, how the medicine is given, and what conditions it has been approved to treat. So off-label drug use is using one of these FDA approved treatments in a manner that has not been officially approved. This can include instances like using the treatment for another condition or using a different dose than is recommended on the drug label. Doctors even consider the use off-label if, for example, one specific treatment combination is often used for patients at stage 3 of their cancer, but is instead utilized for a patient at an earlier stage of that same type of cancer.
Off-label drug use is especially common in cancer treatment. Experts say this is largely the case for older, established drugs, like the chemotherapy drug carboplatin which has been widely used since it was developed in the 1980s. Though drugs like this have proven their efficacy for a number of cancers and diseases, it can be a costly and lengthy process for pharmaceutical companies and researchers to go through all the formal testing for the FDA to approve the drug for its new uses.
For instance, though carboplatin is not FDA approved for the treatment of mesothelioma, it is often used in combination with alimta and other chemotherapy drugs for these patients. It has been actively tested in clinical trials for mesothelioma with positive results, extending life expectancy to 9 and 12 months in two separate trials. However, there would still be much more testing and cost involved to get this chemotherapy drug approved for the rare cancer.
Off-Label Drugs and Mesothelioma
In the case of mesothelioma, there are many promising drugs in addition to carboplatin that are often used to treat patients, but have yet to be approved officially. Rare cancers and diseases in general often face fewer approved treatment options, which causes many oncologists to turn to other promising drugs that have been approved for other cancers. For instance, some treatments that have been approved for non-small cell lung cancer or other lung cancers have been used in the treatment of some mesothelioma cases because of their similarities.
According to the National Cancer Institute, there are only a handful of cancer drugs that are officially approved for the treatment of malignant mesothelioma. Alimta, or pemetrexed, is the only chemotherapy drug that is FDA approved for mesothelioma. The NCI also lists chemotherapy combination gemcitabine and cisplatin, even though the standard of care for mesothelioma is considered to be the combination of alimta and cisplatin. As such, there are many chemotherapy drugs that are used individually and in various combinations to treat mesothelioma that are off-label uses.
Among the most promising off-label drug uses for mesothelioma treatment is immunotherapy. These newer drugs, like Keytruda, have emerged as one of the most important areas of cancer research today. It has proven efficacy in treating a number of cancers, but has only been approved for a few. Keytruda, for instance, has officially been approved for the treatment of melanoma, head and neck cancers and hodgkin lymphoma. Just earlier this year, the FDA also approved Keytruda as a first-line treatment for non-small cell lung cancer, which researchers hope is another step in the right direction for the promising treatment to gain approval for mesothelioma.
Unfortunately, immunotherapy and many other promising drugs approved for other cancers are only available for mesothelioma patients through clinical trials. While understandable, since more testing is needed to really prove efficacy and determine the best administration of these newer treatments, this can severely limit the number of patients eligible to receive these treatments.
The Importance of Being Informed
It can be difficult to know about all the promising drugs and treatments being used today that haven’t been approved because while unapproved uses are legal, marketing of these unapproved uses is not. Overall, there’s a lack of information readily available about these treatments and their potential in unapproved cases, for both researchers and patients. In general, investigating clinical trials and other researchers’ findings are crucial in better understanding the off-label uses of these treatments alone and in combination.
In some cases, it can also be difficult to have these treatments covered or reimbursed by health insurance. Sometimes, the companies explain that because the drug is still “experimental,” it will not be covered. If patients are receiving these treatments in clinical trials, some clinical trials have coverage, while others might have other funding available to cover patient costs.
Along the same lines as being “experimental treatments,” the FDA does not regulate unapproved uses of these drugs, so it’s important to research potential side effects of these drugs and any risks seeking this type of treatment may entail.
Take the time to understand approved and unapproved treatments and what clinical trials may be available for your case. Overall, being well-versed in all your treatment options will help you and your loved ones make the best decision.