Melanoma 

On September 4, 2014, the FDA approved the use of the immunotherapy drug Keytruda (pembrolizumab or MK-3475) for patients with advanced melanoma. The drug is meant to be used after previous treatments of Yervoy (ipilimumab) and BRAF inhibitors. Keytruda is the first drug on the market that targets the PD-1 protein on the body’s T-cells. PD-1, or programmed cell death one, serves to supress the body’s autoimmune system. When Keytruda attaches to PD-1 it stops it from functioning, thereby increasing the sensitivity of the patients immune system and allowing more T-cells to fight the tumor.

 

 

Clinical trials of Keytruda have yet to be completed. However, selective FDA approval was nonetheless approved using the administration’s accelerated approval program.  This accelerated program approved the drug only for patients in the most advanced stages of melanoma; those who did not respond to previous treatments of Yervoy and BRAF inhibitors. The FDA’s accelerated approval program is created to promote an earlier release of, “ drugs that treat serious conditions, and that fill an unmet medical need”  based on evidence that the drug is likely (but not guaranteed) to benefit patients. (FDA) The principle behind this policy is that patients with no alternative treatment and a serious condition should not be deprived of a drug that has any chance of helping them.

 

 

 

Of course, before the FDA approval, patients had much more difficulty accessing Keytruda. Many Stage III or Stage IV melanoma patients chose to participate in clinical trials because it was their only option when they had exhausted other treatment methods. It is important to note that for some clinical trials patients were given pre-existing treatments so that researchers could compare the results of their drug to other treatments. In these cases, some volunteers are given the new treatment, which improves their condition, while others undergo a second treatment of an ineffective drug that is already available on the market. This simply wastes their time, of which they have precious little remaining.

 

 

 

Sometimes patients are unqualified for a clinical trial because their condition has progressed too far, or they have another illness which would complicate their situation. If the condition is serious, then they may apply for compassionate exemption. Compassionate exemption means that the patients are provided access to treatments not yet approved by the FDA, as long as those treatments could possibly prolong their lives, or at least improve their quality of life during the time that they have.

 

 

Cases in which patients for one reason or another do not receive the latest treatment and end up passing away often spark heated discussion about the way that drugs are distributed. Many times the FDA is criticized for withholding treatments that could have saved lives.