Clinical trials for drugs have two basic goals – to determine whether the drug works for a specific indication and what adverse effects it has. I can’t think of any drug that has only one effect in the body. The goal is that advantage of the desired effect far outweighs any adverse (unwanted) effects that it may have.
The information is collected on case report forms. Our knowledge (facts) is determined by the information that is systematically collected in the forms. There are often important and interesting questions that could be answered in the trial but there is significant tension between the amount of data collected and the participant burden of participating in a trial. The more burden (time it takes to complete the data gathering) the less likely it is that the participant will stay in the trial. Drop outs from the study are BAD and expensive so studies strive to gather only the necessary information so everyone completes the study.
Studies will always gather basic information like weight, blood pressure, pulse, oxygenation and tests for the target of the drug if different from weight, blood pressure, pulse, oxygenation – for example a drug for psoriasis, additional information will be gathered such as examination of the skin. This determines the benefit of the treatment.
Adverse events or side-effects can be determined in one of two ways – spontaneous report or prompted. Spontaneous report occurs in response to a general question – “have you noticed other unwanted symptoms or changes in your health since you started the treatment” or asking about specific symptoms – “have you experienced nausea”. Most studies use spontaneous reporting not prompted. In the past, a spontaneously reported positive unexpected outcome would not have been systematically gathered on the case report forms so it can take a while to learn that a drug has additional positive benefits.
Sildenafil (Viagra) was initially in clinical trials for treating angina. When men were done with the study, they did not want to return their unused medication because they found that it improved their erections. It wasn’t an adverse event for them so they did not report it spontaneously because it was not unwanted or negative. So the clinical program for development of drug treatment for impotence started with patient observation rather than an animal study that is the normal drug discovery process.
This is what is happening with the GLP receptor agonists now. Enough people have noted unexpected positive benefits that more research is being done. In the meantime, doctors can prescribe an approved medication for reasons other than what it is FDA approved for. However, insurance often may not pay for drugs outside of their approved indication. Those who can afford to pay the full cost of the drug will be the front line of observing the full range of benefits of the drug (such as Hollywood stars).
Tamara Unleashed 