Please There was a time before the 1950s when a drug was thought to work for a certain disease because wise, old doctors said it would work. We have come a long way since that time. Now you need hard evidence. A common term now is “evidence based medicine”. In the 1950s, it was Bradford Hill who started this process of doing proper drug trials to determine their efficacy and, importantly also their side effects in the treatment of human diseases.
As I mentioned, prior to Bradford Hill’s time, a famous doctor would proclaim the usefulness of a certain medicine and many of us in the medical profession would follow suit.
The evidence- seeking concept is now called randomized controlled trial ( RCT). In the classic RCT, to determine if drug x works for a disease y, first you need an adequate sample size of patients with disease y who will be administered the drug x. To a similar number of patients you need to administer a sugar pill or a dummy pill, called a placebo in medical speak. Then over a certain period of time, if you can show that those in the drug arm of the trial did better than in the placebo arm of the trial, you may be able to conclude that the drug X is effective against the disease y. But the difference in effectiveness has to be what is called, “significant”.
And this significance is determined by statistical testing which tries to eliminate “chance” as the cause for drug x being more effective. Even to eliminate the chance factor, the importance of the term random cannot be overemphasized.
For example in determining who receives the placebo versus the drug in question, the choice has to be random. If the patient is pre selected to receive the drug or the placebo then you have introduced bias into the study and therefore will not be a proper study. In many instances even the investigators will not know whether the patient received the drug or the placebo. This is an example of maths and science magnificently merging together.
Now, just the effectiveness or efficacy as it is often called of drug X is not enough. The drug may be effective for the time being but it may have unacceptable side effects. In 1960 the devastating side effects of the drug thalidomide forced governments around the world to insist that all new drugs should be tested using RCT method to determine efficacy and to check for side effects. So a world of regulations has spawned around RCTs to make sure they are properly carried out.
One of the first diseases to be subjected to human trials was tuberculosis. At one time only one drug was used for TB, then RCTs revealed that the disease responded significantly better to combination of drugs than just one drug. This lesson was carried over for a more modern scourge, the HIV virus against which a combination therapy is now utilized.
RCTs are now also made use of for studying the efficacy of vaccines. The injectable typhoid vaccine that is used worldwide was first studied here in Kathmandu by Dr I L Acharya and colleagues in the early 1980s using the RCT concept.
RCTs then are the gold standard for testing drug efficacy and side effects. In Ayurvedic, Tibetan and other forms of alternative medicine, RCTs could be potentially useful to determine efficacy of treatments.