The first indication of the influence that people can have in clinical trials when acting as a community may have occurred in the 1980s.
As reported recently in Viral World News U.K., many 1970s gay rights activists in the United States took part in the AZT clinical trial. They were determined to take the drug and not the placebo.
The participants took their pills to chemists for analysis. If the pills were found to be placebos they would leave the trial. The sponsors decided to terminate the trial early.
As a result of this breach of protocol, the FDA decided to re-evaluate its testing standard.
The Protocol
Most often clinical trials will the mask the drug that is given to a portion of the participants. In many instances, the trial will be “double blinded” meaning that neither the subject nor the researcher will know if they are taking the placebo or the drug. This is to prevent “bias” (factors that influence the results of the trial).
The trial’s credibility depends in large measure on maintaining the randomized clinical trial, a standard that was devised in the 1960s. The protocol was established to determine the efficacy and safety of a new drug.
In a “blind” trial, one group of patients is treated with the drug. Their response is compared to the second group in the trial who receives the placebo. In blind trials, both the drug and the placebo must be similar in taste, appearance, and smell so that the subjects may only have assumptions about their treatment.
The Phases of Clinical Trials
In order for a drug to be tested on humans in the U. S., the maker of the drug must apply to the FDA for an IND (Investigational New Drug) designation.
The typical path of clinical trials begins with a small-scale trial. It is followed by three phases with each succeeding phase adding a larger number of participants. All three phases check the drug for efficacy and side effects.
Phase I studies are normally of little therapeutic benefit to the participant who is usually in good health. An exception is chemotherapy because it cannot be administered to healthy individuals. Only patients may be tested with these drugs.
One goal of a Phase I study is familiarization with the side effects of the drug at low doses (safety). Another goal is to determine how the drug is metabolized and excreted. About seventy percent of drugs continue on to Phase II.
Phase II trials evaluate how the drug affects the disease and identifies its side effects. The patients are actually giving the scientists the opportunity to evaluate the effects of the drug on their body to determine the merit of continuing the study. Approximately two-thirds of drugs fail in Phase II.
Phase III trials determine the tolerable dose. This will be studied extensively in future studies. Phase III trials may involve up to 3000 participants at various locations. They may last up to four years in order to monitor the long-term adverse reaction.
At this point, the FDA may grant one or more designations to a pharmaceutical company in order to expedite treatment of a drug that shows the potential of satisfying urgent, unmet needs.
To fulfill legal requirements, the risk to the patient must be outweighed by the benefits of the drug. When the pharmaceutical company proves that the drug is safe and effective, it files an application with the FDA. If approved, the drug can be marketed.
Phase IV which is requested by the FDA involves the sponsor following patients who have been prescribed the drug once it has been approved. (additional information about clinical trials is available here.)
And Now the Age of the Internet
The internet has enabled trial participants to locate each other. Then they potentially “unblind” the trial by comparing treatment among themselves. The test is now considered biased and does not contribute with any accuracy to decisions made on the merits of new drugs under development.
Unblinding the trial would mean that the test was no longer neutral. It would mean that patients may have swapped their treatment or that doctors may have administered their favorite drug to a group.
Pharmaceutical regulators must have a true estimate of all treatment effects. The trial disclosures must show as much neutrality as possible with respect to any conflict of interest of investigators or sponsors.
Note that the sponsor can recover its investment for developing the drug, which could amount to millions of dollars, only if the trial is successful.
Patient Communities
Patient communities can range from mailing lists or groups on Facebook or dedicated websites.
A well-known site is a digital platform called PatientsLikeMe. In 2011 ALS patients who were subjects in a clinical trial compared their test results on the site’s message board. Their interaction unblinded the test and broke protocol.
Some patients concocted a homebrew designed to mimic the trial drug. Both the experimental trial and its parallel trial were completed. PatientsLikeMe researchers warned of the risks involved with homebrew compounds and made a concerted effort to see that patients and researchers work together in the future.
Yet we have witnessed similar situations with muscular dystrophy and Hepatitis C on Facebook and other social media platforms. It is obvious that patients can easily communicate without the aid of an organization or patient group.
There may be some advantages to the introduction of social media in testing new drugs. But for now, there is substantial evidence of the critical need for blinding due to the necessity of controlling bias in clinical trials.
Have you or anyone you know been involved with social media groups for health reasons?
