Clinical Trials: Hero or villain?

Only 1 in 10,000 compounds ever reach the market! Drug development is a scientific endeavour that is highly regulated because of legitimate public health concerns.

It takes a lot of time and money to develop a new drug/vaccine - 2 to 12 years from discovery to market and costs that could run into billions of dollars.

Clinical trials (CTs) are at the centre of all medical advances, and a required step in bringing new breakthroughs from the bench to the bedside, that is from the researcher to the patient.

The clinical trial (CT) normally consists of three distinct phases.

Phase I trials (first in human stage) aim to find the best dose of a new drug with the fewest side effects.

Phase II trials (proof of concept stage) further assess safety as well as if a drug works.

Phase III trials (safety and effectiveness) compare a new drug to the standard-of-care drug (if a treatment exists).

It is only when these 3 Phases are completed successfully that the new treatment can be approved by the regulators for use in the general population.

These studies evaluate the effects of an experimental test or treatment, and allow the regulatory authorities to determine if it’s safe and effective, and should be made available to the public. And these trials won’t happen without the participation of volunteers in clinical trials. Thus, participation is critical in helping prevent, diagnose, and cure diseases and illnesses.

When you participate in a CT, you are contributing to research that could bring new treatments one step closer to reaching patients across the World. The trial may not only help advance the science for your disease or illness but could also make progress for related areas of science.

I must admit that CTs do have downsides, and a few are listed below;

The treatment may not work as well as the researchers expected, or at all. It may not work even as well as currently available treatments.

You may experience side effects. Those who stop treatment (dropout rates) can be high - as high as 30 percent. One reason for dropouts may be side effects. Often these are temporary or minor, but some can be severe. In rare cases, participants are seriously injured or have died of complications resulting from their participation in trials of experimental therapies.

You may get a placebo. If you do, while you may experience a positive "placebo effect," you won’t get the full benefit of the treatment.

You may need to spend a considerable amount of your time at a hospital or clinic to satisfy the trial requirements.

It is not all doom and gloom though! The positive sides to participating in clinical trials include;

CTs can save your loved ones - If you've got a disease or illness that’s genetic, your participation could find yourself helping your loved ones by making new treatments available and advancing scientists’ understanding of the disease or illness. The study could even be the one that leads to a cure. In other words, clinical trials are not necessarily done to treat the participating patient.

CTs offer you access to new treatments - participating during a clinical test may offer you access to experimental, cutting-edge treatment options. Plus, you'll have access to a medical team that carefully monitors your disease and your overall health.

CTs need participants from all backgrounds as some conditions are more prevalent within certain populations! It is also known that certain groups of people traditionally underrepresented in research are “denied” the benefits of the application of the knowledge gained through research.

During CT, differences in the efficacy of a drug/treatment in different age groups, sexes and races could become apparent.

Not everyone shares my excitement about clinical trials.

In fact, the general public has doubts, misunderstandings, reservations, and concerns about participating in clinical research!

How is the Safety of Clinical Trial Participants Protected?

This is a very important question. The history of clinical research is not perfect. Currently, every clinical investigator is required to monitor and make sure that every participant is safe. These safeguards are an essential part of the research. To ensure the safety of participants every CT has a data safety monitoring board (DSMB) whose job is to continuously monitor and advice on patients' safety during the clinical trial. CTs are conducted under strict ethical regulations.

According to the framework in which CTs must be conducted, they must satisfy the following requirements to be ethical; social and scientific value, validity, fair participant selection, favourable risk-benefit ratio, independent review, informed consent and respect for the enrolled participant.

The world is currently struggling with COVID 19 (we have lost nearly 500,000 souls as of now) and different companies are busy working round the clock to develop vaccines or find treatments for this virus. None of these will become a reality without clinical trials.

It is my fervent wish and hopes that at the appropriate time, Ghanaians will take part in any clinical trials that are approved by our regulatory authorities.

Let’s not give any credence to the backward comments by those two French scientists, Dr Jean-Paul Mira and Camille Locht (suggesting Africa could be the ideal place to conduct a clinical trial because there are no [face]masks, no treatments and no ICUs).

Several clinical trials have been carried out in Ghana with success – Genetics of Type 2 Diabetes in West Africans, Effect of GBT440 on TCD in Pediatrics with Sickle Cell Disease, SMART Africa (Strengthening Mental Health Research and Training), Collaborative Wilms Tumour Africa Project, just to mention a few.

Participating in clinical trials is the right thing to do!