Ciaran Williams 

Science and Environment Editor

Over the past couple of years, there has been a surge of misinformation, and general apprehension towards drugs and the pharmaceutical industry. Conspiracies that “Big Pharma is out to get you” swirl through social media algorithms, sowing seeds of distrust in the malleable minds of doom-scrollers all around. For those who fall victim to this grift, let me explain how this rhetoric neglects to acknowledge the long, mandatory, and difficult process that researchers go through to get drugs approved in Canada, and how that system ensures effectiveness and safety, in the hopes of putting your anxieties about the pharmaceutical boogie-man to rest.

The first step in this long process is often called “bench science”, where experts working in labs develop the first prototypes of the drug. Once they have isolated one they believe could yield good results results, they begin testing it on in vitro cells– basically the cells in a petri dish– to gauge its rough effectiveness and potential toxicity. This work allows experts to understand whether or not they are on the right track.

From here, if they find a drug they feel confident in moving on with, they can commence preclinical trials, which can involve animal testing or more work on cell lines. In order to gain approval for animal testing specifically, researchers follow incredibly strict guidelines set by the Canadian Council on Animal Care. Once they have obtained approval, they begin testing on animals. The process of preclinical trials works out many of the practical issues of the drug in production. This includes determining the dose and timing of the treatment, assessing side effects and risks, finding the best method of administering the treatment, and determining how the treatment affects the body. Because animals are not perfect replicas of humans, findings from preclinical trials are never taken as definite, and researchers are required to move on to phase 1 clinical trials to begin human testing.

However, before the drug can move from animals to humans in phase 1 testing, there are numerous hoops that researchers must jump through to get approval for human trials. First, they must obtain Research Ethics Board (REB) approval. The REB is the body responsible for protecting participants in the study, ensures that the potential benefits of the drug outweigh the risks, and that there is a real requirement for the drug. They also ensure that the study adheres to the restrictions laid out in Canada’s Tri-Council Policy Statement (2022), which details ethical conduct for research involving humans. 

Once trials have passed REB review, and have received a No Objection Letter from Health Canada, they are greenlit to begin phase 1 trials in humans. In phase 1 studies, the goal is to assess the safety, side effects, best dose, and timing of a new drug. The sample size is relatively small so as to lower the possible risks, with groups consisting of 20 to 80 people on average according to Health Canada.

Once a drug passes phase 1 testing, researchers can move on to phase 2 of the clinical trial. The principal goal of phase 2 trials is to look at how well the drug works and to see what adverse health effects the drug may have. A larger group of participants would be included in phase 2 studies, ranging from 100 to 300 people depending on the scale of the clinical trial. 

Following a successful phase 2 trial, researchers enter the phase 3 clinical trials, which continue the methods of testing established in phase 2, but with a much larger sample size. Phase 3 clinical trials make sure the drug is still effective, will look for adverse effects, compare the new drug to commonly used and approved drugs, and collect information about the drug that will allow it to be used safely on the market. If, at the end of the phase 3 trial, researchers prove that their drug is just as effective or better than the current standard of care, Health Canada will do a full analysis of this and will give them drug approval for use in Canada. While precise numbers for Canada are not easy to find, a vast majority of drugs do not make it past phase 3 clinical trials and do not end up on the market due to the strict criteria for approval set out by Health Canada, meaning that ineffective and dangerous treatments are often weeded out.

To ensure everyone’s continued safety, Health Canada continues to monitor the drug even after it is approved and on the market, noting any adverse health outcomes reported to them. Health Canada may even request that the new drug be studied further so they can formally collect adverse event data in an additional clinical trial: phase 4. 

Information regarding ongoing studies or drugs that have already been approved is available on the website clinicaltrials.gov. The purpose of these publications is to provide access to information about all ongoing clinical trials in Northern America for transparency and transfer of knowledge purposes.

This long and difficult process ensures that all drugs that enter the market are safe and effective. While the process is not infallible, it is an incredibly strong safeguard for the public against ineffective and dangerous treatments. Hopefully understanding the breadth of the process inspires some faith in the medical system. They are responsible for your health, after all.