To understand how a medication works, much analysis must be done. Research studies, often called drug trials, are designed with great care. They help us to know how medications work, how they work best and on whom, and what we can expect from them.
For any new medication that comes to market, several phases of clinical research must take place. This process is very long and expensive. Governments all over the world set standards for these trials. Health Canada approves and follows all of the ones done in Canada.
A new medication starts with a hunch, a thought, or an idea. Often a great development begins in a lab, under a microscope, inside a test tube, or growing in a special dish. Wherever it starts, the next steps are very clear and are the same for all new developments.
At first, studies are done in a lab. Preclinical work involves seeing how a medicine works in animals such as mice, rats, monkey or dogs. Once this is done, the company can go to Health Canada for approval to start testing the drug with humans.
Both Health Canada and a research ethics board must approve the drug trial. The board is an independent committee that reviews all trials. It makes sure that the rights and safety of those involved are protected. Participants must understand all of the risks and benefits of being in a trial. Research ethics boards are often based in universities.
Phase 1 studies are done with normal healthy volunteers. These very short studies measure the way the drug is absorbed into the body and how a ‘normal’ body reacts.
After this rigorous first stage, results are again presented to Health Canada. If the drug appears to be safe, Health Canada will approve the next phase.
Phase 2 studies are done in people who have the disease the drug is meant to target. These people are otherwise quite healthy. Phase 2 trials decide whether the drug can be safely given to this population.
In this stage, many different doses of medication are usually tested to see what dose is effective and has the least possible side effects. Once this phase is complete, Health Canada reviews the findings and allows the company to move to Phase 3.
Phase 3 is where the studies get larger and longer. It is often divided into Phase 3A and Phase 3B. Large numbers of people affected with the disease are enrolled. They are watched for a longer period of time, often years, to study the drug’s long-term safety. These trials are done with more ‘real life’ participants, who may have more complicated medical conditions.
Phase 3A trials are also known as registration trials. The information collected is submitted to government agencies like Health Canada for approval in marketing and selling the drug.
The second part, 3B, is done with patients of special interest to the drug maker. This helps answer specific questions about whether the new drug can be safely combined with other medications.
Once a company has approval to sell the medication, they often begin Phase 4 trials. These are considered experience or marketing trials. Often the purpose is to help more doctors become comfortable with prescribing the medication. Real life data is collected, including side effects and benefits.
Sound complicated and very long? It is, and there are costs involved. From the time an idea evolves until a drug gets to the market, 10 or more years and multi-millions (or billions) of dollars often go by.
However, in certain cases the rules change. Medications for life-threatening illnesses for which there is no known cure do not always follow this long path. Although a drug is still being researched, it may be given in an attempt to save a life. Certain types of cancer medications are often streamed to this faster path.
Not every hunch, thought or idea becomes a reality. More potential drugs never get past pre-clinical or phase 1 than ever make it to market.
Not all research studies involve drugs. These studies can answer many different questions.
You may enter into a research study at the suggestion of your health care provider, you might respond to an advertisement, or you might hear about it from a friend. Research can be done in many different settings such as clinics, hospital, universities or research facilities. Where it takes place can also be important, but in particular you want to be sure that the study has been approved by a Research Ethics Board.
Research trials for new medications require long-term investment from developers, sponsors, research teams, governments and most importantly, volunteers. Without research participants, new tools would never reach the treatment toolbox.
Have you ever thought about volunteering for a research trial? People do so for a variety of reasons, including:
Benefits can include receiving free medicine, having more frequent contact with health care providers, extra monitoring of a disease, and getting a second opinion or access to a specialist.
Keep the following in mind when making a decision about being a research volunteer.
Whatever the reason you consider participating in a research trial, you should be comfortable with your final decision. Feel free to ask questions throughout the process. A well-informed person is an excellent research participant.
Most importantly, thank you for considering the possibility. Without volunteers, research studies would not be possible.