Demystifiying Research

The medical research process comprises three stages:

1) Basic research

Basic research involves experiments and studies that occur in a laboratory setting. It focuses on uncovering and pursuing fundamental new knowledge about disease. While it can take a long time before patients see the results of basic research, it is the essential first step leading to vital medical breakthroughs and advances which provide the starting point for developing new therapies and treatment regimens as well as new research tools and techniques.

Investing in basic research supports the next generation of knowledge that can then be translated into preventive, therapeutic and diagnostic benefits for patients. For example, clinicians will use these discoveries to create a treatment regimen, which is then tested in clinical trials. Support for basic research comes mainly from government funding agencies, such as the Canadian Institutes of Health Research (CIHR) and the Fonds de la recherche Québec – Santé (FRQS). Such funding is provided on the basis of scientific excellence as determined by national peer review, but the money available each year is decreasing.

2) Translational research (3-6 years)

Translational research refers to the translation of knowledge from basic research into new treatments, as well as the translation of findings from clinical trials into everyday practice. The process is actually a two-way street, as novel observations about the nature and progression of disease made by clinician researchers can also inform or drive basic research. The whole process goes from bench-to-bedside-and-back. Funding for translational research comes mainly from medical research foundations and private sources. One reason for this is that translational research requires the concerted efforts of multidisciplinary and interdisciplinary teams of experts, while research grants from public funding agencies focus mainly on basic research and are usually awarded to individual principal investigators. Another reason is that the pharmaceutical industry prefers to invest in later stage research. Therefore, private support plays a significant role in filling this funding gap.

3) Clinical research (6-7 years)

Clinical research is research on human subjects aimed at ascertaining the efficacy of a treatment when measured against existing standards of care. It will determine whether a particular drug or procedure is as effective in people as it appeared in the laboratory and if there are potential adverse side effects that need to be weighed against the potential benefits. Clinical trials involve collaboration between clinician-researchers and study volunteers and help set the standards for patient care. Clinical trial volunteers, who participate only after giving informed consent, benefit by gaining access to new treatments before they are widely available. Clinical research is broken into the following key phases, each treated as a separate clinical trial:

In Phase 1 trials, researchersevaluate the safety of an experimental drug or treatment in a small group of volunteers (20-80). This phase also serves to determine a safe dosage range and to identify potential side effects.

In Phase 2 trials, researchers further evaluate the safety and assess the efficacy and correct dosing of the experimental drug or treatment in a larger group of patients (100-300).

In Phase 3 trials, researchers test the experimental drug or treatment in a much larger, more diverse population (1,000-3,000) to confirm its effectiveness, monitor side effects, develop safe usage guidelines, and compare it with existing products used for the same condition or disease.

If the experimental drug or treatment successfully passes through these three key phases – a process that can take many years – it will usually be approved by Health Canada for use in the general population. In some cases, Phase 4 trials may be required by Health Canada to evaluate long-term safety, the use of the drug or treatment on specific population groups, such as children, or to further assess optimal conditions for use.

Clinical research is by far the most expensive stage in the medical research process. Funding for clinical research comes mainly from the pharmaceutical and biotechnology industry or medical research foundations with substantial research and development budgets and, to a much lesser degree, by public granting agencies and private sources. Owing to the high cost of conducting clinical trials, many clinical research funders and sponsors prefer to invest in trials that offer small improvements over existing drug or treatment options rather than on new drugs or treatments which may have more potential. Furthermore, the funding is not always in sync with prevalent needs in the patient population, especially in cases where the disease is relatively rare or does not provide sufficient market opportunity.