The MS Australia Clinical Trials Network was established to encourage high quality clinical MS research in Australia and New Zealand and to increase awareness of clinical trials and clinical research for people with MS and other demyelinating diseases.
Understanding Clinical Trials
Clinical trials are special research studies to test new treatments for diseases like MS. To make sure the results are fair and accurate, trial plans are made very carefully. They use techniques like ‘blinding’, ‘randomisation’, and placebos.
What is a Placebo?
A placebo is a treatment with no active ingredients, which looks like the treatment being tested. Placebos serve two important purposes in clinical trials:
- Blinding: When a participant, doctor, or assessor doesn’t know if the patient is getting the real treatment or the placebo, it’s called “blinding.” This ensures that any effects observed are genuinely due to the treatment and not influenced by expectations.
- Understanding Treatment Effects: Placebos are essential for comparing the actual impact of a treatment. To prove that a new treatment works, it must show better results than the placebo group. This comparison helps researchers identify the real treatment’s effects, including any side effects.
Why Placebos Matter
Placebos are a vital part of clinical studies as it is well-documented that providing any form of medical care causes a strong, temporary, physiological response in the body – the ‘placebo effect’. To prove a treatment’s effectiveness, it must surpass what’s seen in the placebo group.
Blinding: There are different types of blinding. Single-blind trials conceal treatment information from either the participants or the assessors (doctors or researchers). Double-blind trials conceal it from both participants and assessors, ensuring that neither knows who is receiving the actual treatment or a placebo. This ensures unbiased assessments and, as a participant, you may not receive the actual drug initially. In rare cases, triple-blind trials include data analysts, enhancing the integrity of results.
Random Assignment: To prevent bias, participants are randomly placed into either the treatment or placebo group. Participants must meet specific criteria, ensuring a fair comparison. Different types of MS might react differently to the same treatment, so this is essential.
Benefits of Reliable Trials: When results from many well-conducted trials are combined, we can be highly confident that a new treatment is safe and effective. It also helps us actively monitor any risks associated with the treatment.
Patients are randomly assigned to either the treatment or placebo group to avoid any bias in patient selection. People who wish to enrol in clinical trials must usually meet certain requirements for a specific trial. This is to ensure that ‘apples are being compared with apples’. For example, an intervention may have different effects in people with relapsing remitting MS compared to people with secondary progressive or primary progressive MS.
Depending on the intervention being trialled, the trial protocol may include some or all of these features, or additional design features.
When the results are put together from several clinical trials that all rigorously follow these international ‘gold-standard’ methods, there can be a high level of confidence that a new treatment is safe and effective, and that any risks are known and can be actively monitored.
Trial stages
Clinical trials progress through four phases: Phase 1 assesses safety and tolerability in healthy volunteers, Phase 2 evaluates efficacy in a larger group with the condition, Phase 3 confirms efficacy in a larger population, and Phase 4 monitors long-term safety post-approval by regulatory agencies. The required participant numbers can vary.
Participating in clinical research benefits individuals with MS by potentially providing access to new treatments and improving their quality of life, while also benefiting the broader MS community.
Further reading
Detailed patient resources are available with further information on the clinical trials process and factors you may wish to take into consideration before participating in a clinical trial. We recommend the following:
