Clinical trials are an essential part of medical research. Before new medical treatments can be approved, they must be tested on humans to prove that they are safe and effective. Deciding to participate in a clinical trial is a personal choice that needs to be made after weighing up the pros and cons of how it may impact your quality of life with systemic mastocytosis (SM).
What is a clinical trial, and how does it work?
Clinical trials test new treatments and interventions on humans to evaluate their efficacy and safety before they are approved for general use. While new drug therapies are often what comes to mind when discussing clinical trials, they are also used to test medical devices, new drug combinations, new dosing schedules or supportive care approaches and preventative measures.
There are four possible phases in a clinical trial. A new phase only begins if the preceding phase saw positive results.
- Phase 1: This early phase tests safety and dosing on a small number of participants.
- Phase 2: This phase includes a larger group of people and focuses on treatment efficacy while monitoring safety.
- Phase 3: This larger phase often compares the trial treatment to the current standard treatment for a disease. If results are positive, the treatment usually goes on to be commercialized.
- Phase 4: This post-approval phase monitors long-term effectiveness and safety.
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What are the potential benefits of participating in a clinical trial?
When participating in a clinical trial, there is always the hope that this treatment could be the magic drug that makes living with your disease much easier — or even cures it altogether. It offers hope when current treatments may be less promising.
The main benefits of joining a clinical trial are:
- Having access high-quality medical care and potentially innovative new treatments without the long wait for them to become commercially available — a process that can take years.
- Contributing to medical research that may benefit others, including future generations.
- In some cases, the organization or company that is funding the trial may pay for your medical care, at least in part.
What are the potential disadvantages?
There are risks involved in participating in a clinical trial. Though strict selection criteria helps keep participants safe, a clinical trial can still have negative impacts on your overall health and well-being.
The drawbacks of joining a clinical trial include:
- Not receiving the treatment: Many trials include a placebo or comparator arm, which means some of the trial participants don’t receive the new treatment so they can be compared to those who do.
- Needing extra follow-up appointments and hospital visits, as well as medical tests, scans and biopsies.
- A lack of certainty in what can be expected from the trial.
- Dealing with side effects may be unpleasant or difficult to manage. Particularly in phase 1 trials, side effects may be largely unknown.
In short, participating in a clinical trial can mean access to treatment options you wouldn’t have otherwise, but it means accepting many unknowns about how that treatment could impact you and your wellbeing. If you’d like to join a clinical trial, talk to your care team about your options and what it might mean for your health.
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