Where to start?
Looking into trials earlier in your treatment may mean that you are more likely to qualify. Your participation could make a difference in your life and in the lives of many others.
All people living with a certain condition considering enrolling in a clinical trial will be given written information and an opportunity to discuss the clinical trial with the clinical research coordinator and/or principal investigator through the informed consent process. The informed consent process includes a discussion of what the researchers hope to learn through the clinical trial, how often you will need to come to the clinic and what instructions you will need to follow, the risks and benefits of the drug being tested and being in the trial, and what other medical treatments are available.
In this age of rapidly advancing scientific discovery, it is fundamental to provide rare disease advocates with information about participating in drug development. Likewise, it is vital to ensure that public policies continue to support patient engagement in drug development, offer incentives to innovators to continue to advance treatments that address unmet medical needs, and support access to those approved treatments.
Global Genes, a leading rare disease patient advocacy organization, that unites experts, advocates and people living with a certain condition of all ages to stand together in hope for treatments and cures for the estimated 7,000 rare and genetic diseases that impact approximately 30 million Americans and over 350 million people worldwide.
This organisation developed a resource to help better understand and engage in clinical research and drug development is available Here
What are the benefits and risks of trials?
Every clinical trial has risks. A trial treatment is still being tested, so some of the risks won’t yet be known. Nor will it be known whether the treatment will work. In early trials, effectiveness isn’t necessarily being tested. However, researchers or their representatives should be able to tell you what they anticipate the risks (including side effects) and benefits to be. In a carefully regulated trial, a medical ethics committee should have evaluated the design of the trial to determine that the possible benefits justify the risks for the participants. If someone tells you there are no risks associated with a treatment, it is a red flag that the trial is not being operated with transparency and proper oversight. All medications have some side effects, even aspirin, one of the safest medications ever developed. The International society for stem cell research developed a section named Things to Consider About Clinical Trials that may help you. Continue reading here.
Some benefits of clinical trials are:
- You may receive a new and better treatment than you would have gotten outside of a clinical trial.
- You can help others who share your diagnosis.
- Expert medical attention: Doctors and care teams must closely monitor health-related changes in clinical trial participants. They are more alert to problems such as side effects. You may receive more one-on-one care and have more checkups.
- Free or low-cost care: The research company may offer the new treatment at no or low cost to you.
Nephcure Kidney International website created an infographic on demystifying the myths of clinical trials to elucidate some facts.
- The drugs or treatment being studied are not always better than the standard treatment methods.
- Even if a new approach has benefits, it may not work for you.
- The treatment could cause side effects or risks that doctors do not yet know about or expect.
- Health insurance and managed care providers might not cover all trial care costs.
Benefits and risks in clinical studies are a major problem. In order to spread the message, follow the next links of some infographics that can help you in this matter.
Unknowns of clinical trials.
Some unknowns of clinical trials are:
- You could take part in a randomized trial, a trial in which you do not know which treatment you are receiving.
- You may have questions about how your treatment will be paid for. Costs of the studied treatment are usually covered by the trial sponsor, while standard treatments are covered by insurance. Ask before you enroll, or refer to your informed consent document.
- You may have other concerns about participating.
- Trials are not just for treatment. Some people only think of clinical trials for last resort treatment options, when other treatments have failed. Not only are many treatment trials geared towards those who have just been diagnosed, there are also numerous trials that are improving other aspects of our healthcare.
You are Not a Guinea Pig
By the time trials are approved by the official medical agencies to start recruiting people living with a certain condition, there has been significant research, frequently years of research, that indicates that this option could possibly improve your health. Researchers have rigorously tested, observed and published medical aspects in many preclinical settings before bringing it to people living with a certain condition.
If you wish to learn more, the nephcure Kidney International, has prepared content about Debunking Clinical Trials Myths
Finding a Trial Does Not Have to Be an Individual Task
The decision to participate in a clinical trial is a personal one. Your goals and lifestyle will help you make your choice. Talk with your healthcare team, family and trusted friends, or get matched with someone who has had an experience similar to yours. Not all doctors are aware of all options available all the time, so being proactive is in your best interest. You can contact clinical trials directly, and you do not need a referral from a doctor. If a clinical trial is right for you, ask about those that are available locally. Also, ask about those that are occurring outside the area in which you live. If the clinical trial is not offered where you live, you would be required to travel for treatment. Participants might be required to have more visits to the health care provider than they would have for standard treatment.
Your age shouldn’t keep you from considering a clinical trial. Approach a clinical trial as you do any other treatment decision, and consider the following questions:
- What’s the treatment?
- How does it work?
- How will I get it?
- How will it affect my everyday life?
- What are the side effects?
- What are the benefits?
When discussing side effects, ask if they will evolve over time. For example, if the fatigue will only last for a couple of weeks, that may be something you’re willing to accept for the long-term benefits.
By weighing the answers to these questions, you can determine if a treatment decision aligns with your values and your desired quality of life.
For more questions: Visit our section Clinical trials FAQ.
Why consider participating in research studies?
All treatments and medications currently available, first went through clinical trials. Without clinical trials, and the many patient volunteers who make them possible, our entire medical research process would stop.
Clinical Trial Consent and Screening Process Pfizer and another video from Office for Human Research Protections.
Several educational references have been used as inspiration to prepare this content for you (all Accessed April 2021)
- https://www.mdanderson.org/cancerwise/what-older-cancer-people living with a certain condition-should-know-about-clinical-trials.h00-159224934.html
- https://www.mskcc.org/pdf/cancer-care/patient-education/we-have-kidney-practical-guide-people living with a certain condition-families
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