People living with chronic disease(s) benefit enormously from the pipeline of medications brought to market by hard-working clinical researchers who design and carry out clinical trials of drugs that show promise after testing in animals.
In the case of arthritis, particularly autoimmune forms like rheumatoid arthritis (RA), there are already many approved medications, including complex biologics and biosimilars that are effective. These drugs are proven to both reduce pain, reduce joint damage, and allow people to live a better quality of life.
Despite these advancements, however, it is incredibly common for people living with one or more chronic diseases to find that they need to cycle through a variety of medications to find one that works them. Sometimes, they may also become less responsive to their current regimen over time. For that reason, and others, there is an ongoing need to research and develop innovative therapies that are safe and effective.
Clinical trials need patients like you
Clinical trials are simply not possible without the participation of patients. Animal studies are not enough. This is because drugs that appear to work in animals may not work in humans. Or they may prove to have too many side effects to be acceptable for human use. There is no getting around it. Drugs have to be tested for safety and efficacy in patients. And that may mean you!
Here are five reasons why you might consider participating in a clinical trial to evaluate a drug candidate or medical device. Remember, when you do this, you are providing real-world evidence about the experience of living with chronic disease.
1. Science is not static
Without scientific research, we cannot advance our understanding of the human mind and body. And, we cannot assess the impact of our interventions on medical conditions that afflict us.
Scientists at major research institutions, government laboratories, and pharmaceutical and biotechnology companies work hard to design clinical trials that test the safety and effectiveness of medications and therapies that may someday benefit the patient population. Volunteering to participate in clinical trials in the hopes that the drug might one day benefit other people who have the same condition as you is a major contribution to our society.
2. Clinical trials may give you early access to new therapies.
Phase 1 clinical trials involve testing drugs to ensure general safety. In Phase 2 trials the aim is to determine if the drug is effective. They are usually done with a relatively small group (25-50) of subjects who have similar conditions. They may be randomized to receive different doses or routes of administration.
Phase 3 trials involve much larger numbers of people. They are used to determine if the new drug is as safe and efficacious as what is already available (the current standard treatment). Participants in Phase 3 trials all have the medical condition that the new drug is targeting. They are usually randomized to receive either the investigative drug plus the current standard treatment or the standard treatment alone. Therefore, not all participants will receive the new therapy unless the trial has a cross-over design.
Before agreeing to participate in Phase 3 clinical trials, it is important to understand that these types of trials require control groups (people who do not receive the drug being studied) in order to determine whether the drug causes benefit (or harm) to patients. So there is always a chance that you will be randomized to the control arm of the trial so that you do not actually receive the new therapeutic.
3. Receive enhanced medical care during and after the trial.
Clinical trials are not designed by researchers alone. It is a co-partnership requiring participation by clinicians, researchers, patients and even an ethical oversight body to protect the rights of those who participate.
This oversight body reviews the structure and management of clinical trials and research involving humans in general. It is called the Institutional Review Board (IRB). The IRB requires that scientific investigators communicate directly with participants to responsibly share and transparently inform them about
- what to expect from the trial
- their rights as patients during the trial.
In turn, patients can then share what matters to them with researchers. This is a collaborative process.
In addition, participants must consent to enroll and share their health data with scientific investigators. Once enrolled, patients are carefully observed, meaning you may see your doctor and healthcare team more frequently and experience benefits not available from standard, approved treatments.
Of course, there is always the possibility that you are assigned to the standard treatment group or that you may not respond to the medication being tested. You could also experience adverse reactions to the medication. In that event, your doctor would make recommendations about whether you should continue in the trial or not.
4. It’s not just medicine, experience matters too.
Before enrolling in any clinical trial, certain conditions need to be met. These conditions may vary from trial to trial. Many times, the people who participate in clinical trials – particularly for medications – must meet very strict physical and mental health criteria so that researchers can tease out real results from a vast amount of data they collect from each and every person.
Sometimes, people living with a disease may not be eligible for clinical trials. Some of the reasons for exclusion include having:
- Co-morbid conditions,
- A family history that precludes participation
- Not having enough time to fully participate in the trial
- Being physically unable to travel to the facility for evaluation
Despite those barriers, there remain ways for patients to contribute to the advancement of science because the unique experience of people living with diseases is of immense value.
The ArthritisPower Research Registry
After obtaining consent to participate, the ArthritisPower Research Registry collects data from patients with arthritis via a free smartphone or desktop application. Participants can select from dozens of validated Patient-Reported Outcomes (PRO) that help people self-assess (measure) their experience of pain, fatigue, and other symptoms.
This data is donated to the registry that researchers can access to study the arthritis population across many different dimensions (e.g., age, specific arthritis diagnosis, men vs. women). This allows researchers to design research studies that are deployed directly in the application.
For example, recently, we asked women with arthritis about their experience, concerns, and preferences with reproductive health issues. Data stemming from that research was presented at a major medical meeting and later published in a peer-review journal, thereby increasing our understanding of arthritis.
Another example is that we designed a study to better understand how the arthritis community accessed and used marijuana for medical use and related CBD products. These results were presented at two medical meetings, which is important because it allowed us to directly communicate with an audience of physicians who need to understand how their patients are using alternative therapies. This is particularly important to assess the risks and benefits of therapies that aren’t well studied, such as marijuana and CBD. These are just two of the many studies that are available to people in this registry and others like it.
5. Put yourself at the center of your treatment journey.
Participation in a clinical trial or a research registry is always optional. By electing to share your personal health data, you are playing a more active role in your own healthcare. You are also contributing to the advancement of our understanding of disease as a society. Participation allows you to advocate for the topics most important to you as well as the patient community.
Ask the right questions
It can take months or even years for researchers to understand the effect of a medication or device. Further, a successful clinical trial may not lead to the therapy being immediately available to you and others via prescription. This is because an additional study may be required by the FDA. There is also a lengthy process related to securing approvals to market and sell medications by the sponsoring companies.
Therefore, it is essential that you ask your health care provider important questions about what to expect from your participation.
Suggested questions might be:
- What is the time commitment for participation?
- Will I need the support of a family member or friend to help me manage the experience?
- What can I expect from the treatment? Will I know if I’m on treatment versus being in the control group?
- What are the risks or side effects I might experience?
- Is there compensation?
- When will the results of the trial be shared and how will I find out about them?
Unfortunately, there aren’t enough people participating in clinical trials. Some of that may be linked to concerns about:
- The time commitment and possible costs related to participation. (Importantly, patients should never be asked to pay to be in a clinical trial. Rather, the study should cover costs and some provide compensation.)
- Experiencing possible adverse events
- Being selected for placebo (the control group) and seeing no benefit from participation; or, being on medication and seeing no benefit from the actual therapy, itself. Patients may not know if they are on placebo or the medication until the end of a study. This type of study design is called “blinded.”
- Lack of support from family and friends regarding participation
An urgent and never-ending need for patient participation in clinical trials
Despite these barriers, there is an urgent and never-ending need for patients to participate in clinical trials. It is also essential that they contribute information about their lived experience with the disease (and associated treatments) to research registries.
The first place to ask for more information is from your doctor. Many doctors participate in research or can refer you to a research center for more information. It’s important to keep your health care team informed of your participation in clinical trials so that they can note it in your electronic health records and coordinate care.
All clinical trials are required to be listed at ClinicalTrials.gov, which has a search function to navigate through over 324,000 currently enrolling trials. People with arthritis who seek more information about clinical trial participation can visit www.ArthritisPower.org or www.CreakyJoints.org. People living with other chronic diseases can find a research registry at the National Institutes of Health or at the Patient-Centered Outcomes Research Institution.
Related Content: 8 Practical Tips to Delay Surgery for Arthritis
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The author does not have any financial disclosures to report.
Seth Ginsberg is the President and Co-Founder of Global Healthy Living Foundation and CreakyJoints, the digital community for millions of arthritis patients and caregivers worldwide who seek education, support, activism, and patient-centered research. An arthritis patient himself, Seth has dedicated his career to advocating for the arthritis and chronic disease community.
Since 2014, Ginsberg has served as a Principal Investigator of the PCORI-funded patient-powered research network ArthritisPower®. As a web and smartphone app, ArthritisPower is an innovative registry engineered for and by people with rheumatoid arthritis and related autoimmune and inflammatory conditions. Research efforts are overseen by a patient governor group as well as a distinguished research and corporate advisory board.
Seth lives in New York City with his wife, two young children, and a dog named Little Dude. Learn more at https://creakyjoints.org/your-team/