We are over 200 years since Parkinson’s disease was first described and nearly 55 years since the discovery of levodopa, a medication that helps control the motor symptoms of PD and is still considered the gold standard treatment for this debilitating, progressive, neurodegenerative illness. However, as the disease progresses, response to treatment becomes unpredictable and less effective.

There is a clear need for better therapeutics. But to get a new neurologic treatment from the lab to the pharmacy shelf and into the hands of patients, it can take well over a billion dollars and 15 to 30 years.  About half of drug development time is spent in clinical trials. Unfortunately, close to 85% of all clinical trials are delayed because of recruitment problems and shockingly up to 30% never make it off the ground because they fail to recruit any subjects. This represents a significant waste of time and resources.

Although there is a worldwide community of academics and researchers devoting a great deal of time and attention to finding better treatments and a cure for what is currently incurable, the research cannot proceed without patient participation and involvement. What the millions of us affected with this chronic, disabling disease need is quite simple yet unbelievably complicated – better treatments and ultimately, a cure. We often think of the search for these elusive endpoints as being far removed from us when in fact, we need to be an integral part of the process.

When asked, most patients are interested in participating in research but there may be any number of concerns that need to be addressed in order for them to enroll. There may be logistical issues to consider as well as more significant worries regarding safety. Common concerns, whether misconceptions or valid risks, act as barriers to research participation.

I didn’t really want to be the “test” subject for a brand new, unproven medication that I knew nothing about. It would be dangerous and too risky.

First of all, not all clinical trials involve testing a new treatment. Some do of course but many are observational where scientists learn about particular disease states by monitoring a group of affected patients. Others may be testing an already approved and safe medication that is normally used for other health issues (known as repurposing an existing drug).

Of course, many trials are directed at testing new medications and any new treatment has its risks. Participants in research must be comfortable with the degree of potential risk involved, in order to give informed consent. An important fact to keep in mind is that the drug development process is heavily regulated and monitored by each country’s health authority. By the time a drug is tested in humans, researchers have already had to show scientific evidence that it will likely work and is safe. And if the study happened to be a Phase 3 Trial, the intervention or treatment has already passed safety studies and has been shown to be potentially effective.

If I participate in a clinical trial, I may get worse.

As with any treatment, you cannot be completely sure of the outcome and there is always the risk that you may not receive any benefit from the treatment being tested. But the premise of new interventions being tested, is that they will result in better outcomes than standard treatments. Participants are also closely monitored for any adverse effects or worsening of their original condition and can withdraw from the study at any time.   

Participating in a clinical trial will disrupt my daily life and will require a significant time commitment.

There is so much variation in the logistics of different clinical studies, those that require more intensive and frequent follow up and others that may be completed in a single visit. Some may even be done at home online or may simply involve sending in a DNA sample by mail for example. Depending on your life circumstances, there is likely a logistically suitable study.

 If I participate in a clinical trial, then I won’t receive the same quality of care that I currently have with my doctor.

In most situations, you continue to receive ongoing care from your own physician, and they are aware of your involvement in a particular study. Also, since many clinical trials involve specific visits and investigations often by leading health care professionals in the Parkinson’s field, you may benefit from this increased monitoring and medical supervision.

There may be painful procedures as part of the clinical trial.

The vast majority of clinical trials are not invasive or painful. You will be informed of all potential procedures ahead of time which allows you to make a decision that is appropriate for you. But keep in mind that it is not the researchers’ intention to cause any discomfort and much thought is put into minimizing any adverse effects.

If I join a clinical study, I won’t be able to drop out without jeopardizing my treatment.

There are many reasons both personal, logistical or specific to the research study itself that may result in a participant withdrawing. It is completely within your rights as a patient to do so at any time without fearing that your care will be jeopardized in any way.

I can only participate in a trial if I have access to a major medical institution.

Many studies take part in the community setting by physicians close to home. Some can even be done online, and this number is increasing given the recent acceleration of telemedicine usage as researchers have had to re-evaluate and adapt to the restrictions that were put in place during the ongoing COVID-19 pandemic.

If it was a good idea for me to participate in a clinical trial, my doctor would have told me about it.

Despite the fact that studies have shown that patients generally would like to participate in clinical trials a relative minority say their physician ever mentioned medical research. There are so many issues that have to be dealt with during the short span of your medical appointment, that there often is not enough time to educate you about the clinical trials that are available or suitable. Therefore, just because a trial is not mentioned, does not mean it is not a viable option. Instead, you must rely on other resources (many available online such as Fox Trial Finder or country specific resources such as ClinicalTrials.gov in the US) to inform yourself of appropriate opportunities.

You should only participate in a clinical study if you have no other treatment options available to you.

There are clinical studies directed at many different stages of disease. Those directed at controlling late-stage symptoms may require subjects, and address issues found, in advanced disease. However, there are many studies (particularly those testing potentially disease-modifying treatments) that aim to include relatively newly diagnosed individuals, particularly those that may have not started medications.

Although many of these concerns can be addressed, there are some that cannot be as easily mitigated. If you are part of a randomized control trial, then you will not be able to choose whether you receive placebo vs. the drug being studied. This may be difficult particularly if you had to change your current regimen and were placed in a control group. You may have to stop or change your normal treatment protocol and the experimental medication may not be as effective resulting in you experiencing greater symptoms. Even if the new treatment you receive in a clinical study works well, you may not have continued access to the treatment once the trial is completed.

Participating in research is vital as is making an informed decision regarding enrolling in a clinical trial. Risks versus benefits must be weighed carefully in the context of your own life circumstances, overall health and comfort level.

There is undoubtedly a certain feeling of empowerment that is gained from being proactive in the choices you make to help advance the science, advance knowledge in Parkinson’s research. And better treatments for PD and a cure are in our future because members of our community who are given the opportunity to be a part of a clinical study, consider it with due diligence and follow through if appropriate. Participation in a research trial requires selfless commitment. Those that participate give of their time, energy and literally in some instances, themselves. We in the patient community have the ability to play an irreplaceable role in moving us closer to ending this disease and can be part of something that has the potential to change the trajectory of the lives of all those who face the challenges that Parkinson’s brings.

Soania Mathur MD, CCFP, founder of Unshakeablemd.com. Dr. Soania will be speaking on the topic of “Why participate in clinical trials“ in May at the WPC Virtual Congress.

Ideas and opinions expressed in this post reflect that of the author(s) solely. They do not necessarily reflect the opinions of the World Parkinson Coalition®