People with rare diseases have a number of clinical trials to choose from. Clinical trials offer new medication and treatments, and in most cases, participants are allowed to use the new drug as they see fit. Withdrawing from a trial can be difficult, but it’s important to weigh the pros and cons before making a decision.
Before joining a clinical trial, it’s essential to know as much as you can about the trial itself. Each trial is different and may have additional requirements for participants. Some clinical trials are open only to patients with specific rare diseases, while others are available to anyone who feels they can benefit from the medication or treatment.
With no other treatments currently available, some people feel joining a trial is the best option for them. However, it’s important to understand everything about the drug you’re being offered before deciding.
What Is a Clinical Trial and How Do They Work?
The first thing people need to understand is what a clinical trial actually entails. The phrase “clinical trial” may bring images of laboratories and large corporations to mind, but small companies or even universities run the majority of trials.
In addition, not all trials revolve around medications. Clinical trials also investigate new surgical techniques, therapies involving physical activity, and alternative treatments such as herbal supplements and acupuncture needles.
Clinical trials are extremely important because they offer patients new treatment options that they would never have access to otherwise. Rare diseases tend to receive the most attention from the medical community and pharmaceutical companies because of how little research has been conducted on them.
If you have a rare disease, you can bet that there are several clinical trials for it at the moment. Be a part of the rare disease recruitment process and determine whether you qualify for any of them.
The Benefits of Enrolling in a Clinical Trial
Most people who sign up for clinical trials cannot be paid to participate, but they do receive a number of benefits.
First and foremost is the new medication or treatment they’ll receive as part of the trial. In most cases, participants are given a supply of this new drug and allowed to use it as they see fit, with whatever frequency their doctor prescribes.
What’s more, if the treatment proves successful during a clinical trial, that doesn’t mean a lifetime of free treatments once it hits pharmacies. In some cases, researchers may even pay for additional follow-up testing if specific procedures need to be completed before applying for FDA approval.
How Has Enrollment Changed in the Past 20 Years?
Patients have every right to be concerned when it comes to medical trials, but that doesn’t mean they should turn down an opportunity because of a few inconveniences.
Today the process has been streamlined so much that enrollment can be completed in about six months. There are no more phone calls or forms to fill out (other than signing consent documents), and patients don’t need to make appointments with several doctors for consultation.
In fact, all most patients will deal with is a questionnaire about their condition and medication history provided by the doctor conducting the trial. Most organizations even allow participants to complete this form online or over the phone before coming into the office for a brief visit and final approval.
What to Expect if You Decide to Enroll in a Clinical Trial?
As we mentioned earlier, almost every clinical trial will involve a questionnaire about your medical history and current medications. It should take no more than 30 minutes to complete this form.
After that, you’ll meet with a representative from the company who will conduct an initial screening process to determine your suitability for the trial. Sometimes this process is done over the phone, but it’s also common for participants to meet with a representative in person before giving their consent.
The Risk Involved in Clinical Trials
The majority of clinical trials don’t pose any significant risk to study participants, but there are always some associated risks that tend not to be as common among those who choose not to enroll in trials.
For example, those who participate may experience headaches or nausea as a result of ongoing treatment, more severe side effects such as seizures, bruises, or infertility if they were taking part in an experimental vaccine, and even death.
Medical trials offer an opportunity for patients to try new treatments that could potentially improve their quality of life. But with any trial, there are associated risks and disadvantages. If you’re considering enrolling in a clinical trial, make sure you understand the short-term and long-term consequences before signing up.
