How to access future treatments now

How to access future treatments now: Clinical trials in a nutshell

Diseases will not give up or disappear

Luckily, neither will science nor researchers. Just as they didn’t in the 1980s when the needle and the syringe were the only known way of injecting insulin, or in the 1990s when HIV meant a death penalty, or in the 2000s when destroying the cancer cells also affected the healthy cells. 

Medical science has been rapidly advancing in the recent decades and many positive examples prove that. However, this usually takes time. It takes around 10-15 years for scientists to evaluate the safety and affects of a certain new molecule on the course of the condition, its prevention or diagnosis. Why so many years, you might ask? Well, this is the average period required for a new drug, a medical device or a procedure to be tested and evaluated whether it works for patients with a particular condition. If you have ever wondered how scientists come to such conclusions and achievements, I can give you a simple answer – through clinical trials.

Clinical trials (also called clinical studies) are an integral part of the drug development process – without them, modern therapies would never reach the ones in need. It is a regulated way for patients to have an innovative treatment years before it is in pharmacies or hospitals. Yes, patients can access these therapies while they are still being developed. 

There are over 70 000 ongoing clinical trials taking place in various countries worldwide.

These trials are not necessarily related to drugs or medications. You might be surprised but there are many studies using behavioral approaches or exercises to determine how they affect a certain conditions or health status (e.g trials measuring the impact of yoga as complementary therapy on patients undergoing radical prostatectomy, or trials related to ensuring healthy marriage through educational programs). As to the so-called interventional clinical trials (a type of clinical studies in which participants are assigned to groups that receive one or more intervention/treatment), they usually aim at proving that the drug is more effective than the standard of care or have fewer side effects that the existing therapies on the market. Being tested on animals, healthy volunteers and then patients with a certain condition, a compound is potentially turned into a promising medicine that could change or even save one’s life. 

In many cases, it turns out that current therapies are not effective enough and do not have the desired impact on patients.

While some people are feeling quite well with a certain medication, others do not find it that effective. No matter how good a treatment is, sometimes it fails a patient. Maybe the patient’s illness was so advanced by the time treatment began that the therapy could not work effectively. Maybe the patient has a genetic mutation or some other factor that prevents a drug that works on many other patients from being effective for this individual. Maybe the treatment initially worked but the illness recurred. Whatever the reason, there may come a time when a patient will need to think about what happens when the standard therapies fail to help. 

This is the moment when patients need to make a choice whether to pursue non-standard treatment options, try something non-scientific based on someone’s advice or a recommendation or consider innovative therapies that medicine is currently exploring. This decision should always be consulted with the patient’s physician and the medical team that is conducting the clinical trial if the latter is considered. 

Only 85% of patients are either unaware or unsure that participation in a clinical trial was an option at the time of diagnosis.

75% of these patients said they would have been willing to enroll had they known it was possible (Harris Interactive Survey). What is more, even if people are interested in searching for available clinical trial options, many of them don’t know where to find such information.

Even if people are interested in searching for available clinical trial options, many of them don’t know where to find such information.

There are public registers where patients can find this information such as ClinicalTrials.gov or the EU Clinical Trials Register. However, people often find them hard to navigate as these websites were not built for patients. What’s more, doctors aren’t also always aware of all clinical trials that could be the best match for their patients, just the trials conducted in their hospital or the city they work at.

Other platforms like FindMeCure allow patients to volunteer for clinical trials while showing them all options nearby and matching them to the most suitable study. Then, patients can get connected with the medical team who is running the study of interest so they can ask all their questions and see if they are eligible to take part in the trial.

For all people that are interested in exploring clinical trials as a treatment option, I would like to stress on a few important things they need to consider:

  • Informed consent

Patients should be informed about all the potential benefits as well as the risks that are related to the particular research study. Knowing all the pros and cons of the investigational therapy, the time period of the study and all other important information is described in details in the Informed consent section that the patient must read carefully. They should not forget that they have the right to stop their participation at any time and no one will urge them to do the opposite.

  • The phase of the study

Clinical trials have three phases before a new therapy can be approved and released to the market. Each phase assesses different aspects of the safety, efficacy, and effectiveness of the drug, procedure, or device. 

Phase one examines how the human body reacts to the investigational treatment. Then phase two assesses the optimal dose for the specific disease. Finally, phase three aims to confirm the effectiveness and benefits of the treatment, while also continuing to monitor the safety of the investigational therapy.

Phase one examines how the human body reacts to the investigational treatment. Then phase two assesses the optimal dose for the specific disease. Finally, phase three aims to confirm the effectiveness and benefits of the treatment, while also continuing to monitor the safety of the investigational therapy.
  • The placebo

Some patients participating in clinical trials receive a placebo and not the investigational treatment. A placebo is an inactive substance designed to resemble the investigational drug. This helps researchers to determine the effects of the investigational drug. The situation in cancer clinical trials is different though as researchers compare the investigational therapy with the standard therapy and a placebo is rarely used (usually only when there is no standard treatment).

As a conclusion, I would quote Mary Elizabeth, a Melanoma cancer patient who is alive thanks to a clinical trial:

In the world of science, we do not move forward without people who believe in progress

Mary Elizabeth, a Melanoma cancer patient

Patients volunteering to take part in research are the real heroes of our time. This is how new and more effective therapies can come us much sooner.

If you are interested in looking at what research studies are being done around you, just enter your condition below and follow the steps.

Further reading

How physics plays a role in the treatment of cancer

Cancer blogs

Current cancer research

The Not So Modern Stigma Of Cancer Research: A Scientists View Point

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