The great thing about medical research is that there are always new clinical trials going on in an attempt to find a cure, or at least new treatment options. However, it can be confusing to understand what actually goes on in these trials, and how they come about in the first place. Read further to how clinical trials work for cystic fibrosis, and why they're so important.
Study sponsor and protocol
Let's start with the basics. Every clinical trial has both a study sponsor and a protocol. The trial sponsor is often the company that develops the therapy or medication. They then have to produce the protocol for the trial, which is a plan that gives an explanation of how the trial will work, and why they are doing it. Every single medical center that takes part in the trial, even if it is being done nationwide, must use the exact same protocol. Normally the protocol outlines how many participants there will be, what makes someone eligible or ineligible, the type of data they will be acquiring, and what types of tests will be administered and how often.
It is extremely important during clinical trials that the results are entirely unbiased. Having bias could be unintentional or accidental, but could dramatically affect the trial's results. For example, when choosing patients to participate in groups to compare in a study, if there became a trend of sicker patients partaking in the treatment group and healthier patients going to the control group where they wouldn't receive treatment, the results of the study would not be conclusive, because there were preexisting factors making the analysis uneven. It could be entirely possible for doctors to make this mistake by accident, which is why randomization is necessary.
So, how does randomization work? It helps to make sure there is no bias by ensuring that tests are done properly by giving half of the participants the medication while half receive only a placebo, but the patients are assigned to each group by chance instead of choice. Ensuring that the distribution is random makes the trial results much more reliable.
An alternative to randomization is called blinding. Like randomization, it helps to avoid bias in clinical testing, but instead of assigning people randomly, the researchers are "blind" or unaware of which patients are actually receiving the medication and which are receiving the placebo.
Four phases of clinical research
It is not easy for a drug to attain approval by the United States Food and Drug Administration, or the FDA. In fact, there are four phases in the process. All new drugs must pass three phases of interventional clinical trials with the intent of displaying the safety and effectiveness of the drug in its use against the disease. After it passes these, and if it passes these, the FDA will likely approve the drug, but that isn't the end of the testing. It will be monitored and analyzed continuously during the "fourth phase" of the study. Clearly, the process of full and complete FDA approval is extremely strenuous and complex, which is part of why there are always so many clinical trials going on for cystic fibrosis. If there weren't, there would never be any new drugs or treatments available on the market, because none would be able to attain proper approval without the clinical trials.
Not only is this a complex process, but it is extremely time consuming. It takes money for the pharmaceutical companies to go through this process, as the investment they make into the research and production of the drug is expensive, and they cannot begin to receive payments for these drugs on a large scale until there has been approval.
The reason it takes so long is largely because of the recruitment of patients. Many want to be assured that the trial they participate in is right for them, and conversely the researchers want to ensure this as well. It is often difficult to find the proper participants, and once they are found, it takes a significant amount of time to analyze the results. It may seem shocking, but due to this, the amount of time it takes from the drug first being discovered in the laboratory to the point where it can become approved by the USFDA and be accessible to cystic fibrosis patients is usually somewhere between ten and fourteen years.
Who are ideal candidates for cystic fibrosis clinical trials?
Each and every clinical trial has different descriptions of who they are looking for, and guidelines about who should apply. Sometimes, healthy people are required in the clinical trial as well. Sometimes, the study will be entirely comprised of people with cystic fibrosis, or a combination of both.
However, guidelines of cystic fibrosis clinical trials are often more complicated than simply needing participants who have cystic fibrosis or not. There are "inclusion criteria" and "exclusion criteria", which are aimed at making sure that the product of the clinical trial will be safe, effective, and able to fight against any risks of those involved in the trial, as their comfort and ongoing security is of utmost importance.
Inclusion criteria and exclusion criteria are used to reference whether or not a person fits within the guidelines of a study, or whether they should be included in or excluded from the clinical trial. So, what are the inclusion and exclusion criteria? There are a variety of factors, but they are usually based on certain factors like age, gender, previous treatment history, disease, and other medical conditions.
These criteria also vary on the different types of clinical trials that one is becoming involved in. If a drug is specifically targeted towards children, then the criteria would likely state that the participants in the trial be aged somewhere between 2-10, whereas a different drug's clinical trial might be more age-inclusive. However, before this can happen, the drug would need to be first tested and shown to be safe and effective in adults with cystic fibrosis, as regulations state that this step is necessary before administering a drug on a child.
There are certain drugs that are meant to combat certain bacteria that are often prevalent in those with cystic fibrosis. Therefore, if researchers want to test the effectiveness of a specific antibiotic to fight against said bacteria, the trial would likely dictate that only people with cystic fibrosis who are already infected by that bacteria would be eligible to join the trial. People who did not have the bacteria already would be excluded from the trial because it would not be safe to subject them to a potential contaminant.
Who sponsors these clinical trials?
There are a variety of ways in which a clinical trial can attain the necessary amount of funding. Often, the sponsorship comes from organizations (medical institutions, foundations, universities, drug companies, federal agencies, etc.) or sometimes individuals. The sponsors of the clinical studies then have the ability to decide which investigators will control the trials. Often, medical care will be given to the participant as well.
The Cystic Fibrosis Foundation is the largest supporter of research regarding cystic fibrosis in the United States.