I am writing this post after answering various questions in various forums regarding the pros and cons of going for treatments which are still in clinical trials phases. Most of the questions I receive are related to Stem cell therapy for various diseases. FDA has approved only Stem cell treatments using hematopoietic stem cells. There have been a lot of reports of various doctors and some clinics claiming they have gained success in stem cell treatments for the certain disease like liver cirrhosis, mitochondrial disease, eye vision treatment etc. Most of those claims are either fake or have not been clinically approved by FDA. In most of the countries, it has been a big challenge for authorities to tackle these kinds of frauds and stop peoples from getting trapped by these kinds of scams.
It is necessary for peoples to get complete knowledge of any kind of treatments they are getting the disease. It is also important to understand the difference between clinical trial treatment and medical treatment.
What is clinical Trail?
The clinical trial is an important phase in clinical research of new drugs or treatments because it involves exposure of new invention to the humans. Clinical trials should not be misunderstood for normal treatment. In order to produce new clinical research products legally, it will take 5 – 10 years. All Clinical Research experiments need to get FDA approval before conducting a clinical trial on humans and all the result should be updated in FDA database for clinical research ClinicalTrials.gov.
Facts of Clinical trials:
- Clinical trials are only means of initial investigation of the new treatments and should not be misunderstood as the substitute of existing treatment.
- As per FDA, all clinical trials should be conducted free of cost and patients should not be charged for any treatments.
- FDA requires that all participants of clinical trials should be given complete information about the study known as “informed consent,” and it must be in writing.
- All the clinical trial investigation with human participants needs prior FDA approval.
- All the clinical investigation and their outcome should be submitted to FDA database for the clinical trial.
- It’s illegal to market for the treatment or drug under investigation in clinical trial phase.
Importance of clinical trial:
- Preclinical research helps to answers basic questions about a drug’s safety and efficiency.
- Researchers design clinical trials to answer specific research questions related to a medical product.
- Clinical researches are done in a small population of people to study the dosage level and its safety in humans.
- Clinical research helps to study the efficiency and side effects of the drug in humans.
- It helps to study the various chemical reaction of the new drug in the human body.
- Positive Clinical trial data helps the researchers to get easy approval from FDA to get easy approval for next stage of the investigation.
Designing Clinical Trials
Before the clinical trial begins it is necessary for the researchers to prepare the Clinical trial protocol with the following details:
- Participant selection criteria and the type of participants selected.
- Number of participants to participate in the study.
- Duration of the clinical trial study.
- The method of administration of the drug (Oral or through intravenous administration).
- Test design and test and control population ratio, and the method of Randomization.
- Percentage of dosage and duration of administration.
- The method of assessments conducted, and the type of data to be collected.
- Data extraction, storage and Analyzation technique and tools to be used.
During Phase 1 studies, researchers test a new drug in normal volunteers (healthy people). In most cases, 20 to 80 healthy volunteers or people with the disease/condition participate in Phase 1. Based on the drug and the target diseases, this phase may be conducted for several months and approximately 70% of drugs move to the next phase.
Phase 1 studies are closely monitored and gather information about how a drug interacts with the human body. Researchers adjust dosing schemes based on animal data to find out how much of a drug the body can tolerate, what its acute side effects are. This phase provides early information about how effective it is to administer the drug to limit risks and maximize possible benefits which are important to the design the Phase 2 studies.
In Phase 2 studies, researchers administer the drug to a group of patients with the disease or condition for which the drug is being developed. In most of the cases, approximately 33% of drugs move to the next phase of study may for several months to maximum 2 years.
Phase 2 studies provide researchers with additional safety data. Researchers use these data to refine research questions, develop research methods, and design new Phase 3 research protocols.
Phase 3 study is conducted to demonstrate whether or not a product offers a treatment benefit to a specific population. Sometimes known as pivotal studies, these studies involve 300 to 3,000 participants with the study conducted up to a maximum of years. Approximately 25-30% of drugs move to the next phase.
Phase 3 studies provide most of the safety data. In previous studies, it is possible that less common side effects might have gone undetected. Because these studies are larger and longer in duration, the results are more likely to show long-term or rare side effects.
This phase is a post-market research and analysis of drug reaction among several thousand of volunteers who have the disease/condition. Phase 4 trials are carried out once the drug has been approved by FDA during the Post-Market Safety Monitoring.
The key focus is to gather information on the drug’s effect in various populations and any side effects associated with long-term use.
What is Randomization?
Randomization in a clinical trial is the method of segregation of patients/volunteer population randomly into two group .i.e. Test and control. Randomization as a method of experimental control has been extensively used in human clinical trials and other biological experiments.
Advantage of Randomization:
- It helps to segregate the volunteers into test and control population.
- It prevents the selection bias and ensures against the accidental bias.
- It produces the comparable groups and eliminates the source of bias in treatment assignments.
- Finally, it permits the use of probability theory to express the likelihood of chance as a source for the difference of end outcome.
Randomization Test Group:
This is the randomly assigned group of the population receiving the treatment under investigation.
Randomization Control Group:
Unlike the test group, this is the group of population which receives standard treatment with placebo medicine.
A placebo is an injection or pill that seems identical to the new treatment, but is inactive with no active treatment agent and produces no side effects. During the clinical trial experiment, Placebo helps the researchers to segregate the population to test and control.
The population of Test and Control for the Randomization experiment will usually be in the ratio of 85:15.
Types of Randomization:
Most common types of Randomization methods are:
Randomization based on a single sequence (event) of random assignments is known as simple randomization.
The block randomization method is designed to randomize subjects into groups that result in equal sample sizes.
The stratified randomization method addresses the need to control and balance the influence of covariates.
Covariate adaptive randomization:
In covariate adaptive randomization, a new participant is sequentially assigned to a particular treatment group by taking into account the specific covariates and previous assignments of participants.
Who are clinical trial volunteers?
It’s important to test medical products in the people they are meant to help. The FDA seeks to ensure that people from many different groups are included in clinical trials.
Volunteering for a clinical trial is no guarantee of acceptance. Similarly, there’s no guarantee that an individual in a clinical trial will receive the drug or medical product being studied.
People volunteer to participate in clinical trials for different reasons.
- Some volunteer participates because they want to help advance medical knowledge.
- Peoples with the disease who have tried all available treatments for their condition without success.
- Few peoples because of tempted monetary benefit enroll for the clinical trial.
Who can participate in the clinical trial?
- Some trials involve people with a particular illness or condition to be studied, while others seek healthy volunteers.
- Pregnant women’s and post-pregnancy breastfeeding women’s are not eligible to participate in clinical trials.
- Peoples using tobacco products, illegal drugs or addicted for heavy alcohol intake are not eligible.
- You are on regular medication unless approved by the study, such as an oral contraceptive.
What are the risks of participating in the clinical trial?
- Some experience temporary and some permanent side effects like unpleasant, serious, or even life-threatening side effects.
- Some experience side effects during treatment and some experience post treatment after few months or years.
- The experimental treatment may not be effective.
- The protocol may require more time and attention than a non-protocol treatment, including trips to the study site, more treatments, hospital stays, or complex dosage requirements.
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