What is clinical research?
Clinical research is an area of biomedical research that studies how humans respond to experimental therapies. This is also known as human subjects research.
Usually, clinical trials are designed and performed. Clinical trials test the efficacy of a specific treatment or intervention in a group of patients (research subjects, which represent a sample) and inferences are drawn about the use of treatment in the larger, general population.
There are earlier forms of experimental research, such as computer simulations and animal models that are typically performed before human subjects research. This is known as preclinical research.
For example, if a new asthma drug was developed, it would typically need to have safety and dosage data from laboratory animal subjects, before it can be tested in a small group of human subjects.
There are also multiple phases of clinical research, which we will discuss below.
There are standards such as Good Clinical Practice (GCP), which seek to set a high standard for the integrity of the collected data and the protection of patient’s rights.
This is part of a larger series known as the Autodidact Project. For more information about this journey and to join — see this article:
Why is it important to learn about clinical research?
Before a new drug or medical procedure can be sold, its claims must be proved in clinical trials. The biotechnology, pharmaceutical and healthcare industries rely on peer-reviewed evidence and data generated from well-conducted clinical trials.
Without this, new therapies from biopharma companies and technology vendors are treated with skepticism — as they should, until they are proven in the clinic to be safe and effective.
If you are a health provider who wants to better evaluate scientific evidence to judge the efficacy of new treatments and health interventions that you may recommend, it is wise to study the basics of clinical research. It also recommended that founders of biopharma and technology companies who seek to develop experimental treatments also study these concepts deeply.
Clinical research is highly regulated, by groups such as the Food and Drug Administration (FDA) and other agencies, before new products can advertise their claims and go to market.
Local institutions have Institutional Review Boards (IRB) which are safety committees that must approve human subjects research before clinical trial participants can enroll in studies. Their job is to ensure study designs are safe and ethical, before they are approved to be launched at the hospital or university (that hosts the IRB). It usually takes 3 to 6 months to get an IRB committee to approve a human subjects research submission.
If your project is human subjects research and offers a new treatment (or combinations of treatments) to patients, odds are it must go through the clinic or hospital’s IRB. Study this deeper to confirm this.
If a clinical study is not approved by an IRB, it cannot enroll patients. It usually cannot be published in a peer-reviewed journal.
Types of clinical research?
There are many types of clinical studies. From small pilots that test how a few patients respond to a new treatment to large multi-center studies that recruit hundreds to thousands of patients.
Multiple study designs exist. These may include various forms of randomization, blinding and control groups.
It is recommended to study the four phases of clinical trials. This process must be followed to get a new drug approved by the FDA.
Phases:
Phase 1 — small studies that test new therapies in humans, usually without a control group.
- Objective is to find the maximum tolerated dose (MTD) to aid in further drug refinement. The MTD is the dose that can be administered without causing unacceptable side effects.
- A priority is to report all adverse events, for various dose levels. What side effects do patients have?
Phase 2 — studies that assess the therapeutic activity of new treatments
- How well does this drug perform?
- Assesses treatment efficacy and treatment.
- Usually recruits a small number of patients.
Phase 3 — large studies, usually randomized, with more than one treatment arm
- These are comparative trials — which compares one treatment group (arm) against other treatment groups in the trial.
- Usually involves random assignment of treatment.
- The control group is usually the “current” standard care for a disease.
- Usually blinding is performed, so patients and/or physicians do not know what treatments the patients receive.
- Often recruit many patients, this large sample size is used to generate statistical power to make higher quality inferences.
Phase 4 — post-marketing surveillance trials
- These collect additional information on short and long-term side effects of the treatment in the general population.
What should I learn?
Use the answers to this question as a study guide and knowledge check. If you seek the answers to these questions and complete the recommended courses below, you can build a strong working familiarity with clinical research.
- What is the difference between research and routine clinical practice?
- What is informed consent?
- Explain the ethical principles in the Belmont Report.
- What are the rights of a research subject in a clinical trial?
- What is HIPAA and the specific patient identifiers that must be protected?
- What is the difference between RHI and PHI?
- Can medical records be reviewed without informed consent?
- Who are the typical members of a clinical trial team and their responsibilities?
- What is the purpose of an IRB?
- What are inclusion and exclusion criteria?
- Why is randomization so important?
- Why is a control vs. intervention group design essential for a high-quality clinical trial?
- What is a conflict of interest and how can it be managed?
- Why should case report forms be developed before a study begins?
- Explain how computers can be used to facilitate the conduct of clinical research.
- What are areas of biomedical research that need special protection?
- What are the typical elements that must be assembled in a research protocol?
- What is GCP?
- What does the FDA regulate and are the phases of clinical trials?
What are recommended courses?
In order to become a better biomedical researcher and designer of medical interventions, I recently took many courses and books to study this field more deeply.
It helped me work with doctors to design a new clinical trial and serve as a Principal Investigator (PI). This knowledge will also help me develop many medical technologies in the future. If you seek to do the same, I would consider studying these.
Here are my recommendations:
CITIprogram.org, Online training
- CITI is a website that offers research ethics and compliance training, for many universities. It is considered an authoritative resource in research training.
- Many universities have bought access licenses for their staff and students to access various online modules for free. Login to your university account and contact your local IRB to learn more.
- My institution requires completing 2 certificates from citiprogram.org, before a staff member can be listed on an IRB submission.
- I completed 10 certificates and recommend starting with “Biomedical Research — Basic / Refresher”. You may complete additional courses as necessary.
- Website Link
Management of Data in Clinical Trials, by Eleanor McFadden [Wiley series]
- Now that you are familiar with the basic concepts of clinical research, it is time to dive much deeper into the logistics of clinical research.
- Topics include design of case report forms such as eligibility checklists, informed consent forms, statistical analysis plans and so much more.
- Amazon Link
Understanding Clinical Research, by Renato Lopes [Lange series]
- To complete your exploratory training, this book will cover a review of major topics studies earlier.
- It is also much more approachable that the other 2 resources and will give you a viewpoint on various topics, such as ethics and clinical trial history in more detail.
- Amazon Link
What are opportunities for further involvement?
If you are a high school or college student, consider interning at a local research laboratory. There are many at academic universities and colleges. Summer internships are also very popular learning opportunities.
Six years ago, I helped develop a mentorship nonprofit to expose students to academic research and social entrepreneurship. If you seek to learn or mentor, check out your local SENDforC chapter (webpage: sendforc.org).
If you are a physician, any kind of clinician or an academic professor — you may consider collaborating with your colleagues to launch your own clinical studies. Contact your academic department, local IRB or other resources to learn more.
Otherwise, you may consider working as a clinical research associate (CRA) to assist in the conduct of clinical trials. You may also consider graduate school if you seek to study biomedical research or other topics much deeper.
My research lab -
I recently started my own online laboratory dedicated to advancing digital health research. It is known as the Umano Lab for Digital Health (webpage: umanolab.org). If you are a clinician or a software engineer with ideas to benefit patient care and want to help design new software pilots to improve clinical outcomes, checkout the website.
Graduate students and opensource engineers are paired to the highest ranked proposals from nurses and physicians. We hope to design new web, mobile and machine learning applications to benefit patients at numerous healthcare organizations.
Good luck with your studies!
Contact me with any questions or comments you have.
