Written by Amy Rehan,
The pharmaceutical industry has experienced an enormous transformation, but one thing that remains undisputed is the importance of safety. Drug development involves rigorous testing, continuous monitoring, and adherence to health standards to ensure human safety.
This is why a new drug or medical product is tested even before it reaches the clinical trial stage. This is called preclinical research, and it has immense importance in the pharmaceutical industry. Here’s a short guide explaining preclinical research, its timeline, and significance:
What is Preclinical Research?
Preclinical research is a broad term that encompasses all the studies conducted on a drug or a medical device before it can be tested in humans.
It typically consists of four steps, including:
Target Identification: This is the process of finding a specific biological molecule (protein, RNA, or gene) that modulates the target disease.
Hit Identification: The compounds that show the most promise during the target identification phase are called “hits.”
Lead Optimization: Drugs that show considerable effects move onto the lead optimization process. Researchers attempt to understand the Structure-Activity-Relationships (SAR) of different hit series.
IND-enabling Studies: Leads with the most supportive preclinical data move to an advanced phase of testing called investigational new drug (IND)-enabling studies.
Learn more about preclinical research and contract research organizations (CROs) that provide these services here.
Vitro and Vito Studies
During preclinical research, both vitro and vivo studies are utilized. Vitro studies are conducted outside of a living organism, such as a laboratory setting. This can be a test tube or a petri dish. Researchers analyze cell cultures and tissue samples. Vitro studies are more controlled. Researchers can analyze specific biological processes without dealing with the complexities of the whole organism.
On the other hand, vivo studies involve testing within a living organism. In case of preclinical research, vivo studies are conducted on animals, mainly mice. Vivo studies allow researchers to evaluate the toxicity, efficacy, and safety of a drug or treatment on a whole organism.
When is Preclinical Research Conducted?
As the name indicates, preclinical research is conducted before any human testing of a drug or a medical device. The main reason is that human clinical trials require a large amount of money and time. They also rely upon the willingness of the participants. This is why extensive vetting of drugs before the start of a clinical trial is important.
Why is Preclinical Research Important?
So how does preclinical research facilitate the advancement of pharmaceutical studies? Here are some ways:
Risk Mitigation and Safety Assessment
Preclinical research involves rigorous safety testing. This helps identify potential toxicities and adverse effects early on. Researchers can also identify safe dosages early and streamline human testing.
Efficacy
Preclinical research heavily focuses on identifying a drug’s pharmacokinetics and pharmacodynamics. In simple terms, it helps identify how a drug reacts in the biological system. Researchers can make modifications to get the desired therapeutic effect in a disease model.
Regulatory Compliance
In the United States, pharmaceutical companies are required to submit IND-enabling studies to the Food and Drug Administration (FDA) before human trials can begin. Therefore, preclinical research can ensure regulatory compliance, saving you from legal issues later on.
That said, make sure you work with a preclinical research provider that complies with Good Laboratory Practice (GLP) guidelines to ensure the reliability and efficacy of data.
Cost and Time Efficiency
Running and managing human clinical trials is time-consuming. You don’t want to face setbacks after such extensive research. Preclinical research identifies problems with the drug early on. This will save time and resources and ensure excellence in healthcare.
Preclinical Research: Challenges and Limitations
Preclinical research comes with its fair share of challenges. This includes:
Inadequate Animal Models
As mentioned earlier, vivo studies are critical for testing the reliability of a drug. Unfortunately, it is difficult to find animal models that accurately reflect human diseases. When models fail to capture the complexity of human functions, researchers end up with false or misleading results.
Moreover, physiological and biochemical differences between animals and humans can make it challenging to acquire accurate findings.
Issues with Reproducibility and Reliability
Preclinical research consists of numerous steps and processes. Not all processes and relevant bodies follow the same protocols. This lack of standardization across different research groups reduces the reliability of preclinical research findings.
In many cases, small sample sizes also make it difficult to apply the results to a general, much larger population.
High Costs
Preclinical research can be expensive due to various reasons, including the need for specialized equipment, facilities, and personnel. Pharmaceutical startups and new companies face the issue of acquiring funding. This can render delays, increasing operational costs.
Regulatory Hurdles
Even after the research side of things is prepared, researchers often face regulatory hurdles. For instance, submitting an Investigational New Drug (IND) application requires time and effort. Without specialized expertise and meticulous documentation, the research timeline can be unnecessarily extended.
Ethical Considerations in Preclinical Research
Understanding and implementing ethical considerations during preclinical research is important. This involves adhering to the 3Rs:
Replacement – Replacing animal testing methods with cell cultures whenever feasible.
Reduction – minimizing the number of animals used for testing during preclinical research.
Refinement – implementing pain-free procedures and optimizing housing and handling.
When human tissue cells and cultures are used, informed consent is critical.
Lastly, ethical considerations make sure that preclinical research findings actively contribute to scientific knowledge and the improvement of animal and human health.
Dedication to Excellence
Preclinical research is the first step towards developing safe and effective drugs. They help pharmaceutical researchers identify shortcomings, mitigate risks, and comply with regulatory guidelines.
Despite facing numerous challenges, such as inadequate animal models, high costs, and regulatory setbacks, researchers and the entire pharmaceutical community are committed to conducting preclinical research before human trials can begin.
Preclinical research is no longer an option: It is critical for safe drug development and usage.
Author’s Biography
Amy Rehan is a healthcare researcher and the Content Head at Globex Outreach, where she has spent the past three years carefully curating content for pharmaceutical research facilities. She brings clinical knowledge and marketing expertise to help companies grow.
Please also review AIHCP’s Health Care Ethics Consultant Certification program and see if it meets your academic and professional goals. These programs are online and independent study and open to qualified professionals seeking a four year certification