Scientists around the world discover new breakthroughs in medicine daily, and the rate is accelerating! 20 years ago, there were roughly 4,000 drugs in research. Today, over 15,000 exist in the pipeline.
The different phases of research looks roughly like this: A new drug compound is analyzed in labs to determine if, based on the chemical structure and function, it is similar to other approved drugs and, therefore, can be expected to perform in a predictable way. The new drug is first given the approval to proceed to animal research. A pig’s biological system is very similar to our own. Decades of prior research tells us that if the drug is processed a certain way in a pig, it should be processed in a similar fashion in a human. A pig’s heart, blood vessels, kidney function, and insulin-production are all similar to humans. Thus, why you are seeing an increase in pig organ transplant research. If animal research confirms a benefit, the FDA will allow it to proceed to formal clinical research trials.
It is important to note, both healthcare and clinical research are heavily regulated by governing agencies. In clinical research, there are regulatory bodies such as the institutional review board (IRB), the ethics review board (ERB), research ethics board (REB), and Federal Drug Administration (FDA) that provide oversight to all clinical trials and research. Only these research, ethics and governmental agencies can decide if a trial supports or rejects an advancement and/or treatment in medicine. The IRB investigates all researchers and ensures that they have the training, expertise, processes and support team to effectively execute a trial. These governing bodies are consistently performing a risk-benefit analysis. Throughout every step in a trial, live data is monitored to verify the effectiveness of the research team, the reliability of the data and to confirm safeguards are in place to ensure the safety of the trial participants or patients. Even if a company thinks they have developed a cure for cancer, they cannot jump ahead of the gated process and start administering it to patients. All must start at the beginning of this process with Phase 1 and can only proceed if the results of each of the three phases of research demonstrate continued safety and efficacy.
There are several phases of research and numerous trials within each phase to evaluate specific populations:
Phase 1 trials are the first time the investigational drug is given to humans – approximately 10-50 patients. The sole purpose of Phase 1 is safety. This trial starts small and proceeds slowly. If it holds to the model, then the request to proceed to Phase 2 goes to the regulatory bodies.
Phase 2 is about safety as well as effectiveness. These trials are larger and include several hundred patients. Based on Phase 1 results, it can be expected that the effective dose will be X, Y, or Z in patients with certain conditions. Questions are asked: Is a certain dose more effective than another? Did it produce the same positive outcomes with fewer side effects? This research takes place globally in hundreds of research sites simultaneously. The results are combined to find the answers to multiple questions. At the end of Phase 2, all data are analyzed for safety and effectiveness. If it meets the criteria, it can proceed to Phase 3.
Phase 3 continues to evaluate safety and efficacy but has the additional purpose of homing in on the perfect dose. This phase is among the largest of the trials and can include thousands to tens of thousands of patients. A typical Phase 3 trial evaluates the specific dose that is believed to be the most effective with the fewest side effects. Phase 3 trials are frequently global and can take years to complete. It is only at the end of Phase 3 that an application can be made to the FDA in the United States, or equivalent regulatory bodies in Europe or Asia. If the FDA agrees the research supports that the new drug is effective and safe for patients, then all the years of hard work pay off. Both for patients and researchers.