Clinical Trials, inDepth

Sarah Taj

Macaulay Honors Seminar Three

Professor Joyner

Clinical trials are done to understand the safety and efficacy of a new drug. In order for a drug to be approved in the clinical trial process, it must undergo and pass a number of stages and steps. The organization most responsible for the drug approval and developmental process is the FDA, food and drug administration.  Established in 1906 by the passage of the Federal Food and Drugs Act, the FDA “supports the pharmaceutical industry in every step of the process” (U.S. Food and Drug Administration, 2018). The reason why this organization is crucial is because it is the final step in the drug approval process.

The pharmaceutical development process begins with extensive hours in the laboratory researching the effects of the potential drug and testing it on animal and human cells. After many months, or even years, if the research is deemed as successful, the data is sent to the FDA (Food and Drug Administration) for further approval to begin testing on human subjects. When the research is approved by the organization, the stages of human testing can begin. This process is typically four phases. Furthermore, after every trial, the researches must submit their findings to the FDA for approval before continuing on to the next stage of the process.

The first stage of the human trials understands the safety of the drug. In this initial stage, the FDA states that “20 to 100 healthy volunteers or people with disease/condition will be chosen to participate in the study.” (Food and Drug Administration, 2018). The length of this stage can last up to several months and it is to understand the levels of safety and effects of dosage. With this, researchers will gain a better grasp of how the pharmaceutical drug is able to be metabolized by the body. It is also reported that “approximately only 70% of drugs move on to the next phase of human trials” (Food and Drug Administration, 2018).

The second stage of human trials’ purpose is to further comprehend any possible side effects the drug may have on the test subjects. Because unknown potential side effects can be incredibly dangerous if left unknown, the FDA requires that “up to several hundred people with the disease/condition” (Food and Drug Administration, 2018) should participate in this step of the study. In this stage of operation, the experimental and control group are formed. From the several hundred participants, half are labeled as the experimental group while the other half are labeled as the control group. In this blinded process, neither the participant nor the researcher knows which group is which. This allows the FDA to understand the drug’s actual effects omitting the effects of any biases.

In this stage, for both the experimental and control groups, samples and tests are taken to track the participant’s progress. Furthermore, to have a closer understanding of the drug’s effect, researchers may even “ask you to keep a log of your daily activities and symptoms” (Cancer Net, September 2017). The FDA notes that “only 33% of drugs move on to the next trial”. This is because the side effects are a major determining factor of whether the drug is safe enough for mass consumption. The level of risks is considered and is the biggest filter in the human trials process that disqualifies a majority of drugs through the next stages of testing.

Phase three understands the drug’s effects relative to the standard treatment available for that specific disease or illness. In this stage, the participants are sectioned into two groups. The first group will be treated with the best, standard method that is already available to the public. The second group will be given the potential, tested drug. In this, tests will be taken to understand the different factors between the two. If the new drug does not offer any new benefit but rather has a longer list of side effects, it will fail this stage and will not move on to the final stage of screening. This phase can take a number of years and because it is close to the final stages of approval, requires several thousand volunteers ranging in various ages, races, and ethnicities. The success rate for this stage ranges from 70%-90%. If the drug passes this level, “a pharmaceutical company can request FDA approval for marketing the drug” (Center Watch, 2017).

The final stage of screening is, according to Dr.Viraj Suvarna, Vice President Medical at Eris Lifesciences, the most “important phase of drug development”. In his article, Phase IV of Drug Development, he explains that the final stages display the “real world effectiveness” of the drug outside the confines of a lab. The FDA often titles this phase as the Post Marketing Surveillance Trials. In this final step, researchers map the long-term effectiveness of the drug without interfering with it. There is close monitorization here simply because it is available to the general mass. If the findings show any sign of unpredicted harmful effects, the drug is quickly taken off the market and further studied to prevent the spread of its effect.

The long, rigorous four phases of drug development require an extensive amount of funding. Majority of the drugs undergoing this operation are funded by the government and philanthropic organizations. The National Center for Biotechnology states, however, that “the period between discovery and proof of concept, however, is considered extremely risky and therefore has been difficult to fund” (NCBI, Drug Development for Rare and Neglected Diseases, 2009).  With governmental funding remaining flat for a number of years now, it becomes more difficult to fund many of the potential drugs in need of undergoing the screening process. This why private pharmaceutical companies and venture capitalists are beginning to become the source of funding for many of the newer drugs on the market. Regardless of what the trials are funded by, however, it remains crucial to prevent any shortcuts regarding the approval process headed by the FDA. The health and safety of the public should remain a priority and every new drug introduced to the market should have an extensive history of screening.