Understanding clinical trials

It takes years of intense data-driven research before an experimental therapy or medical device is commercially available. Before these treatments hit the market they must be vetted through clinical trials.

Those trials explore whether or not a medical treatment or device is safe and effective for patients to use in their treatments. The studies follow strict standards that are overseen by regulatory authorities, including independent monitoring committees and other boards. Additionally, the U.S. Food and Drug Administration plays a key role in each step of the clinical process and determines whether or not the experimental treatment can move forward. Of all the programs that move into “the clinic,” as human trials are often called, only about 10 percent make it through to regulatory approval — which demonstrates the rigors of the process.

Products begin in the developmental phase then move through preclinical work, typically using animal models. If the preclinical data is strong enough the FDA will give the OK for human testing, which is typically performed in three phases. If a potential drug or device makes it through all phases, then the product will move before multiple FDA panels before it is approved for marketing in the U.S.

Trial endpoints are quite specific in order to test the safety and efficacy of the treatments. Likewise there are strict protocols on what type of patient can be enrolled. In an ideal setting the trial patient population should be as homogenous as possible. Trials typically begin with small groups until safety has been established. Once safety goals are met, the patient population will expand before efficacy is determined. Patients will typically be divided into two groups, a control group that receives a placebo and a group that receives the experimental drug. The second group can be further divided based on dosing strength of the developmental treatment. This way data results will have a similar baseline and researchers can determine how effective the treatment works in the patient pool. When the research data is accumulated at the end of the trial, homogeneity of patient population will help researches be more certain that any differences in the patient groups can be attributed only to the drugs.

Selecting patients for a trial has become a science of its own, particularly as researchers have learned that men and women of various ethnicities can respond differently to medical treatments. Children can also respond differently to a treatment than an adult. As such, children are typically in their own patient populations.

Patients who agree to participate in a trial can have access to some potentially cutting-edge medicines, such as the latest in immuno-oncological treatments such as the recently approved chimeric antigen receptor T-Cell Therapies (CAR-T). The T-cells are engineered in a laboratory to recognize a specific antigen in a cell and then administered into the cancer patient. The CAR-T cells then multiply within the body and target the antigen and eliminate the cancer threat. So far though CAR-Ts have only shown efficacy in targeting B-Cell lymphomas and not solid tumors. Still, the benefits are enormous for patients who can receive the therapy, with some patients seeing an elimination of their cancers.

When a new treatment is finally approved and available for patients, prescribers will have to carefully note the indications for which a drug is approved. Many medical treatments, particularly in oncology, are highly specific as to what type of patient will benefit given the specific nature of the disease. Prescribers, as well as benefit providers, will also be keen to note the cost of a newly approved drug and whether or not there are benefits programs offered by the manufacturer.

Oftentimes, once a drug is approved by the FDA its journey of discovery is not over. Researchers often take the drug back into the clinic to determine whether or not it is effective at treating other disease indications. Drugs can also continue through a post-approval trial to gather long-term data about the drug’s safety and efficacy.