How Drugs, Treatments Get Approval

Clinical trials help researchers identify and refine better treatments

By Deborah Jeanne Sergeant

Every pill, vaccine and therapy prescribed to you by your medical provider once underwent a clinical trial to prove its safety and efficacy. Once the drugs are approved for distribution, pharmaceutical companies use temperature controlled pharmaceutical transport services to have their products transported. While it is reassuring that the process exists, most people know little about how it works.

“Clinical trials are the best way to measure whether a new medication, device or other type of treatment works and is safe,” said Carrie Dykes, Ph.D., director of research services for the Clinical and Translational Science Institute at URMC.

“Clinical trials allow researchers to compare different types of treatment to advance our understanding of medicine and to improve the health of our community. Clinical trials are not just something used when you have no other treatment options. They help researchers identify and refine better treatments.” Pharmaceutical companies may also need the services of an FDA compliance consultant from an FDA regulatory consulting firm to ensure that their facilities and processes fully comply with FDA regulations.

Researchers must carefully select volunteers who fit a profile of the patients that the new drug or treatment would affect. Other factors of eligibility may include absence of certain conditions, age, gender and more.

Dykes said that people lacking good access to healthcare are often those hardest to recruit for trials, such as those underinsured, living in rural areas, older adults, minorities and people economically disadvantaged.

“Since clinical trial visits are often conducted at a healthcare location for safety reasons, people who cannot easily access care are often not included. However, one of the positive outcomes of the COVID-19 pandemic is the switch that many studies have made to conducting visits remotely,” Dykes said. “While not all steps of a trial can be done remotely, this has made it easier for populations without easy access to healthcare to participate.”

Trials are usually conducted in six steps: approval of the research protocol, screening, informed consent, data collection, study closure, and reporting of findings.

Dykes said that an institutional review board reviews every trial conducted in the US. The board is comprised of experts who look at the risks and benefits of the study to protect those participating. Volunteers are screened for their eligibility. The informed consent ensures that the participants understand the study. As the study ensues, researchers carefully monitor patients. In some cases, they remain at a healthcare facility. In most cases, they participate in a number of visits to the facility so researchers can gather information.

“Once all participants have completed all study visits, the trial is closed,” Dykes said. “The researcher then analyzes the data and reports outcome of the trial.”

Participants typically receive a stipend for their time spent.

Trials typically research in phases. The earliest phase is in a lab Petri dish and then with animals. The first human trials use only a small, healthy group and focus on safety. As the phases continue, more individuals are added to the group and some receive the treatment and others (the control group) do not. This allows researchers to fairly compare the efficacy of what they are testing. As the phases continue, larger groups become involved, which can help prove efficacy among a variety of people.

Some groups are difficult to study for ethical reasons, such as pregnant women and babies.

“We don’t do a lot of those types of trials because it’s difficult to recruit patients for,” said Adam Larrabee, president of Rochester Clinical Research. “Companies don’t want to risk the health of pre-term babies.”

Older adults are also challenging to study because they may have a long list of medications and health issues that could skew the results if their group size is too small. But it is also important to accurately represent their demographic which statistically does have more health issues than younger people.

“The elderly are an important part of our community,” Larrabee said. “They need to be represented in clinical trials.”

He reassures anyone interested in volunteering for a study that “you’re not a guinea pig. Participating in a study is a positive experience. We’re not a typical doctor’s office. You don’t come in and wait half an hour or an hour to see someone. We realize people are contributing their time to promote science. We realize their time is valuable and we see them right away.”

He views participating in studies as an opportunity to receive healthcare as well as experience the satisfaction of contributing to health improvements that will affect generations of people.

Medical studies are also highly regulated.

“It’s come a long way for safety protocols,” Larrabee said. “Patients are very closely monitored throughout their participation and their primary care providers are kept informed. We’re not replacing anyone’s doctor but working with them. They’re human beings contributing to the advancement of new medicines. Medications are very, very expensive. And there are a lot of people who are underinsured. It’s expensive to pay for a medication. If you can get that through participating through a study in addition to receiving closer medical surveillance, why not?”

Look for clinical trials at

What You Should Know About Clinical Trials and Research

Submitted by Rochester Clinical Research

What Are Clinical Trials?

• Clinical trials are research studies performed in people that are aimed at evaluating a medical, surgical or behavioral intervention. They are the primary way that researchers find out if a new treatment, like a new drug or diet or medical device (for example, a pacemaker) is safe and effective in people.
• Through clinical studies, doctors find new and better ways to prevent, detect, diagnose, control and treat illnesses.
• Often a clinical trial is used to learn if a new treatment is more effective or has less harmful side effects than the standard treatment.

Clinical Trials: Imperative to New Medical Treatments

• No new medicine, vaccine, therapy or device can make it to market without clinical trials
• Clinical trials ensure safe, effective treatments making it to market for patients

Clinical Trials Used for Myriad of Treatments, Medical Devices and More. Examples:

• Vaccine
• Drug (treatment: migraine relief, arthritis relief, drugs for certain disease, every drug has gone through clinical trials) includes oral, topical, injection
• Device (ex: pacemaker)
• Lab test (for example: a blood test to detect a certain protein)
• Trials for healthy volunteers to find out average base levels of things (ex: specific blood proteins etc.)

Anyone Can Be Eligible for a Clinical Trial

• Clinical trials are not just for those who are diagnosed with a certain illness searching for a new treatment
• Vaccine trials especially seek healthy volunteers

The Process of a Clinical Trial

• Patient is pre-screened (usually by phone)
— Each study has inclusion and exclusions that permit what people can participate (ex: age groups, allergies, etc.)
• If patient gets through the pre-screen with no exclusions (conditions that would exclude them from being eligible to participate in the study) they come in for a screening visit
— At this screening visit they meet with a nurse and doctor or physician assistant or nurse practitioner for a physical assessment, medical history assessment
— If volunteer goes through screening with no exclusions they are enrolled into the study
— Each study timeline is different, volunteer may receive the treatment or drug at this first visit or may not
— Labs are also done at this visit (most studies have labs: blood draws)
• Once patient is enrolled they complete their first visit and come in for visits based upon the study timeline given by the pharmaceutical sponsor
— Timelines range in lengths and frequency
— Length of entire study
— How often the volunteer comes in
— Each study is different!
• At each visit there is certain assessments and labs done
• Typically blood work
• Making sure there are no changes in medications and health
• Depending on the study (if it is to treat something specific) assess how the condition is doing (ex: migraines, are they better? Worse? Gone away completely?)

Why Clinical Trials

• Clinical trials are a great way for people to do their part in advancing medicine and creating a healthier future. It gives back to the world.
• During clinical trials all volunteers receive study-related medical care. Typically a doctor, physician assistant or nurse practitioner will come in and do a physical exam and medical history evaluation to ensure the volunteer is a good candidate for the study.
• Volunteers are compensated for their time and travel. Typically around $100 per visit.
• It’s a way to receive cutting edge medical treatment, whether it’s a drug or a vaccine that is not yet on the market.
• Clinical trials allow patients to be more involved in their care and educated on their health. Education, informed consent and safety of the volunteer is always the top priority.