New prescription-based medications and medical devices undergo a long, rigorous evaluation process before they ever reach patients. Many require years of research and development, followed by comprehensive analysis during multiple phases of clinical trials. These trials evaluate the safety and efficacy of the new product at hand, and the Food and Drug Administration (FDA) will only approve those that provide both safe and effective treatment. Thus, knowing how to optimize a clinical trial design is crucial for scientists working to bring new medical products to market.
What are Clinical Trials?
Clinical trials are one of the last stages of development before a new medical intervention, such as a drug or medical device, can be approved and sold to consumers. These research studies evaluate the intervention’s safety and efficacy in humans, paving the way for further refinements or FDA approval. Companies wishing to test a new drug partner with a principal investigator, usually a physician, and a team of nurses and other researchers to complete their multi-phase clinical trial. These organizations can then use the results of the trial to determine their drug’s safety, efficacy, and readiness for the market.
These trials require huge investments of time and money, so careful planning is essential. Optimized trial design can help scientists run trials in an accurate, safe, and timely manner while improving their chances for marketing approval by global regulatory agencies, such as the FDA in the U.S. and Health Canada.
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Clinical Trial Design Basics
Asking a few basic questions before designing a clinical trial can set researchers on the right track. Here are three factors that can lay the foundation for a successful clinical trial.
1. Determine your market.
Your first task is to determine your market. Does the target disease have a high or low prevalence, and how much information about the disease is available as a result? Are you studying a drug or medical device? The answers to these questions will help to determine the general parameters of the study.
“In a chronic disease state for which there’s a high level of prevalence, like diabetes, designing a clinical trial follows more or less the same process,” says Stephen Amato, a teaching professor in Northeastern University’s Master of Science in Regulatory Affairs program. In other words, because these diseases are more common and may have been extensively studied already, researchers can typically base the design of their trials on previous trials related to the target disease.
While all researchers have access to FDA support throughout their clinical trials, those studying diseases with unmet clinical needs because of their lower prevalence can take advantage of a closer collaborative relationship with the FDA while designing trials.
“When you’re working in lower prevalence areas, the FDA enables you to work more directly with them to design your study, so the possibility of making a mistake in the design is relatively lower than it would be without the aforementioned collaboration,” Amato says.
2. Define your patient population.
After narrowing down your market, you’ll need to find your patient population. This determination often goes hand in hand with the location in which the study will be completed. Researchers are not limited to their own cities or even countries when conducting clinical trials, and many choose to relocate to areas that have a larger number of qualified patients for them to study.
“Smaller hospitals may not do as many studies, but they may have the patients you’re looking for because that’s often where patients are being treated,” Amato says.
Patients are selected for participation in clinical studies based on specific inclusion-exclusion criteria, which researchers determine based on the specifics of the disease and drug or medical device being studied. These strict criteria help to more specifically define the clinically appropriate patient population and avoid negative side effects that may impact the study’s overall results. For example, researchers may choose to only work with patients who do not smoke to minimize the risk of adverse health effects during the course of the study.
3. Create robust statistical plans.
Statistics play a crucial role in the evaluation of a new drug or device’s safety and efficacy, and the FDA uses statistically analyzed data to determine whether the intervention should be approved for sale. Creating a statistical plan that accurately answers questions about the drug’s side effects, risk factors, and results is therefore a crucial part of the overall design process.
“The statistical plan has to be done way ahead of time, because the FDA doesn’t want either manufacturers or their own organization to waste time to generate data that may not be relevant,” Amato says. “It’s a waste of precious time on the clinical side and could unnecessarily place patients at risk.”
Early planning in this area increases the likelihood that a study’s results will accurately reflect a drug or device’s safety and efficacy, saving time and money that would otherwise need to be spent on additional trials.
Optimizing Your Clinical Trial Design
It’s important to go beyond these basic questions when developing a study to improve its odds of success. Because clinical trials are meant to demonstrate the safety and efficacy of a drug or medical device, each factor involved must be carefully considered to avoid adverse outcomes.
Proper trial design can also save valuable money and time. The median cost of a clinical trial is $19 million, so repeating the trial can be a huge expense. Amato recommends considering the following factors while designing a trial to optimize its results.
Identifying the right group of patients for your study goes beyond determining whether participants have the disease being studied. Additional characteristics, such as whether patients smoke or drink alcohol, how long they’ve had this disease and its severity, and whether they have additional medical concerns all play a role in selection criteria. Understanding a patient’s full medical history helps avoid interactions between the drug being tested and any medications the patient is already taking, creating a safer study for all involved.
“The last thing you want is for the participants in your study to have a toxicity reaction because of what you’re giving them,” Amato says. “That reaction may not occur in folks that don’t smoke, for example.”
A carefully chosen patient population also mitigates the effects of any variables that would make statistical analysis more difficult. Because statistics are so important in proving a drug’s safety and efficacy, variables that could have otherwise been controlled can damage the study’s results and credibility.
Hundreds of thousands of clinical trials are conducted in the U.S. each year, and larger hospitals tend to host a larger percentage of those trials than small, community-based hospitals. While a nationally recognized hospital may lend the study more prestige, it may not be the ideal location if there are not enough patients available to participate.
“Getting the right patients is going to enable you to finish more quickly and get medicines out to people who need them more quickly,” Amato says.
Seeking out smaller hospitals that have higher concentrations of eligible patients, rather than choosing a large one based solely on its reputation, is one way to optimize a study from the very beginning.
Any research team can contact the FDA for guidance while creating and conducting their clinical trials, and Amato recommends getting in touch early in the process.
“Make the FDA a partner in your study process,” he says. “They’d rather have you reach out at the front end and clear something up rather than go all the way through and get to a problem at the end.”
The FDA offers many guidance documents to help researchers optimize their clinical trials. Still, scientists can also reach out directly for personalized support to ensure that their trials are meeting the FDA’s strict requirements for drug approval.
How to Become a Clinical Trial Designer
“Clinical trial design is a team effort,” Amato says. Regulatory and clinical professionals, statisticians, and marketing groups work together to develop the trial, recruit participants, and manage each stage of the trial on a daily basis.
Each role requires a different type of training and specialization. A bachelor’s degree in a field like biotechnology, chemistry, or statistics can help you begin working towards clinical trial participation. Experience is also critical, and Amato notes that it typically takes several years of experience in an area of specialization—along with advanced degrees in regulatory affairs and related fields—to qualify for design roles.
Earning Your Master’s Degree in Regulatory Affairs
Scientists interested in conducting high-quality clinical trials should consider earning their master’s in regulatory affairs, which provides real-world experience and training in various stages of the drug development process.
Northeastern students must have two years of experience before enrolling in the clinical research regulatory affairs concentration within the regulatory affairs master’s program. Students without this experience can instead enroll in one of four additional concentrations and transfer later. All concentrations provide the opportunity to immediately gain experience through Experiential Network projects, internships, and co-ops that can help students advance.
“Even if you come in with a bare minimum of clinical research experience, you’re going to get more professional experience just by going through our program,” Amato says.
Northeastern students can also take advantage of the program’s extensive alumni network. Founded in 2007, the regulatory affairs program has thousands of alumni who can offer career networking and mentorship opportunities to students at all levels.
The program also recognizes the importance of the international medical community. The FDA has encouraged U.S.-based drug manufacturers to conduct trials abroad to ensure their products address the needs of diverse populations, and international students often plan to continue their careers outside of the United States. Northeastern’s master’s in regulatory affairs takes this into consideration, giving students the skills they need to work in a global marketplace.
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