Clearly defining the research question, minimizing variation, planning for interim analyses, choosing entry criteria, selecting participants and implementing monitoring are critical aspects of clinical trial design. This course will give you the practical knowledge to contribute effectively to a clinical trial. This program follows the activities of a fictional clinical study team to illustrate key clinical trial concepts and regulations.
Definition of a Clinical Trial
Clinical trials are studies that look at how well medical therapies work on actual individuals. The process might be a new drug, device or procedure. It might also compare an existing treatment to a placebo. Some trials involve only healthy people, while others require people with a particular disease or condition. It can take months, sometimes years, to determine whether a new drug or other intervention effectively treats a specific health condition.
Before a trial begins, the researchers prepare a study plan. The plan explains what will be done during the trial and why it is necessary. Then they find the right people to participate in the trial. It can take time, as the researchers must find volunteers who meet the specific criteria for each study. It includes ensuring that the number of healthy people is sufficient to show any effect and that enough patients with the disease or condition are included so the researchers can make accurate comparisons.
The people participating in clinical trials and studies must represent the population using the tested medical products. It is why the FDA encourages participation by people of all ages, races and ethnicities. Some of these efforts include setting up clinical trial networks and working with racial and ethnic minority community organizations.
Planning a Trial
The research team must carefully plan each step to conduct the trial ethically and safely. It includes designing the study and choosing participants based on specific health conditions. All clinical trials have eligibility criteria guidelines describing who can or cannot participate in the study. These criteria help reduce the number of people who might get sick or react from participating in the study. The study must also be monitored by a group of experts known as the data monitoring committee (DMC).
The DMC keeps track of how the trial is going and ensures that the trial is safe for patients. Proper planning can be seen as tedious and time-consuming, but it is vital to the success of a clinical trial. Designing a clinical trial requires a lot of expertise in areas like medical and regulatory requirements, project management and more. It is especially important for multicenter and multinational trials.
To plan a successful trial, a feasibility study should be performed to assess the viability of the study. It is important to identify a lead investigator and prepare the protocol for the study. The study should also be discussed with potential sites and designed for site qualification. Lastly, a resource plan should be prepared, including contingency plans for unforeseen events.
Conducting a Trial
In some countries, a local ethics board or Institutional Review Board (IRB) reviews and approves clinical trials before researchers can begin. The IRB ensures that the study has a low risk of harm to participants and that the potential benefits outweigh any risks. The IRB also may require changes in how researchers explain the study to prospective participants and describe any possible dangers.
Some studies are done in a series of steps, called phases, to keep the risk to participants as low as possible and to help answer different research questions. Each phase usually includes a smaller group of participants than the previous one. The number of participants in a study is important because it affects the ability of the researchers to detect differences between groups. It is referred to as the statistical power of the study.
Participants must understand the key aspects of the study, including its purpose, duration, required procedures, risks, and benefits, and they must give their informed consent before participating in the trial. Informed consent is often documented by signing a document that contains this information. It is a legal requirement in most countries, although it is only sometimes required in noninterventional studies (those using previously collected data). The IRB must review the informed consent process before the study begins, and the investigator must submit a yearly Continuing Review Report to the IRB detailing any changes to the study.
Managing a Trial
A large clinical trial requires a significant investment of human and financial resources. It also involves risk, so many things can go wrong during a test. One way to mitigate these risks is to create a detailed project plan before the study launches. It can include a timeline, key dates, and a task schedule. Several online tools are also available to help teams keep track of details and stay on track. Another important part of managing a clinical trial is monitoring and responding to issues that arise during the study.
It can include finding potential solutions, analyzing the impact on the timeline and budget, and communicating with affected parties. A project manager can be instrumental in this process by creating an issue management plan and establishing an ongoing communication process. A project manager can also use the RACI method to organize stakeholders into four categories based on their roles and communication needs.
Finally, managing a clinical trial requires ensuring appropriate steps are taken to recruit participants. It can include utilizing a database of potential participants, placing ads in newspapers or on the radio, putting flyers at places where patients may be found (such as doctor’s offices), and personal recruitment by investigators. Clinical trial sponsors and contract research organizations that work on their behalf may also use outside contractors to help with this procedure.