What are clinical trials?
Clinical trials are designed to determine whether a medical, surgical, or behavioral intervention is safe and effective in humans.
Regulators and researchers are constantly looking for ways to speed up the pace of clinical trials and reduce overall costs, with the goal of bringing life-saving treatments to patients faster.
As of September 2022, ClinicalTrials.gov, a database of private and publicly funded clinical trials worldwide, had more than 20,000 trials registered.
These studies spanned 221 countries, recruited millions of volunteers worldwide, and tested everything from simple behaviors like exercise routines to complex interventions like gene therapy. All followed a similar multi-step process to assess their impact on human health.
Types of Clinical Trials
All clinical trials have a primary goal. These can be divided into the following categories:
Treatment: Testing new treatments, new combinations of drugs, or new approaches to surgery or treatment
Prevention: Researching ways to prevent or improve the recurrence of disease using, for example, drugs, vitamins, or vaccines.
Diagnosis: Finding Better Testing Techniques and Methods for Diagnosing Diseases and Conditions
Examination: Finding the Best Method for Detecting Specific Diseases or Health Conditions
Supportive Care: Investigating Interventions to Improve Patients’ Comfort and Quality of Life with Chronic Diseases Health Services
Basic Science: Overview of Intervention Activity Research: Assessment of Health Care Delivery, Process, Management, Organization, or Financing
From Discovery to Treatment: The Clinical Process
Basic research can often lead to the discovery of new drugs, whether on purpose or by accident. Between basic research and the start of a clinical trial are “preclinical studies,” which involve laboratory tests on cells or animal models to determine whether a drug is beneficial and toxic before testing it in humans.
This process takes several years and ends when researchers submit their data and detailed clinical trial plans to a regulatory agency, such as the US Food and Drug Administration (FDA).
Protocols for Clinical Trials
Clinical trials follow a plan known as a protocol. The protocol is carefully designed to balance the potential benefits of the trial against the risks for the participants. It also answers specific research questions. The protocol describes Information about tests, procedures, and treatments Eligibility requirements
Expected Duration of the Study:
- How long will the study last
- Study objectives
- Information collected
- protection of participants from risks.
- The clinical trial team is led by a principal investigator ( PI). Members of the research team regularly monitor the health of participants to determine the safety and effectiveness of the study.
Clinical Trials Design
There are different types of clinical trials and research plans. However, many clinical trials include standard design elements.
Blinded or masked studies are designed to prevent research teams and study participants from influencing the results. Blinding allows for the collection of scientifically accurate data.
In single-blind (single-mask) studies, you are not told what you are being given, but the research team does.
In double-blind studies, neither you nor the research team are told what you are being given; only the pharmacy knows that. Members of the research team are not told which participants will receive which treatment to reduce bias. However, if it is medically necessary, it is always possible to find out what treatment you are receiving.
Randomization is the process by which participants are randomly assigned to a treatment rather than one or the other. This is done to avoid bias in the assignments. The effects of each treatment are compared at specific points during the trial. If one treatment turns out to be better, the study is stopped so that all volunteers receive the more beneficial treatment.
Clinical Trials Phases
Researchers conduct clinical trials in stages called “phases. Each step has a different purpose and helps researchers answer different questions.
- Researchers test a drug or other treatment on a small group of people for the first time. The goal is to obtain information about the best dose of a drug or other treatment, as well as about its safety and side effects.
- Researchers study a new drug or treatment in a larger group to determine its effectiveness and further study its safety.
- Researchers give a new drug or treatment to a larger group of participants to make sure it works, monitor side effects, compare it to conventional or similar treatments or a placebo, and gather the information that makes the new drug or treatment possible and safe to use.
- After a drug or treatment is approved by the US Food and Drug Administration (FDA) and available to the public, researchers check its safety in the general population to learn more about the benefits and optimal use of the drug or treatment. Phase 4 is designed to monitor long-term safety and detect side effects that may not appear until years later.
Although timelines vary widely, the first three phases of clinical trials can take 10 to 15 years, costing companies anywhere from $1 million to $2 million to more than $30 million.
This process may seem overly complicated when determining something as simple as a drug’s health effects, but every step is carefully calculated, including sterile manufacturing practices, ethical patient consent, and clearly defined endpoints and measurements. But even within this tight structure, there is room to experiment and experiment with new experimental designs aimed at speed and cost reduction.
New Advancements
About 86% of drugs in clinical trials do not receive FDA approval.
This has significant implications for how drug companies approach clinical trials, as the average total cost of developing a new drug is estimated to be between $2 billion and $3 billion.
Although clinical trials are highly regulated processes, innovation in trial design and active collaboration with regulatory agencies can create flexibility to test new ways of evaluating drugs.
Unlike traditional clinical trials, which are fixed and unchanging during the trial, adaptive trials are flexible and adaptive, sometimes testing multiple drugs at the same time. As new information emerges, adaptive trials can reduce or increase doses, change the length of the study, or change patient populations, which has proven essential to the rapid response to the COVID-19 pandemic.
Advances in wearable health devices and artificial intelligence technologies to collect and store large amounts of real-world health data are impacting the design and implementation of clinical trials.
Companies work with regulatory agencies to turn this data into real evidence, providing new information about a drug’s safety and long-term effects that traditional studies may not detect in such detail.
The COVID-19 pandemic has accelerated the adoption of decentralised clinical trials, bringing some of the research activity to patients rather than bringing patients to the study site.
Virtual technologies and services such as telehealth and remote patient monitoring have enabled decentralization, potentially expanding access to research and improving participant diversity, a perennial problem in drug development.
Increasing the diversity of individual clinical trial participants, especially in the United States, to reach communities with the highest burden of disease requires the transfer of clinical trials to other countries.
Making better medicine for more patients
According to a World Health Organization report, in 2021 the number of trials registered in high-income countries (HIC) was almost 100 times higher than in low-income countries.
Medicines developed in HIC regions should not be expected to perform similarly in low- and middle-income countries (LMICs) due to the regions’ enormous biodiversity. To identify and address these disparities, international collaboration and adaptive clinical trials will be used to create sites across LMICs to help pool resources, share knowledge, and accelerate knowledge acquisition.
Long-term efforts to build clinical trial capacity (e.g., regulatory systems, manufacturing capacity) in LMICs are critical to developing drugs that are safe and effective for specific populations and can be used in LMIC health systems.
