For patients, new medicines offer fewer side effects, fewer hospitalizations, improved quality of life, increased productivity, and importantly, extended lives. We have partnered with Antidote to help you find and take part in a clinical trial that is right for you. Simply answer a few questions and Antidote’s smart search engine will match you to a trial, quickly and easily.
The rapid pace of scientific advances is enabling a greater understanding of diseases at the molecular level. The drug development process begins when researchers perform laboratory and animal tests to discover how the drug works and whether it’s likely to be safe and work well in humans. Next, a phase of clinical trials begins, and this is where studies are performed on people to determine whether the drug is safe when used to treat a disease and whether it provides a real health benefit.
Along the way, investigators uncover important milestones that may lead to new treatments, but there are also often many dead ends and setbacks, which may lead researchers down a new route, or force them to take a step back. While these stumbling blocks can be disappointing, they are an integral part of a complex research and development process; both the setbacks and successes provide invaluable knowledge that help guide and direct researchers to get one step closer to the next advance.
On average, it takes at least ten to fifteen years for a new medicine to complete the journey from initial discovery to the marketplace, with clinical trials alone taking six to seven years on average. The average cost to research and develop each successful drug is estimated to be $2.6 billion.
Ultimately, though, the process of drug discovery brings hope and relief to millions of patients.
Clinical trials are research studies that use human volunteers. Clinical trials are conducted to collect data regarding the safety and efficacy of new drug and device development. There are several steps and stages of approval in the clinical trials process before a drug or device can be sold in the consumer market, if ever.
Understanding what they are can help you decide if a clinical trial might be an option for you. Alternatively, maybe you have a friend or family member with peripheral neuropathy and are wondering if a clinical trial is right for them.
Why are Clinical Trials so Important to Drug Development?
The U.S. FDA requires that a potential therapy’s safety and efficacy be tested extensively in a large group of human volunteers before it can receive approval to be manufactured and made available to patients. Yet clinical testing can — and often does — fail because not enough people volunteer. Without sufficient numbers of trial participants, the drug development process stalls and a trial must be repeated, scaled back or, even worse, the potential new therapy is abandoned. This lengthens the time it takes for new treatments to come to market. No amount of funding or other resources can compensate for the lack of clinical research volunteers. That’s why volunteers can play a truly unique role at this pivotal stage of drug development, which is crucial for new treatments to reach pharmacy shelves.
Why is it critical that more people volunteer for trials?
Clinical trials play a critical role in the development of new and better therapies. Under-enrollment in trials is one of the greatest challenges clinical researchers face. Under-enrollment in trials slows research progress and deters potential funders from investing in research. We all pay the price in terms of higher costs and longer time horizons to get to therapeutic breakthroughs. Across all diseases, 85 percent of clinical trials finish late due to difficulties enrolling participants and nearly one-third of trials fail to recruit a single subject and cannot ever begin.
Clinical trials are conducted in a series of steps, called phases – each phase is designed to answer a separate research question.
We’ve provided information about clinical trials to help you understand what a clinical trial is, its different stages and how you can get involved. To TAKE ACTION and find a clinical trial near you visit ClinicalTrials.gov a registry of publicly and privately supported clinical studies of human participants conducted around the world.
Phase 1 Clinical Trials
In Phase 1 trials the candidate drug is tested in people for the first time. These studies are usually conducted with a small number of healthy volunteers, generally 100 or less. The main goal of a Phase 1 clinical trial is to assess the safety of the medicine when used in humans. Doctors/researchers look at the pharmacokinetics of a drug: How is it absorbed? How is it metabolized and eliminated from the body? They also study the drug’s pharmacodynamics: Does it cause side effects? These closely monitored trials are designed to help researchers determine what the safe dosing range is and if the candidate medicine should move on to the next stage of development.
Phase 2 Clinical Trials
In Phase 2 clinical trials, doctors/researchers evaluate the candidate drug’s effectiveness in 100 to 500 patient volunteers. Many Phase II trials study patients receiving the drug compared with patients receiving a different treatment, either an inactive substance (placebo), or a different drug that is usually considered the standard of care for the disease. Doctors/researchers also analyze optimal dose strength and schedules for using the drug and examine the possible short-term side effects (adverse events) and risks associated with the drug. If the drug continues to show promise, they prepare for the much larger Phase 3 trials.
Phase 3 Clinical Trials
Phase 3 clinical trials generate statistically significant data about the safety, efficacy and the overall benefit-risk relationship of the investigational medicine. Phase 3 trials may enroll 1,000 to 5,000 patients or more across numerous clinical trials sites around the world. This phase of research is essential in determining whether the drug is safe and effective.
Each patient enrolled in a Phase 3 clinical trial has a chance of being in one of the following groups:
- Control group – the group that gets the standard treatment
- Study group – the group that gets the new treatment being tested
- Doctors/researchers do not know if the new treatment is better than the standard treatment, but they believe it is as good and may be better.
After the Phase 3 trial, the FDA reviews the clinical trial results to make sure the treatment is safe and effective for people to use. The FDA decides whether to approve the treatment so that it is available for all patients.
There are many frequently asked questions about Phase 3 trials. These include:
How are patients put into groups?
A computer decides which patients are in the control group and which patients are in the study group. Patients have a chance of being in either group. The patient and doctor do not decide. It is random and due to chance alone. This helps to avoid bias in the clinical trial. (Bias happens when human choices affect a study’s results.)
Would my doctor know which group I am in?
In single blind studies, patients do not know whether they are in the control or study group, but the doctor does. In double blind studies, neither the patients nor the doctors know which patients are in each group. (In case of an emergency, doctors can find this information in the study file.)
Would I be given a placebo?
A placebo is something that looks like medicine, but is not. If a placebo is used, it is given together with the best standard treatment. This allows doctors/researchers to compare standard treatment alone to standard treatment with a new drug.
Phase 4 Clinical Trials
In Phase 4 trials, doctors/researchers study treatments that the FDA has already approved. The goal of Phase 4 clinical trials is to continue studying side effects of a new treatment.
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