About Clinical Trials

About Clinical Trials

Clinical Trials

Clinical trials are the final step in a long process that begins with research in a lab. Before any new treatment is used with people in clinical trials, researchers work for many years to understand its effects on cancer cells in the lab and in animals. They also try to figure out the side effects it may cause.

Before any new treatment can be made widely available to patients, it must be studied in clinical trials (research studies) and found to be safe and effective in treating disease. Clinical trials for children and adolescents with cancer are generally designed to compare potentially better therapy with therapy that is currently accepted as standard. Most of the progress made in identifying curative therapies for childhood cancers has been achieved through clinical trials.

A cancer treatment clinical trial is an investigative study of a particular treatment plan. It can study new drugs, combinations of drugs and therapies, modes of drug administration, etc. The clinical trial documentation given to all the doctors administering the trial is a detailed protocol so that exactly the same treatment will be given to all of the enrolled patients.

Today, people are living longer lives from successful cancer treatments that are the results of past clinical trials. Through clinical trials, doctors determine whether new treatments are safe and effective and work better than current treatments. Clinical trials also help find new ways to prevent and detect cancer. And they help improve the quality of life for people during and after treatment.

What is a Clinical Trial?

Clinical trials are human research studies that test new drugs or treatments in a lab environment and compares them to current, standard treatments. Patient testing occurs if lab results suggest the treatment is effective and safe. Study participation is voluntary and often sought after by cancer patient caregivers as trials aim to improve survival rates or reduce the side effects or late effects of treatment.

Clinical trials are standard practice in cancer treatment for children, adolescents and young adults, and the primary treatment option for children facing cancer. About 60% of children with cancer are enrolled in a trial, as opposed to less than 5% of adult patients.  Participation in clinical trials spans an average of two to three years and requires a lifetime of follow-up care.

Types of clinical trials

Different clinical trials have different eligibility criteria for patients, such as diagnosis, previous treatments, and a child’s age. Clinical trials are typically grouped into four different types, or phases:

  • Phase I trials help determine the optimal amount or dose of a new therapy that can be given safely to participants. These studies typically have a small number of participants, often fewer than 20.
  • Phase II studies continue to evaluate safety, but also begin to assess a therapy’s effectiveness. These trials typically have more participants than phase I trials.
  • Phase III studies compare the effectiveness of a new therapy or combination of therapies with a standard treatment, and may include hundreds or thousands of participants.
  • Phase IV trials continue to study the safety and effectiveness of a treatment after it has been approved by the FDA.

Cost of Clinical Trials

The cost of conducting multicenter clinical trials is extraordinary and likely represents the most important barrier to the number of clinical trials available for any age group. It is challenging to acquire public funding to study a rare disease in a population that has a reasonable chance of cure (eg, testicular cancer in a 16-year-old), compared with spending dollars studying hepatocellular carcinoma or lung cancer, from which thousands of adults die each year. However, others argue that the relative number of years of life gained in studying cancers in young people far surpasses that gained in older adults with metastatic carcinomas.

The costs of running a specific trial have also substantially increased over time, in part due to the cost of novel molecularly targeted therapies and the sophisticated biological correlatives needed to measure their specific impact. With today’s absence of adequate state resources, there is a need to lobby donors, philanthropy, and special interest groups to generate the infrastructure needed to develop new studies for the rarer cancers of adolescents. Funds could be channeled through specialized trial consortiums, such as the Sarcoma Alliance for Research, which offer the collective intellectual resources to refine and conduct new trials. This paradigm of specialized groups is illustrated by many new drug studies. These studies begin with basic science exploration involving the collection of rare tumor tissue samples and multifaceted efforts to discover new targets. This process is expensive and generally precedes the selection of the therapeutic agent. Selection of the agent then requires further validation of efficacy with larger and even more expensive clinical trials. To achieve accrual goals, these clinical trials must remain open for years due to the rare nature of the disease in question. In such a scenario, when return on investment is distant and improbable, commercial interest may not be reliable. Therefore, the need for state or philanthropic funding strengthens.