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Clinical Trials Lecturer – Lakshmi Rajdev, MD, MS New York Stomach Cancer Education Symposium Clinical Trials Lecturer – Lakshmi Rajdev, MD, MS Albert Einstein Cancer Center Bronx, NY Debbie’s Dream Foundation Medical Advisory Board Member

Bridging the Gap Between Laboratory Science and Human Health

What Is a Clinical Trial? Clinical trials are research studies that involve people. Through clinical trials, doctors find new ways to improve treatments and the quality of life for people with disease.

Clinical Trials Researchers design cancer clinical trials to test new ways to: Find and diagnose cancer Treat cancer Prevent cancer Manage symptoms of cancer and side effects from its treatment

Many treatments used today are the results of past clinical trials!! These studies are designed to answer questions about new treatments or ways of using existing treatments better. These trials test many types of treatments such as new: Drugs or vaccines Ways to do surgery or give radiation therapy Combinations of treatments Many treatments used today are the results of past clinical trials!!

Clinical Trials Advancing Cancer Care Basic research findings demonstrated the anti-tumor effects of tamoxifen in lab animals. Clinical researchers showed that tamoxifen shrank breast tumors in afflicted women and reduced the risk of tumor recurrence. Clinical researchers also demonstrated that healthy women treated with tamoxifen were less likely to develop breast cancer. Tamoxifen is now widely used as a breast cancer treatment and prevention.

Clinical Trial Phases Clinical trials take place in phases. For a treatment to become standard, it must first go through 3 or 4 clinical trial phases. The early phases make sure the treatment is safe. Later phases show if it works better than the standard treatment. You do not have to take part in all phases. Phase I, II, III, IV

Phase I and II Phase I Phase II Purpose: To find a safe dose To decide how the new treatment should be given To see how the new treatment affects the human body Number of people taking part: 15–30 Phase II To determine if the new treatment has an effect on a certain cancer Number of people taking part: Fewer than 100

Phase III and IV Phase III Phase IV Purpose: To compare the new treatment (or new use of a treatment) with the current standard treatment Number of people taking part: From 100 to several thousand Phase IV To further assess the long-term safety and effectiveness of a new treatment Number of people taking part: Several hundred to several thousand

Clinical Trials Follow Strict Guidelines. Every trial has a person in charge, usually a doctor, who is called the principal investigator. The principal investigator prepares a plan for the study, called a protocol, which is like a recipe for conducting a clinical trial. The protocol explains what the trial will do; how the study will be carried out; why each part of the study is necessary; the reason for doing the study; who can join the study; how many people are needed for the study; any drugs they will take, the dose, and how often; what medical tests they will have and how often; and what information will be gathered about them. http://clinicaltrials.gov/ct/info/whatis#types

Clinical Trial Procedure Who can join a clinical trial? Based on the questions the research is trying to answer, each clinical trial protocol clearly states who can or cannot join the trial. Common criteria for entering a trial include: having a certain type or stage of cancer or having received a certain kind of therapy in the past. Criteria such as these help ensure that people in the trial are as alike as possible.

Clinical Trial Criteria These criteria also help ensure: Safety: Some people have health problems besides cancer that could be made worse by the treatments in a study. If you are interested in joining a trial, you will receive medical tests to be sure that you are not put at increased risk. Accurate and meaningful study results: You may not be able to join some clinical trials if you already have had another kind of treatment for your cancer. Otherwise, doctors could not be sure whether your results were due to the treatment being studied or other factors.

Randomization Randomization is used in all phase III and some phase II trials. These trials are called randomized clinical trials. Comparing these groups to each other often clearly shows which treatment is more effective or has fewer side effects. If you are thinking about joining a randomized clinical trial, you need to understand that there is an equal chance you will be assigned to either group. Neither you nor your doctor chooses which group you will be in.

Placebo A placebo is designed to look like the medicine being tested, but it is not active. Placebos are almost never used in cancer treatment trials. In some cases, a study may compare standard treatment plus a new treatment to standard treatment plus a placebo. You will be told if the study uses a placebo.

Patient Safety Federal rules help ensure that clinical trials are run in an ethical manner. Your rights and safety are protected through: Informed consent Careful review and approval of the clinical trial protocol by: Scientific experts An institutional review board (IRB) Ongoing monitoring of the trial by: The IRB Data and Safety Monitoring Boards (DSMBs) for phase 3 trials The organization sponsoring the trial The research team

Informed Consent Informed consent is a process through which you learn details about the trial before deciding whether to take part. The team explains the trial's: Purpose Procedures Risks and benefits You can: Make a decision about participating Leave the study at any time Learn about all your treatment options Learn all that is involved in the trial including all details about treatment, tests, and possible risks and benefits Discuss the trial with the principal investigator and other members of the research team Both hear and read the information in language you can understand

Ending Trials Early In some cases, a trial might be stopped because: It is going very well. If there is clear evidence early-on that a new treatment or intervention is effective, then the trial may be halted so that the new treatment can be made widely available as soon as possible. If participants are experiencing unexpected and severe side effects or if there is clear evidence that the harms are outweighing the benefits Enough patients cannot be recruited. The results of other trials are published that answer the question or make it irrelevant.

Possible Benefits Of Clinical Trials You will have access to a new treatment that is not available to people outside the trial. The research team will watch you closely. If the treatment being studied is more effective than the standard treatment, you may be among the first to benefit. The trial may help scientists learn more about cancer and help people in the future. This graphs shows the source of funding for all biomedical research from 1994 through 2003. The data were compiled from public and private sources, and all figures were adjusted for inflation using the biomedical research and development price index (BRDPI). According to this analysis, total funding for biomedical research increased from $37.1 billion in 1994 to $94.3 billion in 2003. Industry is the largest and NIH is the second largest supporter of biomedical research. Private funding makes up only a small portion of total funding for biomedical research.

Possible Risks Of Clinical Trials The new treatment may not be better than, or even as good as, the standard treatment. New treatments may have side effects that doctors do not expect or that are worse than those of the standard treatment. You may be required to make more visits to the doctor than if you were receiving standard treatment. You may have extra expenses related to these extra visits such as travel and childcare costs. You may need extra tests. Some of the tests could be uncomfortable or time consuming. Even if a new treatment has benefits in some patients, it may not work for you. Health insurance may not cover all patient care costs in a trial.

Paying for Clinical Trials Patient care costs: Doctor visits Hospital stays Standard cancer treatments Treatments to reduce or eliminate symptoms of cancer or side effects from treatment Lab tests X-rays and other imaging tests Research costs: The study drug Lab tests performed purely for research purposes Additional x-rays and imaging tests performed solely for the trial

Insurance Coverage and Clinical Trials Federal law requires most health insurance plans to cover routine patient care costs in clinical trials under certain conditions. Such conditions include: You must be eligible for the trial. The trial must be an approved clinical trial. Which costs are not covered? Health plans are not required to cover the research costs of a clinical trial. Examples of these costs include extra blood tests or scans that are done purely for research purposes. Often, the trial sponsor will cover such costs. Plans are also not required to cover the costs of out-of-network doctors or hospitals if the plan does not usually do so. But if your plan does cover out-of-network doctors or hospitals, they are required to cover these costs if you take part in a clinical trial.

Who Funds Clinical Research? Federal Government National Institutes of Health Department of Veterans Affairs Department of Defense State and Local Governments Private Organizations Foundations and other not-for-profits Industry

How to Join a Cancer Clinical Trial Step 1: Gather Details about Your Cancer Step 2: Find Clinical Trials Step 3: Take a Closer Look at the Trials that Interest You Step 4: Contact the Team Running the Trial Step 5: Ask Questions Step 6: Make an Appointment

Finding Clinical Trials The National Cancer Institute, drug companies, medical institutions, and other organizations sponsor clinical trials. Clinical trials take place in many settings such as cancer centers, large medical centers, small hospitals, and doctors’ offices. The National Cancer Institute maintains a list of thousands of cancer clinical trials. Contact us to see which ones you might be eligible for. To reach the National Cancer Institute: Call: 1-800-4-CANCER (1-800-422-6237) Visit: http://www.cancer.gov Chat: http://www.cancer.gov/livehelp E-mail: cancergovstaff@mail.nih.gov https://clinicaltrials.gov/ Clinical Advocacy Groups

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