Frequently asked questions about cancer care

Talk with your original oncologist about your desire to receive a second opinion. They can guide you about how long you can safely wait before having surgery or beginning treatment.

Multidisciplinary care means that you will have a cancer team consisting of different specialists working together to create a personalized treatment plan that meets your individual needs. Your team will consist of medical oncologists, radiation oncologists, pathologists, surgeons, nurses, rehabilitation specialists, patient navigators, dietitians, and counselors. Together we ensure you receive the most comprehensive care possible.

In order to provide you with the best care possible, your first consultation will be with the specialist who best fits your needs based on your circumstances. For example, if it would benefit you to have additional diagnostic tests conducted first, then your first appointment will be with the specialist who can perform those tests for you. Then you would see the doctor you are referred to. This will help you have the most valuable and meaningful appointments. Please ask questions if you need clarification on who to see.

Understanding your insurance benefits can help ease the financial stress of undergoing cancer treatment. The best way to achieve this is to contact your insurance company and verify your coverage, deductible, co-pay, and any other special considerations specific to your health plan.

Clinical trials are studies conducted to find new ways to prevent, detect and treat cancer. A physician called an investigator carefully designs each clinical trial.

Clinical trials are carried out according to strict scientific and ethical principles:

• Each clinical trial has a protocol (action plan) that explains how the study will be carried out.
• The investigator, or person in charge of the study, prepares the protocol for the study. This protocol explains what will be done, why, how many people will participate, what medical treatments participants will receive and how often, as well as the treatment plan.

Each doctor who takes part in the study uses the same protocol.

Protocols go through a strict approval process for patient safety. This process includes review by the organization sponsoring the study. At Penn, the protocols are reviewed by Penn’s Institutional Review Board (IRB) and the Abramson Cancer Center’s Clinical Trials and Scientific Review and Monitoring Committee.

Our physicians are committed to providing you with the highest level of care, regardless of if you do or do not participate in a clinical trial. If you choose not to participate, your doctor will continue your treatment along the acceptable clinical guidelines. Your participation in a particular clinical trial is voluntary. Your decision not to participate will not affect your care.

Should you choose to participate in a trial, your team of physicians will meet with you to discuss the benefits and risks associated with the specific trial. In general, participation in a clinical trial provides you with closer monitoring and more frequent follow up visits with your doctor and study team. Participating in a clinical trial may offer you access to experimental technologies, therapies, and/or medications new to the field that could benefit you.

During your appointment, please share your interest in participating in a clinical trial if possible. Your doctor will ask you many questions to determine your eligibility to participate in a clinical trial because each clinical trial has very specific requirements.

Enrollment

Each study's protocol describes the characteristics all patients in the study must have. These key characteristics are called eligibility criteria. Eligibility criteria differ from study to study, depending on the research purpose. They may include such factors as age, gender, and the type and stage of cancer.

Today, more and more people are surviving cancer. It is because of clinical trials—many of which are conducted at Penn Medicine—that patients around the world are benefiting from breakthrough therapies and treatments.

A clinical trial is one of the final stages of a long and careful cancer research process. Studies are done with cancer patients to find out whether new approaches to cancer prevention, diagnosis, and treatment are safe and effective.

Clinical trials are important for:

• Providing options for people with cancer, while allowing physicians to improve the way they prevent, diagnose and treat cancer
• Giving patients access to the latest advances available in cancer care
• Benefiting future generations with new and improved treatments

As an academic medical center, Penn Medicine holds research as an important component of its three–part mission, which is patient care, education and research. Clinical research teaches doctors and nurses how to help patients live longer, healthier lives. All medical advancements are the result of organized, academic investigation.

Choosing to participate in clinical research is a personal decision. Many patients choose to participate, but enrolling in a clinical trial may not be the best decision for every cancer patient. Clinical trials also give patients access to the latest advances available in cancer care. Others participate as a way to prevent or treat cancer that may arise in their family or community at large.

There is no guarantee that you will experience any direct benefit from participating in a study, but there is the potential that you could experience adverse events. The clinical research team discusses the potential problems (and how they can be handled) with you before you agree to be in the study.

You should discuss the risks and benefits of clinical trials with your physician and family since each study is unique.

The Abramson Cancer Center oversees one of the largest clinical trials programs in the country, with more than 200 trials available at any one time.

The National Cancer Institute (NCI) named the Abramson Cancer Center as an "exceptional comprehensive care centers dedicated to creating new models for cancer treatment and prevention with cancer research. This exceptional rating is the highest designation given by the NCI, the U.S. government's principal agency for cancer research. The Abramson Cancer Center receives $10 million annually from the NCI to maintain and obtain resources to support groundbreaking research that gives hope to cancer patients.

Because of its excellence in clinical trials, the Abramson Cancer Center has been selected to participate in important national clinical trials for such recognized groups as:

• Eastern Cooperative Oncology Group
• Gynecologic Oncology Group
• National Surgical Adjuvant Breast Bowel Project
• Radiation Therapy Oncology Group
• Southwest Oncology Group

• Treatment trials test new treatments. New treatments for cancer may include a new cancer drug, new approaches to surgery or radiation therapy, new combinations of treatments, or new treatment methods such as immunotherapy.
• Prevention trials test new approaches to cancer prevention. New approaches may include medicines, vitamins, minerals, or other supplements that doctors believe may lower the risk of a certain type of cancer. These trials look for the best way to prevent cancer in people who have never had cancer, prevent cancer from coming back or prevent a new cancer from occurring in people who have already had cancer.
• Screening trials test the best way to find cancer, especially in its early stages.
• Quality of life trials (also called supportive care trials) explore ways to improve comfort and quality of life for cancer patients.

Most clinical research that involves the testing of a new drug progresses in an orderly series of steps, called phases.

Phases allow researchers to ask and answer questions in a way that results in reliable information about the drug and protects the patients.

Most clinical trials are classified into one of three phases:

Cancer clinical trials are carried out in three different phases. Each phase answers different questions about the new treatment.

• Phase I trials are the first step in testing a new treatment. In these studies, researchers look for the best way to give a new treatment, and dosage for that treatment. They also monitor for treatment side effects. Because less is known about the possible risks and benefits, these studies usually include only a small number of patients who would not be helped by other known treatments.
• Phase II trials focus on learning whether the new treatment has an anticancer effect. It answers questions such as: Does the treatment shrink tumors or improve blood test results? Because less is known about the possible risks and benefits, these studies usually include only a small number of patients.
• Phase III trials compare the results of people receiving the new treatment with results of people receiving standard treatment. In most cases, studies move into phase III testing only after a treatment shows promise in phases I and II. Phase III trials may include hundreds of people around the country.

Every clinical trial is managed by a research team that includes doctors, nurses, research assistants, data analysts and other specialists. The research team works closely with other health professionals, including other doctors and nurses, laboratory technicians, pharmacists, dietitians, and social workers, to provide medical and supportive care to people who take part in a clinical trial.

The research team closely monitors the health of people taking part in the clinical trial and gives them specific instructions when necessary. To ensure the reliability of the trial's results, it is important for the participants to follow the research team's instructions. The instructions may include keeping logs or answering questionnaires. The research team may also seek to contact the participants regularly after the trial ends to get updates on their health.

Every clinical trial has a protocol, or action plan that describes what will be done in the trial, how the trial will be conducted, and why each part of the trial is necessary. The protocol also includes guidelines for who can and cannot participate in the trial. These guidelines, called eligibility criteria, describe the characteristics that all interested people must have before they can take part in the trial.

Eligibility criteria can include age, sex, medical history, and current health status. Eligibility criteria for cancer treatment trials often include the type and stage of cancer, as well as the type(s) of cancer treatment already received.

Enrolling people who have similar characteristics helps ensure that the outcome of a trial is due to the intervention being tested and not to other factors. In this way, eligibility criteria help researchers obtain the most accurate and meaningful results possible.

The total costs of participating in clinical trials are rarely covered for U.S. residents by public or private insurance programs due the experimental nature of the treatments being tested. Coverage by foreign government programs for non-U.S. residents is even less likely.

In order to participate in a clinical trial at Penn Medicine, you will need to be able to pay for needed services not provided as part of the clinical trial directly or through your insurance company/health plan, if coverage can be secured. You will also need to be able to secure living arrangements and pay for living expenses throughout the course of the trial if you do not live within the Philadelphia region.

The costs of care for people participating in a clinical trial fall into two general categories: routine care costs and research costs.

• Routine care costs are costs associated with treating a person's cancer whether or not they are in a trial. These costs are usually covered by health insurance, but requirements vary by state and type of health plan.
• Research costs are costs associated with conducting a clinical trial; these costs may include the costs of extra doctor visits, extra tests, and procedures that are required for the trial but would not be part of routine care. Research costs are usually covered by the organization that sponsors the trial.

National and international regulations and policies have been developed to help ensure research involving people is conducted according to strict scientific and ethical principles. In these regulations and policies, people who participate in research are usually referred to as "human subjects."

Clinical trials that are conducted or supported by agencies of the U.S. federal government or that evaluate new drugs or medical devices that are subject to regulation by the U.S. Food and Drug Administration (FDA) must be reviewed and approved by an Institutional Review Board (IRB). The IRB reviews all aspects of a clinical trial to make sure that the rights, safety, and well-being of trial participants will be protected. The IRB must also review ongoing trials at least yearly and, based on those reviews, can decide whether the trial should continue as initially planned or if changes should be made to improve participant protection. An IRB can stop a clinical trial if the researchers are not following the protocol or if the trial appears to be causing unexpected harm to the study participants.

An IRB must have at least five members, including one scientist, one person who is not a scientist, and one person who is not affiliated with the institution where the trial is taking place and who is not an immediate family member of someone who is affiliated with that institution. The non-scientist and the non-affiliated member can be the same person. IRBs can also include doctors, nurses, social workers, chaplains, patient advocates, and other health care or community professionals. All members of an IRB are required to be educated about the IRB's purpose, functions, and responsibilities, as outlined in federal regulations. Trials taking place at multiple locations can involve multiple IRBs.

In addition, some clinical trials, especially phase III clinical trials, use a Data and Safety Monitoring Board (DSMB) to monitor the safety and progress of the trials. In contrast with IRBs, each trial has only one DSMB.

A DSMB is a committee of doctors, statisticians, and others who are independent of the people, organizations, and institutions that are sponsoring, organizing, and conducting the clinical trial. Similar to IRBs, DSMBs review the progress of a clinical trial and participant safety, but they also review data on the effectiveness of the trial interventions. DSMB members are experts in clinical research and clinical trials. They ensure that trial data are complete, and they can stop a trial early if safety concerns arise or if an answer to the main research question is obtained earlier than expected. Stopping a trial early because the main research question has been answered may make it possible for people who are not in the trial to get access to an effective intervention sooner. DSMBs have scheduled meetings to review clinical data, and their meeting minutes or recommendations are forwarded to the IRBs.

The use of placebos (treatment that appears to be real but has no therapeutic benefit) as comparison or "control" interventions in cancer treatment trials is rare.

If a placebo is used by itself, it is because no standard treatment exists. In this case, a trial would compare the effects of a new treatment with the effects of a placebo.

More often, however, placebos are given along with a standard treatment. For example, a trial might compare the effects of a standard treatment plus a new treatment with the effects of the same standard treatment plus a placebo.

The benefits of participating in a clinical trial include the following:

• Trial participants have access to promising new interventions that are generally not available outside of a clinical trial.
• The intervention being studied may be more effective than standard therapy. If it is more effective, trial participants may be the first to benefit from it.
• Trial participants receive regular and careful medical attention from a research team that includes doctors, nurses, and other health professionals.
• The results of the trial may help other people who need cancer treatment in the future.
• Trial participants are helping scientists learn more about cancer (e.g., how it grows, how it acts, and what influences its growth and spread).

The potential harms of participating in a clinical trial include the following:

• The new intervention being studied may not be better than standard therapy, or it may have harmful side effects that doctors do not expect or that are worse than those associated with standard therapy.
• Trial participants may be required to make more visits to the doctor than they would if they were not in a clinical trial and/or may need to travel farther for those visits.
• Health insurance may not cover all patient care costs in a trial.

After a clinical trial is completed, researchers look carefully at the data collected during the trial to understand the meaning of the findings and to plan further research. After a phase I or phase II trial, the researchers decide whether or not to move on to the next phase or stop testing the intervention because it was not safe or effective. When a phase III trial is completed, the researchers analyze the data to determine whether the results have medical importance and, if so, whether the tested intervention could become the new standard of care.

The results of clinical trials are often published in peer-reviewed scientific journals. Peer review is a process by which cancer research experts not associated with a trial review the study report before it is published to make sure that the data are sound, the data analysis was performed correctly, and the conclusions are appropriate. If the results are particularly important, they may be reported by the media and discussed at a scientific meeting and by patient advocacy groups before they are published in a journal. Once a new intervention has proven safe and effective in a clinical trial, it may become a new standard of care.

The FDA Amendments Act of 2007 requires that researchers report the "basic" results of clinical trials that tested FDA-regulated and FDA-approved chemical or biologic agents or medical devices in ClinicalTrials.gov, a publicly accessible database maintained by the U.S. National Library of Medicine (NLM). Basic trial results must be submitted whether or not the results are published in a peer-reviewed scientific journal and include the following items:

• Demographic and baseline information about the participants
• The progress of the participants through each stage of the trial (e.g., the number of participants who left the trial and at which stage)
• Results for the primary and secondary outcomes (also called endpoints) measured in the trial (e.g., tumor response, disease-free survival, overall survival, quality of life, etc.)
• A point of contact for the trial (to obtain additional information about the trial and its results)

More information is available on the About the ClinicalTrials.gov Results Database page. NLM's website also has an FAQ that lists other ways to find clinical trial results.