Clinical Trials: Questions and Points to Consider From Patients and a Caregiver

Some Questions To Ask Before Entering a Clinical Trial

The Study

  • What is the purpose of the study?
  • Why do researchers think the approach may be effective?
  • Who will sponsor the study?
  • Who has reviewed and approved the study?
  • How are study results and safety of participants being checked?
  • How long will the study last?
  • What will my responsibilities be if I participate?
  • What is the protocol if I’m part of the control group instead of the clinical trial?

Possible Risks and Benefits

  • What are my possible short-term benefits?
  • What are my possible long-term benefits?
  • What are my short-term risks, such as side effects?
  • What are my possible long-term risks?
  • What other (treatment) options do people with my risk level or type of cancer have?
  • How do the possible risks and benefits of this trial compare with those options?

Participation and Care

  • What kinds of therapies, procedures and/or tests will I have during the trial?
  • Will they hurt, and if so, for how long?
  • How do the tests in the study compare with those I would have outside of the trial?
  • Will I be able to take my regular medications while in the clinical trial?
  • Where will I have my medical care?
  • Who will be in charge of my care?

Personal Issues

  • How could being in this study affect my daily life?
  • Can I talk to other people in the study?

Cost Issues

  • Will I have to pay for any part of the trial such as tests or the study drug?
  • If so, what will the charges likely be?
  • What is my health insurance likely to cover?
  • Who can help answer any questions from my insurance company or health plan?
  • Will there be any travel or child care costs that I need to consider while I am in the trial?

Basic Definitions

  • Clinical Trials: research studies that test how well new medical approaches work in peopleProtocol: describes what will be done, how it will be done & why it is necessary
  • Eligibility criteria: guidelines for who can or cannot participate in the study
    Informed consent: the process by which people learn the important facts about a trial
  • Phase I studies evaluate what dose is safe, how it should be administered, & how often
  • Phase II trials study the safety & effectiveness and evaluate how it affects the human body
  • Phase III trials compare a new drug with the current standard therapy
  • Phase IV studies evaluate long-term safety & effectiveness (usually after FDA approval)

Only about 5% of cancer patients participate in clinical trials

Clinical trials can be sponsored by National Cancer Institute, National Institutes of Health, medical institutions, pharmaceutical companies, federal agencies, private companies, and nonprofit organizations

Find Clinical Trials

Clinical Trials: Thoughts from a Patient and CaregiverBy Clayton and Rose Twigg

(Clayton, a survivor of anaplastic thyroid cancer, and Rose have also contributed to the Anaplastic Thyroid Cancer web site.

There are several types of clinical trials:

  • Prevention trials test new approaches that doctors believe may reduce the chance of developing cancer. Most involve healthy participants without cancer. Others involve participants who have had cancer to find ways to prevent second cancers.
  • Screening trials test methods to better detect cancer at earlier stages.
  • Diagnostic trials seek to discover new approaches to diagnosing cancer at earlier stages.
  • Treatment trials seek to find new treatments or techniques that may be better than current treatments.
  • Supportive Care or Quality of Life trials investigate ways to improve the comfort of people with cancer. They may also be designed to investigate better ways to manage treatment side effects.
  • Genetic Studies focus on how genetic makeup can impact detection, or response to treatment.

Participation in a clinical trial is a very individual choice and because it is your choice, you may choose to leave the study at any time for any reason.

Since there is much to be considered, you may want to discuss the decision with family and friends. Also consider getting a second medical opinion about where you are in your treatment, whether you should consider participating in a clinical trial, and, this specific clinical trial.

Though your main concern may be, how do risks, side effects and possible benefits compare to current available treatment, no less important is how participation may impact daily life:

  • How often must I see the doctor / receive treatment / come in for testing?
  • How long will I be in the study if I stay until completion?
  • Will I be hospitalized to receive treatment?
  • Who pays for the treatment - does the study sponsor or insurance cover some or all of the cost?
  • What expenses are my / my insurance company’s responsibility, might I be
  • for some?
    - What are the benefit and the risks?

Benefits to consider:

  • Close monitoring by cancer experts
  • A chance to receive a promising therapy before it is (Food & Drug Administration) FDA approved
  • A chance to ‘make a difference’
  • Study results may help others in the future

Risks to consider:

  • Unknown side effects
  • New treatment may be no better than standard care or it may not be effective
  • If I am in a randomized trial, I will not be able to choose to receive the new treatment

Treatment Clinical Trials Phase I –Is the treatment safe? The first step in testing a new investigational drug in humans.

  • Is generally not disease specific
  • Looks for the best way to give treatment, the best dose and how often to treat
  • Has a small study population (20-30), generally at research institutions where patients are closely monitored
  • Evaluates treatment safety and identifies side effects, looks for dose limiting toxicity, the maximum tolerated dose (MTD) above which side effects become difficult to manage

Phase II – Does the treatment work? Focus is on learning whether new treatment has an anti cancer effect and may or may not be disease specific.- May involve FDA drugs or procedures in a new combination

  • As in a Phase I trial, participation is still limited, generally 50 to 100 participants
  • Though the dose has generally been established in the Phase I trial, side effects continue to be closely monitored
  • Intent to treat may be implied, often based upon Phase I trials; this trial has specific endpoints– does it shrink a tumor, improve blood test results, improve quality of life, etc.

Phase III – Is the new treatment better? Generally a comparison of new treatment with results of people receiving standard treatment. Studies move to Phase III only after a treatment shows promise in Phases I and II.

  • Larger, generally multi-center study, often not restricted to research institutions
  • No one in a Phase III study is left without any treatment when standard treatment is available; the study may have multiple treatment arms, comparing current ‘standard of care’ to a new therapy.
  • When no standard treatment exists for a cancer, studies may compare new treatment with a placebo.

Phase IV - Are there better ways to use the treatment? Researchers may continue to study a drug after it has been FDA approved. These trials are generally designed to discover additional risks and benefits in a much larger population, helping doctors understand long-term safety & efficacy of a new drug.

Informed Consent

Informed Consent is the process by which you agree to participate in a clinical trial after having had the purpose, treatment and testing procedures, risks, and benefits explained to you.

You may want to have a family member or friend with you during the consent process. Much information will be shared. Two pairs of ears are better than one.

Informed Consent will be conducted in the following manner. It should:

  • take place without undue influence or pressure from the study staff,
  • allow subjects time to consider the research before signing the consent,
  • be conducted in a private place and manner,
  • be conducted with words understandable to subjects & written at an 8th grade reading level
  • allow subjects the opportunity to ask questions
  • allow the subject to take home an unsigned copy of the consent form to be shared with family/friends prior to enrollment if he/she so chooses
  • provide a certified translator for those who are non English speaking in the subject’s first language

Once enrolled, you will be given a signed & dated copy of the consent form for your records.

Consent Forms

Consent forms will vary depending upon several factors, including disease site & disease stage, intervention proposed and individual Institutional Review Boards (IRB).

A separate consent form may be required if you are being asked to provide blood or tissue samples for testing.

Each consent form should:

  • honestly express realistic expectations for participating in the research study, avoiding inducement by raising false hope
  • contain information in simple, non-technical lay terms (but, ask any question you may have)

Components of a consent form
Section headers are usually written as questions. Information provided below each header will likely be in paragraph form, will answer these questions, and be similar to the following:

  • Purpose / Invitation should contain a statement that:
    • this study involves research
    • encourages you to ask questions about anything you do not understand
  • Description of Study should provide the following information:
    • what is the study’s purpose- what will be done, timeline of screening procedures, treatment cycles, testing, follow-up
    • where will I receive treatment – doctor’s office, as an out-patient or will I be hospitalized
    • when will it be done – length of study participation for me
    • what are my responsibilities as a participant
    • will I be told if new information (good or bad) about the drug is discovered during the study
  • Selection of Subjects will likely include:
    • whether there are restrictions as to who may or may not participate in the study
    • why have I been asked to join the study
    • how many participants will there be
  • Benefits associated with study — are there any (known drug) benefits
  • Risks associated with study — is there any (known drug) hazard
  • Are There Alternative Therapies:
    • alternatives to study participation may be noted in the consent or the study doctor will discuss them with you during the informed consent process
    • if I decide not to participate or choose to leave the study, will I lose benefits, medical treatment or legal rights to which I am otherwise entitled
  • Statement of Confidentiality, Health Information Portability & Accountability (HIPAA) section:
    • who may receive study results
    • will my consent authorization expire
    • will my name ever be made public
  • Compensation:
    • will I be paid for participation or reimbursed for travel, parking, etc.
    • will compensation be made for injury/adverse reactions and who will pay for this treatment
  • Costs —will participation result in extra cost to me for tests, medications, doctor visits, etc.
  • Voluntary Participation:
    • is my participation voluntary / can I choose to leave the study
    • can I be asked to leave the study— detail why this might occur
  • Contact Information:
    • the name & contact information of doctor and/ or study nurse conducting the study
    • telephone number for legal questions: Institutional Review Board (IRB)
    • a telephone number to be used in the event of an out-of-hours medical emergencies
  • Signature Section— contains places for the following persons who must sign and date this section of the consent form
    • participant
    • person conducting the informed consent process
    • principal investigator (study doctor) if he / she is not the one obtaining patient’s consent

You will receive a copy of the signed consent form to keep for your records.