Give Input On The Informed Consent Process


Consent Requirements
Informed Consent for Clinical Trials  U.S. Federal Drug Administration (FDA) (link)
Informed Consent FAQs U.S. Department of Health and Human Services (HHS) (link)
Research versus Clinical Care
Clinical Research Versus Medical Treatment U.S. Federal Drug Administration (FDA) (link)

Informed consent allows clinical trial participants to understand what will happen during a clinical study, including the possible risks and whether participants will receive their personal results. Your patient group can provide input during the development of informed consent documents and help clinical trial participants better prepare for the consent process.

The goal of informed consent for clinical trials is to make sure participants are fully aware of all aspects of the research study from their perspective. Taking the time to learn the elements of the informed consent process as it applies to clinical trials can help you understand information you can share with the Sponsor about the target participant population that may affect this process. 

The Code of Federal Regulations states that, for research in humans, informed consent must include the following elements:

  • A basic description of the study:
    • Explicitly states that the individuals are participating in research and explains the purpose of the study, a proposed timeline, and gives a general overview of the procedures (including a basic description and timeframe for each).  
    • Can also provide more specific information such as why the participant is eligible to be involved in the study.  
    • Must also include any possible future use (secondary research use) of personal identifiable information (PII).
      • PII is any data that could potentially be used to identify a particular person. 
      • Examples include a person’s full name, Social Security number, address, and email address
  • Risks or discomforts: 
    • Explains any reasonably foreseeable risks or discomforts that may result from participation in the study and the steps the investigators have taken to minimize these risks.  
    • Risks can include psychological stressors, financial burdens, and population specific difficulties.
  • Benefits:
    • Describes potential benefits to participants that may reasonably be expected.
    • Explains, if applicable, that there may be no foreseeable benefit to the participants. 
  • Alternatives to participation: 
    • Discloses any currently available procedures or treatments that may offer an effective alternative to participating in the clinical trial.
    • For many treatment studies, especially for rare diseases, there may be no other effective alternative.
  • Compensation if more than minimal risk: 
    • Explains if compensation and/or medical treatment is provided in the event a  research-related injury occurs.
    • Includes  a description of  the compensation or medical treatment that may be provided or where more information about the process can be found. 
  • Confidentiality:
    • Provides information about privacy protection.
    • Explains how records that include personal identifying information will be stored and/or disposed.
    • Discloses the possibility that the FDA may access records which include PII.
  • Contact Information:
    • Lists who to contact if a potential participant has more questions or wants to understand more about research participants’ rights.
    • Explains who to contact if a research-related injury occurs.
  • Voluntary participation statement and procedures for withdrawal:
    • Explicitly states that all participation is voluntary and that the subject can stop participating at any time during the study. 
    • Outlines whether there are possible consequences of leaving a trial before it is completed. For example, the investigational therapy may require a slow tapering of dosage to avoid withdrawal symptoms.
  • When applicable, the informed consent must also address:
    • The potential for commercial profit based on the research.
    • If and how clinically relevant information will be returned to participants.
    • The inclusion of whole genome sequencing.
    • Any reasons why the research participant’s inclusion in the clinical trial may be ended by the clinical trial investigator. For example, a participant may be excluded from the study if they fail to follow the procedures of the clinical trial or have changes in lab values that fall outside of the clinical trial limits.
    • The approximate number of research participants that will be enrolled in the study.
  • Beyond the actual written content of the consent, requirements include: 
    • Information in the informed consent must be easily understood by potential participants (or their guardians). Complex scientific or research terms should be avoided.
    • There should be no pressure to sign. Participants should be given ample time to discuss the information with friends and relatives, as well as their own doctor or other trusted medical professionals.
    • A contact person who is able to answer any relevant questions about the clinical trial should be available to any potential participant. There should be ample time to discuss any questions with the contact person prior to signing the informed consent. 
  • You can read more details about elements of informed consent in the following resources:

Although there is a clear distinction between a research clinical setting and clinical care, the distinction becomes more complicated when the research is taking place in a clinical care setting. Since many rare disease clinical trials take place in a clinical setting, it is important for you to understand what this means and how it may affect the informed consent process, including the return of test results. 

  • Research clinical trial setting:
    • Protects participants from harm. 
    • Focuses on understanding the effectiveness and safety of investigational therapy.
    • May not directly benefit participants, but instead benefit future patients.
    • May limit the return of an individual’s results. 
    • Requires Institutional Review Board (IRB) approval to return a result that may be potentially medically relevant to a participant, but falls outside the original scope of the approved clinical study protocol. 
    • It is the participant’s responsibility to follow-up on disclosed results with their own medical provider, including having the result verified by a clinical laboratory.
    • Is not the same as health care provided by a personal medical care provider.
    • Gathers and assesses certain measures or outcomes at specific times.
    • Publishes results, although participants are not identified individually.
    • Does not enter a participant’s data into their individual medical records.
  • Clinical care setting, no research involved:
    • Provides care that is in the best interest of the patient.
    • Addresses the needs of individual patients.
    • Makes real-time decisions about health care plans, including trying different treatments.
    • Uses treatments and therapies known to be safe and effective.
    • Maintains confidentiality of medical records.
    • Records health care in a patient’s individual medical records.
    • Permits patients to access all test results and information included in the medical records.
  • Research in a clinical care setting:
    • Provides the investigational therapy as part of participants’ health care.
    • May provide the investigational therapy as an addition to current treatment rather than replacing current treatment.
    • Follows protocols of a research study and the therapy may not directly benefit the participant.
    • Gathers and assesses certain measures and outcomes at specific times.
    • Enters data from measures and outcomes as part of participant’s medical records.
    • Permits patient to access all test results and information included in the medical records.
    • May require IRB approval to return results of potential medical significance that fall outside the original scope of approved study protocol. The result will be verified in a clinical lab prior to being disclosed and the clinicians involved in the study will provide next-step guidance  
    • Publishes results, although participants are not identified individually.
    • Researcher may be part of the clinical team. 
    • Needs very clear informed consent to distinguish what is research and what is part of clinical care.
  • You can learn more about the differences between research, clinical care, different research settings, and the return of potentially medically significant results in the following resources.

You will know the strengths, limitations, needs, and desires of the target population for a clinical trial better than the Sponsor. Now that you understand the informed consent process and obligations of different settings, you can provide insight into elements of the informed consent process that can be proactively addressed to minimize concerns and increase informed participation. You may discuss:

  • Reading level appropriate for target participants and/or guardians.
  • Language requirements, for example, does a significant part of the population speak a language other than English.
  • Potential concerns of the target participants. For example, participants may be concerned about having to discontinue current treatment or it may be important for them to know if they can still go to school or work during the clinical trial.
  • Predictable acceptability to target participant population if individual results may not be returned. 
  • Clearness of the legal and ethical obligations depending on the setting of research.
  • Expectations of clinical care.
  • Terminology that the target participants or their guardians may or may not understand.

Helping potential participants understand their rights prior to beginning the informed consent process can help mitigate any concerns that may jeopardize participation. It can be helpful to provide information about the informed consent process before a clinical trial is ready to recruit. Your knowledge of the informed consent process together with your knowledge of your disease community can help you determine the best approach to ensure the process reaches its goal of informed consent. 

  • Provide general information about clinical trials and informed consent on your website.
  • Explain possible future benefits of studies that may not provide immediate benefit to participants, for example what the study hopes to discover, mitigate, or develop.
  • Hold a webinar about informed consent and make it available to those who can’t attend. 
  • Bring in speakers at an upcoming conference who can talk about the informed consent process.
  • Develop InfoGraphs or pamphlets that can be downloaded from your website or mailed to members who may not have easy internet access.
  • Post links to relevant information, events, or webinars provided by large umbrella organizations like NORD, Global Genes, and FasterCures.
  • Utilize information from natural history studies and patient preference studies to inform an investigational therapy’s Sponsor about any predictable concerns of the study design or eligibility criteria that can be directly addressed in the informed consent.
  • Start educational outreach about the informed consent process to your disease community before the clinical trial is ready to begin accepting participants.
  • Work with the Sponsor to develop a Frequently Asked Questions document that is relevant to the target participant population.  
  • Offer resources developed for your members to the Sponsor to help with their outreach efforts.


Consent Requirements
Informed Consent for Clinical Trials  U.S. Federal Drug Administration (FDA) (link)
Informed Consent FAQs U.S. Department of Health and Human Services (HHS) (link)
Research versus Clinical Care
Clinical Research Versus Medical Treatment U.S. Federal Drug Administration (FDA) (link)