Serve On Boards To Review And Monitor Clinical Trials

Overview

Members of your patient group can help review and monitor clinical trials by serving on Institutional Review Boards (IRBs) and Data Safety and Monitoring Boards (DSMBs). IRBs approve, monitor, and review biomedical and behavioral research involving humans to assure that appropriate steps are taken to protect the rights and welfare of human participants. DSMBs are independent groups of experts who monitor patient safety and treatment efficacy during clinical trials.

Institutional Review Board

Federal regulations require that research projects involving human subjects, including clinical trials, be reviewed by an IRB, but you may not be aware of the exact workings of the review process, for example, that a non-scientist and a person not affiliated with the institution are required members. IRB is actually a general term used by the Federal government, so individual institutions and organizations may have their own name, such as independent ethics committee (IEC), ethical review board (ERB), and research ethics board (REB). 

  • IRB Review Process: The IRB review process is in place to protect the rights and welfare of humans participating as subjects in research studies. 
    • IRB approval is required when research involving human subjects:
      • Receives federal funds either directly or indirectly.
      • Takes place at a university or hospital.
      • Tests therapies that will require U.S. Food and Drug Administration (FDA) marketing approval. 
      • Funded privately, but performed with the intention of publication or as a first step to future IRB-regulated research; in this case the IRB review is voluntary. 
    • The review occurs prior to the start of the research study and periodically throughout the study, and assesses research protocols and related materials, including (but not limited to):
      • Informed consent documents and process.
      • Investigator brochures used to provide information about the research study.
    • The IRB may also:
      • Determine whether a project is exempt from the IRB process. 
      • Require changes prior to granting approval.
      • Grant approval.
      • Deny approval. 
    • The IRB also monitors the safety of study volunteers at a local level.
    • Most changes in protocols and/or the informed consent process and other related materials will require review by the IRB prior to being implemented. 
    • An institution or organization may have its own IRB or may use an outside IRB, which may include institutionally or independently established IRBs.
    • Federal registry of IRBs is required when the IRB will review clinical trials that are:
    • The FDA provides more detailed information about IRBs in the Guidance Document: Institutional Review Boards Frequently Asked Questions
  • IRB Members: Members of an IRB must include at least 1 non-scientist as well as a member not affiliated with the institution or organizations forming the IRB.
    • You or members of your staff or patient community may serve as a non-scientist member of an IRB. 
      • Be aware that nurses, pharmacists, and other biomedical health professionals cannot fill the role of non-scientist even if they are a member of the patient community or your group. They can, however, still be a member of the IRB in a scientific role. 
      • The Human Health and Services Office of Human Research provides further guidance in: Recommendation on IRB Membership and Definition of Non-scientist
    • Members of your Scientific/Medical Advisory Board can also serve as members of IRBs either due to their scientific expertise or as the non-affiliated member. 
    • Due to potential conflict of interest, IRB members associated with your group may not be able to directly review a research proposal focused on your disease depending on the level of involvement in the funding of the research and design or active involvement in the clinical trial.
      • However, having members from your group on 1 or more IRB can help your group better understand the IRB and research process.
  • IRB Appointments: Appointments to an IRB may differ depending on the institution or organization. It is best to contact a specific university, hospital, National Institutes of Health (NIH) Institute, and so on, to determine who appoints members to their affiliated IRB and how you or others from your group could become a potential appointee.

Data Safety and Monitoring Board

You may not be as familiar with the role of DSMBs, also known as Data and Safety Monitoring Committees (DSMCs). A DSMB monitors the safety of study participants and the effectiveness of the study investigational therapy  during a clinical trial. Members of the disease community are often a member of a DSMB for a clinical trial for their disorder.  

  • DSMB process: DSMBs are required as part of a clinical study that poses more than minimal risk to participants.
    • A DSMB provides ongoing monitoring of the progress of the trials and reviews data collected at regular intervals (interim data) throughout the trial.
    • Interim data is evaluated for: 
      • Participant safety.
      • Study conduct and progress. 
        • For example, is data being properly collected, analyzed, reported by the correct parties, and at the frequency outlined in the protocol?
      • Effectiveness of trial investigational therapy.
    • Recommendations by the DSMB regarding the trial include whether the trial should be:
      • Continued, for example if the study is progressing as expected. 
      • Modified, for example, a randomized study may be changed so that all participants receive the treatment if the data find the treatment outcomes are overwhelmingly positive. 
      • Terminated, for example if:
        • The investigational therapy is causing harm and the risk and/or seriousness of harm outweighs long term benefits.
        • Expected benefits are not demonstrated. This could mean the control/placebo group has the same outcome as the group receiving the investigational therapy.
        • There are not enough participants enrolling in the study.
        • Too many participants are withdrawing from the study. 
        • The study protocol is not being followed.
    • The DSMB provides recommendations to the Sponsor or a steering committee or other group delegated by the Sponsor to make decisions about the trial. 
      • Strict requirements and deadlines are in place for reporting unanticipated adverse events to both the sponsor and specific regulatory agencies.
    • The DSMB may have other tasks as determined by the Sponsor.
    • The FDA provides more information about DSMB/DSMC in their Guidance Document: Establishment and Operation of Clinical Trial Data Monitoring Committees
    • Each of the NIH Institutes may provide their own additional guidelines. For example:
    • The Global Advocacy for HIV Prevention provides information about DSMBs in plain language in their Fact Sheet: Data Safety Monitoring Boards.
  • DSMB members: The Sponsor chooses members of the DSMB. 
    • Many Sponsors choose members from the patient community to be on the DSMB. In general, the non-scientist DSMB member would:
      • Not participate in the trial, because awareness of the accumulating data could affect compliance or other aspects of trial participation.
      • Be someone with the disease or condition under study, a close relative of such an individual, or a leader from the patient group.
    • Sponsors also choose medical personnel who specialize in the disease being studied. This may include:
      • Members of your Scientific/Medical Advisory Board who are not involved in the current study.
      • Specialists in your medical network who are not involved in the current study. 
  • DSMB appointments: Because appointments to DSMB for a clinical study are made by the study Sponsor, you may wish to contact Sponsors of upcoming clinical trials for your disease to determine how you, members of your disease community, or specialists or other medical professionals affiliated with your group may be appointed to the DSMB for the clinical trial. 

Tips for Success

  • Build strong, positive relationships with pharmaceutical companies and other potential Sponsors early in the therapy development process to ensure your patient community is represented in the DSMB.
  • Work with your Scientific/Medical Advisory Board as well as researchers focused on your disease to learn more about the IRB process at their respective institutes.
  • Encourage your Scientific/Medical Advisory Boards and researchers to serve on their own institutional IRBs.
  • Introduce Sponsors to your Scientific/Medical Advisory Board as well as other known specialists for your disease and select members of your patient community to potentially increase the expertise on the DSMB.
  • Communicate the protections that IRB and DSMB provide participants of clinical trials to your disease community.

Resources

Institutional Review Board
Recommendation on IRB Membership and Definition of Non-scientist Human Health and Services (HHS) Office of Human Research (link)
Data Safety and Monitoring Board
Fact Sheet: Data Safety Monitoring Boards Global Advocacy for HIV Prevention (link)

Members of your patient group can help review and monitor clinical trials by serving on Institutional Review Boards (IRBs) and Data Safety and Monitoring Boards (DSMBs). IRBs approve, monitor, and review biomedical and behavioral research involving humans to assure that appropriate steps are taken to protect the rights and welfare of human participants. DSMBs are independent groups of experts who monitor patient safety and treatment efficacy during clinical trials.

Federal regulations require that research projects involving human subjects, including clinical trials, be reviewed by an IRB, but you may not be aware of the exact workings of the review process, for example, that a non-scientist and a person not affiliated with the institution are required members. IRB is actually a general term used by the Federal government, so individual institutions and organizations may have their own name, such as independent ethics committee (IEC), ethical review board (ERB), and research ethics board (REB). 

  • IRB Review Process: The IRB review process is in place to protect the rights and welfare of humans participating as subjects in research studies. 
    • IRB approval is required when research involving human subjects:
      • Receives federal funds either directly or indirectly.
      • Takes place at a university or hospital.
      • Tests therapies that will require U.S. Food and Drug Administration (FDA) marketing approval. 
      • Funded privately, but performed with the intention of publication or as a first step to future IRB-regulated research; in this case the IRB review is voluntary. 
    • The review occurs prior to the start of the research study and periodically throughout the study, and assesses research protocols and related materials, including (but not limited to):
      • Informed consent documents and process.
      • Investigator brochures used to provide information about the research study.
    • The IRB may also:
      • Determine whether a project is exempt from the IRB process. 
      • Require changes prior to granting approval.
      • Grant approval.
      • Deny approval. 
    • The IRB also monitors the safety of study volunteers at a local level.
    • Most changes in protocols and/or the informed consent process and other related materials will require review by the IRB prior to being implemented. 
    • An institution or organization may have its own IRB or may use an outside IRB, which may include institutionally or independently established IRBs.
    • Federal registry of IRBs is required when the IRB will review clinical trials that are:
    • The FDA provides more detailed information about IRBs in the Guidance Document: Institutional Review Boards Frequently Asked Questions
  • IRB Members: Members of an IRB must include at least 1 non-scientist as well as a member not affiliated with the institution or organizations forming the IRB.
    • You or members of your staff or patient community may serve as a non-scientist member of an IRB. 
      • Be aware that nurses, pharmacists, and other biomedical health professionals cannot fill the role of non-scientist even if they are a member of the patient community or your group. They can, however, still be a member of the IRB in a scientific role. 
      • The Human Health and Services Office of Human Research provides further guidance in: Recommendation on IRB Membership and Definition of Non-scientist
    • Members of your Scientific/Medical Advisory Board can also serve as members of IRBs either due to their scientific expertise or as the non-affiliated member. 
    • Due to potential conflict of interest, IRB members associated with your group may not be able to directly review a research proposal focused on your disease depending on the level of involvement in the funding of the research and design or active involvement in the clinical trial.
      • However, having members from your group on 1 or more IRB can help your group better understand the IRB and research process.
  • IRB Appointments: Appointments to an IRB may differ depending on the institution or organization. It is best to contact a specific university, hospital, National Institutes of Health (NIH) Institute, and so on, to determine who appoints members to their affiliated IRB and how you or others from your group could become a potential appointee.

You may not be as familiar with the role of DSMBs, also known as Data and Safety Monitoring Committees (DSMCs). A DSMB monitors the safety of study participants and the effectiveness of the study investigational therapy  during a clinical trial. Members of the disease community are often a member of a DSMB for a clinical trial for their disorder.  

  • DSMB process: DSMBs are required as part of a clinical study that poses more than minimal risk to participants.
    • A DSMB provides ongoing monitoring of the progress of the trials and reviews data collected at regular intervals (interim data) throughout the trial.
    • Interim data is evaluated for: 
      • Participant safety.
      • Study conduct and progress. 
        • For example, is data being properly collected, analyzed, reported by the correct parties, and at the frequency outlined in the protocol?
      • Effectiveness of trial investigational therapy.
    • Recommendations by the DSMB regarding the trial include whether the trial should be:
      • Continued, for example if the study is progressing as expected. 
      • Modified, for example, a randomized study may be changed so that all participants receive the treatment if the data find the treatment outcomes are overwhelmingly positive. 
      • Terminated, for example if:
        • The investigational therapy is causing harm and the risk and/or seriousness of harm outweighs long term benefits.
        • Expected benefits are not demonstrated. This could mean the control/placebo group has the same outcome as the group receiving the investigational therapy.
        • There are not enough participants enrolling in the study.
        • Too many participants are withdrawing from the study. 
        • The study protocol is not being followed.
    • The DSMB provides recommendations to the Sponsor or a steering committee or other group delegated by the Sponsor to make decisions about the trial. 
      • Strict requirements and deadlines are in place for reporting unanticipated adverse events to both the sponsor and specific regulatory agencies.
    • The DSMB may have other tasks as determined by the Sponsor.
    • The FDA provides more information about DSMB/DSMC in their Guidance Document: Establishment and Operation of Clinical Trial Data Monitoring Committees
    • Each of the NIH Institutes may provide their own additional guidelines. For example:
    • The Global Advocacy for HIV Prevention provides information about DSMBs in plain language in their Fact Sheet: Data Safety Monitoring Boards.
  • DSMB members: The Sponsor chooses members of the DSMB. 
    • Many Sponsors choose members from the patient community to be on the DSMB. In general, the non-scientist DSMB member would:
      • Not participate in the trial, because awareness of the accumulating data could affect compliance or other aspects of trial participation.
      • Be someone with the disease or condition under study, a close relative of such an individual, or a leader from the patient group.
    • Sponsors also choose medical personnel who specialize in the disease being studied. This may include:
      • Members of your Scientific/Medical Advisory Board who are not involved in the current study.
      • Specialists in your medical network who are not involved in the current study. 
  • DSMB appointments: Because appointments to DSMB for a clinical study are made by the study Sponsor, you may wish to contact Sponsors of upcoming clinical trials for your disease to determine how you, members of your disease community, or specialists or other medical professionals affiliated with your group may be appointed to the DSMB for the clinical trial. 
  • Build strong, positive relationships with pharmaceutical companies and other potential Sponsors early in the therapy development process to ensure your patient community is represented in the DSMB.
  • Work with your Scientific/Medical Advisory Board as well as researchers focused on your disease to learn more about the IRB process at their respective institutes.
  • Encourage your Scientific/Medical Advisory Boards and researchers to serve on their own institutional IRBs.
  • Introduce Sponsors to your Scientific/Medical Advisory Board as well as other known specialists for your disease and select members of your patient community to potentially increase the expertise on the DSMB.
  • Communicate the protections that IRB and DSMB provide participants of clinical trials to your disease community.

Resources

Institutional Review Board
Recommendation on IRB Membership and Definition of Non-scientist Human Health and Services (HHS) Office of Human Research (link)
Data Safety and Monitoring Board
Fact Sheet: Data Safety Monitoring Boards Global Advocacy for HIV Prevention (link)