Providing Testimony

As mentioned previously, FDA Ad Comm meetings provide 2 ways for members of the public, including patients, patient advocates, caregivers, and medical professionals involved in the care of patients, to provide information to the Committee to be considered during their discussions and recommendations to the FDA. Both are highly valued and can be a way for your patient community’s voice to be heard at a critical time in the regulatory decision making process. 

• Written testimony: Copies of all written testimony are distributed to the members of the committee prior to the meeting. 
  • One of the advantages of written testimony is the provided information can be referenced repeatedly by the committee members. 
  • Written testimony can be submitted by those who:
    • Cannot attend the meeting.
    • Plan on attending the meeting.
    • Will be also presenting oral testimony.
    • Are from outside the United States.
  • The meeting announcement in the Federal Register will include the details required to submit written testimony, including
    • The FDA staff receiving the submissions.
    • The deadline for submissions to be included in the meeting.
    • Email address, mailing address, and FAX number for submission. 
• Oral testimony: The impact of your patient community’s voice speaking directly to committee members, as well as the FDA staff attending the meeting, can be powerful.
  • One of the advantages of oral testimony is allowing the committee members to put faces to the patients, families, and clinicians affected by the final decision of the FDA.
  • To during the open public hearing, interested persons need to contact the designated FDA staff by the deadline in the Federal Register announcement. Contact should include:
    • Name and contact information of the person wishing to provide testimony.
      • If representing a group, then the spokesperson’s name and contact information, name of group, population represented by group, and mission statement of group.
    • A brief statement or outline of the general nature of the evidence or arguments the person wishes to present.
    • The approximate amount of time requested to make your presentation (usual allotment is between 5 to 10 minutes, but is dependent on the number of presentations and the information being presented).
  • A confirmation will be sent to individuals indicating that their testimony will be among those included during the Open Public Hearing. 
    • Allotted time for an individual’s oral testimony is usually included in the confirmation.
    • In rare instances where public demand is high, the FDA may opt to implement a lottery and randomly select from among the requests to speak.
    • To allow more speakers, individuals with similar evidence or arguments may be asked to group their presentation into one.
    • FDA may decline a request to speak at an Open Public Hearing if the person wishes to address a matter that is unrelated to the advisory committee’s work. 
  • After acceptance, if the presenter would like to use slides or videos during their presentation, they should inform the designated FDA staff to determine the best format to be used.
    • All videos or slides should be sent at least a week prior to the FDA Ad Comm so that playability can be ensured and corrected if there are any technical issues.
    • Due to software security concerns, videos or slides will not be accepted on a thumb drive the day of the FDA Ad Comm meeting.
  • When arriving on the day of the FDA Ad Comm meeting, designated speakers should identify themselves to FDA staff members and provide the staff with any handouts or other materials for distribution to committee members, and be included in the meeting’s permanent record.
    • A table will be made available near the registration desk for any printed materials you wish to make available to those attending the meeting.
  • Designated speakers will be assigned a number to establish order of speakers.
  • Special seating is usually reserved for registered speakers from the public.
  • Those who speak are encouraged to disclose any financial relationships they may have with the topic of the meeting or with the sponsors or competitors of the therapies under discussion.
  • After a person has made their statement, the FDA Ad Comm members may ask the person questions.
  • The Open Public Hearing most often is held the hour after the lunch break. 
  • For more details, you may wish to review the FDA Guidance: The Open Public Hearing at FDA Advisory Committee Meetings
  • The FDA also provides a more public friendly version of the Guidance, which summarizes steps to becoming a designated speaker and some tips for speaking: Guidelines for Speakers at the Open Public Hearing of an FDA Advisory Committee Meeting
• Testimony content: 
  • Clinical trial participants (and their guardians, if the child is a minor) and physicians involved in the clinical trial should consult the agreement signed with the Sponsor of the therapy clinical trial prior to providing written or oral testimony. 
    • In some cases, there may be limitations on what can be shared.
    • The FDA Ad Comm meeting does not nullify the signed agreement.
  • Limit the amount of general statistics and data that is provided. 
    • For example, in most cases the committee members will already be aware of how many people have the disease.
    • Depending on the concerns of the particular therapy, you or a member of your Scientific/Medical Advisory Board may wish to present data that you wish to be considered in the decision in either written or oral testimony rather than being presented by multiple members of your patient community.
  • Encourage members of your patient community and associated clinicians to share their direct experience with the symptoms being targeted by the current therapy. For example, discuss the impact of: 
    • Targeted symptom(s) on quality of life and daily living, including not only patients, but also family and friends.
    • Current treatment option(s). For example the current treatment option:
      • May not be effective for most.
      • Is difficult to administer.
      • Has multiple or serious adverse effects.
    • The potential of the investigational therapy to improve quality of life and daily living.
  • Share specific, personal experiences that demonstrate the impact of symptom(s), disease, and/or an investigational therapy rather than general statements. 
  • Describe the hopes and dreams of the patient community and how this medical treatment may be a step towards realizing those dreams. 
  • Consider grouping individual oral testimonies that are similar together and be presented by 1 speaker
    • The FDA Ad Comm may make this request if there are many speakers wishing to provide oral testimony.
    • Grouping similar testimonies allows more time for different aspects or perspectives to be presented.
    • One person speaking for multiple patients or families can actually have a greater impact, then each individual presenting a similar story. 
    • The spokesperson can name the other patients/families or state how many patients/families they are representing.
    • Each person or family can still provide written testimony.

Members of an FDA Ad Comm

An FDA Ad Comm ordinarily consists of 10 to 15 fixed or “standing” members including the chairperson. Members are selected because they possess expertise specific to the committee’s function. Most committees will also have a consumer representative and an industry representative. FDA Patient Representatives and expert consultants are often added as temporary voting members.

• Scientific members: 
  • Physician-scientists whose specialties or research involves the kinds of therapies being reviewed, including: 
    • Statisticians
    • Epidemiologists
    • Medical faculty
    • Chemists
    • Biologists
    • Engineers
    • Nutritionists
    • Toxicologists
    • Experts in preclinical (animal) studies
  • As core members of FDA Ad Comms, responsibilities will not be limited to a single disease or a specific therapy of interest. However members of your Scientific/Medical Advisory Board may be interested in learning more about the opportunity:
    •  Applying for Membership on FDA Advisory Committees
• Industry representatives:
  • Address global concerns for industry, not their specific employer.
  • Are not the industry Sponsor for the therapy being reviewed.
  • May or may not have the ability to vote, depending on the specific committee.
• Consumer representatives:
  • Offer broad knowledge of consumer rights and needs.
  • Have an affiliation with and/or active participation in consumer or community-based organizations.
  • Consumer representatives are core members and therefore are not focused on a single disease or specifictherapy. However, the FDA does provide more information about applying to be a consumer representative if you or someone you know is interested:
    • Consumer Representatives on FDA Advisory Committees
• FDA Patient Representatives:
  • FDA Patient Representatives are recruited and then specially trained to participate as temporary voting members for FDA Ad Comm meetings and to provide input earlier in the regulatory therapy development and review process by engaging directly with Division review staff. 
  • They are appointed as Special Government Employees (temporary employees) to provide direct input to agency staff as they share valuable insight on their experiences with various diseases, conditions, and devices while gaining access to confidential information.
  • Qualifications include:
    • Personal experience with the disease either as a patient or primary caregiver.
    • Ability to be objective while representing the concerns of others in their communities.
    • Willingness and ability to communicate in public their views and perspectives.
    • Knowledge about most treatment options and research for the areas of experience they are representing.
    • Impartiality with minimal to no financial conflicts of interest or ethical issues for self or close family members, for example, a financial interest, such as stock, in companies that may be affected by FDA decisions.
  • FDA Patient Representatives are added to the FDA Ad Comm panel for a meeting when a therapy concerns their disease or a disease within their area of expertise.
  • Due to the required training, you or a member of your patient community cannot apply to be a Patient Representative for an already scheduled or soon to be scheduled FDA Ad Comm meeting for a therapy for your disorder. 
  • The FDA provides more information about FDA Patient Representatives including how to apply.
    • About the FDA Patient Representative Program
    • How to Apply to the FDA Patient Representative Program
• Consultants: Scientists or medical personnel whose expertise is not represented by the fixed voting membership may be added to an FDA Ad Comm.
  • World experts on the topic being discussed are often added as temporary voting members.
  • These experts are present only for special meetings.
  • Members of your Scientific/Medical Advisory Board may meet the qualifications to be a consultant if there is no conflict of interest.

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 therapy if the data find the clinical outcomes are overwhelmingly positive in the experimental arm. 
    • 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 clinical 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.
    • For example, the National Eye Institute (NEI) lists additional responsibilities for their DSMC including review and approval of the primary trial manuscript(s): NEI Guidelines for Data and Safety Monitoring of Clinical Trials
  • 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:
    • NINDS Guidelines for Monitoring in Clinical Trials
    • NIAMS Data and Safety Monitoring Guidelines
  • 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.

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:
    • Regulated by the FDA. 
    • Intended to support applications for research or marketing permits for FDA-regulated products.
    • Learn more in the Guidance for Institutional Review Boards: Frequently Asked Questions – IRB Registration
  • 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.

Educating Your Disease Community

Educating your disease community about clinical trials can begin even before a clinical trial for your disease is ready to be opened to participants, but it is especially important when a new therapy for your disease is ready to be trialed. 

• Choose what information to include: You want to both help your disease community understand the importance of participating in clinical trials, but also provide them with general information about the clinical trial process and the importance of making an informed decision about participating.
  • Address what it means to be in a clinical trial. For example:
    • Importance of clinical trial process for therapy development. Clinical trials, unlike preclinical research, are designed to answer basic questions about the ways an investigational therapy will interact in people, especially those with a specific disease, instead of, for example, animals.
    • Essential role participants play to potentially bring new treatments to your disease community. Clinical trials help maintain a high standard of medical care across the  entire disease community, not just to the participants. 
    • Responsibilities of participants in clinical trials, including following the protocol for the trial.
  • Explain what a clinical trial is.
    • Clinical trial definition: Broadly, a clinical trial is simply a research study involving human participants, but clinical trials are only undertaken when there is reason to believe the new therapy will improve patient care, quality of life,  and other things.
    • Types of clinical trials: Clinical trials broadly fall into 2 groups, observational or interventional (also called investigational). 
      • Observational studies are just what they sound like; they observe. Generally, researchers monitor participants over a period of time and use the information to better understand the natural history of the disease. 
      • Interventional studies test the safety and effectiveness of a specific therapy to treat a specific disease or symptom associated with the disease.  
    • Phases of interventional clinical trials: Clinical trials can run from early phase 1 to phase 4, with each phase enrolling more participants. Early phase 1 trials use as few as 10 participants; phase 4 can enroll thousands. The number of required participants for each of the phases may be fewer for rare diseases. 
      • Early phase 1 trials (formerly phase 0) are exploratory studies to make certain the therapy is tolerable to humans and does not cause immediate adverse effects or serious harm.  
        • Do not include therapeutic or diagnostic goals. 
        • Enroll up to 10 healthy volunteers 
        • Run a few months (short time frame of studies).
      • Phase 1 trials are usually conducted with healthy volunteers and emphasize safety. In some circumstances, the study will enroll patients who have the disease.
        • Find out what the therapy’s most frequent and serious adverse events are.
        • Understand how the therapy  is metabolized and excreted. 
        • Determine safe levels to be administered.
        • Enroll a small group of participants, most often healthy individuals who do not have the disease.
        • Collect data for a few months.
      • Phase 2 trials gather preliminary data on the effectiveness of the therapy in patients. 
        • Continued evaluation of safety and monitoring of short-term adverse events. 
        • Determine whether the therapy works as expected in people who have a certain disease or condition. 
        • May compare participants receiving the therapy to similar participants receiving a different treatment, usually an inactive substance (called a placebo) or a different previously-approved therapy.
        • Enroll a small group of patients.
        • Collect data for a few months to 2 years. 
      • Phase 3 trials gather more information about safety and effectiveness.
        • Continued evaluation of safety and monitoring of short-term and longer-term adverse events.
        • Determine if the therapy continues to work as expected in a larger group of patients.
        • May include a more diverse patient population.
        • May include gathering data to determine the most effective dosage.
        • May use the investigational therapy in combination with other previously-approved therapies.
        • Enroll a larger group of patients.
        • Collect data for 1 to 4 years.
      • Phase 4 trials are conducted using a therapy that is newly approved by the U.S. Food and Drug Association (FDA) to continue to monitor safety and effectiveness. 
        • Occur after FDA has approved a therapy for marketing. 
        • May be a requirement of the FDA approval or due to a Sponsor commitment. Sponsors may also perform these studies voluntarily. 
        • Gather additional information about a therapy’s safety, efficacy, and optimal use.
        • Enroll a large patient population, with eligibility requirements determined by the FDA approved indications.
        • Can collect data for many years.
    • Clinical trial team: A clinical trial team is made up of many different key members. The team may vary depending on several factors including the type and phase of clinical trial and whether the trial involves multiple centers. Key roles include: 
      • Principal Investigator (PI): A PI  is the lead researcher or clinician who is conducting the clinical trial. The PI is responsible for: 
        • Ensuring that an investigation is conducted according to the signed investigator statement, the investigational plan, and applicable regulations.
        • Protecting the rights, safety and welfare of the subjects under the investigator’s care.
        • Control of the therapy(s) under investigation.
      • Clinical Research Coordinator (CRC): A CRC manages and conducts the day-to-day study activities in accordance with the protocol, applicable regulations, and Good Clinical Practice (GCP) and Institutional Review Board (IRB ) requirements.
      • Sub Investigator (Sub I): The Sub I is a member of the research team designated and supervised by the PI to perform critical study-related procedures and/or to make important study-related decisions.
      • Clinical Research Nurse Coordinator (CRNC):  A CRNC may be required for certain protocol-related activities.
      • Regulatory Coordinator: The regulatory coordinator prepares and maintains IRB submissions and FDA  documents. May also track study progress. 
  • Define unfamiliar terminology. For example, the following terms may be unfamiliar to your disease community.
    • Protocol: Every clinical trial has a protocol, which is an official document that describes the study in detail. The content of protocols varies depending on several factors including the type and phase of trial. Phase 2 and 3 interventional protocols usually describe: 
      • Background and rationale for conducting the study.
      • Risk benefit assessment.
      • Objective(s), including the specific research questions being addressed.
      • Clinical trial design, including overall design, justification of dose, end of study definition, inclusion and exclusion criteria, strategies for recruitment, how the study intervention will be administered.
      • Methodology to minimize bias, such as randomization and blinding, as well as measures addressing protection of participants safety and consideration of any ethical issues. 
      • Statistical considerations, including how the sample size (number of participants) was determined and how the data will be analyzed.
      • Organization of the clinical trial.
    • Sponsor: The  Sponsor of a clinical trial is different from the investigator. The Sponsor manages or finances the clinical trial, but doesn’t conduct investigation. Pharmaceutical Industry Sponsors are common for newly developed therapies. 
    • Eligibility criteria: Listed in the protocol, these are the factors that allow someone to participate in a clinical trial. 
    • Exclusion criteria: These are the criteria that would not allow someone to participate. 
    • Control group: The control group is a subset of clinical trial participants who do not receive the investigational therapy so that their data can be compared to those who do. A control group can be given a placebo or continued on currently accepted therapies.  
    • Placebo: A placebo is an inactive drug, treatment, or therapy, that is given to a group participating in a clinical trial for the purpose of comparison to the group receiving the therapy. The Protocol document will outline whether placebos will be used; they are never used in studies where their use would be inappropriate, unethical, or harmful; in those cases, historical controls are often used. 
    • Informed consent: In clinical trials, informed consent is a complex, multi-stage process. The goal of the informed consent process is to provide sufficient information so that a potential participant can make an informed decision about whether or not to enroll in a study or to continue participation.  It requires:
      • Informing potential participants and/or their guardians thoroughly about:
        • The clinical trial protocol.
        • A participant’s responsibilities.
        • The responsibilities and limitations of the responsibilities of the clinical trial team and Sponsor.
      • Providing the information in easily understood language and in a way that potential participants and/or their guardians can understand.
        • Translating the information to a participant’s native language may be required if participants are not required to speak the primary language being used by the clinical trial team to join the trial.
      • Disclosing potential risks and benefits of the medical intervention under investigation.
      • Allowing potential participants and/or their guardians:
        • A significant amount of time to think about participating.
        • The ability to ask questions and discuss their concerns.
      • Obtaining a patient or their guardian’s voluntary agreement. 
      • NCATS Toolkit provides more information about the informed consent process in the following section: Giving Input on the Informed Consent Process.
    • An IRB or research ethics committee is composed of a group of people independent of the specific research who are tasked with reviewing proposed research plans and related documents before a study involving human participants can begin and then periodically (usually annually) for the study duration. 
      • Not all studies involving human participants must have an IRB review, however any studies involving medical interventions that will require FDA approval prior to marketing will. 
      • NCATS Toolkit provides more information about IRB and the review process in Serve on Review and Monitoring Boards: Institutional Review Boards.
  • Describe common benefits and risks of clinical trials.
    • Specific benefits and risks should be included in information a potential participant receives prior to joining a clinical trial. 
  • Provide links to more information. It can be difficult to cover everything someone may need to know in the depth that everyone may want to know it. Therefore it is helpful to provide links to other reliable resources to allow further exploration.  For example: 
    • Clinical Trials: What Patients Need to Know (periodically updated) is a webpage developed by the FDA and provides links to information about clinical trials. Links include information about the informed consent process, institutional review boards, and the differences between clinical trials and medical treatment.
    • From Molecules to Medicine: Clinical Research (2018) is one of the Global Genes web-based information rare toolkits. It provides basic information about clinical trials, how to find them, and what it means to participate in a trial in an easy to understand language and format.
    • ClinicalTrials.gov is a searchable online registry of recent and active clinical trials developed by the NIH through the National Library of Medicine. The website also includes information about how to find studies and learning about clinical trials in general.
    • Five Principles: Returning Genetic Testing Results to Research Participants (2014) is a paper containing the five basic principles that the authors propose should be followed when returning research results to individual participants. 
• Develop an FAQ: An FAQ can be a webpage or in printed format that can be shared at conferences or in mailings.
  • Dispel any common myths. For example:
    • Participants in clinical trials are not guinea pigs; there are clear safety regulations.
    • Participants can withdraw from a clinical trial before its conclusion.
  • Address concerns that might be unique to your disease community.
  • Provide some of the general information and terminology considered above in “Choose what to include.”
• Share the information: There are different ways to provide information, each potentially requiring different amounts of time and possibly reaching different people. You know those affected by your disease community best, so you will want to choose the method or combination of methods that will work best for your group. 
  • Post on your website. Depending on the current availability of clinical trials for your disease, you may wish to consider sharing general information about clinical trials by:
    • Highlighting internal webpages on the home page.
    • Dedicating a full tab section to information about clinical trials.
    • Including under a tab dedicated to Research or What You Can Do.
    • Sending emails with links to your webpages focused on research periodically to your group members or including in e-newsletters.
    • Posting the links on social media associated with your group. 
  • Hold an information webinar or virtual meeting. 
    • Include the experience of individuals or families who have participated in a trial.
    • Allow questions to be asked by those attending the webinar.
    • A video recording of the webinar can be posted on your website. 
  • Schedule sessions into your annual scientific or family conference.
  • Also use “snail” mail. Not everyone in your disease community has internet access, so doing a periodic mailing may help reach those who might not have easy access to websites and webinars
  • If there are dedicated clinics for your rare disease, provide FAQs, brochures, or infographics that can be picked up by patients or their families .

Overview

Patients and their families often need help learning about the purpose of clinical trials, as well as how to find and evaluate studies that are enrolling participants. Your patient group can help identify patients who may be eligible to participate in clinical trials when a clinical trial holds promise of an effective, safe new treatment.

Tips for Success

• Keep presentations balanced. Although you want your disease community to participate in clinical trials, you also want each person or family to make the right choice for their situation. Balanced information is key to retaining your patient community’s trust.
• Set realistic expectations for any clinical trial. It is important for your disease community to understand that even interventions that make it to Phase 3 may not gain FDA approval. 
• Consider an opt-out for emails and social media about new clinical trials for those that do not want to participate. 
• Explore the websites of other groups to see how they provide general information about clinical trials as well as information about specific trials. 
• Reach out to the study coordinator and/or principal investigator if a trial pops up for your disease community that you were not expecting.

Increasing recruitment

You can decrease the time it takes to recruit participants for clinical trials by alerting your disease community to upcoming and newly launched trials. However, you may want to establish a review process to determine which clinical trials your group will support. Including members of your Medical/Scientific Advisory Board or outside independent researchers and clinicians in your review process will strengthen your decision. If you decide to support recruitment, you can: 

• Email members when a new trial is added.
• Highlight new trials on the homepage of your website.
• Post on your group’s social media.
• Send alerts through “snail mail” or include in your printed newsletter if you have one.
• Identify members of your disease community who are most likely to meet eligibility criteria and are interested in joining clinical trials using your registry.