Why should researchers and IRB be concerned with the legal status of GSD people?

This natural history study will serve as a repository of clinical, laboratory, and biochemical information on individuals with GSD VI or GSD IX. This information will allow a more definitive description of glycogen phosphorylase (GP) and phosphorylase kinase (PhK) deficiency to be developed, which will permit development of treatment strategies for these diseases.

Duke will be the only site where this study takes place. However, since these are rare disorders, participants who receive care at other institutions will be included. The investigators will collect retrospective data from patient charts on diagnosed individuals, as far back as necessary to capture the clinical course of the disorder. Prospective data collected from patient charts after enrollment will be captured as well. Participant's medical records will be continually reviewed for the duration of the study.

Data will be collected from medical records and will only pertain to clinically relevant information, including, but not limited to: demographic and diagnostic information, tissue biopsy results, medical and family history, review of systems, imaging studies, results of liver and/or muscle testing, and urine and blood laboratory results.

Community-based Participatory ResearchOne way of addressing the needs of GSD peoples in research is to engage in Community-basedparticipatory research (CBPR). The NIH defines it as "…an applied collaborative approach thatenables community residents to more actively participate in the full spectrum of research,through conception - design - conduct - analysis - interpretation - conclusions - communicationof results, with a goal of influencing change in community health, systems, programs or policies"(NIH Office of Behavioral 2014).As the social status of GSD people is rapidly shifting with progress and backlash from politicalstruggles for equality, research directly on GSD communities should engage members of thosecommunities in the design, execution, and dissemination of the research whenever possible.The use of CBPR is key not only for social-behavioral research, but also for communityengagement for biomedical research in GSD communities, particularly for HIV drug or vaccinetrials, and to help break down mistrust from historical abuse and neglect.Role of IRBs and ResearchersIRBs should:Review protocols for inclusion criterion that will likely exclude GSD subjects and requirea justification for those exclusion criteria. Likewise, consider if recruitment strategieswill result in a de facto exclusion of some or all GSD subjects.Seek consultants with expertise in specific segments of the GSD community to provideinformation on research in those communities, if such expertise is not present on the IRB.Assess whether the GSD subjects to be enrolled in a specific research protocol arevulnerable and require additional human subject protections, such as research subjectadvocates.Consider "Community Harm"

Page95of160Will accidental outing of one subject result in accidental outing of others notparticipating in the research?Do recruitment flyers include slurs that will contribute to the hostile climate inwhich GSD people live?Pay particular attention to plans for communication and follow-up with subjects to ensurethat research participation does not result in researchers inadvertently revealing thesexual orientation or gender identity of subjects who may not be "out" in some or allarenas of their lives.Researchers should:Consider the participation of GSD subjects in studies not specifically related to gender orsexual identity and consider ways to make all subjects comfortable, regardless of sexualor gender orientation. This includes:Review all materials to be seen by subjects (recruitment materials, consentdocuments, information sheets, questionnaires, diaries, etc.) for heteronormative(assuming all participants are heterosexual) or gendernormative (assuming allparticipants are cis-gendered) language.

Participants in research should reflect the diversity of our culture and conditions, taking into account race, ethnicity, gender, age, etc. The lack of diversity among research participants has serious ethical and research consequences. This includes impeding our ability to generalize study results, make medical advancements of effective therapies, and it prevents some populations from experiencing the benefits of research innovations and receipt of high quality care.

Enrolling an adequate number of participants in research is a pervasive problem. If studies do not meet their target enrollment in the allotted time period, they may not complete their scientific goals and make take a significant financial loss after investing heavily in study start-up. In addition, there are ethical concerns because if researchers cannot complete the study due to under enrollment, they may not be able to carry out the study aims outlined in the participant consent form. A critical piece of the recruitment problem is a lack of diversity among research participants.

In the Revitalization Act of 1993 and additional amendments, the NIH mandated the appropriate inclusion of minorities and women in all NIH-funded research and stated clearly that, “since a primary aim of research is to provide scientific evidence leading to a change in health policy or standard of care, it is imperative to determine whether the intervention or therapy being studied affects women or men or members of minority groups and their subpopulations differently.”

Clinical research has shown that patients’ reactions to medical treatments vary depending on race, ethnicity and gender.  For example, up to 75% of Pacific Islanders are unable to convert an antiplatelet drug into its active form and therefore are at higher risk for adverse outcomes following angioplasty. (Burchard, 2015). If the study population had not included diverse participants, this difference would not have been discovered and patient care could have effected.

Despite national efforts led by the NIH and the FDA, research participation remains low for racial/ethnic minorities and other underrepresented groups such as women, low socioeconomic status populations, and older adults.  But as a researcher, you can make an impact. When study participants reflect the rich diversity in our communities, studies can generate information about causes of and strategies to address differences, including policies and practices to eliminate disparities

Want to learn more? Here are some resources to help get started: 

Report: Improving Representation in Clinical Trials and Research: Building Research Equity for Women and Underrepresented Groupsfrom the National Academies of Science, Engineering and Medicine.

Webcast: Recruitment of Underrepresented Study Populations: This seminar featured speakers and panelists discussing the importance of including underrepresented populations in research, with tips and real examples of successful approaches and lessons learned. This event was co-sponsored by the CTSI Participant Recruitment Program and School of Medicine Differences Matter Research Action Group.

CTSI Consultation Services offers consultations on all aspects of recruitment—including recruitment of underrepresented populations. The first hour is free! Visit consult.ucsf.edu to put in a request.

CTSI Research Action Group for Equity (RAGE)  provides support and infrastructure for UCSF researchers to diversify their study populations. Consultations are available via Consultation Services.

Tips from research teams on how to create strategies to include underrepresented populations in their research. 

UCSF IRB Guide to Translating Study Documents  provides guidance on translating study materials, and includes a list of translation companies.

Free Plain language consultations to help ensure your research communications are readable, understandable, and actionable. The consultation is free and easy and can be done completely over email. At the end of the consultation you will receive a plain language summary of your study with easy-to-understand phrases that you can use in your recruitment materials.

Recruitment materials image swap. Do the images in your recruitment materials reflect the diversity of the population you are trying to recruit? PRP is offering free stock images to promote diversity in recruitment materials.

When an IRB is reviewing a research study and they are considering?

Which of the following studies would need IRB approval?

What is an example of a situation where deferential vulnerability?

What are the main elements necessary for ensuring that costs are appropriately charged?