Resources for Researchers and Clinicians
A variety of government agencies and other organizations offer information, resources, and services for rare disease researchers and clinicians. Click on the accordions below to find a variety of these resources. See also our pages offering resources for early stage investigators and funding opportunities.
So Many Procedures, So Little Time: How to Develop an Effective Schedule of Events
Regulatory Webinar Series: Presented by Mike Fusakio, PhD; Nathan Hawk, BSPS; Samantha Sonoda, MS, CCRA; and Lisa M. Tully, MA, CCRP, all affiliated with Cincinnati Children's Hospital Medical Center.
Single IRB (Institutional Review Board) Presentation & Panel Discussion
Regulatory Webinar Series: Features Nichelle Cobb, PhD, Smart IRB Director at University of Wisconsin-Madison. Panelists include James Cnota MD (Cincinnati Children's), Jeremy Corsmo, MPH (Cincinnati Children's), Pooja Khatri, MD, MSC (University of Cincinnati) and Mike Linke, PhD (University of Cincinnati).
COVID-19: Information for NIH Applicants and Recipients of NIH Funding
A centralized information page that includes links to a variety of guidance, information, resources, FAQs and NIH funding opportunities relating to COVID-19, from the NIH Central Resource for Grants and Funding Information.
NCATS: A Translational Approach to Addressing COVID-19
Offers an overview of research activities that the NIH's National Center for Advancing Translational Sciences is supporting, spanning the translational science spectrum to address the novel coronavirus 2019 (SARS-CoV-2) and the disease it causes (COVID-19).
Building a Community Engagement Model for Rare Disease: A Community Ambassador Approach
This presentation will address the unique concerns of diversity, equity, inclusion, and accessibility in the rare disease space. Dr. Washington will share a number of rare disease DEIA projects that she has spearheaded, highlighting grassroots collaborative engagement approaches.
DEIA Guest Speakers: Reporting Race and Ethnicity and Inclusive Language in Medical Publication
Terminology, usage, word choice, and representativeness are critically important in medical publication, especially when describing people and research participants and when discussing race and ethnicity and other demographic characteristics or variables. This session will provide recommendations and suggestions from the AMA Manual of Style that encourage fairness, equity, consistency, and clarity in use and reporting of race and ethnicity in medical publication, with specific examples.
Diversity, Equity, Inclusion, Accessibility Webinar: Community Engagement in Translational Research
Rare diseases overall are still unrecognized, especially in Hispanic communities where access to sub-specialist care and diagnostic tools is limited or unavailable. This webinar features Wilfredo De Jesús Rojas, MD, FAAP, MSc, fellow of the American Academy of Pediatrics and a board certified pediatric pulmonologist/physician-scientist. Dr. Wilfredo De Jesús Rojas was inspired to become a pediatric pulmonologist and a physician-scientist to be an advocate and study rare lung diseases in Puerto Rican children.
Diversity, Equity & Inclusion Webinar: The Redlining of Healthcare
How have housing, education and economic policies influenced social determinants of health? This webinar features Tonya Winders, MBA, of the Allergy & Asthma Network and explores explores how we can move forward to reduce systemic barriers to ensure better health outcomes.
NIH-funded NASEM report recommends best practices for use of population descriptors in genetics and genomics research
This report shares NIH's ongoing efforts to safeguard scientific integrity in genomics and promote the responsible design of research studies so that all populations benefit from scientific advances.
Conceptualizing, Contextualizing, and Operationalizing Race in Quantitative Health Sciences Research
This paper outlines pitfalls in the conceptualization, contextualization, and operationalization of race in quantitative population health research and provides recommendations on how to appropriately engage in scientific inquiry aimed at understanding racial health inequities.
Dismantling "Race" in Health Research
In this paper, we first present arguments for inappropriate use of race in health research and then discuss alternative explanations for health disparity findings that use race as a predictor.
Inclusion of People with Disabilities in Research to Improve Medication Adherence: A Systematic Review
In this review, the inclusion and exclusion criteria of the 182 studies included in the Cochrane Review on Interventions for Enhancing Medication Adherence were evaluated for their inclusion of people with disabilities.
Updated Guidance on the Reporting of Race and Ethnicity in Medical and Science Journals
The goal of this guidance is to provide recommendations and suggestions that encourage fairness, equity, consistency, and clarity in use and reporting of race and ethnicity in medical and science journals.
Race and Genetic Ancestry in Medicine — A Time for Reckoning with Racism
In the United States, race, ancestry, genetics, and medicine are inextricably linked in a complex and fraught history. Medicine is replete with examples of racial injustice inflicted by the use of race and ethnicity as biologic constructs to engender hierarchical discrimination.
The evolution of disability language: Choosing terms to describe disability
This publication discusses the history and evolution of disability language, explores current trends, and recommends language for academic articles.
Race, ethnicity and racism in the nutrition literature: an update for 2020.
Reviews the limitations of classifying research participants by race and recommends a series of steps for authors, researchers and policymakers to consider.
Enhancing the Diversity of Clinical Trial Populations – Eligibility Criteria, Enrollment Practices, and Trial Designs Guidance for Industry
US Food and Drug Administration guidance which recommends approaches that sponsors of clinical trials intended to support a new drug application or a biologics license application can take to increase enrollment of underrepresented populations in their clinical trials.
NIH Data Management and Sharing policy
New NIH Policy for Data Management and Sharing that goes into effect January 25, 2023 and replaces the existing 2003 NIH Data Sharing Policy. Updated to keep pace with scientific and technological advancements and emphasizes the NIH’s commitment to data management and sharing.
FDA CDER & NIH NCATS Regulatory Fitness in Rare Disease Clinical Trials Workshop
On May 16 and 17, 2022, the FDA’s Center for Drug Evaluation and Research (CDER) and NIH’s National Center for Advancing Translational Sciences (NCATS) hosted a jointly sponsored virtual workshop on regulatory fitness in rare disease clinical trials.