Who We Are?
The Organizations Board of Directors is composed of highly educated Ph.Ds. and M.D.s who have specific credentials and are qualified to evaluate research proposals and, based on the scientific evaluation of the research area of the proposals, determine which research studies to fund. All board members have more than 20 years of drug development experience in the manufacturing, nonclinical and clinical areas.
- We help with strategies for druggability, manufacturing, nonclinical proof of concept and toxicity studies, clinical design, and regulatory pathway.
- We provide a free research fund to help you move faster.
- We will work with you to achieve ultimate successful rare disease treatment.
Departments
FAQ
The Organization may fund strong research labs overseas, in Europe and or Asia, if there are studies on rare diseases conducted abroad. There will be no relationship between the Organization and the foreign recipient except the shared desire to support the shared mission. No foreign organization will accept earmarked contributions.
The Organization will make grants to other organizations that conduct research into causes, cures, and treatments of rare children’s diseases. The recipient organizations will be required to submit a detailed proposal about their research study. The Organizations Board will review the proposal and determine whether to fund it. The recipient organization will have to submit progress reports and a written accounting to the Organization as to what, where, and how the granted funds were utilized.
Funding will come from fundraising efforts including personal solicitations from the board members to individuals in the medical profession, private foundations, public charities as well as fundraising events.
Each submission from a recipient will include an acknowledgment that the Organization has the authority to stop, withhold, and recover any funds provided if the recipient organization is deficient in demonstrating that all funds are used properly, and in a manner consistent with the purpose and intent of the funding. If the funds are misused, or appear to be misused, the recipient organization will be required to make restitution of any misused funds and must agree to this as a condition of the grant. Regular reports will be required for all funded programs.
Progress reports and final reports will include specific detailed explanations as to what the organization accomplished with the funds. In addition, the Organization may conduct site visits on future visits to verify that the funds are being used for its stated purposes.
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EVERY CHILD, EVERYWHERE. EQUAL VALUE.
DONATE NOWNews
April 20, 2021
Rare Disease Institute in Children’s National Hospital now is seeing patients at their their new location on the Research & Innovation Campus, 7125 13th Pl NW Washington, DC 20012.
January 20, 2021
You can search any pediatric rare disease designation at FDA database
April 21, 2020
Audrey Jia, M.D., Ph.D.
Dr. Jia holds a Master degree in Bioscience Regulatory Affairs from Johns Hopkins University, and a Ph.D. degree in Microbiology and Molecular Genetics from Emory University. Prior to that, Dr. Jia obtained her medical degree from Peking University, and passed USMLE. Dr. Jia has 20 years of combined experience in biologic drug development and regulatory review. Dr. Jia spent 10 years working on biological product research and production in Pfizer, AbbVie and Protein Design Labs Inc. Additionally, Dr. Jia was a full-time senior CMC reviewer for IND/BLA review of biological products in US FDA. She performed numerous IND reviews, BLAs (including post approval reviews), and US/international cGMP pre-approval inspections (PAIs).
Yan Bai, M.D., Ph.D.
Dr. Bai holds a Ph.D. degree in Epidemiology from Emory University, and a medical degree from Peking University Health Science Center. He had his post-doc training in Epidemiology from NIH. Dr. Bai has twenty years of experience in pharmacoepidemiology, clinical development, drug safety and cancer research with strong skills in epidemiology strategy, medical safety evaluation and understanding of the US and global pharmacovigilance processes. He is a regulatory expert in clinical oncology and endocrinology, and currently serves as a senior consultant in DataRevive USA LLC. Before that, He worked at Allergan Inc., Vertex Pharmaceuticals, Sanofi-Aventis, and Johnson & Johnson.
Julia Carrier, Ph.D.
Dr. Carrier holds a Ph.D. degree in Immunology from Emory University, and a bachelor of science from Peking University Health Science Center. She had her post-doc training in Immunology from Harvard University. Dr. Carrier has twenty years of experience in Immunology, nonclinical PK/PD and toxicology. She has extensive experience in nonclinical studies and study designs in oncology, infectious disease, liver disease, cardiology, and ophthalmology disease. She is a regulatory expert in nonclinical area and currently serves as a lead consultant in DataRevive USA LLC. Before that, she worked at Pfizer Centers for Therapeutic Innovation for many years as nonclinical lead.