Orphan Grants Spotlight
Experts from FDA and the orphan products program grantees often publish articles in scientific journals to share information about rare disease product development, policies and results from funded studies with the scientific community. Some recent publications as well as historical grant announcements and awards are below.
- The effect of the COVID-19 pandemic on US Food and Drug Administration funded clinical trials and natural history studies for rare diseases by Kathleen L. Miller and Katherine Needleman
- FDA orphan products clinical trial grants: assessment of outcomes and impacts on rare disease product development by Kathleen L. Miller, Christine Mueller, Gumei Liu, Katherine Needleman, Janet Maynard
- An analysis of follow-on development in new drug classes, January 1986-June 2018 by Michael L. Lanthier, Kirk W. Kerr and Kathleen L. Miller
- FDA’s Office of Orphan Products Development: providing incentives to promote the development of products for rare diseases by Soumya Patel and Katherine Needleman
- Precision Medicines’ Impact on Orphan Drug Designation by Christine M. Mueller, Gayatri R. Rao, and Katherine Needleman
- Dr. Neil Desai, Aadi LLC
“nab-Sirolimus for Patients With Malignant Perivascular Epithelioid Cell Tumors”
Journal of Clinical Oncology
https://ascopubs.org/doi/10.1200/JCO.21.01728?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200pubmed - Dr. David Lynch, Children’s Hospital of Philadelphia
“Simultaneous Quantification of Mitochondrial Mature Frataxin and Extra Mitochondrial Frataxin Isoform E in Friedreich’s Ataxia Blood”
Frontiers in Neuroscience
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9098139/ - Dr. Kelly Dooley, Johns Hopkins University School of Medicine
“Pretomanid Pharmacokinetics in the Presence of Rifamycins: Interim Results from a Randomized Trial among Patients with Tuberculosis”
Antimicrobial Agents in Chemotherapy
https://pubmed.ncbi.nlm.nih.gov/33229425/ - Dr. Phillip Low, Mayo Clinic
“Intrathecal administration of autologous mesenchymal stem cells in multiple system atrophy”
Neurology
https://n-neurology-org.fda.idm.oclc.org/content/93/1/e77.long - Dr. Dung Le, Johns Hopkins University School of Medicine
“A Phase II Study of Allogeneic GM-CSF–Transfected Pancreatic Tumor Vaccine (GVAX) with Ipilimumab as Maintenance Treatment for Metastatic Pancreatic Cancer”
Clinical Cancer Research
https://aacrjournals.org/clincancerres/article/26/19/5129/82710/A-Phase-II-Study-of-Allogeneic-GM-CSF-Transfected - Dr. Guido Magni, River Division Development Corporation
“Teprotumumab for Thyroid-Associated Ophthalmopathy”
New England Journal of Medicine
https://www.nejm.org/doi/10.1056/NEJMoa1614949?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200www.ncbi.nlm.nih.gov
- RFA-FD-23-030: Systematic Review of Clinical Outcome Assessments (COAs) for Communication Brain-Computer Interface Devices (cBCIs) in Amyotrophic Lateral Sclerosis (ALS) (UH2/UH3) Clinical Trials Not Allowed (nih.gov)
- RFA-FD-23-028: Natural History and Biomarker Studies of Rare Neurodegenerative Diseases (U01) Clinical Trials Optional (nih.gov)
- RFA-FD-21-001: Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01) Clinical Trials Required (nih.gov)
- RFA-FD-20-001: Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01) (nih.gov)
- RFA-FD-19-001: Natural History Studies Addressing Unmet Needs of Rare Diseases: Orphan Products Research Project Grant (R01) (nih.gov)
- RFA-FD-15-001: Clinical Studies of Safety and Effectiveness of Orphan Products Research Project Grant (R01) (nih.gov)
- RFA-FD-16-043: Natural History Studies for Rare Disease Product Development: Orphan Products Research Project Grant (R01) (nih.gov)
- NOT-FD-18-006: Notice of Cancellation of the October 2018 Receipt Date for RFA-FD-16-043 (nih.gov)
- RFA-FD-11-001: Clinical Studies of Safety and Effectiveness of Orphan Products Research Project Grant (R01) (nih.gov)
- RFA-FD-09-001: Clinical Studies of Safety and Effectiveness of Orphan Products Research Project Grant (R01) (nih.gov)
October 2022
October 2021
October 2020
October 2019
- FDA awards 12 grants to fund new clinical trials to advance the development of medical products for the treatment of rare diseases - October 8, 2019
- FDA awards 2 grants for natural history studies in rare diseases (October 8, 2019)
October 2017
- FDA awards 6 grants for natural history studies in rare diseases - October 6, 2017
- FDA awards 15 grants for clinical trials to stimulate product development for rare diseases - October 6, 2017
October 2016
September 2015
October 2013
September 2013
2012