Orphan Grants Scientific Publications

FDA Office of Orphan Product Development (OOPD)

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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.

OOPD Publications

Publications by staff in the FDA Office of Orphan Product Development.

Trends in drug development for amyotrophic lateral sclerosis (February 2024, Nature Reviews Drug Discovery)
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 (June 2023, Contemporary Clinical Trials)
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 (September 2020, Orphanet Journal of Rare Diseases)
Precision Medicines’ Impact on Orphan Drug Designation by Christine M. Mueller, Gayatri R. Rao, and Katherine Needleman (November 2019, Clinical and Translational Science)
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 (July 2019, Clinical Pharmacology & Therapeutics)
FDA’s Office of Orphan Products Development: providing incentives to promote the development of products for rare diseases by Soumya Patel and Katherine Needleman (July 2019, Journal of Pharmacokinetics and Pharmacodynamics)

Grantee Publications

Nivedita L. Bhushan PhD, RTI International
Remote-Use Applications of the ALSFRS-R Clinical Outcome Assessment Tool: A Scoping Review
Value in Health (May 2024)
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 (April 2022)
Dr. Neil Desai, Aadi LLC
nab-Sirolimus for Patients With Malignant Perivascular Epithelioid Cell Tumors
Journal of Clinical Oncology (October 2021)
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 (January 2021)
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 (October 2020)
Dr. Phillip Low, Mayo Clinic
Intrathecal administration of autologous mesenchymal stem cells in multiple system atrophy
Neurology (May 2019)
Dr. Guido Magni, River Division Development Corporation
Teprotumumab for Thyroid-Associated Ophthalmopathy
New England Journal of Medicine (May 2017)