Americans depend on the U.S. Food and Drug Administration to help ensure that the foods they buy and consume are safe.
Our teams routinely work with food producers on voluntary recalls, and when necessary and where applicable, mandate recalls in order to keep people from getting sick or being harmed. To promote these goals, FDA is advancing an important new policy.
The U.S. Food and Drug Administration announced that it has awarded 12 new clinical trial research grants totaling more than USD18 million over the next four years to enhance the development of medical products for patients with rare diseases. These new grants were awarded to principal investigators from academia and industry across the country.
The FDA awarded the grants through the Orphan Products Clinical Trials Grants Program.
This program is funded by Congressional appropriations and encourages clinical development of drugs, biologics, medical devices or medical foods for use in rare diseases. The grants are intended for clinical studies evaluating the safety and effectiveness of products that could either result in, or substantially contribute to, the FDA approval of products targeted to the treatment of rare diseases. Grant applications were reviewed and evaluated for scientific and technical merit by more than 100 rare disease experts, which included representatives from academia, the National Institutes of Health and the FDA.
The grant recipients, principal investigators and approximate funding amounts, listed alphabetically, are: Alkeus Pharmaceuticals, Inc. (Cambridge, Massachusetts), Leonide Saad, phase 2 study of ALK-001 for the treatment of Stargardt disease – USD1.75 million over four years; Arizona State University-Tempe Campus (Tempe, Arizona), Keith Lindor, phase 2 study of oral vancomycin for the treatment of primary sclerosing cholangitis – USD2 million over four years; Cedars-Sinai Medical Center (Los Angeles), Shlomo Melmed, phase 2 study of seliciclib for the treatment of Cushing disease – USD2 million over four years; Columbia University of New York (New York), Yvonne Saenger, phase 1 study of talimogene laherparepvec for the treatment for advanced pancreatic cancer – USD750,000 over three years; Emory University (Atlanta), Eric Sorscher, phase 1/ 2 study of Ad/PNP fludarabine for the treatment of head and neck squamous cell carcinoma – USD1.5 million over three years.
Fibrocell Technologies, Inc. (Exton, Pennsylvania), John Maslowski, phase 1/2 study of gene-modified ex-vivo autologous fibroblasts for the treatment of dystrophic epidermolysis bullosa – USD1.5 million over four years; Johns Hopkins University (Baltimore), Amy Dezern, phase 1/2 study of CD8-reduced T cells for the treatment of myelodysplastic syndrome or acute myeloid leukemia – USD750,000 over three years; Oncolmmune, Inc. (Rockville, Maryland) Yang Liu, phase 2b study of CD24Fc for the prevention of graft versus host disease – USD2 million over four years; Patagonia Pharmaceuticals, LLC (Woodcliff Lake, New Jersey), Zachary Rome, phase 2 study of PAT-001 (isotretinoin) for the treatment of congenital ichthyosis – USD1.5 million over three years; The General Hospital Corporation (Boston), Stephanie Seminara, phase 2 study of kisspeptin for the treatment of dopamine agonist intolerant hyperprolactinemia – USD1.4 million over four years; University of Minnesota (Minneapolis), Kyriakie Sarafoglou, phase 2a study of subcutaneous hydrocortisone infusion pump for the treatment of congenital adrenal hyperplasia – USD1.4 million over three years; University of North Carolina at Chapel Hill (Chapel Hill, North Carolina), Matthew Laughon, phase 2 study of sildenafil for the prevention of bronchopulmonary dysplasia – USD2 million over four years
One-third (33 percent) of the new awards aim to accelerate cancer research by enrolling patients with rare forms of cancer, including advanced pancreatic cancer, head and neck squamous cell carcinoma, myelodysplastic syndrome and acute myeloid leukemia. Another 25 percent of the new awards fund studies evaluating drug products for rare endocrine disorders, including Cushing disease, dopamine agonist intolerant hyperprolactinemia and congenital adrenal hyperplasia. Another study addresses an unmet need in primary sclerosing cholangitis, a rare, chronic and potentially serious bile duct disease.
About 42 percent of the grants fund studies which enroll children and adolescents, targeting a variety of rare diseases in children such as Stargardt disease, a juvenile genetic eye disorder that causes progressive vision loss; dystrophic epidermolysis bullosa, a genetic condition that causes the skin to be fragile resulting in painful blisters; and bronchopulmonary dysplasia, a serious lung condition that affects infants.
To date, the program’s grants have supported research that led to the marketing approval of more than 60 orphan products. Among the recent product approvals which were supported by studies funded by this grants program are a marketing approval for a much-needed treatment of human immunodeficiency virus type 1 (HIV-1) infection in adults with multidrug resistant HIV-1 infection and another approval to reduce the acute complications of sickle cell disease in adult and pediatric patients.
The FDA is also currently supporting six natural history studies for rare diseases to further advance the mission of bringing new therapies to market.
U.S. Food and Drug Administration took new steps as part of its broader efforts to address the opioid crisis by approving the final Opioid Analgesic Risk Evaluation and Mitigation Strategy (REMS). This new plan includes several measures to help better communicate the serious risks about the use of opioid pain medications to patients and health care professionals. This expanded REMS now, for the first time, applies to immediate-release (IR) opioid analgesics intended for use in an outpatient setting.
Sun Pharma Advanced Research Company Ltd. announced U.S. Food and Drug Administration (USFDA) approval for the New Drug Application (NDA) of XELPROSTM (latanoprost ophthalmic emulsion) 0.005% for the reduction of elevated intraocular pressure (IOP, or pressure inside the eye) in patients with open-angle glaucoma or ocular hypertension. This approval is from Sun Pharma’s Halol (Gujarat, India) facility.
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Epidemics don’t occur overnight. U.S. Food and Drug Administration must remain vigilant and aggressive against trends that threaten to reverse our progress, or substances that have the potential to cause new epidemics of abuse.
The U.S. Food and Drug Administration announced that it has awarded five grants totaling up to USD 6 million per year over the next five years to Pediatric Device Consortia (PDC) across the country that will provide advice and support services to innovators of children's medical devices. The program aims to enhance the development, production and distribution of pediatric medical devices and has awarded USD 37 million to various consortia since 2009.
The PDC grant recipients and their principal investigators for 2018 are the following:
• Philadelphia Pediatric Medical Device Consortium, Matthew Maltese, Ph.D.
• National Capital Consortium for Pediatric Device Innovation 2.0, Kolaleh Eskandanian, Ph.D., M.B.A.
• Southwest National Pediatric Device Consortium, Chester Koh, M.D.
• University of California San Francisco-Stanford Pediatric Device Consortium, Michael Harrison, M.D.
• West Coast Consortium for Technology and Innovation in Pediatrics, Juan Espinoza, M.D.
Specific areas of expertise provided by the consortia to medical device innovators include advising on issues related to: intellectual property, prototyping, engineering, laboratory and animal testing, grant-writing and clinical trial design to help foster and guide the advancement of medical devices specifically for children.
Of the estimated USD 6 million granted this year, approximately USD 1 million will be used for the Real World Evidence (RWE) Demonstration Project, in which three of the consortia will conduct RWE projects in the pediatric space that develop, verify and operationalize methods of evidence generation, data use and scalability across device types, manufacturers and the health care system. The FDA intends to use the information gathered through this initiative to further efforts to incorporate RWE into the agency’s work.
Legislation passed by Congress in 2007 established funding to be distributed as grants for nonprofit consortia to help stimulate projects to promote the development and availability of pediatric medical devices. This legislation was re-authorized as part of the FDA Safety and Innovation Act of 2012 and again in the FDA Reauthorization Act of 2017 to run through fiscal year 2022.
The PDC Grants Program was launched in 2009, and this is the fourth time the FDA has awarded grants. Each group’s grant runs for five consecutive years. Funding for fiscal year 2018 is approximately USD 1 million to USD 1.35 million per consortium. Support for the four additional years will be contingent upon annual appropriations, availability of funding and satisfactory awardee performance.
The consortia have assisted or advised more than 1,000 medical device projects since the program began. There are now 19 pediatric medical devices available to patients as a result of this grants program, including a needle-free blood collection device that attaches to peripheral IV systems for use as a direct blood draw device; a surgical vessel sealing system for use in open and laparoscopic general surgical procedures to seal blood vessels and vascular bundles and a rapid infusion device that delivers fluids to a patient’s vascular system.
The U.S. Food and Drug Administration approved Cassipa (buprenorphine and naloxone) sublingual film (applied under the tongue) for the maintenance treatment of opioid dependence. This action provides a new dosage strength (16 milligrams/4 milligrams) of buprenorphine and naloxone sublingual film, which is also approved in both brand name and generic versions and in various strengths.
Advancing better nutrition is one of my top priorities and implementing the update to the iconic Nutrition Facts label — the first overhaul in 20 years — is a key part of that commitment.
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The practice of compounding medicines can provide important public health opportunities. The FDA’s compounding program – including implementation of the compounding provisions of the law – is a priority for the agency.
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