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APRIL 2019    A Monthly Review of Articles of Interest for the Clinical Community

..This Month's Clinical Focus:  ORPHAN DRUGS..

 

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  Orphan Designation  - Not Just for Drugs Anymore 

The Humanitarian Device Exemptions (HDE) are devices that have been approved to treat or diagnose a disease or condition that affects fewer than 4,000 individuals in the United States per year.

FDA LogoIn addition to drugs and biologics, there has been progress in the development of medical devices for rare diseases. Since 1990, the FDA has approved 76 medical devices for orphan indications under the Agency’s Humanitarian Device Exemption program. In 2018, the FDA approved 2 devices in the program. One device helps patients with acute perforations of native coronary arteries and coronary bypass graft vessels and the other device received approval to expand its use in patients with nephrotic syndrome associated with focal segmental glomerulosclerosis.

Humanitarian Device Exemption (HDE) Program draft guidance provides clarity about HDE program changes resulting from the 21st Century Cures Act and the FDA Reauthorization Act of 2017, in addition to explaining the criteria the FDA considers to determine if “probable benefit” has been demonstrated as part of the Agency’s decision-making process regarding marketing authorization for a humanitarian use designation.

Playing kids-3171905_640 by gustavorezende PIXABAY Free Lic CC0
Pediatric Device Consortia Grant Program
FDA funds consortia which provide expert advising and support services to innovators of children's devices. These services include business and regulatory consulting, as well as device testing capabilities.  Specific areas of expertise provided by the consortia include intellectual property advising; prototyping; engineering; laboratory and animal testing; grant-writing; and clinical trial design.  The PDC Grants Program was launched in 2009 after Section 305 of the Pediatric Medical Device Safety and Improvement Act of 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.

 

SOURCE: Used by Permission of FDA (Online Public Domain)
 


Grants Awarded to Principal Investigators from Academia and Industry

 

12 new clinical trial research grants totaling more than $18 million over the next four years

 

“Since its creation in 1983, the Orphan Products Grants Program has provided more than $400 million to fund more than 600 new clinical studies,” said Debra Lewis, O.D., acting director of the FDA’s Office of Orphan Products Development. “We are encouraged to see so much interest in our grants program and are pleased to support research for a variety of rare diseases that have little, or no, treatment options for patients.” The program is funded by Congressional appropriations and encourages clinical development of drugs, biologics, medical devices or medical foods for use in rare diseases.

science-and-industry-1182713_640 by markmags PIXABAY Free Lic CC0

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.
 

New scientific advances offer more opportunities to develop these potential cures. With efficient regulation, proper incentives for product development and the continued support of patients, providers and researchers have more opportunities to pursue these advances than ever before. For 35 years, the FDA has provided much-needed financial support for clinical trials of potentially life-changing treatments for patients with rare diseases. This funding helps support early-stage development activities targeting rare diseases that don’t have effective treatments.

By providing seed capital, these FDA-administered grants enable researchers to prove out important concepts. The FDA grants also provide some important recognition to promising development programs that ultimately can help researchers attract additional funding.

 

SOURCE: Used by Permission of FDA (Online Public Domain)


Image credits: Kids playing courtesy of gustavorezende; Brilliant ideas courtesy of markmags; both via PixaBay.com Free Lic CC0

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