FDA to Close Loophole Allowing Companies to Skirt Pediatric Study Requirements
Thursday, September 14, 2017 12:00 AM

FDA commissioner Scott Gottlieb announced Tuesday that the agency will release guidance to close a loophole that allows companies to avoid their obligation to study pharmaceuticals in pediatric populations. 

The situation, according to Gottlieb, arises if sponsors received an orphan designation for a pediatric subtype of an otherwise common and non-orphaned adult disease.

For example, with a condition like inflammatory bowel disease (IBD), a drug may be approved to treat the large population of adults with the condition but then the same drug may be granted an orphan designation to treat a subset of children suffering from IBD.

"But once a drug receives an orphan designation for a pediatric population of the adult disease, the drug then becomes statutorily exempt from the requirements" of the Pediatric Research Equity Act (PREA), Gottlieb explains.

"It’s a loophole that is in direct opposition to what Congress intended," he wrote. "Nobody envisioned this unintended conflict between the original ODA [Orphan Drug Act] and the provisions outlined in PREA. In effect, by letting sponsors designate pediatric subpopulations of drugs intended to treat adult diseases, the drug makers receive an unintended ‘free pass’ from having to study drugs in these or other pediatric uses. Thus, rather than ensuring more pediatric research, as Congress envisioned, we can end up with fewer pediatric studies. FDA will soon issue a draft guidance document that’s aimed at closing this inadvertent loophole."

Nancy Goodman of Kids v Cancer, whose RACE for Children's Act was included in the FDA user fee reauthorization law (FDARA), explained to Focus that the language on closing the loophole was initially going to be included in FDARA. 

A public meeting announced by Gottlieb will also address some of the statute from FDARA and get input on complex scientific and regulatory issues, such as those raised by molecularly targeted drugs and biologics and the appropriate application of orphan incentives.

In addition to closing that loophole and the meeting, Gottlieb said FDA has now reviewed all orphan drug designation requests older than 120 days and will put in place new policies to improve the efficiency of FDA’s review process to ensure that we meet our new 90-day mandate to prevent new backlogs.


Source: raps.org

Georgia Tech-led group gets $20M federal grant to fund biomedical engineering research center
Wednesday, September 13, 2017 12:00 AM

A Georgia Tech-led research consortium received a nearly $20 million National Science Foundation grant to fund a new engineering research center.

The NSF Engineering Research Center for Cell Manufacturing Technologies (CMaT) will develop tools and technologies to help clinical facilities reproducibly manufacture efficient, safe and affordable cell-therapy products, Georgia Tech said in a statement.

The Georgia Tech-led CMaT lab 

Examples of these highly promising therapies include T cell-based immunotherapies for blood cancers and a gene-modified stem cell therapy recently approved in Europe for a form of the so-called “bubble boy” syndrome.

Unlike pharmaceuticals and other products now used in medical treatments, cells are living entities whose properties can change depending on the way they are grown, stored or manipulated, CMaT Director Krishnendu Roy said in the statement.

“The center will develop new engineering tools and scalable methods to better characterize, expand, differentiate, separate, transport and store high-quality cells so they provide consistent therapeutic effects, allowing them to be used in standardized therapies by clinicians to serve large numbers of patients worldwide,” Roy said.

CMaT will develop models for a supply chain, storage and distribution system for these therapeutic cell products. The public-private initiative will also help develop a skilled bio-manufacturing workforce through education and training activities at the K-12, technical college, undergraduate, graduate and postdoctoral levels.

The Georgia Tech led partnership includes the University of Georgia, the University of Wisconsin-Madison and the University of Puerto Rico, Mayaguez Campus. Affiliate partners include University of Pennsylvania, Emory University, the Gladstone Institutes and Michigan Technological University.

CMaT is expected to speed up the development of new therapies and the testing needed to bring them into the clinic, said Steven Stice, director of the University of Georgia’s Regenerative Bioscience Center.

Regenerative medicine applications could offer new ways of treating diseases for which there are now essentially no treatments, including Parkinson's, Alzheimer’s, heart disease and stroke.

“There are a significant number of cell therapy clinical trials and investments in the field,” Stice noted. “But there is little or no investment in a set of consistent standardization methods to optimize how these therapies should work."

Spaser Can Detect, Kill Circulating Tumor Cells to Prevent Cancer Metastases, Study Finds
Wednesday, August 30, 2017 12:00 AM

ATLANTA—A nanolaser known as the spaser can serve as a super-bright, water-soluble, biocompatible probe capable of finding metastasized cancer cells in the blood stream and then killing these cells, according to a new research study.

The study found the spaser can be used as an optical probe and when released into the body (possibly through an injection or drinking a solution), it can find and go after circulating tumor cells (CTCs), stick to them and destroy these cells by breaking them apart to prevent cancer metastases. The spaser absorbs laser light, heats up, causes shock waves in the cell and destroys the cell membrane. The findings are published in the journal Nature Communications.

The spaser, which stands for surface plasmon amplification by stimulated emission of radiation, is a nanoparticle, about 20 nanometers in size or hundreds times smaller than human cells. It has folic acid attached to its surface, which allows selective molecular targeting of cancer cells. The folate receptor is commonly overexpressed on the surface of most human cancer cells and is weakly expressed in normal cells.

The discovery was made by researchers at Georgia State University, the University of Arkansas for Medical Sciences, the University of Arkansas at Little Rock and the Siberian Branch of the Russian Academy of Science.

“There is no other method to reliably detect and destroy CTCs,” said Dr. Mark Stockman, director of the Center for Nano-Optics and professor of physics at Georgia State. “This is the first. This biocompatible spaser can go after these cells and destroy them without killing or damaging healthy cells. Any other chemistry would damage and likely kill healthy cells. Our findings could play a pivotal role in providing a better, life-saving treatment option for cancer patients.”

Metastatic cancer occurs when cancer spreads to distant parts of the body, often to the bone, liver, lungs and brain, through a process called metastasis. Many types of cancers refer to this as stage IV cancer. Once cancer spreads, it can be difficult to control, and most metastatic cancer can’t be cured with current treatments, according to the National Institute of Health’s National Cancer Institute. One of the most dangerous ways metastasizing occurs is through the CTCs, which this study aims to detect and destroy using spasers.

The spasers used in this study measure just 22 nanometers, setting the record for the smallest nanolasers. A nanometer is one-billionth of a meter. Most results were obtained with a gold, spherical nanoparticle surrounded by a silica shell and covered with a uranine dye, which is widely used for tracing and biomedical diagnostics.

The researchers studied the spaser’s capabilities in vitro in human breast cancer cells with high folate receptor expression and endothelial cells with low folate receptor expression, as well as in mouse cells in vivo.

They found cells with spasers demonstrated high image contrasts with one or many individual “hot spots” at different laser energies above the spasing threshold. The presence of spasers was confirmed with several optical and electron microscopy techniques, which revealed an initial accumulation of individual spasers on the cell membrane followed by their entrance into the cell cytoplasm.

The study also found low toxicity of the spasers for human cells. At the same time, the spasers subjected to laser irradiation selectively killed the tumor cells without damaging the healthy ones.

Based on the study’s results, spaser-based therapeutic applications with high-contrast imaging is a promising field. The data suggest spasers have high potential as therapeutic and diagnostic agents that integrate optical diagnosis and photothermal-based cell killing, using just a few laser pulses to kill cancer cells.

The study is funded by the National Institutes of Health, the National Science Foundation, the University of Arkansas for Medical Sciences and the U.S. Office of Naval Research.

To read the paper, visit https://www.nature.com/articles/ncomms15528.

Augusta University researcher gets $12.8 million grant to lead consortium on diabetic complications
Thursday, August 24, 2017 12:00 AM

Augusta University researcher Dr. Richard A. McIndoe received a $12.8 million grant from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), a division of the National Institutes of Health.

The funds will allow McIndoe, a bioinformatics expert and associate director of the Center for Biotechnology and Genomic Medicine at Augusta University,to continue leading a national research initiative that aims to reduce diabetic complications.

Augusta University researcher Dr. Richard A. McIndoe received a $12.8 million grant from… more

This is the fourth time McIndoe has been chosen to lead the Diabetic Complications Consortium, an NIKKD program.

The consortium is focused on advancing science and patient care, including funding studies in animals and humans to better understand and prevent complication.

It also supports summer programs for medical students.

According to Atlanta-based Centers for Disease Control and Prevention, nearly 10 percent of the U.S. population has diabetes. Complications from the disease are the leading reason to diabetes-related deaths.

Amgen and Humana Partner for Improved Health Outcomes and Efficiency
Tuesday, August 22, 2017 12:00 AM

Partnership to Target Multiple Serious Diseases Using Real World Data Studies

THOUSAND OAKS, Calif. and LOUISVILLE, Ky. (Aug. 17, 2017) -- Two of the nation’s leading health organizations, health and well-being company Humana Inc. (NYSE: HUM) and biotechnology company Amgen (NASDAQ:AMGN), have teamed up to identify opportunities to improve health outcomes and improve efficiency by unlocking new insights from the real world health care experiences of Humana’s 13 million members. 

In the collaboration agreement between Amgen and Humana, six projects are currently underway or planned, with more expected over the term of the agreement. Both organizations are committed to expanding the work into further therapeutic areas where the partnership can bring value to both Amgen and Humana. The goal is the same across all projects: deliver increased value to patients and the health care system by focusing on working to improve quality and outcomes in the context of total health care costs.

The collaboration will initially target multiple serious conditions, including  cardiovascular disease, osteoporosis, neurologic disorders, inflammatory diseases and cancer. Researchers will combine available sources of real world evidence (RWE) with data from wearable technology, digital apps and Bluetooth-enabled drug delivery devices. Prospective observational studies are also planned.

Planned research will identify patients whose serious medical conditions are likely to result in an adverse patient outcome, which may lead to development of algorithms that can predict risk and drive to early intervention. The partnership will also dive deeply into specific therapeutic areas, from defining the burden of osteoporatic fractures to understanding the impact of wearable technology on medication adherence for inflammatory diseases. 

“The rising cost of disease is challenging the sustainability of our health care system in the U.S., and is motivating innovators to urgently develop new therapeutic options and partner on opportunities to improve the quality and efficiency of care and reduce financial burden to the system,” said Joshua Ofman, M.D., M.S.H.S., senior vice president of Global Value, Access & Policy at Amgen. “It is our hope that this collaboration with Humana, a first of its kind for Amgen, will cultivate value-based, integrated approaches to care that will focus on patients and benefit the healthcare system more broadly.” 

“Humana is focused on the holistic health of our members and by teaming up with Amgen we can study new ways to improve the health outcomes for our members,” said Laura Happe, Pharm.D. M.P.H., chief pharmacy officer for Humana. “At the same time, we hope this research results in new tools and technology that support our provider partners who are on the journey to population health and value-based care.”

Amgen and Humana have proven experience in value-based initiatives. Globally, Amgen has engaged in more than 75 distinct value-based programs that have focused on improving clinical outcomes, patient experience and population health. 

As of June 30, 2017, Humana has 1.8 million individual Medicare Advantage members and 142,000 commercial members who are cared for by 50,700 primary care providers, in more than 900 value-based relationships across 43 states and Puerto Rico. By focusing on quality and health, Humana experienced 20 percent lower costs in total in 2015 for members who were treated by providers in a value-based reimbursement model setting versus an estimation of original fee-for-service Medicare costs using CMS Limited Data Set Files.

About Amgen 

Amgen is committed to unlocking the potential of biology for patients suffering from serious illnesses by discovering, developing, manufacturing, and delivering innovative human therapeutics. This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology.

Amgen focuses on areas of high unmet medical need and leverages its expertise to strive for solutions that improve health outcomes and dramatically improve people's lives. A biotechnology pioneer since 1980, Amgen has grown to be one of the world's leading independent biotechnology companies, has reached millions of patients around the world and is developing a pipeline of medicines with breakaway potential.

For more information, visit www.amgen.com and follow us on www.twitter.com/amgen.

Amgen and Value-Based Programs

As a collaborative partner within the healthcare system, Amgen believes that engaging in value-based programs with stakeholders creates mutually beneficial opportunities to improve costs, quality of care and the patient experience 

Globally, Amgen is engaged in over 75 distinct, value-based projects, spanning disease state collaborations, risk sharing, cost-cap guarantee, pay-for-performance and outcomes-based agreements. 

About Humana

Humana Inc. is committed to helping our millions of medical and specialty members achieve their best health. Our successful history in care delivery and health plan administration is helping us create a new kind of integrated care with the power to improve health and well-being and lower costs. Our efforts are leading to a better quality of life for people with Medicare, families, individuals, military service personnel, and communities at large.

To accomplish that, we support physicians and other health care professionals as they work to deliver the right care in the right place for their patients, our members. Our range of clinical capabilities, resources and tools – such as in-home care, behavioral health, pharmacy services, data analytics and wellness solutions – combine to produce a simplified experience that makes health care easier to navigate and more effective.

More information regarding Humana is available to investors via the Investor Relations page of the company’s web site at www.humana.com, including copies of:


•               Annual reports to stockholders

•               Securities and Exchange Commission filings

•               Most recent investor conference presentations

•               Quarterly earnings news releases and conference calls

•               Calendar of events

•               Corporate Governance information





Amgen, Thousand Oaks
Kristen Neese, 805-313-8267 (media)
Arvind Sood, 805-447-1060 (investors)



Marina Renneke, 602-760-1758, [email protected]

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