Former FDA Commissioner Discusses Government's Role In Drug Discovery.

Medscape (10/16) publishes the transcript from a Medscape One-on-One session between Sanford-Burnham Medical Research Institute CEO Dr. John C. Reed and Samaritan Health Initiatives President Andrew C. von Eschenbach, MD, who formerly held positions as FDA commissioner and NCI director. At the discussion, which took place at NIH's Celebration of Science Conference at the National Institutes of Health, Dr. von Eschenbach noted that "academic centers and researchers" are working to discover the "underlying mechanisms" of the diseases; and the "government has to play a critical role in catalyzing and fostering that collaboration. ... We can bring the parts and pieces together as a team to use the brilliance of the science that you, Dr. Reed, have been doing, and others here at NIH and in academic institutions all around the world have been doing, and recognize that science is the means. The end is that we solve people's problems, and we do it together," von Eschenbach emphasized.

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US Conference on Rare Diseases and Orphan Products - Special Challenges in Rare Diseases

October 22-24, 2012
Washington, DC

Special Challenges in Rare Diseases

• Learn how the government and private sector are addressing the special challenges faced by patients and companies in the new health care environment
• Meet individuals who face the same challenges that you do
• Gain a better understanding of how the FDA and NIH work and how they interact with the drug and device development processes
• Learn ways to de-risk your investments in orphan products and understand better the timelines for research and regulatory reviews

Featured Speakers Include
Keynote:

• John J. Castellani: President & CEO Pharmaceutical Research and Manufacturers of America (PhRMA)

The US Conference on Rare Diseases and Orphan Products is Going Mobile! Download the Mobile App Today. The US Conference on Rare Diseases and Orphan Products app is FREE and available for all registered attendees on Android, iPhone, iPad, and Mobile Web version. Download the app to access a wide range of Rare Diseases and Orphan Products information as well as the ability to:      • Create and Manage your Agenda and Appointments      • Receive News and Announcements      • Network with Fellow Attendees      • Receive Event Information in Real-time      • View a List of Exhibiting Companies Download from the Android Market | Download from the Apple Store | Download Mobile Web Version To download, go to your mobile device’s app store and search for DIA/NORD RARE DISEASES. Table Top Exhibits View Exhibiting Companies Eager to Meet with You. Maximize Your Learning in Washington, DC Register for the post-conference workshop,Clinical Trial Endpoints: Methods and Practice in Developing Measurements which has a special emphasis on rare diseases - and save $200 off your registration.* Contact Customer Service if you would like to attend this one-day workshop. Clinical Trial Endpoints: Methods and Practice in Developing Measurements October 25 | 8:30 AM – 5:00 PM *Discounts taken from your Clinical Trial Endpoints: Methods and Practice in Developing Measurements registration fee. Follow #DIARAREDISEASES on Twitter for real-time updates.

Meeting Program

 

US Conference on Rare Diseases and Ophan Products - October 22-24

Gain a Common Understanding of the Current and Emerging State of Rare Diseases and Orphan Products. Join patients, patient organizations, drug and device companies, researchers, government professionals, and investors to discuss ways to bringing rare disease and orphan product research, development and access into the spotlight. Sessions will be organized around three central themes: policy, research and regulation, and special challenges. These sessions are intended to promote advancements in science, patient care and other considerations that will address the needs of the 30 million Americans suffering from a rare disease. Last year’s event brought together more than 300 stakeholders, and this year’s event promises even more.  FEATURED TOPICS: Policies affecting rare diseases in this evolutionary time Legislation that, by the time of the meeting, Congress is likely to have enacted, including special provisions applicable to orphan products How to be a more effective advocate How to work with federal agencies on grants and orphan designations Ways to de-risk your investments in orphan products and better understand better the timelines for research and regulatory reviews How the FDA and NIH work and interact with the drug and device development processes How to develop efficient clinical rare disease programs and avoid common pitfalls How the government and private sectors are addressing the special challenges faced by patients and companies in the new health care environment And more... Register 3 from your company and the 4th is FREE! TABLETOP EXHIBIT OPPORTUNITIES:  Showcase your company's products or services. Submit your application today to Shannon.Lewis@diahome.org for details.   Program Overview  |    Register Patient organizations and patients are invited to attend the US Conference on Rare Diseases and Orphan Products at a reduced registration fee of $400. Contact Carrie.Dunn@diahome.org for details. Email Preferences    |    My DIA    |    Forward to a Friend    |

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FDA Voice Interviews Jesse Goodman, M.D., M.P.H., on the DARPA and NIH Project Collaboration: Human on a Chip

FDA Voice: FDA has embarked on an exciting collaboration with the Defense Advanced Research Projects Agency (DARPA) and NIH—to develop a groundbreaking tool that could help bring new treatments to patients faster, more cheaply, and more safely. Can you talk about this new technology?
Dr. Goodman: Yes, it’s what we’re calling Human on a Chip. This is an ambitious project to create a tool that could revolutionize toxicology testing and it’s something I’m really excited to talk about. Scientists have relied largely on animal studies to determine if a drug is toxic before testing it in humans. And while animal testing is useful, it’s also expensive, time consuming, and has drawbacks. For example, it doesn’t always detect toxic effects specific to humans and doesn’t usually provide information about the role that genetic differences within human populations play in toxicity. It can also generate false alarms, showing an effect in animals that doesn’t predict an actual effect in people, which leads us to abandon promising new drugs. FDA is collaborating with DARPA, NIH, and the scientific community to spur innovation in this field by exploring how tools like Human on a Chip can be integrated into our development tool box to improve testing for toxicity and potentially reduce the need for animal testing.

FDA Voice: Can you describe Human on a Chip?

Dr. Goodman: Researchers are developing microsystems using human cells to test the effects of drugs or other substances. For example, scientists have developed a micro machine chip with human lung cells that grow on a surface to form a lung-like tissue that has both air spaces and blood circulation. FDA is supporting the coupling of this chip to a heart-like chip that beats and pumps blood. We can use this type of system to evaluate, with human cells, how specialized organs like the lung and heart react to a specific chemical.

more of the interview here... http://blogs.fda.gov/fdavoice/index.php/2012/04/fda-voice-interviews-jesse-goodman-m-d-m-p-h-on-the-darpa-and-nih-project-collaboration-human-on-a-chip/

NIH’s 1,000 Genomes Project gains wider access, from amednews.com

Researchers and physicians now will have access to the largest-known database of genetic variations thanks to a partnership announced in March between Amazon Web Services and the National Institutes of Health.

Through the partnership, the 200-terabyte 1000 Genomes Project database, equivalent to more than 30,000 standard DVDs, will be stored on Amazon’s cloud and accessible to anyone with an Internet connection and a computer capable of processing that amount of data. Tools to process the data also are available on Amazon for a fee that varies according to the data access and analyses needed. Amazon created a website explaining the computing requirements or tools that can be used to access the database (aws.amazon.com/1000genomes/).
The 1,000 Genomes Project was launched in 2008 to create the most comprehensive map of human genetic variation available anywhere in the world. The goal is to collect data from the genomes of more than 2,600 people from 26 populations around the world and find the majority of all genome variations in existence. The database now has the genome sequencing of 1,700 people.
Whole genome sequencing allows researchers to identify genetic variations that increase a person’s risk of developing any one of a variety of conditions or diseases.
“The explosion of biomedical data has already significantly advanced our understanding of health and disease. Now we want to find new and better ways to make the most of these data to speed discovery, innovation and improvements in the nation’s health and economy,” said Francis S. Collins, MD, PhD, NIH director.
Lisa Brooks, PhD, program director of the Genetics Variation Program at the NIH, said most research labs do not have access to such a large data set or the computing power to work with one. The NIH is investing this money to advance disease study that otherwise would be difficult to carry out, she said.
The 1,000 Genomes Project database is geared toward researchers as opposed to the practicing physician. But if a physician already deals with genetic testing, he or she could use the database to determine whether a patient’s genetic variation may have led to them developing a particular disease or condition, Brooks said. A physician’s office computer would not likely be able to process the data, but a doctor could get access through a teaching hospital that does research.

more... http://www.ama-assn.org/amednews/2012/04/16/bise0418.htm

NIH grabs share of Obama's $200M big data bet,

http://www.fiercebiotechit.com/story/nih-grabs-share-obamas-new-200m-bet-big-data/2012-04-02?utm_medium=nl&utm_source=internal

By Ryan McBride

Big data has won a $200 million endorsement from the White House, and the National Institutes of Health (NIH) stands to gain a sizable share of funding. The NIH revealed March 29 that it is one of 6 federal agencies in line to reap benefits from the initiative to find solutions for taming huge datasets.
The NIH and National Science Foundation have teamed up to award up to $25 million from the initiative for 15 to 20 projects in science and engineering fields. In the biomedical arena, for instance, the NIH wants to fund projects that could enable the crunching of massive amounts of data to aid scientific investigations. Also, the agency announced that digital data from the international 1000 Genomes Project will be hosted in the cloud by Amazon Web Services and be available for free.
President Obama's big data bet follows criticism from the scientific community that, despite the billions of dollars invested in genomic research and molecular biology studies, a relative pittance has gone into supporting the gigantic datasets that have resulted from those efforts. For example, cheap and fast DNA sequencing has motivated federally funded labs to explore sequencing, yet few of them have the internal computing power to manage and analyze the massive genomic datasets.

How massive? The 1000 Genomes Project eats up 200 terabytes of storage, equal to some 16 million filing cabinets stuffed with text or 30,000 DVDs, according to the NIH.

"Improving access to data from this important project will accelerate the ability of researchers to understand human genetic variation and its contribution to health and disease," stated Dr. Eric Green, director of the NIH's National Human Genome Research Institute, which is one of the backers of the 1000 Genomes Project.

- here's the NIH's release
- check out InformationWeek's article

Amazon, NIH put 1000 Genomes Project in the cloud

I highly recommend getting their newsletter.-W

March 29, 2012 — 2:57pm ET | By Gienna Shaw

It took 10 years and billions of dollars to sequence and publish the first human genome. Now,

anyone with an Internet connection can, in theory, access 200 terabytes of genomic data,

including DNA sequenced from more than 1,700 individuals.

Amazon and the U.S. National Institutes of Health (NIH) announced today that the complete 1000 Genomes Project will be available on Amazon Web Services as a public data set. The announcement, made at the White House Big Data Summit, will make the largest collection of human genetics available free of charge, according to a TechCrunch article.

Read more: Amazon, NIH put 1000 Genomes Project in the cloud - FierceHealthIT http://www.fiercehealthit.com/story/amazon-nih-put-1000-genomes-project-cloud/2012-03-29#ixzz1qbaL2qAv Subscribe: http://www.fiercehealthit.com/signup?sourceform=Viral-Tynt-FierceHealthIT-FierceHealthIT