Children’s Rare Disease Network Partners With Medpedia.com To Create Rarespace

Online Knowledge Share Platform to Provide Valuable Information to Rare Disease Community

Dana Point, Calif. (June 29, 2010) – Every parent needs a supportive, collaborative network full of information where medical professionals, researchers, patients, parents, advocates and the general public share knowledge about the rare childhood diseases that affect 22.5 million American families. RareSpace is an online knowledge sharing platform designed in partnership with R.A.R.E. Project, the Children’s Rare Disease Network and Medpedia.com to help grow resources for children with special needs. This resource is available online now at RareSpace.

“RareSpace is a unique and valuable tool for families caring for children with rare diseases,” says Jonathan Jacoby, CEO of the R.A.R.E Project. “With the help of Medpedia.com, RareSpace will become a safe place to share important information aggregated from the rare disease community at large, which is vital to finding hope for children.”

It’s the collaborative structure of RareSpace that makes it truly unique. The site will educate and connect users about critical resources, innovations in research, standards of care as well as best practices in treating rare diseases and disorders. Creating a community filled with medical professionals, policy advocates, researchers and parents catalyzes a free exchange of information, increasing the general knowledge quotient of every stakeholder. The goal is to supply real resources and connections for those who are in the care of children living with rare diseases and disorders, and help each family not feel isolated in their struggles to care, treat and advocate for these sick children.

“It’s so critical for families and caregivers to have a knowledge bank like RareSpace to draw from,” says Devon Watts, community manager at Medpedia.com. “We are excited that Medpedia.com is a part of connecting a community and granting an open space for dialogue and education. It’s the widespread sharing of information that will benefit patients most.”

Different from other community sites, one key advantage of RareSpace is that people can share documents and resources very easily. Discussions of genetics and genetic diseases in general happen on RareSpace, with the understanding that research on all these diseases could lead to cures for other genetic disorders as well. Articles on translational research and discussions of possible cures for diseases can be found on RareSpace. A major benefit is that medical professionals will answer questions posed by site users about treatment, best practices and how to best help these children and their families. Users will create profiles and have the opportunity to upload documents and links and connect with other users all within the RareSpace portal. In addition to connecting within RareSpace, users will have the ability to create disease specific groups that can all be linked with RareSpace. This robust platform provides numerous ways to share critical information and knowledge across the rare disease community, which can be done in a safe environment that includes representation from all of the key stakeholders involved in rare disease.

R.A.R.E. Project’s, Jonathan Jacoby to present at FDA Hearing on Rare Disease

On Tuesday, June 29, 2010, and Wednesday, June 30, 2010, FDA’s Office of Orphan Products Development will host a two-day public hearing and Webcast on the Development of Articles for Rare Diseases.

This public hearing is intended to gain from health care providers, academia, industry, patients, and other interested persons their perspectives on various aspects of the development of medical products for the diagnosis, treatment, or management of rare diseases. The input from this public hearing will help inform the work of FDA’s committee for rare diseases

Jonathan Jacoby, CEO R.A.R.E Project will present case studies on therapeutic candidate generation, focusing on;

• Challenges of patient-driven R&D
• Challenges and successes with biomarker identification
• Experience with Orphan Drug Designations
• Individual investigator INDs and IND exemptions
• NIH clinical trials
• Barriers to recruitment and participation in CTs

Following the presentation, Jacoby will provide recommendations that include the importance of very small CTs as a pathway to regulatory approval. Jacoby will present this testimony from 11:40 – 12:00, June 29, 2010.

The meeting will be webcasted and any interested persons are encouraged to join:

• Dates: June 29-30, 2010
• Time: 9:00 AM to 5:00 PM ET
• Webcast addresses (will be active at the time of webcast):
  June 29 – collaboration.fda.gov
  June 30 – collaboration.fda.gov

FDA Database Aims to Spark Orphan-Disease Drug Development

By Amy Dockser Marcus

For months now, the Food and Drug Administration has been trying novel ways of encouraging drug makers to develop drugs for rare diseases.
Today the agency’s Office of Orphan Products Development is launching a new gambit—a database of FDA-approved compounds and products that show promise in rare diseases too.

The concept is called “repurposing” — finding new ways to use products that have already been okayed by the FDA for some other use.

The already-approved products in the Rare Disease Repurposing Database are unique in that they’ve also previously received orphan-drug designation, meaning they’ve shown potential to treat one or more of the diseases affecting 200,000 or fewer Americans. (While orphan-drug status doesn’t guarantee FDA approval as a treatment for the rare disease, it’s a prerequisite for getting incentives such as tax breaks and marketing exclusivity if the agency does okay the drug for that purpose.)

Timothy Coté, director of the orphan products development office, says that testing an already-approved drug as a treatment for a rare disease has significant advantages — for one, it’s already been found safe by the FDA. And running trials on an existing drug is much cheaper than trying to develop a totally new compound.

Despite the incentives in the Orphan Drug Act, there are only about 350 such drugs that have been approved — and there are some 7,000 rare diseases.

Information in the database is already publicly available through the FDA but until now has been scattered. Coté says officials went through more than 2,000 orphan drug designations to compile the new 235-drug database, which he says is likely to be used not only by drug companies but also by venture capitalists looking for promising investments and patient advocacy groups eager to drive drug development for their rare condition.

Coté says he hopes the new database will spark drug development efforts. But he plans to do some repurposing of his own. He says the next step after the database is released is for his office to carefully study the list, identify some particularly promising products, and urge the companies to move forward.

Link to article

Orphan Drugs Rare Disease Opportunity, Investors Eye

Investors are warming up to the economics of rare disease orphan drugs as potentially big moneymakers.  As much as we love to bash Wall Street, the financial sector has found gold in orphan drugs for treating rare disease, and that might just fill in a deficit of research capital, which would otherwise be unavailable where and when its needed for orphan disease and disorder treatment drugs.

From The Motley Fool | 5/25/10 :

Orphan drugs don’t have the potential patient numbers that cholesterol or diabetes drugs have, but that doesn’t mean you should ignore them either.  What they lack in volume they make up for in price.

Technically speaking, a drug can get orphan drug status if it treats a disease that affects less than 200,000 people in the U.S. A lot of cancers fall into that range — for instance, GlaxoSmithKline’s (NYSE: GSK) Tykerb has orphan drug status for gastric cancer. But the true orphan drugs treat diseases you’ve probably never have heard of.

A little protection goes a long way

There really are some financial incentives to gaining rare disease orphan drug treatment status. The government kicks back up to 50% of the cost of clinical trials in the form of tax credits. And the Food and Drug Administration comps the fee established by the Prescription Drug User Fee Act (PDUFA) that drugmakers pay when submitting their orphan disease marketing application.

But the biggest advantage comes from the exclusive sales of the rare disease orphan drug treatment for seven years in the U.S. and six to 10 years in the EU that governments give orphan drugs. It doesn’t matter whether patents expire during that time, the regulatory agencies will hold off approving generic competition until the rare disease orphan drug treatment exclusivity has expired.

The Motley Fool:

Being the first to treat a disease also offers an indirect lockup of patients from other branded drugs. If the drug works well enough, patients are less likely to enter a clinical trial for a competitor’s experimental drug, making it harder for the competitor to get on the market. Being first doesn’t provide an absolute monopoly — in addition to Genzyme’s (Nasdaq: GENZ) Cerezyme, Shire’s Vpriv is approved for Gaucher disease and Protalix and Pfizer (NYSE: PFE) are also working on a Gaucher disease treatment — but a little protection can go a long way.

On the road to bigger things

Sometimes the orphan disease drug status is just a starting point. Because the proteins that drugs interact with are often involved with other diseases, treating an orphan disease can be a good starting point before launching into another disease that may not be so rare.

READ THE COMPLETE ARTICLE AT MOTLEY FOOL

Orphan Drug Development for dummies! Well not really!

Keck Institute to host a ‘How To’ workshop on developing Orphan Drugs – this is one parents perspective and quest to build an effective treatment to help save her daughters lives.

http://addiandcassi.com/wordpress/the-road-to-building-a-new-orphan-drug-am-i-mad/

Make Rare Disease a Top USA Health Care Priority, Vote Today at Change.org – 25 Million+, It is time to care about rare disease.

For the millions and millions of kids and adults, brothers and sisters, moms and dads, families of every sort, friends, doctors, researchers, non-profits, foundations, research institutions, hospitals and advocacy groups,

for everyone touched by rare disease,

put on your blue jeans,

put on your blue jeans denim ribbon, and

vote for rare disease at Change.org – vote here ”25 Million+, It is time to care about rare disease.”

Ask family, friends, and strangers to vote.  The first round vote ends at 5 p.m. (ET) on Thursday, February 18, 2010.

Right now we are in seventh place for “health care” ideas, we need to be in the top three ideas to make it to the finals.

Our votes can put rare disease into the top ten real American priorities – I say it’s time, don’t you?  Start your World Rare Disease Day celebration today, vote for rare disease as a true health care priority.  And pass it on (and on and on).

Catherine Calhoun

Biotechs Adjust to the New Normal

Biotechs Adjust to the New Normal (.mp3, 16.34MB)

Chris Starr has lived through biotech windows, cycles, and bubbles. An industry veteran, today he is CEO and co-founder of Raptor Pharmaceuticals, which works on improving existing drugs through its targeting platform and formulation expertise. Like many executives, Starr has had to prioritize his pipeline and find ways to raise money during tough times. We talked to Starr about the changing environment for biotechs, how he’s adapted, and as times improve, whether or not there’s a new normal.

PATIENTS EMBRACE VIRTUAL BIOTECHS TO DEVELOP DRUGS FOR THEIR CONDITIONS

As a business model, the notion of the virtual biotech by some estimates has been around for as long as 25 years. But for people afflicted with a rare disease and their families, the notion of the virtual biotech is generating energy and enthusiasm around raising funding to create ad hoc teams to conduct specific drug development projects. We spoke to Jonathan Jacoby, CEO of the RARE Project, about what it takes to create a virtual biotech, what successes there have been to date, and how this reflects a broader change in the role of patients and their families in drug development.

http://www.crdnetwork.org/news/podcasts/virtualbiotechs.html

Nominate Your Rock Star of Science!

Geoffrey Beene Gives Back®’s hip new campaign, “Rock Stars of Science” (a.k.a Rock S.O.S) is making its mark, and The Children’s Rare Disease Network is proud to be affiliated with this important initiative!
Geoffrey Beene Gives Back® teamed up with GQ Magazine to create a philanthropic campaign pairing A-list rock stars and celebrities with the country’s leading Medical Researchers to:
• bridge the recognition gap that makes Britney Spears more influential than Stephen Hawking
• celebrate America’s most important Scientists
• heighten public awareness of the dire need for additional funding for scientific research
• gain recognition for the breadth of Geoffrey Beene causes
• collect signatures in support of a trip to Capitol Hill on September 24th to meet with House and Senate leadership to further these goals
We are asking you to do two things – Please sign the Rock S.O.S Declaration (hyperlink to: http://www.rockstarsofscience.org/declaration.asp), a simple way to show our community support and Nominate Your Rock Stars (hyperlink to: http://www.rockstarsofscience.org/nominate.asp) today! Let’s do it!!!