NORD e-News: July 2011 Edition Send a Message to Congress on NORD Website Federal budget cuts have been proposed to Medicare reimbursement for drugs and biologics as part of a compromise to raise the debt ceiling. One proposal under consideration is to move orphan products from Medicare Part B coverage to Part D. This could greatly increase the cost for some patients and families dependent on lifesaving orphan drugs and biologics. You can quickly and easily submit a message to President Obama and all members of Congress to let them know you oppose cuts to Medicare reimbursement that reclassify orphan drugs from Part B to Part D. It takes just a few minutes. Read NORD’s basic letter, personalize it with your message, and submit it on the NORD website. Register Now for U.S. Rare Diseases/Orphan Products Conference Registration is now open for the 1st Annual Rare Diseases/Orphan Products Conference to be held Oct. 11-13 in Washington DC. Sponsored by NORD and DIA, this conference is also supported by FDA, NIH, EURORDIS, and Duke University. It will feature three tracks—one for patient organizations, one for researchers, and one for industry—and it will include the well-received Rare Disease Investigator Training Course offered by FDA for the first time last year. Join all stakeholders—patients, researchers, companies, investors, and government—in this summit focused on providing a common understanding of emerging challenges and strategies for the future. Timothy Coté Joins NORD as Chief Medical Officer Timothy R. Coté, MD, MPH, formerly Director of the FDA Office of Orphan Products Development, has joined NORD as Chief Medical Officer. His role will be to provide medical guidance in all areas—and particularly advocacy and education—on behalf of patients and families. Dr. Coté will divide his time between NORD and the Keck Graduate Institute in Claremont, California. More. SSA Expands Compassionate Allowances List In a July 14 event on Capitol Hill, Social Security Commissioner Michael Astrue announced the addition of 12 conditions to the Compassionate Allowances list. This is a list of medical diagnoses that qualify patients for expedited review when they apply for disability assistance. The July 14 event celebrated the fact that there are now 100 medical diagnoses on the list. Read NORD press release. Policy News Supreme Court Rules Vermont Law is Unconstitutional The U.S. Supreme Court has overturned a Vermont law that sought to limit the practice of selling information about physicians’ prescribing habits for marketing purposes. Pharmacists collect information when filling prescriptions that may ultimately be sold to pharmaceutical companies. In 2007, the state of Vermont enacted a law banning this practice. However, NORD and Genetic Alliance had filed an amicus brief contending that the use of such data was important in helping companies recover their investment in new drugs. Woodcock Presents Testimony on PDUFA V, Innovation, and Transparency Janet Woodcock, MD, Director of the Center for Drug Evaluation and Research at FDA, presented testimony before the House Energy and Commerce Subcommittee on Health on July 7. She told the subcommittee that it is critical that the reauthorization of PDUFA occur seamlessly, without any gap between the old law and the new one. Read her testimony, which also dealt with benefit-risk analysis, innovation, and challenges posed by globalization. PCORI Seeks Public Input on Patient-Centered Outcomes The Patient-Centered Outcomes Research Institute is seeking public responses to five questions to clarify its working definition of patient-centered outcomes research. PCORI was created by Congress through the 2010 Patient Protection and Affordable Care Act as an independent, non-profit research organization to help patients and those who care for them make informed health decisions. The questions are posted on the PCORI website. Responses must be submitted by Sept. 2. NORD Public Policy VP Diane Dorman presented a statement to the PCORI Board of Governors at a July 18 meeting. Read the statement. NIH News Advocacy Groups Urge Congress to Fund NCATS NORD and many of its members signed on to a letter being circulated last week by the Parkinson’s Action Network in support of funding in the NIH FY 2012 budget for NCATS, the National Center for Advancing Translational Sciences proposed by NIH Director Francis Collins, MD, PhD. The PAN letter was to be submitted to House and Senate appropriations subcommittees. As Dr. Collins has described it, the role of NCATS would be to create a focused, integrated, and systematic approach to link basic science research with therapeutics development and clinical care. More. Undiagnosed Diseases Program (UDP) Temporarily Suspends Applications The NIH UDP has announced a temporary suspension in accepting new applications as of July 1 in order to thoroughly review the large volume of applications already received. More. NIH Physician Named Researcher of the Year by the Physicians Professional Advisory Committee (PPAC) The National Institute of Environmental Health Sciences physician-scientist and clinical researcher Capt. Lisa Rider, M.D., was named 2011 Physician Researcher of the Year by PPAC of the United States Public Health Service (USPHS). Rider’s award is one of three presented to senior USPHS medical officers each year honoring clinicians, researchers, and applied public health executives. More. FDA News Register Now for Drug & Device Workshops Registration deadlines are approaching for two FDA Office of Orphan Products Development “Do A Designation” workshops at the University of Minnesota in early August. The first-ever Humanitarian Use Device (HUD) workshop will take place August 1-2. More. On August 2-4, OOPD will host a similar workshop on orphan drug designation. More. Center for Drug Evaluation and Research (CDER) Report This new report discusses the current regulatory science needs that will guide the CDER’s strategic planning. It is available for public comment for 60 days. Read the press release. Recent Orphan Drug Designations CytRx Corp has received orphan drug designation for INNO-206 to treat soft tissue sarcomas. 4SC has received orphan drug designation for its oncology drug resminostat (4SC-201) as a treatment for hepatocellular cancer. News from NORD Member Organizations Acid Maltase Deficiency Association (AMDA) The AMDA is announcing a 2011 research grant opportunity. If you have a proposal for a research project on Pompe disease, please visit their website and submit the proposal by Sept. 2. More. The AMDA and the International Pompe Association (IPA) are hosting the 2011 Patient/Scientific Conference in San Antonio, Texas on October 7-9. More. American Brain Tumor Association (ABTA) The ABTA will hold its annual Patient-Family Connections Meeting & Retreat in Lincolnshire, Illinois July 29-30. Diane Dorman of NORD will speak on healthcare public policy and advocacy. This year’s event also includes a Support Group Facilitator Training for social workers, nurses and other healthcare professionals. Victoria Reggie Kennedy, the widow of U.S. Senator Edward M. Kennedy, who passed away from a brain tumor in 2009, will headline the Annual Meeting Dinner. More. American Partnership for Eosinophilic Disorders (APFED) Hope Faculty Grants APFED is seeking Letters of Intent for research proposals leading to improved diagnosis, management and treatment of rare eosinophil associated diseases. Letters must be received by September 30. Selected investigators will be invited to submit proposals. One grant of $150,000 spanning 3 years will be awarded. Contact Kathleen Sable at [email protected]. More. HOPE Pilot Grants APFED is seeking applications from investigators from a variety of disciplines to initiate new projects relevant to eosinophilic disorders. Successful applications will focus on the development of new ideas likely to lead to future external funding. Deadline: Nov, 1. One-time grant(s) of $50,000 will be awarded. Contact Kathleen Sable at [email protected] . More. Amyloidosis Support Groups The 2011 Familial Amylodosis Conference will be held October 28-30 in Itasca, IL. Confirmed speakers include Merrill Benson, MD, Martha Skinner, MD, John Berk, MD, Morie Gertz, MD, Steven Zeldenrust, MD, and Peter Dyck, MD. Contact Muriel Finkel for more details at [email protected] , 847-350-7540, or 866-404-7539 (toll-free). GBS/CIDP Foundation International The Foundation is seeking candidates for a new executive director. Board President Phil Kinnicutt has shared a letter about this executive search. More. National Ataxia Foundation Pioneer SCA Translational Research Award The Pioneer SCA Translational Research Award is intended for a senior investigator for a one year grant of $100,000 focusing on research investigations to facilitate the development of treatments for the spinocerebellar ataxias. Letters of intent are due August 15. More. Young Investigator awards for SCA Research The Young Investigator awards for SCA Research are intended to encourage young investigators to pursue careers in spinocerebellar ataxia research. Letters of intent are due August 1. More. National Fragile-X Foundation (NFXF) In celebration of National Fragile-X Awareness Month, the NFXF is enlisting help to spread awareness that those affected by Fragile X are more than their diagnosis. Please visit the Awareness Day 2011 web page to see inspirational stories from Fragile X families and then share your own! Help show the many ways that those with Fragile X are all unique, self-determining individuals. Tourette Syndrome Association (TSA) The TSA is requesting research grant proposals from PhD and MD researchers in basic and clinical studies on all aspects of Tourette syndrome. Pre-proposals are due by September 1. More. Patient Recruitment Anyone considering participating in a clinical trial should discuss the matter with his or her physician. NORD does not endorse or recommend any particular studies. Chronic Pain Management Clinical Trials Pediatric Patients If you are a parent with a child experiencing chronic pain who would like to know more about pediatric chronic pain clinical studies being conducted, or a healthcare professional aware of pediatric patients requiring analgesia who might be appropriate for and interested in participating in clinical studies, please contact Purdue Pharma L.P.’s Medical Services Department: Email: [email protected] Phone: (Toll-Free) 1-888-726-7535; Option#1 Business hours: Monday-Friday 8am-5pm EST (USA) Lupus Nephritis If you have been diagnosed with lupus nephritis and are experiencing symptoms despite your current therapy, you may be interested in learning more about this study, currently seeking eligible patients, to evaluate the effectiveness and safety of an investigational medication for the treatment of active lupus nephritis. You may be eligible to participate if you are between 18 and 70 years of age and have persistently active lupus nephritis. To learn more about the study, view a list of site locations, and see if you pre-qualify, visit clinicaltrials.gov. Upcoming Meetings and Webcasts Community-Engaged Research: Exploring the Unique Community-Academic Relationship This Office for Human Research Protections (OHRP) National Research Community Forum will be held September 26 in St. Louis, MO.  More information. Websites and Blogs of Interest International Clinical Trials Portal The National Cancer Institute (NCI) recently launched the International Clinical Trials Portal on Cancer.gov. This section of the NCI website provides online resources that will help investigators outside the United States navigate the legal and regulatory issues that come with collaborating on clinical trials with U.S.-based research groups. Visit the site.

NORD and the NORD Logo are registered trademarks of National Organization for Rare Disorders, Inc. NORD – 55 Kenosia Ave – PO Box 1968 – Danbury, CT 06813-1968. – ph 1.800.999.NORD