Federal Research Public Access Act of 2010 (FRPAA)

FRPAA , HR5037, was recently introduced in the House of Representatives by Mike Doyle (D-PA) and a group of bipartisan cosponsors. This proposed legislation would require free online access to results from publicly-funded research within six months of publication in a peer-reviewed journal. This is similar to the Senate bill introduced in 2009 by Senators Lieberman (I-CT) and Cornyn (R-TX) and the open-access mandate at the NIH that requires posting of NIH-sponsored research in PubMed Central within 12 months of publication. Open access to scientific data will help researchers make progress in the development of disease treatments and diagnostics. FRPAA will now be sent to the House Committee on Oversight and Government Reform for discussion. More information.

Cures Acceleration Network (CAN) to be Established

The Health Care Reform Act includes a provision to create the CAN at the National Institutes of Health (NIH) to reduce the time it takes to move drugs and therapies from the research phase to the marketplace. Funding will be made available to accelerate the development of drugs, biological products or devices for the diagnosis, prevention or treatment of diseases that may have a small commercial market. More information.

Second FDA Orphan Designation Workshop Planned

FDA’s Office of Orphan Products Development held the first Orphan Drug Designation Workshop on February 25-26 at Keck Graduate Institute (KGI). This workshop, co-sponsored by FDA, NORD and the KGI Center for Rare Disease Therapies, was a success. NORD helped recruit 47 participants seeking orphan drug designation for various products. Based on their individual experiences, 90 percent said it was beneficial and 94 percent would recommend the workshop.

The next workshop will be held August 3-4 at the University of Minnesota. Participants are encouraged to bring specific product proposals for at least one candidate orphan drug that holds promise for the treatment of a rare disease. A significant portion of the workshop will be dedicated to preparing applications, including one-on-one guidance sessions with FDA staff members. FDA will keep product and disease information confidential. Final applications can be submitted to the FDA at the close of each workshop.

Families USA Launches Health Reform Central

Health Reform Central is a new website designed to assist health advocates in understanding the comprehensive health reform law and to help states prepare for implementation. Visit the site.

NORD and EURORDIS Launch Advocacy Blog

NORD and the European Rare Disease Organisation, EURORDIS, recently launched an international advocacy blog at www.rarediseaseblogs.net. Invited bloggers include experts from the U.S. and Europe on topics related to rare disease research and the development of orphan products. Recent posts include one from NORD President Peter L. Saltonstall on the recent expansion of the Social Security Compassionate Allowances program and one from EURORDIS CEO Yann LeCam entitled "Rare Diseases at the Forefront to Address the Healthcare Challenges of the Future." Everyone with an interest in rare diseases and orphan products is invited to visit the blog often and post comments.

NORD Applauds NIH and FDA Joint Initiative

NORD President and CEO Peter L. Saltonstall today praised the announcement by the Food and Drug Administration (FDA) and National Institutes of Health (NIH) of a new collaborative effort to accelerate medical innovation and more effectively evaluate new products. "We applaud the action of FDA and NIH in seeking to work together more closely to bridge the gap between promising early research and the development of marketable therapies," Saltonstall said. "We're very excited and encouraged by today's announcement, and we stand ready to help in any way we can." Specifically, the new joint initiative will involve collaboration in two areas---translationship and regulatory science. It will include the establishment of a Joint Leadership Council to spearhead collaborative work on important public health issues. FDA and NIH also will jointly make available $6.75 million over the next three years for regulatory science research.

Database of Rare Disease Experts

As part of the Rare Disease Day observance, NORD has launched a project to create a database of physicians with expertise on various rare diseases. To nominate a physician (either a clinician or researcher or both), provide basic information on this online form. NORD will contact the physician to determine whether he/she is interested in being included in the database and to gather additional information.

NORD Applauds NIH and FDA Joint Initiative

NORD President and CEO Peter L. Saltonstall today praised the announcement by the Food and Drug Administration (FDA) and National Institutes of Health (NIH) of a new collaborative effort to accelerate medical innovation and more effectively evaluate new products. "We applaud the action of FDA and NIH in seeking to work together more closely to bridge the gap between promising early research and the development of marketable therapies," Saltonstall said. "We're very excited and encouraged by today's announcement, and we stand ready to help in any way we can." Specifically, the new joint initiative will involve collaboration in two areas---translationship and regulatory science. It will include the establishment of a Joint Leadership Council to spearhead collaborative work on important public health issues. FDA and NIH also will jointly make available $6.75 million over the next three years for regulatory science research.

FDA Funding for 2011

President Obama's budget request for FY 2011 was released on February 1. He has proposed an inflationary increase of about 6 percent for FDA, in a year in which most domestic discretionary programs will be frozen or cut.

NORD is a founding member of the Alliance for a Stronger FDA, which has taken the position that a 6% increase is insufficient. The Alliance cites three reasons:

• The growing imbalance between FDA’s funding and its expanding portfolio of public health responsibilities.
• Three good appropriations cycles have not fully undone a decade of budgetary neglect.
• Further strengthening of FDA is vital to our nation’s public health, economy and national security.

The Alliance, which is composed of patient groups, consumer advocates, biomedical research advocates, health professionals and industry, will be working with the Congress and the Administration to get FDA a larger increase. The Alliance’s position is that despite the enormous federal budget deficit, FDA must be a public priority in order to ensure that consumers have access to safe and effective therapies, foods, cosmetics, and medical products.

Social Security Expands Compassionate Allowances List

The Social Security Administration announced on February 11, 2010, that it is adding 38 additional medical conditions to the “Compassionate Allowances” program. This program provides expedited review of applications for disability assistance from people with severely disabling diseases. Previously, there were 50 conditions on the Compassionate Allowances list.

NORD President Peter L. Saltonstall was to have appeared at a press conference with Social Security Commissioner Michael Astrue to announce the additions to the list. The press conference was canceled because of snowstorms in Washington DC, but in a press release Saltonstall said “NORD applauds SSA for creating and now expanding the Compassionate Allowances program.” Astrue announced early in his tenure at SSA that he wanted to address problems related to processing of applications from people with very disabling rare diseases. SSA plans to continue to expand the Compassionate Allowances program in the future.

Research Survey for Individuals with Rare Disorders

Medical students at Michigan State University, College of Human Medicine are conducting a research project designed to measure barriers and improvements to diagnosis and treatment in people with rare disorders

http://www.surveymonkey.com/s/LGBDNNT