NORD News for November - The Voice of the Community

Guest post by NORD

News from NORD and its Member Organizations

NORD and Coalition Support Advancing Targeted Therapies for Rare Diseases Act  

More than 100 patient organizations signed a letter submitted by NORD to the Senate HELP Committee on Nov. 12th in support of the Advancing Targeted Therapies for Rare Diseases Act. The letter states that the legislation “could greatly accelerate the pace of therapeutic development for rare diseases” by streamlining the regulatory review process without compromising safety and efficacy standards.

Specifically, the legislation clarifies the authority of the Food and Drug Administration (FDA) to leverage data previously used in the approval of a targeted drug when approving a new therapy that incorporates or utilizes the same or similar genetically targeted technology or the same variant protein targeted technology.

The position of NORD and its advocacy partners on this issue is that this authority is critically important to accelerate the development of treatments for rare diseases or subsets of rare diseases that otherwise are unlikely to be developed as a result of small patient populations. Only the sponsor generating the original data may leverage the data for future uses so intellectual property and patent protections would not be affected.

NORD and Partners Enthusiastically Endorse FDA Commissioner Nomination

The American Academy of Pediatrics (AAP), FasterCures, American Society of Clinical Oncology (ASCO), and Friends of Cancer Research are among the organizations signing onto a letter submitted by NORD to the Senate HELP Committee in support of President Obama’s nomination of Dr. Robert Califf as FDA Commissioner.

“The United States is at a pivotal moment in terms of public health, and the leadership that Dr. Califf will bring to the FDA will be paramount to assuring the safety, effectiveness, and security of human drugs, and helping to speed innovations that make medicines more effective, safer, and more affordable,” the letter states.

NORD Releases First-ever State Progress Report

With patient access to health care becoming increasingly dependent upon state policies, NORD has developed and released the first-ever State Progress Report to evaluate how states are serving the 30 million Americans with rare diseases. In this initial release, the State Progress Report focuses on four key areas: medical foods coverage, prescription cost-sharing requirements, newborn screening, and Medicaid eligibility levels.

“By creating the State Progress Report, NORD hopes to provide a road map of where states can improve to help people living with rare diseases,” said NORD President and CEO Peter L. Saltonstall. “Until now, there has been no comprehensive resource to help make sense of the inconsistencies between states.”

New Physician Guide Focuses on Rare Lung Disease

NORD has published a Physician Guide to Nontuberculous Mycobacterial (NTM) Lung Disease to promote earlier diagnosis and optimal treatment of this rare disorder. The new guide, written by Leah Lande, MD, of Lankenau Medical Center and Jefferson University Medical College, is free and available on NORD’s Physician Guide website.

Resources For Medical Professionals From NORD Member Organizations

NORD members provide expert-reviewed information and resources for medical professionals. In addition, many provide funding for study of rare diseases. Here is a brief sampling of news and resources from NORD members:

CurePSP and Tau Consortium Forge Landmark Collaboration

CurePSP and the Tau Consortium have brought together a team of prominent investigators who will launch the definitive study of the gene variants involved in progressive supranuclear palsy (PSP). This deadly brain disorder afflicts about 20,000 people in the U.S., and is a leading target for researchers seeking to understand all neurodegenerative diseases, including Alzheimer’s and Parkinson’s diseases.

International PC Consortium Welcomes All Working on Pachyonychia Congenita and Related Disorders

The IPCC welcomes all scientists and physicians interested in collaborative efforts to develop and deliver effective treatments for PC and related conditions. Sponsored by PC Project, the registry now has more than 150 participants. All are welcome to join in collaborative research efforts, genetic discoveries, and patient care. The 13th annual IPCC Symposium will take place May 10-11, just prior to the Society of Investigative Dermatology annual meeting in Phoenix, AZ.

Applications Are Due Feb. 29th for Congenital Central Hypoventilation Syndrome (CCHS) Pilot Grant Awards

The CCHS Pilot Grant Awards represent a collaboration between the CCHS Family Network and CCHS Foundation to encourage and support basic, clinical, translational, or epidemiological research to impact the lives of patients with CCHS. CCHS is a multisystem disorder of the central nervous system in which the automatic control of breathing is absent or impaired.

Pancreatic Cancer Action Network Research Grants to Total $9.7 Million in 2016

The Pancreatic Cancer Action Network’s research investment for 2016 will total $9.7 million, up 40% over last year. These awards target applicants at all stages of their careers and support projects across the research continuum – basic, translational, and clinical. View current opportunities.

Tele-Ichythosis: An Online Resource Available to Dermatologists & Other Healthcare Professionals

The Foundation for Ichythosis & Related Skin Types provides a tele-dermatology service to help dermatologists and other healthcare professionals with the treatment and diagnosis of patients with ichthyosis and related skin types.  The service is made possible by the FIRST Medical and Scientific Advisory Board.  Questions may be submitted and responses provided in a secure, HIPAA-compliant environment to facilitate communication between dermatologists.

Osteogenesis Imperfecta Foundation Develops Toolbox for Medical Professionals

The Osteogenesis Imperfecta Foundation has developed an online Information Center for Medical Professionals with the assistance of its medical advisers. The kit includes videos, fact sheets, and other resources to assist with diagnosis and treatment of OI patients. Resources are designed for physicians with new OI patients as well as those with current OI patients who have new issues.   
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