Obama Signs FDA User Fee Legislation Bringing Hope to Rare Disease Patients
FOR IMMEDIATE RELEASE
Obama Signs FDA User Fee Legislation Bringing Hope to Rare Disease Patients
EveryLife Foundation for Rare Diseases Applauds Congress for Including Provision to Empower the FDA to Accelerate Approval of Lifesaving TreatmentsJuly 10, 2012, Washington, DC – Yesterday President Obamasigned into law The Food and Drug Administration Safety and Innovation Act (FDASIA), S. 3187, bipartisan legislation that will spur the development of lifesaving treatments for 30 million Americans suffering from rare diseases.
“We are thrilled the language to improve access to the FDA’s Accelerated Approval pathway for rare diseases has been included in FDASIA,” said Emil Kakkis, MD, President, EveryLife Foundation for Rare Diseases. “We wish to thank Representatives Cliff Stearns (R-FL) and Ed Towns (D-NY) for being champions for the rare disease community.”
Stearns and Towns first introduced Unlocking Lifesaving Treatments for Rare Diseases Act (ULTRA) to empower FDA to use all the science available for allowing surrogate endpoints in clinical trials for rare diseases to determine whether a drug is working, significantly decreasing the development time and cost. Stearns and Towns later introduced Faster Access to Specialized Treatments (FAST) Act that improved Accelerated Approval for life-threatening diseases while maintaining high safety and efficacy standards.
FDA’s Accelerated Approval has been successful in getting treatments approved for cancer and AIDS patients, but has been essentially unavailable for rare disease treatments. There are currently fewer than 400 FDA-approved treatments for nearly 7000 rare diseases. Investment and interest in development will surge for rare diseases if there is access to the Accelerated Approval pathway.
“We would not have been successful if it were not for the great work of Energy and Commerce Chairman Fred Upton (R-MI), Biotechnology Industry Organization (BIO), and more than 300 patient organizations that advocated for improving the FDA’s regulatory process,” added Kakkis.
FDASIA is the culmination of more than a year of negotiations between industry and FDA and includes the reauthorization of the drug and device user fees.
The Foundation will host its fourth Rare Disease Workshop on “Developing Guidance and Policy Recommendations for Accelerated Approval in Rare Diseases” on November 15th in Washington, D.C. FDA, NIH, industry and academic scientists are invited to participate.
The EveryLife Foundation for Rare Diseases is dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy. We can do more with the science we already have and bring life saving treatments to millions of people suffering from rare diseases.——————————————————
Contact: Julia Jenkins, Director of Public & Government Relations
Email: jjenkins@EveryLifeFoundation.org Phone: 415-884-0223
www.EveryLifeFoundation.org
A framework for human microbiome
The Human Microbiome Project Consortium (HMPC) two interesting papers about the human microbiome.
First of all ithas established a population-scale framework to develop metagenomic protocols, resulting in a broad range of quality-controlled resources and data including standardized methods for creating, processing and interpreting distinct types of high-throughput metagenomic data available to the scientific community.(1)The constructed catalogue is
the largest and most comprehensive reference set of human microbiome data associated with healthy adult individuals. (1)The HMPC has also analized the micro-diversity in our bodies, producing the first map of our microbiota… but the story isn’to closed:
Many details remain for further work to fill in, building on this reference study. How do early colonization and lifelong change vary among body habitats? Do epidemiological patterns of transmission of beneficial or harmless microbes mirror patterns of transmission of pathogens? Which co-occurrences among microbes reflect shared response to the environment, as opposed to competitive or mutualistic interactions? How large a role does host immunity or genetics play in shaping patterns of diversity, and how do the patterns observed in this North American population compare to those around the world? Future studies building on the gene and organism catalogues established by the Human Microbiome Project, including increasingly detailed investigations of metatranscriptomes and metaproteomes, will help to unravel these open questions and allow us to more fully understand the links between the human microbiome, health and disease.(2)In the image (via Matthew Herper for Forbes): Principal coordinates plot showing variation among samples demonstrates that primary clustering is by body area, with the oral, gastrointestinal, skin and urogenital habitats separate; the nares habitat bridges oral and skin habitats.(2)
(1) The Human Microbiome Project Consortium (2012). A framework for human microbiome research Nature, 486 (7402), 215-221 DOI: 10.1038/nature11209
(2) The Human Microbiome Project Consortium (2012). Structure, function and diversity of the healthy human microbiome Nature, 486 (7402), 207-214 DOI: 10.1038/nature11234
Scientists are commencing the largest genetic mapping project for a single disease this week. They are investigating over 800 participants with Alzheimer’s disease to map the full genetic make-up of the individuals in an aim to determine if there are common genetic links between them and the disease. The researchers expect that the mapping project will point them in new directions of study in an effort to understand and cure the disease. Alzheimer’s disease affects over 26 million people worldwide and the prevalence is expected to double within a generation. For more information on Alzheimer’s disease or to learn more about your brain health, please contact me at www.mybraintoday.com
ATP, easy as 123.
ATP is the renewable energy source for our cells. It consists of 3 phosphates, a sugar and an adenine ring. The bond between the last 2 phosphate groups is a weak unstable bond. When broken, ATP is changed to ADP and a phosphate, releasing energy to be used in cellular reactions. ATP stores regenerate when energy from respiration reactions is used to reattach the phosphate a an ADP. ATP is soluble and hence easily transported. Every chemical reaction in every cell requires or produces ATP molecules. Most ATP is made in the mitochondria and diffuses into the cytoplasm and to membranes. ATP is used in movement, locomotion, structure, metabolism, synthesis, active transport, cellular signalling, DNA and RNA synthesis.
