RDLA’s 3rd Annual Rare Disease Cocktail Reception & Movie Screening

Join RDLA for our 3rd Annual Rare Disease Cocktail Reception & Movie Screening to kick-off Rare Disease Day (week) in Washington DC

Monday, February 25, 2013
5:00pm Cocktail Reception * 6:30 Movie Screening * 8:00 Panel Discussion
Carnegie Institution of Science, 1530 P Street Northwest, Washington DC

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Feb. 6 Briefing: The Blueprint of Medical Research: How New Medicines Get from the Lab to the Patient

Improving and accelerating the R&D path requires all stakeholders in the medical research ecosystem to work together. Co-hosted by FasterCures and Friends of Cancer Research, this briefing is designed to educate members of Congress, legislative staff and advocates about the vital role federal agencies and the medical research sector play in advancing medical progress, and to emphasize the importance of prioritizing this work.

Wednesday, February 6, 2013, 12:00pm – 1:30pm
Dirksen Senate Office Building, Room 106, Washington, DC

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PhRMA Report: Innovation in the Biopharmaceutical Pipeline: A Multi-Dimensional View

For Immediate Release:  January 17, 2013

Today, the Pharmaceutical Research and Manufacturers of America (PhRMA) released a report entitled “Innovation in the Biopharmaceutical Pipeline: A Multi-Dimensional View.”  The report was conducted by researchers at the Analysis Group, and examines the drug pipeline to illustrate the type of research conducted by biopharmaceutical companies.

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Congressional bickering is deeply damaging to biomedical research

The inability of Congress to pass a budget is damaging the NIH, the core of biomedical research in the U.S, which has cut or delayed funding for most new top projects, biomedical researcher Steven Salzberg writes in Forbes. He says this has left scientists and researchers looking for work, possibly in the private sector, and he calls on members of Congress to set aside their fighting and just pass the NIH budget without cuts. 

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From FDA Matters: Orphan Drugs at 30: Will Success Become Too Expensive?

The Orphan Drug Act (ODA) turned 30 this month, demonstrating that good laws really can have an enduring impact.  Amidst the celebrations, a reporter asked me a provocative question: can we afford more orphan drugs costing hundreds of thousands of dollars per year? FDA Matters answered “yes.”

From The Alliance for a Stronger FDA: Points to Keep in Mind for Next 8 Weeks

The 8-week reprieve from sequestration has benefited all federal discretionary programs. “Live to fight another day” is not to be taken for granted.  Sequestration still, however, looms as an existential threat to the mission of the FDA. If it occurs — as presently scheduled, on March 1 — we estimate the agency will have to generate about $250 to $270 million in savings over 7 months.

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Orphan Drug Prices To Face Fiscal Cliff Scrutiny, NORD Expects

From The Pink Sheet:   If the debate does include a focus on entitlement spending, NORD does not plan on taking sides.

The fight to avert the next fiscal cliff may affect orphan drugs and their escalating prices, according to the National Organization for Rare Disorders.

Concerns among rare disease patients about increases in orphan drug costs have been percolating for a few years. But Peter Saltonstall, NORD president and CEO, said he expects the issue to gain even more attention as Capitol Hill continues to fight over spending and budgets.

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RareVoice Awards Gala, Washington DC

On November 14, 2012, RDLA hosted its inaugural RareVoice Awards Gala to honor and thank Members of Congress, Congressional Staff and Patient Advocates for their advocacy to ensure The Food and Drug Administration Safety and Innovation Act (FDASIA), S. 3187 included provisions that spur the development of lifesaving treatments for rare disease patients.  Abbey Meyers, Founder of the National Organization for Rare Disorders, Inc. (NORD) was honored with a lifetime achievement RareVoice Award for her work to pass the Orphan Drug Act. In her honor, the award has been named an “Abbey.”

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Rare Disease Congressional Caucus Briefing Nov. 2012

RDLANational Health Council, and Shire, in coordination with Rare Disease Caucus Co-Chairs Representatives Leonard Lance (R-NJ) and Joe Crowley (D-NY) hosted a briefing on Wednesday, November 14, 2012

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Medical Innovation Top Priority for Likely Voters in U.S.

We are very pleased to feature a guest blog post from Mary Woolley, President and CEO, Research!America, about support for medical innovation and why it is an important issue for the majority of likely voters during this year’s election.

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