Read About the Legislation Featured at Lobby Day

During Rare Disease Week 2014 in Washington D.C. over 160 rare disease advocates lobbied for 3 main pieces of legislation and asked their legislators to join the Rare Disease Legislative Caucus.

Read more

Action Alert for Organizations: Sign on to support the MODDERN Cures Act

U.S. Representative Leonard Lance (R-NJ) has introduced legislation that would “modernize our drug and diagnostics evaluation and regulatory network” – the MODDERN Cures Act, H.R. 3497.  The lead cosponsor is U.S. Representative Jay Inslee (D-WA).

Read more

FightSMA Announces Passage of National Pediatric Research Network Act

Bill authorizes the National Institutes of Health (NIH) to support the creation of up to 20 pediatric research consortia focused on diseases such as Spinal Muscular Atrophy.

Read more

HR 2725 – FDA SOS Act of 2013

The FDA Safety over Sequestration Act is bipartisan legislation that would exempt FDA user fees from sequestration.  The bill amends the Budget Control Act.  To sign onto the letter to support FDA SOS, contact Jenny Carey by Friday Dec. 6th.

Recent update:  At the Energy & Commerce Congressional Hearing on FDASIA, Dr. Janet Woodcock, FDA’s Director of the Center for Drug Evaluation & Research (CDER) explained how the rare diseases programs at the FDA have been hindered due to Sequestration.  The EveryLife Foundation is encouraging all rare disease patient organizations to support FDA-SOS.

Read more

The Burrill Report: Threatening Orphans

With everything on the table for tax reform, rare disease advocates and drug developers sound an alarm.

DANIEL S. LEVINE The Burrill Report 

“It’s the biggest issue facing the rare disease community right now.”

Sign-on to Protect the Orphan Drug Act

One of the key provisions from the Orphan Drug Act is at risk of begin eliminated!  The Orphan Drug Tax Credit (ODTC) is part of a package of incentives enacted in 1983 in the Orphan Drug Act that encourage drug companies to develop products for rare diseases.

The Senate’s plan is to start at zero, eliminating all tax credits, meaning the rare disease community must fight to get ODTC put back in the bill.  In the House, everything is on the table for elimination, including ODTC.

Drug companies need more incentives to develop rare disease treatments, not less.  Congress must hear from the patient community that the Orphan Drug Tax Credit is important to them.  To add your organization to letter to protect the Orphan Drug Act, please contact Lauren Neff:  Deadline to sign on is COB, July 24th.

Read more

Cantor Balancing Research Priorities, Fiscal Concerns – Kids First Research Act (HR 2019)

House Majority Leader Eric Cantor is calling for medical research to gain precedence over some other issues, as Congress tries to balance competing priorities with limited dollars.

Cantor said his interest in medical research partly comes from watching his father suffer for more than a decade from a rare neurological disorder known as multiple system atrophy or Shy-Drager syndrome.

By Melissa Attias, Roll Call Staff  June 3, 2013, 3:51 p.m.

Read more

Mindful of Previous Defeat, Cantor Pushes Bill to Increase Pediatric Research

CQ NEWS – June 7, 2013 – 3:24 p.m.  By Daniel Newhauser, CQ Roll Call

House Majority Leader Eric Cantor and his staff are working behind the scenes to avoid another embarrassment this month when they bring to the floor a bill that would increase funding for pediatric research.

The measure is part of the Virginia Republican’s agenda to soften the Republican image. But it is running up against the same hardline conservatives who forced leaders in April to pull from the House floor a bill (HR 1549) that would have reprioritized funding within President Barack Obama’s health care law (PL 111-148,PL 111-152).

Read more

Sign on to Support Newborn Screening Act

To sign on to support this legislation, email Melissa at

The Newborn Screening Save Lives Reauthorization Act (H.R. 1281) reauthorizes critical federal programs that provide assistance to states to improve and expand their newborn screening programs; support parent and provider education; and ensure laboratory quality and surveillance for newborn screening.

Read more

H.R. 2090, the Patient Choice Act of 2013

Congressman Morgan Griffith (R-VA), Congressman Scott Peters (D-CA), and Congressman Michael McCaul (R-TX) today introduced H.R. 2090, the Patient Choice Act of 2013, which aims to speed up the Food and Drug Administration’s (FDA) approval of drugs for patients. The Patient Choice Act would create a provisional approval process for innovative drugs and treatments while giving patients with terminal diseases the option to purchase these new therapies at their own expense.

Read more