Building on Policy Momentum

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The rare disease community has seen several policy wins in the past few months, some of which may have gone unnoticed. First, in November, Congress passed the National Pediatric Research Network Act as part of the PREEMIE Reauthorization Act. President Obama signed the measure into law on November 27th. This legislation will strengthen collaborations among pediatric researchers across the United States by creating a network and database housed within the National Institutes of Health (NIH) for rare pediatric diseases. This bill solidifies the NIH’s research into pediatric diseases by authorizing 20 National Pediatric Research Consortia across the country. These consortia, while centered in institutions across the United States, will seek to forge inter-institutional bonds. The rare disease community should celebrate the enactment of this initiative as it strengthens research into rare pediatric diseases.

In early December, the Centers for Medicaid and Medicare Services revised their benefit policies to reflect a recent settlement agreement in the case Jimmo v. Sebelius. Previous to this case, CMS upheld an “improvement standard” when determining Medicare coverage within skilled nursing facilities, or skilled care in other settings. This meant that the patient must have a reasonable chance of “improving” from his or her current state in order to qualify. After a group of chronic disease patients successfully proved this as discriminatory in front of a U.S. District Court, CMS revised its standards to permit Medicare to cover skilled nursing care if a patient would deteriorate without the coverage, which qualifies many chronic disease patients for coverage. Thus, the chronic disease community, which includes many people affected by rare diseases, should celebrate this change in Medicare standards.

Finally, on January 16th, Congress passed an omnibus spending bill to fund the government for FY 2014. This bill was quite favorable for the FDA, as appropriations were raised to $2.552 billion for FY 2014 compared to $2.335 in FY 2013. Congress also reinstated $85 million in user fees the FDA lost in 2013 while avoiding the sequestration of user fees in 2014. Overall, this is the strongest level of appropriations the FDA has ever seen, reaffirming Congress’s belief in the importance of the FDA’s mission. This is also a big win for the Alliance for a Stronger FDA, which has long advocated for increased appropriations for the FDA. NORD is a founding member of the Alliance and our VP of Public Policy, Diane Dorman, served as president of the Alliance in 2013.

The rare disease community should celebrate the policy successes of the past few months, especially as we move closer to Rare Disease Day on February 28th. Let’s take this policy momentum into Rare Disease Day and build on the progress we’ve made!