Cooley’s Anemia Advocacy Forum Update: July

July 7, 2015The Cooley’s Anemia Advocacy Forum (CAAF) is a Cooley’s Anemia Foundation project aimed at helping thalassemia patients and families learn how to make their voices heard on federal issues of importance to the thalassemia community. (A brochure about CAAF can be downloaded by clicking here.) Following is a report from Lyle Dennis and Katie Schubert.

This month we’re looking at the successful advocacy efforts of the Foundation, and giving you an update on the 21st Century Cures Initiative. Before Congress goes on a break for August, there is much work to be done!

Patient-Family Conference Success Mirrored in Advocacy

This year’s Patient-Family conference, entitled “Celebrating Self-Empowerment,” offered participants a unique opportunity to meet other members of our community and learn important information that could have a positive impact on their health and their lives.

And by all accounts, self-empowerment has been reflected in the progress that the thalassemia community has made in both the scientific and the policy spheres.

First, with regard to blood safety, CAF has taken a firm and aggressive position that any changes in blood donation policy must be accompanied by a vast improvement in the quality of the blood monitoring system. That system is currently in development by the Food and Drug Administration and the National Heart, Lung and Blood Institute at NIH. CAF’s position is simply that no changes should be made in blood donation policy until and unless there is an improved safety system in place.

Second, with regard to insurance, the Affordable Care Act has now been fully implemented for 18 months. With the recent Supreme Court decision (King v. Burwell), it is unlikely that there will be any successful challenges to the law as the two most promising opportunities to overturn it have failed. The CAF is continuing to work hard to assure that all phases of the law’s implementation-and any future legislative or regulatory changes that are considered-will take into consideration the needs of the rare disease community.

Finally, there is some good news with regard to funding. Both the House and Senate Appropriations Committees are recommending increases in NIH funding: $1.1 billion and $2.0 billion, respectively. This level of investment should help make more research grants available. For the Centers for Disease Control and Prevention (CDC), the overall budget is relatively flat. However, we are delighted to see that the line item for “thalassemia,” which is the blood safety surveillance program, is funded at about $2.0 million in both appropriations bills.

Moving forward, CAF’s plan is to stick to its guns on the blood safety issue; work to maintain maximum coverage for patients and families dealing with rare, chronic disorders; and, to continue to fight for resources for patients and researchers.

Update: 21st Century Cures Legislation Faces Hurdles

Sponsors of a bipartisan package to speed new medical cures are working to put the 21st Century Cures legislation, which we reported on in the last newsletter, back on a fast track.

Energy and Commerce Chairman Fred Upton (R-MI) had hoped to have the measure on the House floor in June after the package was unanimously advanced by his panel in May. But efforts have stalled amid protests over a provision that would have paid for $5 billion to $7 billion of the measure’s cost by adjusting the timing of Medicare payments to Part D prescription drug sponsors so that the government keeps interest revenue instead of insurers.

A Congressional Budget Office (CBO) estimate has also raised concerns among biomedical research advocates that the bill’s wording would not make NIH and FDA funding mandatory, i.e. not separate from the annual appropriations process, as lawmakers intended.

“This proposed budgetary change would result in increased Part D costs that would ultimately be passed on to the Medicare Trust Fund and its beneficiaries,” a bipartisan group of more than 40 lawmakers wrote in a letter to House leaders last week. “This change is effectively a tax on America’s seniors.”

After weeks of negotiations, advocates say that bill sponsors signaled that they would remove the offset and are now close to finalizing revised language while keeping their alternatives close to the vest.

The latest hiccup for lawmakers is the bill’s cost. The CBO estimates that the bill would cost $106.4 billion over five years, a number higher than expected because it includes the entire NIH budget that would be reauthorized under the measure. The estimate also includes a separate $10 billion fund for NIH and a $550 million fund for FDA, implying that such money would still be subject to appropriations and thus require future action from Congress.


Secured By miniOrange