Legislative Call to Action

Take Action and Join the Committee on Federal Legislation:

The legislative committee tracks, reviews, and supports or opposes legislation important to the MPS and related diseases community.  Additionally, the committee reviews governmental agency policy and guidelines that may impact families and individuals with MPS and strategizes on how to make a positive impact. Annually, the committee travels to D.C. to advocate with legislators, representatives, health legislative aides, NIH, and other various governmental decision makers important to accelerating our progress to treatments and cures and protecting the rights individuals with disabilities.

The legislation committee consists of volunteer parents, patients, and staff which convenes by monthly conference calls, attends relevant patient advocacy meetings in local areas, and educates key decision makers about MPS and related disease.  To join the Legislative Committee and/or learn more, contact Stephanie Bozarth.

Questions?  contact Stephanie Bozarth, MPS Board of Director and Chair of Committee on Federal Legislation at stephanie.bozarth@mpssociety.org

We Need You to Take a Stand and Let Your Voice Be Heard!


Call to Action:

National MPS Society advocates for Congressional Response to FDA Draft Guidance

Call for Action: The FDA recently issued a draft guidance on the Expedited Programs for Serious Conditions, to fulfill FDASIA mandate on Accelerated Approval, however the guidance does not address issues specific for rare diseases, like MPS, as required by the 2012 FDA Safety and Innovation Act.  The MPS Society signed on a letter to the FDA stating the guidance does not meet the congressional intent outlined in FDASIA.  In addition, the Society sent out several call for actions to all members to urge their Representatives to sign on to the FDA draft guidance.  On September 20, 2013 the goal of over 100 representative signatures was achieved. To read more, Click here.  To read letter, click here.

Ask your Congressman to Join the Rare Disease Caucus

Help us strengthen the rare disease community’s voice on Capitol Hill!  Please take 3 minutes to ask your Member of Congress to join the Rare Disease Caucus at bit.ly/Rare-Caucus.

It’s easy – the Action Center has a draft letter.
Read More.

How to Attend a Congressional Town Hall

Taking part in a public or town hall meeting is a great way to build a relationship with your Member of Congress and his or her staff and to raise the profile of the rare disease community to policy-makers.

Patient Impact of Regulatory Challenges and Roadblocks to Participation in Rare Disease Early Phase Clinical Trials


National MPS Society signs on to Support Legislative Efforts: (updated 9/22/2013)

* The Kids First Research Act of 2013 (H.R. 2019). This bipartisan bill would eliminate taxpayer financing of presidential campaigns and party conventions and reprogram those savings to provide for a 10-year pediatric research initiative through the Common Fund administered by the National Institutes of Health.

* Compassionate Allowances within SSA-A NORD letter to Acting SSA Commissioner Carolyn W. Colvin, as well as the U.S. Senators and Representatives with jurisdiction over SSA, urging continuation of this important program and continued expansion of the list of diseases.
Read a draft of the letter with names of those who have signed on to date.
Read about the Compassionate Allowances Program and view the list of medical conditions approved to date.

* Orphan Drug Tax Credit (ODTC) is part of a package of provisions enacted in 1983 in the Orphan Drug Act that provide incentives for drug companies to develop products for rare diseases.  Tax reform is planned for this Congress and the ODTC is on the table for elimination.  Drug companies need more incentives to develop rare disease treatments, not less.  Read the letter sent by NORD in support of ODRC. Congress must hear from the patient community that this incentive is important.  Read More.

* Undiagnosed Diseases Research & Collaboration Network (HR 1591)The “Network” will help reduce the length of time and number of physicians it currently takes to diagnose patients with a rare disease by creating a professional physician collaboration network and providing a means to collect and analyze information on undiagnosed cases. Read More.

* 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

* Rare Disease White Pages on Accelerated Approval

* Life Span Respite Appropriations Letter

* Coalition Letter:  Health Information Technology Framework Proper oversight of health IT is critical to ensuring patient safety, however such oversight must be implemented in a balanced way that also fosters innovation and encourages adoption of these technologies.  A coalition is encouraging the Administration to ensure health IT is regulated in a way that achieves the goals of improving the quality, safety and cost-effectiveness of care.
Read the letter & see the long list of supporters here:  Coalition Sign-on Letter Regarding Policy Associated with Health IT.

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