Annie Kennedy's Blog (85)

ACTION ALERT: Tell Your Senator to Vote NO on Today's Healthcare Repeal!

Today, the Senate is expected to start floor debate and motion for a vote on either the Better Care Reconciliation Act (BCRA), which would repeal and immediately replace the Affordable Care Act (ACA), or the Obamacare Repeal Reconciliation Act (ORRA), which would repeal portions of the ACA after a two-year delay. The Senate is working to pass whichever bill is more likely to attain 50 votes. 

 

The Congressional Budget Office released reports last week that…

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Added by Annie Kennedy on July 25, 2017 at 9:05am — No Comments

FY 18 Labor HHS Report Language Released: Our Duchenne Community is Stronger Than Ever!

Transitions are hard. That’s the truth whether you’re talking about a new school year, a new job, or even a new Congress. And, in particular, new Presidential Administrations are always complex political climates in Washington.

 

But this year, our Duchenne community came together once again and embraced the challenge of this latest transition. With so many new Members of Congress and Federal Agency leadership in flux, we knew that our 2017 February…

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Added by Annie Kennedy on July 19, 2017 at 12:00pm — No Comments

FDA Makes Determination Regarding In-Dwelling Ports

Time and again in our Duchenne community, we see brave kids and their warrior parents work to change the landscape. After all, it’s that exact spirit upon which PPMD was founded. And with that same spirit that family after family has transformed their personal moments into broader movements that have benefited us all.

 

On May 18, the FDA Pediatric Advisory Committee convened to determine whether to allow for the protocol of Sarepta’s ESSENCE trial to…

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Added by Annie Kennedy on May 26, 2017 at 2:45pm — No Comments

FDA Pediatric Advisory Committee Unanimously Votes YES — Now Moves to Commissioner

Today’s Pediatric Advisory Committee Meeting resulted in a unanimous decision from committee members to recommend the use of in-dwelling ports in Sarepta's ESSENCE clinical trial. This is an important step forward for our community,…

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Added by Annie Kennedy on May 18, 2017 at 4:30pm — No Comments

Today’s FDA Pediatric Advisory Committee Meeting

As our clinical trial pipeline unfolds and therapies become available, navigating the landscape is becoming increasingly complex. But today our landscape shone a bit brighter as the sun rose on the FDA campus early this morning and we watched members of our Duchenne community arrive.



Because today – our Duchenne…

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Added by Annie Kennedy on May 18, 2017 at 10:00am — No Comments

PPMD Publishes & Testifies on Newborn Screening

PPMD's SVP of Legislation & Public Policy, Annie Kennedy and Michelle Puryear, MD, PhD, testified at the Advisory Committee on Heritable Disorders in Newborns and Children last week.



On the heels of a recent publication in the International Journal of Neonatal Screening, Dr. Puryear provided an update of the therapeutic pipeline and some of PPMD's activities around developing infrastructure to support a pilot for newborn screening for…

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Added by Annie Kennedy on May 16, 2017 at 4:40pm — No Comments

PPMD Signs on to FDA User Fee Letter

PPMD is proud to join dozens of other rare disease nonprofit organizations in asking Congressional leaders to reauthorize the FDA user fee agreements.

The current FDA user fee agreements are the culmination of months of negotiation between FDA and the medical product industry, with significant input from the patient advocacy community. 

Read the…

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Added by Annie Kennedy on May 16, 2017 at 4:00pm — No Comments

PPMD Helps Author Expanded Access Principles

Earlier this month, PPMD was proud to be among seven patient advocacy organizations who took a lead in laying out a joint set of principles to guide any efforts that seek to change the process of accessing unapproved therapies outside of a clinical trial, also known as compassionate use or expanded access.

The patient advocacy organizations include:

  • Alliance for Aging Research
  • American Cancer Society Cancer Action Network
  • Friends of…
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Added by Annie Kennedy on May 16, 2017 at 4:00pm — No Comments

Senate Introduces BENEFIT Act

PPMD is thrilled to announce that on May 4 the BENEFIT Act was introduced in the Senate by longtime Duchenne community champions, Sen. Roger Wicker (R-MS) and Sen. Amy Klobuchar (D-MN).

 

Over the past five years, Congress has made considerable progress in driving forward policies and procedures to ensure the patient perspective is considered by Food and Drug Administration (FDA) reviewers evaluating candidate drugs and other medical products. As a result of…

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Added by Annie Kennedy on May 16, 2017 at 4:00pm — No Comments

PPMD Response to the House Passage of the American Health Care Act

We may have lost the battle — but the war is far from over.

Some days are hard to make sense of. Today feels like one of those for many as we watched the House votes tally this afternoon.

This community knows better than most that our healthcare system needs repair and innovation. But the proposals that have been brought forward have not represented enhancements to healthcare…

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Added by Annie Kennedy on May 4, 2017 at 6:00pm — No Comments

ACTION ALERT: ­Immediate Action Needed – Revised AHCA Bill Threatens Duchenne Community’s Access to Health Care Coverage

The current version of American Health Care Act (AHCA) directly threatens essential health benefits (EHBs), Medicaid eligibility and funding, and vital health care protections for our Duchenne community. 

 

I wish there was a way to sugar coat this. Find the silver lining. See the glass as…

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Added by Annie Kennedy on April 28, 2017 at 9:30am — No Comments

Members of Congress Re-Introduce ABLE Improvement Bills

Great news – The ABLE program enhancements have been re-introduced in Congress and are gaining support. This package of bills include improvements to the original ABLE program that directly impact our community, here’s how:



On April 4th, a bi-partisan group of Members of Congress, including Senators Richard Burr (R-NC), Bob Casey (D-PA), Jerry…

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Added by Annie Kennedy on April 14, 2017 at 9:00pm — No Comments

PPMD Joins Letter to Protect Medicaid

In late March, PPMD joined 87 organizations to issue a letter calling on Congress to take a hard look at the likely significant and life-threatening consequences of the American Health Care Act on millions of…

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Added by Annie Kennedy on April 13, 2017 at 9:30pm — No Comments

PPMD Signs Appropriation Letter to Fund Newborn Screening

PPMD is proud to sign on to letters sent both to the Senate and the House asking that they provide at least $29.8 million to the Centers for Disease Control and Prevention’s (CDC) Newborn Screening Quality Assurance Program (NSQAP) and at least $19.9 million to the Health Resources and Services Administration’s (HRSA) Heritable Disorders…

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Added by Annie Kennedy on April 12, 2017 at 9:00pm — No Comments

PPMD's Duchenne Drug Development Roundtable Meeting Series, Part 3: Clinical Trial Readiness from the Viewpoint of Clinicians and Infrastructure

Accelerating trials. Protecting our robust therapeutic pipeline. Ensure as many hits on goal as we can.



Priorities shared by all of us.

And to achieve this, PPMD has convened our industry partners through our Duchenne Drug Development Roundtable and held a series of small, targeted meetings this winter, each focused…

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Added by Annie Kennedy on April 5, 2017 at 11:00am — No Comments

PPMD Working with FDA to Plan Landmark Pediatric Advisory Committee Meeting to Improve Clinical Trial Experience

Later this spring – likely on or about May 18 – FDA’s Pediatric Advisory Committee will convene to consider the issue of allowing in-dwelling ports in Sarepta’s Essence trial.

Once again, our community is at a pivotal moment in our history where we can affect change and improve the clinical trial experience for our…

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Added by Annie Kennedy on April 5, 2017 at 10:00am — No Comments

At Long Last, PDUFA VI Hearings Have Begun – But There’s a Twist

Wow...what a week in Washington! President Trump released his budget proposal, the American Health Care Act moves to the floor of the House for a vote today, the U.S. Supreme Court ruled on a landmark case around the rights of students with disabilities within the public school system, and at long last after months of our Duchenne…

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Added by Annie Kennedy on March 23, 2017 at 10:30am — No Comments

ACTION ALERT: Make Your Voice Heard on New Healthcare Proposal

“Who’s on first?”

Remember that classic Abbott & Costello routine? Well, trying to track the quickly-shifting healthcare bill has felt much like watching that routine. Complex, confusing, and exhausting. But the stakes here are no laughing matter.

 

We recognize that each member of our community views these…

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Added by Annie Kennedy on March 21, 2017 at 10:00am — No Comments

PPMD Joins Rare Disease Advocates in Letter to President

PPMD joined 200 patient organizations in a letter to President Trump about the challenges facing rare disease patients in America and the vital role that federal agencies play in helping to accelerate the…

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Added by Annie Kennedy on February 27, 2017 at 7:30pm — No Comments

Meet the 2017 PPMD Adult Advisory Committee (PAAC)

Years ago, a friend of mine named Tom Bailey who had Duchenne wrote a memoir that he entitled, Moving Mountains Without Muscles. As I look at the tremendous accomplishments of PPMD’s Adult Advisory Committee since we first launched this leadership group 2 years ago, I am astounded by how much passion, expertise, and effort each member has dedicated to…

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Added by Annie Kennedy on January 16, 2017 at 7:30pm — No Comments

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