Nominations for Fifth Annual RareVoice Awards Due by July 31st

Do you know anyone who has gone above and beyond to become a rare disease policy leader or advocate in a state or in our nation? Rare Disease Legislative Advocates (RDLA) is seeking nominations for the RareVoice Awards to be given on November 16th in Washington, DC.

Nomination categories include:

Congressional Staff

Has a staff member of a member of the U.S. House, Senate or Congressional committee been instrumental in helping to create and/or advance legislation that benefits the rare disease community?

Government Agency Staff

Do you know a government agency staff member who has gone out of his or her to listen to rare disease patients and then taken action to benefit the community?

Patient Advocate – Federal Level Advocacy

Do you know an advocate or organization that has worked with Congress or federal agencies to spearhead change on behalf of the rare disease community?

Patient Advocate – State Level Advocacy

Do you know an advocate or organization that has crusaded on the state level to support and advance rare disease legislation or regulatory reform?

RareVoice Award nominations are open to the public. Winners will be selected by an independent committee composed of representatives from the rare disease community including previous RareVoice winners.

To nominate a rare disease champion, please click HERE. Nominations close on July 31st, 2016.

The winners will be announced LIVE at the fifth annual RareVoice Awards ceremony on November 16that the Arena Stage in Washington, DC and will receive the coveted Abbey award.

The award is named after Abbey Meyers, the founder of the National Organization for Rare Disorders (NORD), and represents the “rare voice” speaking on behalf of patients, especially children, who might not otherwise be heard. The Abbey statue was commissioned for the RareVoice Awards from the renowned sculptor Nobe, who crafts each statue by hand in bronze.

Senate Holds Third and Final Markup of Biomedical Innovation Legislation

On April 6th, the Senate Health, Education, Labor and Pensions (HELP) Committee held the last of three markups to discuss biomedical innovation legislation.  The legislation considered and approved in those mark-ups will be conferenced with the 21st Century Cures Act which passed the House of Representatives with broad bipartisan support last summer. In addition to approving the five bills under consideration, members of the Committee offered several amendments of particular interest to the rare disease community. Read more

RECAP: White House Meeting On 21st Century Cures

From the House Energy and Commerce Committee:

WASHINGTON, DC – House Energy and Commerce Committee Chairman Fred Upton (R-MI), Ranking Member Frank Pallone, Jr. (D-NJ), and Rep. Diana DeGette (D-CO) issued the following statement following a meeting at the White House with Vice President Joe Biden on H.R. 6, the 21st Century Cures Act. Senate HELP Committee Chairman Lamar Alexander (R-TN) and Ranking Member Patty Murray (D-WA) also attended this afternoon’s meeting.

Upton, Pallone, and DeGette commented:

“Today’s meeting with Vice President Biden and Senate leaders was positive and productive. The #Path2Cures connects both the White House and U.S. Capitol, and we welcome the vice president’s enthusiastic participation in this bipartisan effort. As many patients and their families are too painfully aware, there’s not much time. But the good news is, we are more than half way there as we’ve already done the legwork in the House and the Senate is making headway on its innovation package.

“We are seeing a tremendous opportunity for Cures, with our legislative efforts coupled with the ‘moon shot’ and the administration’s Precision Medicine Initiative, there is no question that 21st Century Cures is the right vehicle to get this done. We will all continue working together – House, Senate, and White House – until we are successful in delivering #CuresNow for patients across America.”

From EveryLife Foundation: Take Action to Support Lifesaving Newborn Screening Legislation in California

The EveryLife Foundation is working to expand the lifesaving potential of newborn screening in California. Last month, SB 1095 was introduced by pediatrician and California State Senator Dr. Richard Pan. This bill would help eliminate the legislative delay in newborn screening and ultimately ensure that patients are treated as early as possible. Early screening plays a vital role in ensuring that rare diseases are detected and can be better treated. Read more

Action ALERT for Pennsylvania Advocates: Contact the Senate to Boost Rare Disease Treatments

Dear Advocate,

As you may know, the House passed the Orphan Products Extension Now, Accelerating Cures & Treatments or OPEN ACT in July as part of a broader legislative package called the 21st Century Cures Act. The OPEN ACT has the potential to double the number of treatments available to rare disease patients.

Since passing the House, the bill has stalled in the Senate. Your Senator, the Honorable Bob Casey (D-PA) is a key decision-maker on this vital legislation. Please take a moment to call his office to ask that they support the bipartisan OPEN ACT, co-sponsored by Sens. Hatch (R-UT) and Klobuchar (D-MN). You can find suggested talking points below: Read more

Senate chairman: Cures bill is next priority

From The Hill:

The Senate Health Committee will be turning its attention to a medical innovation bill now that it has completed the overhaul of No Child Left Behind, Chairman Lamar Alexander (R-Tenn.) said.

“No. 1, what we want to turn our attention to next is what we call our innovation bill,” Alexander said on C-SPAN’s “Newsmakers.”  The innovation bill is the Senate’s version of a measure that has already passed the House, called 21st Century Cures. The idea behind both bills is to speed up the Food and Drug Administration’s approval process for new drugs and boost funding for research at the National Institutes of Health (NIH).

The 21st Century Cures bill passed the House on a bipartisan vote in July, but since then the momentum has slowed.

Alexander had previously said that the committee would finish its work on the bill by the end of the year, but the panel has not released a bill, much less advanced it to the full Senate.

Still, Alexander noted the bipartisan support for the idea behind the measure and said the panel is moving forward.

One of the major points of contention has been deciding whether the funding increase for the NIH will be in the form of mandatory spending, meaning that it is guaranteed over a number of years and not subject to the annual appropriations process. Sen. Patty Murray (Wash.), the panel’s top Democrat, has made her support for the bill contingent on mandatory NIH funding.

Alexander reiterated on C-SPAN that he is willing to have mandatory funding but acknowledged that not all Republicans are. He also said he wants the NIH funds to be offset by cutting other mandatory spending, which could cause Democrats to balk.

“I’m willing to consider [mandatory funding] under some circumstances,” Alexander said, if it “replaces other mandatory funding.”

“Not all my Republican colleagues are, but I’m willing to do it,” he said. “And I’m willing to do it because this is such an exciting time in science. We’re coming up with so many things to help people, and we’ve got a person like Francis Collins, who’s a genius heading the National Institutes of Health, we ought to take advantage of that.

“It affects every American, so I’m willing to do something that I normally wouldn’t do, and that’s going to be one of the toughest parts of the innovation bill that we have to decide next year.”

Action Alert from the EveryLife Foundation: Ask Your Senators to Co-Sponsor the OPEN ACT to Repurpose Drugs for Rare Disease Patients

Take action to support the OPEN ACT – Orphan Product Extensions Now, Accelerating Cures & Treatments (HR 971/S 1421).  The OPEN ACT could bring hundreds of safe, effective, and affordable medicines to rare disease patients within the next several years by incentivizing drug makers to “repurpose” therapies for the treatment of life-threatening rare diseases and pediatric cancers. EveryLife Foundation, NORD, Global Genes, Genetic Alliance and an additional 155 patient organizations support this bipartisan legislation.

 

Click here to take action!

Coalition Support letter for the Advancing Targeted Therapies for Rare Diseases Act of 2015

Coalition Support Letter for the Advancing Targeted Therapies for Rare Diseases Act of 2015

Patient-Focused Impact Assessment Act Sign-on Letter

PFIA Senate Sign On Letter, June 2015

Read more about the Patient-Focused Impact Assessment below:

1597: The Patient-Focused Impact Assessment Act

     Overview

The Food and Drug Administration Safety and Innovation Act (FDASIA) and its related user fee agreement contained a number of provisions intended to strengthen the meaningful engagement of the patient and other critical voices within the medical product review process. These provisions included the strengthening of existing tools and creation of new ones needed to fill gaps, policies to allow for greater engagement of external experts in the review process and a requirement for FDA to engage in multiple stakeholder meetings on the important issue of benefit/risk assessments. While patient stakeholders strongly supported these reforms, concerns exist as to how these new tools are being used by the FDA.

   Need for Greater Accountability & Transparency

To address these concerns and ensure that the FDA uses existing and future patient-focused drug development tools and authorities to the greatest extent possible, Congress should enact additional reforms through the 21st Century Cures Initiative focused on achieving this goal. The reforms should recognize the progress of recent years, including the high levels of enthusiasm and innovation among the patient stakeholder community to play a more active role in the product review process. The Patient- Focused Impact Act is bipartisan legislation sponsored by Sen. Roger Wicker and Sen. Amy Klobuchar to do just that through two provisions:

  • Development of the Patient-Focused Impact Assessment: Many stakeholders are questioning exactly how the FDA using PFDD tools and authorities when it comes to making critical decisions on whether or not to approve a product. To address this concern, the PFIA will require the development of a patient engagement assessment tool whose results would be included within the publicly disclosed safety and effectiveness data package of any approved drug. The assessment will be a simple and straightforward survey that asks reviewers how a number of PFDD tools and authorities informed their decision on a new drug application. Topics would include benefit/risk data for the indicated populations, draft or final guidances, patient-preference data, patient-reported outcomes data, and the views of patients and other external experts on the application. The assessment would shed light into how PFDD tools and authorities are or are not being used and would help identify gaps or shortcomings in the space.
  • Issuance of Draft & Final Guidance to Patients and Industry on PFDD: This provision recognizes the immense potential possible through greater use of PFDD tools as well as the need for greater clarity and guidance from the FDA to both patient organizations and industry sponsors. It will require the FDA to issue draft and final guidance with the final guidance informed by a public comment period. The guidance would seek to encourage patient and industry collaborations on the development of PFDD tools.

Cosponsor S. 1597

The Patient-Focused Impact Act builds upon the success in recent years to strengthen the patient voice in the medical product development process. It would enact two targeted reforms to provide greater clarity and transparency to all stakeholders and help demonstrate how the FDA is using these tools and authorities.

To cosponsor S. 1597, please contact Sarah Lloyd Allred with Sen. Wicker at 4-6253 or Sarah Smith with Sen. Klobuchar at 4-3244 today!

PFIA Senate Sign On Letter, June 2015

Advocates in North Carolina Pass State-Level legislation to Aid the Fight Against Rare Diseases

Taylor’s Tale, a Charlotte-based advocacy group in the fight against Batten disease and other rare diseases, spearheaded new legislation in North Carolina to create a new advisory council housed within the UNC-Chapel Hill School of Medicine to work towards finding more treatments for rare diseases.

North Carolina House Bill 823 will establish the council that will give guidance on research, diagnosis, treatment and education related to rare diseases. The council will consist of rare disease advocates including providers, researchers and patients.

NC State House

In short, the council is directed by the state to, “Advise on coordinating statewide efforts for the study of the incidence of rare diseases within the State and the status of the rare disease community.”

It can be hard to find a specialist who knows how to treat your rare disease. Disease advocacy groups, rare disease organizations and genetics clinics may help you to find one. A partnership between advocacy groups including Taylor’s Tale, academic medical centers, the National Institutes of Health and others keenly interested in the diagnosis and treatment of rare diseases can be a model for the nation as they tackle some of the most challenging diseases affecting our citizens. To learn more visit taylorstale.org

(Left) Sharon King and Representative Marilyn Avila, who was one of the bill’s main sponsors. sharon kin