Action ALERT from the EveryLife Foundation: Protect NIH & FDA Funding in 21st Century Cures

We are less than 24 hours away from the historic House vote on the 21st Century Cures Act.

Unfortunately, several Members of Congress have introduced an amendment that would strip away billions in needed funding for NIH & FDA, which would undue much of what we have fought for. We cannot allow this amendment to stand! I know many of you called on Tuesday and your calls are making a difference and getting through. We’re asking you to call again to ensure that Congress gets the message.

Please take a moment to call your Representative and urge them to vote YES on 21st Century Cures and to protect the Cures Innovation Fund. 

As always, THANK YOU for your support and for keeping up the fight! And don’t forget to share this with your networks. We’re close to the finish line!

Action ALERT from the EveryLife Foundation: Urge Congress to Vote YES on the 21st Century Cures Act

As soon as you read this, please click here to call your Member of Congress and urge them to vote YES on the 21st Century Cures Act HR 6.

This week the House will hold a floor vote that will determine the fate of the 21st Century Cures Act HR 6. This historic legislation holds the potential to dramatically advance rare disease research and includes the OPEN ACT, legislation that could double the number of rare disease treatments. But we need your help to ensure the bill passes the House.

Your call and your voice could help make the difference. Please take a few moments to call and take a stand for rare disease patients everywhere.

THANK YOU for your support and for keeping up the fight on behalf of the rare disease community. Please remember to share this alert with your networks and on social media. We need all hands on deck!

Urgent Action Needed: Help Save 21st Century Cures in Georgia

We need your help to pass a historic piece of legislation called the 21st Century Cures Act (HR 6). The bill contains billions in new funding for the Food & Drug Administration, The National Institutes of Health, and a provision that could double the number of treatments available to rare disease patients (The OPEN ACT).

The bill will be coming up for a vote next week and we’ve received reports that Congressman Price in Georgia is not supportive of the legislation.  We urgently need you to send a short email to Kyle Zebley in Rep. Price’s office to tell him how important this bill is to you and your family and for all rare disease patients.  Below, I’ve included a sample message, that can be sent to Rep. Price’s Legislative Director, Kyle Zebley, at kyle.zebley@mail.house.gov

Read more

In-District Lobby Days

Presented by:

 

We hope you join us for our 2nd annual In-District Lobby Days! In August, Congress will return home to their districts to connect with constituents.  This is the perfect opportunity for the rare disease community to meet with Members of Congress.As you read this, Congress is considering a historic legislative package called 21st Century Cures, that could be a game changer for the rare disease community. Your advocacy could help shape policy and advance the development of treatments and cures for rare diseases. Click below to sign up for In-District Lobby Days:
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Deadline to register was Friday, July 17th. Please stayed tuned for more opportunities to advocate for patients with rare diseases!

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Meetings will take place between August 3rd and September 4th in the district offices of your legislators.  Once you register, RDLA with Advocacy Associates, will begin scheduling your meetings based on your availability. To prepare advocates for their meetings, we ask that you to join us for one of our Regional Legislative Conferences (separate registration required):

 

 

 

or
attend our webinars on July 8th & Aug 5th (you will receive call-in information in the confirmation email)

If you have any questions feel free to contact Andy Russell @ (415) 884-0223, arussell@everylifefoundation.org

 

Sponsored by:

 

Action Alert: Support 21st Century Cures!

Would you like to help Congress pass the 21st Century Cures Act?

Congress is on the verge of voting on a historic bill called the 21st Century Cures Act (HR 6) – a piece of legislation that could be a game changer for the rare disease community. The bill contains several provisions to incentivize the development of treatments for rare diseases, and holds the potential to double the number of treatments available to rare disease patients.

Rare Disease Legislative Advocates’ West-Coast Conference

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Presented by: 

Friday, July 31, 2015

University of California San Francisco, Mission Bay, Betty Irene Women’s Hospital

Rare disease patient advocates and other stakeholders from the rare disease community came together on Friday, July 31th, 2015 in San Francisco, CA for our inaugural West Coast Legislative Conference. The goal of our new regional legislative conferences was to update advocates about important legislation advancing through Congress and to prepare them to meet with their Representatives during August Recess.

Thanks to our co-hosts and sponsors, advocates were updated and discussed important legislation advancing through Congress and are prepared to meet with their Representatives during August Recess. We were joined by the esteemed Dr. Keith Yamamoto, Rep. Eric Swalwell (CA-15), & Willie Brown. Lunch was provided, and a networking reception followed the event.

Click here for the Agenda

This event was a great success view photos here:

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Rare Disease Legislative Advocates’ North-East Conference

 

Monday, July 20, 2015

Stanley Bergen Building, Rutgers University, Newark, NJ

Rare disease patient advocates and other stakeholders from the rare disease community came together on Monday, July 20th, 2015 in Newark, NJ for our inaugural North-East Legislative Conference. The goal of our new regional legislative conferences was to update advocates about important legislation advancing through Congress and to prepare them to meet with their Representatives during August Recess.
This year Congress is working on legislation that will have a huge impact on drug development and approval, and as a community we must join together to ensure the legislation benefits rare disease patients and is passed in both the House and the Senate.

 

 

This event was a great success view photos here:
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Thank you to our sponsors:NJ Leg Conf Logo Mashup 7.13.3

NIH Innovation Fund

The NIH innovation fund would raise NIH funding to $31.8 billion in fiscal 2016 and to $34.85 billion in fiscal 2018. Congress appropriated $30.3 billion for the NIH in fiscal 2015, and President Barack Obama requested (PDF) $31.3 billion for fiscal 2016.

The bill would also create an NIH Innovation Fund of roughly $2 billion per year between fiscal years 2016 and 2020. The fund would support precision medicine, young emerging scientists and other unidentified sources of medical innovation.

Research!America, said it was “thrilled” by the new version of the bill but warned that the final legislation must allow the NIH to decide how to allocate the dollars.

Precision Medicine in 21st Century Cures

From FDA Law Blog by James E. Valentine:

Precision Medicine:  Congress finally provided some detail about precision medicine in the second version of the 21st Century Cures Act (Sec. 2041).  While the second version of the bill provided a definition of precision medicine, the latest version would have FDA define the term.  The revised provision would require FDA to issue a guidance document to assist sponsors in the development of such drugs.

This provision also allows for applications of a precision medicine that has been designated “for a rare disease for a serious condition” to rely upon data or information previously developed by the sponsor for a prior approved drug or indication in order to expedite clinical development of a precision medicine or indication that is using the same or similar approach as that of the prior approved drug.  In addition, the provision specifies that applications for precision medicines should be considered as to whether they are eligible for accelerated approval.

Patient Focused-Drug Development in 21st Century Cures

From Alexander Gaffney in a RAPS article:

Title II of the 21st Century Cures legislation seeks to build on FDA’s recent launch of a Patient-Focused Drug Development (PFDD) program. That program, launched in 2012 under the Food and Drug Administration Safety and Innovation Act (FDASIA), is meant to incorporate patient preferences into FDA’s regulatory decision-making. The idea behind the proposal is that different patients—even those suffering from the same disease—have varying levels of risk they are willing to accept.

For more on the Patient-Focused Drug Development Program, please see our tracker here.

As legislators explained in a white paper accompanying the last draft of the bill, “No one understands a particular condition or disease better than patients living with it.” Accordingly, the bill would require FDA to establish a structured framework for the meaningful incorporation of patient experience data into the regulatory decision-making process, including the assessment of desired benefits and tolerable risks associated with new treatments.

That measure is likely to win support at FDA, which has sought to develop similar approaches in the past and has been receptive to patient feedback. Under the approach, FDA would have to, within two years, develop and implement a process to allow companies to submit “patient experience data,” which could be used to develop a “structured risk-benefit assessment framework.” A draft guidance document would also be due from FDA within two years.