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Advocacy

Advocacy is central to our core values and our mission. We believe it is important to be a champion for the community and to be a driving force of positive change. NNPDF advocates on issues related to and impacting the Niemann-Pick community at the federal, state, and regional levels. 

The NNPDF will keep you up-to-date on events related to Niemann-Nick Disease via email and social media as they develop.


BENEFIT Act of 2021

NNPDF recently signed on to a letter to US Senators and Representatives in support of the BENEFIT Act of 2021. The Better Empowerment Now to Enhance Framework and Improve Treatments Act of 2021 (BENEFIT Act of 2021) is a bill that requires the US Food and Drug Administration (FDA) to consider relevant patient-focused drug development data, such as data from patient preference studies and patient-reported outcome data, in the risk-benefit assessment framework used in the process for approving new drugs.

Read the letter

03/21/2022

Addendum to Endpoint Considerations to Facilitate Drug Development for NPC Workshop Summary

The Duke Margolis Center for Health has shared an addendum to the January NPC workshop summary we previously released. This addendum provides a brief summary of the comments received through the workshop docket.

As a reminder, the FDA is hosting a August webinar to share and discuss the high level results of the summary document. Advance registration is needed.

07/19/2022

Endpoint Considerations to Facilitate Drug Development for NPC Workshop Summary

Duke-Margolis Center has shared an update related to the January workshop on Endpoint Considerations to Facilitate Drug Development for NPC. Your contributions prior to and during the workshop helped ensure that patients and families were at the center of the conversation and that the event was informative for all NPC drug development stakeholders. The workshop summary is now available.

A brief webinar along with the FDA will be held on Thursday, August 4, from 3:00-4:00 pm ET to provide an overview of the key themes and future directions shared during the January 2022 workshop and presented in this newly released workshop summary report.

The webinar is intended to be another touchpoint for NPC drug development stakeholders on this important topic. Stakeholders will be able to submit questions about the workshop summary in advance of the webinar using this form, and we will get to as many as we can in the webinar format. Please note that the webinar is not intended to cover any new content beyond the January workshop and the workshop summary.

Workshop Summary
Webinar Registration
Questions Submission Form

06/13/2022

Duke-Margolis NPC Public Workshop Update

A public workshop was previously held by Duke-Margolis Center for Health Policy, sponsored by the FDA, allowing participants the opportunity to discuss clinical endpoints relevant to NPC clinical trials and innovative strategies to support therapeutic development for patients with NPC.

Following this workshop, NNPDF submitted a letter and supporting materials to the FDA and Duke Margolis Center for Health Policy in response to the docket.  We are currently awaiting the Summary Document from this workshop which we will share widely once it is available.

04/29/2022

Duke-Margolis NPC Public Workshop Update

The public workshop was recently held by Duke-Margolis Center for Health Policy, sponsored by the FDA. This virtual public workshop allowed participants the opportunity to discuss clinical endpoints relevant to NPC clinical trials and innovative strategies to support therapeutic development for patients with NPC.

Several of our Niemann-Pick community members were able to share their stories during this event. As a community, we thank them for advocating on behalf of the entire NPC community to ensure that our families and expert clinicians voices are heard and incorporated into decision making and outcomes.

Submit workshop comments: Docket FDA-2021-N-1297. Docket closes April 25, 2022.
Draft Agenda for NPC Public Workshop

02/11/2022

Duke-Margolis NPC Public Workshop Update

A reminder that a public workshop is being held by Duke-Margolis Center for Health Policy, sponsored by the FDA. It is a public workshop, and all are welcome to register to attend this virtual event. Participants will discuss clinical endpoints relevant to NPC clinical trials and innovative strategies to support therapeutic development for patients with NPC.

The event will be held on January 24, 2022 from 12:00pm – 3:30pm EST and January 25, 2022 from 12:00pm – 3:30pm EST. The public workshop is open to all, you can register here.

You may submit comments for this workshop to Docket FDA-2021-N-1297. Docket will be open until April 25, 2022.

Draft Agenda for NPC Public Workshop
Register for Virtual Workshop
Submit workshop comments: Docket FDA-2021-N-1297

01/11/2022

Endpoints Consideration to Facilitate Drug Development for NPC

Duke-Margolis NPC Public Workshop Update

On behalf of the NPC community and in conjunction with partner organizations, NNPDF sent the following letter to Dr. Mark Clellan regarding the January 2022 public workshop for endpoints consideration in drug development for Niemann-Pick type C. We are advocating to ensure that our families and expert clinicians voices are heard and incorporated into decision making and outcomes.

The public workshop on January 24-25, 2022 is open to all, you can register here.

Letter to Dr. Mark Clennan

12/15/2021

FDA and Duke-Margolis NPC Workshop
FDA and Duke-Margolis will host a virtual public workshop on January 24-25, 2022 on endpoint considerations to facilitate drug development for Niemann-Pick Type C. The workshop is open to the public. Learn more here.

12/06/2021

FDA Listening Session

NPC Community Listening Session Report – August 3, 2021

FDA Listening Session on Niemann-Pick Disease – April 9, 2021
NNPDF hosted an FDA listening session on Niemann-Pick Disease on Friday, April 9th, 2021. The listening session was a platform for the NNPDF to present to the FDA/CDER and to share the concerns and priorities from the entire Niemann-Pick Disease community.

The Listening Session Summary Report was submitted to the FDA along with several patient voice statements and the arimoclomol support letter which was signed by 500+ Niemann-Pick community members! Thank you for supporting your Niemann-Pick community! Review the NNPDF FDA Listening Session Summary Report.


Institute for Clinical and Economic Review (ICER)

NNPDF recently supported a coalition letter, led by the Haystack Project, in response to the Institute for Clinical and Economic Review’s (ICER) national call for suggestions on how to improve its value assessment framework.

Haystack Project is a non-profit organization enabling rare and ultra-rare disease patient advocacy organizations to coordinate and focus efforts that highlight and address systemic reimbursement obstacles to patient access.

2020 Update to ICER Value Framework


National Organization for Rare Disorders (NORD)

NNPDF recently supported a letter as representatives of the 30 million Americans living with rare diseases. Our organizations wrote to urge the U.S Senate committee on the Judiciary to take steps to protect the lives of individuals with rare diseases and their caregivers who come to the United States to participate in clinical trials or receive life-saving care. The Department of Homeland Security’s United States Citizenship and Immigration Services (USCIS) recently announced a policy shift that left these individuals facing deportation.

Read complete letter.


Rare & Ready Genetic Condition Coalition

NNPDF has partnered with a statewide grassroots advocacy coalition of 25 non-profit rare disease organizations called Rare & Ready: A Genetic Condition Coalition. We believe action must be taken to improve timely access to FDA-approved medicines in Medicaid for those with rare and genetic conditions. Our efforts are not disease- or treatment-specific; rather, we seek to address concerns of the broader rare community. Follow the links below to learn more.

Rare & Ready to State of Florida
Rare & Ready to State of Georgia

Rare & Ready Infographic


Rare Disease Advisory Councils

NNPDF continues to support Rare Disease Advisory Council’s (RDAC) across the country. RDACs give rare disease community members a unified voice in state government and provide a valuable resource to elected officials on rare disease education and challenges. Currently, only 21 states have RDACs. NNPDF has signed on to RDAC’s in the states of Georgia, Mississippi, and Wisconsin. Read the letters below and learn more about this issue.

Georgia RDAC
Mississippi RDAC
Wisconsin RDAC


WORLDSymposium 2022

Joslyn Crowe, Executive Director and Justin Hopkin, Board Chair attended WORLDSymposium 2022 last week where they heard the latest updates on research in lysosomal storage diseases. They presented a poster co-authored by NNPDF on “A qualitative study to understand caregivers’ burden of acid sphingomyelinase deficiency” and were able to connect with several colleagues in the rare disease space.

POSTER: A qualitative study to understand caregivers’ burden of acid sphingomyelinase deficiency

Supporting one another. Supporting our community.