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
- Duke-Margolis NPC Public Workshop
- Endpoints Consideration to Facilitate Drug Development for NPC
- FDA Listening Session
- ICER – Institute for Clinical and Economic Review
- Interstate Medical Licensure Compact
- National Organization for Rare Disorders (NORD)
- Newborn Screening
- NNPDF Community Response: FDA Review of Arimoclomol
- NPC Community Letter of Support: FDA Review of IB1001
- Payer Matrix – Response
- Rare & Ready Genetic Condition Coalition
- RARE Act
- Rare Disease Advisory Councils
- WORLDSymposium 2022
NPC Community Letter of Support: FDA Review of IB1001
On behalf of the NPC community, NNPDF and partner organizations including, APMRF, DART, FireFly Fund, Hadley Hope, Hide & Seek Foundation, Hope for Marian, and SOAR, have produced a Community Letter of Support for the FDA review of IB1001. This letter affirms our confidence in robustness of the application as it is supported by strong efficacy and safety results and our commitment to supporting IB1001 application throughout the review process.
Read the Community Letter of Support.
04/01/2024
NNPDF Community Response: FDA Review of Arimoclomol
On behalf of the NPC community, NNPDF submitted the arimoclomol community response and support statement to the FDA earlier this month. Our intent was to ensure the FDA heard from families living with NPC and other care partners, clinicians, and community supporters, since any decision FDA makes regarding arimoclomol will impact you and your loved ones. Thank you to all that signed on and shared your voice in support of arimoclomol for the treatment of NPC.
Read the community response and support statement.
02/21/2024
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.
03/21/2022
Duke-Margolis NPC Public Workshop
Webinar on Endpoint Considerations to Facilitate Drug Development for Niemann-Pick Type C (NPC): Key Themes and Future Directions from the January 2022 Public Workshop – August 4, 2022
08/08/2022
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.
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
Interstate Medical Licensure Compact
The Interstate Medical Licensure Compact is an agreement among participating U.S. states to work together to significantly streamline the licensing process for physicians who want to practice out-of-state. It offers a voluntary, expedited pathway to licensure for physicians who qualify. The Compact has the potential to increase access to health care and extend the reach of physicians through telemedicine.
As a partner of the Rare & Ready Genetic Condition Coalition, NNPDF has joined with 46 non-profit rare disease patient organizations in support of the Interstate Medical Licensure Compact. Read the letter below and learn more about this issue.
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.
Payer Matrix
Letter Regarding: Payer Matrix identifying itself as both a ‘Patient Advocacy Company’ and a ‘Leading Patient Advocate’ is untrue and misleading to consumers.
Rare & Ready Genetic Condition Coalition
NNPDF has partnered with a statewide grassroots advocacy coalition of 46 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.
Website: rareandready.org
Rare & Ready Information Flyer
Rare & Ready to State of Florida
Rare & Ready to State of Georgia
Rare & Ready Infographic
RARE Act
NNPDF is among 78 organizations that joined in support of S. 1214, the Retaining Access and Restoring Exclusivity (RARE) Act, as introduced by Senator Tammy Baldwin, in the upcoming markup for HELP Committee consideration. The RARE Act would maintain the original intent of the Orphan Drug Act, making clear that orphan drug exclusivity is tied to the approved indication, while ensuring proper incentives remain in place to ensure that robust rare disease drug development continues for all rare disease patients.
Read the RARE Act letter of support.
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, 27 states have RDACs. NNPDF has signed on to RDAC’s in the states of Georgia, Indiana, Mississippi, Texas, and Wisconsin. Read the letters below and learn more about this issue.
Georgia RDAC
Indiana RDAC
Mississippi RDAC – Senate Bill 2156 (Blackwell)
Mississippi RDAC – House Bill 616 (McKnight)
Texas 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