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-Pick Disease via email and social media as they develop.
May 15, 2024
NNPDF joins the broader rare disease community in signing on to preserve the federal-level committee focused on newborn screening. This committee plays a vital role in the early diagnosis of rare and serious genetic conditions in newborns, and its continued work is essential to the future of this important effort. We’re proud to stand with our partners in support of this initiative.
April 1, 2024
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 the robustness of the application as it is supported by strong efficacy and safety results, and our commitment to supporting the IB1001 application throughout the review process.
February 21, 2024
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.
March 21, 2022
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.
August 8, 2022
July 19, 2022
The Duke-Margolis Center for Health Policy 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.
June 13, 2022
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 was held on Thursday, August 4, 2022, to provide an overview of the key themes and future directions shared during the January 2022 workshop and presented in the workshop summary report.
April 29, 2022
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.
February 11, 2022
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.
January 11, 2022
A public workshop was held by Duke-Margolis Center for Health Policy, sponsored by the FDA, on January 24 and 25, 2022. Participants discussed clinical endpoints relevant to NPC clinical trials and innovative strategies to support therapeutic development for patients with NPC.
December 15, 2021
On behalf of the NPC community and in conjunction with partner organizations, NNPDF sent the following letter to Dr. Mark McClellan 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.
December 6, 2021
FDA and Duke-Margolis hosted a virtual public workshop on January 24–25, 2022 on endpoint considerations to facilitate drug development for Niemann-Pick Type C. The workshop was open to the public.
FDA Listening Session on Niemann-Pick Disease – April 9, 2021
NNPDF hosted an FDA listening session on Niemann-Pick Disease on Friday, April 9, 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!
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.
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.
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.
Letter regarding: Payer Matrix identifying itself as both a “Patient Advocacy Company” and a “Leading Patient Advocate” is untrue and misleading to consumers.
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.
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.
NNPDF continues to support Rare Disease Advisory Councils (RDACs) 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 RDACs in the states of Georgia, Indiana, Mississippi, Texas, and Wisconsin. Read the letters below and learn more about this issue.
Joslyn Crowe, Executive Director, and Justin Hopkin, Board Chair, attended WORLDSymposium 2022, 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.
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