In this issue: Advocacy Impact: Shwachman-Diamond Syndrome and the community's ideas were part of last week's kick-off meeting of FDA’s Rare Disease Innovation Hub
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Shwachman-Diamond Syndrome Alliance (SDS Alliance) shares ideas at new FDA Rare Disease Innovation Hub kick-off Meeting. FDA leaders launch rare disease hub to increase cross-center collaboration.
It was an incredible opportunity for the SDS Alliance to represent the SDS community at the FDA for the public kick-off meeting of the new Rare Disease Innovation Hub! Advocacy with Impact. Thank you to the Reagan-Udall Foundation for the FDA for organizing the meeting and making it possible for many rare disease organizations and advocates to be heard. The day was filled with insights from passionate stakeholders, advocates, and members of the rare disease community. FDA officials, including the directors of CBER and CDER, launched a rare disease innovation hub at a public meeting on October 16, 2024, aiming to enhance collaboration and communication across the two centers and externally, to serve as a single point of entry to engage with FDA, and to streamline and accelerate therapy development for rare diseases.
The Reagan-Udall Foundation for the FDA, in collaboration with FDA’s Rare Disease Innovation Hub, hosted a public meeting on October 16, 2024. The meeting brought together rare disease patient advocates, academic researchers, regulated industry, and other key stakeholders to discuss how the recently announced Rare Disease Innovation Hub can best engage with members of the rare disease community and prioritize its work.
During this public meeting, FDA sought to gather public comment in the following areas:
Cross-cutting rare disease-related, scientific, regulatory, or policy issues (those not related to a particular disease or condition) that should be prioritized for consideration by the Rare Disease Innovation Hub
Rare disease-specific (but not application-specific) scientific, regulatory, or policy issues that should be prioritized for consideration by the Rare Disease Innovation Hub
Rare disease-related activities or initiatives currently being undertaken independently by CDER or CBER that you believe would benefit from being undertaken by the Rare Disease Innovation Hub as a joint activity
Approaches that the Rare Disease Innovation Hub should follow for engagement with patients and caregiver groups, industry organizations, and scientific/academic organizations
In addition to the presentations during the hybrid meeting, comments can be submitted to the docket by October 31st as described in the Federal Register Notice.
The full meeting recording is now available on the meeting page and on YouTube, here:
The hub will work across different rare diseases, the vast majority of which do not have any treatment. The FDA said it will “particularly focus on products intended for smaller populations” or for diseases in which the natural history is not well understood. The hub “will bring us the coordination between the centers that I think will really make sure that things like biomarkers are coordinated,” Marks said in his introductory remarks. He added that CDER and CBER reporting structures won’t be disrupted but there will be more collaboration. CDER Director Patrizia Cavazzoni highlighted that this meeting already demonstrates how the hub “will turbo-charge our activities across centers. We really want to listen.” At the same time, they emphasized that they are in startup mode. Dynamic, strategic, AND still looking for a director to lead the effort.
“There will be differences between gene therapies and protein therapeutics and small molecules,” Marks said. “That may mean that sometimes an endpoint or policy may have to be different, but again, we’re committed to being transparent about this.”
The remainder of the meeting included 41 speakers — from patient groups to industry — including Shwachman-Diamond Syndrome Alliance's President and CEO Dr. Eszter Hars, discussing everything from the rare diseases of their children to ways that the FDA can be more nimble. The agency said it received more than 200 requests to participate in the meeting.
Some speakers sought improved consistencies across the FDA drug and biologic centers, citing the need to establish regular and transparent lines of communication with sponsors. Others requested a roadmap for the hub’s short and long-term goals. Comments on the docket are still open, according to the FDA. Dr. Hars highlighted the need of the SDS and many other rare disease communities to collaborate with the FDA, researchers, and other stakeholders to support the development of biomarkers and outcome measures, which could support the development of new therapies using different modalities and could accelerate the work and impact across multiple agencies.
More about the Rare Disease Innovation Hub from the meeting organizers and its impact on SDS Alliance's mission
"An estimated 10,000+ rare diseases affect more than 30 million people – approximately one out of every 10 people – in the United States, and about half of these people are children. While there have been important advances in treatments for rare diseases, more needs to be done. Many rare diseases lack treatment options and therefore many patients have high unmet medical needs. This requires new approaches to expedite development and approval of safe and effective drugs and biologics. The FDA sees huge potential in establishing a new model, within the FDA, to leverage cross-agency expertise and greatly enhance our inter-center connectivity to spur the development of treatments for rare diseases.
To that end, the Center for Biologics Evaluation and Research (CBER) and the Center for Drug Evaluation and Research (CDER) are launching a Rare Disease Innovation Hub to advance rare disease therapy development through greater communication, collaboration, and coordination across CBER and CDER, in coordination with other Centers and Offices across FDA. Helmed by senior leadership from CBER and CDER, the Hub will work to develop and implement a comprehensive cross-center rare disease strategic agenda that takes full advantage of our current clinical and scientific expertise across both centers and is based on a shared vision and comprehensive approach to 1) align methods and approaches to rare disease programs, both during IND development, and in application review; 2) identify and enable innovative approaches in the areas of novel endpoints, biomarker development, and innovative trial designs; and 3) streamline communications with the rare disease community. The Hub will work across rare diseases but will especially focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood, as we recognize that development of therapies for these conditions can be particularly challenging."
This gives us at the SDS Alliance reasons for hope and momentum to implement our strategies to no only make therapy development possible, but fast and likely.
The Role of SDS Alliance
Organizations like SDS Alliance provide vital support for individuals and families affected by Shwachman-Diamond Syndrome, in particular offering access to information on the latest research and foundational understanding of the basics of research participation. They offer resources, connect patients with medical experts and researcher, and foster a community where individuals can share experiences and information and turn hope into action, together.
Why Community Matters
Being part of a community provides emotional support and access to the latest research and treatment options. SDS Alliance advocates for awareness, research funding, and educational resources that can greatly enhance the quality of life for those affected by SDS.
Conclusion
Shwachman-Diamond Syndrome is a complex genetic disorder that requires coordinate care, caring community support, and strategic investment into research and research infrastructure to de-risk therapy development. Organizations like Shwachman-Diamond Syndrome Alliance (SDS Alliance) play a crucial role in providing support and resources for those affected and the SDS community at large. Join us and raise awareness to help others learn about this important condition.
For more information, visit SDS Alliance and join a community dedicated to improving the lives of those impacted by Shwachman-Diamond Syndrome.
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