News

Forwarded to COMBINEDBrain by Vanessa Vogel-Farley, REN:

On October 28, 2026, the U.S. Food and Drug Administration, in partnership with the Reagan-Udall Foundation for the FDA, will convene a hybrid public meeting to seek input related to the FDA’s Rare Disease Framework Initiative to reimagine the agency’s approach to rare disease drug development. The intent of this new initiative is to develop a fit-for-purpose Framework that reflects the realities of rare disease drug development by considering the nuances of each disease, patient needs, product type, and the evidence that realistically can be generated in small patient populations. 

The public meeting will be an opportunity for members of the rare disease community – including patients, disease advocates, sponsors, and others – to provide input on the direction and focus of the Framework. Insights from the public’s remarks will inform the agency’s upcoming work. 

The FDA welcomes all feedback related to the Framework Initiative and in particular seeks input on the following topic areas: 

1. Fit-for-purpose review of rare disease therapies based on attainable evidence, particular disease state, and population. 
2. FDA consistency and predictability of drug development and review regulatory requirements.  
3. FDA engagements with external parties – including patients, disease experts, and researchers – to inform review decision-making. 
4. Considerations for chemistry, manufacturing, and controls (CMC) specific to rare disease therapies.

This link also allows you to register for in-person or virtual attendance and request to provide public comment.

https://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/fda-rare-disease-framework-initiative-public-meeting-10282026

For more information, please go to the event website:

https://therorybellefoundation.org/events/a-night-for-nars1

Announcement of Grant Funding Opportunity for Rare Disease Research:

The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) is pleased to announce a new funding opportunity for the Orphan Products Grants Program to support clinical trials for rare diseases and conditions.  The purpose of this funding opportunity announcement is to fund clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in labeling to address unmet needs in rare diseases or conditions. 

Through the funding of collaborative, efficient, and/or innovative clinical trials, FDA expects to increase the number of approved treatments for rare diseases and exert a broad and positive impact on rare disease drug development. 

Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01)

Receipt Date: October 20, 2026

FOA Number: RFA-FD-25-020

•    Purpose: To fund clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in labeling to address unmet needs in rare diseases or conditions. 

•    Focus: Efficiency, innovation, impact, collaboration, leveraging patient input, infrastructure and financial resources

•    Efficiency: Encourage efficient and innovative clinical trials in all phases of product development through adaptive and seamless trial designs, basket, umbrella and platform trials studying multiple rare diseases/products and the use of data modeling and simulations. Allow additional total costs up to $250,000 per year for studies using efficient and innovative designs.

•    Contact: Katherine Needleman, Director, Orphan Products Grants Program

                     E-mail: katherine.needleman@fda.hhs.gov

•    Additional Information: OOPD Website

Please forward this announcement to any parties who may have need and interest in this grant program and please post the information on social media to alert interested parties.  

Thank you for your help in bringing awareness to this important opportunity.

Bridget Nugent, Ph.D.

Health Scientist, Office of Orphan Products Development

Office of the Commissioner

U.S. Food and Drug Administration

U.S. Department of Health and Human Services

Tel: 301-796-9443

 

From Terry Jo Bichell via LinkedIn:

What a historic 2 days!! The very first RR-RGND launched in Arlington, Virginia on Sept 28-29 and the right people were in the room when it happened!! All the stakeholders: Industry, Patient Advocates, Clinicians, FDA, Funders, Statisticians, Psychometricians, even Payers were in the room with one problem to solve: “Is the child's life better because they took the medication? And how do we measure that?” It is a notoriously thorny problem to measure benefit in people with cognitive disability who have a hard time speaking for themselves. The historic shift was for collaboration rather than competition between all these players to help get good treatments to kids who need them. I am so proud to have worked closely with Elizabeth Berry-Kravis, Michelle Campbell Emma James Stéphane Auvin, MD, PhD, FAES Konrad J. Werhahn Kimberly Goodspeed, MD Sunitha Malepati, Charlene Son Rigby, Annie Kennedy, Berit Powers, Laura Pisani, MD, MBA, FACMG, Nicola Williamson, Robert Komorowski, Scott Demarest, Bryce Reeve, Smitha Jagadish, Rachael Hawtin and the COMBINEDBrain team to make this meeting come together. It was built on the shoulders of giants, Jacqueline French and the Epilepsy Foundation Research Roundtable for Epilepsy.

THANK YOU TO OUR GENEROUS ANNUAL SUMMIT SPONSORS!

There are more opportunities available! Click HERE to request information!

We did not get the required 10 participants signed up for a roadshow at the Annual Summit so we won't be having one this year.

THANK YOU!

From a soldier to a musician, from an immigrant family to a mother of twins, meet the unsuspecting heroes waging war against rare disease. Love drives change in this rare disease revolution, where parents become scientists and warriors who wage battles in labs, Congress, and their own homes to drive scientific breakthroughs, hope and cures for countless others. Rent the documentary here!

Paige Kalik, Genetic Counseling student, has authored a book about being the sibling of a child affected by a rare genetic neurodevelopmental condition. Emily Amerson, PAG Leader and Governing Board Member, illustrated it! Paige is donating profits from the sale of this book to COMBINEDBrain. You may order your copy here or at the QR code below!

Shared by Jared Bancroft, TPRM3 Foundation

Thought this would be of interest to folks here ...

Broad, Boston Children’s, and Jackson Labs have just launched the Center for Therapeutic Genetics. It is aimed at finding treatments for rare and ultra-rare diseases. One of their early areas of focus will be in CNS specifically around genetic epilepsies. Definitely something to keep an eye on as they get off the ground. Click the link above and go to the "Connect" tab to sign up for their newsletter to keep apprised of their progress!

Press release with more details - 

https://www.broadinstitute.org/news/broad-institute-boston-childrens-hospital-and-jackson-laboratory-launch-center-therapeutic

COMMUNITY HEALTH EQUITY GRANT OPPORTUNITY!

Apply to Our 2026 Grant Cycle by August 10th RTW Foundation's 2026 grant cycle is still open! If your organization is committed to advancing health equity in New York City for communities of color, low-income neighborhoods, recent immigrants, and other underserved populations, be sure to submit your LOI before the deadline. This year, our community health team is expanding services beyond funding to support communities and organizations particularly impacted by changes in safe, affordable access to care. We look forward to providing grant funding, co-hosting health fairs, and expanding health education with local health organizations.  LOI Deadline: Monday, August 10th - https://www.tfaforms.com/5226638 Learn more and submit an LOI here Please feel free to share this opportunity with local health equity organizations in New York City. If you have any questions or want to connect, please reach out to our Director of Community Engagement, Sarah Garwood at sg@rtwf.org. LinkedIn Instagram Facebook YouTube Copyright © 2026 RTW Foundation, All rights reserved. You are receiving this email because you opted in via our website. Our mailing address is: RTW Foundation 40 10th Ave Fl 3 New York, NY 10014-1115 Add us to your address book Want to change how you receive these emails? You can update your preferences or unsubscribe from this list.