In this issue: Join the community webinar and Q&A session to learn all about the EL-PFDD meeting on Shwachman-Diamond Syndrome and have all your questions answered! This is your chance to make a real impact on future treatments and a brighter future for all SDS patients. Be heard by the FDA, researchers, doctors, and all stakeholders.
This is the latest issue of the SDS Alliance Blog! Welcome to timely updates on all things SDS, Science, and Advocacy. We bring you a digest of recent scientific publications, conferences, and other newsworthy content - all relevant to SDS - with links to more details and learning opportunities. Are you interested in anything specific? Did we miss something? Let us know. Email connect@SDSAlliance.org or message us on Facebook! This is all for you!
Dear patient/caregiver,
Have you heard about the Externally-Led Patient Focused Drug Development Meeting for Shwachman-Diamond Syndrome planned for June 4th, 2025 in a hybrid format (Zoom and in-person in Cincinnati, OH)? See all the details at www.SDSAlliance.org/pfdd
This is a big deal for all of us and we want the entire SDS community to be involved and active! To prepare, we are hosting a community webinar and Q&A session next Thursday, January 23, 4 pm ET, to answer all your questions! Meet your host, and learn from the expert consultant James Valentine who is supporting the planning process.
Register for the community webinar and Q&A today, here:https://zoom.us/meeting/register/g7CpXvQMSkS8PiOs4XicXw
After registering, you will receive a confirmation email containing information about joining the meeting.
Why you should join the community webinar and Q&A to learn more about the SDS PFDD meeting?
Externally-Led Patient Focused Drug Development (EL-PFDD) Meeting is a special types of meeting developed and overseen by the US Food and Drug Administration (FDA) to facilitate capturing the lived experiences of people who have a certain health condition, in this case Shwachman-Diamond Syndrome, in order to facilitate regulatory decision making, research prioritization, and clinical trial design in a way that benefits us, the patients.
This community webinar will provide an overview of what this type of meeting entails, and how you can get involved. As we shared on the SDS PFDD meeting page, there are several ways to get involved. The webinar is a great opportunity to get all your questions answered.
We will hear from PFDD experts, our consultants, who are helping us plan and implement a high-impact PFDD meeting for Shwachman-Diamond Syndrome
After a brief introduction by your host, Dr. Eszter Hars/SDS Alliance, we will hear from out PFDD consultants James Valentine and Larry Bauer, of Hyman, Phelps, and McNamara. James Valentine assists medical product industry and patient advocacy organization clients in a wide range of regulatory matters, including new drug and biologic development and approval issues. Mr. Valentine also works with clients on clinical trials operations and compliance matters.
About our speaker and consultant, James Valentine, and his role in the SDS PFDD meeting
We chose James as our partner and guide in planning the SDS PFDD meeting due to his unparalleled experience and dedication to the community and this mechanism to accelerate therapy development for rare diseases like SDS. Working with James, we are also benefitting from the support of his colleague Larry Bauer. They are both former FDA officials who were involved with the launch of the PFDD initiative in 2012 and in private practice have helped with over 70 EL-PFDD meetings to date, which is the majority of EL-PFDD meetings ever to take place! More details about the SDS PFDD meeting (planned for June 4th, 2025) and opportunities to get involved at www.SDSAlliancce.org/pfdd.
Our speakers at this webinar (Ask an Expert series): Larry Bauer and James Valentine from Hyman, Phelps & McNamara
James has worked the last 13 years as a champion for the patient voice as part of the regulatory process. James previously worked at the FDA where he was a patient liaison, helping to incorporate the patient voice into medical product review across the FDA’s various medical product centers and review divisions. There, he helped to develop and launch the Patient-Focused Drug Development initiative.
Larry worked at the NIH for 17 years in clinical research, followed by a position at the FDA as a Regulatory Scientist in the Center for Drug Evaluation and Research’s Rare Diseases Program, a group he co-founded and worked for 10 years, where he advanced rare disease drug development.
In private practice, James and Larry have worked with many patient organizations to ensure their community’s voices were heard by decision-makers. Relevant to our EL-PFDD meeting, they has been involved in helping plan and moderating three-fourths of the over 75 externally-led PFDD meetings.
Before joining the firm in 2014, Mr. Valentine worked in FDA’s Office of Health and Constituent Affairs (previously Office of Special Health Issues) where he facilitated patient input in benefit-risk decision-making and served as a liaison to stakeholders on a wide range of regulatory policy issues. Mr. Valentine administered the FDA Patient Representative Program, facilitated stakeholder consultations during the reauthorization of PDUFA and MDUFA, helped launch the Patient-Focused Drug Development program, and developed the FDA Patient Network.
Mr. Valentine also worked at the Center for Drug Evaluation and Research’s (CDER) Office of Regulatory Policy where he coordinated the implementation of the medical gases certification scheme that was established in FDASIA and handled a variety of postmarket safety issues including REMS and safety labeling changes.
The Patient Voice is absolutely critical for patient focused drug development and advancement of any treatment. This is true for Shwachman-Diamond Syndrome (SDS) as well.
EL-PFDD meetings give the FDA and other key stakeholders, including medical product developers, healthcare providers, and federal partners, an important opportunity to hear directly from people living with SDS, their families, caregivers, and patient advocates about the symptoms that matter most to them, the impact the disease has on people’s daily lives, and their experiences with currently available treatments. This input can help inform the FDA’s decisions and oversight during drug development and new drug review, which is crucial for expanding options for improving the lives of those living with SDS. It will also help medical product developers, researchers, clinicians, and the general public gain an in-depth understanding of what living with SDS really means.
The EL-PFDD meeting is a once-in-a-lifetime opportunity for you—patients and caregivers—to use your voice and make an impact on the future of SDS therapy development. It is an opportunity to share your experience and perspectives on living with SDS, particularly as they relate to the community's unmet needs and consideration for future therapy development efforts. Learn more on the EL-PFDD for SDS information page.
There will be several ways and opportunities for you to contribute your voice, from speaking at the EL-PFDD meeting live or virtually, responding to polls and discussion during the meeting, filling out surveys ahead of the meeting, and participating in a qualitative research interview (SDS Patient LENS study).
Right now, we are specifically looking for patient and caregiver speakers/panelists for the EL-PFDD meeting - in person in Cincinnati, OH on June 4th. Applications are now open through January 26, 2025. Apply using this survey. Selected speakers/panelists will receive support in developing and delivering their stories through professional coaches, and their travel/accommodation will be covered. Childcare may be available upon request.
Other ways to get involved are available on the SDS PFDD website at www.SDSAlliance.org/pfdd
Please note that the EL-PFDD meeting is scheduled to take place on the day before the SDS Congress (11th International Schwachman Diamond Syndrome Scientific Congress in Cincinnati, OH), at the same or nearby location (and via Zoom). While we are coordinating logistics with the SDS Congress organizers, the two events are independent of each other. Registrations don’t carry over from one to the other.
We look forward to hearing from you about how you would like to contribute to the EL-PFDD meeting to elevate the patient voice and use it to drive change and improve therapeutic options.
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