WASHINGTON, May 16, 2024
/PRNewswire/ -- The EveryLife Foundation for Rare Diseases is
excited to announce its Scientific Workshop slated for May 21, 2024, in Washington, D.C., with both virtual and
in-person attendance options.
This year, the workshop emphasizes identifying and
characterizing the challenges in developing therapies for
conditions affecting exceedingly small populations, often termed
'ultra-rare' diseases. While significant strides have been made
with legislative tools like the Orphan Drug Act, only a small
fraction of rare diseases have FDA-approved treatments.
In response to the ongoing debate within the community regarding
the necessity and potential framework of a formal definition for
ultra-rare diseases, the EveryLife Foundation aims to ensure that
any conclusions drawn are well-informed by evidence and community
needs, while also considering the potential for unintended
consequences.
The workshop aims to explore the unique hurdles in therapy
development, regulatory processes, and funding, aiming to delineate
the considerations and implications of establishing a statutory
definition for ultra-rare diseases. The event will also evaluate
the current state of therapy development to document existing
disparities and pinpoint opportunities for targeted policy
changes.
"We are proud to convene leading experts from industry,
academia, policy, and patient organizations who are pioneers in
ultra-rare disease research and advocacy," said Michael Pearlmutter, CEO at the EveryLife
Foundation. "Our workshop will drive crucial discussions that
inform and refine policy and regulatory frameworks, ensuring that
any new initiatives enhance rather than segment the rare disease
therapy development landscape."
Confirmed Speakers:
Federal Agencies
- Peter Marks, MD, PhD –
Director, Center for Biologics Evaluation and Research (CBER),
FDA
- Janet Maynard, MD, MHS – Director, Office of Rare
Diseases, Pediatrics, Urologic and Reproductive Medicines, FDA
- Joni Rutter, PhD – Director,
National Center for Advancing Translational Sciences (NCATS),
NIH
- Janet Woodcock, MD – Former
Principal Deputy Commissioner, FDA
Think Tank Innovators
- Kishore Hari – Senior Program
Manager, Chan Zuckerberg Initiative
- Esther Krofah, MPP – Executive Vice President, Milken
Institute Health
- Ethan Perlstein, PhD – CEO, Perlara PBC & Maggie's
Pearl
Patient Advocacy Experts
- Lynn Albizo, JD – Chief Public Policy Officer, Immune
Deficiency Foundation
- Matthew Ellinwood, DVM, PhD – Chief Scientific Officer,
National MPS Society
- Justin Hopkin, MD – Chairman Emeritus, National
Niemann-Pick Disease Foundation
- Annie Kennedy – Chief of Policy,
Advocacy, and Patient Engagement, EveryLife Foundation for
Rare Diseases
- Sharon King – President,
Taylor's Tale
- Paul Melmeyer, MPP – Vice President, Muscular Dystrophy
Association
- Emily Milligan, MPH – Executive Director, Barth Syndrome
Foundation
- Jamie Sullivan, MPH – Senior
Director, Advocacy, and Patient Engagement, EveryLife
Foundation for Rare Diseases
Academic and Regulatory Experts
- A.J. Allen, MD, PhD – Chief
Medical Officer, IACT
- Tim Franson, MD – Principal, Faegre Drinker
Consulting
- Aaron Goldenberg, PhD, MPH – Professor and Vice Chair,
Department of Bioethics, Director, Center for Community Health and
Genomics, Case Western Reserve
University School of Medicine
- Frank Sasinowski, MS, MPH, JD – Director, Hyman, Phelps,
& McNamara P.C.; Vice Chair of the Board, EveryLife Foundation
for Rare Diseases
Pharmaceutical Industry and Investment Experts
- Teresa Fecteau, PhD – Director, U.S. Medical for Rare
Disease, Biogen
- Reenie McCarthy, JD – Chief
Executive Officer, Stealth Biotherapeutics
- Adora Ndu, PharmD, JD – Chief
Regulatory Officer, BridgeBio
- Sal Rais, MD, MBA – Biotech
Analyst, T. Rowe Price
- Kevin Thorneloe, PhD – Sr. Medical Director, Pharming
The workshop will serve as a platform for in-depth discussions,
allowing attendees to network with industry leaders and contribute
to the shaping of the future of ultra-rare disease therapy
development.
Registration and Media Inquiries:
The workshop is accessible to all, with no-cost registration
options for both virtual and in-person participation. Limited media
registrations are available. For more details about the workshop
and to register, please visit EveryLife Foundation's Scientific
Workshop Schedule.
About EveryLife Foundation for Rare Diseases:
EveryLife Foundation for Rare Diseases is a nonprofit,
nonpartisan organization dedicated to advancing the development of
treatment and diagnostic opportunities for rare disease patients
through science-driven public policy. The Foundation works to
improve the lives of the millions of Americans suffering from rare
diseases by advocating for policies that foster innovation and
remove barriers to life-saving treatments. Founded in 2009 and
headquartered in Washington, DC,
the EveryLife Foundation for Rare Diseases has programs throughout
the United States. To learn more,
visit EveryLifeFoundation.org.
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SOURCE EveryLife Foundation for Rare Diseases