PARDAC Stakeholder Summit

SEPTEMBER 22, 2022

Pennsylvania Rare Disease Advisory Council Stakeholder Summit

Special Thank You to Our Sponsors

Presenting Sponsor

Rare Sponsor

Thank You For Our Continued Support Of Our Council

Agenda

10:00 AM - STAKEHOLDER SUMMIT REGISTRATION

10:30 AM – PLENARY SESSION ONE

Topic: Gene Therapy
Panel:

Patrick Collins, CSL Behring

Patrick Collins
Sr. Director for Healthcare Policy and External Affairs at CSL Behring

Patrick is currently the Sr. Director for Healthcare Policy and External Affairs at CSL Behring, based in King of Prussia, Pennsylvania. CSL Behring is a manufacturer of protein replacement therapies derived from human plasma and their analogs therapies for the treatment of rare diseases. Patrick was responsible for building CSL Behring’s U.S. government affairs program and has been with the company for 21 years in positions of growing public policy responsibility.

Currently, Patrick also serves as a member of the Pennsylvania Rare Disease Advisory Council, having been appointed by the Secretary of Health.

Prior to joining CSL Behring, Patrick served as Director of Government Relations for the National Hemophilia Foundation (1997-2001) where he oversaw their government affairs activities. Chief accomplishments during this time included a principal role in the congressional passage and funding of the $750 million Ricky Ray Hemophilia Relief Trust Fund and the creation of the Patient Notification System.

A native of New York City, Patrick served earlier in his career as a liaison to the New York City Council for a city agency. He also served on the staff of a former Chairman of the New York State Senate Insurance Committee.

Patrick holds a B.A in Government and Politics and an M.A in Public Policy from St. John’s University in Queens, New York. He currently lives in Exton, Pennsylvania with his wife and daughter.

Brett Logan, Alliance for Regenerative Medicine

Brett Logan
Director, U.S. Market Access & State Government Relations, Alliance for Regenerative Medicine (ARM)

Brett Logan is the Director of U.S. Market Access & State Government Relations at the Alliance for Regenerative Medicine (ARM). Brett has 15+ years of experience working in policy and advocacy, specializing in healthcare payment policy.

As the lead of ARM’s U.S. market access portfolio, he leverages his experience to advance patient access by shaping public policy. In this capacity, he manages ARM’s U.S. Market Access & Value Committee which is focused primarily on coverage and reimbursement of cell and gene therapies.

Prior to ARM, Brett worked at the Centers for Medicare and Medicaid Services (CMS) where he served as a senior adviser to the Principal Deputy Administrator and Director of the Center of Medicare. In this position, he advised on policy development and strategy, managed COVID-19- related Medicare workflows, and collaborated with senior leadership on developing payment rules and regulations. Before that, he represented United Healthcare (UHC) where he focused on advocacy strategy, policy development, and engagement with key stakeholders such as CMS.

Early in his career, Brett served as a legislative advisor for a ranking member of the U.S. Senate, where he specialized in healthcare and appropriations. Brett graduated from Union University with a Bachelor of Arts in Political Science and English. He currently resides in the D.C. Metropolitan area.

Krupa Sivamurthy, CSL Behring

Krupa Sivamurthy
Sr Director, Global Medical Lead, Hematology, CSL Behring

Krupa Sivamurthy is a senior pharmaceutical professional, with more than a decade of experience in Medical Affairs. She is a Pediatric Hematologist Oncologist, with extensive clinical experience, both in the US and in India.

Krupa started her career as Pediatrician in India, working in a large tertiary care center in Bangalore. Her interest in Hematology lead her to pursue training as a Registrar at Christian Medical College, Vellore – one of the premier institutions for Clinical Hematology in south-east Asia.

Following relocation to the United States in 2001, Krupa pursued a residency in Pediatrics at Albert Einstein Medical Center, Philadelphia. A fellowship in Pediatric Hematology Oncology at St Christopher’s Hospital followed this.

Krupa continued at St Christopher’s Hospital as an Attending in Hematology, focused on clinical care of children with Hemophilia, Sickle Cell Disease and other disorders.

Krupa then joined Pfizer Rare Disease as Global Medical Director where she was in charge of Global Medical Affairs for Hematology. After a decade at Pfizer, she joined CSL Behring as Therapeutic Area Medical Lead where she is responsible for creating and overseeing execution of Medical Affairs strategy, including publications, congress presence, physician & patient engagement.

Krupa received her MD from MS Ramaiah Medical College, India and completed her Ped HemeOnc Fellowship from St Christopher’s Hospital, Philadelphia.

Dan Price, Patient

11:30 AM - STAKEHOLDER SUMMIT LUNCHEON REGISTRATION

12:00 PM – PARDAC LUNCHEON

Unveiling of the FOR THE FIRST TIME the Patient Needs Assessment Survey Themes
Topic: Panel Discussion
Panel:

William C. Welch, MD, FACS, FICS

William C. Welch, MD, FACS, FICS
Chair, Department of Neurosurgery, Pennsylvania Hospital

Dr. Welch is the Frederick A. Simeone Professor of Neurosurgery at the University of Pennsylvania Perelman School of Medicine. His primary appointment is in the Department of Neurosurgery, where he serves as Vice Chair- Academic Affairs. He is the Chair of Neurosurgery at Pennsylvania Hospital ("Nations First Hospital"). His clinical practice is in spinal surgery, and he routinely performs surgeries on patients with rare diseases from the Commonwealth and throughout the nation.

April Adley, DNP, MHA, RN, CNML, NEA-BC

April Adley, DNP, MHA, RN, CNML, NEA-BC
Vice President of Nursing Services for the Children’s Hospital and Women Health at Penn State Health Milton S. Hershey Medical Center

April Adley, DNP, MHA, RN, CNML, NEA-BC, currently serves as the Vice President of Nursing Services for the Children's Hospital and Women Health at Penn State Health Milton S. Hershey Medical Center. Currently, she is accountable for 196 inpatient beds and nearly 600 nursing staff, overseeing clinical, operational and financial leadership for nursing. April received her Bachelors in Nursing from Bloomsburg University in 2004, her Masters in Health Care Administration in 2011 from Walden University, and her Doctorate in Nursing Practice in 2020 from the University of Pittsburgh. She holds two certifications in leadership and was named the Central Penn Women of Influence in 2019. She is a dynamic and motivated nursing executive with a proven record of generating and building relationships, managing projects from concept to completion, and coaching individuals to success. Serving in various healthcare leadership roles over the last 10 years, she is skilled in building cross-functional teams, demonstrating exceptional communication skills, and making critical decisions during challenging times. April is an adaptable and transformational leader who can work in inter-professional settings, creating highly effective teams, focusing on employee wellness and developing opportunities that further enhance an organization's goals, mission, vision, and values.

Marie Conley, Principle, Conley Consulting

Marie Conley
Principle, Conley Consulting

Marie Conley is the founder of The Conley Cushing’s Disease Fund, which was established on July 17, 2014, and is a project of The Foundation for Enhancing Communities, fiscal sponsor. The funds raised are used in part to create awareness, advocacy and support for patients with this disease and their loved ones, as well as support institutions and organizations focused on research and treatment surrounding Cushing’s Disease. In 2012, Marie was diagnosed with Cushing’s Disease – a disease so rare it affects less than 10 people per million each year. She is also adrenal insufficient. She is a consultant focusing on engagement and stakeholder strategies for a variety of clients through her company Conley Consulting, LLC. Marie hails from Bucks County, Pennsylvania, and lives in Elizabethtown, Pennsylvania, with her husband, Chris Lammando, and their son Carter.

2:00 PM – PLENARY SESSION TWO

Topic: Newborn Screening
Panel:

Jodie Vento, MGC, CGC

Jodie Vento, MGC, CGC
Assistant Professor of Human Genetics and the Genetic Counseling Training Program Director
at the University of Pittsburgh

Jodie M. Vento, is currently an Assistant Professor of Human Genetics and the Genetic Counseling Training Program Director at the University of Pittsburgh. After completing undergraduate degrees in biology and psychology at the University of Pittsburgh in 2005, Jodie earned a Masters in Genetic Counseling from the University of Maryland Medical School in Baltimore in 2008. She worked as a genetic counselor and neurogenetics program coordinator at Children’s National Medical Center in Washington, DC, before joining Children’s Hospital of Pittsburgh in 2012. At UPMC Children’s Hospital of Pittsburgh, Jodie managed an innovative program called the Center for Rare Disease Therapy. In her role at Children’s Hospital, Jodie worked extensively with payers to develop shared processes and resources to increase the coverage and reimbursement of genetic testing. Jodie is the Insurance Alignment Committee Chair for the Patient-Centered Laboratory Guidance Services (PLUGS) Group out of Seattle Children’s Hospital. Jodie is also an active member of NSGC and is currently on the Board of Directors. In her role at NSGC, she has served as the chair of the Access and Service Delivery committee and the co-chair of the Test Utilization Subcommittee within the Industry SIG. Jodie is also the Founder and Past President of the Pennsylvania Association of Genetic Counselors. Jodie serves as the Genetics Faculty for the Leadership Education in Neurodevelopmental Disabilities and Related Disorders (LEND) Program at the University of Pittsburgh. Throughout her career, Jodie has worked closely with rare disease community and will continue this work through the Pennsylvania Rare Disease Advisory Council (PARDAC).

Dr. Laurie Varlotta, MD

Dr. Laurie Varlotta, MD
Chairperson, NBS Advisory Board Pediatric Pulmonologist; Director, Cystic Fibrosis Care Center;
Director, Pediatric Pulmonology Fellowship Program, St. Christopher Hospital for Children; Professor, Department of Pediatrics Drexel University College of Medicine

Laurie Varlotta is a board certified physician in Pediatrics and Pediatric Pulmonology. She has been an Attending Physician in Pediatric Pulmonology at St. Christopher’s Hospital for Children, Philadelphia, PA since 1992 and is also Professor of Pediatrics at Drexel University College of Medicine. Since 1999 Dr. Varlotta has been the Director of the Cystic Fibrosis (CF) Center at St. Christopher’s Hospital for Children. St. Christopher’s Hospital CF Center had been designated as a Therapeutics Development Center (TDC) for research. Dr. Varlotta has been Co-Director of the TDC at St. Christopher’s from 2007 until 2018. Dr. Varlotta is involved in a number of clinical research studies, including multi-center trials involving individuals with CF. Dr. Varlotta has lectured nationally and internationally.

Dr. Varlotta received a BA in Chemistry at Emory University and her medical degree at the State University of New York Downstate Medical School in Brooklyn, NY. Dr. Varlotta did her Pediatric Residency at Einstein/ Montefiore Hospital in New York and was Chief Resident in Pediatrics at Bronx Lebanon Hospital in New York. She did her Fellowship in Pediatric Pulmonology at Children’s Hospital of Philadelphia.

Dr. Varlotta is currently a Member of the Quality Improvement Consortium for the Cystic Fibrosis Foundation Newborn Screening Group, CFF National Newborn Screening Quality Initiative (SIP) Consortium as well as a member of the Categorical Diagnosis in Newborn Screening in Cystic Fibrosis US Department of Health and Human Services, Health Resources and Services Administration (HRSA). Regionally, she is a member and Chair of the Commonwealth of Pennsylvania Technical Advisory Board for Newborn Screening. She is a Board Member of the Delaware Valley Cystic Fibrosis Foundation Chapter. She is founder and Co-Chair of the Mid- Atlantic Cystic Fibrosis Consortium and the Cystic Fibrosis Mid-Atlantic Research Study Group. She has been named in Philadelphia magazine Top Doctors™ 2017, 2018, 2019, 2020, 2021 and 2022- Pediatric Pulmonology.

Nicole Engelhardt, MS LCGC

Nicole Engelhardt, MS LCGC
Genetic Counselor, Children Hospital of Philadelphia

Nicole is a Genetic Counselor who trained at Arcadia University. She has been working in biochemical genetics at the Children's Hospital of Philadelphia since 2019 where she focuses on helping parents handle newborn screening results and guides genetic testing for undiagnosed children. Her work also includes two specialty clinics: Williams Syndrome and Congenital Disorders of Glycosylation. Nicole has been involved in research for rare diseases, including case reports for new disorders like MDH2 and expanding the phenotype of known disorders such as Fabry disease and ALG3-CDG. She also helped obtain compassionate use for an orphan drug for children with Niemann-Pick type B while FDA approval was pending. Nicole believes that everyone should have the ability to understand the nuances of their medical information and make decisions that work best for their family.

Claire Ellis, Everylife Foundation

Claire Ellis
Policy Fellow, Everylife Foundation

Claire Ellis is the Policy Fellow with the EveryLife Foundation for Rare Diseases. Claire advocates for newborn screening legislation at the state and federal level, creates advocacy resources and tools to facilitate patient and organization engagement within the newborn screening ecosystem, and assists in the management of the Foundation’s Community Congress Newborn Screening working group. Previously Claire worked with the Indiana House of Representatives as a Legislative & Constituent Services Intern and with Zimmer Biomet as a Government Affairs Intern. Claire graduated from the O’Neill School of Public and Environmental Affairs at Indiana University with a degree in Law & Public Policy and a concentration in Healthcare Policy.

Lesa Brackbill

Lesa Brackbill is a wife, mother, author, and advocate.

She and her husband Brennan became involved with Krabbe-related advocacy when their daughter Victoria was diagnosed with Krabbe Disease in 2015. Using her knowledge and experience, Lesa helped to reform the Newborn Screening (NBS) program in Pennsylvania, making the program stronger; as a result of her efforts, Pennsylvania began screening for Krabbe in 2021 (Act 133 of 2020). After successfully seeing Act 133 of 2020 enacted, Lesa guided a group of passionate moms to success with Act 29 of 2022 (related to cCMV screening), which was signed in June 2022.

Lesa is the Outreach Coordinator for Policy and Advocacy for the Leukodystrophy Newborn Screening Action Network (LDNBS.org) and is working toward building a coalition with all interested parties as they work toward a world where every baby is screened equally for all possible leukodystrophies.

Lesa serves on the Board of Directors for KrabbeConnect and is the co-chair of the Krabbe Stakeholders Group – a collaboration between advocacy, industry, and clinicians seeking to improve many aspects of life with Krabbe.

She is an ambassador for the Family Education program of Expecting Health, and the parent advocate on the LSD Subcommittee of the Pennsylvania Newborn Screening Technical Advisory Board. Additionally, she is on the State Policy Committee for Life Sciences PA and a member of the EveryLife Foundation’s Public Policy and NBS Community Congresses.

Lesa is the author of Even So, Joy: Our Journey Through Heartbreak, Hope, and Triumph which chronicles her family’s journey with Krabbe Disease and finding hope and joy in the journey. She blogs regularly and is also a co-author on two journal articles.

In her “free time” Lesa is a tour guide at High Point – the home of Milton S. Hershey and a weekend manager at The Hershey Story. Most importantly, she is raising identical twin boys, Isaiah and Caleb with her husband, Brennan, in Hershey, PA.

Lesa is originally from Red Bluff, California and moved to Pennsylvania in 2008 to pursue a career in lobbying. Lesa holds a Bachelor of Arts in Political Science and is currently working toward a Master of Arts in Strategic Communication, both from Azusa Pacific University.

SPEAKERS

PA Rare Disease Caucus Co-Chairs
Sen. Judy Ward
Rep. Mark Longietti
Sen. Maria Collett
Rep. Barbara Gleim
Acting Sec. Meg Snead, DHS

PARDAC Stakeholder Summit

SEPTEMBER 22, 2022

Pennsylvania Rare Disease Advisory Council Stakeholder Summit

Special Thank You to Our Sponsors

Presenting Sponsor

Rare Sponsor

Thank You For Our Continued Support Of Our Council

Agenda

10:00 AM - STAKEHOLDER SUMMIT REGISTRATION

10:30 AM – PLENARY SESSION ONE

11:30 AM - STAKEHOLDER SUMMIT LUNCHEON REGISTRATION

12:00 PM – PARDAC LUNCHEON

2:00 PM – PLENARY SESSION TWO

SPEAKERS

Improving the lives of Pennsylvanianswho live with a rare disease

Improving the lives of Pennsylvanians
who live with a rare disease