5th Expanded Access Programmes World Congress 2023 Americas

5th Expanded Access Progammes World Congress 2023 Americas

Strategies and Considerations in Launching an Expanded Access Programmes

Revere Hotel Boston Common, Boston, MA, USA

Thursday 16th - Friday 17th November 2023

KEY INDUSTRY EXPERT SPEAKERS 2023

Jennifer M. Joe, Global Medical Strategy & Population Health Director, AstraZeneca
Norah Xiao, Director, Digital Health Partnership, Astrazeneca
Bhaskar Dutta, Head, Digital Health & Medical Affairs Technologies, Alexion
Isabelle Lousada, CEO and President, Amyloidosis Research Consortium
Graham Sidorowicz, Executive Vice President Commercial , Bionical Emas
Siddharth Jain, Medical Director –LSDs & HAE, Global Medical Affairs, Takeda
Donna Cowan, Associate Director, EAP & Registry, Stealth Biotherapeutics*
Amber Bifolck Fisher, Clinical Research Manager, Kura Oncology, Inc.
Alan J. Balch, Chief Executive Officer, Patient Advocate Foundation
Rachel Harisson, Associate Director, Early Access Program, Apellis Pharmaceuticals
Monica L Weldon, President/CEO/Founder, SYNGAP1 Foundation
Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech Evaluation, Independence Blue Cross
Richard Klein, Director, EAP & Policy, GE2P2 Global Foundation
Jennifer E. Miller, PhD, Associate Professor , Yale School of Medicine
Dawn Sullivan, L.E., Founder/CEO, Sullivan Associates, LLC
Emily M. Parks, Founder, CEO, POP!
Christine Von Raesfeld, Founder & CEO, People with Empathy
Annmarie Galli, Head, Global Medical Affairs Research and Compassionate Use / ManagedAccess Programs Oncology, GSK
Wyndi Phillips, Vice President, Project Management, WEP Clinical
Chisa Nosamiefan, Co-founder, The Labalaba Foundation for Lupus
Roberta Smith, President , Alagille Syndrome Alliance
Robert Donnell, EVP - Medicines Access - Smartway Pharmaceuticals
Lee Sutton, Vice President, Business Development, Expanded Access Programs, WEP Clinical

Graham Sidorowicz

EVP Commercial

Bionical Emas

Graham Sidorowicz

EVP Commercial

Graham is an experienced senior leader with extensive experience in commercial, market access and medical functions working in small and large pharmaceutical organizations. He has worked implementing EAPs, both for pharmaceutical companies directly and through a provider organization. During his time working in Medical Affairs, Graham led multiple Real-World Data collection projects and has published data on prospective observational studies and a global registry. Graham currently leads commercial activities across the business and is responsible for external partnerships.

JENNIFER M. JOE

Global Medical Strategy & Population Health Director

AstraZeneca

JENNIFER M. JOE

Global Medical Strategy & Population Health Director

Jennifer Joe MD is the Population Health Director within global medical affairs, where she sits at the intersection of population health, digital, education, and clinical trials. Her role is disease state agnostic, and her current work is supporting the amyloidosis teams. She believes in putting the voice of the patient at the center of every discussion. Trained in nephrology at the Harvard-affiliated Mass General and Brigham and Women’s Hospital, she currently practices part-time at the Boston VA healthcare center.

DONNA COWAN

Associate Director, Expanded Access Programs

Stealth BioTherapeutics

DONNA COWAN

Associate Director, Expanded Access Programs

Donna Cowan is a Certified Clinical Research Coordinator/Project Manager with more than 20 years of biopharmaceutical experience. For the past several years Donna has concentrated on the management of Investigator Initiated Studies and Expanded Access Programs (Compassionate Use, Managed Access, Named Patient), leveraging her passion for patient care into providing early access to investigational therapies to those with serious or life-threatening conditions who may have no other treatment options. She has experience in various therapeutic areas including oncology, rare and orphan disease, endocrinology, neuroendocrinology, and infectious disease, participating in several successfully executed NDA applications in these areas. Ms. Cowan is the Associate Director of the Expanded Access Programs and Investigator Initiated Studies at Stealth BioTherapeutics (SBT), a Boston area-based biopharmaceutical company developing therapies to treat the mitochondrial dysfunction associated with genetic mitochondrial diseases and many common age-related diseases.

Norah Xiao

Director, Digital Health Partnership

Astrazeneca

Norah Xiao

Director, Digital Health Partnership

Norah Xiao is the Director of Digital Health Partnership in Global Commercial Digital Health at AstraZeneca. As a digital product strategist, patients’ needs are her northstar. She leads the development of global commercial digital health strategy with Global Medical Affair and Global Brand and the implementation with marketing companies by building partnerships with external ecosystems. Amyloidosis is the rare disease she is currently working on.

EMILY M. PARKS

Founder, CEO

POP!

EMILY M. PARKS

Founder, CEO

Emily Parks is a young professional based out of the Washington D.C. area working in behavioral health and patient advocacy and student of Johns Hopkins University Carey School of Business pursuing an MBA in Healthcare Management. Emily received a small bowel transplant recipient in 2020 from MedStar Georgetown Transplant Institute after 20 years on TPN. Combining her expertise in behavioral health with her lived experience of navigating multiple tertiary hospitals firsthand has led her down the path of studying the impact of medical trauma and medical PTSD on patients and its influence on treatment outcomes. Emily founded the organization POP!, an organization dedicated to raising awareness of medical PTSD and medical trauma. In partnership with Stanford University School of Medicine and University of Washington- Seattle School of Medicine, Emily is part of a team constructing a new curriculum for budding anesthesiologists to use trauma-informed care to increase emotional intelligence in providers and decrease further retraumatization for patients living with anesthesiology related Medical PTSD. Emily is coordinating development of a new podcast in which anonymous letters from both patients and providers about their negative interactions with one another. Then, with the support of industry experts, the podcast host will examine both the facts and subjective emotions from either side to determine the tools needed to help people think and/or act differently in similar situations going forward. Lastly, she is working on a publication detailing what Medical PTSD is,common responses and/or protocols patients face in the healthcare system that leads to undermining feelings of patient empowerment, and how providers can respond to patients with Medial PTSD to promote true patient-centered care. In her free time, Emily enjoys cuddling with her cats, Sassy and Mickey, watching true crime documentaries, and sleeping. Site: popmedicalptsd.org LinkedIn: https://www.linkedin.com/in/emilyprk/

SIDDHART JAIN

Medical Director, Global Medical Lead

Takeda

SIDDHART JAIN

Medical Director, Global Medical Lead

Siddharth is an accomplished Medical Affairs leader (14+ years) with a diverse, multicultural experience in reputed organizations with successful stints in varied therapies (including Rare diseases). His strengths are collaborating with stakeholders from varied cultural & ethnic diversities & different work environments, reputation for agility to adapt towards rapidly evolving ecosystem, quickly earning confidence of key stakeholders and establishing a positive, growth-minded working culture by providing ongoing mentoring and support for colleagues. He has conceptualized and executed various digital, omnichannel & medical excellence projects, partnering with external societies/ patient advocacy groups, market access & leading large multi-country RWE studies and driving publications.

EUGEAN JIWANMALL

Senior Research Analyst for Medical Policy & Technology Evaluation

Independence Blue Cross

EUGEAN JIWANMALL

Senior Research Analyst for Medical Policy & Technology Evaluation

Eugean Jiwanmall’s past professional experiences include basic science and clinical research. In his current role as a Senior Research Analyst for Medical Policy & Technology Evaluation at Independence Blue Cross’ Medical & Claim Payment Policy Department within Facilitated Health Networks and Medical Affairs, he has served as the subject-matter-expert, writer, reviewer, communicator, presenter, consultant, adviser/facilitator, and decision-maker for hundreds of medical policy topics and technology assessments within dozens of clinical disciplines and multiple business areas. Eugean has participated in numerous roles & capacities in a number of conferences, summits, and other national & international events etc. upon invitations. He has been invited by renowned organizations, evaluator bodies, & thinktanks etc. to be key informant on a variety of complex and challenging topics, and recognized in communications & publications for these works. Eugean has taken and completed graduate coursework in bioethics at the University of Pennsylvania School of Medicine. He holds a bachelor’s degree in biological sciences with honors from Drexel University (minor was in business administration), a master’s degree of public health in evaluative clinical sciences from Dartmouth Medical School, and an MBA in healthcare management and economics with honors from Drexel University.

AMBER BIFOLCK FISHER

Clinical Research Manager

Kura Oncology, Inc.

AMBER BIFOLCK FISHER

Clinical Research Manager

Amber Bifolck Fisher has been supporting Oncology clinical trials since 2014. She entered the field at the Massachusetts General Hospital, where she supported Lymphoma and Multiple Myeloma trials and then provided department training as the Clinical Research Education Manager. In 2022 she joined Kura Oncology as a Clinical Trial Associate, and transitioned to the role of Clinical Research Manager. She presently manages operations for the Compassionate Use and Investigator Sponsor Studies programs. She is also enrolled in a MS program at Brandeis University, with anticipated graduation in December 2023.

MONICA L WELDON

President/CEO/Founder Bridge the Gap

SYNGAP Education and Research Foundation

MONICA L WELDON

President/CEO/Founder Bridge the Gap

In November of 2012, Monica’s twin son, Beckett, was the first to be diagnosed at Texas Children’s Genetics Clinic with the gene mutation SYNGAP1 (6p21.3). When Beckett was 4 months old, she noticed he was not meeting the same milestones as his twin sister. Monica then began a journey to find answers to help her son. She started to blog about his progress and this led to building a community of parents and caregivers that are now a strong support group. She is the Founder and President/CEO of Bridge the Gap – SYNGAP Education and Research Foundation. It is her passion to help support these families by raising awareness and creating a strong foundation that will accelerate a path to better therapies. She retired in 2016 after 23 years in education teaching secondary science. Her new focus is on building the programs and mission of Bridge the Gap – SYNGAP Education and Research Foundation. She is the Primary Investigator on the SYNGAP1 (MRD5) Registry and Natural History Study. She is a life member of the Worldwide Association of Female Professionals and a member of the first class of 2017 Illumina Ambassadors established in the United States. In addition to leading the foundation, she is an author, public speaker, consultant on rare disease business strategies and advocates for rare disease legislation at both the federal and state levels. Several of her authored scientific publications include Nature Neuroscience, The Journal of Neurodevelopmental Disorders, and The Journal of Pediatrics. She has authored a book about her son Beckett’s diagnostic journey called “Slow Moving Stream – My Special Boy”. She is a graduate from East Texas Baptist University with a Bachelor’s of Science in Biology/Psychology (1991) and Secondary Certification in Education (1995). She will be attending Northwestern University Pritzker School of Law in the Fall of 2020 to earn her Masters in Science Law. She has five beautiful children, Haleigh (28), Taylor, USMC (25), Sawyer (23), and the twins Beckett & Pyper (12). She was a Global Genes 2015 & 2016 RARE Champion in Advocacy Award nominee for her work in the rare disease community and WeGo Health Awards Nominee for years 2015, 2017, 2018, 2019, & 2020. She was named 2020 Global Shakers Rare Disease Champion.

RACHEL HARISSON

Associate Director, Early Access Program

Apellis Pharmaceuticals

RACHEL HARISSON

Associate Director, Early Access Program

Early Access Program (EAP) Professional and an emerging leader in the EAP field who lived and worked overseas for 11 years. Committed to connecting individuals, motivating cross-functional teams and the pursuit of breakthroughs in medical care frontiers. Dedicated to collaborating and supporting peers and managers, have earned the trust and admiration of professionals both in America and abroad. In addition, am devoted to supporting the research, innovation and delivery of medical treatment to help professional stakeholders and patients around the world.

Isabelle Lousada

CEO and President

Amyloidosis Research Consortium (ARC)

Isabelle Lousada

CEO and President

Isabelle Lousada is Founder and CEO of the Amyloidosis Research Consortium (ARC). She is an experienced nonprofit director and pioneer in patient advocacy in the rare disease field. She has been the driving force behind ARC, building successful collaborations and funded research programs across the sectors to advance the science and understanding of the amyloidosis diseases. She was diagnosed with AL amyloidosis, and was one of the first patients to successfully undergo a stem cell transplant. For the past 25 years, she has worked to bring patients, academia, regulators and industry together to develop partnerships, promote awareness, and address the critical unmet needs. She has been committed to empowering patients, increasing access, and improving care. Her work has led to positive outcomes for patients, families, and the wider medical community.

Alan J. Balch

Chief Executive Officer

Patient Advocate Foundation

Alan J. Balch

Chief Executive Officer

Dr. Balch has over fifteen years of executive leadership in the non-profit sector with an emphasis on consensus-building and collaboration. He has led numerous federal advocacy efforts on a range of issues both at the legislative and regulatory level. He became the CEO of both NPAF and PAF in 2013. From 2006 - 2013, he served as the Vice President of the Preventive Health Partnership -- a national health promotion collaboration between the American Cancer Society, American Diabetes Association, and American Heart Association. Prior to 2006 Dr. Balch was the Executive Director of Friends of Cancer Research. Dr. Balch has served on the Executive Board of the Patient Advocate Foundation and National Patient Advocate Foundation since 2007. He also serves on numerous advisory boards and committees. He earned his PhD in environmental studies with a concentration in political economy in 2003 from the University of California, Santa Cruz; master’s degree in environmental sciences in 1997 from the University of Texas in San Antonio; his bachelor’s degree (cum laude) in biology in 1994 from Trinity University in San Antonio.

Wyndi Phillips

Vice President, Project Management

WEP Clinical

Wyndi Phillips

Vice President, Project Management

Wyndi is responsible for overseeing and leading the WEP Clinical global Project Management team, ensuring strong customer service and timely delivery of medications to patients around the world through Expanded Access and Post-Approval Named Patient Programs. Previously, she successfully led global teams in the management of clinical drug supply as Executive Director of Clinical Supplies at PRA Health Sciences and as a Project Group Manager at Almac Clinical Services. Wyndi earned her Doctor of Pharmacy degree from Campbell University and completed a residency at Mercer University. She has worked as a clinical pharmacist in hospital, ambulatory care, and community settings.

CHRISTINE VON RAESFELD

Founder & CEO

People with Empathy

CHRISTINE VON RAESFELD

Founder & CEO

As Founder/CEO of People with Empathy, Christine Von Raesfeld is a leader in bringing a critically needed patient perspective to cutting-edge medical innovations. Committed to providing patients with chronic and rare diseases with the support they need, Christine works with patient advocacy organizations, industry representatives, and individual patients and their loved ones. Living with many rare and chronic diseases, she believes that the only way to ensure health equity is to leverage one’s own data. As a patient involved in Stanford’s Humanwide program, she speaks on the benefits and advantages of precision medicine, with a special interest in pharmacogenomics. Christine’s drive to make lives better for patients has been nationally recognized. In 2019, she spoke on stage at the Startup Health Festival as an invited guest of Sanguine Biosciences. In 2018, Christine served on the Team of Patient Advisors for PatientsLikeMe. She has been nationally recognized for her work in advocacy and when several awards including Silicon Valley Business Journal’s100 Women of Influence, 2019 and a 2020 Community Hero by Assemblyman Kansen Chu. In addition, Christine serves as a Virtual Advisor Team member for the All of Us research program, a Technical Expert Panelists for CMS and a patient advisor/consultant or multiple pharmaceuticals off branded campaigns and startup ventures.

Bhaskar Dutta

Head, Digital Health & Medical Affairs Technologies

Alexion

Bhaskar Dutta

Head, Digital Health & Medical Affairs Technologies

Chisa Nosamiefan

Co-founder

The Labalaba Foundation for Lupus

Chisa Nosamiefan

Co-founder

Chisa Nosamiefan, a co-founder of The Labalaba Foundation for Lupus, is a global patient leader dedicated to raising awareness, advocating for improved healthcare, and supporting individuals affected by this autoimmune disease. She is currently co-leading Minority Patient X, a patient experience initiative that is focused on understanding and improving minority patients’ experience of healthcare, by ensuring a better understanding and thoughtful incorporation of unmet needs, language and culture impacts, and patient preferences. Chisa serves as a member of the Patient Advisory Board of the Brigham and Women's Hospital Lupus Center in Boston, Massachusetts, contributing her patient perspective to shape research, treatment protocols, and policies. Her unique insights and experiences help bridge the gap between patients and the healthcare system, medical professionals, and industry to drive inclusive patient-centered care. Chisa is also a member of The Light Collective Advisory Board, further expanding her impact in the patient digital and health data space. She advances the issues pertaining to collective rights, interests, and voices of patient communities in health tech. With her personal experience and dedication to improving healthcare outcomes, Chisa strives to make a positive difference in the lives of people affected by rare and chronic diseases and promote a more inclusive and patient-centric healthcare system.

Annmarie Galli

Head, Global Medical Affairs Research and Compassionate Use / Managed Access Programs Oncology

GSK

Annmarie Galli

Head, Global Medical Affairs Research and Compassionate Use / Managed Access Programs Oncology

Roberta Smith

President

Alagille Syndrome Alliance

Roberta Smith

President

Roberta has been with ALGSA since 2013, her work in advocacy inspired by her twin daughters, one having Alagille Syndrome. Roberta has worked closely with industry through the drug development of now two FDA approved drugs for Alagille Syndrome. This has included intricate involvement in expanded access and patient recruitment, elevating the patient voice to improve access and understanding of the patient experience.

Robert Donnell

Executive Vice President, Medicines Access

Smartway Pharmaceuticals

Robert Donnell

Executive Vice President, Medicines Access

Robert has designed and led some of the most innovative and largest EAPs in recent years including in rare diseases and gene therapy. He has experience across Europe, USA and globally and has advised major blue-chip Pharma as well as smaller biotech companies. He started his career at P&G and AZ before moving to a number of international service providers working with pharma and biotech companies on supply chain solutions. He currently leads all global development and implementation of EAPs at Smartway where he specializes in working with manufacturers to develop the best Medicines Access plan for them.

Lee Sutton

Vice President, Business Development, EAP

WEP Clinical

Lee Sutton

Vice President, Business Development, EAP

Lee has been in the pharmaceutical & logistics industry for over 10 years. He joined WEP Clinical in 2020, after spending 7 years with Thermo Fisher Scientific where he led global commercial operations within a supply chain business unit. In his current role, Lee is responsible for driving the strategy & growth of WEP’s Expanded Access and Post-Approval Named Patient Programs. Lee is a highly experienced, results-oriented global business development leader who possesses strong strategic capabilities and knowledge of managing clinical and commercial supply chains. His innovative thinking and straightforward business approach drives value and exceptional service to our clients and their patients.

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