Featuring

3rd Expanded Access Programmes Global Congress 2022 Americas

Obtaining the much needed access to therapies for lifechanging treatment

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Hours
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29th - 30th November 2022 - 2 Day Conference
Hyatt Regency Boston, Boston, MA, USA

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Venue

Hyatt Regency Boston

Address

1 Ave de Lafayette, Boston, MA 02111, United States

Phone

+1 617-912-1234

Global Congress 2022 Americas

Expanded Access Programmes

Expanded access or compassionate use is the use of an unauthorized drug or medical device under special forms of investigational new drug applications (IND) or IDE application for devices, outside of a clinical trial, by the patient with an immediately life-threatening illness or severely debilitating disease or condition who do not meet the enrollment criteria for the clinical trial in progress. These programs go under various names, including early access, special access, or managed access program, compassionate access, named-patient access, temporary authorization for use, cohort access, and pre-approval access.

When patient participation in clinical trials is not possible due to ineligibility or scarcity of ongoing trials, or if enrollment in clinical trials is not conceivable because the distance to the trial inhibits access, expanded access offers a possible route for gaining access to an investigational medical product. These are investigational drugs, biologics or medical devices that are being tested to see whether they are safe and effective. As investigational drugs have not yet received regulatory approval from FDA, the agency has not found these products to be safe or effective for their specific use. It is very important to remember that their potential risks and benefits are not yet established and their effectiveness to treat the condition is not yet known.

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Insights & Attendees

Previous Key Industry Experts

Donna Cowan

Associate Director, Expanded Access Programs Stealth BioTherapeutics

Alan J. Balch, PhD

CEO, National Patient Advocate Foundation

Robert Donnell

Executive Vice President, Medicines Access, Smartway Pharma

Neena Nizar

President & Founder, The Jansens Foundation

All Expert Speakers

From a patient advocacy organization perspective, was a very informative meeting. Learned a lot from presentations and networking that will inform how we can add value to the advocacy-patient-drug developer dialogue.

Dr. Nadine Tutton

Scientific Director, Research, Association for Frontotemporal Degeneration

I was very impressed with the format, the content was interesting and well done. I felt I learned a lot and was glad to be able to attend.

Senior Director

Business Development at Pharm-Olam, LLC

View Conference Agenda

EXPANDED ACCESS PROGRAMMES

SPONSORS

Gold Sponsor

Gold Sponsor

Gold Sponsor

Exhibitor

Expanded access or compassionate use is the use of an unauthorized drug or medical device under special forms of investigational new drug applications (IND) or IDE application for devices, outside of a clinical trial, by the patient with an immediately life-threatening illness or severely debilitating disease or condition who do not meet the enrollment criteria for the clinical trial in progress. These programs go under various names, including early access, special access, or managed access program, compassionate access, named-patient access, temporary authorization for use, cohort access, and pre-approval access.

When the patient participation in clinical trials is not possible due to ineligibility or scarcity of ongoing trials, or if enrollment in clinical trials is not conceivable because the distance to the trial inhibits access, expanded access offers a possible route for gaining access to an investigational medical product. These are investigational drugs, biologics, or medical devices that are being tested to see whether they are safe and effective. As investigational drugs have not yet received regulatory approval from FDA, the agency has not found these products to be safe or effective for their specific use. It is very important to remember that their potential risks and benefits are not yet established and their effectiveness to treat the condition is not yet known.

With patients thoroughly becoming knowledgeable, Pharmaceutical companies are bound to receive unsought requests. However, there are challenges and obstacles such as a diverse regulatory landscape, logistical differences, and complexities from planning an application to approval and implementation. Sponsors need to plan carefully before undertaking an Expanded Access Programme so that they have the resources, processes, and tools in place to support the inevitable demand.

The objective of this 2-day interactive congress is to provide the audience with much-needed skills, best practices, innovations, technologies, and concepts in obtaining the much-needed access to these therapies for life-changing treatment, led by key expert stakeholders with intimate knowledge in the industry.

We look forward to welcoming you at the Congress! 

Jocelyn Raguindin
Jocelyn Raguindin

Jocelyn is Conference Director at Paradigm Global Events (PGE). She has over 17+ years experience in organizing pharma-industry conferences and has been at the helm of every PGE conference since 2013. Jocelyn is based in London, UK.

Gain Latest Insights on:

  • The current compassionate use, or expanded access-related mechanisms
  • The benefits and issues surrounding expanded access programme
  • Understanding the varying regulatory landscape, policies and guidelines to ensure a smooth execution of the programme
  • Criteria that must be met to authorize expanded access use, requirements for expanded access submissions
  • Understanding operational complexities, best practices and strategies in running a global EAP
  • Successfully navigating logistical intricacies
  • Incorporating patient voice from early planning to ensure a patient-centric access programme
  • Engaging representatives from all stakeholder groups to understand their perspective and expectations of the programme

Who Should Attend?

  • This Congress will provide information to pharmaceutical and biotech companies, researchers, physicians, institutional review boards (IRBs), patient organisations and advocates and regulatory agencies about the implementation of expanded access to investigational drugs.
  • Network with representatives involved in the following areas:
  • Expanded Access
  • Managed Access
  • Early Access
  • Market Access
  • Medical Affairs
  • Regulatory Affairs and Policy
  • Patient Support and Patient Engagement
  • Corporate Development
  • RWE, and Data Management
  • Quality and Compliance
  • Clinical Development
  • Programme Management
  • Supply Chain Management
  • Patient Access
  • Clinical Research
  • Sourcing Logistics
  • Consulting

Donna Cowan

Associate Director, Expanded Access Programs Stealth BioTherapeutics

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.

Alan J. Balch, PhD

CEO, National Patient Advocate Foundation

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.

Robert Donnell

Executive Vice President, Medicines Access, Smartway Pharma

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.

Neena Nizar

President & Founder, The Jansens Foundation

Dr. Nizar serves as Founder and Executive Director of the Jansen’s Foundation. She is a rare disease advocate and Change Leader in the rare disease community, specifically in the field of ultra-rare skeletal dysplasias. Dr. Nizar has a Doctoral degree in Educational Leadership from Creighton University, Nebraska, is a TEDx speaker, a blogger and passionate voice for the special needs community. She is a Trailblazer for the Rare Advocacy Movement (RAM) and Nebraska’s Mother of The Year, 2018. Dr. Nizar is also a rare disease patient and a mother to two boys with Jansen’s Metaphyseal Chondrodysplasia – a disease that affects less than 30 people worldwide.

Monica L Weldon

President/CEO/Founder Bridge the Gap - SYNGAP Education and Research Foundation

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.