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3rd Expanded Access Programmes Global Congress 2022 Americas

Obtaining the much needed access to therapies for lifechanging treatment

Days
Hours
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20th - 21st September 2022 - 2 Day Conference
Boston, MA, USA

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Venue

Hilton Boston Back Bay

Address

40 Dalton St, Boston, MA 02115, United

Phone

+1 617-236-1100

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

Lisa Kearns

Division of Medical Ethics, NYU Grossman School of Medicine

Anne B. Cropp

Chief Scientific Officer, Early Access Care

Jean Godin,

Head, Global Access Programme, Sanofi

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

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EXPANDED ACCESS PROGRAMMES

SPONSORS

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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

Lisa Kearns

Division of Medical Ethics, NYU Grossman School of Medicine

Lisa Kearns, MS, MA, is a senior researcher in the Division of Medical Ethics at NYU Grossman School of Medicine. She is a member of the Division’s working groups on Compassionate Use and Preapproval Access (CUPA) and Pediatric Gene Therapy and Medical Ethics (PGTME). For the past six years she has studied the ethical issues surrounding pre approval/expanded access to investigational drugs, including state and federal “right to try” legislation. More recently she has been studying the ethics of “bespoke” N-of-1 gene therapies and of pre approval access to gene therapies. Since March she has been researching the ethical allocation of drugs, vaccines, and medical equipment during the novel coronavirus pandemic and access to investigational and unproven therapies for Covid-19. She is the deputy chair of the Compassionate Use Advisory Committees (CompAC), which advise Janssen Pharmaceuticals on the ethical allocation of its investigational drugs.

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.

Anne B. Cropp

Chief Scientific Officer, Early Access Care

Anne Cropp Pharm.D. is Chief Scientific Officer for Early Access Care, a company exclusively providing global Expanded Access/Compassionate Use services.  Anne has led the development of Expanded Access programs for small, mid, and large pharma companies based in the US, Europe, and Asia. Prior to founding Early Access Care, Anne was Vice President in the Global Product Development Group of Pfizer Inc responsible for Phase 2 and 3 Clinical Development Programs and Head of the Compassionate Access Group for Pfizer.  She is a globally recognized expert in expanded access and compassionate use and has over 25 years experience in early access programs. 

Jean Godin

Head, Global Access Programme, Sanofi

Jean Godin holds a Doctorate in Medicine (MD) from the University of Montreal, a post Graduate diploma in Family Medicine from the University of Montreal and a MBA in Business Administration from McGill University. He is a licensed physician of the Quebec College of Physician & Medical Council of Canada (LMCC). He practiced medicine full time in the Montreal area in both university and community hospitals until he joined the pharmaceutical industry in December of 1999.

Jean headed scientific affairs in Canada at Teva Pharmaceuticals (innovative division) from 1999 to 2007. In June 2007, he became General Manager for the branded activities of Teva in Canada; he was then responsible for the development and commercialization of the innovative portfolio. In July 2012, Jean joined Novartis Canada in the position of Chief Scientific Officer and Vice-President, Clinical and Regulatory Affairs, reporting to the CEO. Jean joined Sanofi in May 2016.  He is currently the Head of Global Access Programs.

Jean currently lives in Cambridge, Massachusetts and maintains a part-time medical practice in Montreal.