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Expanded Access Programmes Global Congress 2021 Americas

Obtaining the much needed access to therapies for lifechanging treatment

Days
Hours
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1st - 2nd December 2021 - 2 Day Conference
Boston MA, USA

Venue

Boston MA, USA

Address

Boston MA, USA

Phone

+44 (0) 207 193 3485

Global Congress 2021 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.

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

EXPANDED ACCESS PROGRAMMES

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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 15+ 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:

Who Should Attend?

Lisa Kearns

Division of Medical Ethics, NYU Grossman School of Medicine

Awaiting bio. 

Neena Nizar

President & Founder, The Jansens Foundation

Awaiting for bio. 

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. 

Khrystal Davis

Founder, Texas Rare Alliance

Awaiting for bio.