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The Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration (FDA) is announcing a public workshop entitled "Facilitating End-to-End Development of Individualized Therapeutics."

Purpose of the Workshop:

The purpose of the public workshop is to foster development of individualized therapeutic products for the treatment of one individual or a very small number of patients, based on engineering a product aimed at the specific molecular mechanism underlying a patient’s (or small group of patients’) illness. 

Background:

Individualized therapeutics (also called Bespoke therapeutics) have become increasingly feasible because of enhanced understanding of individual patient variations and because of the implications of these variations for treatment and dose selection.  There are manufacturing technologies, referred to here as manufacturing platform technologies, that allow a treatment to be engineered for a specific individual (i.e., engineering an individualized therapeutic product).  Manufacturing platform technologies may be beneficial in gene therapies based on use of a specific vector (e.g., adeno-associated viral vector) to deliver a specifically engineered transgene insert that may vary between patients; in genome editing technologies; for genetically engineered bacteriophages or custom formulated bacteriophage cocktails for multidrug-resistant infections; and in manufacturing NGS-based therapeutic products for the treatment of cancer. 

Development of individualized therapeutics represents a new paradigm in drug development and there may be increasing numbers of stakeholders beyond traditional pharmaceutical companies with a vested interest in this endeavor.  The challenges and opportunities for individualized therapeutics span the development process, including manufacturing and assurance of product quality, preclinical testing, clinical safety and effectiveness evaluation, and sustainability.  Patient access to individualized therapies may advance more rapidly with development of new manufacturing approaches using a common technology platform, and innovative approaches to collaboration among stakeholders, including the patients who may receive these therapies. 

The goal of the public workshop is to receive input from stakeholders on issues that are limiting development of individualized therapeutics, and on actions that FDA and other critical stakeholders can take to facilitate end-to-end development and sustainable access to these promising, novel products.  These issues will be explored through presentations and panel discussions by experts from academic institutions, industry, government agencies, non-government organizations, and patient groups.  Each panel discussion will include a question and answer session.

Objectives of the Workshop:

1. To discuss scientific challenges to development of individualized therapeutics, and ongoing efforts to adapt products manufactured based on a technology platform to treat diseases affecting one individual or a very small number of patients; explore lessons learned, logistical challenges, failures, and successes.

2. To examine challenges under the current U.S. regulatory framework that need to be addressed to have a clear pathway for access to individualized therapeutics, including licensed manufacture and consistent delivery of safe and effective products.

3. To explore challenges from the patient perspective that limit development and access to individualized therapeutics; consider research and development, ethical, and product availability and access issues.

4. To identify new models of collaboration or synergies that may facilitate aspects of development, including manufacturing, preclinical testing, clinical development, and continuing evaluation for consistent production, availability, and delivery of individualized therapeutic products.

Webcast

The Webcast for this event can be viewed at http://fda.yorkcast.com/webcast/Play/b9c6ac08f3d040eba768ef43befb498f1d.

Transcripts

Transcript for the Facilitating End-to-End Development of Individualized Therapeutics Public Workshop

DATE:

Tuesday March 3, 2020
8:30 AM – 5:00 PM EST

LOCATION:

White Oak Campus
10903 New Hampshire Ave
Building 31 Conference Center, Great Room
Silver Spring, MD, 20993-0002

Entrance for the workshop is through Building 1, where routine security check procedures will be performed.  For parking and security information, please refer to https://www.fda.gov/about-fda/white-oak-campus-information/public-meetings-fda-white-oak-campus

This public workshop will also be webcast.  Individuals who wish to view the webcast should register for the workshop.  A link to the live webcast will be provided upon registration.

REGISTRATION:

Registration: To register for the public workshop, please visit the following website: https://www.eventbrite.com/e/facilitating-end-to-end-development-of-individualized-therapeutics-tickets-83345822599

Please provide complete contact information for each attendee, including name, title, affiliation, and email address.  Registration is free and based on space availability, with priority given to early registrants.  Persons interested in attending this public workshop must register by February 18, 2020, midnight Eastern Daylight Time. Early registration is recommended because seating is limited. If time and space permit, on-site registration on the day of the public workshop will be provided, beginning at 7:30 AM.   If you need special accommodations due to a disability, please contact Loni Warren Henderson or Sherri Revell no later than February 18, 2020.

LUNCH AND REFRESHMENTS:

Box lunches will be available for purchase.  Cash and credit cards accepted.  Lunch should be pre-ordered when signing in for the workshop in the morning or during the morning break.  Pre-paid lunches will be ready for pick-up at the lunch break.

FOR FURTHER INFORMATION CONTACT:

Loni Warren Henderson or Sherri Revell, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993, 240-402-8010, email [email protected] (subject line: Individualized Therapeutics Workshop).

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