Expanded Access at a Crossroads - Webinar Recording/Transcript

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Expanded Access at a Crossroads: How Drug Companies Can Benefit from Pre-Approval Access Programs

Expanded Access is the hottest topic in new-drug development, and now the FDA appears ready to loosen the reins. In rapid succession:

  • Congress passed FDARA and included language encouraging drug developers to engage wider ranges of patients in the clinical development process
  • The Senate approved “Right to Try” legislation, which could allow drug developers to provide pre-approval access to patients without FDA oversight
  • The Expanded Access Drug Summit took place. Its mission was to draft best practices for integration of pragmatic Expanded Access programs into the clinical development process.

Now, the organizer and chair of that summit meeting, Jess Rabourn CFA, explains how you can get in on the ground floor.

Mr. Rabourn explains the new proposed best practices for integrating cohort Expanded Access into the drug-development process in breakthrough disease, and a lot more besides:

  • Unpacking the nest of regulations that govern pre-approval access, including key provisions on cost recovery
  • Debunking common misconceptions that may affect your business operations
  • Learning how pre-approval access programs have affected clinical research
  • Parsing prevailing business practices for sponsoring Expanded Access programs, including those that go beyond single-patient scenarios
  • The growing recognition of data value: capturing Real World Evidence and the differentiating factors of treatment response
  • And much more!

So many unanswered questions. How do you balance the demand for Expanded Access while still supporting clinical research? What are best practices for forging ahead on these programs without slighting ongoing new-drug development? You’ll hear answers to those questions — and more — which keep the focus on cost recovery, protocol design and data collection. Don’t miss out!

This presentation targets:

  • Small, pre-revenue drug and biologics makers with therapeutics in development for serious unsolved diseases; i.e. companies with limited resources
  • Large therapeutics companies with multiple marketed products in addition to investigational-stage products; i.e., companies with high sensitivity to public perception
  • Precision medicine, big data, and diagnostics companies
  • Patient advocacy organizations

Within such organizations, job titles include:

  • Director of Clinical Development
    Director of Medical Affairs
  • CSO (for precision medicine/data companies)
  • CSOs and Public Policy Directors (for advocacy and policy-support groups)

Jess Rabourn CFA founded WideTrial, a company that supports and directly sponsors cohort Expanded Access programs in the U.S. He also is Principal Partner of Ax-S Pharma LLC, a consultancy specializing in pre-market access strategies in the U.S.  He is a prominent voice on commercial factors of breakthrough drug development and making early-access programs commercially feasible. He is a member of the American Academy of Neurology and was lead author of the Expanded Access section of the submitted draft for FDA's Guidance on ALS Drug Development. He produced and chaired the 2017 Expanded Access Summit, a groundbreaking event focusing on the role of pragmatic access programs in the drug development process.