The 2017 WideTrial Expanded Access Summit

Integrating Expanded Access Programs into the Clinical Drug Development Process

Engaging More Patients / Capturing More Data

 

September 15, 2017
Royal Sonesta Boston
Cambridge, MA

Agenda

8:30am – 8:45am

Welcome / Opening Remarks

8:45am – 9:15am

Keynote: The Regulation of Expanded Access, Past and Present
David Farber, FDA Life Science Partner, King & Spaulding

9:20am – 10:30am

Session 1: “Doing it Right”: Ethical considerations, medical appropriateness and non-interference with research

  • Medical appropriateness and non-promotion
  • The role of the “honest third party” broker
  • The historical relationship between access and research
  • Advance planning of supply chain and clinical resources
  • Protocol considerations

Moderator: Anne Cropp, PharmD, VP and Clinical Group Head, Pfizer Expert Panelists TBA

10:30am – 10:45am
BREAK
10:45am – 12:00pm

Session 2: Addressing the traditional challenges head-on

  • Regulatory hazards, real and perceived
  • Authorization for early, meaningfully sized programs
  • Cost drivers: Supply, Program Management, Medical Treatment, Data & Clinical Duties
  • Billing and Payer policy: CMS Parts B and D, Private Insurers, Patient Assistance Programs

Moderator:  Jess Rabourn, CFA, Managing Director, WideTrial
Pat Furlong, Executive Director, Parent Project Muscular Dystrophy
Richard Moscicki, MD, Deputy Center Director, CDER, FDA

12:15pm – 1:30pm

LUNCH

Featured Speaker: Learning from Patients; using outcomes data and molecular diagnostics to guide targeted drug development 

1:45pm – 2:45pm

Session 3: Cost recovery; now and the near future

  • Defining medical value and s.o.c. in unsolved diseases
  • Charitable partnerships, wholesale repurchase of study drug
  • Initiatives for tiered pricing of investigational products
  • CMS, group plans, and critical paths for policy change
2:45pm – 3:45pm

Session 4: Expanded Access trials in response to contagion

  • Process development (PD) funding for large scale supply
  • Preventative treatment for high-risk populations
  • Integrating access trials into adaptive study designs (expanded ring trials)
  • Partnering with governments and NGOs abroad
3:45pm – 4:00pm
BREAK
4:00pm – 5:30pm

Session 5: Generating clinical data for biomarker discovery and subgroup targeting

  • Accumulation of longer-term safety & efficacy data
  • Consenting, analyzing, and storing participants’ biosamples & clinical endpoint data
  • Discovery & validation of novel target engagement markers
  • Discovery of super-responder groups and response-predictive markers.
  • Optimal dosing in population subsets
  • Guiding pivotal trial design