2009 Annual Conference Daylong Symposium: Research

GRANDRx
Crafting a Plan for Rare Disease Drug Development
• North Bethesda Marriott • July 17, 2009 • 8:00 AM - 6:00 PM •
• Followed by Reception and Dinner •

The Time is Now!
Rare diseases are therapeutically “neglected” because the costs of drug development cannot be recouped by drug sales, either due to small patient numbers or inability to pay for medicines. Of the more than 6000 rare diseases, fewer than 200 have any therapy. This problem is compounded by the extreme costs, failure rates, & development times of new drugs. Now is the time to engage in an initiative similar in its audacity and importance to the Human Genome Project. We must accelerate transformation in the systems of discovery. The new TRND Program of NIH will be presented. This workshop will result in work groups to tackle these issues and create collaborative solutions.

A Problem‐Focused Paradigm
In this workshop, we will focus on solutions to the specific limiting problems of rare disease drug development, with a deliverable, milestone, and results‐oriented approach. Those problems are:

Candidate drug development from chemical probe to IND
Assay development for rare diseases
Clinical cohorts and biomarkers
Regulatory sufficiency throughout

Presenters:

Melissa Ashlock - Therapeutics for Rare and Neglected Diseases, NHGRI
Chris Austin - NIH Chemical Genomics Center
Kyle Brown - Inolyst
Francis Collins - former director of NHGRI
Tim Cote - Director of the Office of Orphan Products, FDA
Felicia DeRosa - Program Director, Friedreich's Ataxia Research Alliance
Steve Groft - Director, NIH Office of Rare Disease Research
Alan Guttmacher - Acting Director, NHGRI
Jim Inglese - NIH Chemical Genomics Center
David Meeker - Executive Vice President of Therapeutics, Genzyme
Eric Olson - Vice President, Vertex Pharmaceuticals, Inc.
Vanessa Rangel Miller - DuchenneConnect
Peter Saltonstall - President & CEO, National Organization for Rare Disorders
Janet Woodcock - Director, Center for Drug Evaluation and Research, FDA

On average, drug sponsors can spend over 13 years studying the benefits and risks of a new compound, and several hundred millions of dollars completing these studies before seeking FDA’s approval. About 1 out of every 10,000 chemical compounds initially tested for their potential as new medicines is found safe and effective, and eventually approved by FDA, making the drug discovery and development process complex, time consuming, and costly.

NEW DRUG DEVELOPMENT: Science, Business, Regulatory, and Intellectual Property Issues Cited as Hampering Drug Development Efforts.

GRANDRx
Crafting a Plan for Rare Disease Drug Development
• North Bethesda Marriott • July 17, 2009 • 8:00 AM - 6:00 PM •
• Followed by Reception and Dinner •

8:00 am		Breakfast
9:00 am		Welcome and Introductions Sharon Terry – President & CEO, Genetic Alliance Steve Groft - Director, Office of Rare Disease Research Peter Saltonstall - President & CEO, National Organization for Rare Disorders
9:15 am		Unprecedented Therapeutic Opportunities for Rare and Neglected Diseases Francis Collins - former director of National Human Genome Research Institute
9:45 am		Assays and Therapeutic Development for Rare and Neglected Diseases Jim Inglese - Deputy Director, NIH Chemical Genomics Center, National Human Genome Research Institute, NIH
10:05 am		Therapeutics for Rare and Neglected Diseases: Creating a Breakthrough Systems Process for Moving from Targets to Clinical Candidates Chris Austin - Director, NIH Chemical Genomics Center, National Human Genome Research Institute, NIH
10:25 am		Opportunities and Challenges in Orphan Disease Drug Development Melissa Ashlock - Senior Consultant, Therapeutics for Rare and Neglected Diseases Program (TRND), National Human Genome Research Institute, NIH
10:45 am		Break
11:00 am		Drug Discovery for Cystic Fibrosis: A Bench to Bedside Collaboration between Vertex Pharmaceuticals and Cystic Fibrosis Foundation Eric Olson - Vice President, Vertex Pharmaceuticals, Inc.
11:20 am		The Orphan Drug Act Tim Cote - Director of the Office of Orphan Products, FDA
11:40 am		Discussion Alan Guttmacher - Acting-Director, National Human Genome Research Institute
12:00 pm		Lunch and Networking
12:45 pm		The Regulatory Issues for Drug Development throughout the Pipeline Sandra Kweder - Deputy Director Office of New Drugs, Center for Drug Evaluation and Research, FDA
1:10 pm		Building a Registry for Rare Disorders: The Importance of Fitting into a Global System Panel:Vanessa Rangel Miller - DuchenneConnect; Felicia DeRosa - Program Director, Friedreich's Ataxia Research Alliance; Kyle Brown - Innolyst
1:55 pm		Systems for a Patient-centered Enterprise for Rare and Neglected Diseases Patrick Terry - Acting-CEO, Grand Therapeutics Foundation
2:15 pm		Discussion Steve Groft - Director, Office of Rare Disease Research
2:40 pm		Engaging Us All to Co-Create Solutions – Work Group Instructions Patrick Terry - Acting-CEO, Grand Therapeutics Foundation
3:00 pm		Break
3:15 pm		Breakouts Session: Three Work Groups 1) Assay Development - Josh Sommer & James O’Leary 2) Preclinical Development - Eric Olson & Steven Bramer 3) Cohort Development - Margaret Anderson & Kurt Christensen
5:00 pm		Group Gathering: Reports from the Work Groups
5:45 pm		Discussion & Next Steps Patrick Terry - Acting-CEO, Grand Therapeutics Foundation
6:00 pm		Reception – cash bar
7:00 pm		Dinner

Sponsors: Office of Rare Disease Research, Genzyme, Genetic Alliance