Organization Daylong Symposium: How Can I Get a Treatment for My Disease?
How Can I Get a Treatment for My Disease?
Fundamentals of Drug Development for Advocates: A Basic Primer
June 24, 2011
Bethesda North Marriott
"In order to change an existing paradigm you do not struggle to try and change the problematic model. You create a new model and make the old one obsolete."
There is no deli line in treatment development. You can’t take a number and wait for someone to call your disease to the front of the line for a treatment.
Despite the enormous amount of time, money, blood, sweat and tears that have gone into treatment development for genetic diseases, only a few hundred have treatments. The successes and the problems have been described over and over. It is time now to forge the solutions.
Some of the answers will come from retooling drug development as it stands today. Some will come from changing the paradigm completely (see What is my Responsibility? for this workshop!).
How have some advocacy organizations successfully moved through the drug development pipeline, resulting in new treatments for their disease? Some have conducted successful natural history studies, and many have robust registries and biobanks – how is that possible? How did they make it happen?
In this daylong, “How Can I Get Treatments for My Disease?” we discussed the fundamentals of drug development for advocacy organizations. We heard from experts about the drug development process and how advocacy organizations can influence it by developing much-needed tools and resources. We examined the drug development map, and showed case studies of organizations who are leading the way through gene identification, diagnostics, disease characterization, clinical endpoints/biomarkers, clinical trials, outreach efforts, registries, and biobanks.
The day concluded with each organization developing an action plan to move research forward. We hope you were able to join us for this unique opportunity to learn about the drug development process and how your organization can lead the way in developing new treatments for your disease or condition.
Agenda
Therapeutic Development from A to Z
8:00 a.m.
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Registration and Breakfast
8:30 a.m.
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Welcome and framing the day
Steve Groft, Office of Rare Disease Research
Liz Horn, Director, Genetic Alliance Registry and BioBank
9:00 a.m.
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What Should Happen?
Bernard Munos, InnoThink Center for Research in Biomedical Innovation
9:30 a.m.
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Novel Efforts to Accelerate Drug Development: examples from rare and neglected diseases
Chris Austin, NCTT, NIH
10:00 a.m.
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Break
10:30 a.m.
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The Map of the World as We Know it: Opportunities for Advocates
Sharon Terry, Genetic Alliance
11:00 a.m.
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Models on the Map
10 minutes each: describe where you are on the map, describe what you did, what it cost, what you learned, what you would do different next time, how you would franchise or industrialize it.
Gene & Mutation Identification – Martin Naley, Life Technologies
Diagnostics – Sherri Bale, GeneDx
Clinical endpoints/Biomarkers – Pat Furlong, Parent Project Muscular Dystrophy
Clinical Trials – Donna Appell, Hermansky-Pudlak Syndrome Network
12:00 p.m.
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Lunch
1:00 p.m.
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How to Get There from Here: Building the Shared Infrastructure Nuts and bolts
Outreach – Brian Loew, Inspire
Registries – Liz Horn, Genetic Alliance Registry and BioBank
BioBanks – Suzanne Vernon, CFIDS Association of America
2:00 p.m.
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Dive In: Create Your Plan of Attack
Completely interactive –newbies, novices and advanced! Goal is a plan – or at least the questions that one needs to answer to create a plan. Get it done!
4:30 p.m.
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Return and share plans, challenges – find resources in the room – determine what shared resources and infrastructures should be created.
5:30 p.m.
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Conclusions, Mandates and Marching Orders
