1. Non For Profit Model for Rare Disease Therapy Development
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2. Rare Disease Therapy Development - Historically
Therapy Development Process
Historically big business
Large investment
Time to Trial 6 to 8 years
Rare disease economically not attractive
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3. Typical Rare Disease “Proactive” Advocacy Groups
Foster collaborations
Liaisons between Basic Research and Biotech companies
Deliver resources to fill funding gaps in the development process
“Managing science” –resources/timeframes/direction/goals
Therapy focus – move what is known today forward
Political Involvement
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4. Standard Therapeutic Options
Enzyme Therapy
Gene Transfer
Stem Cell Therapy
Small Molecule Pharmaceuticals
High Through-put Screening
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5. Therapy Development Process
Proof of Concept
Investigational New Drug Application (IND)
Protocol Development
Markers and Endpoints
Toxicology
Short Term Studies
Long Term Studies
Pharmacology
Chemistry/Manufacturing
Dosage and Delivery Studies
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6. Therapy Development Process (cont.)
Institutional Review Board (IRB)
Informed Consent Forms
Approvals
Recombinant Advisory Committee (RAC)
Food and Drug Administration (FDA)
Clinical Trial Initiation
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7. Rare Disease Therapy Development Process Commonality
A B C
Proof of Concept X X X
IND X X X
IRB X X X
RAC X X X
FDA X X X
Clinical Trial Initiation X X X
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8. Rare Disease Synergies
Regardless of the therapy we all face the same procedural development issues.
Uniting empowers rare diseases
What is good for one of us is good for all
How do we leverage the commonality?
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9. Non-For-Profit Therapy Development Model
Non-For-Profit foundation sponsors the therapy development
Academic society is the Principle Investigator
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10. Non-For-Profit Therapy Development Model Benefits
Goal is to develop therapies verses commercialization of a drug
Quickly take proof of concept from the lab to the clinic
NPF assume risk and responsibility
Leveraging infrastructure and regulatory knowledge
Provide data to companies for future clinical phases for long term solutions
Funding sources: foundations, companies, or NIH
Cost savings in therapy development for companies to prove concept before large scale investment
Business issues not a concern to achieve the NFP goals regarding intellectual properties or patents
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11. Advancing Rare Diseases to the Clinic in a United Approach
Rare diseases have a platform to move to a clinical program
Together we have stronger voice to regulatory committees
Quicker to clinic
Develop NFP models for each type of therapy
We all face the same issues
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