Transcript Rare Disease Therapy Development

Non For Profit Model
for Rare Disease Therapy
Development
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Rare Disease Therapy
Development - Historically
• Therapy Development Process
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Historically big business
Large investment
Time to Trial 6 to 8 years
Rare disease economically not attractive
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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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Standard Therapeutic Options
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Enzyme Therapy
Gene Transfer
Stem Cell Therapy
Small Molecule Pharmaceuticals
– High Through-put Screening
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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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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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Rare Disease Therapy Development
Process Commonality
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Proof of Concept
IND
IRB
RAC
FDA
Clinical Trial Initiation
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Disease
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C
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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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Non-For-Profit Therapy
Development Model
• Non-For-Profit foundation sponsors the
therapy development
• Academic society is the Principle
Investigator
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Non-For-Profit Therapy
Development Model Benefits
• Goal is to develop therapies verses commercialization of
a drug
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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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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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