non for profit model for rare disease therapy development
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04/19/23 1
Non For Profit Modelfor Rare Disease Therapy
Development
04/19/23 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
04/19/23 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
04/19/23 4
Standard Therapeutic Options
• Enzyme Therapy
• Gene Transfer
• Stem Cell Therapy
• Small Molecule Pharmaceuticals– High Through-put Screening
04/19/23 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
04/19/23 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
04/19/23 7
Rare Disease Therapy Development Process Commonality
Disease
AB 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
04/19/23 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?
04/19/23 9
Non-For-Profit Therapy Development Model
• Non-For-Profit foundation sponsors the therapy development
• Academic society is the Principle Investigator
04/19/23 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
04/19/23 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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