Non for profit model for rare disease therapy development
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Non For Profit Model for Rare Disease Therapy Development PowerPoint PPT Presentation

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Non For Profit Model for Rare Disease Therapy Development. Therapy Development Process Historically big business Large investment Time to Trial 6 to 8 years Rare disease economically not attractive. Rare Disease Therapy Development - Historically. Foster collaborations

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Non For Profit Model for Rare Disease Therapy Development

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Non For Profit Modelfor Rare Disease Therapy Development

Therapy Development Process

Historically big business

Large investment

Time to Trial 6 to 8 years

Rare disease economically not attractive

Rare Disease Therapy Development - Historically

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

Typical Rare Disease “Proactive” Advocacy Groups

Enzyme Therapy

Gene Transfer

Stem Cell Therapy

Small Molecule Pharmaceuticals

High Through-put Screening

Standard Therapeutic Options

Proof of Concept

Investigational New Drug Application (IND)

Protocol Development

Markers and Endpoints


Short Term Studies

Long Term Studies



Dosage and Delivery Studies

Therapy Development Process

Institutional Review Board (IRB)

Informed Consent Forms


Recombinant Advisory Committee (RAC)

Food and Drug Administration (FDA)

Clinical Trial Initiation

Therapy Development Process (cont.)



Proof of ConceptXXX





Clinical Trial InitiationXXX

Rare Disease Therapy Development Process Commonality

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?

Rare Disease Synergies

Non-For-Profit foundation sponsors the therapy development

Academic society is the Principle Investigator

Non-For-Profit Therapy Development Model

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

Non-For-Profit Therapy Development Model Benefits

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

Advancing Rare Diseases to the Clinic in a United Approach

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