Margaret AndersonMargaret AndersonCOOCOO

June 25, 2009June 25, 2009

BioEconomy SummitHealthcare Policy Session 2: Affordability

and Access

Fostering innovation while delivering affordable treatments and technologies to

large markets

• Research roadmaps • New technology applications• Patient data utilization in research• Human capital• Clear federal guidance on technologies & approaches• Guidelines for public-private partnerships• Global perspective of the drug discovery and

development process• Collaboration on patient registries and

recruitment for clinical trials

• Online tools to facilitate collaboration & interaction

• Increased awareness of the importance of translational research programs

• Effective communication of the complexity and value of medical research to the public

• Advocacy behind key public policy issues (i.e., privacy, IP)

• Organizational transformation to address emerging research needs

• Risk-sharing on pre-competitive tools

• More focus on translational research at NIH

• Increased funding and predictable rewards structure for very novel, high-risk research

• Standardization of clinical terms

•Information portal for best practices•Goal-oriented intellectual property arrangements

Components Essential to Successful Components Essential to Successful Innovation in Disease ResearchInnovation in Disease Research

RESEARCHRESOURCES

RESEARCHINFRASTRUCTURE

RESEARCHENVIRONMENT

Addressing Challenges Addressing Challenges within the Traditional within the Traditional

Research SystemResearch System

Key Issues Affecting ProgressKey Issues Affecting Progress

Infrastructure• Institutional stakeholders’ resistance to

changing infrastructure and rewards systems – in areas such as publication, tenure, grants, and intellectual property – to promote collaboration and innovation;

• Lack of institutionalized communication and data exchange between basic and clinical researchers;

• Inadequate opportunities for cross-disciplinary training and practice

Key Issues Affecting ProgressKey Issues Affecting ProgressResearch Environment

• Highly specialized, reductionist approach to scientific inquiry

• Little funding or reward available for high-risk research• Limited collaboration on “big picture” problems• Conflict-of-interest challenges in public-private

partnerships• Lack of public understanding of the disease research

challenges• Insufficient focus on translating basic research into

clinical application;• Inadequate dissemination of previous research efforts –

especially failures;• Failure to aggregate funding across organizational lines

to achieve larger scale impact

Valley of DeathValley of Death

Basic research is the earliest stage of research, carried out for the advancement of knowledge, without necessarily any regard to its application to practical problems.

Translational research is the process of applying ideas, insights, and discoveries generated through basic scientific inquiry to the treatment and prevention of human disease – the critical bridge between basic research and clinical research.

Clinical research is research in human subjects aiming toward approved treatments for patients.

Finding Big Ideas in Small Finding Big Ideas in Small Spaces: New Business Spaces: New Business

Models to Models to Accelerate ResearchAccelerate Research

Philanthropic Research Funding: Philanthropic Research Funding: Small in Size, Unique in its RoleSmall in Size, Unique in its Role

25%

32%

16%

8%

8%2%

2%

7%

Foundations and other private funds($2.5B)

Estimated health research expenditure in the US by source, 2006

USD billions, percent (100%=$116.1B)

Pharmaceuticals ($37.0B)

Medical device industry ($9.5B)

Biotech($18.2B)

NIH($28.5B)

Universities ($8.3B)

State and local funds ($2.9B)

Other federal funds ($9.2B)

Source: “2006 Investment in U.S. Health Research”, Research!America; Moses, H. et al, “Financial Anatomy of Biomedical Research,” Journal of American Medical Association, vol.294, No.11, September 21, 2005

Philanthropic money accounts for only 2% of total funding; however, it fills a unique niche by:• Filling gaps in funding research that is high risk but also with potential of high return • Being virtually the only source of funding for innovative research in the current environment of flat NIH funding

Finding Big Ideas in Small Finding Big Ideas in Small SpacesSpaces

The Redstone Acceleration and Innovation NetworkThe Redstone Acceleration and Innovation Network

Passion Capital De-Risking Investments

Patient AccessClinical Trial and Research Networks

Intellectual CapacityResearch Discipline

Science ManagementAdvocacy

TThe he RRedstone edstone AAcceleration and cceleration and IInnovation nnovation NNetworketwork

Venture Philanthropies’ Innovative Venture Philanthropies’ Innovative PracticesPractices

• Strategic use of capital• Building collaborations• Streamlining the grantmaking process• Sharing information

High-Risk, High-Reward VenturesHigh-Risk, High-Reward VenturesNew Business Models to Accelerate ResearchNew Business Models to Accelerate Research

• Funded $126 million in research to date, 2/3 for translational research

• Funded testing of over 100 therapeutic targets for Parkinson’s Disease

• Partnership with Elan Corp. gives them first look at promising projects

• Funded more than $66 million since 2005

• Actively supports more than 30 new treatments in development – 4 FDA-approved

• Partners include Vertex Pharmaceuticals, Parion Sciences, PTC Therapeutics, Inspire Pharmaceuticals, Gilead Sciences, Altus Pharmaceuticals

• Raised $102 million to fund 70 labs worldwide

• 4 new treatments approved in 4 years

• 10 clinical trials facilitated by Multiple Myeloma Research Consortium

• 30 MMRF-funded compounds now in preclinical studies and clinical trials

Separating what we Separating what we need to do in scienceneed to do in science

and what we need to doand what we need to doin the culture of sciencein the culture of science

to accelerate curing to accelerate curing diseasesdiseases

Urgent Need to Refocus the Urgent Need to Refocus the GlobalGlobalMedical Research EnterpriseMedical Research Enterprise

Current research infrastructure has led to great advances in knowledge of human health and disease;

but it is not a good system for developing therapies for patients

studying biologystudying biology curing diseasescuring diseases

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