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PCAST Report: Priorities for Personalized Medicine


... (PCAST) Personalized Medicine ... Biomarkers to facilitate product ... Projected Diagnostic Product Pipeline Survey conducted by Coalition for 21st Century ... – PowerPoint PPT presentation

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Title: PCAST Report: Priorities for Personalized Medicine

PCAST Report Priorities for Personalized Medicine
Presidents Council of Advisors on Science and
Technology (PCAST)
  • Member 2001-2009
  • Chair, Subcommittee on Personalized Medicine
  • Study commenced January 2007
  • Report Published September 2008, met with
    President and Congressional Staff September 16
    and 17, 2008
  • http//www.ostp.gov/galleries/PCAST/pcast_report_v

Presidents Council of Advisors on Science and
Technology (PCAST)
  • Personalized Medicine Subcommittee
  • hosted more than ten meetings and workshops
  • over 110 individuals participated
  • Representatives from
  • Academic institutions, diagnostic, DTC, service
    and imaging companies
  • Biotech, pharma, tools, IT companies
  • Insurance companies and providers
  • Patient advocates
  • VCs, trade and professional associations, govt

Policy Areas Studied by PCAST
  • Regulation of therapies and diagnostics by FDA,
  • Reimbursement of therapies and diagnostics by CMS
    and private insurance companies
  • Genomic diagnostics, intellectual property and
    related emerging patent issues (PTO)
  • Patient privacy
  • Information technology and issues of electronic
    patient records and associated data/databases
  • Economics of personalized medicine
  • Personalized medicine technology/tools
  • Patient, physician and public education.

PCAST Findings
  • High level of policy interest attributable to
    promise of improved patient care and disease
    prevention, plus potential to positively impact
    increasing cost of healthcare and decreasing rate
    of new medical product development
  • Ability to distinguish patients most likely to
    benefit from treatment or suffer harm should
    improve quality of care and result in cost
  • Ability to stratify patients by disease
    susceptibility or likely response to treatment
    could reduce size, duration and cost of clinical
    trials to facilitate treatment, diagnostic and
    prevention strategies development.

PCAST Findings
  • Identified specific policy actions related to
    genomics-based molecular diagnostics with
    greatest potential to accelerate progress in
    personalized medicine
  • Parallel developments in genomics-linked
    therapeutics also important, though pace of
    change in molecular diagnostics most rapid and
    policy hurdles greatest in the latter at present

PCAST Findings--continued
  • Narrowed policy recommendations to three areas
    technology/tools, regulation and reimbursement
  • Other areas, while very important to long term
    progress, deemed less urgent due to
  • Significant ongoing government activity
    (information technology and privacy)
  • Early stage of personalized medicine development
    (physician and patient education and economics)
  • Need for more comprehensive policy
    recommendations beyond the scope of personalized
    medicine (intellectual property and privacy).

PCAST Findings Technology and Tools
  • Challenges to validating genomic/clinical
    correlations necessary to advance products into
    clinical use
  • Concerns for imbalance between discovery and

PCAST Recommendations Technology and Tools
  • Federal government should develop strategic
    long-term plan by coordinating public and private
    sector efforts to advance RD
  • HHS should lead effort to create public/private
    Personalized Medicine RD Roadmap
  • NIH, DOE and other agencies should evaluate
    relative funding for discovery versus
    translational research relevant to personalized
  • NIH should coordinate process to identify and
    prioritize diseases and common therapies that
    would benefit from applications of genomics-based
    molecular diagnostics.

PCAST Recommendations Technology and Tools
  • Federal government should make critical
    investments in enabling tools and resources
    moving beyond genomic discoveries to personalized
    medicine products and services of patient and
    public benefit
  • NIH should spearhead public and private efforts
    to develop a nationwide network of standardized
    biospecimen repositories
  • NIH should promote methods for validating the
    clinical utility of molecular diagnostics based
    on genomic correlations with disease
  • NIH should establish large US population cohort
    for investigating genetic and environmental
    health impacts.

PCAST Findings Regulation
  • FDA progress via Guidances to defining approach
    to regulation of genomics-based molecular
    diagnostics, with following outstanding issues
  • Risk definition for device versus information
  • Standards for study design and product
  • Coordination between CLIA (CMS) and FDA
  • Regulatory approach to co-development
  • Criteria and procedures for therapy labeling with
    diagnostic information
  • Regulatory approach to Clinical Decision Support

PCAST Recommendations Regulation
  • FDA should implement more transparent, iterative
    approach to regulation
  • Final guidance for IVDMIAs should clarify risk
  • FDA and CMS should clarify roles and potential
    regulatory redundancy
  • FDA should finalize draft co-development paper
  • FDA should clarify criteria and procedures for
    incorporating genetic data on labels for
  • FDA should issue Guidance for regulation of
    automated Clinical Decision Support Systems

PCAST Recommendations Regulation
  • FDA Critical Path should be funded to include
    support of personalized medicine progress
  • Biomarkers to facilitate product development,
    clinical trial design and validation for
    molecular diagnostics should be priority
  • Reagan-Udall should be funded and expanded to
    include venture capital and molecular diagnostic
    company representation.

PCAST Recommendations Regulation
  • Industry should adopt proactive and constructive
    role with FDA to fulfill its regulatory
  • When possible, responses to draft guidances
    should include alternative approaches
  • Industry should provide FDA with annual
    projections for number and type of products in
    development pipeline to better address staffing
    and resource needs.

PCAST Findings Reimbursement
  • Three challenges to meeting cost-containment
    objectives and not obstructing molecular
    diagnostic progress
  • Reimbursement based upon historical low-margin
    commodity pricing
  • Need for standards related to evidence
  • Procedural hurdles associated with coding
    systems, bundled payment systems and complex
    billing procedures.

PCAST Recommendations Reimbursement
  • Public and private payors should determine
    coverage policies and payment rates for genomic
    molecular diagnostics in light of their overall
    impact on patient caredemonstrated by evidence
    from clinical trails and other studies
  • Reimbursement should be commensurate with
    clinical benefits and payors should collaborate
    in establishing more flexible coding approaches
  • Payors should collaborate to expand coverage
    with evidence development programs
  • Payors should collaborate in developing standards
    for evidence requirements from clinical trials.

PCAST Findings and RecommendationsHHS
  • Secretary Leavitt encouraged the progress of
    personalized medicine at many federal agencies
    within HHS
  • Continuity is important to realizing the long
    term promise of personalized medicine and to make
    the most effective use of limited resources
  • HHS should establish a Personalized Medicine
    Coordination Office (PMCO)
  • PMCO should facilitate progress while ensuring
    safety, efficacy and utility and
  • Monitor progress to ensure all HHS agencies
    address emerging needs.

Personalized Medicine Projected Diagnostic
Product Pipeline
  • Survey conducted by Coalition for 21st Century
    Medicine provided to FDA in May 2007 in response
    to Guidances on IVDMIAs and ASRs
  • 70 commercial, academic and private research
    laboratories developing nearly 200 unique tests
    qualifying as IVDMIAs
  • Include Laboratory Developed Tests serving as
    treatment decision tools for 20 cancers, as
    well as prediction tools incorporating lab and
    other data for patient specific values to predict
    conditions, outcomes, or risks

Personalized Medicine Progress Overview
  • Genomic-based molecular diagnostics represent a
    first wave of personalized medicine
    toolsforcing active dialogue on regulation and
  • Most pharmaceutical and biopharmaceutical
    companies now have biomarker programs associated
    with early drug RD, some with companion products
    in pipeline
  • FDA is actively integrating personalized medicine
    into clinical trial design over 200 drug labels
    have genetic info
  • Programs in multiple federal agencies should
    facilitate personalized medicine progress (e.g.
    AHIC and PHR, EMR)

Personalized Medicine Progress Overview
  • Imaging/diagnostic companies are making a
    significant commitment to field, with new imaging
    products expected in pipeline
  • Community based delivery systems are integrating
    personalized medicine into research and patient
  • Teaching and research institutions are
    collaborating to create delivery systems that
    exchange laboratory and other patient data
    between/among institutions
  • Community, teaching institutions and companies
    are developing Clinical Decision Support Systems
    to facilitate uptake of complicated genomic data
    into patient care
  • Consumer oriented IT companies are providing new
    electronic patient records to provide control and
    access to individuals.

Personalized Medicine PCAST Conclusion
  • Personalized Medicine is changing the way we
    think about diagnostic information in patient
  • Patient stratification to enhance quality of
    care, whether by genomic technology or other
    means, will become more widely implemented in the
    coming years
  • New molecular technologies are raising the
    visibility of the relative value of critical
    diagnostic information versus that of treatment
  • Evidence development will play a key role in how
    the value proposition is sorted out.

Recent EventsPotential to Influence California
  • American Recovery Reinvestment Act provides 1.1B
    in Cost Effectiveness Research (CER), 19B to
    accelerate adoption of HIT systems by doctors and
    hospitals, plus 1B for Prevention and Wellness
  • New genomics position in FDAs Office of Chief
    Scientist announced February 5, 2009
  • Deloitte ROI report January 2009
  • CMS evaluating 2 personalized medicine NCDs in
  • Private sector efforts to accelerate patient
    stratification, e.g. First Medco publication
    heart attack risk with Plavix and PPIs, November

2009 California Biomedical Industry
  • 2,042 California biomedical companies
  • 74.5 billion total estimated revenues
  • 271,000 total estimated employees
  • 20.3 billion estimated wages and salaries
  • Nearly 75,000 average wage
  • 3.2 billion total NIH grants awarded
  • 4.3 billion total est. venture capital
  • _____________
  • California Biomedical Industry 2009 Report,
    California Healthcare Institute and
    PricewaterhouseCoopers LLP.

Californias Personalized Medicine Industry
  • California companies represent majority of new
    molecular diagnostic firms
  • Bay Bio lists 101 companies representing all
    areas (i.e. therapy, diagnostics, tools, DTC,
  • Most active California VCs (3) financed 15
    companies with 1000 employees
  • 2 largest molecular diagnostic firms 2008
    combined revenue 170 Million and 600

Current Private Sector Investment Environment
  • Limited access to capital pressure on long-term
    RD spending and efficiency at all sizes of
  • Potential healthcare reform, increasing risk of
    margin erosion and reducing pharma policy
  • Limited liquidity, squeezing venture and
    innovation and
  • Speed and degree of consolidation unknown.

Role of Personalized Medicine in Future of
Californias Biomedical Industry
  • Combination of economic and healthcare crises
    threatens Californias biomedical industry
  • Personalized medicine represents
  • Leading edge of genomics applications
  • Tools to facilitate CER
  • Beneficiary of HIT adoption and Prevention and
  • Laboratory jobs cannot be outsourced
    internationallyprovides educational and
    employment opportunities

California Resources
  • Molecular diagnostics and other companies at
    front of innovation cycle
  • CIRM
  • UC and State University systeminterests in
    business, science, legal and employment issues
  • TRANSPERS Center at UCSF catalyzing study of
    clinical utility, access and economicsplus
    assembly of representative stakeholders
  • Historically, national delivery-of-care models
    follow where California leads

Policy/Research Areas to Consider
  • Reminder--
  • Healthcare has many stakeholders in heavily
    regulated businesses that are likely to be
    adverse to change. The status quo serves many
    constituencies very well.

Policy/Research Areas to Consider
  • Big Picture
  • Assemble different stakeholders to inform,
    educate and act
  • Implement delivery studies and other pilot
    programs to better inform policy
  • Conform state-funded research programs to provide
    future access and standards for personalized
    medicine research and
  • Drive privacy, patient consent, access, education
    and training incentives and standards.
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