PCAST Report: Priorities for Personalized Medicine

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PCAST Report Priorities for Personalized Medicine
2
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
  2.pdf

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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
  agencies

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Policy Areas Studied by PCAST

• Regulation of therapies and diagnostics by FDA,
  CMS
• 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.

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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
  savings
• 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.

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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
  time

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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)
  or
• Need for more comprehensive policy
  recommendations beyond the scope of personalized
  medicine (intellectual property and privacy).

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PCAST Findings Technology and Tools

• Challenges to validating genomic/clinical
  correlations necessary to advance products into
  clinical use
• Concerns for imbalance between discovery and
  validation.

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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
  medicine
• NIH should coordinate process to identify and
  prioritize diseases and common therapies that
  would benefit from applications of genomics-based
  molecular diagnostics.

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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.

11
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
  performance
• 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
  Systems

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PCAST Recommendations Regulation

• FDA should implement more transparent, iterative
  approach to regulation
• Final guidance for IVDMIAs should clarify risk
  definition
• 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
  therapeutics
• FDA should issue Guidance for regulation of
  automated Clinical Decision Support Systems

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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.

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PCAST Recommendations Regulation

• Industry should adopt proactive and constructive
  role with FDA to fulfill its regulatory
  responsibilities
• 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.

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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
  development
• Procedural hurdles associated with coding
  systems, bundled payment systems and complex
  billing procedures.

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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.

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PCAST Findings and RecommendationsHHS
Coordination

• 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.

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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

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Personalized Medicine Progress Overview

• Genomic-based molecular diagnostics represent a
  first wave of personalized medicine
  toolsforcing active dialogue on regulation and
  reimbursement
• 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)

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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
  care
• 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.

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Personalized Medicine PCAST Conclusion

• Personalized Medicine is changing the way we
  think about diagnostic information in patient
  care
• 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.

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Recent EventsPotential to Influence California
Perspective

• 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
  Fund
• 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
  2009
• Private sector efforts to accelerate patient
  stratification, e.g. First Medco publication
  heart attack risk with Plavix and PPIs, November
  2008

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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
  investment
• _____________
• California Biomedical Industry 2009 Report,
  California Healthcare Institute and
  PricewaterhouseCoopers LLP.

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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,
  etc.)
• Most active California VCs (3) financed 15
  companies with 1000 employees
• 2 largest molecular diagnostic firms 2008
  combined revenue 170 Million and 600
  employees.

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Current Private Sector Investment Environment

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

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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
  Wellness
• Laboratory jobs cannot be outsourced
  internationallyprovides educational and
  employment opportunities

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California Resources

• Molecular diagnostics and other companies at
  front of innovation cycle
• CIRM
• CALPERS
• 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

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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.

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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.