Transcript PCAST Report: Priorities for Personalized Medicine

PCAST Report: Priorities for
Personalized Medicine
President’s Council of Advisors on
Science and Technology (PCAST)
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Member 2001-2009
Chair, Subcommittee on Personalized
Medicine
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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_v2.pdf
President’s Council of Advisors on
Science and Technology (PCAST)
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Personalized Medicine Subcommittee
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hosted more than ten meetings and workshops
over 110 individuals participated
Representatives from:
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Academic institutions, diagnostic, DTC, service and
imaging companies
Biotech, pharma, tools, IT companies
Insurance companies and providers
Patient advocates
VC’s, trade and professional associations, gov’t agencies
Policy Areas Studied by PCAST
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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.
PCAST Findings
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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.
PCAST Findings
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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;
PCAST Findings--continued
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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:
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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).
PCAST Findings: Technology and Tools
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Challenges to validating genomic/clinical
correlations necessary to advance products
into clinical use;
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Concerns for imbalance between discovery
and validation.
PCAST Recommendations:
Technology and Tools
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Federal government should develop strategic longterm plan by coordinating public and private
sector efforts to advance R&D;
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HHS should lead effort to create public/private
“Personalized Medicine R&D 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.
PCAST Recommendations:
Technology and Tools
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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
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FDA progress via Guidances to defining approach
to regulation of genomics-based molecular
diagnostics, with following outstanding issues:
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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
PCAST Recommendations: Regulation
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FDA should implement more transparent, iterative
approach to regulation:
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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
PCAST Recommendations: Regulation
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FDA Critical Path should be funded to include support of
personalized medicine progress:
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Biomarkers to facilitate product development, clinical
trial design and validation for molecular diagnostics
should be priority;
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Reagan-Udall should be funded and expanded to
include venture capital and molecular diagnostic
company representation.
PCAST Recommendations: Regulation
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Industry should adopt proactive and constructive role with
FDA to fulfill its regulatory responsibilities:
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When possible, responses to draft guidances should
include alternative approaches;
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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
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Three challenges to meeting cost-containment objectives
and not obstructing molecular diagnostic progress:
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Reimbursement based upon historical low-margin
commodity pricing;
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Need for standards related to evidence development;
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Procedural hurdles associated with coding systems,
bundled payment systems and complex billing
procedures.
PCAST Recommendations:
Reimbursement
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Public and private payors should determine coverage
policies and payment rates for genomic molecular
diagnostics in light of their overall impact on patient care—
demonstrated by evidence from clinical trails and other
studies:
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Reimbursement should be commensurate with clinical benefits and
payors should collaborate in establishing more flexible coding
approaches;
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Payors should collaborate to expand “coverage with evidence
development” programs;
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Payors should collaborate in developing standards for evidence
requirements from clinical trials.
PCAST Findings and Recommendations:
HHS Coordination
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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):
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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
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Survey conducted by Coalition for 21st Century
Medicine provided to FDA in May 2007 in
response to Guidances on IVDMIAs and ASRs:
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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
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Genomic-based molecular diagnostics represent a “first
wave” of personalized medicine tools—forcing active
dialogue on regulation and reimbursement;
Most pharmaceutical and biopharmaceutical companies
now have biomarker programs associated with early drug
R&D, 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
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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.
Personalized Medicine: PCAST
Conclusion
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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.
Recent Events—Potential to
Influence California Perspective
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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 FDA’s 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
PPI’s, November 2008
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
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California Biomedical Industry 2009 Report, California Healthcare Institute and
PricewaterhouseCoopers LLP.
California’s Personalized Medicine
Industry
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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 VC’s (3) financed 15+
companies with ~1000 employees;
2 largest molecular diagnostic firms 2008
combined revenue ~$170+ Million and 600+
employees.
Current Private Sector Investment
Environment
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Limited access to capital; pressure on long-term
R&D spending and efficiency at all sizes of
companies;
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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.
Role of Personalized Medicine in Future of
California’s Biomedical Industry
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Combination of economic and healthcare crises
threatens California’s biomedical industry;
Personalized medicine represents:
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Leading edge of genomics applications
Tools to facilitate CER
Beneficiary of HIT adoption and Prevention and
Wellness
Laboratory jobs cannot be outsourced internationally—
provides educational and employment opportunities
California Resources
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Molecular diagnostics and other companies at
front of innovation cycle;
CIRM;
CALPERS;
UC and State University system—interests in
business, science, legal and employment issues;
TRANSPERS Center at UCSF catalyzing study of
clinical utility, access and economics—plus
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
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Big Picture:
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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.