Consideration of Net Benefit as applied to Organ Transplantation

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Consideration of Net Benefit as applied to Organ
Transplantation

• Workgroup 3 UNOS Strategic Planning Retreat,
  Boston 10/05

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Worgroup 3 Net Benefit

• Dale Distant Introduction to Net Benefit
• Bob Wolfe Net Benefit Calculation and
  application
• Mark Stegall Net Benefit and KARS
• Bill Harmon Workgroup 3 recommendations

3
Workgroup 3 - Members

• Dale Distant
• Don Hillebrand
• Maggie Allee
• Dan Hayes
• Bill Harmon
• Abraham Shaked
• Jill McMaster
• Dolph Chianchiano

• Geoffrey Land
• Mark Stegall
• Jorge Reyes
• Clyde Barker
• Rich Fine
• Barry Kahan
• Doug Heiney

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Program Assessment and Rating Tool

• In July 2004, the HRSA Division of
  Transplantation (DoT) participated in the Office
  of Management and Budget (OMB) Program Assessment
  Rating Tool (PART).
• PART is a systematic method of assessing the
  performance of program activities across the
  Federal government.
• The PART is a diagnostic tool the main objective
  of which is to improve program performance.

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Program Assessment and Rating Tool

• PART strengthens and reinforces performance
  measurement by encouraging careful development of
  performance measures according to outcome
  oriented standards and by requiring that agency
  goals be appropriately ambitious.
• Because the ability to meet these targets is
  directly dependent on the efficient and effective
  operations of the OPTN, these goals will become
  the performance goals of the OPTN contract.
• Because achieving these goals is among DoTs most
  important priorities, the OPTN contractor must be
  a strategic partner in this effort.

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HHS Transplantation Goals
HHS Organ Transplantation Program Goals Targets Actual Performance
II. IMPROVE HEALTH OUTCOMES A. Expand the availability of health care, particularly to underserved, vulnerable, and special needs populations Long-Term Goal By 2013, increase the number of deceased donor organs transplanted to 42,800, an increase of 110 over baseline. (Outcome) Short-Term Goals 1. Increase the annual number of organs transplanted in accordance with projections until 42,800 organs are transplanted in 2013. (Outcome) 2. Increase the annual number of ?non-cardiac death? donors by 333 until the number of 9,251 ?non-cardiac death? donors is achieved in 2013. (Outcome) 3. Increase the annual number of ?cardiac death? donors by 175 until the number of 2,018 ?cardiac death? donors is achieved in 2013. (Outcome) 4. Increase the average number of organs transplanted per ?non-cardiac death? donor each year by 0.08 until the average of 4.00 is achieved in 2013. (Outcome) 5. Increase the average number of organs transplanted per ?cardiac death? donor each year by 0.096 until the average of 3.00 is achieved in 2013. (Outcome) FY 13 42,800 FY 06 25,651 FY 05 23,512 FY 04 21,459 FY 06 6,920 FY 05 6,587 FY 04 6,254 FY 06 793 FY 05 618 FY 04 443 FY 06 3.44 FY 05 3.36 FY 04 3.28 FY 06 2.328 FY 05 2.232 FY 04 2.136 FY 03 20,392 (baseline) FY 04 (4/05) FY 03 20,392 (baseline) FY 04 (4/05) FY 03 6,187 (baseline) FY 04 (4/05) FY 03 268 (baseline) FY 04 (4/05) FY 03 3.20 (baseline) FY 04 (4/05) FY 03 2.04 (baseline)
Long-Term Goal By 2013, increase the total expected life-years gained for kidney transplant recipients in the first 5 years after the transplant to 8,543 compared to what would be expected for these recipients had they remained on the waiting list. (Outcome) Short-Term Goals 6. Increase the average number of life-years gained in the first 5 years after transplantation for deceased kidney/kidney-pancreas transplants by 0.003 life-years until the goal of 0.436 life-years gained per transplant is achieved in 2013. (Outcome) 7. Increase the total number of expected life-years gained in the first 5 years after the transplant for all deceased kidney and kidney-pancreas transplant recipients compared to what would be expected for these patients had they remained on the waiting list. (Outcome) FY 13 8,543 FY 06 0.415 FY 05 0.412 FY 04 0.409 FY 06 5,048 FY 05 4,641 FY 04 4,257 FY 03 3,871 (baseline) FY 04 (4/05) FY 03 0.406 (baseline) FY 04 (4/05) FY 03 3,871 (baseline)
Efficiency Measure 1. Decrease the total OPTN operating costs per deceased organ transplanted. FY 06 774 FY 05 789 FY 04 808 FY 04 (4/05) FY 03 795 (baseline)
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HHS Transplantation Goals

• Long-Term Goal By 2013, increase the total
  expected life-years gained for kidney transplant
  recipients in the first 5 years after the
  transplant to 8,543 compared to what would be
  expected for these recipients had they remained
  on the waiting list.
• Short-Term Goal Increase the average number of
  life-years gained in the first 5 years after
  transplantation for deceased kidney/kidney-pancrea
  s transplants by 0.003 life-years until the goal
  of 0.436 life-years gained per transplant is
  achieved in 2013.

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HHS Transplantation Goals

• Short-Term Goal Increase the total number of
  expected life-years gained in the first 5 years
  after the transplant for all deceased kidney and
  kidney-pancreas transplant recipients compared to
  what would be expected for these patients had
  they remained on the waiting list

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Role of the OPTN
Stakeholders Advancing the Science of
Transplantation
Transplant Clinicians
Government Agencies OPTN/SRTR DSAs
Academic Centers
Corporate Enterprise
Funding Agencies
Immunology/ Immunosuppression
Donor Management/ Donation/ Preservation
Policy/ Allocation Collaboration
Recipient Management
Research
Research
History of Continously Improving Results
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OPTN Strategic Plan

• Benefit Net Benefit
• Equity
• Allocation Policy Directives to committees
• Data Collection
• Continual Improvement

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Net Benefit

• New terminology for transplant community but not
  a new concept
• Net benefit describes utility
• Utility considerations are already integral to
  organ allocation
• HLA in kidney transplantation
• MELD in liver transplantation
• Lung Allocation Score net benefit and waitlist
  mortality

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Net Benefit

• A more complete description of transplant utility
• Can synthesize multiple metrics describing the
  benefits and harms of transplantation into a
  single concept
• Readily quantifiable
• May describe individual or group benefit
• Allows comparison between individuals

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Net Benefit

• Not a single concept but a consistent methodology
  for answering questions of utility (maximizing
  benefit/reducing burdens)
• Patient and Graft Survival
• Best data serial data for some organs not
  others
• Waitlist mortality MELD
• Quality of Life Limited data
• Data collection beginning
• Burden of disease data in other databases

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Net Benefit

• Useful for all organs
• Utility goals need to be determined for each
  organ
• Components of net benefit calculation are organ
  specific
• Projected estimates of net benefit are time
  dependent
• Lung is the most advanced
• Aids determination of futility or harm as a
  result of transplant

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Lung Allocation - Definitions
USA New Lung Allocation Policy Pulmonary
Medicine Conference 2005

• Waitlist Urgency Measure Expected number of
  days lived without a transplant during an
  additional year on the waitlist
• Post-transplant Survival Measure Expected
  number of days lived during the first year
  post-transplant
• Transplant Benefit Measure Post-transplant
  Survival Measure minus Waitlist Urgency Measure
  i.e. expected extra days of life over the next
  year if that candidate receives a transplant
  rather than remaining on the waitlist

Lung Allocation Subcommittee, Thoracic Organ
Committee, UNOS
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Factors predicting survival after ltx
USA New Lung Allocation Policy Pulmonary
Medicine Conference 2005
Factors predicting waitlist survival

• Forced vital capacity (FVC)
• PA systolic (Group A, C, D)
• O2 required at rest
• (Group A, C, D)
• Age
• Body mass index (BMI)
• IDDM
• Functional status (NYHA)
• 6-minute walk distance
• Ventilator use
• Diagnosis

• Forced Vital Capacity (FVC) (Group B, D)
• PCW pressure ? 20 (Group D)
• Ventilator use
• Age
• Creatinine
• Functional Status (NYHA)
• Diagnosis

Lung Allocation Subcommittee, Thoracic Organ
Committee, UNOS
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Expected Waitlist Survival vs. Transplant Benefit
USA New Lung Allocation Policy Pulmonary
Medicine Conference 2005
based on number of transplant organs available
for current blood type within 1 year
1
2
2
3
3
1
transplant benefit threshold
Lung Allocation Subcommittee, Thoracic Organ
Committee, UNOS
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Expected Waitlist Survival vs. Transplant Benefit
USA New Lung Allocation Policy Pulmonary
Medicine Conference 2005
allocation balancing urgency benefit
2
1
patients dont stay in one place!
3
Lung Allocation Subcommittee, Thoracic Organ
Committee, UNOS
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Net Benefit - Caution

• New organ specific data elements or data
  collection intervals may be required
• The language, assumptions, and methods are
  utilitarian
• ethical dilemmas regarding distributive justice
• Equity is a necessary separate discussion

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Net Benefit - Caution

• One fundamental difficulty is that the
  foundations of the economic analysis are
  ethically biased towards utilitarianism. In the
  choice between different health care allocations
  both economic and ethical aspects must be
  considered. If this ethical bias inherent in
  economic theories is not recognised, the choice
  could be dubious from an ethical point of view.

Malmgren K, Hedström A, Granqvist R, Malmgren H
Ben-Menachem E, Cost analysis of epilepsy
surgery and of vigabatrin treatment in patients
with refractory partial epilepsy. Epilepsy
Research 25 (1996), 199-207.
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Here is a story based on Foot (1978) Five people
are in a hospital, dying. One can be saved only
by a kidney transplant, another by a heart
transplant, another by a brain transplant, etc.
They are all young and will lead full lives if
they are saved. But no donors are available.
Then, one day, Harry wanders into the emergency
room to ask directions... So the question for a
utilitarian is, why not?
Heuristics and biases in equity judgments a
utilitarian approach Jonathan BaronDepartment
of PsychologyUniversity of Pennsylvania