Consideration of Net Benefit as applied to Organ Transplantation Workgroup 3 – UNOS Strategic Planning Retreat, Boston 10/05

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

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

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.

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 DoT’s most important priorities, the OPTN contractor must be a strategic partner in this effort.

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 (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 (Outcome) 3. Increase the annual number of  cardiac death  donors by 175 until the number of 2,018  cardiac death  donors is achieved in (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 (Outcome) 5. Increase the average number of organs transplanted per  cardiac death  donor each year by until the average of 3.00 is achieved in (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: FY 05: FY 04: 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 life-years until the goal of life-years gained per transplant is achieved in (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: FY 05: FY 04: FY 06: 5,048 FY 05: 4,641 FY 04: 4,257 FY 03: 3,871 (baseline) FY 04: (4/05) FY 03: (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)

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-pancreas transplants by life-years until the goal of life-years gained per transplant is achieved in 2013.

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

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

OPTN Strategic Plan Benefit – Net Benefit Equity Allocation Policy – Directives to committees Data Collection Continual Improvement

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

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

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

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

Lung Allocation - Definitions 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 USA New Lung Allocation Policy: Pulmonary Medicine Conference 2005 Lung Allocation Subcommittee, Thoracic Organ Committee, UNOS

Factors predicting survival after ltx Forced vital capacity (FVC) PA systolic (Group A, C, D) O 2 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 Factors predicting waitlist survival USA New Lung Allocation Policy: Pulmonary Medicine Conference 2005 Lung Allocation Subcommittee, Thoracic Organ Committee, UNOS

2 2 Expected Waitlist Survival vs. Transplant Benefit allocation by benefit allocation by urgency based on number of transplant organs available for current blood type within 1 year transplant benefit threshold USA New Lung Allocation Policy: Pulmonary Medicine Conference 2005 Lung Allocation Subcommittee, Thoracic Organ Committee, UNOS

Expected Waitlist Survival vs. Transplant Benefit allocation balancing urgency & benefit allocation balancing urgency & benefit patients don’t stay in one place! USA New Lung Allocation Policy: Pulmonary Medicine Conference 2005 Lung Allocation Subcommittee, Thoracic Organ Committee, UNOS

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

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),

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 Baron Department of Psychology University of Pennsylvania