In This Week’s Issue:
1. Value Assessment in the News, see details below.
2. Is the QALY Fit for Purpose? Click here to read the journal.
3. 2021 PCORI Annual Meeting Scheduled for Nov. 17-19, click here for details.
4. Public Comment on PCORI Proposed National Priorities for Health, see details below.
5. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
6. States Rely on Template Legislation that References ICER and Discriminatory Metrics, see details below.
7. Emerging Threats in States for Use of Discriminatory Metrics, see details below.
8. IVI to Hold Second Methods Summit, click here to learn more.
9. ICER's QALY-Based Study Topics: Hypertrophic Cardiomyopathy, Myasthenia Gravis, Atopic Dermatitis, Alzheimer's Disease, Asthma, Type 2 Diabetes, click here to provide patient input.
10. Upcoming Events and Webinars, see details below.
11. Medical Journal Articles, see details below.
12. AHRQ Effective Program Updates, see details below.
1. Value Assessment in the News, see details below.
2. Is the QALY Fit for Purpose? Click here to read the journal.
3. 2021 PCORI Annual Meeting Scheduled for Nov. 17-19, click here for details.
4. Public Comment on PCORI Proposed National Priorities for Health, see details below.
5. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
6. States Rely on Template Legislation that References ICER and Discriminatory Metrics, see details below.
7. Emerging Threats in States for Use of Discriminatory Metrics, see details below.
8. IVI to Hold Second Methods Summit, click here to learn more.
9. ICER's QALY-Based Study Topics: Hypertrophic Cardiomyopathy, Myasthenia Gravis, Atopic Dermatitis, Alzheimer's Disease, Asthma, Type 2 Diabetes, click here to provide patient input.
10. Upcoming Events and Webinars, see details below.
11. Medical Journal Articles, see details below.
12. AHRQ Effective Program Updates, see details below.
1. Value Assessment in the News
Federal policymakers are considering value assessment options, from domestic reference pricing to foreign reference pricing. As policymakers consider alternatives to QALYs and the evLYG which is derived from a QALY, it is important that policymakers to understand value assessment, how discriminatory metrics are used in value assessment and how innovators like PCORI and IVI are developing new methods for assessing cost and economic data related to care and treatment. For more information about evLYG, please view this PIPC Chairman blog and this Twitter thread that explains the evLYG method in more detail. We urge you to share widely the concerns from the National Council on Disability related to QALYs and evLYG (p. 61) and CBO’s use of QALYs to score H.R. 3 as a reminder that patients and people with disabilities oppose the use of this metric and metrics related to it like evLYG. Click here to view principles for value assessment centered on patients and people with disabilities.
2. AJMC: Is the QALY Fit for Purpose?
The American Journal of Managed Care (AJMC) published a journal article that examines some of the flaws of the quality-adjusted-life-years metric. "We argue that the QALY does not currently pass these 3 tests to an acceptable standard," the authors write. "We argue for methods to achieve incremental improvement in both the scientific and ethical standards used in constructing measures such as the QALY and for an end to the inertia in improving a measurement system that is widely considered inadequate." Click here to read the journal.
3. 2021 PCORI Annual Meeting Scheduled for Nov. 17-19
PCORI announced that its 2021 annual meeting will be held virtually on Wednesday, November 17 through Friday, November 19. The event serves as a report to the nation on PCORI’s progress in funding initiatives to determine which care approaches work best, for whom, and under what circumstances. Click here for details.
4. Public Comment on PCORI Proposed National Priorities for Health
In 2020, PCORI started an initiative to identify new priorities to direct its work in the coming years. PCORI's Board of Governors approved posting for public comment of five proposed National Priorities for Health developed in consultation with stakeholders, advisors, and experts. Public comment on the priorities began June 28 and will run through August 27. The five priorities are:
Click here to read more and to submit comments.
5. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage?
Other countries are often referenced as examples of how the use of QALYs or similar cost-based thresholds impact access to care.
6. States Rely on Template Legislation that References ICER and Discriminatory Metrics
We are seeing many states reference pieces of template legislation put forward by the National Association of Health Policy (NASHP). Though the intent of these bills is to lower healthcare costs, several implicate the discriminatory Quality-Adjusted Life Year (QALY) and others rely on the Institute for Clinical and Economic Review (ICER) as the sole source for evidence. The three primary bills are:
7. Emerging Threats in States for Use of Discriminatory Metrics
Several states are considering policies that would reference entities such as the Institute for Clinical and Economic Review (ICER), which calls the discriminatory quality-adjusted life year (QALY) the “gold standard” for value assessment or are considering policies to import QALY-based decisions from other countries. Yet, federal policymakers have emphasized that the use of discriminatory metrics is subject to civil rights laws such as the Americans with Disabilities Act. Click here to view the Value Our Health state template legislation that would protect people with disabilities and chronic conditions from the use of QALYs and similar metrics developed by third parties such as ICER in decisions related to reimbursement and coverage, as well as ensure their engagement in decision-making. Click here to view a one pager about the flaws in ICER’s methodology. Click here to view information from experts on the downside of referencing foreign countries. Click here to learn about statutory protections against use of QALYs. Other states are taking positive steps to ban the use of the QALY and other discriminatory metrics.
Maryland
The Maryland legislature passed legislation earlier this year creating a Prescription Drug Affordability Board. The legislation did not include protections for patients and people with disabilities such as barring the use of discriminatory QALYs. The Board has begun meeting to hear from stakeholders, including hearing a presentation from the Institute for Clinical and Economic Review which relies on QALYs to assess treatment value. Legislation was introduced this year to bar the use of QALYs and implement patient protections that has not advanced in the legislature. Click here to view the Board meeting agenda and here to view reference to the discussion on how to leverage ICER studies. Click here to view HB 1167 which would bar the use of QALYs in Maryland.
Colorado
The Governor of Colorado has signed into law SB 21-175, which creates a prescription drug affordability review board. SB 21-75 did include an amendment that prohibits the use of QALYs in the section of the bill that determines an upper payment limit, but it does not include similar protections in the sections establishing the commission’s authority to perform value assessments for treatments would prevent QALYs from being used to target treatments for people with disabilities and older adults. In some states these types of boards and commissions have led to referencing value assessments based on QALYs from the Institute for Clinical and Economic Review (ICER). In Massachusetts, the Health Policy Commission went so far as to contract with ICER. In order to mitigate this, it is important that the Board has representation from patients and people with disabilities. The Governors office is currently accepting applications for both the Colorado Prescription Drug Affordability Review Board and the Colorado Prescription Drug Affordability Advisory Council.
Massachusetts
The Massachusetts House and Senate have each taken the positive step of introducing An Act Advancing Health Care Research and Decision-Making Centered on Patients and People with Disabilities, H.201 and S.753. This bill would enshrine essential patient protections including a ban on the use of the QALY, a requirement for research to meet patient-centeredness criteria, and robust engagement of the patient and disability communities in health care decision making.
California
CA AB 1130, the California Health Care Quality and Affordability Act, is being considered in California to create an Office of Health Care Affordability. Similar to the Massachusetts Health Policy Commission, the legislation would authorize reviewing health costs and "require the office to set priority standards for various health care metrics.” In Massachusetts, the Health Policy Commission went so far as to contract with ICER. We understand the legislation is likely to pass the Appropriations Committee this session, and amendments to protect patients from discrimination has not been considered. It was originally proposed by Governor Gavin Newsom in the California Budget for 2021-22 which included a proposal for a new Office of Health Care Affordability to be housed within the Office of Statewide Health Planning and Development (OSHPD) that would seek to promote “cost efficient” care. It is anticipated that this office could advance as an independent bill, or as part of the California budget. The Value Our Health template language has not been introduced or passed in California to protect patients from the use of QALYs and discriminatory considerations of cost effectiveness.
8. IVI to Hold Second Methods Summit
The Innovation and Value Initiative (IVI) announced that it will hold a second methods summit to drive patient-centered value assessment. The multi-stakeholder event is intended to drive consensus on priority patient inputs, methods, and research that more fully represent the patient perspective in comparative effectiveness research and economic evaluations of health care interventions. The three-part series will take place in Fall 2021. Overall, the goal of IVI’s summit is to define an action agenda for patient-centered outcomes research and cost-effectiveness research by exploring patient outcomes, beyond traditional clinical factors, that represent important impacts to patients and must be accounted for in economic analyses. Click here to learn more.
9. ICER's QALY-Based Study Topics: Hypertrophic Myocardiopathy, Myasthenia Gravis, Atopic Dermatitis, Alzheimer's Disease, Asthma, Type 2 Diabetes
The Institute for Clinical Economic Review (ICER) conducts cost effectiveness studies for insurers using the cost-per-QALY methodology. ICER provides guidance on its website for patients and patient advocates to provide direct input related to their experiences with the disease. Click here to provide patient input. Click here to view the topics and deadlines.
10. Upcoming Events and Webinars
Real World Evidence in the COVID Era: The National COVID Cohort Collaborative
July 28, 2021
Click here to view.
PCORI Advisory Panel on Clinical Trials Summer 2021 Meeting
August 10, 2021
Click here to view.
Whistleblowers of America: Workplace Promise Institute 2021 Conference
September 9-10, 2021
Click here to view.
PCORI Annual Meeting
November 17-19, 2021
Click here to view.
11. Medical Journal Articles
Toward Better Data Dashboards for US Drug Value Assessments, click here to view.
Prevalence of Avoidable and Bias-Inflicting Methodological Pitfalls in Real-World Studies of Medication Safety and Effectiveness, click here to view.
Association of Co-Pay Elimination With Medication Adherence and Total Cost, click here to view.
The Utility of Patient Engagement in Drug Research and Development, click here to view.
Patient-Powered Research Networks of the Autoimmune Research Collaborative: Rationale, Capacity, and Future Directions, click here to view.
Enhancing Patient Research Partner Engagement: Research in Psoriatic Arthritis, click here to view.
Patient Voices in Value-Based Cancer Care: Priorities for the Biden Administration, click here to view.
The Dollar or Disease Burden: Caps on Healthcare Spending May Save Money, but at What “Cost” to Patients?, click here to view.
12. AHRQ Effective Program Updates
Systematic Review: Breast Reconstruction After Mastectomy, click here to view.
Surveillance Report: Living Systematic Review on Cannabis and Other Plant-Based Treatments for Chronic Pain. Click here to view.
Systematic Review: Prehospital Airway Management. Click here to view.
Systematic Review: Radiation Therapy for Brain Metastases. Click here to view.
Systematic Review: Safety of Vaccines Used for Routine Immunization in the United States: An Update. Click here to view.
Research Protocol: Evaluation of Mental Health Applications. Click here to view.
Maternal, Fetal, and Child Outcomes of Mental Health Treatments in Women: A Systematic Review of Perinatal Pharmacologic Interventions. Click here to view.
Federal policymakers are considering value assessment options, from domestic reference pricing to foreign reference pricing. As policymakers consider alternatives to QALYs and the evLYG which is derived from a QALY, it is important that policymakers to understand value assessment, how discriminatory metrics are used in value assessment and how innovators like PCORI and IVI are developing new methods for assessing cost and economic data related to care and treatment. For more information about evLYG, please view this PIPC Chairman blog and this Twitter thread that explains the evLYG method in more detail. We urge you to share widely the concerns from the National Council on Disability related to QALYs and evLYG (p. 61) and CBO’s use of QALYs to score H.R. 3 as a reminder that patients and people with disabilities oppose the use of this metric and metrics related to it like evLYG. Click here to view principles for value assessment centered on patients and people with disabilities.
2. AJMC: Is the QALY Fit for Purpose?
The American Journal of Managed Care (AJMC) published a journal article that examines some of the flaws of the quality-adjusted-life-years metric. "We argue that the QALY does not currently pass these 3 tests to an acceptable standard," the authors write. "We argue for methods to achieve incremental improvement in both the scientific and ethical standards used in constructing measures such as the QALY and for an end to the inertia in improving a measurement system that is widely considered inadequate." Click here to read the journal.
3. 2021 PCORI Annual Meeting Scheduled for Nov. 17-19
PCORI announced that its 2021 annual meeting will be held virtually on Wednesday, November 17 through Friday, November 19. The event serves as a report to the nation on PCORI’s progress in funding initiatives to determine which care approaches work best, for whom, and under what circumstances. Click here for details.
4. Public Comment on PCORI Proposed National Priorities for Health
In 2020, PCORI started an initiative to identify new priorities to direct its work in the coming years. PCORI's Board of Governors approved posting for public comment of five proposed National Priorities for Health developed in consultation with stakeholders, advisors, and experts. Public comment on the priorities began June 28 and will run through August 27. The five priorities are:
- Increase Evidence for Existing Interventions and Emerging Innovations in Health
- Enhance Infrastructure to Accelerate Patient-Centered Outcomes Research
- Advance the Science of Dissemination, Implementation, and Health Communication
- Achieve Health Equity
- Accelerate Progress Toward an Integrated Learning Health System
Click here to read more and to submit comments.
5. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage?
Other countries are often referenced as examples of how the use of QALYs or similar cost-based thresholds impact access to care.
- Australia: Cystic fibrosis patients are being hamstrung by the PBS’ refusal to cover life-changing medications. Similarly, cancer patients face hurdles in access to drugs that could potentially improve their long-term health outcomes.
- New Zealand: Access to Trikafta for cystic fibrosis patients in New Zealand has been severely limited, forcing patients to self-fund their own treatment. Click here to read more.
- United Kingdom: Arbitrary restrictions on treatments in the U.K. has put families with children that suffer from spinal muscular atrophy in a difficult position.
6. States Rely on Template Legislation that References ICER and Discriminatory Metrics
We are seeing many states reference pieces of template legislation put forward by the National Association of Health Policy (NASHP). Though the intent of these bills is to lower healthcare costs, several implicate the discriminatory Quality-Adjusted Life Year (QALY) and others rely on the Institute for Clinical and Economic Review (ICER) as the sole source for evidence. The three primary bills are:
- Canadian Reference Pricing -- This piece of template legislation directly references the prices paid for drugs in five Canadian provinces. Before applying for coverage by the provinces, all drugs must complete a Common Drug Review by CADTH, which uses QALYs. The result of this is that in Canada is that many individuals living with disabilities are unable to receive the treatments and care they need. The National Council on Disability (NCD) warned in its 2019 report that similar coverage denials and loss of access to care could also be the outcome if the United States if we reference other countries.
- Unsupported Price Increase -- This piece of template legislation relies on one report by ICER to determine whether a price increase on certain therapies were supported by additional evidence. This is concerning as it codifies reference to an independent entity lacking oversight and accountability as a sole source of information. ICER is the sole arbiter as to what evidence it uses to develop this report, and patients have consistently expressed concern with ICER’s lack of transparency and omission of real-world evidence. ICER also has the ability to change the inputs and methodology of the report without oversight. Though the 2020 report does not use the QALY, it is possible that future reports will.
- Drug Pricing Review Board -- This piece of template legislation establishes a drug review board or commission. The goal of the board is to allow the state to review and evaluate the reimbursement rate and/or coverage for pharmaceuticals. Most have fairly broad parameters of how they can assess a drug’s value, and specifically permit the state to rely on third-party research or contract directly with a third-party for the purpose of fulfilling its duties. As has happened in other states such as New York and Massachusetts, without patient protections, these bills allow the new state board or commission to reference value assessments that rely on QALY and similar metrics. It is imperative that these boards has patient and disability representation and that a QALY ban is included in the legislation.
7. Emerging Threats in States for Use of Discriminatory Metrics
Several states are considering policies that would reference entities such as the Institute for Clinical and Economic Review (ICER), which calls the discriminatory quality-adjusted life year (QALY) the “gold standard” for value assessment or are considering policies to import QALY-based decisions from other countries. Yet, federal policymakers have emphasized that the use of discriminatory metrics is subject to civil rights laws such as the Americans with Disabilities Act. Click here to view the Value Our Health state template legislation that would protect people with disabilities and chronic conditions from the use of QALYs and similar metrics developed by third parties such as ICER in decisions related to reimbursement and coverage, as well as ensure their engagement in decision-making. Click here to view a one pager about the flaws in ICER’s methodology. Click here to view information from experts on the downside of referencing foreign countries. Click here to learn about statutory protections against use of QALYs. Other states are taking positive steps to ban the use of the QALY and other discriminatory metrics.
Maryland
The Maryland legislature passed legislation earlier this year creating a Prescription Drug Affordability Board. The legislation did not include protections for patients and people with disabilities such as barring the use of discriminatory QALYs. The Board has begun meeting to hear from stakeholders, including hearing a presentation from the Institute for Clinical and Economic Review which relies on QALYs to assess treatment value. Legislation was introduced this year to bar the use of QALYs and implement patient protections that has not advanced in the legislature. Click here to view the Board meeting agenda and here to view reference to the discussion on how to leverage ICER studies. Click here to view HB 1167 which would bar the use of QALYs in Maryland.
Colorado
The Governor of Colorado has signed into law SB 21-175, which creates a prescription drug affordability review board. SB 21-75 did include an amendment that prohibits the use of QALYs in the section of the bill that determines an upper payment limit, but it does not include similar protections in the sections establishing the commission’s authority to perform value assessments for treatments would prevent QALYs from being used to target treatments for people with disabilities and older adults. In some states these types of boards and commissions have led to referencing value assessments based on QALYs from the Institute for Clinical and Economic Review (ICER). In Massachusetts, the Health Policy Commission went so far as to contract with ICER. In order to mitigate this, it is important that the Board has representation from patients and people with disabilities. The Governors office is currently accepting applications for both the Colorado Prescription Drug Affordability Review Board and the Colorado Prescription Drug Affordability Advisory Council.
Massachusetts
The Massachusetts House and Senate have each taken the positive step of introducing An Act Advancing Health Care Research and Decision-Making Centered on Patients and People with Disabilities, H.201 and S.753. This bill would enshrine essential patient protections including a ban on the use of the QALY, a requirement for research to meet patient-centeredness criteria, and robust engagement of the patient and disability communities in health care decision making.
California
CA AB 1130, the California Health Care Quality and Affordability Act, is being considered in California to create an Office of Health Care Affordability. Similar to the Massachusetts Health Policy Commission, the legislation would authorize reviewing health costs and "require the office to set priority standards for various health care metrics.” In Massachusetts, the Health Policy Commission went so far as to contract with ICER. We understand the legislation is likely to pass the Appropriations Committee this session, and amendments to protect patients from discrimination has not been considered. It was originally proposed by Governor Gavin Newsom in the California Budget for 2021-22 which included a proposal for a new Office of Health Care Affordability to be housed within the Office of Statewide Health Planning and Development (OSHPD) that would seek to promote “cost efficient” care. It is anticipated that this office could advance as an independent bill, or as part of the California budget. The Value Our Health template language has not been introduced or passed in California to protect patients from the use of QALYs and discriminatory considerations of cost effectiveness.
8. IVI to Hold Second Methods Summit
The Innovation and Value Initiative (IVI) announced that it will hold a second methods summit to drive patient-centered value assessment. The multi-stakeholder event is intended to drive consensus on priority patient inputs, methods, and research that more fully represent the patient perspective in comparative effectiveness research and economic evaluations of health care interventions. The three-part series will take place in Fall 2021. Overall, the goal of IVI’s summit is to define an action agenda for patient-centered outcomes research and cost-effectiveness research by exploring patient outcomes, beyond traditional clinical factors, that represent important impacts to patients and must be accounted for in economic analyses. Click here to learn more.
9. ICER's QALY-Based Study Topics: Hypertrophic Myocardiopathy, Myasthenia Gravis, Atopic Dermatitis, Alzheimer's Disease, Asthma, Type 2 Diabetes
The Institute for Clinical Economic Review (ICER) conducts cost effectiveness studies for insurers using the cost-per-QALY methodology. ICER provides guidance on its website for patients and patient advocates to provide direct input related to their experiences with the disease. Click here to provide patient input. Click here to view the topics and deadlines.
- Type 2 Diabetes: Draft Scoping Document available, comment period open through 7/21/2021.
- Hypertrophic Cardiomyopathy: Model Analysis Plan available. 8/18/2021: Draft Evidence Report. Meeting 10/22/2021: CTAF will deliberate and vote on evidence presented in ICER’s report on therapies for obstructive hypertrophic cardiomyopathy.
- Myasthenia Gravis: Model Analysis Plan available. 7/22/2021: Draft Evidence Report. Meeting 9/24/2021: New England CEPAC will deliberate and vote on evidence presented in ICER’s report on therapies for myasthenia gravis.
- Atopic Dermatitis: Evidence Report AVAILABLE. Meeting 7/23/2021: An assessment of treatments for atopic dermatitis by the New England CEPAC.
- Hereditary Angioedema: 7/27/2021: Final Updated Assessment with RWE Update.
- Unsupported Price Increase: 11/16/2021: Final Report.
- Asthma: Research Protocol available. 8/2/2021: Model Analysis Plan. Meeting 11/19/2021: New England CEPAC will deliberate and vote on evidence presented in ICER’s report on therapies for severe asthma.
- Alzheimer's Disease: Evidence Report available. Final Evidence Report to be published on 8/5/21.
- Fair Access: Coverage Policies in 2020: Protocol Available. 10/20/2021: Final Report.
10. Upcoming Events and Webinars
Real World Evidence in the COVID Era: The National COVID Cohort Collaborative
July 28, 2021
Click here to view.
PCORI Advisory Panel on Clinical Trials Summer 2021 Meeting
August 10, 2021
Click here to view.
Whistleblowers of America: Workplace Promise Institute 2021 Conference
September 9-10, 2021
Click here to view.
PCORI Annual Meeting
November 17-19, 2021
Click here to view.
11. Medical Journal Articles
Toward Better Data Dashboards for US Drug Value Assessments, click here to view.
Prevalence of Avoidable and Bias-Inflicting Methodological Pitfalls in Real-World Studies of Medication Safety and Effectiveness, click here to view.
Association of Co-Pay Elimination With Medication Adherence and Total Cost, click here to view.
The Utility of Patient Engagement in Drug Research and Development, click here to view.
Patient-Powered Research Networks of the Autoimmune Research Collaborative: Rationale, Capacity, and Future Directions, click here to view.
Enhancing Patient Research Partner Engagement: Research in Psoriatic Arthritis, click here to view.
Patient Voices in Value-Based Cancer Care: Priorities for the Biden Administration, click here to view.
The Dollar or Disease Burden: Caps on Healthcare Spending May Save Money, but at What “Cost” to Patients?, click here to view.
12. AHRQ Effective Program Updates
Systematic Review: Breast Reconstruction After Mastectomy, click here to view.
Surveillance Report: Living Systematic Review on Cannabis and Other Plant-Based Treatments for Chronic Pain. Click here to view.
Systematic Review: Prehospital Airway Management. Click here to view.
Systematic Review: Radiation Therapy for Brain Metastases. Click here to view.
Systematic Review: Safety of Vaccines Used for Routine Immunization in the United States: An Update. Click here to view.
Research Protocol: Evaluation of Mental Health Applications. Click here to view.
Maternal, Fetal, and Child Outcomes of Mental Health Treatments in Women: A Systematic Review of Perinatal Pharmacologic Interventions. Click here to view.
