In this week's edition...
— RSVP Today for #PIPCForum2023! See details below.
— The Future State of Value-Based Care. Click here to watch the video.
— AHRQ: Vanquishing Healthcare Disparities by Advancing Healthcare Equity. See details below.
— How to Use PCORnet® in Your Research. Click here to learn more.
— PCORI Funds 4 New Large Studies Focused on Maternal Health. Click here to read the announcement.
— PCORI Seeks Nominations for Advisory Panels. Click here to learn more, to apply to serve on an advisory panel, or submit a nomination.
— Emerging Threats in States for Use of Discriminatory Metrics. See details below.
— What Happens in Countries Using QALYs and Cost-Based Thresholds? See details below.
— ICER's QALY-Based Study Topics. See details below.
— Upcoming Events and Webinars. See details below.
— AHRQ Effective Program Updates. See details below.
— RSVP Today for #PIPCForum2023! See details below.
— The Future State of Value-Based Care. Click here to watch the video.
— AHRQ: Vanquishing Healthcare Disparities by Advancing Healthcare Equity. See details below.
— How to Use PCORnet® in Your Research. Click here to learn more.
— PCORI Funds 4 New Large Studies Focused on Maternal Health. Click here to read the announcement.
— PCORI Seeks Nominations for Advisory Panels. Click here to learn more, to apply to serve on an advisory panel, or submit a nomination.
— Emerging Threats in States for Use of Discriminatory Metrics. See details below.
— What Happens in Countries Using QALYs and Cost-Based Thresholds? See details below.
— ICER's QALY-Based Study Topics. See details below.
— Upcoming Events and Webinars. See details below.
— AHRQ Effective Program Updates. See details below.
What We're Reading
1. RSVP Today for #PIPCForum2023! The Partnership to Improve Patient Care (PIPC) will air its 14th annual forum virtually on Monday, December 11 at 12pm ET.
For #PIPCForum2023, we will be welcoming participants to discuss with us how to improve patient engagement in policy decisions as well as efforts to address how value assessments are developed and used in health decisions. We look forward to learning about the experiences of patients that have engaged with policymakers leading implementation of the IRA’s Medicare Drug Negotiation Program and state Prescription Drug Affordability Programs.
We also look forward to a lively discussion about the need for value assessment of health care to be centered on patients and people with disabilities with protections against their use to discriminate or promote health inequities. PIPC will engage in a conversation about the importance of policies that advance the collection of data and conduct of research centered on the needs of people with disabilities. Please visit the PIPC website here to RSVP.
2. The Future State of Value-Based Care. In his keynote address, Dr. Stefan Larsson, the Chairman and Co-founder of the International Consortium for Health Outcomes Measurement, describes the need for value-based care models over fee-for-service to improve health care quality, and the evolution mindset needed for health care leaders to shape care of the future that focuses on four levers central to adaptive systems. He stresses the importance of outcomes measurement and that tools already exist for clinicians and leaders to begin the journey toward value and outcomes that actually matter to patients.
3. AHRQ: Vanquishing Healthcare Disparities by Advancing Healthcare Equity. A blog from AHRQ leaders notes that Executive Order 13985, Advancing Racial Equity and Support for Underserved Communities Through the Federal Government, mandated federal agencies to launch a whole-of-government approach to advance equity. In response, AHRQ has thoroughly examined equity and its connections to Agency activities in alignment with its mission. As part of that work, AHRQ explicitly recognized that patients and communities must be centered in all efforts to advance equity. AHRQ also noted research literature identifying the use of healthcare algorithms as impacting racial/ethnic disparities in care and discussing approaches to identify and mitigate existing biases. Such efforts are consistent with eliminating the use of biased value assessments in health decisions, a point made last year by advocates in California in response to the Attorney General’s efforts against use of biased algorithms. Click here to view the AHRQ blog. Click here to view the letter from CA advocates.
4. How to Use PCORnet® in Your Research. PCORnet®, the National Patient-Centered Clinical Research Network, is now offering four new tools that can empower you to maximize every aspect research and conduct patient-centered CER more efficiently. These tools will: Speed prep-to-research queries; Speed the contracting process for PCORI-funded studies; Support fast and easy search of crucial details about PCORnet-participating sites; Facilitate fast and consistent IRB applications in your PCORnet-leveraged study.
5. PCORI Funds 4 New Large Studies Focused on Maternal Health. The Patient-Centered Outcomes Research Institute (PCORI) has announced funding awards of $80.5 million to support four new studies focused on disparities in maternal health. The clinical effectiveness research studies will take into account the clinical and social factors that contribute to inequities in maternal morbidity and mortality. The trials will focus on populations disproportionately experiencing adverse outcomes, as well as those in rural areas or with lower incomes.
6. PCORI Seeks Nominations for Advisory Panels. PCORI is currently seeking nominations for appointments to its advisory panels. These multi-stakeholder advisory panels must include patients, caregivers, or patient advocates. PCORI advisory panels do not serve in an official decision-making capacity, but their recommendations and advice will be taken into consideration by PCORI. PIPC encourages patients, caregivers, and patient advocates to apply or to submit nominations to serve on PCORI's five advisory panels on: Clinical Effectiveness and Decision Science, Clinical Trials, Healthcare Delivery & Disparities Research, Patient Engagement, and Rare Disease. The application is open through March 29, 2024. Click here to learn more, to apply to serve on an advisory panel, or submit a nomination.
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, and others are considering policies to import QALY-based coverage and reimbursement decisions from other countries that restrict access to care. We encourage you to keep track of all state-based threats using this new website tracking state activities. Key issues are highlighted below.
Oregon
The Oregon legislature passed SB 844 in 2021 creating a Prescription Drug Affordability Board, which was updated based on the Board’s recommendations in 2023 by SB 192. While the legislation included language barring consideration of QALYs and similar formulas, the Board has engaged the Program on Regulation, Therapeutics, and Law (PORTAL), per their meeting agenda for November 15. PORTAL has a subcontract with the Institute for Clinical and Economic Review (ICER) for its work with the Massachusetts Health Policy Commission and has presented on the merits of the QALY and evLYG measures to the Colorado Prescription Drug Affordability Board. This raises concerns about the evidentiary basis for the Board’s decisions and the potential for reference to discriminatory measures of cost effectiveness.
The Oregon Board voted to advance an affordability review of 26 drugs at the October 18, 2023 meeting, with an ambitious plan to conduct the reviews by February. The Board’s rules for conducting an affordability review does not include a robust process for engaging patients and people with disabilities in their decisions. The Oregon PDAB will hold a public hearing on December 13, 2023 from 1:30-4pm Pacific time. Guidance for providing public testimony is available here. Separately, the Oregon Drug Price Transparency Program will hold their annual meeting on December 7, 2023, with registration available here. The Pacific Northwest Advocates Confab is a helpful resource - click here to view their webinar.
Click here to join Disability Rights Oregon in advocating for legislation banning use of QALYs.
Colorado
Colorado passed legislation in 2021 creating a Prescription Drug Affordability Review Board. The legislation included language stating that the Upper Payment Limit for selected drugs "shall not consider research or methods that employ a dollars-per-quality adjusted life year, or similar measure, that discounts the value of a life because of an individual's disability or age.” However, this limitation did not clearly extend to the process for determining the selected drugs or the affordability review. Presentations to the Colorado Board from entities such as the Program on Regulation, Therapeutics, and Law (PORTAL) related to cost effectiveness analyses have referenced the merits of using of a cost-per-QALY or the equal value of life-year gained (evLYG) in estimating cost effectiveness of treatments, indicating that these discriminatory measures may have influenced how Colorado selected the drugs and may influence the affordability review. Concerns about the potential for discrimination were described in a letter to the Board from 16 patient and disability organizations. The PDAB offers several opportunities for patients to engage and ensure their voice is heard on these topics. The next PDAB Meeting will be held Friday, December 8 at 10 am MT. Click here to register. Click here to learn more from a Colorado patient with Cystic Fibrosis.
Massachusetts
The Massachusetts Senate’s Ways and Means Committees was referred the “PACT Act”, reported favorably on October 30, 2023 by the Joint Committee on Health Care Financing. The legislation includes a provision calling for the health policy commission not to base its determinations on measures such as QALYs. The Senate rejected an amendment (#19) pushed by Massachusetts advocates calling for transparency related to the use of cost effectiveness analyses by the commission, as well as increased requirements for stakeholder engagement. The Senate passed the bill on November 15, 2023. Massachusetts advocates will work with the House toward language that more clearly provides for public transparency of the evidence under consideration and opportunities for engagement from patients and people with disabilities.
International News: What Happens in Countries Using QALYs and Cost-Based Thresholds?
Other countries are often referenced as examples of how the use of QALYs or similar cost-based thresholds impact access to care.
ICER's QALY-Based Study Topics
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.
Upcoming Events and Webinars
Ensuring Equity in Implementation of IRA Drug Price Negotiations
December 5, 2023
Click here to view.
PCORI Board of Governors Meeting
December 12, 2023
Click here to view.
Medical Journal Articles
Alternative approaches to measuring value: an update on innovative methods in the context of the United States Medicare drug price negotiation program. Click here to read the article.
Economic Outcomes in Patient-Centered Outcomes Research: A Paradigm Shift. Click here to read the article.
Visualization of Evidence for Shared Decision Making. Click here to read the article.
Avenues for Strengthening PCORnet’s Capacity to Advance Patient-Centered Economic Outcomes in Patient-Centered Outcomes Research. Click here to read the article.
Linking Medicare-Medicaid Claims for Patient-Centered Outcomes Research Among Dual-Eligible Beneficiaries. Click here to read the article.
Gene Therapies for Sickle Cell Disease: Effectiveness and Value. Click here to read the article.
White Paper: Improving Patient Subgroup Representation with Real-World Data. Click here to read the article.
A Clinical Pathway to Well-Being: Putting Patient Priorities at the Center of Care. Click here to read the article.
Comparative Effectiveness of Treatments for Rheumatoid Arthritis in Clinical Practice: A Systematic Review. Click here to read the article.
QALYs: The Math Doesn’t Work. Click here to read the article.
The Implementation of Value-Based Frameworks, Clinical Care Pathways, and Alternative Payment Models for Cancer Care in the United States. Click here to view.
Perspectives on Patient-Reported Outcome Data After Treatment Discontinuation in Cancer Clinical Trials. Click here to view.
AHRQ Effective Program Updates
Technical Brief: Measuring Healthcare Organization Characteristics in Cancer Care Delivery Research. Click here to view.
Systematic Review: Postpartum Care up to 1 Year After Pregnancy: A Systematic Review and Meta-analysis. Click here to view.
Draft Report: Impact of Healthcare Algorithms on Racial and Ethnic Disparities in Health and Healthcare. Click here to view.
Systematic Review: Partian Breast Irradiation for Breast Cancer. Click here to view.
Systematic Review: Use of Telehealth During the COVID-19 Era. Click here to view.
Research Report: Analysis of Requirements for Coverage with Evidence Development (CED) - Topic Refinement. Click here to view.
Technical Brief: Infection Prevention and Control for the Emergency Medical Services and 911 Workforce. Click here to view.
Systematic Review: Management of Infantile Epilepsies. Click here to view.
1. RSVP Today for #PIPCForum2023! The Partnership to Improve Patient Care (PIPC) will air its 14th annual forum virtually on Monday, December 11 at 12pm ET.
For #PIPCForum2023, we will be welcoming participants to discuss with us how to improve patient engagement in policy decisions as well as efforts to address how value assessments are developed and used in health decisions. We look forward to learning about the experiences of patients that have engaged with policymakers leading implementation of the IRA’s Medicare Drug Negotiation Program and state Prescription Drug Affordability Programs.
We also look forward to a lively discussion about the need for value assessment of health care to be centered on patients and people with disabilities with protections against their use to discriminate or promote health inequities. PIPC will engage in a conversation about the importance of policies that advance the collection of data and conduct of research centered on the needs of people with disabilities. Please visit the PIPC website here to RSVP.
2. The Future State of Value-Based Care. In his keynote address, Dr. Stefan Larsson, the Chairman and Co-founder of the International Consortium for Health Outcomes Measurement, describes the need for value-based care models over fee-for-service to improve health care quality, and the evolution mindset needed for health care leaders to shape care of the future that focuses on four levers central to adaptive systems. He stresses the importance of outcomes measurement and that tools already exist for clinicians and leaders to begin the journey toward value and outcomes that actually matter to patients.
3. AHRQ: Vanquishing Healthcare Disparities by Advancing Healthcare Equity. A blog from AHRQ leaders notes that Executive Order 13985, Advancing Racial Equity and Support for Underserved Communities Through the Federal Government, mandated federal agencies to launch a whole-of-government approach to advance equity. In response, AHRQ has thoroughly examined equity and its connections to Agency activities in alignment with its mission. As part of that work, AHRQ explicitly recognized that patients and communities must be centered in all efforts to advance equity. AHRQ also noted research literature identifying the use of healthcare algorithms as impacting racial/ethnic disparities in care and discussing approaches to identify and mitigate existing biases. Such efforts are consistent with eliminating the use of biased value assessments in health decisions, a point made last year by advocates in California in response to the Attorney General’s efforts against use of biased algorithms. Click here to view the AHRQ blog. Click here to view the letter from CA advocates.
4. How to Use PCORnet® in Your Research. PCORnet®, the National Patient-Centered Clinical Research Network, is now offering four new tools that can empower you to maximize every aspect research and conduct patient-centered CER more efficiently. These tools will: Speed prep-to-research queries; Speed the contracting process for PCORI-funded studies; Support fast and easy search of crucial details about PCORnet-participating sites; Facilitate fast and consistent IRB applications in your PCORnet-leveraged study.
5. PCORI Funds 4 New Large Studies Focused on Maternal Health. The Patient-Centered Outcomes Research Institute (PCORI) has announced funding awards of $80.5 million to support four new studies focused on disparities in maternal health. The clinical effectiveness research studies will take into account the clinical and social factors that contribute to inequities in maternal morbidity and mortality. The trials will focus on populations disproportionately experiencing adverse outcomes, as well as those in rural areas or with lower incomes.
6. PCORI Seeks Nominations for Advisory Panels. PCORI is currently seeking nominations for appointments to its advisory panels. These multi-stakeholder advisory panels must include patients, caregivers, or patient advocates. PCORI advisory panels do not serve in an official decision-making capacity, but their recommendations and advice will be taken into consideration by PCORI. PIPC encourages patients, caregivers, and patient advocates to apply or to submit nominations to serve on PCORI's five advisory panels on: Clinical Effectiveness and Decision Science, Clinical Trials, Healthcare Delivery & Disparities Research, Patient Engagement, and Rare Disease. The application is open through March 29, 2024. Click here to learn more, to apply to serve on an advisory panel, or submit a nomination.
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, and others are considering policies to import QALY-based coverage and reimbursement decisions from other countries that restrict access to care. We encourage you to keep track of all state-based threats using this new website tracking state activities. Key issues are highlighted below.
- Click here to learn more about the potential for Prescription Drug Affordability Boards to discriminate by incorporating the use of QALYS.
- 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.
Oregon
The Oregon legislature passed SB 844 in 2021 creating a Prescription Drug Affordability Board, which was updated based on the Board’s recommendations in 2023 by SB 192. While the legislation included language barring consideration of QALYs and similar formulas, the Board has engaged the Program on Regulation, Therapeutics, and Law (PORTAL), per their meeting agenda for November 15. PORTAL has a subcontract with the Institute for Clinical and Economic Review (ICER) for its work with the Massachusetts Health Policy Commission and has presented on the merits of the QALY and evLYG measures to the Colorado Prescription Drug Affordability Board. This raises concerns about the evidentiary basis for the Board’s decisions and the potential for reference to discriminatory measures of cost effectiveness.
The Oregon Board voted to advance an affordability review of 26 drugs at the October 18, 2023 meeting, with an ambitious plan to conduct the reviews by February. The Board’s rules for conducting an affordability review does not include a robust process for engaging patients and people with disabilities in their decisions. The Oregon PDAB will hold a public hearing on December 13, 2023 from 1:30-4pm Pacific time. Guidance for providing public testimony is available here. Separately, the Oregon Drug Price Transparency Program will hold their annual meeting on December 7, 2023, with registration available here. The Pacific Northwest Advocates Confab is a helpful resource - click here to view their webinar.
Click here to join Disability Rights Oregon in advocating for legislation banning use of QALYs.
Colorado
Colorado passed legislation in 2021 creating a Prescription Drug Affordability Review Board. The legislation included language stating that the Upper Payment Limit for selected drugs "shall not consider research or methods that employ a dollars-per-quality adjusted life year, or similar measure, that discounts the value of a life because of an individual's disability or age.” However, this limitation did not clearly extend to the process for determining the selected drugs or the affordability review. Presentations to the Colorado Board from entities such as the Program on Regulation, Therapeutics, and Law (PORTAL) related to cost effectiveness analyses have referenced the merits of using of a cost-per-QALY or the equal value of life-year gained (evLYG) in estimating cost effectiveness of treatments, indicating that these discriminatory measures may have influenced how Colorado selected the drugs and may influence the affordability review. Concerns about the potential for discrimination were described in a letter to the Board from 16 patient and disability organizations. The PDAB offers several opportunities for patients to engage and ensure their voice is heard on these topics. The next PDAB Meeting will be held Friday, December 8 at 10 am MT. Click here to register. Click here to learn more from a Colorado patient with Cystic Fibrosis.
Massachusetts
The Massachusetts Senate’s Ways and Means Committees was referred the “PACT Act”, reported favorably on October 30, 2023 by the Joint Committee on Health Care Financing. The legislation includes a provision calling for the health policy commission not to base its determinations on measures such as QALYs. The Senate rejected an amendment (#19) pushed by Massachusetts advocates calling for transparency related to the use of cost effectiveness analyses by the commission, as well as increased requirements for stakeholder engagement. The Senate passed the bill on November 15, 2023. Massachusetts advocates will work with the House toward language that more clearly provides for public transparency of the evidence under consideration and opportunities for engagement from patients and people with disabilities.
International News: What Happens in Countries Using QALYs and Cost-Based Thresholds?
Other countries are often referenced as examples of how the use of QALYs or similar cost-based thresholds impact access to care.
- Australia: The opportunity for survival for hundreds of Australians living with a deadly type of blood cancer is in the hands of a government committee, which will decide whether to support public funding for CAR-T cell therapy.
- Canada: Madi and Beth Vanstone have chosen to participate in the I Am Number 12 campaign, which aims to bring much-needed attention to the challenges people living with rare diseases are facing in Canada.
- United Kingdom: Cystic fibrosis patients could see "miracle drug" cut from NHS due to "unjustifiable" costs.
ICER's QALY-Based Study Topics
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.
- Chronic Obstructive Pulmonary Disease: Revised Scoping Document available. Public meeting: June 2024
- Post-Traumatic Stress Disorder: Revised Scoping Document available. Main review: May 30, 2024.
- Paroxysmal Nocturnal Hemoglobinuria: Evidence Report available.
- Schizophrenia: Draft Evidence Report available.
- Pulmonary Arterial Hypertension: Main review: December 1, 2023.
Upcoming Events and Webinars
Ensuring Equity in Implementation of IRA Drug Price Negotiations
December 5, 2023
Click here to view.
PCORI Board of Governors Meeting
December 12, 2023
Click here to view.
Medical Journal Articles
Alternative approaches to measuring value: an update on innovative methods in the context of the United States Medicare drug price negotiation program. Click here to read the article.
Economic Outcomes in Patient-Centered Outcomes Research: A Paradigm Shift. Click here to read the article.
Visualization of Evidence for Shared Decision Making. Click here to read the article.
Avenues for Strengthening PCORnet’s Capacity to Advance Patient-Centered Economic Outcomes in Patient-Centered Outcomes Research. Click here to read the article.
Linking Medicare-Medicaid Claims for Patient-Centered Outcomes Research Among Dual-Eligible Beneficiaries. Click here to read the article.
Gene Therapies for Sickle Cell Disease: Effectiveness and Value. Click here to read the article.
White Paper: Improving Patient Subgroup Representation with Real-World Data. Click here to read the article.
A Clinical Pathway to Well-Being: Putting Patient Priorities at the Center of Care. Click here to read the article.
Comparative Effectiveness of Treatments for Rheumatoid Arthritis in Clinical Practice: A Systematic Review. Click here to read the article.
QALYs: The Math Doesn’t Work. Click here to read the article.
The Implementation of Value-Based Frameworks, Clinical Care Pathways, and Alternative Payment Models for Cancer Care in the United States. Click here to view.
Perspectives on Patient-Reported Outcome Data After Treatment Discontinuation in Cancer Clinical Trials. Click here to view.
AHRQ Effective Program Updates
Technical Brief: Measuring Healthcare Organization Characteristics in Cancer Care Delivery Research. Click here to view.
Systematic Review: Postpartum Care up to 1 Year After Pregnancy: A Systematic Review and Meta-analysis. Click here to view.
Draft Report: Impact of Healthcare Algorithms on Racial and Ethnic Disparities in Health and Healthcare. Click here to view.
Systematic Review: Partian Breast Irradiation for Breast Cancer. Click here to view.
Systematic Review: Use of Telehealth During the COVID-19 Era. Click here to view.
Research Report: Analysis of Requirements for Coverage with Evidence Development (CED) - Topic Refinement. Click here to view.
Technical Brief: Infection Prevention and Control for the Emergency Medical Services and 911 Workforce. Click here to view.
Systematic Review: Management of Infantile Epilepsies. Click here to view.
