In this week's edition...
— Recapping #PIPCForum2023. See details below.
— Cassidy Requests Information on Improving Access to Gene Therapies. See details below.
— Ban Quality-Adjusted Life-Years in Oregon. Click here to sign the petition.
— 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.
— Recapping #PIPCForum2023. See details below.
— Cassidy Requests Information on Improving Access to Gene Therapies. See details below.
— Ban Quality-Adjusted Life-Years in Oregon. Click here to sign the petition.
— 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. Recapping #PIPCForum2023. The Partnership to Improve Patient Care (PIPC) recently aired its 14th annual forum virtually on December 11, 2023.
The first panel discussed the need for better engagement tactics by CMS, including how PCORI and FDA could be models and raising concerns about payers potentially leaning more, not less, on utilization management. The panel emphasized the need for processes to allow patients and patient groups to weigh in on the evidence under consideration, as well as to help CMS keep their finger on the pulse of any unintended consequences related to implementing the provisions of the Inflation Reduction Act.
The second panel focused on value assessment and how measures of quality of life and life extension differ among patients and people with disabilities. While it may seem obvious to us, the panel highlighted for the audience how people with ALS or Down Syndrome value their own lives, with an emphasis on protecting their quality of life. The need for federal legislation, H.R. 485 the Protecting Health Care for All Patients Act, was highlighted as a priority for addressing the use of biased and discriminatory value assessments in health programs.
Lastly, Dr. Bonnie Swenor shared some timely updates related to the fight for disability inclusion in research and data collection. She was at the forefront of the fight for people with disabilities to be included as a health disparities population and continues the fight for every disabled person to be counted in the Census, an issue that is very personal to me as a former co-chair of the former Census Monitoring Board. As a researcher who is also disabled, Dr. Swenor provided insights as to how bias seeps into research when people with disabilities are not included and researchers assume people with disabilities have a low quality of life - baking in ableism and bias from the start of health care research and value assessment.
Click here to watch the forum in its entirety.
2. Cassidy Requests Information on Improving Access to Gene Therapies. Sen. Bill Cassidy (R-LA), Ranking Member of the Senate HELP Committee, is seeking stakeholder feedback on ways to improve and protect access to gene therapies for patients with rare diseases. Cassidy hopes to use the feedback to inform future legislation to modernize and improve the market structure for gene therapies to ensure that the U.S. commercial health insurance market supports Americans with ultra-rare diseases, while ensuring the continued development of innovative gene therapies. Feedback is due to [email protected] by January 22, 2024.
3. Ban Quality-Adjusted Life-Years in Oregon. Disability Rights Oregon is circulating a petition to ban quality adjusted life years (QALYs) by payers in Oregon, a flawed scoring system economists use to determine cost-effectiveness of proposed medical treatments. Payers—including the Oregon Health Plan—use QALY scores to determine who is eligible for healthcare resources. The problem is the scores are calculated in inherently discriminatory ways. DRO states in their petition, "QALY violates both the Rehabilitation Act and the Americans with Disabilities Act. These types of scores have no place influencing what conditions and treatments the Oregon Health Plan will cover.” DRO hopes that the state legislature will act during a short legislative session. Click here to sign the petition.
4. 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 take place on December 15, 2023 at 1 pm MT to adopt the final Affordability Review Report for Trikafta. The Board determined during its December 8 meeting that Trikafta was not unaffordable for Colorado consumers. Click here to register for the December 15 meeting. Colorado’s Prescription Drug Affordability Advisory Council (PDAAC) will hold a meeting on January 25, 2024 at 9 am MT. Click here to register. The PDACC has also noticed that it is currently accepting applications for new council members. Click here to apply.
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
PCORI Board of Governors Meeting
February 13, 2024
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. Recapping #PIPCForum2023. The Partnership to Improve Patient Care (PIPC) recently aired its 14th annual forum virtually on December 11, 2023.
The first panel discussed the need for better engagement tactics by CMS, including how PCORI and FDA could be models and raising concerns about payers potentially leaning more, not less, on utilization management. The panel emphasized the need for processes to allow patients and patient groups to weigh in on the evidence under consideration, as well as to help CMS keep their finger on the pulse of any unintended consequences related to implementing the provisions of the Inflation Reduction Act.
The second panel focused on value assessment and how measures of quality of life and life extension differ among patients and people with disabilities. While it may seem obvious to us, the panel highlighted for the audience how people with ALS or Down Syndrome value their own lives, with an emphasis on protecting their quality of life. The need for federal legislation, H.R. 485 the Protecting Health Care for All Patients Act, was highlighted as a priority for addressing the use of biased and discriminatory value assessments in health programs.
Lastly, Dr. Bonnie Swenor shared some timely updates related to the fight for disability inclusion in research and data collection. She was at the forefront of the fight for people with disabilities to be included as a health disparities population and continues the fight for every disabled person to be counted in the Census, an issue that is very personal to me as a former co-chair of the former Census Monitoring Board. As a researcher who is also disabled, Dr. Swenor provided insights as to how bias seeps into research when people with disabilities are not included and researchers assume people with disabilities have a low quality of life - baking in ableism and bias from the start of health care research and value assessment.
Click here to watch the forum in its entirety.
2. Cassidy Requests Information on Improving Access to Gene Therapies. Sen. Bill Cassidy (R-LA), Ranking Member of the Senate HELP Committee, is seeking stakeholder feedback on ways to improve and protect access to gene therapies for patients with rare diseases. Cassidy hopes to use the feedback to inform future legislation to modernize and improve the market structure for gene therapies to ensure that the U.S. commercial health insurance market supports Americans with ultra-rare diseases, while ensuring the continued development of innovative gene therapies. Feedback is due to [email protected] by January 22, 2024.
3. Ban Quality-Adjusted Life-Years in Oregon. Disability Rights Oregon is circulating a petition to ban quality adjusted life years (QALYs) by payers in Oregon, a flawed scoring system economists use to determine cost-effectiveness of proposed medical treatments. Payers—including the Oregon Health Plan—use QALY scores to determine who is eligible for healthcare resources. The problem is the scores are calculated in inherently discriminatory ways. DRO states in their petition, "QALY violates both the Rehabilitation Act and the Americans with Disabilities Act. These types of scores have no place influencing what conditions and treatments the Oregon Health Plan will cover.” DRO hopes that the state legislature will act during a short legislative session. Click here to sign the petition.
4. 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 take place on December 15, 2023 at 1 pm MT to adopt the final Affordability Review Report for Trikafta. The Board determined during its December 8 meeting that Trikafta was not unaffordable for Colorado consumers. Click here to register for the December 15 meeting. Colorado’s Prescription Drug Affordability Advisory Council (PDAAC) will hold a meeting on January 25, 2024 at 9 am MT. Click here to register. The PDACC has also noticed that it is currently accepting applications for new council members. Click here to apply.
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.
- Canada: A patient with abdominal cancer was forced to leave the country after the British Columbia Cancer Agency told her she was not a candidate for treatment.
- United Kingdom: Cystic fibrosis patients are rallying against a proposal to scale back access to a life-changing drug.
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: Draft Evidence Report available.
- Schizophrenia: Draft Evidence Report available.
- Pulmonary Arterial Hypertension: Final Evidence Report and Meeting Summary available.
Upcoming Events and Webinars
PCORI Board of Governors Meeting
February 13, 2024
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.
