In this week's edition...
— Senate Aging to Hold Hearing on Rare Diseases, Asks for Patient Stories. See details below.
— PCORI Engagement Projects Focus on Mental Health. Click here to read the blog.
— Chairman Coelho: Better Research Can Prevent Discriminatory Outcomes. Click here to read the letter.
— Powerful State Panel Poses Threat to Patients with Rare Diseases. Click here to read the column
— 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.
— Job Openings. See details below.
— Senate Aging to Hold Hearing on Rare Diseases, Asks for Patient Stories. See details below.
— PCORI Engagement Projects Focus on Mental Health. Click here to read the blog.
— Chairman Coelho: Better Research Can Prevent Discriminatory Outcomes. Click here to read the letter.
— Powerful State Panel Poses Threat to Patients with Rare Diseases. Click here to read the column
— 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.
— Job Openings. See details below.
What We're Reading
1. Senate Aging to Hold Hearing on Rare Diseases, Asks for Patient Stories. Ahead of an October 26 hearing that will focus on unmet medical needs for patients with rare or progressive diseases, Senate Aging Committee Ranking Member Mike Braun (R-IN) issued a request for patient stories. "Have you or a loved one been affected by a rare, progressive, or similar serious disease? Senator Braun, Ranking Member of the Senate Special Committee on Aging, will be holding a hearing on the unmet medical need experienced by people with rare, progressive, and serious diseases face, on Thursday, October 26 at 10:00AM. We want to amplify your experiences and make sure that your story is heard by Congress. You can submit your stories to the Committee for entry into the record at [email protected]."
2. PCORI Engagement Projects Focus on Mental Health. A new PCORI blog post outlines how patient-centered outcomes research and comparative clinical effectiveness research (PCOR/CER) emphasizes patient engagement and mental health. “Mental health conditions impact individuals across age, income, racial, and ethnic groups. One in five people in the United States will experience a mental health condition within a given year, and more than 50 percent will receive a mental health diagnosis at some point in their lifetime. Using engagement tools to build patient and other stakeholders’ capacity for patient-centered outcomes research and comparative clinical effectiveness research (PCOR/CER), two PCORI-funded project teams have made significant advances regarding mental health amongst two distinct population groups: graduate students and older adults.”
3. Chairman Coelho: Better Research Can Prevent Discriminatory Outcomes. PIPC Chairman Tony Coelho recently penned a letter to the editor for Bloomberg in response to their recent editorial characterizing opposition to QALYs as "dubious." "The problem lies in selecting a one-size-fits-all measure of clinical or cost-effectiveness to serve as a benchmark for coverage or even a fair price. Most of us don’t fit into an average. Yet traditional research doesn’t do a great job of analyzing how subgroups fare on treatment." Click here to read the letter.
4. Powerful State Panel Poses Threat to Patients with Rare Diseases. In a column for Colorado Politics, Amber Freed outlines concerns about the state's Prescription Drug Advisory Board (PDAB) "fair pricing" policies. "Colorado is going it alone among the states and setting drug prices in a bureaucratic vacuum. Therefore, certain medicines may not be available at the prices Colorado sets. If a hospital or pharmacy cannot buy a drug for the price the government demands, that medicine will not be available. Or in many cases an alternative drug may be available — even if that is not the medicine preferred by doctors for specific patients. There is nothing 'fair' about a law that denies access to medicines to the patients who need them." Click here to read the column.
5. 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.
Michigan
In Michigan, legislators introduced, S. 483, which would create a Prescription Drug Affordability Board (PDAB) and institute upper payment limits for certain prescription drugs. The bill passed the Senate and has moved to the House. Advocates have provided testimony urging lawmakers to slow down and ensure any efforts include patient protections, including a QALY ban and Board and Advisory Committee seats for patients and people with disabilities. Click here to view testimony from Laura Bonnell, Bonnell Foundation.
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. Presentations related to cost effectiveness analyses to the Colorado Board have referenced the use of a cost-per-QALY or the equal value of life-year gained (evLYG) in estimating cost effectiveness of treatments. This indicates that these metrics may have influenced how Colorado selected the drugs for review. The PDAB offers several opportunities for patients to engage and ensure their voice is heard on these topics:
Pennsylvania
In the last legislative session, the legislature introduced, HB 1722, a bill creating a Prescription Drug Affordability Board that would not bar reference to QALYs and would explicitly authorize the Board to contract with a third party that could include ICER. The bill has not been reintroduced in 2023. The Pennsylvania legislature held an informational meeting on Prescription Drug Boards in the House Health Committee on April 12 at 10am. Click here to learn more about Prescription Drug Affordability Boards and the implications of discriminatory measures of cost effectiveness. Click here to learn more about the committee chair, Rep. Dan Frankel.
Massachusetts
An Act Advancing Health Care Research and Decision-Making Centered on Patients and People with disabilities, SD. 2117 and HD. 3356 has been introduced in both the House and Senate. On September 20, 2023, the Massachusetts Joint Committee on Health Care Financing considered the legislation, including provisions that would prohibit reliance on discriminatory measures, including quality-adjusted life years (QALYs) to determine whether a particular health care treatment is cost effective, recommended, the value of a treatment, or in determining coverage, reimbursement, appropriate payment amounts, cost-sharing, or incentive policies or programs. PIPC Chair Tony Coelho testified in support of this legislation, alongside the ALS Foundation and the Epilepsy Foundation of New England.
Oregon
The Oregon legislature held a hearing on March 27, 2023 on SB 492, legislation that would address the use of biased measures of quality of life, such as quality-adjusted life years or QALYs. PIPC Chairman Tony Coelho submitted testimony stating, "This legislation reflects decades of work to advance disability rights and to end the use of discriminatory measures of the effectiveness of health care that too often drive barriers to care for people with disabilities. In the past this may have been considered just a disability issue, but today we recognize the implications of these biased algorithms for health equity more broadly.” Click here to view testimony from PIPC Chairman Tony Coelho. Click here to view testimony submitted by the Caring Ambassadors Program. Click here to view the list of organizations in Oregon supporting the bill.
On October 6, 2022, the Oregon Health Evidence Review Commission (HERC) discussed a proposed plan for using QALYs. The meeting followed CMS approval of Oregon’s waiver application, which does not bar the use of QALYs in the interim. Click here to read comments submitted by PIPC along with 62 other groups asking CMS to reject the waiver. Click here to view the CMS-approved waiver. At the HERC meeting, the Oregon Health Authority expressed its intent to continue using a prioritized list, shifting it from use under a waiver to use as part of the State Plan Amendment after the waiver expires in 2027. The HERC decided to accept comments on their use of QALYs but has not made a decision as to whether they will adjust their methodology. Click here to view the letter from almost 50 advocacy groups asking the HERC to stop using QALYs. Click here to view testimony from PIPC Chairman Coelho to the Oregon HERC expressing his support for the Americans with Disabilities Act. Click here to view the options that continue to be under consideration by the HERC to continue using QALYs. Click here to view all of the comments shared with PIPC.
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
Real World Evidence Conference
October 16-17, 2023
Click here to view.
2023 Annual Meeting - National Forum for Heart Disease & Stroke Prevention
October 26, 2023
Click here to view.
Medical Journal Articles
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.
The Implementation of Value-Based Frameworks, Clinical Care Pathways, and Alternative Payment Models for Cancer Care in the United States. Click here to view.
Federal Data for Conducting Patient-Centered Outcomes Research on Economic Outcomes. Click here to view.
Patient-Reported Outcomes for People with Diabetes: What and How to Measure? A Narrative Review. Click here to view.
Patient Engagement in Research; Benefits, Challenges, Importance, and Implications. Click here to view.
The Opportunity for Greater Patient and Public Involvement and Engagement in Drug Development and Regulation. 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. Senate Aging to Hold Hearing on Rare Diseases, Asks for Patient Stories. Ahead of an October 26 hearing that will focus on unmet medical needs for patients with rare or progressive diseases, Senate Aging Committee Ranking Member Mike Braun (R-IN) issued a request for patient stories. "Have you or a loved one been affected by a rare, progressive, or similar serious disease? Senator Braun, Ranking Member of the Senate Special Committee on Aging, will be holding a hearing on the unmet medical need experienced by people with rare, progressive, and serious diseases face, on Thursday, October 26 at 10:00AM. We want to amplify your experiences and make sure that your story is heard by Congress. You can submit your stories to the Committee for entry into the record at [email protected]."
2. PCORI Engagement Projects Focus on Mental Health. A new PCORI blog post outlines how patient-centered outcomes research and comparative clinical effectiveness research (PCOR/CER) emphasizes patient engagement and mental health. “Mental health conditions impact individuals across age, income, racial, and ethnic groups. One in five people in the United States will experience a mental health condition within a given year, and more than 50 percent will receive a mental health diagnosis at some point in their lifetime. Using engagement tools to build patient and other stakeholders’ capacity for patient-centered outcomes research and comparative clinical effectiveness research (PCOR/CER), two PCORI-funded project teams have made significant advances regarding mental health amongst two distinct population groups: graduate students and older adults.”
3. Chairman Coelho: Better Research Can Prevent Discriminatory Outcomes. PIPC Chairman Tony Coelho recently penned a letter to the editor for Bloomberg in response to their recent editorial characterizing opposition to QALYs as "dubious." "The problem lies in selecting a one-size-fits-all measure of clinical or cost-effectiveness to serve as a benchmark for coverage or even a fair price. Most of us don’t fit into an average. Yet traditional research doesn’t do a great job of analyzing how subgroups fare on treatment." Click here to read the letter.
4. Powerful State Panel Poses Threat to Patients with Rare Diseases. In a column for Colorado Politics, Amber Freed outlines concerns about the state's Prescription Drug Advisory Board (PDAB) "fair pricing" policies. "Colorado is going it alone among the states and setting drug prices in a bureaucratic vacuum. Therefore, certain medicines may not be available at the prices Colorado sets. If a hospital or pharmacy cannot buy a drug for the price the government demands, that medicine will not be available. Or in many cases an alternative drug may be available — even if that is not the medicine preferred by doctors for specific patients. There is nothing 'fair' about a law that denies access to medicines to the patients who need them." Click here to read the column.
5. 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.
Michigan
In Michigan, legislators introduced, S. 483, which would create a Prescription Drug Affordability Board (PDAB) and institute upper payment limits for certain prescription drugs. The bill passed the Senate and has moved to the House. Advocates have provided testimony urging lawmakers to slow down and ensure any efforts include patient protections, including a QALY ban and Board and Advisory Committee seats for patients and people with disabilities. Click here to view testimony from Laura Bonnell, Bonnell Foundation.
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. Presentations related to cost effectiveness analyses to the Colorado Board have referenced the use of a cost-per-QALY or the equal value of life-year gained (evLYG) in estimating cost effectiveness of treatments. This indicates that these metrics may have influenced how Colorado selected the drugs for review. The PDAB offers several opportunities for patients to engage and ensure their voice is heard on these topics:
- Colorado’s next Prescription Drug Affordability Board meeting is October 27.
- Patient and caregiver survey due date has been extended to October 12.
Pennsylvania
In the last legislative session, the legislature introduced, HB 1722, a bill creating a Prescription Drug Affordability Board that would not bar reference to QALYs and would explicitly authorize the Board to contract with a third party that could include ICER. The bill has not been reintroduced in 2023. The Pennsylvania legislature held an informational meeting on Prescription Drug Boards in the House Health Committee on April 12 at 10am. Click here to learn more about Prescription Drug Affordability Boards and the implications of discriminatory measures of cost effectiveness. Click here to learn more about the committee chair, Rep. Dan Frankel.
Massachusetts
An Act Advancing Health Care Research and Decision-Making Centered on Patients and People with disabilities, SD. 2117 and HD. 3356 has been introduced in both the House and Senate. On September 20, 2023, the Massachusetts Joint Committee on Health Care Financing considered the legislation, including provisions that would prohibit reliance on discriminatory measures, including quality-adjusted life years (QALYs) to determine whether a particular health care treatment is cost effective, recommended, the value of a treatment, or in determining coverage, reimbursement, appropriate payment amounts, cost-sharing, or incentive policies or programs. PIPC Chair Tony Coelho testified in support of this legislation, alongside the ALS Foundation and the Epilepsy Foundation of New England.
Oregon
The Oregon legislature held a hearing on March 27, 2023 on SB 492, legislation that would address the use of biased measures of quality of life, such as quality-adjusted life years or QALYs. PIPC Chairman Tony Coelho submitted testimony stating, "This legislation reflects decades of work to advance disability rights and to end the use of discriminatory measures of the effectiveness of health care that too often drive barriers to care for people with disabilities. In the past this may have been considered just a disability issue, but today we recognize the implications of these biased algorithms for health equity more broadly.” Click here to view testimony from PIPC Chairman Tony Coelho. Click here to view testimony submitted by the Caring Ambassadors Program. Click here to view the list of organizations in Oregon supporting the bill.
On October 6, 2022, the Oregon Health Evidence Review Commission (HERC) discussed a proposed plan for using QALYs. The meeting followed CMS approval of Oregon’s waiver application, which does not bar the use of QALYs in the interim. Click here to read comments submitted by PIPC along with 62 other groups asking CMS to reject the waiver. Click here to view the CMS-approved waiver. At the HERC meeting, the Oregon Health Authority expressed its intent to continue using a prioritized list, shifting it from use under a waiver to use as part of the State Plan Amendment after the waiver expires in 2027. The HERC decided to accept comments on their use of QALYs but has not made a decision as to whether they will adjust their methodology. Click here to view the letter from almost 50 advocacy groups asking the HERC to stop using QALYs. Click here to view testimony from PIPC Chairman Coelho to the Oregon HERC expressing his support for the Americans with Disabilities Act. Click here to view the options that continue to be under consideration by the HERC to continue using QALYs. Click here to view all of the comments shared with PIPC.
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: MS patients in Australia face limited choices with respect to effective treatments covered, and some are forced to raise money for treatment overseas.
- U.K: The family of a patient with MS has had to turn to crowdfunding for treatment that is not funded through the NHS.
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.
- Paroxysmal Nocturnal Hemoglobinuria: Draft Scoping Document Available. Comments due August 14, 2023.
- Schizophrenia: Draft Scoping Document available. Comments due on July 26, 2023.
- Metachromatic Leukodystrophy: Draft Evidence Report now available.
- Pulmonary Arterial Hypertension: Draft Scoping Document available. Open for comments until June 5, 2023.
- Sickle Cell Disease: Evidence Report Available. California Technology Assessment Forum (CTAF) to meet on July 27, 2023, to review.
- Non-Alcoholic Steatohepatitis: Final Evidence Report now available.
Upcoming Events and Webinars
Real World Evidence Conference
October 16-17, 2023
Click here to view.
2023 Annual Meeting - National Forum for Heart Disease & Stroke Prevention
October 26, 2023
Click here to view.
Medical Journal Articles
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.
The Implementation of Value-Based Frameworks, Clinical Care Pathways, and Alternative Payment Models for Cancer Care in the United States. Click here to view.
Federal Data for Conducting Patient-Centered Outcomes Research on Economic Outcomes. Click here to view.
Patient-Reported Outcomes for People with Diabetes: What and How to Measure? A Narrative Review. Click here to view.
Patient Engagement in Research; Benefits, Challenges, Importance, and Implications. Click here to view.
The Opportunity for Greater Patient and Public Involvement and Engagement in Drug Development and Regulation. 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.
