In this week's edition...
— National Health Council Comments Urge CMS Not to Use QALYs in Medicare Drug Price Negotiations. Click here to read the letter.
— Fatty Liver Foundation Submits Comments on ICER's Assessment of NASH Treatments. Click here to read more.
— Chairman's Corner: In Medicare Drug Price Negotiations, Avoid Metrics Steeped In Stigma. Click here to read the column.
— Follow PIPC on LinkedIn! Click here to view the page.
— 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.
— National Health Council Comments Urge CMS Not to Use QALYs in Medicare Drug Price Negotiations. Click here to read the letter.
— Fatty Liver Foundation Submits Comments on ICER's Assessment of NASH Treatments. Click here to read more.
— Chairman's Corner: In Medicare Drug Price Negotiations, Avoid Metrics Steeped In Stigma. Click here to read the column.
— Follow PIPC on LinkedIn! Click here to view the page.
— 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. National Health Council Comments Urge CMS Not to Use QALYs in Medicare Drug Price Negotiations. In a comment letter to the Centers for Medicare and Medicaid Services (CMS), the National Health Council urges CMS not to use the discriminatory QALY metric as a part of its Medicare Drug Negotiation Program. "The NHC is appreciative that CMS will not use QALYs as part of the Medicare Drug Negotiation Program. QALYs can be discriminatory in practice, and the NHC applauds CMS decision to not use them as metrics in the negotiation process. We also expressed concern that CMS may not effectively eliminate QALYs from analysis by utilizing studies that use QALY-related data from secondary sources, or that CMS may over- exclude analyses that are otherwise helpful in establishing the value of a drug. We asked CMS for more clarity on how they will exclude QALYs from these studies." Click here to read the letter.
2. Fatty Liver Foundation Submits Comments on ICER's Assessment of NASH Treatments. The Fatty Liver Foundation has published a comment letter to ICER outlining concerns with its evidence report on treatments for nonalcoholic steatohepatitis (NASH). Specifically, Co-founder and CEO Wayne Eskridge highlighted concerns about the underestimation of NASH impact and implications for obeticholic acid and resmetirom. "These benefits are crucial for halting disease progression and are currently underappreciated in ICER's evidence report and cost/benefit analyses." Click here to read more.
3. Chairman's Corner: In Medicare Drug Price Negotiations, Avoid Metrics Steeped In Stigma. In a column for Health Affairs, PIPC Chairman Tony Coelho responds to a January 4 piece discussing pricing methodologies based on how much a drug improves health, emphasizing the need to avoid discriminatory assessments that harm patients and people with disabilities "...The authors overemphasize reliance on frameworks that, especially for people with disabilities, are red flags due to their reliance on valuing people’s lives in dollars. Instead, the Centers for Medicare and Medicaid Services (CMS) has an opportunity to achieve a level of patient-centeredness that other programs lack." Click here to read the column.
4. Follow PIPC on LinkedIn! Visit and follow PIPC's new page on LinkedIn to stay informed on all of our latest comment opportunities, letters, and other key resources. Click here to view the page.
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.
Pennsylvania
The Pennsylvania legislature held an informational meeting on Prescription Drug Affordability Boards in the House Health Committee on April 12 at 10am. While new text has not been released, a version of legislation creating a Board was introduced in 2021, HB 1722. In the prior Congress, the legislation did not bar the use of quality-adjusted life years (QALYs) in making determinations of the treatments subject to a cost review and upper payment limit. The bill explicitly allowed for consideration of "the estimated value or cost-effectiveness of the prescription drug product” without any restriction on use of discriminatory measures of cost effectiveness such as those using QALYs. 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.
Nevada
Nevada has introduced AB 250 which would set prices for prescription drugs in line with the new federally determined maximum fair price (MFP). The MFP price will be newly established through implementation of the IRA, and CMS has not yet released their final methodology to determine the MFP. It will be important that CMS avoid the use of QALYs and similar measures with implications for bias and discrimination and appropriately engage impacted populations in determining the outcomes for measuring a drug's therapeutic benefit. Advocates are urging CMS to use a transparent methodology that is centered on outcomes that matter to patients and people with disabilities and that explicitly does not rely on discriminatory QALYs and similar measures. Therefore, the pending legislation raises serious concerns about states directly referencing prices that may rely on biased or discriminatory measures. Click here to view testimony from Nevada Chronic Care Collaborative. Click here to view testimony from Epilepsy Foundation Nevada.
Minnesota
The Minnesota state legislature has introduced a bill to create a Prescription Drug Affordability Board, SF168 and HF17. It does not include a bar on using QALYs or other metrics that discriminate as a consideration for selecting the treatments to be considered, nor does the bill bar the use of QALYs as a consideration in establishing an upper payment limit, instead explicitly referencing prices established by third parties that may consider QALYs. The bill specifically references Canadian prices as a consideration for establishing the upper payment limit for drugs, prices which are explicitly based on QALYs. Click here to view the testimony from the ALS Association.
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. This bill includes important patient protections including a ban on the discriminatory Quality-Adjusted Life Year (QALY).
California
California Attorney General Rob Bonta has solicited information from hospital CEOs across the state about how healthcare facilities and other providers are identifying and addressing racial and ethnic disparities in commercial decision-making tools. In response to this letter, advocates urged Attorney General Bonta to include QALYs and similar metrics in his investigation. Click here to view the Attorney General’s letter. Click here to view a response from advocates.
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, including proposals to redact the word QALY from the HERC’s deliberations without barring the use of the metric in making decisions. 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
PCORI Advisory Panel on Patient Engagement Spring 2023 Meeting
May 4, 2023
Click here to view.
National Council on Disability Quarterly Meeting
May 4, 2023
Click here to view.
2023 Science of Patient Engagement Symposium
May 8, 2023
Click here to view.
Connecting Real-World Data and Domain Expertise to Enhance Trial Design and Planning
May 18, 2023
Click here to view.
PCORnet Studies Applicant Workshop
May 18, 2023
Click here to view.
PCORI Board of Governors Meeting
June 5-6, 2023
Click here to view.
PCORI Board of Governors Meeting
September 11, 2023
Click here to view.
2023 PCORI Annual Meeting
October 4-6, 2023
Click here to view.
Medical Journal Articles
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.
Data Governance for Real-World Data Management: A Proposal for a Checklist to Support Decision Making. Click here to view.
A Patient-Centered Comparative Effectiveness Research Study of Culturally Appropriate Options for Diabetes Self-Management. Click here to view.
Payer–patient Engagement Framework to Strengthen Ethical Formulary Decision-making in Rare Disease Arena in the USA. Click here to view.
The Impact on Cost-Effectiveness of Accounting for Generic Drug Pricing: Four Case Studies. Click here to view.
A Perspective on Life-Cycle Health Technology Assessment and Real-World Evidence for Precision Oncology in Canada. Click here to view.
AHRQ Effective Program Updates
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.
Systematic Review: Pharmacologic and Nonpharmacologic Treatments for Posttraumatic Stress Disorder. Click here to view.
Systematic Review: Living Systematic Review on Cannabis and Other Plant-Based Treatments for Chronic Pain. Click here to view.
Job Openings
1. National Health Council Comments Urge CMS Not to Use QALYs in Medicare Drug Price Negotiations. In a comment letter to the Centers for Medicare and Medicaid Services (CMS), the National Health Council urges CMS not to use the discriminatory QALY metric as a part of its Medicare Drug Negotiation Program. "The NHC is appreciative that CMS will not use QALYs as part of the Medicare Drug Negotiation Program. QALYs can be discriminatory in practice, and the NHC applauds CMS decision to not use them as metrics in the negotiation process. We also expressed concern that CMS may not effectively eliminate QALYs from analysis by utilizing studies that use QALY-related data from secondary sources, or that CMS may over- exclude analyses that are otherwise helpful in establishing the value of a drug. We asked CMS for more clarity on how they will exclude QALYs from these studies." Click here to read the letter.
2. Fatty Liver Foundation Submits Comments on ICER's Assessment of NASH Treatments. The Fatty Liver Foundation has published a comment letter to ICER outlining concerns with its evidence report on treatments for nonalcoholic steatohepatitis (NASH). Specifically, Co-founder and CEO Wayne Eskridge highlighted concerns about the underestimation of NASH impact and implications for obeticholic acid and resmetirom. "These benefits are crucial for halting disease progression and are currently underappreciated in ICER's evidence report and cost/benefit analyses." Click here to read more.
3. Chairman's Corner: In Medicare Drug Price Negotiations, Avoid Metrics Steeped In Stigma. In a column for Health Affairs, PIPC Chairman Tony Coelho responds to a January 4 piece discussing pricing methodologies based on how much a drug improves health, emphasizing the need to avoid discriminatory assessments that harm patients and people with disabilities "...The authors overemphasize reliance on frameworks that, especially for people with disabilities, are red flags due to their reliance on valuing people’s lives in dollars. Instead, the Centers for Medicare and Medicaid Services (CMS) has an opportunity to achieve a level of patient-centeredness that other programs lack." Click here to read the column.
4. Follow PIPC on LinkedIn! Visit and follow PIPC's new page on LinkedIn to stay informed on all of our latest comment opportunities, letters, and other key resources. Click here to view the page.
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.
Pennsylvania
The Pennsylvania legislature held an informational meeting on Prescription Drug Affordability Boards in the House Health Committee on April 12 at 10am. While new text has not been released, a version of legislation creating a Board was introduced in 2021, HB 1722. In the prior Congress, the legislation did not bar the use of quality-adjusted life years (QALYs) in making determinations of the treatments subject to a cost review and upper payment limit. The bill explicitly allowed for consideration of "the estimated value or cost-effectiveness of the prescription drug product” without any restriction on use of discriminatory measures of cost effectiveness such as those using QALYs. 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.
Nevada
Nevada has introduced AB 250 which would set prices for prescription drugs in line with the new federally determined maximum fair price (MFP). The MFP price will be newly established through implementation of the IRA, and CMS has not yet released their final methodology to determine the MFP. It will be important that CMS avoid the use of QALYs and similar measures with implications for bias and discrimination and appropriately engage impacted populations in determining the outcomes for measuring a drug's therapeutic benefit. Advocates are urging CMS to use a transparent methodology that is centered on outcomes that matter to patients and people with disabilities and that explicitly does not rely on discriminatory QALYs and similar measures. Therefore, the pending legislation raises serious concerns about states directly referencing prices that may rely on biased or discriminatory measures. Click here to view testimony from Nevada Chronic Care Collaborative. Click here to view testimony from Epilepsy Foundation Nevada.
Minnesota
The Minnesota state legislature has introduced a bill to create a Prescription Drug Affordability Board, SF168 and HF17. It does not include a bar on using QALYs or other metrics that discriminate as a consideration for selecting the treatments to be considered, nor does the bill bar the use of QALYs as a consideration in establishing an upper payment limit, instead explicitly referencing prices established by third parties that may consider QALYs. The bill specifically references Canadian prices as a consideration for establishing the upper payment limit for drugs, prices which are explicitly based on QALYs. Click here to view the testimony from the ALS Association.
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. This bill includes important patient protections including a ban on the discriminatory Quality-Adjusted Life Year (QALY).
California
California Attorney General Rob Bonta has solicited information from hospital CEOs across the state about how healthcare facilities and other providers are identifying and addressing racial and ethnic disparities in commercial decision-making tools. In response to this letter, advocates urged Attorney General Bonta to include QALYs and similar metrics in his investigation. Click here to view the Attorney General’s letter. Click here to view a response from advocates.
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, including proposals to redact the word QALY from the HERC’s deliberations without barring the use of the metric in making decisions. 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: Australian cancer patients are forced to wait two years longer for access to breakthrough medicines compared to patients in the U.S. Some estimates show that patients have had to spend $200 million per year as a result.
- France: Cystic fibrosis patients are facing restrictions in accessing treatment, either because of their age or because they have a rare genetic profile.
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.
- Sickle Cell Disease: Draft Evidence Report Available. Public Comments open until 5/9/2023.
- Non-Alcoholic Steatohepatitis: Evidence Report available. Evidence Presentation to be held 4/28/2023
- Metachromatic Leukodystrophy: Revised Scoping Document and Research protocol available. Draft Evidence Report to be released 7/26/2023
Upcoming Events and Webinars
PCORI Advisory Panel on Patient Engagement Spring 2023 Meeting
May 4, 2023
Click here to view.
National Council on Disability Quarterly Meeting
May 4, 2023
Click here to view.
2023 Science of Patient Engagement Symposium
May 8, 2023
Click here to view.
Connecting Real-World Data and Domain Expertise to Enhance Trial Design and Planning
May 18, 2023
Click here to view.
PCORnet Studies Applicant Workshop
May 18, 2023
Click here to view.
PCORI Board of Governors Meeting
June 5-6, 2023
Click here to view.
PCORI Board of Governors Meeting
September 11, 2023
Click here to view.
2023 PCORI Annual Meeting
October 4-6, 2023
Click here to view.
Medical Journal Articles
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.
Data Governance for Real-World Data Management: A Proposal for a Checklist to Support Decision Making. Click here to view.
A Patient-Centered Comparative Effectiveness Research Study of Culturally Appropriate Options for Diabetes Self-Management. Click here to view.
Payer–patient Engagement Framework to Strengthen Ethical Formulary Decision-making in Rare Disease Arena in the USA. Click here to view.
The Impact on Cost-Effectiveness of Accounting for Generic Drug Pricing: Four Case Studies. Click here to view.
A Perspective on Life-Cycle Health Technology Assessment and Real-World Evidence for Precision Oncology in Canada. Click here to view.
AHRQ Effective Program Updates
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.
Systematic Review: Pharmacologic and Nonpharmacologic Treatments for Posttraumatic Stress Disorder. Click here to view.
Systematic Review: Living Systematic Review on Cannabis and Other Plant-Based Treatments for Chronic Pain. Click here to view.
Job Openings
- Associate Director, Public Engagement, Patient-Centered Outcomes Research Institute. Details.
- Engagement Officer, Public and Patient Engagement, Patient-Centered Outcomes Research Institute. Details.
- Program Officer, Science of Engagement, Patient-Centered Outcomes Research Institute. Details.
