In this week's edition...
— Comments Due Friday! Stakeholder Views on Components of "Patient-Centered Value” in Health and Health Care. Click here to view the report.
— PCORI Seeks Nominations for Advisory Panels. Click here to learn more, submit a nomination, or apply to be on an advisory panel.
— Interview: A Bipartisan End to QALYs? Click here to view the report.
— New NCD Funding Opportunity: The Absence of People with Disabilities in Clinical Trials. Click here to view the report.
— House Labor-HHS Subcommittee Announces Public Witness Hearing. See details below.
— PCORI Request for Information to Enhance Workforce Development Now Open. Click here to view the RFI.
— 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.
— Comments Due Friday! Stakeholder Views on Components of "Patient-Centered Value” in Health and Health Care. Click here to view the report.
— PCORI Seeks Nominations for Advisory Panels. Click here to learn more, submit a nomination, or apply to be on an advisory panel.
— Interview: A Bipartisan End to QALYs? Click here to view the report.
— New NCD Funding Opportunity: The Absence of People with Disabilities in Clinical Trials. Click here to view the report.
— House Labor-HHS Subcommittee Announces Public Witness Hearing. See details below.
— PCORI Request for Information to Enhance Workforce Development Now Open. Click here to view the RFI.
— 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. Comments Due Soon! Stakeholder Views on Components of "Patient-Centered Value” in Health and Health Care. PCORI has recently released draft report on potential components of patient-centered value in health and health care. The report, Stakeholder Views on Components of "Patient-Centered Value” in Health and Health Care, summarizes the result of a multi-step process by PCORI to gather views of patients, families, caregivers, and the broader healthcare community on the potential components, attributes, and measurement of patient-centered value and related data collection. Comments on the report can be submitted through Friday, March 3. Click here to view the report.
2. PCORI Seeks Nominations for Advisory Panels. PCORI is currently seeking nominations for appointments to its advisory panels. The advisory panels must include patients or patient advocates. PCORI's staff, board, and methodology committee take advisory panels' recommendations into account when making decisions and determinations. PIPC encourages patients and patient advocates to submit nominations to serve on PCORI's advisory panels, including on the Advisory Panel on Clinical Effectiveness and Decision Science, Advisory Panel on Patient Engagement, and Advisory Panel on Rare Disease. Nominations are open through March 31, 2023. Click here to learn more, submit a nomination, or apply to be on an advisory panel.
3. Interview: A Bipartisan End to QALYs? Virginia Buckingham recently interviewed Dr. William S. Smith, the Senior Fellow and Director of the Life Sciences Initiative at the Pioneer Institute, on his forthcoming book on QALYS and rationing care. "The QALY has this simplistic two–poled goal of extending life and improving the quality of life, and I don’t think valuing drugs is that simple. It’s not that easy when you look at specific conditions. The same thing applies to rare disease. A rare disease drug is going to help a patient where there’s only 1000 patients in the whole country with that particular disease." Click here to read the interview.
4. New NCD Funding Opportunity: The Absence of People with Disabilities in Clinical Trials. The National Council on Disability (NCD) announced a Notice of Funding Opportunity for a report on increasing participation among the disability community in clinical trials. The purpose of this report is to address the explicit and implicit exclusion of people with disabilities in clinical trials, the repercussions of exclusion on the disability community, the legal requirements of clinical trial investigators to include PWDs in clinical trials and repercussions for not abiding by those requirements. The final report will provide recommendations and promising practices to increase the acceptance and participation rates of people with disabilities in clinical trials. Applications are due on March 22, 2023. Click here for additional details.
5. House Labor-HHS Subcommittee Announces Public Witness Hearing. The House Appropriations Subcommittee on Labor, Health and Human Services (HHS), Education, and Related Agencies will hold a hearing to receive public witness testimony on Thursday, March 23, 2023. Requests to testify are due on Friday, March 3 at 5:00 PM EST. Written testimony can be submitted up until the hearing date of March 23. Instructions on submitting requests to testify in-person can be viewed here.
6. PCORI Request for Information to Enhance Workforce Development Now Open. For this RFI, PCORI is seeking input on the opportunities, gaps, and challenges in the current PCOR workforce landscape, and input on additional programmatic initiatives, investments, and/or partnerships that would accelerate the development of the PCOR workforce and facilitate the conduct of PCOR in alignment with PCORI’s National Priorities for Health, Research Agenda, and Strategic Plan. Comments should be submitted to [email protected]. by March 17, 2023. Click here to view the RFI.
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.
Minnesota
The Minnesota state legislature has introduced a bill to create a Prescription Drug Affordability Board, HF 17. It was heard by the Commerce Finance and Policy Committee on January 23, 2023. It does not include a bar on using QALYs or other metrics that discriminate. Also, the bill specifically references Canadian prices as a consideration for establishing the upper payment limit for drugs, prices which are explicitly based on QALYs.
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
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
MedPAC March 2023 Public Meeting
March 2-3, 2023
Click here to view.
MACPAC March 2023 Public Meeting
March 2-3, 2023
Click here to view.
PCORI Board of Governors Meeting
March 7, 2023
Click here to view.
Virtual V-BID Summit 2023: Accelerating Health Equity
March 8, 2023
Click here to view.
How to Supercharge Real World Evidence Generation in Phase IV Trials
March 16, 2023
Click here to view.
PCORnet Studies Applicant Workshop
May 18, 2023
Click here to view.
Medical Journal Articles
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.
Identifying And Exploring Bias in Public Opinion on Scarce Resource Allocation During The COVID-19 Pandemic. Click here to view.
Machine Learning Applied to Patient-Reported Outcomes to Classify Physician-Derived Measures of Rheumatoid Arthritis Disease Activity. Click here to view.
Patient-Centered Pain Care Using Artificial Intelligence and Mobile Health Tools: A Randomized Comparative Effectiveness Trial. 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.
1. Comments Due Soon! Stakeholder Views on Components of "Patient-Centered Value” in Health and Health Care. PCORI has recently released draft report on potential components of patient-centered value in health and health care. The report, Stakeholder Views on Components of "Patient-Centered Value” in Health and Health Care, summarizes the result of a multi-step process by PCORI to gather views of patients, families, caregivers, and the broader healthcare community on the potential components, attributes, and measurement of patient-centered value and related data collection. Comments on the report can be submitted through Friday, March 3. Click here to view the report.
2. PCORI Seeks Nominations for Advisory Panels. PCORI is currently seeking nominations for appointments to its advisory panels. The advisory panels must include patients or patient advocates. PCORI's staff, board, and methodology committee take advisory panels' recommendations into account when making decisions and determinations. PIPC encourages patients and patient advocates to submit nominations to serve on PCORI's advisory panels, including on the Advisory Panel on Clinical Effectiveness and Decision Science, Advisory Panel on Patient Engagement, and Advisory Panel on Rare Disease. Nominations are open through March 31, 2023. Click here to learn more, submit a nomination, or apply to be on an advisory panel.
3. Interview: A Bipartisan End to QALYs? Virginia Buckingham recently interviewed Dr. William S. Smith, the Senior Fellow and Director of the Life Sciences Initiative at the Pioneer Institute, on his forthcoming book on QALYS and rationing care. "The QALY has this simplistic two–poled goal of extending life and improving the quality of life, and I don’t think valuing drugs is that simple. It’s not that easy when you look at specific conditions. The same thing applies to rare disease. A rare disease drug is going to help a patient where there’s only 1000 patients in the whole country with that particular disease." Click here to read the interview.
4. New NCD Funding Opportunity: The Absence of People with Disabilities in Clinical Trials. The National Council on Disability (NCD) announced a Notice of Funding Opportunity for a report on increasing participation among the disability community in clinical trials. The purpose of this report is to address the explicit and implicit exclusion of people with disabilities in clinical trials, the repercussions of exclusion on the disability community, the legal requirements of clinical trial investigators to include PWDs in clinical trials and repercussions for not abiding by those requirements. The final report will provide recommendations and promising practices to increase the acceptance and participation rates of people with disabilities in clinical trials. Applications are due on March 22, 2023. Click here for additional details.
5. House Labor-HHS Subcommittee Announces Public Witness Hearing. The House Appropriations Subcommittee on Labor, Health and Human Services (HHS), Education, and Related Agencies will hold a hearing to receive public witness testimony on Thursday, March 23, 2023. Requests to testify are due on Friday, March 3 at 5:00 PM EST. Written testimony can be submitted up until the hearing date of March 23. Instructions on submitting requests to testify in-person can be viewed here.
6. PCORI Request for Information to Enhance Workforce Development Now Open. For this RFI, PCORI is seeking input on the opportunities, gaps, and challenges in the current PCOR workforce landscape, and input on additional programmatic initiatives, investments, and/or partnerships that would accelerate the development of the PCOR workforce and facilitate the conduct of PCOR in alignment with PCORI’s National Priorities for Health, Research Agenda, and Strategic Plan. Comments should be submitted to [email protected]. by March 17, 2023. Click here to view the RFI.
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.
Minnesota
The Minnesota state legislature has introduced a bill to create a Prescription Drug Affordability Board, HF 17. It was heard by the Commerce Finance and Policy Committee on January 23, 2023. It does not include a bar on using QALYs or other metrics that discriminate. Also, the bill specifically references Canadian prices as a consideration for establishing the upper payment limit for drugs, prices which are explicitly based on QALYs.
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
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: Families of children with cystic fibrosis are forced to pay tens of thousands per month for Trikafta because the PBS has not funded the treatment for kinds under 12.
- Canada: Patient advocates Beth and Madi Vanstone, CF Canada, and the Canadian Organization for Rare Diseases are rallying for a rare disease drug strategy that promotes access and affordability.
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.
- Amyotrophic Lateral Sclerosis (ALS): Final Evidence Report and Meeting Summary available. Evidence Presentation available. Draft Evidence Report available. Public comments closed. Model Analysis Plan available. Research Protocol available. Revised Scoping Document available.
- Sickle Cell Disease: Research Protocol available. 02/27/2023: Model Analysis Plan. Revised Scoping Document available. Draft Scoping Document available. Public Comments closed.
- Non-Alcoholic Steatohepatitis: Draft Evidence Report available. Public comments open until 3/16/2022. Model Analysis Plan available. Research Protocol available. Revised Scoping Document available. Draft Scoping Document available. Public comments closed.
- Menopause: Vasomotor Symptoms: Final Evidence Report and Meeting Summary available. Evidence Report and Presentation available. Draft Evidence Report available. Model Analysis Plan available.
- Alzheimer's Disease: Draft Evidence Report available. Public comments open until 2/2/2023. 3/1/2023: Evidence Report. Model Analysis Plan available. Research Protocol available. Revised Scoping Document available.
- Beta Thalassemia: Final Evidence Report and Meeting Summary available. Evidence Presentation available. Draft Evidence Report available. Public comments closed. Model Analysis Plan available. Research Protocol available. Revised Scoping Document available.
- COVID-19: Final Evidence Report and Meeting Summary available. Evidence Presentation and Report available. Draft Evidence Report Available. Public comments closed. Model Analysis Plan available. Draft Scoping Document and Research Protocol available.
- Obesity Management: Final Evidence Report and Meeting Summary available. Evidence Presentation available. Draft Evidence Report available. Model Analysis Plan available. Research Protocol available.
- Multiple Sclerosis: CIS, RRMS, and SPMS: Evidence Presentation available. 2/17/2023: Final Evidence Report and Meeting Summary. Evidence Report available. Draft Evidence Report available. Model Analysis Plan available. Research Protocol available. Revised Scoping Document available. Draft Scoping Document available.
- Gene Therapies for Hemophilia A and B: Final Evidence Report and Meeting Summary available. Evidence Presentation available. Evidence Report available. Draft Evidence Report available. Public comments closed. Model Analysis Plan available. Revised Scoping Document available. Draft Scoping Document available.
Upcoming Events and Webinars
MedPAC March 2023 Public Meeting
March 2-3, 2023
Click here to view.
MACPAC March 2023 Public Meeting
March 2-3, 2023
Click here to view.
PCORI Board of Governors Meeting
March 7, 2023
Click here to view.
Virtual V-BID Summit 2023: Accelerating Health Equity
March 8, 2023
Click here to view.
How to Supercharge Real World Evidence Generation in Phase IV Trials
March 16, 2023
Click here to view.
PCORnet Studies Applicant Workshop
May 18, 2023
Click here to view.
Medical Journal Articles
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.
Identifying And Exploring Bias in Public Opinion on Scarce Resource Allocation During The COVID-19 Pandemic. Click here to view.
Machine Learning Applied to Patient-Reported Outcomes to Classify Physician-Derived Measures of Rheumatoid Arthritis Disease Activity. Click here to view.
Patient-Centered Pain Care Using Artificial Intelligence and Mobile Health Tools: A Randomized Comparative Effectiveness Trial. 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.
