In this week's edition...
— IVI 2023 Methods Summit Begins Today! Click here for additional details.
— E&C Closes in on QALY Ban. See details below.
— Comments Due Friday! PCORI Request for Information to Enhance Workforce Development Now Open. Click here to view the RFI.
— Applications Due Soon! PCORI Seeks Nominations for Advisory Panels. Click here to learn more, submit a nomination, or apply to be on an advisory panel.
— Op-Ed: The Eugenic Roots of 'Quality Adjusted Life Years' and Why They Matter. Click here to read the op-ed.
— PIPC Submits Comments on PCORI Stakeholder Views on Components of 'Patient-Centered Value' in Health Care. Click here to read the letter.
— IVI Requesting Papers for Valuing Innovation Project. Click here for additional details.
— 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.
— IVI 2023 Methods Summit Begins Today! Click here for additional details.
— E&C Closes in on QALY Ban. See details below.
— Comments Due Friday! PCORI Request for Information to Enhance Workforce Development Now Open. Click here to view the RFI.
— Applications Due Soon! PCORI Seeks Nominations for Advisory Panels. Click here to learn more, submit a nomination, or apply to be on an advisory panel.
— Op-Ed: The Eugenic Roots of 'Quality Adjusted Life Years' and Why They Matter. Click here to read the op-ed.
— PIPC Submits Comments on PCORI Stakeholder Views on Components of 'Patient-Centered Value' in Health Care. Click here to read the letter.
— IVI Requesting Papers for Valuing Innovation Project. Click here for additional details.
— 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. IVI 2023 Methods Summit Begins Today! The Innovation and Value Initiative (IVI) will hold its 4th Annual Methods Summit starting today at 10:00 AM, and continuing through tomorrow. This year's summit will identify actionable changes and outline immediate actions, roles, and accountability metrics needed for substantive change to achieve health equity and true value in health technology assessment. IVI CEO Jason Spangler will also host a conversation with Dr. Dara Hughes, the Chief Medical Officer at the CMS Innovation center, on how CMS is approaching health equity for the largest healthcare system in the country and what they have learned so far. Click here for additional details.
2. E&C Closes in on QALY Ban. House Energy and Commerce Committee Chair Cathy McMorris Rodgers (R-WA) and Health Subcommittee Ranking Member Anna Eshoo (D-CA) are closing in on a an bipartisan agreement on legislation that would ban the QALY after the Protecting Health Care for Patients All Act passed the subcommittee with strong bipartisan support last Wednesday. The bill will now head to the full committee for a markup at a date and time to be announced. “There’s a bit more work needed to fully vet and agree to this proposed language," said Rep. Eshoo. "I think we’re nearly there, and I look forward to voting for a bipartisan amendment when the full committee convenes at our next markup."
3. Comments Due Friday! PCORI Request for Information to Enhance Workforce Development. 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.
4. Applications Due Soon! PCORI 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.
5. Op-Ed: The Eugenic Roots of 'Quality Adjusted Life Years' and Why They Matter. In an op-ed for The Washington Post, Pepper Stetler explains how the QALY has discriminated against patients and people with disabilities. "Perhaps QALY wouldn’t seem acceptable if we weren’t already so used to calculated predictions that maximize functioning and performance outcomes but minimize the value of the lives of people with disabilities. But this calculation is not inevitable. Several alternatives to QALY have been proposed that acknowledge the complexity of health-care decisions and calculate the cost of medical treatment without penalizing disability. The best alternative would require us to accept disability as a valuable part of the diversity of human life, rather than a strange distortion in time.” Click here to read the op-ed.
6. PIPC Submits Comments on PCORI Stakeholder Views on Components of 'Patient-Centered Value' in Health Care. PIPC submitted comments to PCORI on its draft report entitled "Stakeholder Views on Components of 'Patient-Centered Value' in Health and Health Care." While the letter applauds PCORI's consistent emphasis on including the patient voice in every step of research, PIPC Chair Tony Coelho expressed concerns that PCORI included disability-adjusted life years (DALYs) as an attribute of life and social impacts per the feedback of some clinicians and payers. As PCORI advances this work, PIPC' urges consideration of the differences among patients based on their condition or disease, age, social determinants of health, etc. so that researchers are prioritizing the collection of data on attributes that matter to the patients in question. Click here to read the letter.
7. IVI Requesting Papers for Valuing Innovation Project. IVI is inviting eligible individuals and research teams to submit papers as a part of its Valuing Innovation Project (VIP). The VIP specifically seeks to advance research on a set of prioritized questions to better define, measure, and reward innovations in the context of health technology assessment (HTA). To submit an entry, applicants should e-mail the required materials to [email protected] by April 2, 2023, 11:59 PM PT. Click here for additional details.
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, SF168 and HF17. 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 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.
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
How to Supercharge Real World Evidence Generation in Phase IV Trials
March 16, 2023
Click here to view.
Virtual Sites—Are They the Future of RWE?
March 29, 2023
Click here to view.
PCORnet Studies Applicant Workshop
May 18, 2023
Click here to view.
Medical Journal Articles
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.
1. IVI 2023 Methods Summit Begins Today! The Innovation and Value Initiative (IVI) will hold its 4th Annual Methods Summit starting today at 10:00 AM, and continuing through tomorrow. This year's summit will identify actionable changes and outline immediate actions, roles, and accountability metrics needed for substantive change to achieve health equity and true value in health technology assessment. IVI CEO Jason Spangler will also host a conversation with Dr. Dara Hughes, the Chief Medical Officer at the CMS Innovation center, on how CMS is approaching health equity for the largest healthcare system in the country and what they have learned so far. Click here for additional details.
2. E&C Closes in on QALY Ban. House Energy and Commerce Committee Chair Cathy McMorris Rodgers (R-WA) and Health Subcommittee Ranking Member Anna Eshoo (D-CA) are closing in on a an bipartisan agreement on legislation that would ban the QALY after the Protecting Health Care for Patients All Act passed the subcommittee with strong bipartisan support last Wednesday. The bill will now head to the full committee for a markup at a date and time to be announced. “There’s a bit more work needed to fully vet and agree to this proposed language," said Rep. Eshoo. "I think we’re nearly there, and I look forward to voting for a bipartisan amendment when the full committee convenes at our next markup."
3. Comments Due Friday! PCORI Request for Information to Enhance Workforce Development. 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.
4. Applications Due Soon! PCORI 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.
5. Op-Ed: The Eugenic Roots of 'Quality Adjusted Life Years' and Why They Matter. In an op-ed for The Washington Post, Pepper Stetler explains how the QALY has discriminated against patients and people with disabilities. "Perhaps QALY wouldn’t seem acceptable if we weren’t already so used to calculated predictions that maximize functioning and performance outcomes but minimize the value of the lives of people with disabilities. But this calculation is not inevitable. Several alternatives to QALY have been proposed that acknowledge the complexity of health-care decisions and calculate the cost of medical treatment without penalizing disability. The best alternative would require us to accept disability as a valuable part of the diversity of human life, rather than a strange distortion in time.” Click here to read the op-ed.
6. PIPC Submits Comments on PCORI Stakeholder Views on Components of 'Patient-Centered Value' in Health Care. PIPC submitted comments to PCORI on its draft report entitled "Stakeholder Views on Components of 'Patient-Centered Value' in Health and Health Care." While the letter applauds PCORI's consistent emphasis on including the patient voice in every step of research, PIPC Chair Tony Coelho expressed concerns that PCORI included disability-adjusted life years (DALYs) as an attribute of life and social impacts per the feedback of some clinicians and payers. As PCORI advances this work, PIPC' urges consideration of the differences among patients based on their condition or disease, age, social determinants of health, etc. so that researchers are prioritizing the collection of data on attributes that matter to the patients in question. Click here to read the letter.
7. IVI Requesting Papers for Valuing Innovation Project. IVI is inviting eligible individuals and research teams to submit papers as a part of its Valuing Innovation Project (VIP). The VIP specifically seeks to advance research on a set of prioritized questions to better define, measure, and reward innovations in the context of health technology assessment (HTA). To submit an entry, applicants should e-mail the required materials to [email protected] by April 2, 2023, 11:59 PM PT. Click here for additional details.
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, SF168 and HF17. 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 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.
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.
- New Zealand: Cancer patients in New Zealand are struggling to raise funds on their own to cover treatments that are not funded by Pharmac.
- United Kingdom: Patients are rallying support for the NHS to cover treatment for cervical and rare eye cancers that are not being funded in the U.K.
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: Revised 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
How to Supercharge Real World Evidence Generation in Phase IV Trials
March 16, 2023
Click here to view.
Virtual Sites—Are They the Future of RWE?
March 29, 2023
Click here to view.
PCORnet Studies Applicant Workshop
May 18, 2023
Click here to view.
Medical Journal Articles
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.
