In this week's edition...
— IVI Releases Health Equity Initiative Update. Click here to view the update.
— ICER Names Linda Goler Blount, MPH, to Governance Board. Click here to read the full announcement.
— 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.
— 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 Releases Health Equity Initiative Update. Click here to view the update.
— ICER Names Linda Goler Blount, MPH, to Governance Board. Click here to read the full announcement.
— 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.
— 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 Releases Health Equity Initiative Update. The Innovation and Value Initiative (IVI) released their Health Equity Initiative (HEI) Update – Synthesis of Cross-Stakeholder Insights with the title No Value Without Equity. After intensive dialogue with patients, caregivers, researchers, payers, industry, and employers, IVI identified four key domains impacting the ability to incorporate equity in HTA, including: (1) Power, People, and Processes; (2) Communications and Use; (3) Data, Inputs, and Infrastructure; and (4) Methods.
“We’re privileged to provide a collaboration forum that facilitates solution-focused discourse to help stakeholders measure and make decisions about the value of health technology, with equity as the primary lens,” noted Jennifer Bright, IVI Chief Strategy and Engagement Officer. “Stakeholders need to lean into the complex changes required to make certain that HTA is patient-centered and equity-focused.” Click here to view the update.
2. ICER Names Linda Goler Blount, MPH, to Governance Board. The Institute for Clinical and Economic Review (ICER) announced the election of Linda Goler Blount, MPH, President of the Black Women’s Health Imperative, to ICER's Governance Board. In a statement regarding her election, Ms. Goler Blount explained that “Health technology assessment (HTA) often serves as a filter through which evidence is judged and given context for policymakers’ decisions about pricing, access, and payment. It’s important to continue to examine how to include diverse voices in the patient community, how to interpret data, and how to help highlight opportunities to address the disparities in access and outcomes that persist in our health care system." Click here to read the full announcement.
3. 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.
4. 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.
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.
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 Releases Health Equity Initiative Update. The Innovation and Value Initiative (IVI) released their Health Equity Initiative (HEI) Update – Synthesis of Cross-Stakeholder Insights with the title No Value Without Equity. After intensive dialogue with patients, caregivers, researchers, payers, industry, and employers, IVI identified four key domains impacting the ability to incorporate equity in HTA, including: (1) Power, People, and Processes; (2) Communications and Use; (3) Data, Inputs, and Infrastructure; and (4) Methods.
“We’re privileged to provide a collaboration forum that facilitates solution-focused discourse to help stakeholders measure and make decisions about the value of health technology, with equity as the primary lens,” noted Jennifer Bright, IVI Chief Strategy and Engagement Officer. “Stakeholders need to lean into the complex changes required to make certain that HTA is patient-centered and equity-focused.” Click here to view the update.
2. ICER Names Linda Goler Blount, MPH, to Governance Board. The Institute for Clinical and Economic Review (ICER) announced the election of Linda Goler Blount, MPH, President of the Black Women’s Health Imperative, to ICER's Governance Board. In a statement regarding her election, Ms. Goler Blount explained that “Health technology assessment (HTA) often serves as a filter through which evidence is judged and given context for policymakers’ decisions about pricing, access, and payment. It’s important to continue to examine how to include diverse voices in the patient community, how to interpret data, and how to help highlight opportunities to address the disparities in access and outcomes that persist in our health care system." Click here to read the full announcement.
3. 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.
4. 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: Patients with lung cancer and cystic fibrosis were forced to wait years for Pharmac to fund the treatments they need to address their conditions.
- Australia: Access to treatment for children with cystic fibrosis is being delayed due to long-winded government negotiations.
- Canada. In a recent op-ed, the Best Medicines Coalition writes "people who are very sick are slipping through the cracks, including Zachary, who is stuck between a private insurer that won’t pay and a public plan he can’t afford. Too many Canadians with rare diseases face prescription costs that are twice what the average Canadian makes in a year.”
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.
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.
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.
