In this week's edition...
— Due Today! End Disability Discrimination in Health Care! Share Your Story with AAPD and the Arc. See details below.
— Patients with Sickle Cell Disease Urge FDA to Greenlight Landmark Gene-Editing Treatment. Click here to read the article.
— PCORI Executive Director: Patient-Centered Research Is Critical to Enhancing Care Coordination. Click here to read the blog
— Listening Sessions Can Help CMS Become More Patient-Centered. Here’s How The Sessions Could Be More Effective. Click here to read the article.
— Introducing a Framework to Understand Economic Impacts on Patients and Caregivers. Click here to read the blog.
— PCORI Seeks Nominations for Advisory Panels. Click here to learn more, to apply to serve on an advisory panel, or submit a nomination.
— Emerging Threats in States for Use of Discriminatory Metrics. See details below.
— What Happens in Countries Using QALYs and Cost-Based Thresholds? See details below.
— ICER's QALY-Based Study Topics. See details below.
— Upcoming Events and Webinars. See details below.
— AHRQ Effective Program Updates. See details below.
— Due Today! End Disability Discrimination in Health Care! Share Your Story with AAPD and the Arc. See details below.
— Patients with Sickle Cell Disease Urge FDA to Greenlight Landmark Gene-Editing Treatment. Click here to read the article.
— PCORI Executive Director: Patient-Centered Research Is Critical to Enhancing Care Coordination. Click here to read the blog
— Listening Sessions Can Help CMS Become More Patient-Centered. Here’s How The Sessions Could Be More Effective. Click here to read the article.
— Introducing a Framework to Understand Economic Impacts on Patients and Caregivers. Click here to read the blog.
— PCORI Seeks Nominations for Advisory Panels. Click here to learn more, to apply to serve on an advisory panel, or submit a nomination.
— Emerging Threats in States for Use of Discriminatory Metrics. See details below.
— What Happens in Countries Using QALYs and Cost-Based Thresholds? See details below.
— ICER's QALY-Based Study Topics. See details below.
— Upcoming Events and Webinars. See details below.
— AHRQ Effective Program Updates. See details below.
What We're Reading
1. Due Today! End Disability Discrimination in Health Care! Share Your Story with AAPD and the Arc. Both the Arc and AAPD are soliciting patient stories to share with policymakers as the Department of Health and Human Services (HHS) looks to update existing rules that address discrimination against people with disabilities in government-funded health care programs. Comments are due TODAY. Click here to share your story with the Arc. Click here to share your story with AAPD.
2. Patients with Sickle Cell Disease Urge FDA to Greenlight Landmark Gene-Editing Treatment. An article in the Boston Globe details the push to get the FDA to approve new gene therapies for sickle cell disease. “...Several sickle cell specialists, however, worry about the cost of the treatments ― and the resulting impact on federal and private insurance plans. Each medicine is expected to cost at least $1 million to $2 million, not including the expense of administering the one-time infusions and other aspects of the treatments."
3. PCORI Executive Director: Patient-Centered Research Is Critical to Enhancing Care Coordination. In a new PCORI blog post, Executive Director Nakela Cook explains the importance of patient-centered research in care coordination. “...Care coordination, especially transitional care, has long been on PCORI’s radar, including in the funding of research studies on maternal care and cardiovascular disease, as well as on the transition from pediatric to adult care for individuals living with sickle cell disease. This issue aligns clearly with PCORI’s mission, touches all our National Priorities for Health, and offers opportunities for fruitful collaboration with other organizations and agencies as the health research community seeks evidence supporting innovative ways to enhance quality and coordination of care for patients, families and caregivers.”
4. Listening Sessions Can Help CMS Become More Patient-Centered. Here’s How The Sessions Could Be More Effective. Elisabeth Oehrlein, Hillary Edwards, Thomas Howarth, and Joe Vandigo offer recommendations on making CMS patient listening sessions more patient-centric in an article for Health Affairs. "Our recommendations are based on the lessons we learned while engaging the patient community about their lived experiences; using patient experience data to guide decisions across drug development, real-world studies, and clinical comparative effectiveness research; and balancing patient perspectives with other stakeholders as part of value assessment. Fully engaging patients will ensure that valuable first-hand expertise and experience on the selected drugs and their alternatives are incorporated and available to support patient-centered decision making.”
5. Introducing a Framework to Understand Economic Impacts on Patients and Caregivers. The Innovation and Value Initiative (IVI) and AcademyHealth recently partnered, through a project supported by a Eugene Washington PCORI Engagement Award, to convene a participatory process and develop a framework for researchers that captures the full range of economic impacts on patients and caregivers. The project included two parts: (1) conducting an environmental scan to identify existing methods for defining economic impacts in health-related research; and (2) iterative stakeholder engagement to develop a draft framework. "This framework offers a powerful new tool for researchers to comprehensively co-create with patients a more holistic approach to understanding the patient and caregiver experience. Applying the framework can enhance how we generate evidence to better meet the needs of patients, providers, and policymakers."
6. PCORI Seeks Nominations for Advisory Panels. PCORI is currently seeking nominations for appointments to its advisory panels. These multi-stakeholder advisory panels must include patients, caregivers, or patient advocates. PCORI advisory panels do not serve in an official decision-making capacity, but their recommendations and advice will be taken into consideration by PCORI. PIPC encourages patients, caregivers, and patient advocates to apply or to submit nominations to serve on PCORI's five advisory panels on: Clinical Effectiveness and Decision Science, Clinical Trials, Healthcare Delivery & Disparities Research, Patient Engagement, and Rare Disease. The application is open through March 29, 2024. Click here to learn more, to apply to serve on an advisory panel, or submit a nomination.
Emerging Threats in States for Use of Discriminatory Metrics
Several states are considering policies that would reference entities such as the Institute for Clinical and Economic Review (ICER), which calls the discriminatory quality-adjusted life year (QALY) the “gold standard” for value assessment, and others are considering policies to import QALY-based coverage and reimbursement decisions from other countries that restrict access to care. We encourage you to keep track of all state-based threats using this new website tracking state activities. Key issues are highlighted below.
Oregon
The Oregon legislature passed SB 844 in 2021 creating a Prescription Drug Affordability Board, which was updated based on the Board’s recommendations in 2023 by SB 192. While the legislation included language barring consideration of QALYs and similar formulas, the Board has engaged the Program on Regulation, Therapeutics, and Law (PORTAL), per their meeting agenda for November 15. PORTAL has a subcontract with the Institute for Clinical and Economic Review (ICER) for its work with the Massachusetts Health Policy Commission and has presented on the merits of the QALY and evLYG measures to the Colorado Prescription Drug Affordability Board. This raises concerns about the evidentiary basis for the Board’s decisions and the potential for reference to discriminatory measures of cost effectiveness.
The Oregon Board voted to advance an affordability review of 26 drugs at the October 18, 2023 meeting, with an ambitious plan to conduct the reviews by February. The Board’s rules for conducting an affordability review does not include a robust process for engaging patients and people with disabilities in their decisions. Comments for the November 15, 2023 meeting may be submitted through a comment form.
Colorado
Colorado passed legislation in 2021 creating a Prescription Drug Affordability Review Board. The legislation included language stating that the Upper Payment Limit for selected drugs "shall not consider research or methods that employ a dollars-per-quality adjusted life year, or similar measure, that discounts the value of a life because of an individual's disability or age.” However, this limitation did not clearly extend to the process for determining the selected drugs or the affordability review. Presentations to the Colorado Board from entities such as the Program on Regulation, Therapeutics, and Law (PORTAL) related to cost effectiveness analyses have referenced the merits of using of a cost-per-QALY or the equal value of life-year gained (evLYG) in estimating cost effectiveness of treatments, indicating that these discriminatory measures may have influenced how Colorado selected the drugs and may influence the affordability review. Concerns about the potential for discrimination were described in a letter to the Board from 16 patient and disability organizations. The PDAB offers several opportunities for patients to engage and ensure their voice is heard on these topics. The next PDAB Meeting will be held Friday, December 8 at 10 am MT. Click here to register.
Michigan
In Michigan, legislators introduced, S. 483, which would create a Prescription Drug Affordability Board (PDAB) and institute upper payment limits for certain prescription drugs. The bill passed the Senate and has moved to the House. Advocates have provided testimony urging lawmakers to slow down and ensure any efforts include patient protections, including a QALY ban and Board and Advisory Committee seats for patients and people with disabilities. Click here to view testimony from Laura Bonnell, Bonnell Foundation.
Pennsylvania
In the last legislative session, the legislature introduced, HB 1722, a bill creating a Prescription Drug Affordability Board that would not bar reference to QALYs and would explicitly authorize the Board to contract with a third party that could include ICER. The bill has not been reintroduced in 2023. The Pennsylvania legislature held an informational meeting on Prescription Drug Boards in the House Health Committee on April 12 at 10am. Click here to learn more about Prescription Drug Affordability Boards and the implications of discriminatory measures of cost effectiveness. Click here to learn more about the committee chair, Rep. Dan Frankel.
Massachusetts
An Act Advancing Health Care Research and Decision-Making Centered on Patients and People with disabilities, SD. 2117 and HD. 3356 has been introduced in both the House and Senate. On September 20, 2023, the Massachusetts Joint Committee on Health Care Financing considered the legislation, including provisions that would prohibit reliance on discriminatory measures, including quality-adjusted life years (QALYs) to determine whether a particular health care treatment is cost effective, recommended, the value of a treatment, or in determining coverage, reimbursement, appropriate payment amounts, cost-sharing, or incentive policies or programs. PIPC Chair Tony Coelho testified in support of this legislation, alongside the ALS Foundation and the Epilepsy Foundation of New England.
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 Board of Governors Meeting
December 12, 2023
Click here to view.
Medical Journal Articles
Gene Therapies for Sickle Cell Disease: Effectiveness and Value. Click here to read the article.
White Paper: Improving Patient Subgroup Representation with Real-World Data. Click here to read the article.
A Clinical Pathway to Well-Being: Putting Patient Priorities at the Center of Care. Click here to read the article.
Comparative Effectiveness of Treatments for Rheumatoid Arthritis in Clinical Practice: A Systematic Review. Click here to read the article.
QALYs: The Math Doesn’t Work. Click here to read the article.
The Implementation of Value-Based Frameworks, Clinical Care Pathways, and Alternative Payment Models for Cancer Care in the United States. Click here to view.
Perspectives on Patient-Reported Outcome Data After Treatment Discontinuation in Cancer Clinical Trials. Click here to view.
The Implementation of Value-Based Frameworks, Clinical Care Pathways, and Alternative Payment Models for Cancer Care in the United States. Click here to view.
Federal Data for Conducting Patient-Centered Outcomes Research on Economic Outcomes. Click here to view.
Patient-Reported Outcomes for People with Diabetes: What and How to Measure? A Narrative Review. Click here to view.
Patient Engagement in Research; Benefits, Challenges, Importance, and Implications. Click here to view.
AHRQ Effective Program Updates
Technical Brief: Measuring Healthcare Organization Characteristics in Cancer Care Delivery Research. Click here to view.
Systematic Review: Postpartum Care up to 1 Year After Pregnancy: A Systematic Review and Meta-analysis. Click here to view.
Draft Report: Impact of Healthcare Algorithms on Racial and Ethnic Disparities in Health and Healthcare. Click here to view.
Systematic Review: Partian Breast Irradiation for Breast Cancer. Click here to view.
Systematic Review: Use of Telehealth During the COVID-19 Era. Click here to view.
Research Report: Analysis of Requirements for Coverage with Evidence Development (CED) - Topic Refinement. Click here to view.
Technical Brief: Infection Prevention and Control for the Emergency Medical Services and 911 Workforce. Click here to view.
Systematic Review: Management of Infantile Epilepsies. Click here to view.
1. Due Today! End Disability Discrimination in Health Care! Share Your Story with AAPD and the Arc. Both the Arc and AAPD are soliciting patient stories to share with policymakers as the Department of Health and Human Services (HHS) looks to update existing rules that address discrimination against people with disabilities in government-funded health care programs. Comments are due TODAY. Click here to share your story with the Arc. Click here to share your story with AAPD.
2. Patients with Sickle Cell Disease Urge FDA to Greenlight Landmark Gene-Editing Treatment. An article in the Boston Globe details the push to get the FDA to approve new gene therapies for sickle cell disease. “...Several sickle cell specialists, however, worry about the cost of the treatments ― and the resulting impact on federal and private insurance plans. Each medicine is expected to cost at least $1 million to $2 million, not including the expense of administering the one-time infusions and other aspects of the treatments."
3. PCORI Executive Director: Patient-Centered Research Is Critical to Enhancing Care Coordination. In a new PCORI blog post, Executive Director Nakela Cook explains the importance of patient-centered research in care coordination. “...Care coordination, especially transitional care, has long been on PCORI’s radar, including in the funding of research studies on maternal care and cardiovascular disease, as well as on the transition from pediatric to adult care for individuals living with sickle cell disease. This issue aligns clearly with PCORI’s mission, touches all our National Priorities for Health, and offers opportunities for fruitful collaboration with other organizations and agencies as the health research community seeks evidence supporting innovative ways to enhance quality and coordination of care for patients, families and caregivers.”
4. Listening Sessions Can Help CMS Become More Patient-Centered. Here’s How The Sessions Could Be More Effective. Elisabeth Oehrlein, Hillary Edwards, Thomas Howarth, and Joe Vandigo offer recommendations on making CMS patient listening sessions more patient-centric in an article for Health Affairs. "Our recommendations are based on the lessons we learned while engaging the patient community about their lived experiences; using patient experience data to guide decisions across drug development, real-world studies, and clinical comparative effectiveness research; and balancing patient perspectives with other stakeholders as part of value assessment. Fully engaging patients will ensure that valuable first-hand expertise and experience on the selected drugs and their alternatives are incorporated and available to support patient-centered decision making.”
5. Introducing a Framework to Understand Economic Impacts on Patients and Caregivers. The Innovation and Value Initiative (IVI) and AcademyHealth recently partnered, through a project supported by a Eugene Washington PCORI Engagement Award, to convene a participatory process and develop a framework for researchers that captures the full range of economic impacts on patients and caregivers. The project included two parts: (1) conducting an environmental scan to identify existing methods for defining economic impacts in health-related research; and (2) iterative stakeholder engagement to develop a draft framework. "This framework offers a powerful new tool for researchers to comprehensively co-create with patients a more holistic approach to understanding the patient and caregiver experience. Applying the framework can enhance how we generate evidence to better meet the needs of patients, providers, and policymakers."
6. PCORI Seeks Nominations for Advisory Panels. PCORI is currently seeking nominations for appointments to its advisory panels. These multi-stakeholder advisory panels must include patients, caregivers, or patient advocates. PCORI advisory panels do not serve in an official decision-making capacity, but their recommendations and advice will be taken into consideration by PCORI. PIPC encourages patients, caregivers, and patient advocates to apply or to submit nominations to serve on PCORI's five advisory panels on: Clinical Effectiveness and Decision Science, Clinical Trials, Healthcare Delivery & Disparities Research, Patient Engagement, and Rare Disease. The application is open through March 29, 2024. Click here to learn more, to apply to serve on an advisory panel, or submit a nomination.
Emerging Threats in States for Use of Discriminatory Metrics
Several states are considering policies that would reference entities such as the Institute for Clinical and Economic Review (ICER), which calls the discriminatory quality-adjusted life year (QALY) the “gold standard” for value assessment, and others are considering policies to import QALY-based coverage and reimbursement decisions from other countries that restrict access to care. We encourage you to keep track of all state-based threats using this new website tracking state activities. Key issues are highlighted below.
- Click here to learn more about the potential for Prescription Drug Affordability Boards to discriminate by incorporating the use of QALYS.
- Click here to view the Value Our Health state template legislation that would protect people with disabilities and chronic conditions from the use of QALYs and similar metrics developed by third parties such as ICER in decisions related to reimbursement and coverage, as well as ensure their engagement in decision-making.
- Click here to view a one pager about the flaws in ICER’s methodology.
- Click here to view information from experts on the downside of referencing foreign countries.
- Click here to learn about statutory protections against use of QALYs.
Oregon
The Oregon legislature passed SB 844 in 2021 creating a Prescription Drug Affordability Board, which was updated based on the Board’s recommendations in 2023 by SB 192. While the legislation included language barring consideration of QALYs and similar formulas, the Board has engaged the Program on Regulation, Therapeutics, and Law (PORTAL), per their meeting agenda for November 15. PORTAL has a subcontract with the Institute for Clinical and Economic Review (ICER) for its work with the Massachusetts Health Policy Commission and has presented on the merits of the QALY and evLYG measures to the Colorado Prescription Drug Affordability Board. This raises concerns about the evidentiary basis for the Board’s decisions and the potential for reference to discriminatory measures of cost effectiveness.
The Oregon Board voted to advance an affordability review of 26 drugs at the October 18, 2023 meeting, with an ambitious plan to conduct the reviews by February. The Board’s rules for conducting an affordability review does not include a robust process for engaging patients and people with disabilities in their decisions. Comments for the November 15, 2023 meeting may be submitted through a comment form.
Colorado
Colorado passed legislation in 2021 creating a Prescription Drug Affordability Review Board. The legislation included language stating that the Upper Payment Limit for selected drugs "shall not consider research or methods that employ a dollars-per-quality adjusted life year, or similar measure, that discounts the value of a life because of an individual's disability or age.” However, this limitation did not clearly extend to the process for determining the selected drugs or the affordability review. Presentations to the Colorado Board from entities such as the Program on Regulation, Therapeutics, and Law (PORTAL) related to cost effectiveness analyses have referenced the merits of using of a cost-per-QALY or the equal value of life-year gained (evLYG) in estimating cost effectiveness of treatments, indicating that these discriminatory measures may have influenced how Colorado selected the drugs and may influence the affordability review. Concerns about the potential for discrimination were described in a letter to the Board from 16 patient and disability organizations. The PDAB offers several opportunities for patients to engage and ensure their voice is heard on these topics. The next PDAB Meeting will be held Friday, December 8 at 10 am MT. Click here to register.
Michigan
In Michigan, legislators introduced, S. 483, which would create a Prescription Drug Affordability Board (PDAB) and institute upper payment limits for certain prescription drugs. The bill passed the Senate and has moved to the House. Advocates have provided testimony urging lawmakers to slow down and ensure any efforts include patient protections, including a QALY ban and Board and Advisory Committee seats for patients and people with disabilities. Click here to view testimony from Laura Bonnell, Bonnell Foundation.
Pennsylvania
In the last legislative session, the legislature introduced, HB 1722, a bill creating a Prescription Drug Affordability Board that would not bar reference to QALYs and would explicitly authorize the Board to contract with a third party that could include ICER. The bill has not been reintroduced in 2023. The Pennsylvania legislature held an informational meeting on Prescription Drug Boards in the House Health Committee on April 12 at 10am. Click here to learn more about Prescription Drug Affordability Boards and the implications of discriminatory measures of cost effectiveness. Click here to learn more about the committee chair, Rep. Dan Frankel.
Massachusetts
An Act Advancing Health Care Research and Decision-Making Centered on Patients and People with disabilities, SD. 2117 and HD. 3356 has been introduced in both the House and Senate. On September 20, 2023, the Massachusetts Joint Committee on Health Care Financing considered the legislation, including provisions that would prohibit reliance on discriminatory measures, including quality-adjusted life years (QALYs) to determine whether a particular health care treatment is cost effective, recommended, the value of a treatment, or in determining coverage, reimbursement, appropriate payment amounts, cost-sharing, or incentive policies or programs. PIPC Chair Tony Coelho testified in support of this legislation, alongside the ALS Foundation and the Epilepsy Foundation of New England.
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: Nearly 20,000 Australians suffer from a severe form of psoriasis, but the PBS only just listed a treatment this month.
- Australia: Families are calling for greater access to a medication that helps manage a severe form of epilepsy, but it is not yet available through the PBS.
- Canada: A cancer patient was forced to leave the country to receive treatment as it is not yet available through Newfoundland and Labrador's health care system.
- Canada: A cystic fibrosis (CF) patient recalls the difficult process of lobbying Health Canada to get Trikafta funded.
- New Zealand: Cancer patients are calling for a complete overhaul of Pharmac amid the board's lagging approval of new therapies.
- United Kingdom: Despite positive results from a life-extending lung cancer drug, the NHS is refusing to buy this treatment.
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.
- Post-Traumatic Stress Disorder: Draft Scoping Document Available. Comments due November 21, 2023.
- Paroxysmal Nocturnal Hemoglobinuria: Draft Scoping Document Available. Main Review to be released February 16, 2024.
- Schizophrenia: Draft Scoping Document available. Man Review to be released February 9. 2024.
- Metachromatic Leukodystrophy: Draft Evidence Report now available.
- Pulmonary Arterial Hypertension: Draft Scoping Document available. Open for comments until June 5, 2023.
- Sickle Cell Disease: Evidence Report Available. California Technology Assessment Forum (CTAF) to meet on July 27, 2023, to review.
- Non-Alcoholic Steatohepatitis: Final Evidence Report now available.
Upcoming Events and Webinars
PCORI Board of Governors Meeting
December 12, 2023
Click here to view.
Medical Journal Articles
Gene Therapies for Sickle Cell Disease: Effectiveness and Value. Click here to read the article.
White Paper: Improving Patient Subgroup Representation with Real-World Data. Click here to read the article.
A Clinical Pathway to Well-Being: Putting Patient Priorities at the Center of Care. Click here to read the article.
Comparative Effectiveness of Treatments for Rheumatoid Arthritis in Clinical Practice: A Systematic Review. Click here to read the article.
QALYs: The Math Doesn’t Work. Click here to read the article.
The Implementation of Value-Based Frameworks, Clinical Care Pathways, and Alternative Payment Models for Cancer Care in the United States. Click here to view.
Perspectives on Patient-Reported Outcome Data After Treatment Discontinuation in Cancer Clinical Trials. Click here to view.
The Implementation of Value-Based Frameworks, Clinical Care Pathways, and Alternative Payment Models for Cancer Care in the United States. Click here to view.
Federal Data for Conducting Patient-Centered Outcomes Research on Economic Outcomes. Click here to view.
Patient-Reported Outcomes for People with Diabetes: What and How to Measure? A Narrative Review. Click here to view.
Patient Engagement in Research; Benefits, Challenges, Importance, and Implications. Click here to view.
AHRQ Effective Program Updates
Technical Brief: Measuring Healthcare Organization Characteristics in Cancer Care Delivery Research. Click here to view.
Systematic Review: Postpartum Care up to 1 Year After Pregnancy: A Systematic Review and Meta-analysis. Click here to view.
Draft Report: Impact of Healthcare Algorithms on Racial and Ethnic Disparities in Health and Healthcare. Click here to view.
Systematic Review: Partian Breast Irradiation for Breast Cancer. Click here to view.
Systematic Review: Use of Telehealth During the COVID-19 Era. Click here to view.
Research Report: Analysis of Requirements for Coverage with Evidence Development (CED) - Topic Refinement. Click here to view.
Technical Brief: Infection Prevention and Control for the Emergency Medical Services and 911 Workforce. Click here to view.
Systematic Review: Management of Infantile Epilepsies. Click here to view.
