1. PIPC Submits Comments to PCORI on Proposed Research Agenda. Click here to view the letter.
2. PIPC Offers Feedback on IVI Draft Model on Major Depressive Disorder. Click here to view the letter.
3. PIPC Submits Comments to HHS on Proposed Notice of Benefit and Payment Parameters Rule. Click here to view the letter.
4. MACPAC Panel Urges Commission to Increase Beneficiary Involvement. Click here to view the article.
5. Lund Report: Oregon Faces Scrutiny Over One-Of-A-Kind Hurdles to Children’s Health Care. Click here to view the article.
6. New Targeted PCORI Funding Announcement: Comparative Effectiveness of Interventions Targeting Mental Health in Individuals with Intellectual and Developmental Disabilities. See below for details.
7. On the Podcast: Chronic Disease Care at Risk in Oregon. Click here to listen.
8. PIPC Holds 12th Annual Forum: The Call to Action to Bar Reference to Discriminatory Metrics in U.S. Health Pricing and Coverage Policy. Click here to view the forum.
9. IHP: Disability Agency Urges Congress to Ban QALYs in All Federal Programs. Click here to read the article ($). Click here to view the NCD letter sent to Congress.
10. PIPC and Morning Consult Publish Poll Showing Strong Support for NCD Recommendations to Ban QALYs. Click here to view the poll results.
11. PCORI Seeks Nominations for Advisory Panels. Click here to learn more, submit a nomination, or apply to be on an advisory panel.
12. DREDF Releases Report Responding to a Flawed ICER Analyses, Instead Finds that QALY Violates Disability Nondiscrimination Law. Click here to view the report.
13. Upcoming opportunity within the Center for Evidence and Practice Improvement at AHRQ, see details below.
14. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
15. Emerging Threats in States for Use of Discriminatory Metrics, see details below.
16. ICER's QALY-Based Study Topics: Alzheimer's Disease, Beta Thalassemia, Chemotherapy-Induced Neutropenia, COVID-19, Type 2 Diabetes, Hypertrophic Cardiomyopathy, Asthma, Unsupported Price Increase, Fair Access: Coverage Policies. Click here to provide patient input.
17. Upcoming Events and Webinars, see details below.
18. Medical Journal Articles, see details below.
19. AHRQ Effective Program Updates, see details below.
In a letter to the Patient-Centered Outcomes Research Institute (PCORI), the Partnership to Improve Patient Care (PIPC) offered feedback on PCORI's proposed research agenda. In the letter, PIPC Chair Tony Coelho doubled down on his recommendation that PCORI engage in direct outreach to organizations representing patients and people with disabilities in its forthcoming research efforts, noting that PCORI must be more specific and clear with respect to its methodologies. The letter also stresses that PCORI should work with experts that do not view the discriminatory quality-adjusted-life-years (QALY) metric as the "gold standard" for value assessment in health care.
"When health care decisions are made without reliable data on the experience of patients and their preferences, we have a systematic problem," wrote Chair Coelho. "The solution to that problem is a clear and credible source for decision-makers to not only directly access outcomes data meeting criteria for being patient-centered, but to also better understand when – or not – the outcomes data they are relying on is indeed credible and representative of diverse patient perspectives."
2. PIPC Offers Feedback on IVI Draft Model on Major Depressive Disorder
In a letter to the Innovation and Value Initiative (IVI), the Partnership to Improve Patient Care (PIPC) submitted comments on IVI's draft model protocol on major depressive disorder (MDD). The letter notes that, as designed, the model is unlikely to be able to address the issue of treatment heterogeneity, suggesting that IVI broaden the question of how to estimate the accrual of "marginal value" from new therapies. PIPC also strongly encouraged IVI to cease using of the quality-adjusted-life-years metric (QALY) in its models.
"We appreciate the process you have set forward providing transparency into and encouraging stakeholder feedback on your model and process," wrote PIPC Chair Tony Coelho. "This type of transparency and robust stakeholder engagement, particularly from patients and providers, leads to stronger models that more accurately convey value to patients and society."
3. PIPC Submits Comments to HHS on Proposed Notice of Benefit and Payment Parameters Rule
In a letter to the Department of Health and Human Services (HHS), Partnership to Improve Patient Care (PIPC) Chair Tony Coelho provided feedback on the Notice of Benefit and Payment Parameters 2023 Proposed Rule. The letter stresses that: (1) plans should not be permitted to reference the discriminatory quality-adjusted-life-years (QALY) metric; (2) research meets the standards of patient-centeredness; and (3) full transparency should be required regarding sources used in decision making. PIPC is grateful to HHS for recognizing that more guidance is needed to ensure that health plans are complying with the Essential Health Benefits (EHB) nondiscrimination policy and would encourage you to consider the following comments.
"PIPC is grateful to HHS for recognizing that more guidance is needed to ensure that health plans are complying with the Essential Health Benefits (EHB) nondiscrimination policy and would encourage you to consider the following comments," wrote Chair Coelho. "We appreciate the Administration’s commitment to nondiscrimination and would be happy to remain a resource as you continue to strengthen non-discrimination policies."
4. MACPAC Panel Urges Commission to Increase Beneficiary Involvement.
At its January meeting, the Medicaid and CHIP Payment and Access Commission (MACPAC) was urged to continue creating ways to increase beneficiaries’ engagement with the policy-making process. According to Inside Health Policy, "Advocates said more engagement with marginalized groups, especially disabled people of color and the queer community, as well as promoting meaningful exchanges with beneficiaries would help build a system that meets beneficiaries’ needs and increases overall retention rates.” Click here to view the MACPAC summary. Click here to view the IHP article (subscription only).
5. Lund Report: Oregon Faces Scrutiny Over One-Of-A-Kind Hurdles to Children’s Health Care.
Scrutiny of the Oregon waiver and its “prioritized list of services” was further explored in a recent article in Oregon’s Lund Report. The article states, "Now, as the state seeks to renew federal approval of the Oregon Health Plan’s specialized waiver from Medicaid requirements, the state’s alone-in-the-nation approach toward children’s benefits has become a target for parents, providers and advocates. National advocates have also put a public spotlight on Oregon.” The article further states, "When the system fails, children fall through the cracks.” The article quoted a recent letter to the state from the Oregon Center for Children and Youth with Special Health Needs, a state public health agency, calling the approach “fundamentally racist” and “unacceptable.” The article also quoted Joan Alker, Medicaid policy researcher and executive director at the Center for Children and Families at Georgetown University McCourt School of Public Policy, who said, "Oregon is the only state in the country that has this broad waiver of (the federal children’s health requirement), and there’s simply no good reason at all for it, and it needs to go away.” Click here to view the article.
6. New Targeted PCORI Funding Announcement: Comparative Effectiveness of Interventions Targeting Mental Health in Individuals with Intellectual and Developmental Disabilities
On Tuesday, January 11, 2022, the Patient-Centered Outcomes Research Institute (PCORI) released the Comparative Effectiveness of Interventions Targeting Mental Health in Individuals with Intellectual and Developmental Disabilities (IDD) Targeted PCORI Funding Announcement (Targeted PFA). This Targeted PFA aims to fund high-quality, rigorous randomized controlled trials or observational, prospective cohort studies that compare the effectiveness of evidence-based approaches (e.g., specific pharmacological and behavioral interventions) that address mental health conditions in individuals with IDD. Proposed studies should compare the effectiveness of pharmacological, behavioral, or other nonpharmacological or combination interventions administered via appropriate delivery modalities (e.g., telehealth, family-based, group, or individual).
Studies focused on pediatric, transitional age, and adult individuals with mild to moderate IDD-related impairment, those with more severe impairment due to IDDs, underrepresented groups, and underserved populations (e.g., individuals in rural settings) are of particular interest.
Here is the funding announcement for more information about this Targeted PFA.
7. On the Podcast: Chronic Disease Care at Risk in Oregon
States are like dominos. When one state enacts a health policy, others replicate. Oregon’s Medicaid draft 1115 waiver has several red flags for patient healthcare access, and all patients should watch this carefully. Meghan Moyer, the public policy director at Disability Rights Oregon, explains why the waiver is a concern for chronic and rare disease patients. From discriminatory healthcare metrics that determine coverage to a closed formulary that would hinder access to FDA accelerated approval drugs, it’s filled with problems for patients. Click here to listen to the podcast.
8. PIPC Holds 12th Annual Forum: The Call to Action to Bar Reference to Discriminatory Metrics in U.S. Health Pricing and Coverage Policy.
The 12th Annual PIPC Forum on The Call to Action to Bar Reference to Discriminatory Metrics in U.S. Health Pricing and Coverage Policy, focused on the recommendation of the National Council on Disability (NCD) for an unambiguous ban on the quality adjusted life year (QALY) within the text of the Build Back Better Act (H.R. 5367), as well as the increasing reference to comparative and cost effectiveness assessments in determining reimbursement, coverage, and programs at the state and federal level. Click here to view the forum.
The Chairman of the National Council on Disability, Andrés Gallegos, stated, “In our most recent advice to Congress we advise that Congress extend those prohibitions [prohibitions against the use of the QALY in the ACA] throughout federal programs. The Build Back Better Act is a tremendous opportunity to do so and to memorialize the protections with an unambiguous ban on the use of the QALY. We remain hopeful that our advice will be heeded as we all await the final text of that bill.”
9. IHP: Disability Agency Urges Congress to Ban QALYs In All Federal Programs
Inside Health Policy highlighted statements made by the Chairman of the National Council on Disability (NCD) at the annual PIPC Forum. The article stated, "After getting the Senate Finance Committee to ban Medicare from using a common metric for appraising drug prices, the government agency National Council on Disability is asking Congress to use the Build Back Better bill to prohibit the use of the quality-adjusted life years metric in all government programs.” The article stated that the NCD sent a letter to Congress encouraging the inclusion of a QALY ban within the bill that applies to all Medicare price negotiation and that is extended to all government programs. The article highlighted that the "U.S. government does not have a uniform policy on QALYs. Some federal agencies are banned from using measurement tools like QALYs, but state Medicaid programs use them.” Chairman Gallegos was quoted as stating, “In our most recent advice to Congress we advise that Congress extend those prohibitions [against the use of the QALY in the ACA] throughout federal programs.” Chairman Gallegos also stated, “The Build Back Better Act is a tremendous opportunity to do so and to memorialize the protections with an unambiguous ban on the use of the QALY.” Click here to view the article (subscription required). Click here to view the NCD letter sent to Congress.
10. PIPC and Morning Consult Publish Poll Showing Strong Support for NCD Recommendations to Ban QALYs
Morning Consult ran a poll on behalf of Partnership to Improve Patient Care focusing on the use of cost-effectiveness assessments to determine the value of coverage and treatment costs. The survey found that Americans want patients and their doctors in charge of health care decision making and are opposed to the use of cost assessments such as Quality-Adjusted Life Year (QALY).
PIPC Chairman Tony Coelho published an opinion piece in Morning Consult about the poll, stating, "Ten years after the Partnership to Improve Patient Care (PIPC) released its first public opinion survey, our latest poll released today underscores how vitally important it is for lawmakers to maintain and strengthen safeguards for patients and people with disabilities in all health care policies.” Chairman Coelho was heartened to see the National Council on Disability — an independent federal agency that advises Congress and the administration on disability policy — recently come out with targeted recommendations to strengthen BBBA by including meaningful protections against government use of discriminatory cost-effectiveness thresholds based on the quality-adjusted life year (QALY). He emphasized that this recommendation represents an important step in protecting patients and patients with disabilities. Despite the long history in the United States acknowledging that metrics like QALYs discriminate in violation of disability and civil rights laws, their use is rising —not falling. He concluded that it is time to finally end the ambiguity and prohibit QALYs altogether.
11. 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, 2022. Click here to learn more, submit a nomination, or apply to be on an advisory panel.
12. DREDF Releases Report Responding to a Flawed ICER Analyses, Instead Finds that QALY Violates Disability Nondiscrimination Law
The Disability Rights Education and Defense Fund ("DREDF") responded directly to a report commissioned for the Institute for Clinical and Economic Review (“ICER”), an organization specializing in clinical cost-effectiveness analyses relying on the QALY, in which its hired legal consultants posit that the use of QALY as a measure of the cost-effectiveness of specific drugs and therapies “poses absolutely no risk of discrimination against any patient group.” DREDF strongly disagrees with this conclusion. In its response, DREDF challenges the factual and legal assumptions of ICER's report and explains how the use of the QALY, even in tandem with alternative measures such as the Equal Value of Life Years Gained (“evLYG”), violates disability nondiscrimination law.
DREDF will join Familia Unida to celebrate National Disabilities Awareness Month and share information about its new report and the ongoing work of the disability community against disability discrimination in healthcare. Click here to view the DREDF report. Click here to view the one-pager. Click here to find summaries in both English and Spanish.
13. Upcoming Opportunity Within the Center for Evidence and Practice Improvement at AHRQ
AHRQ will soon be hiring a Senior Staff Service Fellow in CEPI’s Office of the Director to support activities funded by the Patient-Centered Outcomes Research Trust Fund (PCOR TF) under the National Center for Excellence in Primary Care Research (NCEPCR). The Senior Staff Service Fellow will support AHRQ’s mandated roles under the PCOR TF in evidence dissemination and implementation in primary care as well as of training and capacity building for comparative effectiveness research in primary care settings.
The Senior Staff Service Fellow designs, executes and evaluates projects funded by the PCOR TF to increase the uptake of evidence in primary care including by using new models of primary/ambulatory care delivery, advancing health equity and engaging learning health systems. AHRQ is looking for a team player to lead a multidisciplinary team to design, that leads national initiatives to disseminate and implement PCOR findings related to transforming primary/ambulatory care delivery to make care more equitable and patient-centered including a focus on people living with multiple chronic conditions and other at-risk populations.
This Senior Staff Service Fellow will also advise other members of CEPI and AHRQ on dissemination and implementation activities related to the PCOR TF; supports and aligns ongoing primary care projects across all divisions within the Center as well as across AHRQ. Serves as a recognized expert within the areas of primary care, primary care research and implementation science.
14. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage?
Other countries are often referenced as examples of how the use of QALYs or similar cost-based thresholds impact access to care.
- Australia: Thousands of patients who are struggling with CF are campaigning for Trikafta, a life-saving drug, to be added to the PBS due to its incredibly expensive cost.
- New Zealand: Former bowel cancer patient is calling for the NZ government to cover Cetuximab, which saved her life, or have it available at hospitals to cut the price.
- United Kingdom: MS patient cannot afford food or heating due to the expensive, life-changing drug, fampridine, which the NHS refuses to cover.
15. 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.
Other states to watch: Rhode Island and Minnesota are also potential targets for future legislation that could trigger the use of discriminatory QALYs.
The Washington State legislature has introduced SB 5532, a bill establishing a prescription drug affordability board. It does not prohibit the use of metrics such as quality-adjusted life years (QALYs) by the Board as part of its process for conducting an affordability review or in the process for determining which drugs will be made subject to an affordability review, with potential implications for a discriminatory impact in the treatments chosen for payment limits. As described by the National Association of State Health Policy, this legislation is modeled on Canada, which uses QALYs to determine coverage and reimbursement of health care treatments and services. The legislation does bar QALYs as part of the methodology for calculating the “upper payment limit” for drugs selected based on the affordability review. The bill was heard in the Senate Committee on Health & Long Term Care. Click here to view the bill.
Recently, the Portland Business Journal published an article on how patient advocates are pushing to overhaul Oregon Medicaid's drug list to ensure people with disabilities are not denied care. The article stated, "The U.S. Department of Health and Human Services in 1992 denied Oregon’s use of the “quality-adjusted life year” metric as discriminatory.” The article also highlighted that, "Disability rights advocates are pushing Oregon’s Medicaid program to change how it decides which treatments to cover in order to ensure that people with disabilities are not discriminated against and denied services. Since then, Disability Rights Oregon (DRO) sent a letter signed by 55 additional organizations full compliance with ESPDT requirements, a prohibition on the use of discriminatory QALY measures, and non-discrimination in suicide prevention services. Seven national disability rights organizations sent a letter making the same recommendations. Oregon’s Medicaid waiver application, to be submitted to the Centers for Medicare and Medicaid Services in February, includes changes to advance health equity, while maintaining the coordinated care model Oregon created nearly a decade ago. Click here to read the full article. Click here to view the DRO letter and click here to view the letter from national disability rights organizations. Click here to view the letter from the Epilepsy Foundation. Click here to view the timeline for providing input.
The Massachusetts House and Senate have each taken the positive step of introducing An Act Advancing Health Care Research and Decision-Making Centered on Patients and People with Disabilities, H.201 and S.753. This bill would enshrine essential patient protections including a ban on the use of the QALY, a requirement for research to meet patient-centeredness criteria, and robust engagement of the patient and disability communities in health care decision making. Advocates are also advancing efforts to include an unambiguous QALY ban in S. 2651, which is scheduled for a floor vote on February 10.
Maine is currently considering LD 1636, which would import QALYs from Canada. The bill directly references the prices paid for drugs in five Canadian provinces. Before applying for coverage by the provinces, all drugs must complete a Common Drug Review by CADTH, which uses QALYs. In Canada, many individuals living with disabilities are unable to receive the treatments and care they need. The National Council on Disability (NCD) warned in its 2019 report that similar coverage denials and loss of access to care could also be the outcome if the United States if we reference other countries. The NCD also highlighted in its report that Section 504 of the Rehab Act and Section 1557 of the ACA also apply to Medicaid programs because they receive federal financial assistance. Click here to view the bill. It will be heard on February 15 at 10am EST in the Committee on Health Coverage, Insurance and Financial Service.
The Maryland legislature passed legislation earlier this year creating a Prescription Drug Affordability Board. The legislation did not include protections for patients and people with disabilities such as barring the use of discriminatory QALYs. The Board has begun meeting to hear from stakeholders, including hearing a presentation from the Institute for Clinical and Economic Review which relies on QALYs to assess treatment value. Legislation was introduced this year to bar the use of QALYs and implement patient protections that has not advanced in the legislature. On August 3, organizations representing patients and people with disabilities sent a letter urging the Board to avoid policies that would potentially discriminate by relying on discriminatory metrics such as QALYs. Click here to view legislation introduced in Maryland to bar the use of QALYs and implement patient protections - it has not yet advanced in the legislature.
16. ICER's QALY-Based Study Topics: Alzheimer's Disease, Beta Thalassemia, Chemotherapy-Induced Neutropenia, COVID-19, Type 2 Diabetes, Hypertrophic Cardiomyopathy, Asthma, Unsupported Price Increase, Fair Access: Coverage Policies
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.
- Alzheimer's Disease: Research Protocol available. Draft Scoping Document available. Public Comments closed. 3/23/2022: Model Analysis Plan.
- Beta Thalassemia: Research Protocol available. Revised Scoping Document available. 2/28/2022: Model Analysis Plan. 4/13/2022: Draft Evidence Report.
- Chemotherapy-Induced Neutropenia: Draft Evidence Report available. Model Analysis Plan available. Draft Scoping Document and Research Protocol available.
- COVID-19: Draft Evidence Report Available. Model Analysis Plan available. Draft Scoping Document and Research Protocol available.
- Type 2 Diabetes: Evidence Presentation available. Evidence Report available. Draft Scoping Document and Research Protocol available. 2/15/2022: Final Evidence Report and Meeting Summary.
- Hypertrophic Cardiomyopathy: Final Evidence Report and Meeting Summary available. Evidence Report Available. Evidence Presentation available.
- Unsupported Price Increase: Final Report available.
- Asthma: Final Evidence Report and Meeting Summary available. Evidence Presentation available. Evidence Report available.
- Fair Access: Coverage Policies in 2020: Protocol Available. Final Report available.
17. Upcoming Events and Webinars
Board of Governors Meeting
February 15, 2022
Click here for details.
PCORI Advisory Panels -- Applicant Town Hall
February 16, 2022
Click here for details.
PCORI Virtual Briefing on Suicide Prevention in Youth and Young Adults
February 23, 2022
Click here for details.
18. Medical Journal Articles
“It’s Time to Represent”: Shifting the Paradigm to Improve the Quality of Inputs into Value Assessment Frameworks, click here to view.
Evaluation of Financial Outcomes Under a Value-Based Payment Program for Community Pharmacies, click here to view.
Limited Role of Patient Input in Specialty Drug Coverage Policies, click here to view.
The Importance of Collaboration in Pursuit of Patient-Centered Value Assessment, click here to view.
Toward Better Data Dashboards for US Drug Value Assessments, click here to view.
Prevalence of Avoidable and Bias-Inflicting Methodological Pitfalls in Real-World Studies of Medication Safety and Effectiveness, click here to view.
Association of Co-Pay Elimination with Medication Adherence and Total Cost, click here to view.
The Utility of Patient Engagement in Drug Research and Development, click here to view.
Patient-Powered Research Networks of the Autoimmune Research Collaborative: Rationale, Capacity, and Future Directions, click here to view.
Enhancing Patient Research Partner Engagement: Research in Psoriatic Arthritis, click here to view.
Patient Voices in Value-Based Cancer Care: Priorities for the Biden Administration, click here to view.
The Dollar or Disease Burden: Caps on Healthcare Spending May Save Money, but at What “Cost” to Patients? click here to view.
19. AHRQ Effective Program Updates
Systematic Review: Prehabilitation and Rehabilitation for Major Joint Replacement. Click here to view.
Integrated and Comprehensive Pain Management Programs: Effectiveness and Harms. Click here to view.
Systematic Review: Management of High-Need, High-Cost Patients: A “Best Fit” Framework Synthesis, Realist Review, and Systematic Review. Click here to view.
Research Protocol: Partial Breast Irradiation for Breast Cancer. Click here to view.
Systematic Review: Physical Activity and the Health of Wheelchair Users: A Systematic Review in Multiple Sclerosis, Cerebral Palsy, and Spinal Cord Injury. Click here to view.
Research Report: Developing and Piloting a Tool to Create Dot Plots to Summarize Pooled Data for Multiple Outcomes in Systematic Reviews. Click here to view.
Systematic Review: Malnutrition in Hospitalized Adults. Click here to view.
Research Protocol: Nutrition as Prevention for Improved Cancer Outcomes. Click here to view.
Research Protocol: Telehealth During COVID-19. Click here to view.
Research Protocol: Pharmacologic and Nonpharmacologic Treatments for Posttraumatic Stress Disorder: An Update of the PTSD Repository Evidence Base. Click here to view.