— Senate HELP Committee to Discuss Importing Value Assessments from Other Countries. See details below.
— Impact of Healthcare Algorithms on Racial and Ethnic Disparities in Health and Healthcare. Click here to view the AHRQ systematic review.
— PIPC Submits Comments on ICER Assessment of PNH Treatments. Click here to read the letter.
— PIPC Comments on Adding a Patient Representative on P&T Committees. Click here to read the letter.
— Due TODAY! Patient-Centered Economic Outcomes Landscape (2023-2024): Request for Public Input. See details below.
— Cassidy Requests Information on Improving Access to Gene Therapies. See details below.
— Ban Quality-Adjusted Life-Years in Oregon. Click here to sign the petition.
— 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.
1. Senate HELP Committee to Discuss Importing Value Assessments from Other Countries. On January 25, 2024, the Senate Health Education Labor and Pensions (HELP) Committee intends to hold a hearing about importing foreign prices for prescription drugs, thereby importing their methods for value assessment and potentially coverage. The National Council on Disability (NCD) has consistently recommended against the policy due to its unintended effect of importing the use of discriminatory measures of effectiveness into the United States, with the potential of restricting access to care for people with disabilities, serious chronic conditions and older adults as is common in other countries.
Click to view the NCD letter to Congress in 2021 stating, "Drug prices need to be lowered. They should not be permitted to be lowered based on the use of a pricing methodology that has unarguably been proven to be discriminatory in its use against persons with disabilities. Acceptance of foreign drug prices set in reliance on the QALY method effectively endorses the use of this discriminatory pricing methodology.” Click here to view the PIPC issue brief raising issues related to Germany’s process for value assessment that lead to 60% of new medicines receiving negative assessments. Click here to view other PIPC resources.
2. Impact of Healthcare Algorithms on Racial and Ethnic Disparities in Health and Healthcare. On December 8, 2023, the Agency for Healthcare Research and Quality published a systematic review examining "the evidence on whether and how healthcare algorithms (including algorithm-informed decision tools) exacerbate, perpetuate, or reduce racial and ethnic disparities in access to healthcare, quality of care, and health outcomes, and examine strategies that mitigate racial and ethnic bias in the development and use of algorithms.” The review concluded that, "Algorithms have been shown to potentially perpetuate, exacerbate, and sometimes reduce racial and ethnic disparities.”
The report further recommended, "Important future steps include increasing transparency in algorithm development and implementation, increasing diversity of research and leadership teams, engaging diverse patient and community groups in the development to implementation lifecycle, promoting stakeholder awareness (including patients) of potential algorithmic risk, and investing in further research to assess the real-world effect of algorithms on racial and ethnic disparities before widespread implementation.” Click here to view the AHRQ systematic review. PIPC is following the use of artificial intelligence and algorithms closely, supporting transparency of the evidence base for their use to avoid reliance on discriminatory measures and cost effectiveness to deny access to care.
3. PIPC Submits Comments on ICER Assessment of PNH Treatments. In a letter to the Institute for Clinical and Economic Review (ICER), PIPC submitted comments on ICER's assessment of Iptacopan and Danicopan for Paroxysmal Nocturnal Hemoglobinuria (PNH). The letter criticizes ICER's choice of model for underestimating the complexity of PNH and ignoring major aspects of disease burden. PIPC also encourages ICER to rely more heavily on real world evidence, as well as make more of an effort to address patient heterogeneity.
"PIPC urges ICER to consider models that do not rely on quality-adjusted life years or equal value of life year gained measures in its studies," wrote PIPC Chair Tony Coelho. "A model that allows for consideration of the complexity of a disease, the impact of treatment for different subpopulations, and the broader set of costs and savings for patients, medical and non-medical, would allow for a more accurate value assessment."
4. PIPC Comments on Adding a Patient Representative on P&T Committees. On January 8, PIPC submitted a comment letter to the Centers for Medicare and Medicaid Services (CMS) on the Notice of Benefit and Payment Parameters for 2025 related to its proposal to require Pharmacy & Therapeutics (P&T) committees to include a consumer representative. PIPC urged the final requirement to use the terminology “patient representative” as opposed to “consumer representative.” A patient perspective should be required to bring a focus on the patient experience of care to the P&T committee, providing additional insight into the practical use of therapies and effect on quality-of-life outcomes. Additionally, the P&T committee process should be required to engage patients and people with disabilities and the organizations representing them as advisors that have experience with the disease or condition to ensure that outcomes that matter to patients are key considerations in payer decisions.
5. Due TODAY! Patient-Centered Economic Outcomes Landscape (2023-2024): Request for Public Input. PCORI has produced a draft Landscape of Patient-Centered Economic Outcomes that aims to lay out, in one place, appropriate and relevant outcomes, that capture costs and other economic burdens from a patient-centered, but multi-stakeholder, perspective. PCORI is also requesting input from patients, families, caregivers, and all of our stakeholders, the broader healthcare community, and the public. Comments must be submitted here by TODAY, January 16, 2024 at 5 PM ET.
6. Cassidy Requests Information on Improving Access to Gene Therapies. Sen. Bill Cassidy (R-LA), Ranking Member of the Senate HELP Committee, is seeking stakeholder feedback on ways to improve and protect access to gene therapies for patients with rare diseases. Cassidy hopes to use the feedback to inform future legislation to modernize and improve the market structure for gene therapies to ensure that the U.S. commercial health insurance market supports Americans with ultra-rare diseases, while ensuring the continued development of innovative gene therapies. Feedback is due to GeneTherapyCoverage@help.senate.gov by January 22, 2024.
7. Ban Quality-Adjusted Life-Years in Oregon. Disability Rights Oregon is circulating a petition to ban quality adjusted life years (QALYs) by payers in Oregon, a flawed scoring system economists use to determine cost-effectiveness of proposed medical treatments. Payers—including the Oregon Health Plan—use QALY scores to determine who is eligible for healthcare resources. The problem is the scores are calculated in inherently discriminatory ways. DRO states in their petition, "QALY violates both the Rehabilitation Act and the Americans with Disabilities Act. These types of scores have no place influencing what conditions and treatments the Oregon Health Plan will cover.” DRO hopes that the state legislature will act during a short legislative session. Click here to sign the petition.
8. 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.
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. The Oregon PDAB will hold a meeting on January 17, 2023. In response, advocates sent a letter to the Board in advance of their December public meeting, available here. Guidance for providing public testimony is available here.
Click here to join Disability Rights Oregon in advocating for legislation banning use of QALYs.
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, February 16 from 1 -5 pm MT. Click here to register or provide comment. Colorado’s Prescription Drug Affordability Advisory Council (PDAAC) will hold a meeting on January 25, 2024 at 9 am MT. Click here to register. The PDACC has also noticed that it is currently accepting applications for new council members. Click here to apply.
The Massachusetts Senate’s Ways and Means Committees was referred the “PACT Act”, reported favorably on October 30, 2023 by the Joint Committee on Health Care Financing. The legislation includes a provision calling for the health policy commission not to base its determinations on measures such as QALYs. The Senate rejected an amendment (#19) pushed by Massachusetts advocates calling for transparency related to the use of cost effectiveness analyses by the commission, as well as increased requirements for stakeholder engagement. The Senate passed the bill on November 15, 2023. Massachusetts advocates will work with the House toward language that more clearly provides for public transparency of the evidence under consideration and opportunities for engagement from patients and people with disabilities.
On January 4, 2024, the Vermont Senate Health and Welfare Committee raised S. 98, legislation that would direct the Green Mountain Care Board to conduct an affordability review using cost-effectiveness studies. The language limiting the use of quality-adjusted life years (QALYs) is modeled on language used in other states to allow for use of cost effectiveness measures such as the equal value of life year gained (evLYG). As drafted, the language limits the use of QALYs only to identifying subopulations and only focuses on discrimination related to life extension, raising concerns that the state could partner with entities such as PORTAL and ICER that support the use of QALYs and evLYGs to value health care.
ICER acknowledges that it developed the language for use in Washington State that is now being used across states. While the legislative language is touted as restricting QALYs, it is not modeled on the Affordable Care Act’s ban on use of QALYs and similar measures in Medicare, which comprehensively bans discriminatory measures. The Value Our Health template for state legislatures includes a provision banning use of discriminatory measures that is modeled on the ACA language (see section (d)). Advocates that want to weigh in may reach out to the committee assistant Kiki Carasi-Schwartz at email@example.com.
At its December, 2023 meeting, the Washington State Prescription Drug Affordability Board acknowledged the limitation in its statute on using quality adjusted life years (QALYs) to establish an upper payment limit, yet also raised the possibility of contracting with the Institute for Clinical and Economic Review (ICER) as a consultant to the Board for its methodologies and analysis (1:03:25). At its October, 2023 meeting, the Board discussed partnering the Program on Regulation, Therapeutics, and Law (PORTAL), acknowledging its education of the Colorado Board and collaboration with the National Association of State Health Policy (NASHP) in development of the legislation creating Washington’s PDAB and supporting materials, further underscoring the potential influence of entities that view QALYs and evLYG measures as useful to the affordability review process (47:55). PIPC is following the PDAB’s implementation in Washington State closely for potential use of discriminatory measures of effectiveness. The next meeting is on January 31, 2024, though the registration and agenda is not yet posted.
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: Cancer and cystic fibrosis patients were forced to pay six-figure costs for key treatments before subsidies were finally provided.
- New Zealand: A patient multiple myeloma must leave the country to gain access to a broader range of life-extending treatments.
- United Kingdom: Cancer patients are pressing the NHS to cover CAR-T cell therapy treatments for more types of this disease, as it is currently restricted.
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.
- Myelodysplastic Syndrome: Revised Scoping Document available. Public meeting: July 2024.
- Chronic Obstructive Pulmonary Disease: Revised Scoping Document available. Public meeting: June 2024
- Post-Traumatic Stress Disorder: Revised Scoping Document available. Main review: May 30, 2024.
- Paroxysmal Nocturnal Hemoglobinuria: Draft Evidence Report available.
- Schizophrenia: Evidence Report available. Public meeting: February 2024.
- Pulmonary Arterial Hypertension: Final Evidence Report and Meeting Summary available.
Upcoming Events and Webinars
PCORI Board of Governors Meeting
February 13, 2024
Click here to view.
Medical Journal Articles
Using A Patient-Centered Value Assessment to Optimize Fair Prices for Inflation Reduction Act’s Medicare Drug Price Negotiation Program. Click here to read the article.
Alternative approaches to measuring value: an update on innovative methods in the context of the United States Medicare drug price negotiation program. Click here to read the article.
Economic Outcomes in Patient-Centered Outcomes Research: A Paradigm Shift. Click here to read the article.
Visualization of Evidence for Shared Decision Making. Click here to read the article.
Avenues for Strengthening PCORnet’s Capacity to Advance Patient-Centered Economic Outcomes in Patient-Centered Outcomes Research. Click here to read the article.
Linking Medicare-Medicaid Claims for Patient-Centered Outcomes Research Among Dual-Eligible Beneficiaries. Click here to read the article.
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.
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.