1. PIPC Chairman, Tony Coelho, reiterates PIPC’s Commitment to Nondiscrimination, see details below.
2. Patient Advocates: International Reference Pricing Would Decimate Alzheimer's Disease Research, see details below.
3. Over 85 Disability and Patient Groups Sign Open Letter: Reject Health Policies that Discriminate, see details below.
4. People With Disabilities Could Soon Face Health Care Discrimination, click here to read the article.
5. IHP: QALYs in the News, see details below.
6. IHP: Advocates Sue To Overturn Trump CMS Approval Of TN Funding Demo, click here to view the article (subscription only).
7. NCD Writes to CBO on Reliance on the QALY for Budget Scores, click here to read the letter.
8. States Rely on Template Legislation that References ICER and Discriminatory Metrics, see details below.
9. IVI Value Model on Major Depressive Disorder, see details below.
10. WEDNESDAY: AAPD Leadership Awards Gala, click here to register.
11. Emerging Threats in States like CO and ME for Use of Discriminatory Metrics, see details below.
12. Vaccination Equity and Communication, see details below.
13. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
14. ICER's QALY-Based Study Topics: Hypertrophic Cardiomyopathy, Myasthenia Gravis, Atopic Dermatitis, Lupus Nephritis, Multiple Myeloma, Alzheimer's Disease, click here to provide patient input.
15. Upcoming Events and Webinars, see details below.
16. Medical Journal Articles, see details below.
17. AHRQ Effective Program Updates, see details below.
PIPC Chairman Tony Coelho issued a statement expressing disappointment that the House reintroduced legislation - modeled on what was known as H.R. 3 when it was introduced in 2019 - that would rely on quality-adjusted life years (QALYs) to value health care, which are well known to discriminate against people with disabling and chronic conditions, especially in communities of color that are not well represented in the research literature. Chairman Coelho stated that the patient and disability community is united in amplifying the work of the National Council on Disability, an independent federal agency that has warned policymakers on the implications of referencing QALYs directly and by reference to third parties and foreign countries, noting that the patient and disability community stand ready to work with Congress on solutions that address the cost of care that do not discriminate.
2. Patient Advocates: International Reference Pricing Would Decimate Alzheimer's Disease Research
The Alliance for Aging Research, PIPC, and other patient advocates sent a letter to Congressional leaders urging them to abandon proposed policies that would import discriminatory QALY-based pricing from other countries. "Use of QALY-based cost-effectiveness analysis is a significant issue for people with Alzheimer's disease since the majority of those with dementia are the oldest old," they write. "Of the estimated 6 million people with Alzheimer's disease who are age 65 and older, 80 percent are 75 years or older, and more than a third are 85 years or older....When applied to health care decision-making by insurance companies, this can mean that treatments for these more vulnerable people are deemed 'too expensive' and therefore 'not cost-effective' to cover." Click here to read the letter. Click here to read more about the impact of international reference pricing on Alzheimer's research.
3. Over 85 Disability and Patient Groups Sign Open Letter: Reject Health Policies that Discriminate
On April 14, 2021, over 85 groups representing people with disabilities, chronic conditions and older adults stated to policymakers, "Every life is valuable. We strongly urge policymakers to reject policies that discriminate against people with disabilities and chronic conditions, older adults, and reinforce health inequities.” In releasing the Open Letter, Maria Town, President and CEO of the American Association of People with Disabilities, expressed disappointment that the disability community still has to fight policies that discriminate by referencing discriminatory metrics to value health care, whether directly or indirectly from foreign governments. Other disability groups including Autistic Self Advocacy Network, the Disability Rights Education & Defense Fund, National Council on Independent Living, and Not Dead Yet expressed similar concerns about the use of discriminatory metrics to value health care, and particularly the quality-adjusted life year (QALY) which devalues lives lived with a disability. Organizations representing patients and people with chronic conditions also joined the Open Letter, with accompanying statements from Genetic Alliance, CancerCare, Alliance for Aging Research and the Cystic Fibrosis Research Institute expressing deep concerns with short-sighted drug pricing policies that rely on QALYs and highlighting their united front opposing their use by policymakers.
Click here to view the Open Letter. Click here to view the statements from disability groups and here to view the statements from patient groups.
4. People With Disabilities Could Soon Face Health Care Discrimination
Angela Williams, CEO of EasterSeals, wrote about growing interest in using QALYs to evaluate new treatments, including breakthroughs for people with disabilities. In particular, she highlighted ICER's use of the metric to evaluate treatments for complex conditions. "ICER’s evaluations impose brutally simplistic categories on complex diseases," she writes. "For instance, the institute splits patients with spinal muscular atrophy that have received treatment into three rudimentary categories: mobile, requiring ventilation and dead....Worryingly, the institute has become very effective at getting large health insurers to utilize its metrics in their coverage decisions. And now New York health officials have begun using ICER metrics in its state-run Medicaid program, specifically to clamp down on access to advanced cystic fibrosis treatments." Click here to read the article.
5. IHP: QALYs in the News
Inside Health Policy reported that the controversial metric called quality-adjusted life years (QALYs) are the subject of ongoing health policy debates in Congress and the administration. In one article, the Institute for Clinical and Economic Review (ICER) was referenced as an entity that "seeks to limit patient access to expensive new treatments and reduce drug costs for payers” thereby putting payers over patients. Another article reported on the QALY Open Letter from almost 90 organizations "calling on the Biden administration to reject QALYs and international reference pricing -- they say other countries use QALYs so tying Medicare prices to those abroad indirectly relies on QALYs.” Click here and here to view the articles (subscription only).
6. IHP: Advocates Sue To Overturn Trump CMS Approval Of TN Funding Demo
Inside Health Policy reported last week that the National Health Law Program and Tennessee Justice Center sued to overturn Tennessee’s capped Medicaid funding demonstration without the required public comment period. The article noted, "The groups argue the demonstration will hurt Tennessee Medicaid beneficiaries. 'Tennessee, and its contracting managed care plans, have repeatedly failed to provide the thirteen Plaintiff Medicaid beneficiaries with access to medically necessary services. By continuing the managed care program, introducing the aggregate cap and shared savings financing scheme, and permitting the State to impose additional limits on prescription drug coverage, the TennCare III approval will only intensify the harm that these beneficiaries have suffered,' the suit says.” As additional background, in January 2020, twenty groups including the Partnership to Improve Patient Care submitted a comment letter to the Centers for Medicare & Medicaid Services (CMS) expressing concern about the demonstration, stating that adopting a closed formulary opens the door to evaluating the “value” of a drug using cost-effectiveness analyses based on the discriminatory quality-adjusted life year (QALY), which would lead to limited access to needed treatments for patients and people with disabilities. The article stated, "NHeLP and others ask the court to vacate the approval and keep the demonstration from being implemented.” Click here to view the letter from advocates. Click here to view the IHP article (subscription only).
7. NCD Writes to CBO on Reliance on the QALY for Budget Scores
The National Council on Disability (NCD) sent a letter to the Congressional Budget Office (CBO) expressing its concern with the use of QALYs to score recent drug pricing legislation. "We are troubled that although CBO recognized that concerns have been raised about the discriminatory design and impact of the QALY, it described the discrimination as 'potential,' and opted to utilize it because it was 'the best available to CBO and is consistent with the approach taken by many countries to negotiate drug prices,'" NCD wrote. "This makes light of the fact that the QALY assigns a lower value to the lives of people with disabilities and chronic illnesses. It also ignores the fact that countries that rely on the QALY to set drug prices have restricted or denied patients with disabilities access to effective drugs used to treat chronic conditions and to breakthrough medications." Click here to read the letter.
8. States Rely on Template Legislation that References ICER and Discriminatory Metrics
We are seeing many states reference pieces of template legislation put forward by the National Association of Health Policy (NASHP). Though the intent of these bills is to lower healthcare costs, several implicate the discriminatory Quality-Adjusted Life Year (QALY) and others rely on the Institute for Clinical and Economic Review (ICER) as the sole source for evidence. The three primary bills are:
- Canadian Reference Pricing -- This piece of template legislation 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. The result of this is that in Canada is that 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.
- Unsupported Price Increase -- This piece of template legislation relies on one report by ICER to determine whether a price increase on certain therapies were supported by additional evidence. This is concerning as it codifies reference to an independent entity lacking oversight and accountability as a sole source of information. ICER is the sole arbiter as to what evidence it uses to develop this report, and patients have consistently expressed concern with ICER’s lack of transparency and omission of real-world evidence. ICER also has the ability to change the inputs and methodology of the report without oversight. Though the 2020 report does not use the QALY, it is possible that future reports will.
- Drug Pricing Review Board -- This piece of template legislation establishes a drug review board or commission. The goal of the board is to allow the state to review and evaluate the reimbursement rate and/or coverage for pharmaceuticals. Most have fairly broad parameters of how they can assess a drug’s value, and specifically permit the state to rely on third-party research or contract directly with a third-party for the purpose of fulfilling its duties. As has happened in other states such as New York and Massachusetts, without patient protections, these bills allow the new state board or commission to reference value assessments that rely on QALY and similar metrics. It is imperative that these boards has patient and disability representation and that a QALY ban is included in the legislation.
9. IVI Value Model on Major Depressive Disorder
The Innovation and Value Initiative (IVI) is holding a public comment period on its draft model scope for its major depressive disorder value model through May 14, 2021. IVI is seeking feedback on factors most important to include, missing data elements, and recommendations for specific use cases in the model. Please send comments and questions to firstname.lastname@example.org or respond to the survey here. IVI will hold an informational webinar on Monday, May 3, from 1:00-2:00 PM to provide more detailed information about the MDD Model Scope. Registration information is available here.
10. WEDNESDAY: AAPD Leadership Awards Gala
The American Association of People with Disabilities (AAPD) will hold its annual Leadership Awards Gala on Wednesday, April 28 at 7:00 p.m. Eastern. This event is free and open to all. AAPD will recognize two outstanding emerging leaders with disabilities, Elijah Armstrong and Noor Pervez, with the 2021 AAPD Paul G. Hearne Emerging Leader Award. The Gala will feature special guest appearances by President Joseph R. Biden, Jr., Academy Award nominated and Golden Globe winning actor, Taraji P. Henson, Majority Whip James E. Clyburn, Valerie Jarrett, President of The Barack Obama Foundation, and Andrés Gallegos, Chairman of the National Council on Disability. AAPD will also honor several advocates and allies, including Stacey Abrams, founder of Fair Fight, Jane Pauley, the host of CBS Sunday Morning, Congresswoman Ayanna Pressley, Congressman Don Young, Thomas P. DiNapoli, the New York State Comptroller, as well as leading organizations AT&T and The New York Times. Click here to register.
11. Emerging Threats in States like CO and ME 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 or are considering policies to import QALY-based decisions from other countries. Yet, federal policymakers have emphasized that the use of discriminatory metrics is subject to civil rights laws such as the Americans with Disabilities Act. 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 Colorado state legislature has introduced SB 21-175 creating a prescription drug affordability review board. In some states these types of boards and commissions have led to referencing value assessments based on QALYs from the Institute for Clinical and Economic Review (ICER). In Massachusetts, the Health Policy Commission went so far as to contract with ICER. An amendment to SB 21-75 was adopted that prohibits the use of QALYs in the section of the bill that determines an upper payment limit. A similar amendment in the sections establishing the commission’s authority to perform value assessments for treatments would prevent QALYs from being used to target treatments for people with disabilities and older adults. The Value Our Health legislative template provides a path for states to protect patients and people with disabilities by also creating standards for the evidence that drives decision-making and assuring engagement of patient and disability stakeholders, in addition to barring QALYs.
Maine has introduced a bill, S.P. 262, which would rely solely on the Institute for Clinical and Economic Review, ICER, to determine whether a price increases on certain therapies were supported by additional evidence. We remain concerned about states referencing ICER, particularly as a sole source of information, and will be watching developments in Maine closely.
12. Vaccination Equity and Communication
There is critical ongoing work to assure equity in the prioritization of vaccinations, as well as communication to address reluctance and ensure access to information and systems. PIPC Steering member the Association of University Centers on Disabilities (AUCD) aggregated the following resources that we wanted to share:
- Elevated COVID-19 Mortality Risk Among Recipients of Home and Community-Based Services: A Case for Prioritizing Vaccination for This Population
- National Council on Disability letter to Governors Association
- COVID-19 Vaccine Prioritization Dashboard
- Measuring the impact of COVID-19 vaccine misinformation on vaccination intent in the UK and USA
- COVID-19 Vaccination Intent, Perceptions, and Reasons for Not Vaccinating Among Groups Prioritized for Early Vaccination — United States, September and December 2020
- KFF COVID-19 Vaccine Monitor: What Do We Know About Those Who Want to “Wait and See” Before Getting a COVID-19 Vaccine?
13. 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: New drugs exist to help migraine sufferers but Australia's health system will not fund them.
- New Zealand: Woman loses job after Pharmac switches her epilepsy drug without her knowledge to save money, leading to serious health problems. Advocates call for district health boards to be able to administer unfunded cancer drugs.
- United Kingdom: While cystic fibrosis patients in Scotland, Wales, or Northern Ireland receive a lifesaving new cystic fibrosis drug, patients in England are kept waiting.
14. ICER's QALY-Based Study Topics: Hypertrophic Myocardiopathy, Myasthenia Gravis, Atopic Dermatitis, Lupus Nephritis, Multiple Myeloma, Alzheimer's Disease
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.
- Hypertrophic Cardiomyopathy: Draft Scoping Document available. Public Comments open through 4/29/2021. Meeting 8/22/2021: CTAF will deliberate and vote on evidence presented in ICER’s report on therapies for obstructive hypertrophic cardiomyopathy.
- Myasthenia Gravis: Research Protocol available. 6/7/2021: Model Analysis Plan. Meeting 9/24/2021: New England CEPAC will deliberate and vote on evidence presented in ICER’s report on therapies for myasthenia gravis.
- Atopic Dermatitis: Model Analysis Plan available. 5/14/2021: Draft Evidence Report. Meeting 7/23/2021: An assessment of treatments for atopic dermatitis by the New England CEPAC.
- Lupus Nephritis: Final Evidence Report AVAILABLE.
- Multiple Myeloma: Meeting 4/16/2021: An assessment of idecabtagene vicleucel, ciltacabtagene autoleucel, and belantamab mafodotin for the treatment of multiple myeloma. 5/11/2021: Final Evidence Report
- Alzheimer's Disease: Model Analysis Plan available. 5/5/2021: Draft Evidence Report.
- Hereditary Angioedema: 7/27/2021: Final Updated Assessment with RWE Update.
- Unsupported Price Increase: Research Protocol available, public input OPEN through 5/10/2021.
15. Upcoming Events and Webinars
Webinar: Understanding the Costs and True Benefits of SDOH Investments
May 4, 2021
Click here to view.
Financial Times US Pharma and Biotech Summit
May 5-6, 2021
Click here to view.
PCORI Advisory Panel on Healthcare Delivery and Disparities Research Spring 2021 Meeting
May 6, 2021
Click here to view.
Building Data Capacity for Patient Centered Outcomes Research: A Comprehensive Ecosystem for PCOR - Workshop 3
June 14, 2021
Click here to view.
PCORI Advisory Panel on Patient Engagement Summer 2021 Meeting
July 15-16, 2021
Click here to view.
16. Medical Journal Articles
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.
Embedding Clinical Trials Within Routine Health-Care Delivery: Challenges and Opportunities, click here to view.
Approval and Coverage of Cancer Drugs in England, Canada, and the US, click here to view.
Experiences of an HCV Patient Engagement Group: A Seven-Year Journey, click here to view.
Choosing Important Health Outcomes for Comparative Effectiveness Research: 6th Annual Update to a Systematic Review of Core Outcome Sets for Research, click here to view.
Health Technology Assessment With Diminishing Returns to Health: The Generalized Risk-Adjusted Cost-Effectiveness (GRACE) Approach, click here to view.
Comparative Effectiveness Research Needs to Consider Optimal Dosing and Scheduling, click here to view.
Are Patients More Adherent to Newer Drugs?, click here to view.
Improving the Quality of Person-Centred Healthcare from the Patient Perspective: Development of Person-Centred Quality Indicators, click here to view.
17. AHRQ Effective Program Updates
Maternal, Fetal, and Child Outcomes of Mental Health Treatments in Women: A Systematic Review of Perinatal Pharmacologic Interventions. Click here to view.
OPEN FOR COMMENT THROUGH MAY 13, 2021: Interventional Treatments for Acute and Chronic Pain. Click here to view.
OPEN FOR COMMENT THROUGH MAY 14, 2021: Key Questions: Accelerated Partial Breast Irradiation for Breast Cancer. Click here to view.
OPEN FOR COMMENT THROUGH APRIL 29, 2021: Research Report: Outcome Measure Harmonization and Data Infrastructure for Patient-Centered Outcomes Research in Depression. Click here to view.
Rapid Evidence Product: Improving Pain Management in the Context of America's Opioid and Substance Use Disorders Crisis. Click here to view.
Systematic Review: Cervical Ripening in the Outpatient Setting. Click here to view.
Surveillance Report: Living Systematic Review on Cannabis and Other Plant-Based Treatments for Chronic Pain. Click here to view.
Rapid Evidence Review: Antibody Response Following SARS-CoV-2 Infection and Implications for Immunity. Click here to view.
White Paper: Standardized Library of Lumbar Spondylolisthesis Outcome Measures. Click here to view.