1. Drug Pricing Policy Must Prioritize Patients, click here to read the op-ed.
2. IfPA Blog: When Economists Decide Heart Patients' Fate, click here to read the blog post.
3. NCD Calls on HHS to Strengthen Rights and Protections for People with Disabilities in Letter, click here to read the letter.
4. PIPC Joins 30 Leading Disability and Civil Rights Stakeholders in Letter Asking HHS to Step Up Nondiscrimination Efforts, click here to read the letter.
5. Emerging Threats in States for Use of Discriminatory Metrics, see details below.
6. PCORI Virtual Briefing on COVID-19 & Telehealth, click here to learn more and register.
7. 2021 AUCD Virtual Gala, click here to learn more and register.
8. EveryLife Foundation for Rare Diseases Rare on the Road Leadership Tour, see details below.
9. Vaccination Equity and Communication, see details below.
10. PCORI Seeks Nominations for Advisory Panels, click here to learn more, submit a nomination, or apply to be on an advisory panel.
11. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
12. ICER's QALY-Based Study Topics: Atopic Dermatitis, Treatments, High Cholesterol, Anemia in Chronic Kidney Disease, Lupus Nephritis, Multiple Myeloma, Alzheimer's Disease, click here to provide patient input.
13. Upcoming Events and Webinars, see details below.
14. Medical Journal Articles, see details below.
15. AHRQ Effective Program Updates, see details below.
Jenn McNary, a dedicated patient advocate who has three children living with rare diseases, wrote that drug pricing policy must center the patient and not rely on simplistic formulas. "The QALY is a concerning metric, as it places a lower value on treatments that extend the lives of people with disabilities and chronic illnesses, like my sons," she wrote. "Other countries, like Canada and the United Kingdom, rely on this type of assessment, and in these countries, rare disease patients generally do not receive access to novel therapeutics." Click here to read the full op-ed.
2. IfPA Blog: When Economists Decide Heart Patients' Fate
Cardiologist Seth Baum writes in a blog for the Institute for Patient Access (IfPA) that ICER's recent evaluation of novel high cholesterol treatments is flawed and could result in patients not having access to treatments that save lives. He noted that the study (1) relies too heavily on clinical trial data; (2) ignores varying patient circumstances; (3) disregards the needs of high-risk patients; and (4) lacked input from health experts. "Each shortcoming increases the likelihood that ICER will come down with a one-size-fits-all recommendation for a high-risk patient population. Such economics-based decisions have the potential to keep patients from accessing the treatment that is best for them." he wrote. Click here to read the blog post.
3. NCD Calls on HHS to Strengthen Rights and Protections for People with Disabilities in Letter
The National Council on Disability (NCD), an independent federal agency charged with providing advice and recommendations regarding disability policy to the President, Congress, and other federal agencies, wrote to HHS calling for it to move ahead with a Request for Information (RFI) on “Discrimination on the Basis of Disability in Critical Health and Human Service Programs or Activities."
"A strong evidence base, shared principles, and strong coalitions make for effective policy, and it is clear that there is such coalescence surrounding this RFI," NCD Chairman Andrés J. Gallegos wrote. "We believe strengthening Section 504 would align with both HHS’s mission, as well as the focus of NCD’s bipartisan membership during my tenure as Chairman: to provide equality of access and care for all individuals with disabilities."
The letter also referenced a letter from 30 leading disability and civil rights stakeholders, including PIPC, also calling for the RFI to be issued. Read more about that letter below.
Click here to read the NCD letter.
4. PIPC Joins 30 Leading Disability and Civil Rights Stakeholders in Letter Asking HHS to Step Up Nondiscrimination Efforts
The Partnership to Improve Patient Care (PIPC) proudly joined 30 leading disability rights and racial justice organizations in a letter to the Department of Health and Human Services' Office of Civil Rights that calls for additional regulatory action aimed at preventing discrimination in health care. In particular, the letter calls on HHS to formally issue its Request for Information (RFI) on “Discrimination on the Basis of Disability in Critical Health and Human Service Programs or Activities."
The RFI addresses disability non-discrimination in a number of key health care areas. This includes life-sustaining care, organ transplants, Crisis Standards of Care, suicide prevention programs and services, the child welfare system, health care value assessment methodologies, and auxiliary aids and accessible medical equipment in the health care system.
“…As recent reports from the National Council on Disability show, people with disabilities still face significant disparities and obstacles to access in healthcare,” the letter states.” “The issues addressed in this RFI look to root out the common thread they all share: too many medical professionals see life with a disability as less worth living and less worthy of care, sometimes so much less so that they view death as the correct course.” Click here to read the letter.
5. 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 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.
North Dakota is currently considering a piece of legislation, SB 2170, 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. 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. 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 S.B. 734. Click here to view the NCD report. Click here to view the NCD's recent letter to CMS against importing QALYs. SB 2170 narrowly passed by vote in the Senate on February, 19, 2021. It has not yet been heard in the House.
Last year, advocates applauded the State of Oklahoma for being the first state to pass legislation explicitly barring the use of quality-adjusted life years (QALYs) or any other metric that would devalue lives lived with a disability, whether from within its agency or a third party. Oklahoma's Health and Human Services Committee passed separate legislation that would import QALYs from Canada, where the Patented Medicine Prices Review Board relies on a cost-utility analysis model in which health outcomes are expressed as QALYs. 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 in 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 S.B. 734. Click here to view the NCD report. Click here to view the NCD's recent letter to CMS against importing QALYs. The bill was passed by the Appropriations committee and will next be heard on the Senate floor. Reach out to Senators to express concern that S.B. 734 would import QALYS from Canada and violate the QALY ban in the Nondiscrimination in Health Care Coverage Act passed in 2020.
The legislature in the State of New Mexico has introduced HOUSE BILL 154 which would create a prescription drug affordability board. The House State Government, Elections, and Indian Affairs Committee voted to table the bill, so it will not come up again this session. The proposed law would authorize the board to review the cost of prescription drugs and vote on whether to impose an upper payment limit on purchases and payer reimbursements of prescription drug products in the state, similar to the State of New York. Similar to New York, the bill explicitly authorizes the board to enter into contracts with qualified, independent third parties (such as the Institute for clinical and Economic Review) for services necessary to carry out the powers and duties of the board. New York explicitly references ICER which is known to refer to the quality-adjusted life year (QALY) as the “gold standard” for value assessment. To mitigate states from this kind of discrimination, organizations supporting Value Our Health developed a template for state 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. This is now an opportune time to educate lawmakers in New Mexico on the QALY and its discriminatory implications.
Maine has introduced a bill, S.P. 262, which would rely 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 and will be watching developments in Maine closely.
In the past, we have reported that Pennsylvania is considering the establishment of a Health Policy Committee, modeled after Massachusetts. The Pennsylvania State Treasurer is proposing to create a Drug Affordability Review Board and to institute “value-based purchasing” of pharmaceuticals. Interestingly, the Treasurer specifically references VBP arrangements in Oklahoma but does not mention that the Oklahoma state legislature subsequently barred the use of QALYs. We will follow these developments closely to see if Pennsylvania similarly seeks to bar QALYs as part of their considerations.
6. PCORI Virtual Briefing on COVID-19 & Telehealth
Join PCORI for important funding updates on telehealth and COVID-19 as well as key takeaways and themes from PCORI’s telehealth portfolio that can inform ongoing conversations on the current and post-COVID landscape of telehealth policy. Hear about the lessons, opportunities, and challenges of the application of telehealth during the pandemic from the provider perspective, along with telehealth policy insights from a state health expert that may be useful to policy makers and Congress. This event is being held virtually on Tuesday, March 23 – 2:30 pm to 3:30 pm Eastern. Click here to learn more and register.
7. 2021 AUCD Virtual Gala
Join AUCD on April 14, 2021 for its AUCD for All Virtual Gala. This year’s Gala is free for everyone to attend and in a virtual, accessible format. This year’s theme is Celebrating 50 Years of Leadership: Looking Back, Moving Forward in recognition of AUCD’s 50th Anniversary. During the Gala, AUCD will reflect on its past accomplishments while amplifying the voices of current and future leaders in the disability community. Click here to learn more and register.
8. EveryLife Foundation for Rare Diseases Rare on the Road Leadership Tour
The Rare on the Road Leadership Tour mobilizes grassroots advocates by bringing the combined expertise of the EveryLife Foundation for Rare Diseases and Global Genes to areas not tapped by other rare disease events, as we work to identify and activate new patients and to ensure the patient voice is larger and louder than ever before. This year, due to the circumstances surrounding COVID-19, the 2021 RARE on the Road Leadership Tour is going virtual with an updated format, which will consist of one interactive webinar and three virtual, state-specific meetings geared toward uniting and activating the rare disease community at the local level. To make this year’s RARE on the Road Leadership Tour as accessible as possible, English language closed caption, in addition to real-time Spanish translation, will be made available in all virtual spaces. The webinar will take place on Tuesday, March 23, 2021 at 11:00 am Eastern. Register here.
9. 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?
10. 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, 2020. Click here to learn more, submit a nomination, or apply to be on an advisory panel.
11. 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: Lifesaving cystic fibrosis drug stuck in approval process limbo.
- Canada: Cystic fibrosis patients continue to wait for access to "game-changing" drug.
- New Zealand: New Zealanders go into debt to access drugs that other countries fund. Bowel cancer patient "desperate" to raise funds for cancer drug that government refuses to fund. Pharmac will not pay for cystic fibrosis drug for patient who will die without it.
- United Kingdom: Family of child with spinal muscular atrophy looking for options abroad as NHS will not fund lifesaving treatment. Thousands of men with prostate cancer will be denied life-extending drug after health experts ruled it was not cost effective.
12. ICER's QALY-Based Study Topics: Atopic Dermatitis, High Cholesterol, Anemia in Chronic Kidney Disease, 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.
- Atopic Dermatitis: Research Protocol available. 3/22/2021: Model Analysis Plan.
- High Cholesterol: Final Evidence Report available.
- Anemia in Chronic Kidney Disease: Final Evidence Report available.
- Lupus Nephritis: Draft Evidence Report available. 3/12/2021: Evidence Report. Meeting 3/26/2021: New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for lupus nephritis.
- Multiple Myeloma: Draft Evidence Report AVAILABLE, Comment Period OPEN through 3/11/2021. Meeting 4/16/2021.
- Alzheimer's Disease: Model Analysis Plan available. 5/5/2021: Draft Evidence Report.
13. Upcoming Events and Webinars
Engagement Awards 2021 Special Cycle -- Applicant Office Hours
March 8, 2021
Click here to view.
National Health Council Webinar: Case Examples of Patient-Centered Real-World Evidence
March 12, 2021
Click here to view.
PCORI Workshop on Methodologic Challenges in Intellectual and Developmental Disabilities Research
March 15, 2021
Click here to view.
Webinar: Priorities on the Health Horizon: Informing PCORI's Strategic Plan
March 15-16, 2021
Click here to view.
PCORI Board of Governors Meeting
March 16, 2021
Click here to view.
14. Medical Journal Articles
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.
Six Ways to Foster Community-Engaged Research During Times of Societal Crises, click here to view.
Improving Comparative Effectiveness Research of Complex Health Interventions: Standards from the Patient-Centered Outcomes Research Institute (PCORI), click here to view.
15. AHRQ Effective Program Updates
OPEN FOR COMMENT THROUGH MARCH 26, 2021: Management of High-Need, High-Cost Patients: A "Best Fit" Framework Synthesis, Realist Review, and Systematic Review. Click here to view.
Technical Brief: Disparities and Barriers to Pediatric Cancer Survivorship Care. Click here to view.
Technical Brief: Automated-Entry Patient-Generated Health Data for Chronic Conditions: The Evidence on Health Outcomes. Click here to view.
REQUEST FOR INFORMATION OPEN UNTIL APRIL 16, 2021: Use of Clinical Algorithms That Have the Potential To Introduce Racial/Ethnic Bias Into Healthcare Delivery. Click here to view.
Systematic Review: Integrating Palliative Care in Ambulatory Care of Noncancer Serious Chronic Illness. Click here to view.
Research Protocol: Management of Infantile Epilepsy. Click here to view.
Systematic Review: Living Systematic Review on Cannabis and Other Plant-Based Treatments for Chronic Pain. Click here to view.
Research Protocol: Improving Rural Health Through Telehealth-Guided Provider-to-Provider Communication. Click here to view.
Systematic Review: Treatments for Acute Pain. Click here to view.
Systematic Review: Acute Treatments for Episodic Migraine. Click here to view.