1. IfPA Blog: When Economists Decide Heart Patients' Fate, click here to read the blog post.
2. President Biden Appoints First White House Disability Policy Director, click here to read more about the appointment.
3. Drug Pricing Policy Must Prioritize Patients, click here to read the op-ed.
4. NYT: How America’s Vaccine System Makes People With Health Problems Fight for a Place in Line, click here to view the article.
5. Emerging Threats in States like CO, ND, and ME 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.
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.
2. President Biden Appoints First White House Disability Policy Director
President Joe Biden appointed Kimberly Knackstedt to serve as the first White House Disability Policy Director, a position which will be part of the Domestic Policy Council (DPC). "Kim is the perfect leader for the disability community!! This is the most significant appointment our community has ever had," tweeted PIPC Chairman Tony Coelho regarding the news. Click here to read more about the appointment.
3. Drug Pricing Policy Must Prioritize Patients
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.
4. NYT: How America’s Vaccine System Makes People With Health Problems Fight for a Place in Line
PIPC member, Laura Bonnell with the Bonnell Foundation, was quoted in the New York Times article on prioritization of vaccines for people with medical conditions that may raise their risk for a severe case of Covid-19. The article states, "In Michigan, Laura Bonnell, who has two daughters in their 20s with cystic fibrosis, said she extracted a promise from officials in her county this week that they would soon offer vaccine appointments to any cystic fibrosis patient over age 16. 'It’s about desperation,' said Ms. Bonnell, who runs a foundation for families affected by cystic fibrosis. 'It’s about being heard.’” Click here to view the article.
5. Emerging Threats in States like CO, ND, 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. As you know, 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. As states consider such boards and commissions, we hope the Value Our Health legislative template provides a path for states to simultaneously protect patients and people with disabilities by creating standards for the evidence that drives decision-making, assuring engagement of patient and disability stakeholders, and barring the use of QALYs and similar metrics.
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.
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.
In good news, Connecticut has introduced the Value Our Health template legislative language! CT HB06242 "An Act Prohibiting Health Insurers From Using A Dollars-per-quality Adjusted Life Year Or Any Similar Measure As A Threshold” was introduced by Representative Michelle Cook. Please share the great news with advocates in Connecticut that may have interest in being supportive! Click here to view the bill.
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.
- Canada: Advocates push province to fund pancreatic cancer drug as surge in late-stage diagnoses predicted.
- New Zealand: Cancer patient told palliative care only option after being rejected Pharmac funding. Spinal Muscular Atrophy sufferer says that Pharmac funding for needed medicine would be "absolutely life changing." New Zealanders with rare diseases, chronic diseases, or cancer say that their care would be vastly better with improved health care and awareness of those diseases in the medical community. Pharmac's decision to force epilepsy patients to switch medications could have led to their deaths. Click here to read more.
- United Kingdom: Getting life-changing cystic fibrosis drug "bittersweet" for UK woman as NHS denies access to her sister. Teenager pleads for access to life-changing drug as NICE prepares to restrict access to save money. Charities call on NICE to revisit its decision on life-changing prostate cancer drug.
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: Evidence Report AVAILABLE. 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. 4/2/2021: Evidence Report and Responses to Public Comments. Meeting 4/16/2021.
- Alzheimer's Disease: Model Analysis Plan available. 5/5/2021: Draft Evidence Report.
13. Upcoming Events and Webinars
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.
AI, Big Data and Deep Real-World Evidence – The Challenges and Opportunities
April 22, 2021
Click here to view.
14. Medical Journal Articles
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.
Six Ways to Foster Community-Engaged Research During Times of Societal Crises, click here to view.
15. AHRQ Effective Program Updates
Research Report: A Prospective Comparison of Evidence Synthesis Search Strategies Developed With and Without Text-Mining Tools. Click here to view.
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.