— Drugs Extending My Life, but Boston Institute Disagrees. Click here to read the op-ed.
— How CMS Can Advance – or Impede – Patient-Centered Decision-Making in its New Drug Pricing Program. Click here to read the column,
— QALYs: The Math Doesn’t Work. Click here to read the article.
— Opinion: Drug Pricing and the 'Impossible Trinity' for Patients. Click here to read the op-ed.
— PIPC Collaborates with Avalere on Investigation of Use of Patient-Centered Outcomes in Value Assessment. See details below.
— CCD Pens Letter of Support for Protecting Healthcare for All Patients Act. Click here to read the letter.
— Chairman's Corner: I introduced the ADA 35 years ago. Now Congress needs to act to ban a practice that devalues disabled lives. Click here to read the op-ed.
— 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. Drugs Extending My Life, but Boston Institute Disagrees. Steve Kowalski details how ICER's assessment of ALS medicines has restricted his accesses to much needed therapies. "I recently testified at an ICER meeting regarding the medicines I’m on. ICER’s final report regarding one of my medicines found that the results from one trial aren’t enough, despite approval by the Federal Drug Administration (FDA). To add insult to injury, ICER made its decision by comparing treatment of a rare, rapidly progressing terminal illness to other livable diseases with more treatment options. It’s simply not a valid comparison and the repercussions are drastic. Already the insurer CIGNA has said it will stop covering that one treatment." Click here to read the op-ed.
2. How CMS Can Advance – or Impede – Patient-Centered Decision-Making in its New Drug Pricing Program. In a column for STAT News, Joe Vandigo and Hillary Edwards discuss the importance of advancing patient-centered care and decision-making in Medicare drug price negotiations. "...It’s disappointing the recent CMS guidance for the implementation of the Medicare Drug Price Negotiation Program, which uses a number of factors including relative clinical benefit of a drug to determine a government-set price for certain drugs, does not outline a clear plan to engage the patient community or prioritize research studies that were guided by meaningful patient engagement....Patients need to be a part of a real back-and-forth, a true dialogue, and need to be able to see in a transparent way how their input will be considered in policy decision-making. CMS has yet to release any substantial information on these “listening sessions” and most importantly has not given any indication how they meaningfully incorporate feedback into their decision-making." Click here to read the column,
3. QALYs: The Math Doesn’t Work. An article in the Journal of Health Economics and Outcomes Research details key flaws of the QALY. "Proponents of the ban say that QALYs undervalue the positive effects of therapeutics on people with disabilities. We share their concerns. Furthermore, our review of the mathematical properties of QALYs, including an analysis of quality-of-life utility (QOL utility) data recently collected from patients with inflammatory bowel disease (IBD), has led us to conclude that QALYs are an inappropriate metric of drug and treatment cost-effectiveness for all people, both disabled and nondisabled, and should not be the basis for US healthcare policy decisions." Click here to read the article.
4. Opinion: Drug Pricing and the 'Impossible Trinity' for Patients. In an op-ed for STAT News, Keith Ericson and Tal Gross demonstrate exclusionary drug pricing formulas impact patient access to treatments in other countries. “...Many Americans envy the British for their low drug prices and the low out-of-pocket costs. But the British reliance on exclusions still imposes tremendous costs on patients. Consider how the NHS handled breakthrough treatments for cystic fibrosis developed by Vertex Pharmaceuticals. Starting in 2016, the NHS refused to cover the drugs because it decided that Vertex’s price was too high. British parents of children with cystic fibrosis protested in the streets, holding up pictures of their children lying in hospital beds. It took three years for the NHS and Vertex to agree on a discounted price. For those three years, British kids with cystic fibrosis knew that new treatments for their disease existed, but they had no access to them.” Click here to read the op-ed.
5. PIPC Collaborates with Avalere on Investigation of Use of Patient-Centered Outcomes in Value Assessment. The Partnership to Improve Patient Care (PIPC) collaborated with Avalere on research to better understand the current role of patient-centered outcomes (PCOs) in the value assessment process, utilizing select assessments from the Institute for Clinical Economic Review (ICER) as case studies. Findings from this analysis highlighted that the use of PCOs in these values assessments were limited and often did not impact the quantitative assessments of value.
PIPC Chairman Tony Coelho stated, “Our opportunity to provide this feedback is now… ICER released its new proposed value framework for 2023-2026, with comments due June 30, 2023. In it, we see ICER struggling to respond to the criticism from patients and people with disabilities that their work fails to meaningfully incorporate a patient perspective of value.” In its draft updated framework, ICER states that after “ongoing considerations” it still will not include additional dimensions of value citing issues related to double counting. Chairman Coelho went on to say, “At best, ICER recognizes the problem. At worst, ICER concludes it’s just too hard to do.” Click here to view the report conducted with Avalere. Click here to view Chairman Coelho’s blog about the report. Click here to view comments to ICER and here to view its updated value framework.
6. CCD Pens Letter of Support for Protecting Healthcare for All Patients Act. In a letter to House leadership, the Consortium for Constituents with Disabilities urged Speaker Kevin McCarthy (R-CA) and Leader Hakeem Jeffries (D-NY) to support H.R. 485, the Protecting Healthcare for All Patients Act. "[The QALY] fails to consider other factors that influence quality of life, such as emotional well-being, the personal wishes and aspirations of the patient, will to live, the personal beliefs of the patient and more...This bill is a great first step in recognizing the equal value of the lives of individuals with disabilities and will help address inequalities people with disabilities face in accessing healthcare." Click here to read the letter.
7. Chairman's Corner: I introduced the ADA 35 years ago. Now Congress needs to act to ban a practice that devalues disabled lives. In an op-ed for STAT News, PIPC Chair Tony Coelho clears up some mischaracterizations of the Protecting Health Care for All Patients Act. In a QALY-based cost-effectiveness assessment, a person living with conditions like heart disease, ALS, or sickle cell disease will be considered “worth less” to treat than others, and studies may not consider their quality of life improvements to be meaningful, thereby giving the treatment for such conditions a lower value and leading payers to give them less priority for coverage and potentially restrict their coverage. The Protecting Health Care for All Patients Act would extend that ban to other federal health care programs, such as Medicaid. Click here to read the op-ed.
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.
Pennsylvania
In the last legislative session, the legislature introduced, HB 1722, a bill creating a Prescription Drug Affordability Board that would not bar reference to QALYs and would explicitly authorize the Board to contract with a third party that could include ICER. The bill has not been reintroduced in 2023. The Pennsylvania legislature held an informational meeting on Prescription Drug Boards in the House Health Committee on April 12 at 10am. Click here to learn more about Prescription Drug Affordability Boards and the implications of discriminatory measures of cost effectiveness. Click here to learn more about the committee chair, Rep. Dan Frankel.
Massachusetts
An Act Advancing Health Care Research and Decision-Making Centered on Patients and People with disabilities, SD. 2117 and HD. 3356 has been introduced in both the House and Senate. This bill includes important patient protections including a ban on the discriminatory Quality-Adjusted Life Year (QALY).
California
California Attorney General Rob Bonta has solicited information from hospital CEOs across the state about how healthcare facilities and other providers are identifying and addressing racial and ethnic disparities in commercial decision-making tools. In response to this letter, advocates urged Attorney General Bonta to include QALYs and similar metrics in his investigation. Click here to view the Attorney General’s letter. Click here to view a response from advocates.
Oregon
The Oregon legislature held a hearing on March 27, 2023 on SB 492, legislation that would address the use of biased measures of quality of life, such as quality-adjusted life years or QALYs. PIPC Chairman Tony Coelho submitted testimony stating, "This legislation reflects decades of work to advance disability rights and to end the use of discriminatory measures of the effectiveness of health care that too often drive barriers to care for people with disabilities. In the past this may have been considered just a disability issue, but today we recognize the implications of these biased algorithms for health equity more broadly.” Click here to view testimony from PIPC Chairman Tony Coelho. Click here to view testimony submitted by the Caring Ambassadors Program. Click here to view the list of organizations in Oregon supporting the bill.
On October 6, 2022, the Oregon Health Evidence Review Commission (HERC) discussed a proposed plan for using QALYs. The meeting followed CMS approval of Oregon’s waiver application, which does not bar the use of QALYs in the interim. Click here to read comments submitted by PIPC along with 62 other groups asking CMS to reject the waiver. Click here to view the CMS-approved waiver. At the HERC meeting, the Oregon Health Authority expressed its intent to continue using a prioritized list, shifting it from use under a waiver to use as part of the State Plan Amendment after the waiver expires in 2027. The HERC decided to accept comments on their use of QALYs but has not made a decision as to whether they will adjust their methodology. Click here to view the letter from almost 50 advocacy groups asking the HERC to stop using QALYs. Click here to view testimony from PIPC Chairman Coelho to the Oregon HERC expressing his support for the Americans with Disabilities Act. Click here to view the options that continue to be under consideration by the HERC to continue using QALYs. Click here to view all of the comments shared with PIPC.
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: MS patients in Australia face limited choices with respect to effective treatments covered, and some are forced to raise money for treatment overseas.
- U.K: The family of a patient with MS has had to turn to crowdfunding for treatment that is not funded through the NHS.
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.
- Paroxysmal Nocturnal Hemoglobinuria: Draft Scoping Document Available. Comments due August 14, 2023.
- Schizophrenia: Draft Scoping Document available. Comments due on July 26, 2023.
- Metachromatic Leukodystrophy: Draft Evidence Report now available.
- Pulmonary Arterial Hypertension: Draft Scoping Document available. Open for comments until June 5, 2023.
- Sickle Cell Disease: Evidence Report Available. California Technology Assessment Forum (CTAF) to meet on July 27, 2023, to review.
- Non-Alcoholic Steatohepatitis: Final Evidence Report now available.
Upcoming Events and Webinars
PCORI Board of Governors Meeting
September 11, 2023
Click here to view.
2023 PCORI Annual Meeting
October 4-5, 2023
Click here to view.
Medical Journal Articles
The Implementation of Value-Based Frameworks, Clinical Care Pathways, and Alternative Payment Models for Cancer Care in the United States. Click here to view.
Perspectives on Patient-Reported Outcome Data After Treatment Discontinuation in Cancer Clinical Trials. Click here to view.
The Implementation of Value-Based Frameworks, Clinical Care Pathways, and Alternative Payment Models for Cancer Care in the United States. Click here to view.
Federal Data for Conducting Patient-Centered Outcomes Research on Economic Outcomes. Click here to view.
Patient-Reported Outcomes for People with Diabetes: What and How to Measure? A Narrative Review. Click here to view.
Patient Engagement in Research; Benefits, Challenges, Importance, and Implications. Click here to view.
The Opportunity for Greater Patient and Public Involvement and Engagement in Drug Development and Regulation. Click here to view.
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.