1. Forbes: A QALY Is A QALY Is A QALY, Or Is It? Click here to read the column.
2. Op-ed: A little known group is making decisions about which Orange County lives are worth living? Click here to read the op-ed.
3. Morning Consult: Emancipating Data — Science in the Open Is Key to Understanding Value, click here to read the op-ed
4. Health Affairs: Putting Patients’ Goals And Needs At The Center Of Care — How Can We Do Better? Click here to read the blog.
5. Chairman Coelho in San Jose Mercury News: Trump Medicare drug plan would hurt most vulnerable, click here to read the op-ed
6. PIPC Submits Comment to CMS on Proposed International Pricing Index, click here to read the letter.
7. PIPC: Don't Discriminate on Care, click here to view.
8. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? Click here to view.
9. Upcoming ICER Studies: Angiodema, Asthma, Opioid Use Disorder, MS, Peanut Allergy, SMA, Depression, click here to provide patient input
10. Upcoming Events and Webinars, see details below.
11. Medical Journal Articles, see details below.
12. AHRQ Effective Program Updates, see details below.
In a column for Forbes, Joshua Cohen writes that the use of QALYs to compare qualitatively different kinds of interventions and disease or condition categories - life-saving or life-threatening versus life-improving - may be problematic. “...Besides the arbitrariness of the numerator in cost-per-QALY thresholds, a central question facing ICER and CVS Caremark is whether a drug's value should depend on not only a measure such as QALYs, but also on which disease or population is being treated, which implies that a QALY may not always have the same weight. In other words, should ICER invoke higher (i.e., more lenient) cost-per-QALY gained cost-effectiveness thresholds in some areas (say, cancer or rare diseases, or young patients) than others and, if so, on what basis?" Click here to read the column.
2. Op-ed: A little known group is making decisions about which Orange County lives are worth living
In an op-ed for The Orange County Register, Chris Buchanan discusses the issues with ICERs assessment of value. “The problems with ICER’s research and methodology are legion, but the fundamental problem is this: ICER is making decisions about the value of a human life – about what a life is worth and whose life is worth saving – based only on financial considerations. If you are an Orange County resident living with a life-threatening or chronic disease, such as cancer, cystic fibrosis, arthritis, or many others, ICER’s formula is likely to determine that your life is worth less than that of a healthy person. Simply because a patient has the misfortune to get a disease does not make his or her life any less valuable than that of a healthy person.”
“Another deficiency in ICER’s approach is that they fail to give meaningful consideration to what patients living with the affected conditions say about what improves their quality of life. There is little transparency into what goes into ICER’s formulations or how they arrive at their conclusions, which means other researchers are unable to analyze and replicate ICER’s results. Finally, in addition to being cruel and punitive, ICER’s one-size-fits-all approach to insurance coverage is likely to discourage efforts to develop innovative new treatments for many chronic and life-threatening conditions. It’s just a bad model.” Click here to read the op-ed.
3. Morning Consult: Emancipating Data — Science in the Open Is Key to Understanding Value
In an op-ed for Morning Consult, IVI’s Jennifer Bright writes that the lack of transparency and collaboration in medical research creates barriers to efficient learning and dissemination to practice. In the health care marketplace, the need for openness goes beyond foundational science and academic publications to data that inform real-world decision-making at the bedside, and in coverage and payment. Without access to all data (both positive and negative) on therapies, for example, clinicians cannot make the best decisions. And without access to real-world data and methods for incorporating it into value assessment, payers and employers cannot maximize value in benefit design. If we are to move toward value-based reimbursement, openness is a prerequisite. That’s why the Innovation and Value Initiative conceived the Open-Source Value Platform as an open laboratory for creating more sophisticated, transparent and flexible systems to assess value in health care. We think the OSVP is an important step toward the openness needed to adequately measure value in health care. But even these efforts can only go so far without open access to the varied data that drives all value assessment. Click here to read the op-ed.
4. Health Affairs: Putting Patients’ Goals And Needs At The Center Of Care — How Can We Do Better?
In a post for Health Affairs Blog, Tara Oakman and Lauren Gerlach highlight patient engagement strategies for making health and health care systems truly focused on the goals and needs of the people they serve. “...In recent years, for example, [the Robert Wood Johnson Foundation] has invested in research to understand what consumers value in health care, supported state and local consumer health advocacy organizations, developed principles of what measurement in health care could look like if it were truly patient centered, and supported research to better understand the information that people, especially low-income populations, are seeking in making decisions related to their coverage and care...Here, we highlight a few strategies discussed at the meeting that may be of particular interest to those in health philanthropy.” Click here to read the blog.
5. Chairman Coehlo in San Jose Mercury News: Trump Medicare drug plan would hurt most vulnerable
PIPC Chairman Tony Coehlo penned an op-ed in The Mercury News outlining his concerns with the Trump administration’s proposed international drug pricing index. “I’m all for reforms to reduce drug prices and improve affordability. I’ve consistently advocated for a seat at the table for patients and people with disabilities in creating a health care system that pays for high-quality care. Data and information from personalized and precision medicine can tell us what treatment works for whom to both improve access and lower prices. What I’m not for, however, are policies that systematically disadvantage those most vulnerable. In effect, the administration proposes to smuggle in the same discriminatory coverage standards and formularies that I and many other leading advocates have fought for the last 30 years.”
“Other countries limit access to medications using one-size-fits-all thresholds based on quality-adjusted-life-years, or ‘QALYs.’ QALYs seek to quantify the extent to which a treatment extends life and improves health on average — but in practice, these algorithms discriminate by assigning higher value to people in ‘perfect health’ than people in less-than-perfect health. Treatments for older individuals with fewer years to live or for people living with disabilities fare badly under algorithms that say, ‘you’re not worth it.’” Click here to read the op-ed.
6. PIPC Submits Comment Letter to CMS on Proposed International Pricing Index
PIPC joined organizations representing patients, people with disabilities, family members, caregivers, veterans, seniors, providers, and others in response to the Advance Notice of Proposed Rulemaking released by the Center for Medicare & Medicaid Services (CMS) to utilize an “International Pricing Index” (IPI) to set reimbursement for medicines in Medicare Part B. The letter highlights concerns that this new policy would import QALY-based standards to key U.S. health programs. "Addressing health care costs, including drug prices, is an important and meaningful effort that should center on achieving outcomes that matter to those being served by health systems (patients, people with disabilities, veterans, seniors and other marginalized communities) such as improved quality of care and lower out-of-pocket costs," the letter states. "We are hopeful the Administration will reconsider their plan to import international cost-effectiveness standards into the U.S. and instead advance patient-centered, non-discriminatory approaches and establish meaningful protections for our communities in future demonstrations." Click here to read the letter.
7. PIPC: Don't Discriminate on Care
Patients and people with disabilities face major threats as private insurers and government programs look to cut costs using quality-adjusted-life-years (QALYs) and other assessments that discriminate against patients, people with disabilities and seniors. Recently, new threats have emerged, including:
- A pharmacy benefit manager’s new plan to limit access to prescription drugs using a rigid cost-per-QALY threshold, similar to that used by the United Kingdom’s National Health Service;
- The use of cost-per-QALY thresholds in New York’s Medicaid program;
- A new Medicare proposal that would set reimbursement for physician-administered medicines based off of decisions made in countries that use WALYs and cost effectiveness thresholds to set national coverage.
8. 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. In New Zealand, patients are not getting access to treatment, and some are forced to leave the country to access treatments. Click here, here and here to read more. In Canada, click here to view the story of a child with thyroid cancer forced to come to the U.S. for care, and here to learn about patients seeking medications for heart pain being denied access. Spinal muscular atrophy advocates are similarly petitioning Health Canada to expand access to treatment. Click here to view the article. Similarly, in the United Kingdom, activists fight for access to treatments for cystic fibrosis and cancer. Click here and here for articles related to cystic fibrosis, and here for an article related to limited access to treatments for lung cancer. Click here for an article related to MS. The Wall Street Journal’s Editorial Board recently opined on "why the U.S. shouldn’t put the world’s most innovative drug market at the mercy of what Greece is willing to pay for a cancer treatment.” Click here to view.
9. Upcoming ICER Studies: Angiodema, Asthma, Opioid Use Disorder, MS, Peanut Allergy, SMA, Depression
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. Please note the following upcoming formal ICER deadlines per their website:
Spinal Muscular Atrophy: 12/20/2018-1/31/19 Open Input on draft report!! 3/7/19 Public meeting.
Depression: Revised scoping document available on treatment-resistant depression. 1/14/19 Research Protocol. Also note interventions of interest for 2019 review: Esketamine (Janssen)
Multiple Sclerosis: Revised scoping document available on treatment for secondary progressive multiple sclerosis. 1/14/19 Research Protocol.
Peanut Allergy: Stakeholder list available. Revised scoping document on 12/20/2018. 4/9/19 Draft Evidence Report.
Duchenne Muscular Dystrophy: Open input period 12/20-1/8/2019!!
10. Upcoming Events and Webinars
Cycle 1 2019 Treatment of Anxiety in Children, Adolescents, and/or Young Adults PFA Applicant Town Hall
January 22, 2019
Click here for details.
Pharmacological Treatment for Anxiety in Children, Adolescents, and/or Young Adults PFA Applicant Town Hall -- Cycle 1 2019
January 22, 2019
Click here for details.
Cycle 1 2019 Broad PFAs Applicant Town Hall
January 23, 2019
Click here for details
Nothing About Us Without Us: How the Need for Cultural Responsiveness is Changing Research
January 24, 2019
Click here for details
PCORI Board of Governors Meeting
January 29, 2019
Click here for details.
Patient Registries and Real-World Evidence Summit J
January 30, 2019
Click here for details.
Patient Registries and Real-World Evidence Summit J
January 30-31, 2019
Click here for details.''
Webinar: How Real-World Evidence Is Playing Out In The Real World
February 14, 2019
Click here for details.
2019 NEC Symposium
June 2 - 5, 2019,
Click here for details.
A New Path Forward for Using Real World Evidence in Randomized Clinical Trials
June 23, 2019,
Click here for details.
11. Medical Journal Articles
A Patient-Centered Approach to Comparative Effectiveness Research Focused on Older Adults: Lessons From the Patient-Centered Outcomes Research Institute, click here to view.
Editorial: Evaluating Patient and Public Involvement in Research, click here to view.
Developing a Patient-Centered Outcome for Targeting Early Childhood Obesity Across Multiple Stakeholders, click here to view.
Cancer Patient Perspectives on the Use of Clinical Pathways and Shared Decision-Making in Cancer Care, click here to view.
In Proportion: Approaches for Displaying Patient-reported Outcome Research Study Results as Percentages Responding to Treatment, click here to view.
If Patients Are the True North, Patient-Centeredness Should Guide Research, click here to view.
Understanding and Improving Value Frameworks With Real-World Patient Outcomes, click here to view.
Multi-Method Patient-Engagement Approach: A Case Example from a PCORI-Funded Training Project, click here to view.
Comparative Effectiveness and Safety of Bariatric Procedures for Weight Loss: A PCORnet Cohort Study, click here to view.
Oh, the Places We'll Go: Patient-Reported Outcomes and Electronic Health Records, click here to view.
Putting Patients at the Centre of Healthcare: Progress and Challenges for Health Technology Assessments, click here to view.
12. AHRQ Effective Program Updates
Patient Navigation Models for Lung Cancer, click here to view.
Stroke Prevention in Patients With Atrial Fibrillation: A Systematic Review Update, click here to view.
Long-term Drug Therapy and Drug Holidays for Osteoporosis Fracture Prevention: A Systematic Review, click here to view.
Addressing Social Isolation to Improve the Health of Older Adults: A Rapid Review, click here to view.
Labor Dystocia, click here to view.
Nonsurgical Treatments for Urinary Incontinence in Women: A Systematic Review Update, click here to view.
Randomized Trial of a Patient-Centered Decision Aid for Promoting Informed Decisions about Lung Cancer Screening: Implementation of a PCORI Study Protocol and Lessons Learned, click here to view.
Collaboration Is Key to Accelerating Diagnostics Access to Optimize Benefits of Precision Medicines, click here to view.
Telehealth for Acute and Chronic Care Consultations, click here to view.
Library of Patient-Centered Outcomes Research Resources, click here to view.