1. PIPC Patient Blog: CancerCare Response to 'Mouse That Roared,' click here to read the blog.
2. Patient Advocates Say ICER Uses Discriminatory Cost Metrics, click here to read the full article.
3. Pioneer Institute Report Addresses Shortcomings of ICER Models to Evaluate Rare Disease Treatments, click here to read the report.
4. Webinar: Value Assessments – the Impact on People with Rare Diseases, see below for details.
5. AAR Seeks Recruits for Annual Senior Patient and Family Caregivers Network Training, click here to learn more and to apply for the training.
6. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
7. ICER Studies: Type 2 Diabetes, Arthritis, Cardiovascular Disease, Duchenne Muscular Dystrophy click here to provide patient input.
8. Upcoming Events and Webinars, see details below.
9. Medical Journal Articles, see details below.
10. AHRQ Effective Program Updates, see details below.
2. Patient Advocates Say ICER Uses Discriminatory Cost Metrics, click here to read the full article.
3. Pioneer Institute Report Addresses Shortcomings of ICER Models to Evaluate Rare Disease Treatments, click here to read the report.
4. Webinar: Value Assessments – the Impact on People with Rare Diseases, see below for details.
5. AAR Seeks Recruits for Annual Senior Patient and Family Caregivers Network Training, click here to learn more and to apply for the training.
6. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
7. ICER Studies: Type 2 Diabetes, Arthritis, Cardiovascular Disease, Duchenne Muscular Dystrophy click here to provide patient input.
8. Upcoming Events and Webinars, see details below.
9. Medical Journal Articles, see details below.
10. AHRQ Effective Program Updates, see details below.
1. PIPC Patient Blog: CancerCare Response to 'Mouse That Roared'
In a PIPC blog, patient advocate Carole Florman responds to a Boston Globe article that characterizes ICER as a "mouse that roared," noting that ICER's methodologies are fatally flawed and discriminate against people with disabilities. "ICER continues to rely on a one-size-fits all approach that does not account for differences among patients. Not every drug or treatment works for every patient, but what ICER may judge to be a “low value” treatment may be the only one that can save a particular patient’s life. My guess is that to that patient and their family, such treatment is seen as very high value indeed. Those of us advocating for patients simply want the patient’s voice included and there are models for doing so," she wrote. Click here to read the blog.
2. Patient Advocates Say ICER Uses Discriminatory Cost Metrics
Inside Health Policy interviewed a number of disability and patient advocates, including PIPC's Ari Ne'eman and Sara van Geertruyden, about how ICER's cost metrics discriminate against people with disabilities. "The metric at issue is the Quality-Adjusted Life-Year, which is a commonly used standard internationally to determine the cost-effectiveness of medical treatments, including drugs. One QALY equals one year in perfect health. Congress spurned the QALY metric when it restricted the partly government-funded Patient-Centered Outcomes Research Institute’s use of similar metrics in the Affordable Care Act....Ari Ne’eman, a consultant and disability rights advocate, said he took issue with the QALY’s use of utility weights, or discounting the value of a year of life for patients who have chronic illness or disabilities," it wrote. Click here to read the full article.
3. Pioneer Institute Report Addresses Shortcomings of ICER Models to Evaluate Rare Disease Treatments
Pioneer Institute released a new report, Looming Challenges for ICER in Assessing the Value of Rare Disease Therapies, that examines why the Institute for Clinical and Economic Review (ICER) and the Quality Adjusted Life Years (QALY) approach to value assessment is particularly ill-suited to assess the cost-effectiveness of orphan and rare disease treatments, which represent a rapidly growing sector of the biopharmaceutical marketplace. In the report, lead author and Pioneer Institute Visiting Fellow in Life Sciences, Dr. William Smith illustrates a number of reasons why ICER is unfit to evaluate the cost-effectiveness of rare disease treatments. "The ICER QALY thresholds are arbitrary enough when applied to traditional medicines but when applied to treatments for rare diseases, they take on additional – and troubling – arbitrariness…These thresholds are not “scientific” in any economic sense but, in asserting a value on human life, they are a kind of theological dogma that must be accepted on faith as an objective standard," wrote Dr. Smith. Click here to read the report.
4. Webinar: Value Assessments – the Impact on People with Rare Diseases
The Partnership to Improve Patient Care (PIPC) and Everylife Foundation for Rare Diseases invites people with rare diseases, as well as their families and providers to join our webinar on July 17 from 1:30-3 PM EST to learn about value assessments, their potential to limit access and innovation, and related public policy threats at the federal and state level.
We are in a very promising time for medical research and development in which true disease altering gene therapies are right around the corner. These therapies have the potential to provide life-altering, and, in some cases, curative treatments for rare disease, which up until recently had no hope for treatment. As these treatments are being developed, organizations like ICER are rushing to determine their value or “cost-effectiveness.” Metrics for measuring the “cost effectiveness” of treatments often relies on discriminatory methods, like the quality-adjusted-life-year (QALY), which values the lives of people with disabilities and serious chronic conditions as worth less than those of non-disabled people. This concerning way of measuring value consistently devalues drugs for rare disease, which can lead to limitations on patient access and a disincentive for investment in rare disease research and development. While no patient is average, average value metrics are increasingly at the center of state and federal discussions about drug pricing, and advocates may be unfamiliar with how to engage with a complicated but important issue area.
Disability and patient advocates have long fought against the QALY, achieving a prohibition against its use in Medicare within the Affordable Care Act. Previous administrations have ruled that using QALYs to allocate healthcare resources may constitute a violation of the Americans with Disabilities Act. But over the last few years, proposals to use average metrics such as QALYs have become increasingly common, threatening access to lifesaving medications for people with disabilities and those with rare diseases.
This event will arm advocates with the information they need to advocate against policies that will limit innovation and access for rare disease patients. We hope you will join us in advocating for policymakers to Value Our Health! Please RSVP to [email protected].
5. AAR Seeks Recruits for Annual Senior Patient and Family Caregivers Network Training
Alliance for Aging Research’s Senior Patient and Family Caregiver Network (SP&FCN) is seeking advocates to participate in a research-advocacy training program designed to empower senior patients and their family caregivers to engage in patient-centered outcomes research (PCOR). The training will be held November 19-21, 2019 in Dallas, TX, and reimbursements for a hotel stay and travel expenses are available. In particular, the Alliance is looking for patients or caregivers of patients with Alzheimer’s disease, sarcopenia, atrial fibrillation, chronic pain, age-related macular degeneration, or heart valve disease. Click here to learn more and to apply for the training.
6. 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.
7. ICER Studies: Acute Migraine, Type 2 Diabetes, Arthritis, Cardiovascular Disease, Duchenne Muscular Dystrophy, Peanut Allergy
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:
8. Upcoming Events and Webinars
National Health Council Webinar on Rubric to Capture the Patient Voice
July 11, 2019
Click here for details.
PQA Quality Forum Webinar: Pharmacists’ Expanding Scope of Practice: Are We Ready?
July 11, 2019
Click here for details.
Leveraging Randomized Clinical Trials to Generate Real-World Evidence for Regulatory Purposes
July 11-12, 2019
Click here for details.
PCORI Board of Governors Meeting
July 23, 2019
Click here for details.
NVHR Hepatitis C Patient Summit
July 29-30, 2019
Click here for details.
PCORI Board of Governors Meeting
August 20, 2019
Click here for details.
Advisory Panel on Rare Disease Fall 2019 Meeting
September 16, 2019
Click here for details.
2019 PCORI Annual Meeting
September 18-20, 2019
Click here for details.
FT Pharma Pricing and Value Summit 2019
September 26, 2019
Click here for details.
2019 AUCD Annual Meeting
November 17-20, 2019
Click here for details.
9. Medical Journal Articles
Ethical Challenges Related to Patient Involvement in Health Technology Assessment, click here to view.
Value-Based Insurance Design: Current Evidence and Future Directions, click here to view.
Novel Approaches to Value Assessment Beyond the Cost-Effectiveness Framework, click here to view.
Navigating Joint HTA, Procurement, and Fair Pricing: Evidence-Based Insights and Practical Recommendations - A Meeting Report from ISPOR Regional Conference in Warsaw, 2019, click here to view.
Health Technology Assessment as Part of a Broader Process for Priority Setting and Resource Allocation, click here to view.
Improving Quality Measure Maintenance: Navigating the Complexities of Evolving Evidence, click here to view.
A Tool for Empirical Equipoise Assessment in Multigroup Comparative Effectiveness Research, click here to view.
As Health Technology Assessment Evolves So Must its Approach to Patient Involvement, click here to view.
Evaluation of Value-Based Insurance Design for Primary Care, click here to view.
Leveraging Patient/Community Partnerships to Disseminate Patient Centered Outcomes Research in Geriatrics, click here to view.
Enabling Individualised Health in Learning Healthcare Systems, click here to view.
10. AHRQ Effective Program Updates
Systematic Review: Achieving Health Equity in Preventative Services, click here to view.
Research Protocol: Impact of Community Health Worker Certification on Workforce and Service Delivery for Asthma and Other Selected Chronic Diseases, click here to view.
Systematic Review: Treatment of Depression in Children, click here to view.
AHRQ EPC Program Helps Health Systems Use Evidence, click here to view.
Technical Brief: Pharmacological and Nonpharmacological Treatments for Post-Traumatic Stress Disorder, click here to view.
Systematic Review: Management of Infertility, click here to view.
Key Questions: Cervical Ripening in the Outpatient Setting, click here to view.
Key Questions: Radiation Therapy for Brain Metastases, click here to view.
Protocol: A Rapid Evidence Review of Retention Strategies for Medications for Addiction Treatment (MAT) in Adults with Opioid Use Disorder, click here to view.
Systematic Review: Diagnosis and Treatment of Clinical Alzheimer's-type Dementia (CATD), click here to view.
Systematic Review: Comparative Effectiveness of Analgesics to Reduce Acute Pain in the Prehospital Setting, click here to view.
Systematic Review: Long-Term Drug Therapy and Drug Holidays for Osteoporosis Fracture Prevention, click here to view.
Systematic Review: Telehealth for Acute and Chronic Care Consultations, click here to view.
In a PIPC blog, patient advocate Carole Florman responds to a Boston Globe article that characterizes ICER as a "mouse that roared," noting that ICER's methodologies are fatally flawed and discriminate against people with disabilities. "ICER continues to rely on a one-size-fits all approach that does not account for differences among patients. Not every drug or treatment works for every patient, but what ICER may judge to be a “low value” treatment may be the only one that can save a particular patient’s life. My guess is that to that patient and their family, such treatment is seen as very high value indeed. Those of us advocating for patients simply want the patient’s voice included and there are models for doing so," she wrote. Click here to read the blog.
2. Patient Advocates Say ICER Uses Discriminatory Cost Metrics
Inside Health Policy interviewed a number of disability and patient advocates, including PIPC's Ari Ne'eman and Sara van Geertruyden, about how ICER's cost metrics discriminate against people with disabilities. "The metric at issue is the Quality-Adjusted Life-Year, which is a commonly used standard internationally to determine the cost-effectiveness of medical treatments, including drugs. One QALY equals one year in perfect health. Congress spurned the QALY metric when it restricted the partly government-funded Patient-Centered Outcomes Research Institute’s use of similar metrics in the Affordable Care Act....Ari Ne’eman, a consultant and disability rights advocate, said he took issue with the QALY’s use of utility weights, or discounting the value of a year of life for patients who have chronic illness or disabilities," it wrote. Click here to read the full article.
3. Pioneer Institute Report Addresses Shortcomings of ICER Models to Evaluate Rare Disease Treatments
Pioneer Institute released a new report, Looming Challenges for ICER in Assessing the Value of Rare Disease Therapies, that examines why the Institute for Clinical and Economic Review (ICER) and the Quality Adjusted Life Years (QALY) approach to value assessment is particularly ill-suited to assess the cost-effectiveness of orphan and rare disease treatments, which represent a rapidly growing sector of the biopharmaceutical marketplace. In the report, lead author and Pioneer Institute Visiting Fellow in Life Sciences, Dr. William Smith illustrates a number of reasons why ICER is unfit to evaluate the cost-effectiveness of rare disease treatments. "The ICER QALY thresholds are arbitrary enough when applied to traditional medicines but when applied to treatments for rare diseases, they take on additional – and troubling – arbitrariness…These thresholds are not “scientific” in any economic sense but, in asserting a value on human life, they are a kind of theological dogma that must be accepted on faith as an objective standard," wrote Dr. Smith. Click here to read the report.
4. Webinar: Value Assessments – the Impact on People with Rare Diseases
The Partnership to Improve Patient Care (PIPC) and Everylife Foundation for Rare Diseases invites people with rare diseases, as well as their families and providers to join our webinar on July 17 from 1:30-3 PM EST to learn about value assessments, their potential to limit access and innovation, and related public policy threats at the federal and state level.
We are in a very promising time for medical research and development in which true disease altering gene therapies are right around the corner. These therapies have the potential to provide life-altering, and, in some cases, curative treatments for rare disease, which up until recently had no hope for treatment. As these treatments are being developed, organizations like ICER are rushing to determine their value or “cost-effectiveness.” Metrics for measuring the “cost effectiveness” of treatments often relies on discriminatory methods, like the quality-adjusted-life-year (QALY), which values the lives of people with disabilities and serious chronic conditions as worth less than those of non-disabled people. This concerning way of measuring value consistently devalues drugs for rare disease, which can lead to limitations on patient access and a disincentive for investment in rare disease research and development. While no patient is average, average value metrics are increasingly at the center of state and federal discussions about drug pricing, and advocates may be unfamiliar with how to engage with a complicated but important issue area.
Disability and patient advocates have long fought against the QALY, achieving a prohibition against its use in Medicare within the Affordable Care Act. Previous administrations have ruled that using QALYs to allocate healthcare resources may constitute a violation of the Americans with Disabilities Act. But over the last few years, proposals to use average metrics such as QALYs have become increasingly common, threatening access to lifesaving medications for people with disabilities and those with rare diseases.
This event will arm advocates with the information they need to advocate against policies that will limit innovation and access for rare disease patients. We hope you will join us in advocating for policymakers to Value Our Health! Please RSVP to [email protected].
5. AAR Seeks Recruits for Annual Senior Patient and Family Caregivers Network Training
Alliance for Aging Research’s Senior Patient and Family Caregiver Network (SP&FCN) is seeking advocates to participate in a research-advocacy training program designed to empower senior patients and their family caregivers to engage in patient-centered outcomes research (PCOR). The training will be held November 19-21, 2019 in Dallas, TX, and reimbursements for a hotel stay and travel expenses are available. In particular, the Alliance is looking for patients or caregivers of patients with Alzheimer’s disease, sarcopenia, atrial fibrillation, chronic pain, age-related macular degeneration, or heart valve disease. Click here to learn more and to apply for the training.
6. 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: It can take five years for Australians to access lifesaving medicine. Delays in accessing medicine can be a matter of life or death.
- New Zealand: NZ Myeloma patients lack access to needed medicine. Breast cancer advocates say that human life is not prioritized by PHARMAC. Click here to read more.
- Canada: B.C. SMA patients fight to access Spinraza. An Ontario woman living with CF advocates for greater access to lifesaving drugs.
- United Kingdom: Patients continue to fight for access to cystic fibrosis drugs. Patients call for coverage of a new migraine drug by NHS Wales.
7. ICER Studies: Acute Migraine, Type 2 Diabetes, Arthritis, Cardiovascular Disease, Duchenne Muscular Dystrophy, Peanut Allergy
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:
- Arthritis: Updated scoping document available. 7/9/2019: Research Protocol. Meeting 11/19/2019: CTAF to an update to its 2017 rheumatoid arthritis assessment.
- Type 2 Diabetes: Research Protocol Available. 7/29/2019: Model Analysis Plan. Meeting 11/14/2019: The New England CEPAC will convene to deliberate on ICER's review of oral semaglutide for the treatment of type 2 diabetes.
- Cardiovascular Disease : Model Analysis Plan available. 7/24/2019: Draft Evidence Report. 9/26/2019 Meeting: Midwest CEPAC to deliberate and vote on ICER's report on evidence presented in ICER's report on additive CVD therapies.
- Peanut Allergy: Evidence Presentation and Report available. 7/10/2019: Final Evidence Report.
- Duchenne Muscular Dystrophy: 7/11/2019: Evidence Report, Revised Voting Questions, and Response to Comments. Meeting 7/25/2019: New England CEPAC to deliberate and vote on evidence presented in ICER's report on treatments for Duchenne muscular dystrophy.
- Unsupported Price Increase Assessment: 10/8/2019: Final Report.
- Acute Migraine: Comments OPEN on Draft Scoping Document, Stakeholder List through 7/19/2019. Meeting 1/23/2020: Midwest CEPAC to review ICER's assessment of acute migraine treatments
- Valuing A Cure Project: White Paper available 8/6/2019. Comment period open 8/6/2019-9/3/2019.
8. Upcoming Events and Webinars
National Health Council Webinar on Rubric to Capture the Patient Voice
July 11, 2019
Click here for details.
PQA Quality Forum Webinar: Pharmacists’ Expanding Scope of Practice: Are We Ready?
July 11, 2019
Click here for details.
Leveraging Randomized Clinical Trials to Generate Real-World Evidence for Regulatory Purposes
July 11-12, 2019
Click here for details.
PCORI Board of Governors Meeting
July 23, 2019
Click here for details.
NVHR Hepatitis C Patient Summit
July 29-30, 2019
Click here for details.
PCORI Board of Governors Meeting
August 20, 2019
Click here for details.
Advisory Panel on Rare Disease Fall 2019 Meeting
September 16, 2019
Click here for details.
2019 PCORI Annual Meeting
September 18-20, 2019
Click here for details.
FT Pharma Pricing and Value Summit 2019
September 26, 2019
Click here for details.
2019 AUCD Annual Meeting
November 17-20, 2019
Click here for details.
9. Medical Journal Articles
Ethical Challenges Related to Patient Involvement in Health Technology Assessment, click here to view.
Value-Based Insurance Design: Current Evidence and Future Directions, click here to view.
Novel Approaches to Value Assessment Beyond the Cost-Effectiveness Framework, click here to view.
Navigating Joint HTA, Procurement, and Fair Pricing: Evidence-Based Insights and Practical Recommendations - A Meeting Report from ISPOR Regional Conference in Warsaw, 2019, click here to view.
Health Technology Assessment as Part of a Broader Process for Priority Setting and Resource Allocation, click here to view.
Improving Quality Measure Maintenance: Navigating the Complexities of Evolving Evidence, click here to view.
A Tool for Empirical Equipoise Assessment in Multigroup Comparative Effectiveness Research, click here to view.
As Health Technology Assessment Evolves So Must its Approach to Patient Involvement, click here to view.
Evaluation of Value-Based Insurance Design for Primary Care, click here to view.
Leveraging Patient/Community Partnerships to Disseminate Patient Centered Outcomes Research in Geriatrics, click here to view.
Enabling Individualised Health in Learning Healthcare Systems, click here to view.
10. AHRQ Effective Program Updates
Systematic Review: Achieving Health Equity in Preventative Services, click here to view.
Research Protocol: Impact of Community Health Worker Certification on Workforce and Service Delivery for Asthma and Other Selected Chronic Diseases, click here to view.
Systematic Review: Treatment of Depression in Children, click here to view.
AHRQ EPC Program Helps Health Systems Use Evidence, click here to view.
Technical Brief: Pharmacological and Nonpharmacological Treatments for Post-Traumatic Stress Disorder, click here to view.
Systematic Review: Management of Infertility, click here to view.
Key Questions: Cervical Ripening in the Outpatient Setting, click here to view.
Key Questions: Radiation Therapy for Brain Metastases, click here to view.
Protocol: A Rapid Evidence Review of Retention Strategies for Medications for Addiction Treatment (MAT) in Adults with Opioid Use Disorder, click here to view.
Systematic Review: Diagnosis and Treatment of Clinical Alzheimer's-type Dementia (CATD), click here to view.
Systematic Review: Comparative Effectiveness of Analgesics to Reduce Acute Pain in the Prehospital Setting, click here to view.
Systematic Review: Long-Term Drug Therapy and Drug Holidays for Osteoporosis Fracture Prevention, click here to view.
Systematic Review: Telehealth for Acute and Chronic Care Consultations, click here to view.
