In This Week’s Issue:
1. Value Assessments – the Impact on People with Rare Diseases, click here to learn more and watch the webinar.
2. The Hill: Health Systems Must Help Patients Make Informed Decisions, click here to read the article.
3. Op-Ed: ICER's Agenda Helped by Media Attention, click here to read the article.
4. FH Foundation Publication on Cardiovascular Outcomes, click here to read the article.
5. ICER Announces Possible 2020 Assessments, click here to see the list of possible assessments.
6. AAR Seeks Recruits for Annual Senior Patient and Family Caregivers Network Training, click here to learn more and to apply for the training.
7. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
8. ICER Studies: Type 2 Diabetes, Arthritis, Cardiovascular Disease, Duchenne Muscular Dystrophy click here to provide patient input.
9. Upcoming Events and Webinars, see details below.
10. Medical Journal Articles, see details below.
11. AHRQ Effective Program Updates, see details below.
1. Value Assessments – the Impact on People with Rare Diseases, click here to learn more and watch the webinar.
2. The Hill: Health Systems Must Help Patients Make Informed Decisions, click here to read the article.
3. Op-Ed: ICER's Agenda Helped by Media Attention, click here to read the article.
4. FH Foundation Publication on Cardiovascular Outcomes, click here to read the article.
5. ICER Announces Possible 2020 Assessments, click here to see the list of possible assessments.
6. AAR Seeks Recruits for Annual Senior Patient and Family Caregivers Network Training, click here to learn more and to apply for the training.
7. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
8. ICER Studies: Type 2 Diabetes, Arthritis, Cardiovascular Disease, Duchenne Muscular Dystrophy click here to provide patient input.
9. Upcoming Events and Webinars, see details below.
10. Medical Journal Articles, see details below.
11. AHRQ Effective Program Updates, see details below.
1. Value Assessments – the Impact on People with Rare Diseases
On July 17, the Partnership to Improve Patient Care (PIPC) and Everylife Foundation for Rare Diseases held a webinar about value assessments, their potential to limit access and innovation for rare disease treatments. The webinar featured Sara van Geertruyden, Executive Director, Partnership to Improve Patient Care; Ari Ne’eman, Disability Advocate, Partnership to Improve Patient Care; Siri Vaeth, Executive Director, Cystic Fibrosis Research, Inc.; and Christina Hartman, Senior Director of Policy and Advocacy, Everylife Foundation for Rare Diseases. The panelists highlighted their concern about the value assessments — like the quality-adjusted life year (QALY) and equal value life-year gained (evLYG) – because the metrics they use treat patients as averages. They noted that cost-effectiveness assessments are particularly concerning when it comes to treatments developed for rare diseases, as they have the potential to impact both patient access and disincentivize investment in rare disease research and development. Click here to learn more and watch the webinar.
2. The Hill: Health Systems Must Help Patients Make Informed Decisions
In an op-ed published in The Hill, the National Health Council's Marc Boutin and the Milken Institute's Tanisha Carino, Ph.D. write that PCORI has made great strides in helping doctors and patients focus on patients' needs and ensure that treatments are in line with patient preferences. They also argue for its continued existence. "In its first decade, PCORI has changed the system for the better. Reauthorization will ensure continued progress toward a health-care system that enables patients to make more informed decisions and achieve the outcomes most meaningful to them," they wrote. Click here to read the article.
3. Op-Ed: ICER's Agenda Helped by Media Attention
Robert Goldberg of the Center for Medicine in the Public Interest wrote that some journalists accept ICER evaluations at face value and fail to ask critical questions, resulting in the public having a skewed view of ICER and its work. "The demonization of small patient groups as somehow being mere mouthpieces for drug companies is consistent with the narrative that pharma funding of organizations who would otherwise have no voice is illegitimate. And it also sidelines a more serious discussion of whether patients believe Exondys work. The fact is, ICER ignores the value of orphan drugs and media coverage of funding enables such willful ignorance," he wrote. Click here to read the article.
4. FH Foundation Publication on Cardiovascular Outcomes
A new study underscores the risk of denying a prescribed treatment and the imperative for patients not to be treated as averages. New research from the FH Foundation highlights that individuals at high risk for cardiovascular events, including those with familial hypercholesterolemia (FH) or atherosclerotic cardiovascular disease (ASCVD), experienced more heart attacks, strokes and other cardiovascular events when they were unable to obtain their prescribed PCSK9 inhibitor, a cholesterol-lowering treatment. The new study, published in Circulation: Cardiovascular Quality and Outcomes, found that high-risk individuals had a 16 percent increased risk of a cardiovascular event during the 11.5-month study period when their PCSK9 inhibitors were rejected by their insurance plan. Click here to read the article.
5. ICER Announces Possible 2020 Assessments
The Institute for Clinical and Economic Research (ICER) published a list of possible assessments for 2020. The possible assessments include drugs to treat cystic fibrosis, breast cancer, multiple sclerosis, and HIV. ICER's QALY-based studies are not transparent, use discriminatory cost-effectiveness measures, and ignore patient preferences and input. Click here to see the list of possible assessments.
6. 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.
7. 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.
8. 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:
9. Upcoming Events and Webinars
NVHR Hepatitis C Patient Summit
July 29-30, 2019
Click here for details.
Webinar: Real World Evidence and Data: A Tufts Study of 30 Pharma Companies
August 15, 2019
Click here for details.
PCORI Board of Governors Meeting
August 20, 2019
Click here for details.
Webinar: How Frequently Should Cancer Survivors be Screened for Disease Recurrence? Is More Always Better?
August 22, 2019
Click here for details.
Health Spending: Moving from Theory to Action
September 11, 2019
Click here for details.
PCORI Improving Methods Applicant Town Hall
September 12, 2019
Click here for details.
Advisory Panel on Rare Disease Fall 2019 Meeting
September 16, 2019
Click here for details.
IMPACCT: Real World Evidence
September 18-19, 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.
10. Medical Journal Articles
Evidence-Based Medicine: A Data-Driven Approach to Lean Healthcare Operations, click here to view.
Patient Registries: An Underused Resource for Medicines Evaluation: Operational Proposals for Increasing the Use of Patient Registries in Regulatory Assessments, click here to view.
Commonly Used Definitions in Real-World Studies May Underestimate the Prevalence of Renal Disease Among Nonvalvular Atrial Fibrillation Patients, click here to view.
Factors Associated with Evidence-Based Decision-Making Among Patients and Providers, click here to view.
Rethinking Bias and Truth in Evidence-Based Medicine, click here to view.
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.
11. AHRQ Effective Program Updates
Research Protocol: Care Interventions for People With Dementia (PWD) and Their Caregivers, 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.
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: Long-Term Drug Therapy and Drug Holidays for Osteoporosis Fracture Prevention, click here to view.
On July 17, the Partnership to Improve Patient Care (PIPC) and Everylife Foundation for Rare Diseases held a webinar about value assessments, their potential to limit access and innovation for rare disease treatments. The webinar featured Sara van Geertruyden, Executive Director, Partnership to Improve Patient Care; Ari Ne’eman, Disability Advocate, Partnership to Improve Patient Care; Siri Vaeth, Executive Director, Cystic Fibrosis Research, Inc.; and Christina Hartman, Senior Director of Policy and Advocacy, Everylife Foundation for Rare Diseases. The panelists highlighted their concern about the value assessments — like the quality-adjusted life year (QALY) and equal value life-year gained (evLYG) – because the metrics they use treat patients as averages. They noted that cost-effectiveness assessments are particularly concerning when it comes to treatments developed for rare diseases, as they have the potential to impact both patient access and disincentivize investment in rare disease research and development. Click here to learn more and watch the webinar.
2. The Hill: Health Systems Must Help Patients Make Informed Decisions
In an op-ed published in The Hill, the National Health Council's Marc Boutin and the Milken Institute's Tanisha Carino, Ph.D. write that PCORI has made great strides in helping doctors and patients focus on patients' needs and ensure that treatments are in line with patient preferences. They also argue for its continued existence. "In its first decade, PCORI has changed the system for the better. Reauthorization will ensure continued progress toward a health-care system that enables patients to make more informed decisions and achieve the outcomes most meaningful to them," they wrote. Click here to read the article.
3. Op-Ed: ICER's Agenda Helped by Media Attention
Robert Goldberg of the Center for Medicine in the Public Interest wrote that some journalists accept ICER evaluations at face value and fail to ask critical questions, resulting in the public having a skewed view of ICER and its work. "The demonization of small patient groups as somehow being mere mouthpieces for drug companies is consistent with the narrative that pharma funding of organizations who would otherwise have no voice is illegitimate. And it also sidelines a more serious discussion of whether patients believe Exondys work. The fact is, ICER ignores the value of orphan drugs and media coverage of funding enables such willful ignorance," he wrote. Click here to read the article.
4. FH Foundation Publication on Cardiovascular Outcomes
A new study underscores the risk of denying a prescribed treatment and the imperative for patients not to be treated as averages. New research from the FH Foundation highlights that individuals at high risk for cardiovascular events, including those with familial hypercholesterolemia (FH) or atherosclerotic cardiovascular disease (ASCVD), experienced more heart attacks, strokes and other cardiovascular events when they were unable to obtain their prescribed PCSK9 inhibitor, a cholesterol-lowering treatment. The new study, published in Circulation: Cardiovascular Quality and Outcomes, found that high-risk individuals had a 16 percent increased risk of a cardiovascular event during the 11.5-month study period when their PCSK9 inhibitors were rejected by their insurance plan. Click here to read the article.
5. ICER Announces Possible 2020 Assessments
The Institute for Clinical and Economic Research (ICER) published a list of possible assessments for 2020. The possible assessments include drugs to treat cystic fibrosis, breast cancer, multiple sclerosis, and HIV. ICER's QALY-based studies are not transparent, use discriminatory cost-effectiveness measures, and ignore patient preferences and input. Click here to see the list of possible assessments.
6. 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.
7. 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: Patients forced to travel to Russia for experimental MS treatment. Children with cystic fibrosis have lifesaving drugs rejected by the government for being too expensive.
- New Zealand: Breast cancer advocates call for review of drug funding problems.
- Canada: Patients are drawing attention to the government's lack of coverage of cystic fibrosis drugs. Lifesaving SMA drugs continue to not be covered.
- United Kingdom: Patients continue to fight for access to cystic fibrosis drugs. NICE finally funds lifesaving breast cancer drugs after long delays.
8. 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: Research Protocol available. 8/5/2019: Model Analysis Plan. 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 : Draft Evidence Report and Draft Voting Questions available and OPEN for public comment through 8/20/2019. 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.
- Duchenne Muscular Dystrophy: Evidence Presentation available. Meeting 7/25/2019: New England CEPAC deliberated and voted on evidence presented in ICER's report on treatments for Duchenne muscular dystrophy. 8/15/2019: Final Evidence Report and Meeting Summary.
- Unsupported Price Increase Assessment: 10/8/2019: Final Report.
- Acute Migraine: Revised Scoping Document available. 9/6/2019: Research Protocol. 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.
9. Upcoming Events and Webinars
NVHR Hepatitis C Patient Summit
July 29-30, 2019
Click here for details.
Webinar: Real World Evidence and Data: A Tufts Study of 30 Pharma Companies
August 15, 2019
Click here for details.
PCORI Board of Governors Meeting
August 20, 2019
Click here for details.
Webinar: How Frequently Should Cancer Survivors be Screened for Disease Recurrence? Is More Always Better?
August 22, 2019
Click here for details.
Health Spending: Moving from Theory to Action
September 11, 2019
Click here for details.
PCORI Improving Methods Applicant Town Hall
September 12, 2019
Click here for details.
Advisory Panel on Rare Disease Fall 2019 Meeting
September 16, 2019
Click here for details.
IMPACCT: Real World Evidence
September 18-19, 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.
10. Medical Journal Articles
Evidence-Based Medicine: A Data-Driven Approach to Lean Healthcare Operations, click here to view.
Patient Registries: An Underused Resource for Medicines Evaluation: Operational Proposals for Increasing the Use of Patient Registries in Regulatory Assessments, click here to view.
Commonly Used Definitions in Real-World Studies May Underestimate the Prevalence of Renal Disease Among Nonvalvular Atrial Fibrillation Patients, click here to view.
Factors Associated with Evidence-Based Decision-Making Among Patients and Providers, click here to view.
Rethinking Bias and Truth in Evidence-Based Medicine, click here to view.
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.
11. AHRQ Effective Program Updates
Research Protocol: Care Interventions for People With Dementia (PWD) and Their Caregivers, 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.
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: Long-Term Drug Therapy and Drug Holidays for Osteoporosis Fracture Prevention, click here to view.