In This Week’s Issue:
1. 2020 NMQF Virtual Leadership Summit Panel on Value Assessment, see details below.
2. PIPC and EveryLife Foundation for Rare Diseases Webinar on the Use of Value Assessments for Cell and Gene Therapy – Recording Available, click here to view a recording of the webinar.
3. PIPC Submits Comment Letter on ICER Draft Evidence Report for Bladder Cancer Treatments, click here to read the letter.
4. RESCHEDULED -- National Health Council Webinar on Multi-Criteria Decision Analysis, click here to learn more and register.
5. PCORI and RAND to Evaluate Measures of Patient and Stakeholder Engagement, click here to learn more.
6. Value Our Health Map on QALYs Around the World, click here to view the map.
7. ADA 25 Advancing Leadership Forum on Disability Justice in the Fight for Racial Equity, click here to learn more and register.
8. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
9. ICER's QALY-Based Study Topics: Hemophilia A, Sickle Cell Disease, Ulcerative Colitis, Bladder Cancer, Opioid Treatments, High Cholesterol, Anemia in Chronic Kidney Disease, Lupus Nephritis, Multiple Myeloma, Alzheimer's Disease, 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.
1. 2020 NMQF Virtual Leadership Summit Panel on Value Assessment, see details below.
2. PIPC and EveryLife Foundation for Rare Diseases Webinar on the Use of Value Assessments for Cell and Gene Therapy – Recording Available, click here to view a recording of the webinar.
3. PIPC Submits Comment Letter on ICER Draft Evidence Report for Bladder Cancer Treatments, click here to read the letter.
4. RESCHEDULED -- National Health Council Webinar on Multi-Criteria Decision Analysis, click here to learn more and register.
5. PCORI and RAND to Evaluate Measures of Patient and Stakeholder Engagement, click here to learn more.
6. Value Our Health Map on QALYs Around the World, click here to view the map.
7. ADA 25 Advancing Leadership Forum on Disability Justice in the Fight for Racial Equity, click here to learn more and register.
8. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
9. ICER's QALY-Based Study Topics: Hemophilia A, Sickle Cell Disease, Ulcerative Colitis, Bladder Cancer, Opioid Treatments, High Cholesterol, Anemia in Chronic Kidney Disease, Lupus Nephritis, Multiple Myeloma, Alzheimer's Disease, 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.
1. 2020 NMQF Virtual Leadership Summit Panel on Value Assessment
At the 2020 NMQF Summit on Health Disparities and Health Braintrust, Dr. LaTasha Lee moderated a panel discussion on Traditional Value Assessment Methods Exacerbating Health Inequities – a Sickle Cell Disease Case Study. Panelists included Dr. Lakiea Bailey, PhD from Sickle Cell Consortium, treating physician Dr. Alexis Thompson, MD, MPH from Northwestern University Feinberg School of Medicine, Sara van Geertruyden, JD from Partnership to Improve Patient Care (PIPC), and Dr. Irene Agodoa, MD from Global Blood Therapeutics. The panel provided information about the discriminatory implications of cost effectiveness analysis and the quality-adjusted life year (QALY) metric used to assess the “value” of treatments, which largely relies on RCT’s that do not include minority participants nor considers the differential triggers for disease and social determinants of health that are unique to communities of color. Dr. Bailey provided a personal account of her experience as a patient, and provided insights into the assessment of sickle cell disease treatments conducted by the Institute for Clinical and Economic Review (ICER), its implications for limiting access to patients in the real world, and how it missed the mark in capturing the patient experience and the value of treatment for patients that have seen so little innovation in their treatment until now. Click here to view the webinar. Click here to view the research paper on how traditional value assessments exacerbate health inequities authored by NMQF, Sickle Cell Community Consortium, Axis Advocacy, Sick Cells and PIPC.
2. PIPC and EveryLife Foundation for Rare Diseases Webinar on the Use of Value Assessments for Cell and Gene Therapy – Recording Available
On Oct. 8, 2020, Partnership to Improve Patient Care (PIPC) and Everylife Foundation for Rare Diseases hosted a webinar on the findings of the new Health Advances White Paper: Value for Whom? Incorporating Patient Perspectives into Value Assessment for Novel Cell and Gene Therapies. The expert speakers discussed the use of traditional cost-effectiveness analyses to determine the “value” of innovative cell and gene therapies. The speakers concluded there were many shortcomings in using CEA on these novel therapies and discussed new and innovative models that are moving in a better direction and trying to truly determine value to patients. Click here to view a recording of the webinar.
3. PIPC Submits Comment Letter on ICER Draft Evidence Report for Bladder Cancer Treatments
The Partnership to Improve Patient Care (PIPC) submitted a comment letter to the Institute for Clinical and Economic Review (ICER) on its draft evidence report for bladder cancer treatments. PIPC Chairman Tony Coelho criticized ICER for continuing to conduct studies prematurely, as well as assessing the value-based price of these drugs in the absence of sufficient evidence. Chairman Coelho also noted that the mixed data sources for measures of effectiveness in the report are likely to lead to biased estimates in ICER's model. "Bladder cancer can present many challenges to a patient’s quality of life, and there are currently very few treatment options for patients with high-risk non-muscle invasive bladder cancer that is unresponsive to BCG," wrote Chairman Coelho. "It is critical that new treatments are evaluated carefully when there is appropriate available evidence." Click here to read the letter.
4. RESCHEDULED: National Health Council Webinar on Multi-Criteria Decision Analysis
On November 19, 2020, the National Health Council will hold a webinar in tandem with release of a white paper summarizing findings from a Roundtable on Patient-Centered Multi-Criteria Decision Analysis (MCDA). Speakers will introduce MCDA and describe comments, suggestions, and takeaways that emerged during the discussion. Researchers, value assessors, and the patient community agree that conventional approaches to value assessment often inadequately account for the many dimensions of value important to patients. Researchers have identified multi-criteria decision analysis (MCDA) as one promising approach to provide a more comprehensive measure of value. The Webinar will take place at 11:00 a.m. on November 19. Click here to learn more and register.
5. PCORI and RAND to Evaluate Measures of Patient and Stakeholder Engagement
PCORI and the RAND Corporation will collaborate to examine the best ways to systematically measure aspects of engagement to help them identify successful practices for engagement in multi-stakeholder research teams. PCORI seeks to advance the use of engagement measures to ultimately produce information about how to best prepare teams to work with one another, how well stakeholders and researchers are working together, and how meaningful patient and stakeholder engagement is to the planning, conduct, dissemination, and use of health research. The project team has convened two advisory groups comprised of patients, clinicians, researchers, industry leaders, and research funders to guide our work. It is also conducting qualitative research with key informants from nine different stakeholder communities. Click here to read more.
6. Value Our Health Map on QALYs Around the World
Quality-Adjusted Life Years (QALYs) are a number which (theoretically) represents the degree to which a drug or treatment extends life and improves quality of life. The QALY assigns a value to a human’s life on a scale between 0 and 1; 0 = dead and 1 = young and in perfect health. Are you worth treating? Is your treatment worth covering? In many countries, the QALY determines the answer. According to a 2019 report from the National Council on Disability, the use of the QALY in many foreign countries to inform benefits and coverage decisions has limited access to lifesaving medications for people with disabilities and those with chronic illnesses. Foreign reference pricing policies would bring QALYs, and the access barriers that come with them, to the U.S. Value Our Health has a new interactive map on how QALYs value patients around the world. Click here to view the map.
7. ADA 25 Advancing Leadership Forum on Disability Justice in the Fight for Racial Equity
Join ADA 25 Advancing Leadership for a transformative multisession, multiday, forum centering in-depth, action-oriented conversations on the intersections of racial equity and disability justice. The forum, which is already underway, will take place over two more days, October 22 and October 27. Those interested can sign up for one or multiple sessions. This four-part series cements the connection between racial equity and disability justice. Local and nationally recognized social justice activists will share their stories, strategies, and hopes for the future. Click here to learn more and register.
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.
9. ICER's QALY-Based Study Topics: Hemophilia A, Sickle Cell Disease, Ulcerative Colitis, Bladder Cancer, Opioid Treatments, 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.
10. Upcoming Events and Webinars
PCORI Advisory Panel on Clinical Trials Fall 2020 Meeting
November 6, 2020
Click here for details.
An Introduction to Real-World Data & Real-World Evidence: A Virtual Training Series for the Patient Community
November 13, 2020
Click here for details.
11. Medical Journal Articles
Pharmaceutical Pricing Benchmarks: Governmental Versus Private Sector, click here to view.
Researchers, Patients, and Other Stakeholders' Perspectives on Challenges to and Strategies for Engagement, click here to view.
Accounting for US Public Funding in Drug Development: How Can We Better Balance Access, Affordability, and Innovation?, click here to view.
Competencies for Professionals in Health Economics and Outcomes Research: The ISPOR Health Economics and Outcomes Research Competencies Framework, click here to view.
Translating Stakeholder-Driven Comparative Effectiveness Research into Practice: The PCORnet Bariatric Study, click here to view.
Registries for Evaluating Patient Outcomes: A User's Guide: 4th Edition, click here to view.
Replication of Randomized Clinical Trial Results Using Real-World Data: Paving the Way for Effectiveness Decisions, click here to view.
Improving Transparency to Build Trust in Real-World Secondary Data Studies for Hypothesis Testing—Why, What, and How: Recommendations and a Road Map from the Real-World Evidence Transparency Initiative, click here to view.
Measuring and Improving Quality in the US: Where Are We Today?, click here to view.
Potential Impact of Missing Outcome Data on Treatment Effects in Systematic Reviews: Imputation Study, click here to view.
Advancing Community-Engaged Research: Increasing Trustworthiness Within Community-Academic Partnerships, click here to view.
12. AHRQ Effective Program Updates
OPEN FOR COMMENT THROUGH 11/4/2020: Technical Brief: Disparities and Barriers for Pediatric Cancer Survivorship Care. Click here to view.
Rapid Evidence Product: Resource Allocation and Pandemic Response: An Evidence Synthesis To Inform Decision Making. Click here to view.
Systematic Review: Management of Colonic Diverticulitis. Click here to view.
OPEN FOR COMMENT THROUGH 10/23/2020: Key Questions: Diagnostic Errors in the Emergency Department. Click here to view.
No-Touch Modalities for Disinfecting Patient Rooms in Acute Care Settings: A Rapid Review. Click here to view.
OPEN FOR COMMENT THROUGH 10/20/2020: Technical Brief: Interventions To Decrease Hospital Length of Stay. Click here to view.
Systematic Review: Therapies for Clinically Localized Prostate Cancer. Click here to view.
Research Report: Developing Consistent and Useful Quality Improvement Study Data Extraction for Health Systems. Click here to view.
Technical Brief: Strategies for Patient, Family, and Caregiver Engagement. Click here to view.
Research Protocol: Interventions To Decrease Hospital Length of Stay. Click here to view.
At the 2020 NMQF Summit on Health Disparities and Health Braintrust, Dr. LaTasha Lee moderated a panel discussion on Traditional Value Assessment Methods Exacerbating Health Inequities – a Sickle Cell Disease Case Study. Panelists included Dr. Lakiea Bailey, PhD from Sickle Cell Consortium, treating physician Dr. Alexis Thompson, MD, MPH from Northwestern University Feinberg School of Medicine, Sara van Geertruyden, JD from Partnership to Improve Patient Care (PIPC), and Dr. Irene Agodoa, MD from Global Blood Therapeutics. The panel provided information about the discriminatory implications of cost effectiveness analysis and the quality-adjusted life year (QALY) metric used to assess the “value” of treatments, which largely relies on RCT’s that do not include minority participants nor considers the differential triggers for disease and social determinants of health that are unique to communities of color. Dr. Bailey provided a personal account of her experience as a patient, and provided insights into the assessment of sickle cell disease treatments conducted by the Institute for Clinical and Economic Review (ICER), its implications for limiting access to patients in the real world, and how it missed the mark in capturing the patient experience and the value of treatment for patients that have seen so little innovation in their treatment until now. Click here to view the webinar. Click here to view the research paper on how traditional value assessments exacerbate health inequities authored by NMQF, Sickle Cell Community Consortium, Axis Advocacy, Sick Cells and PIPC.
2. PIPC and EveryLife Foundation for Rare Diseases Webinar on the Use of Value Assessments for Cell and Gene Therapy – Recording Available
On Oct. 8, 2020, Partnership to Improve Patient Care (PIPC) and Everylife Foundation for Rare Diseases hosted a webinar on the findings of the new Health Advances White Paper: Value for Whom? Incorporating Patient Perspectives into Value Assessment for Novel Cell and Gene Therapies. The expert speakers discussed the use of traditional cost-effectiveness analyses to determine the “value” of innovative cell and gene therapies. The speakers concluded there were many shortcomings in using CEA on these novel therapies and discussed new and innovative models that are moving in a better direction and trying to truly determine value to patients. Click here to view a recording of the webinar.
3. PIPC Submits Comment Letter on ICER Draft Evidence Report for Bladder Cancer Treatments
The Partnership to Improve Patient Care (PIPC) submitted a comment letter to the Institute for Clinical and Economic Review (ICER) on its draft evidence report for bladder cancer treatments. PIPC Chairman Tony Coelho criticized ICER for continuing to conduct studies prematurely, as well as assessing the value-based price of these drugs in the absence of sufficient evidence. Chairman Coelho also noted that the mixed data sources for measures of effectiveness in the report are likely to lead to biased estimates in ICER's model. "Bladder cancer can present many challenges to a patient’s quality of life, and there are currently very few treatment options for patients with high-risk non-muscle invasive bladder cancer that is unresponsive to BCG," wrote Chairman Coelho. "It is critical that new treatments are evaluated carefully when there is appropriate available evidence." Click here to read the letter.
4. RESCHEDULED: National Health Council Webinar on Multi-Criteria Decision Analysis
On November 19, 2020, the National Health Council will hold a webinar in tandem with release of a white paper summarizing findings from a Roundtable on Patient-Centered Multi-Criteria Decision Analysis (MCDA). Speakers will introduce MCDA and describe comments, suggestions, and takeaways that emerged during the discussion. Researchers, value assessors, and the patient community agree that conventional approaches to value assessment often inadequately account for the many dimensions of value important to patients. Researchers have identified multi-criteria decision analysis (MCDA) as one promising approach to provide a more comprehensive measure of value. The Webinar will take place at 11:00 a.m. on November 19. Click here to learn more and register.
5. PCORI and RAND to Evaluate Measures of Patient and Stakeholder Engagement
PCORI and the RAND Corporation will collaborate to examine the best ways to systematically measure aspects of engagement to help them identify successful practices for engagement in multi-stakeholder research teams. PCORI seeks to advance the use of engagement measures to ultimately produce information about how to best prepare teams to work with one another, how well stakeholders and researchers are working together, and how meaningful patient and stakeholder engagement is to the planning, conduct, dissemination, and use of health research. The project team has convened two advisory groups comprised of patients, clinicians, researchers, industry leaders, and research funders to guide our work. It is also conducting qualitative research with key informants from nine different stakeholder communities. Click here to read more.
6. Value Our Health Map on QALYs Around the World
Quality-Adjusted Life Years (QALYs) are a number which (theoretically) represents the degree to which a drug or treatment extends life and improves quality of life. The QALY assigns a value to a human’s life on a scale between 0 and 1; 0 = dead and 1 = young and in perfect health. Are you worth treating? Is your treatment worth covering? In many countries, the QALY determines the answer. According to a 2019 report from the National Council on Disability, the use of the QALY in many foreign countries to inform benefits and coverage decisions has limited access to lifesaving medications for people with disabilities and those with chronic illnesses. Foreign reference pricing policies would bring QALYs, and the access barriers that come with them, to the U.S. Value Our Health has a new interactive map on how QALYs value patients around the world. Click here to view the map.
7. ADA 25 Advancing Leadership Forum on Disability Justice in the Fight for Racial Equity
Join ADA 25 Advancing Leadership for a transformative multisession, multiday, forum centering in-depth, action-oriented conversations on the intersections of racial equity and disability justice. The forum, which is already underway, will take place over two more days, October 22 and October 27. Those interested can sign up for one or multiple sessions. This four-part series cements the connection between racial equity and disability justice. Local and nationally recognized social justice activists will share their stories, strategies, and hopes for the future. Click here to learn more and register.
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.
- Canada: Sisters undergo time-consuming and trying treatments for cystic fibrosis, and accessing a life-changing drug could give them a new normal.
- New Zealand: New Zealand uses old treatments for diabetes and modern drugs remain unavailable. Pharmac refuses to fund drug that could save 17-year-old with spinal muscular atrophy.
9. ICER's QALY-Based Study Topics: Hemophilia A, Sickle Cell Disease, Ulcerative Colitis, Bladder Cancer, Opioid Treatments, 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.
- Ulcerative Colitis: Final Policy Recommendations and Final Evidence Report AVAILABLE.
- Sickle Cell Disease: Evidence Report and Responses to Comments AVAILABLE. Meeting POSTPONED: New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for sickle cell disease.
- Hemophilia A: Evidence Report AVAILABLE. 11/20/2020: Final Evidence Report. Meeting 10/30/2020: New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for hemophilia.
- Bladder Cancer: Draft Evidence Report available. 11/6/2020: Evidence Report and response to public comments. Meeting 11/20/2020: Midwest CEPAC will convene virtually to deliberate and vote on evidence presented in ICER's report on digital therapeutics for bladder cancer.
- Opioids: Digital Apps: Draft Evidence Report available. 11/6/2020: Revised Evidence Report and Responses to Public Comments. Meeting 11/18/2020: Midwest CEPAC will convene virtually to deliberate and vote on evidence presented in ICER's report on digital therapeutics for opioid use disorder (OUD).
- Opioids: Supervised Injection Centers: Draft Evidence Report AVAILABLE, comment period OPEN through 10/22/2020. Meeting 12/3/2020: The New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on supervised injection facilities.
- High Cholesterol: Model Analysis Plan available. 11/12/2020: Draft Evidence Report.
- Anemia in Chronic Kidney Disease: Model Analysis Plan available. 11/30/2020: Draft Evidence Report.
- Unsupported Price Increase Assessment: 1/8/2021: Final Assessment and Report.
- Lupus Nephritis: Research Protocol available. 11/20/2020: Model Analysis Plan. 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 Scoping Document available. 10/21/2020: Revised Scoping Document.
- Service Dogs for PTSD: 11/30/2020: Final Assessment and Report.
- Alzheimer's Disease: 10/21/2020: Draft Scoping Document. Meeting 5/7/2020: CTAF will convene virtually to deliberate and vote on evidence presented in ICER's report on Alzheimer's disease.
10. Upcoming Events and Webinars
PCORI Advisory Panel on Clinical Trials Fall 2020 Meeting
November 6, 2020
Click here for details.
An Introduction to Real-World Data & Real-World Evidence: A Virtual Training Series for the Patient Community
November 13, 2020
Click here for details.
11. Medical Journal Articles
Pharmaceutical Pricing Benchmarks: Governmental Versus Private Sector, click here to view.
Researchers, Patients, and Other Stakeholders' Perspectives on Challenges to and Strategies for Engagement, click here to view.
Accounting for US Public Funding in Drug Development: How Can We Better Balance Access, Affordability, and Innovation?, click here to view.
Competencies for Professionals in Health Economics and Outcomes Research: The ISPOR Health Economics and Outcomes Research Competencies Framework, click here to view.
Translating Stakeholder-Driven Comparative Effectiveness Research into Practice: The PCORnet Bariatric Study, click here to view.
Registries for Evaluating Patient Outcomes: A User's Guide: 4th Edition, click here to view.
Replication of Randomized Clinical Trial Results Using Real-World Data: Paving the Way for Effectiveness Decisions, click here to view.
Improving Transparency to Build Trust in Real-World Secondary Data Studies for Hypothesis Testing—Why, What, and How: Recommendations and a Road Map from the Real-World Evidence Transparency Initiative, click here to view.
Measuring and Improving Quality in the US: Where Are We Today?, click here to view.
Potential Impact of Missing Outcome Data on Treatment Effects in Systematic Reviews: Imputation Study, click here to view.
Advancing Community-Engaged Research: Increasing Trustworthiness Within Community-Academic Partnerships, click here to view.
12. AHRQ Effective Program Updates
OPEN FOR COMMENT THROUGH 11/4/2020: Technical Brief: Disparities and Barriers for Pediatric Cancer Survivorship Care. Click here to view.
Rapid Evidence Product: Resource Allocation and Pandemic Response: An Evidence Synthesis To Inform Decision Making. Click here to view.
Systematic Review: Management of Colonic Diverticulitis. Click here to view.
OPEN FOR COMMENT THROUGH 10/23/2020: Key Questions: Diagnostic Errors in the Emergency Department. Click here to view.
No-Touch Modalities for Disinfecting Patient Rooms in Acute Care Settings: A Rapid Review. Click here to view.
OPEN FOR COMMENT THROUGH 10/20/2020: Technical Brief: Interventions To Decrease Hospital Length of Stay. Click here to view.
Systematic Review: Therapies for Clinically Localized Prostate Cancer. Click here to view.
Research Report: Developing Consistent and Useful Quality Improvement Study Data Extraction for Health Systems. Click here to view.
Technical Brief: Strategies for Patient, Family, and Caregiver Engagement. Click here to view.
Research Protocol: Interventions To Decrease Hospital Length of Stay. Click here to view.