In This Week’s Issue:
1. PIPC Webinar on the Use of Value Assessments for Cell and Gene Therapy, see details below.
2. Sickle Cell Disease and Traditional Value Assessment Discussion at the 2020 NMQF Summit on Health Disparities, see details below.
3. Value Our Health Map on QALYs Around the World, click here to view the map.
4. PCORI Announces New Board of Governors Members, click here to read the press release.
5. PCORI Proposed Principles for the Consideration of the Full Range of Outcomes Data and Webinar Series, see details below.
6. SWHR Migraine Patient Toolkit: Living Well with Migraine, click here to view the toolkit.
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. PIPC Webinar on the Use of Value Assessments for Cell and Gene Therapy, see details below.
2. Sickle Cell Disease and Traditional Value Assessment Discussion at the 2020 NMQF Summit on Health Disparities, see details below.
3. Value Our Health Map on QALYs Around the World, click here to view the map.
4. PCORI Announces New Board of Governors Members, click here to read the press release.
5. PCORI Proposed Principles for the Consideration of the Full Range of Outcomes Data and Webinar Series, see details below.
6. SWHR Migraine Patient Toolkit: Living Well with Migraine, click here to view the toolkit.
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. PIPC Webinar on the Use of Value Assessments for Cell and Gene Therapy
Please join the Partnership to Improve Patient Care (PIPC) and the EveryLife Foundation for Rare Diseases for a webinar on Thursday, October 8 at 2pm ET highlighting 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 webinar will explore the topic of the use of traditional cost-effectiveness analyses to determine the “value” of innovative cell and gene therapies and explore some new and innovative models that are moving in a better direction and trying to truly determine value to patients. Click here to read the white paper. Please RSVP for the webinar to [email protected].
2. Sickle Cell Disease and Traditional Value Assessment Discussion at the 2020 NMQF Summit on Health Disparities
This Tuesday, October 6 at 10:00 a.m. Eastern, The National Minority Quality Forum (NMQF) will hold a discussion with health care experts to discuss how traditional value assessment methods exacerbate health inequities. The panel will use sickle cell disease as a case study. Panelists include PIPC Executive Director Sara van Geertruyden and Lakiea Bailey, Ph.D., the Executive Director of the Sickle Cell Consortium. Click here to learn more and watch the session.
3. 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.
4. PCORI Announces New Board of Governors Members
The Comptroller General of the United States announced seven new appointments to PCORI's Board of Governors, including Danny van Leeuwen, the Founder and Principal of Health Hats. Mr. van Leeuwen was appointed to fill a seat on the Board of Governors that is reserved for patients. PIPC offers its heartfelt congratulations to Mr. van Leeuwen and looks forward to his tenure representing patients on PCORI's Board of Governors. PCORI’s Board is made up of 19-21 representatives of the patient, health care consumer, clinical, health insurance, employer, research, industry, policy and other health care sectors, plus the directors of the National Institutes of Health and Agency for Healthcare Research and Quality. Click here to read the press release.
5. PCORI Proposed Principles for the Consideration of the Full Range of Outcomes Data and Webinar Series
At its September 2020 meeting, PCORI’s Board of Governors approved the release of the proposed Principles for the Consideration of the Full Range of Outcomes Data for public comment. This is one component in PCORI’s plan to implement its new statutory mandate to consider the full range of outcomes data in PCORI-funded research.
These principles will serve as a point of reference for PCORI, and as a basis for developing future guidance to potential applicants for PCORI funding on what is included in “the full range of clinical and patient-center outcomes relevant to, and that meet the needs of, patients, clinicians, purchasers, and policy-makers” consistent with its authorizing law. Comment will be open through November 13, 2020. Click here to read the principles and to submit comments.
During the comment period, PCORI will hold two panels highlighting perspectives on the new Principles. On October 5 from 2:30-4:00 p.m. Eastern, PCORI will hold a webinar focusing on patients, caregivers, and consumers. The following day, October 6 from 2:30-4:00 p.m. Eastern, PCORI will hold a webinar focusing on payer, provider, health system, and pharmaceutical industry perspectives.
6. SWHR Migraine Patient Toolkit: Living Well with Migraine
The Society for Women's Health Research released a new toolkit focused on helping those with migraine, their caregivers, and their health providers to facilitate the journey towards wellness. Vetted by migraine experts and patients, SWHR’s Living Well with Migraine toolkit offers a starting point for those at the beginning of their wellness journey as well as important information for patients who may already be working to improve their quality of life in the face of migraine. Click here to view the toolkit.
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 three more days, October 14, 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 Webinar Series: Considering the Full Range of Outcomes in PCORI Research -- Patients, Caregivers and Consumers
October 5, 2020
Click here for details.
Real World Data Mapping – Identifying and Navigating Data Sources Globally
October 5, 2020
Click here for details.
PCORI: New Evidence to Inform Decisions
October 16, 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
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.
Patient and Other Stakeholder Engagement in Patient-Centered Outcomes Research Institute Funded Studies of Patients with Kidney Diseases, click here to view.
Patient-Reported Outcomes: Central to the Management of COVID-19, click here to view.
How to Include Patient-Reported Outcome Measures in Clinical Trials, click here to view.
12. AHRQ Effective Program Updates
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.
Rapid Evidence Product: Retention Strategies for Medications for Addiction Treatment in Adults With Opioid Use Disorder. Click here to view.
Please join the Partnership to Improve Patient Care (PIPC) and the EveryLife Foundation for Rare Diseases for a webinar on Thursday, October 8 at 2pm ET highlighting 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 webinar will explore the topic of the use of traditional cost-effectiveness analyses to determine the “value” of innovative cell and gene therapies and explore some new and innovative models that are moving in a better direction and trying to truly determine value to patients. Click here to read the white paper. Please RSVP for the webinar to [email protected].
2. Sickle Cell Disease and Traditional Value Assessment Discussion at the 2020 NMQF Summit on Health Disparities
This Tuesday, October 6 at 10:00 a.m. Eastern, The National Minority Quality Forum (NMQF) will hold a discussion with health care experts to discuss how traditional value assessment methods exacerbate health inequities. The panel will use sickle cell disease as a case study. Panelists include PIPC Executive Director Sara van Geertruyden and Lakiea Bailey, Ph.D., the Executive Director of the Sickle Cell Consortium. Click here to learn more and watch the session.
3. 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.
4. PCORI Announces New Board of Governors Members
The Comptroller General of the United States announced seven new appointments to PCORI's Board of Governors, including Danny van Leeuwen, the Founder and Principal of Health Hats. Mr. van Leeuwen was appointed to fill a seat on the Board of Governors that is reserved for patients. PIPC offers its heartfelt congratulations to Mr. van Leeuwen and looks forward to his tenure representing patients on PCORI's Board of Governors. PCORI’s Board is made up of 19-21 representatives of the patient, health care consumer, clinical, health insurance, employer, research, industry, policy and other health care sectors, plus the directors of the National Institutes of Health and Agency for Healthcare Research and Quality. Click here to read the press release.
5. PCORI Proposed Principles for the Consideration of the Full Range of Outcomes Data and Webinar Series
At its September 2020 meeting, PCORI’s Board of Governors approved the release of the proposed Principles for the Consideration of the Full Range of Outcomes Data for public comment. This is one component in PCORI’s plan to implement its new statutory mandate to consider the full range of outcomes data in PCORI-funded research.
These principles will serve as a point of reference for PCORI, and as a basis for developing future guidance to potential applicants for PCORI funding on what is included in “the full range of clinical and patient-center outcomes relevant to, and that meet the needs of, patients, clinicians, purchasers, and policy-makers” consistent with its authorizing law. Comment will be open through November 13, 2020. Click here to read the principles and to submit comments.
During the comment period, PCORI will hold two panels highlighting perspectives on the new Principles. On October 5 from 2:30-4:00 p.m. Eastern, PCORI will hold a webinar focusing on patients, caregivers, and consumers. The following day, October 6 from 2:30-4:00 p.m. Eastern, PCORI will hold a webinar focusing on payer, provider, health system, and pharmaceutical industry perspectives.
6. SWHR Migraine Patient Toolkit: Living Well with Migraine
The Society for Women's Health Research released a new toolkit focused on helping those with migraine, their caregivers, and their health providers to facilitate the journey towards wellness. Vetted by migraine experts and patients, SWHR’s Living Well with Migraine toolkit offers a starting point for those at the beginning of their wellness journey as well as important information for patients who may already be working to improve their quality of life in the face of migraine. Click here to view the toolkit.
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 three more days, October 14, 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.
- Australia: Government refuses to provide subsidy for revolutionary new medicine that shrank an eight-year-old's brain stem tumor in just days.
- United Kingdom: A new type 2 diabetes drug could stop kidney failure but Pharmac refuses to fund it.
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: Revised Voting Questions and Evidence Report AVAILABLE. 10/16/2020: Final Policy Recommendations and Final Evidence Report.
- 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: 10/16/2020: 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. Comment period OPEN through 10/15/2020. 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, comment period OPEN through 10/15/2020. 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: Research Protocol available. 10/15/2020: Model Analysis Plan.
- Unsupported Price Increase Assessment: 1/8/2021: Final Assessment and Report.
- Lupus Nephritis: Revised Scoping Document available. 10/6/2020: Research Protocol. 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, comment period OPEN through 10/13/2020.
- Service Dogs for PTSD: 11/30/2020: Final Assessment and Report.
- Alzheimer's Disease: Open Input Period through 10/16/2020. 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 Webinar Series: Considering the Full Range of Outcomes in PCORI Research -- Patients, Caregivers and Consumers
October 5, 2020
Click here for details.
Real World Data Mapping – Identifying and Navigating Data Sources Globally
October 5, 2020
Click here for details.
PCORI: New Evidence to Inform Decisions
October 16, 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
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.
Patient and Other Stakeholder Engagement in Patient-Centered Outcomes Research Institute Funded Studies of Patients with Kidney Diseases, click here to view.
Patient-Reported Outcomes: Central to the Management of COVID-19, click here to view.
How to Include Patient-Reported Outcome Measures in Clinical Trials, click here to view.
12. AHRQ Effective Program Updates
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.
Rapid Evidence Product: Retention Strategies for Medications for Addiction Treatment in Adults With Opioid Use Disorder. Click here to view.
