In This Week’s Issue:
1. Most Favored Nation’s Executive Order Released Yesterday Would Import Discriminatory QALYs, see details below.
2. The Bonnell Foundation Discusses Access Challenges for Cystic Fibrosis Patients Abroad, click here to listen to the podcast.
3. NMQF, Axis Advocacy, Sick Cells, and PIPC Release a White Paper on Value Assessments' Impact on Health Inequities, click here to read the paper.
4. AAPD, NCD, and NCIL Caution About the Discriminatory Implications of the QALY and Foreign Reference Pricing, click here to view the webinar.
5. IVI Unveils New Major Depressive Disorder Value Assessment Initiative, click here to read more.
6. Advocates Call for Focus on People with Disabilities in Vaccine Allocation Framework, see details below.
7. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
8. PCORI Annual Meeting September 16-17, click here to learn more and to register.
9. ICER's QALY-Based Study Topics: Hemophilia A, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, Bladder Cancer, Opioid Treatments, High Cholesterol, Anemia in Chronic Kidney Disease, Lupus Nephritis, Multiple Myeloma, 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. Most Favored Nation’s Executive Order Released Yesterday Would Import Discriminatory QALYs, see details below.
2. The Bonnell Foundation Discusses Access Challenges for Cystic Fibrosis Patients Abroad, click here to listen to the podcast.
3. NMQF, Axis Advocacy, Sick Cells, and PIPC Release a White Paper on Value Assessments' Impact on Health Inequities, click here to read the paper.
4. AAPD, NCD, and NCIL Caution About the Discriminatory Implications of the QALY and Foreign Reference Pricing, click here to view the webinar.
5. IVI Unveils New Major Depressive Disorder Value Assessment Initiative, click here to read more.
6. Advocates Call for Focus on People with Disabilities in Vaccine Allocation Framework, see details below.
7. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
8. PCORI Annual Meeting September 16-17, click here to learn more and to register.
9. ICER's QALY-Based Study Topics: Hemophilia A, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, Bladder Cancer, Opioid Treatments, High Cholesterol, Anemia in Chronic Kidney Disease, Lupus Nephritis, Multiple Myeloma, 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. Most Favored Nation’s Executive Order Released Yesterday Would Import Discriminatory QALYs
The Trump administration released the text of an Executive Order allowing the Department of Health and Human Services (HHS) to rely on foreign governments to set prices for drugs covered under Medicare, regardless of the metrics used by those countries or implications for access to care for patients. The Partnership to Improve Patient Care (PIPC) released a statement expressing strong opposition to this concept, as it will have the impact of limiting and delaying access to needed treatments for Americans.
PIPC Chairman Tony Coelho along with leading patient and disability groups have long advocated against this type of discriminatory policy. Click here to read a February blog post from Chairman Coelho on the topic. The National Council on Disability (NCD) released a statement upon first news of this Executive Order in July calling out the harms of referencing international pricing -- which relies on the use of discriminatory measures like QALYs. Click here to read the statement.
2. The Bonnell Foundation Discusses Access Challenges for Cystic Fibrosis Patients Abroad
Laura Bonnell, executive director of the Bonnell Foundation and mother of two daughters with Cystic Fibrosis, hosted a podcast interviewing Beth Vanstone of Get Loud for CF, a Canadian mother of a daughter with Cystic Fibrosis. They discussed Beth’s long and arduous process to get access to novel CF therapies that are not covered in Canada for her daughter, Maddy. Laura expressed concern as to what modeling the pricing policies of Canada and other countries that rely on the QALY could mean for American patients in the context of the Most Favored Nations Executive Order. They also hit on a wide range of topics impacting CF families around the world. Click here to listen to the podcast.
3. NMQF, Axis Advocacy, Sick Cells, and PIPC Release a White Paper on Value Assessments' Impact on Health Inequities
National Minority Quality Forum, Axis Advocacy, Sick Cells, and Partnership to Improve Patient Care released a white paper investigating the limitations of applying traditional value assessments to communities of color. The paper finds that the pervasive issues that concern the patient and disability communities about value assessments, like over reliance on averages and the use of the Quality-Adjusted Life Year, become even more concerning when assessing treatments for conditions that disproportionately impact communities of color. If relied on in coverage decisions, these assessments could have the effect of exacerbating health inequities. Click here to read the paper.
4. AAPD, NCD, and NCIL Caution About the Discriminatory Implications of the QALY and Foreign Reference Pricing
The American Association of People with Disabilities (AAPD) released a webinar featuring an interview between Maria Town, the President/CEO of AAPD, and Ana Torres Davis, Senior Attorney for the National Council on Disability (NCD), as well as a presentation by Kelly Buckland, Executive Director of the National Council on Independent Living (NCIL), about dangers of quality-adjusted life years (QALYs) for discrimination against people with disabilities and their importation from foreign countries into Medicare. The webinar focused on the National Council on Disability’s 2019 report that recommended that the administration rescind a proposal to use an international pricing index in Medicare and strongly advocated against President Trump’s proposed Executive Order due to its implications for importing discrimination and restricted access to care for people with disabilities. Click here to view the webinar.
5. IVI Unveils New Major Depressive Disorder Value Assessment Initiative
The Innovation and Value Initiative (IVI) recently announced a multi-year initiative to build and test an open-source value assessment model to facilitate evaluation of health interventions indicated for major depressive disorder (MDD). IVI’s MDD model will feature both cost-effectiveness analysis and the Multi-Criteria Decision Analysis. The model is designed to be a lab to test and optimize approaches to incorporate novel value elements and methods. Both pharmacologic and non-pharmacologic treatments will be evaluated. Click here to read more about the model.
6. Advocates Call for Focus on People with Disabilities in Vaccine Allocation Framework
Last week, the National Academies of Sciences, Engineering, and Medicine published a discussion draft of its Preliminary Framework for an Equitable Allocation of COVID-19 Vaccine. Stakeholders had just four days to comment on the discussion draft, with comments closing on Friday, September 4. Several disability groups commented, calling for a greater focus on people living with disabilities to ensure that they are not discriminated against when a COVID-19 vaccine is available. The Association of University Centers on Disabilities (AUCD) strongly recommended to include individuals with disabilities and the array of direct care professionals – paid and unpaid – who serve them in the first phase allocation of COVID-19 vaccine. The Consortium for Citizens with Disabilities (CCD) also provided comments stating that people who live or work in congregate settings should receive priority in allocation of a vaccine and expressed concerns about the need for more input related to health disparities. Not Dead Yet further stated that people with disabilities who faced potential COVID-19 triage should be prioritized. Click here to read the AUCD letter. Click here to view the CCD letter which will be posted later today. Click here to view the Not Dead Yet letter.
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. PCORI Annual Meeting September 16-17
Make plans now to attend PCORI's sixth Annual Meeting, Accelerating Impact on Care and Patient-Centered Outcomes, being held virtually September 16-17. The meeting will highlight results from several PCORI-funded studies and explore how we can shorten the time from research to improved health for patients. During the meeting, attendees will:
Click here to learn more and to register.
9. ICER's QALY-Based Study Topics: Hemophilia A, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, Bladder Cancer, Opioid Treatments, High Cholesterol, Anemia in Chronic Kidney Disease, Lupus Nephritis, Multiple Myeloma
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 Board of Governors Meeting
September 14-15, 2020
Click here for details.
2020 PCORI Annual Meeting (A Virtual Event): Accelerating Impact on Care and Patient Outcomes
September 16-17, 2020
Click here for details.
Cayenne Wellness Center and Axis Advocacy -- 12th Annual Sickle Cell Disease Educational Seminar
September 17-19, 2020
Click here for details.
STAT News: Getting Real World Data about Covid-19
September 17, 2020
Click here for details.
SWHR Virtual Panel on Uterine Fibroids
September 22, 2020
Click here for details.
PCORI: New Evidence to Inform Decisions
October 16, 2020
Click here for details.
11. Medical Journal Articles
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.
Evaluation of the Use of Cancer Registry Data for Comparative Effectiveness Research, click here to view.
Outcome-Based Payment Schemes: What Outcomes Do Patients with Cancer Value?, click here to view.
Engaging Patients and Stakeholders in Preresearch: Findings from the Pipeline to Proposal Awards Initiative, click here to view.
Why Clinical Trials May Not Help Patients Make Treatment Decisions: Results from Focus Group Discussions with 22 Patients, click here to view.
Defining Patient Engagement in Research: Results of a Systematic Review and Analysis: Report of the ISPOR Patient-Centered Special Interest Group, click here to view.
12. AHRQ Effective Program Updates
OPEN FOR COMMENT THROUGH 9/28/2020: Systematic Review: Acute Treatments for Episodic Migraine. Click here to view.
OPEN FOR COMMENT THROUGH 9/28/2020: Systematic Review: Treatments for Acute Pain. Click here to view.
OPEN FOR COMMENT THROUGH 9/16/2020: Key Questions: Transitions of Care from Pediatric to Adult Services for Children with Special Healthcare Needs. 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.
OPEN FOR COMMENT THROUGH 9/28/2020: Systematic Review: Cervical Ripening in the Outpatient Setting. Click here to view.
OPEN FOR COMMENT THROUGH 9/14/2020: Systematic Review: Radiation Therapy for Brain Metastases. Click here to view.
OPEN FOR COMMENT THROUGH 9/18/2020: Systematic Review: Integrating Palliative Care in Ambulatory Care of Non-Cancer Serious Chronic Illness. Click here to view.
The Trump administration released the text of an Executive Order allowing the Department of Health and Human Services (HHS) to rely on foreign governments to set prices for drugs covered under Medicare, regardless of the metrics used by those countries or implications for access to care for patients. The Partnership to Improve Patient Care (PIPC) released a statement expressing strong opposition to this concept, as it will have the impact of limiting and delaying access to needed treatments for Americans.
PIPC Chairman Tony Coelho along with leading patient and disability groups have long advocated against this type of discriminatory policy. Click here to read a February blog post from Chairman Coelho on the topic. The National Council on Disability (NCD) released a statement upon first news of this Executive Order in July calling out the harms of referencing international pricing -- which relies on the use of discriminatory measures like QALYs. Click here to read the statement.
2. The Bonnell Foundation Discusses Access Challenges for Cystic Fibrosis Patients Abroad
Laura Bonnell, executive director of the Bonnell Foundation and mother of two daughters with Cystic Fibrosis, hosted a podcast interviewing Beth Vanstone of Get Loud for CF, a Canadian mother of a daughter with Cystic Fibrosis. They discussed Beth’s long and arduous process to get access to novel CF therapies that are not covered in Canada for her daughter, Maddy. Laura expressed concern as to what modeling the pricing policies of Canada and other countries that rely on the QALY could mean for American patients in the context of the Most Favored Nations Executive Order. They also hit on a wide range of topics impacting CF families around the world. Click here to listen to the podcast.
3. NMQF, Axis Advocacy, Sick Cells, and PIPC Release a White Paper on Value Assessments' Impact on Health Inequities
National Minority Quality Forum, Axis Advocacy, Sick Cells, and Partnership to Improve Patient Care released a white paper investigating the limitations of applying traditional value assessments to communities of color. The paper finds that the pervasive issues that concern the patient and disability communities about value assessments, like over reliance on averages and the use of the Quality-Adjusted Life Year, become even more concerning when assessing treatments for conditions that disproportionately impact communities of color. If relied on in coverage decisions, these assessments could have the effect of exacerbating health inequities. Click here to read the paper.
4. AAPD, NCD, and NCIL Caution About the Discriminatory Implications of the QALY and Foreign Reference Pricing
The American Association of People with Disabilities (AAPD) released a webinar featuring an interview between Maria Town, the President/CEO of AAPD, and Ana Torres Davis, Senior Attorney for the National Council on Disability (NCD), as well as a presentation by Kelly Buckland, Executive Director of the National Council on Independent Living (NCIL), about dangers of quality-adjusted life years (QALYs) for discrimination against people with disabilities and their importation from foreign countries into Medicare. The webinar focused on the National Council on Disability’s 2019 report that recommended that the administration rescind a proposal to use an international pricing index in Medicare and strongly advocated against President Trump’s proposed Executive Order due to its implications for importing discrimination and restricted access to care for people with disabilities. Click here to view the webinar.
5. IVI Unveils New Major Depressive Disorder Value Assessment Initiative
The Innovation and Value Initiative (IVI) recently announced a multi-year initiative to build and test an open-source value assessment model to facilitate evaluation of health interventions indicated for major depressive disorder (MDD). IVI’s MDD model will feature both cost-effectiveness analysis and the Multi-Criteria Decision Analysis. The model is designed to be a lab to test and optimize approaches to incorporate novel value elements and methods. Both pharmacologic and non-pharmacologic treatments will be evaluated. Click here to read more about the model.
6. Advocates Call for Focus on People with Disabilities in Vaccine Allocation Framework
Last week, the National Academies of Sciences, Engineering, and Medicine published a discussion draft of its Preliminary Framework for an Equitable Allocation of COVID-19 Vaccine. Stakeholders had just four days to comment on the discussion draft, with comments closing on Friday, September 4. Several disability groups commented, calling for a greater focus on people living with disabilities to ensure that they are not discriminated against when a COVID-19 vaccine is available. The Association of University Centers on Disabilities (AUCD) strongly recommended to include individuals with disabilities and the array of direct care professionals – paid and unpaid – who serve them in the first phase allocation of COVID-19 vaccine. The Consortium for Citizens with Disabilities (CCD) also provided comments stating that people who live or work in congregate settings should receive priority in allocation of a vaccine and expressed concerns about the need for more input related to health disparities. Not Dead Yet further stated that people with disabilities who faced potential COVID-19 triage should be prioritized. Click here to read the AUCD letter. Click here to view the CCD letter which will be posted later today. Click here to view the Not Dead Yet letter.
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.
- Canada: Canadian teen fights for reform to drug access processes. Woman with cystic fibrosis talks of "heartbreaking" fight to get government to provide access to life-changing medication.
- United Kingdom: UK cystic fibrosis sufferer only alive because drug manufacturer gave her life-saving medicine on compassionate grounds, as government continues to refuse to fund it.
8. PCORI Annual Meeting September 16-17
Make plans now to attend PCORI's sixth Annual Meeting, Accelerating Impact on Care and Patient-Centered Outcomes, being held virtually September 16-17. The meeting will highlight results from several PCORI-funded studies and explore how we can shorten the time from research to improved health for patients. During the meeting, attendees will:
- Learn about our efforts to disseminate research findings
- Network with others
- Examine PCORI’s emphasis on engagement
- Explore participation in developing our research agenda
- Listen to powerful, inspiring keynote speakers
Click here to learn more and to register.
9. ICER's QALY-Based Study Topics: Hemophilia A, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, Bladder Cancer, Opioid Treatments, High Cholesterol, Anemia in Chronic Kidney Disease, Lupus Nephritis, Multiple Myeloma
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. Meeting 9/24/2020: CTAF will convene to deliberate and vote on evidence presented in ICER's report on ulcerative colitis therapies.
- Cystic Fibrosis: CTAF convened 8/27/2020 to deliberate and vote on evidence presented in ICER's report on treatments for cystic fibrosis. 9/23/2020: 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: Draft Evidence Report AVAILABLE. Comment period OPEN through 9/23/2020.
- Bladder Cancer: Model Analysis Plan available. 9/17/2020: Draft Evidence Report.
- Opioids: Digital Apps: Model Analysis Plan available. 9/17/2020: Draft Evidence Report.
- Opioids: Supervised Injection Centers: Model Analysis Plan available. 9/24/2020: Draft Evidence Report.
- High Cholesterol: Research Protocol available. 9/24/2020: Model Analysis Plan.
- 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: Draft Scoping Document AVAILABLE, Comment Period OPEN through 9/21/2020, 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: Open Input Period through 9/17/2020.
- Service Dogs for PTSD: Open Input Period through 10/2/2020.
10. Upcoming Events and Webinars
PCORI Board of Governors Meeting
September 14-15, 2020
Click here for details.
2020 PCORI Annual Meeting (A Virtual Event): Accelerating Impact on Care and Patient Outcomes
September 16-17, 2020
Click here for details.
Cayenne Wellness Center and Axis Advocacy -- 12th Annual Sickle Cell Disease Educational Seminar
September 17-19, 2020
Click here for details.
STAT News: Getting Real World Data about Covid-19
September 17, 2020
Click here for details.
SWHR Virtual Panel on Uterine Fibroids
September 22, 2020
Click here for details.
PCORI: New Evidence to Inform Decisions
October 16, 2020
Click here for details.
11. Medical Journal Articles
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.
Evaluation of the Use of Cancer Registry Data for Comparative Effectiveness Research, click here to view.
Outcome-Based Payment Schemes: What Outcomes Do Patients with Cancer Value?, click here to view.
Engaging Patients and Stakeholders in Preresearch: Findings from the Pipeline to Proposal Awards Initiative, click here to view.
Why Clinical Trials May Not Help Patients Make Treatment Decisions: Results from Focus Group Discussions with 22 Patients, click here to view.
Defining Patient Engagement in Research: Results of a Systematic Review and Analysis: Report of the ISPOR Patient-Centered Special Interest Group, click here to view.
12. AHRQ Effective Program Updates
OPEN FOR COMMENT THROUGH 9/28/2020: Systematic Review: Acute Treatments for Episodic Migraine. Click here to view.
OPEN FOR COMMENT THROUGH 9/28/2020: Systematic Review: Treatments for Acute Pain. Click here to view.
OPEN FOR COMMENT THROUGH 9/16/2020: Key Questions: Transitions of Care from Pediatric to Adult Services for Children with Special Healthcare Needs. 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.
OPEN FOR COMMENT THROUGH 9/28/2020: Systematic Review: Cervical Ripening in the Outpatient Setting. Click here to view.
OPEN FOR COMMENT THROUGH 9/14/2020: Systematic Review: Radiation Therapy for Brain Metastases. Click here to view.
OPEN FOR COMMENT THROUGH 9/18/2020: Systematic Review: Integrating Palliative Care in Ambulatory Care of Non-Cancer Serious Chronic Illness. Click here to view.
