In This Week’s Issue:
1. Patient Advocate: Socialized Medicine and Rationing COVID Care Defy American Principles, click here to read the op-ed.
2. PhRMA Foundation Challenge Award Program Funding Opportunity, see details below.
3. PCORI Seeks Nominations for Advisory Panels, click here to learn more, submit a nomination, or apply to be on an advisory panel.
4. Study: Step Therapy Can Lead to Higher OOP Costs for Crohn’s Disease Patients, click here to read more.
5. PCORI Proposed Principles for the Consideration of the Full Range of Outcomes Data and Webinar Series -- Comments Open through November 13, 2020, see details below.
6. RESCHEDULED -- National Health Council Webinar on Multi-Criteria Decision Analysis, click here to learn more and register.
7. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
8. ICER's QALY-Based Study Topics: Hemophilia A, Sickle Cell Disease, Bladder Cancer, Opioid Treatments, High Cholesterol, Anemia in Chronic Kidney Disease, Lupus Nephritis, Multiple Myeloma, Alzheimer's Disease, 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. Patient Advocate: Socialized Medicine and Rationing COVID Care Defy American Principles, click here to read the op-ed.
2. PhRMA Foundation Challenge Award Program Funding Opportunity, see details below.
3. PCORI Seeks Nominations for Advisory Panels, click here to learn more, submit a nomination, or apply to be on an advisory panel.
4. Study: Step Therapy Can Lead to Higher OOP Costs for Crohn’s Disease Patients, click here to read more.
5. PCORI Proposed Principles for the Consideration of the Full Range of Outcomes Data and Webinar Series -- Comments Open through November 13, 2020, see details below.
6. RESCHEDULED -- National Health Council Webinar on Multi-Criteria Decision Analysis, click here to learn more and register.
7. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
8. ICER's QALY-Based Study Topics: Hemophilia A, Sickle Cell Disease, Bladder Cancer, Opioid Treatments, High Cholesterol, Anemia in Chronic Kidney Disease, Lupus Nephritis, Multiple Myeloma, Alzheimer's Disease, 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. Patient Advocate: Socialized Medicine and Rationing COVID Care Defy American Principles
Khrystal Davis, the mother of a child with spinal muscular atrophy, wrote in Inside Sources last week that a movement towards socialized medicine, exemplified by President Trump's executive order calling for "most-favored nation" drug pricing, will hurt children like hers. "COVID-19 has resurrected this un-American idea [the QALY] of rationing healthcare. States have sought to advance Crisis Standards of Care that allow them to deny care for people with disabilities and serious medical conditions. This logic is not dissimilar from the devaluation of life that underpins the QALY," she wrote. "Meanwhile, as states actively ration care, the Trump administration issued an executive order that ties the prices we pay for prescription drugs to prices in foreign, socialist countries where QALYs provide the rationale for restricting coverage and access. This is the same concept as a recent bill pushed by House Speaker Nancy Pelosi and progressive democrats.... I worry that due to the president’s executive order, I may not be able to protect my son, even by returning to my conservative roots in St. Louis." Click here to read the op-ed.
2. PhRMA Foundation Challenge Award Program Funding Opportunity
The PhRMA Foundation announced a new Challenge Award program soliciting research proposals to evaluate how value assessment can better serve diverse populations and address the drivers of health disparities. The foundation will award a total of $85,000 to researchers whose papers advance solutions to that question. Interested candidates should submit letters of intent to the Foundation by no later than December 1, 2020, to initiate the application process. Qualified applicants will be contacted no later than December 21, 2020, with a request to submit the full paper describing their response to the challenge question. To learn more about the application process, click here. For more details about the award, click here to view a brochure.
3. PCORI Seeks Nominations for Advisory Panels
PCORI is currently seeking nominations for appointments to its advisory panels. The advisory panels must include patients or patient advocates. PCORI's staff, board, and methodology committee take advisory panels' recommendations into account when making decisions and determinations. PIPC encourages patients and patient advocates to submit nominations to serve on PCORI's advisory panels, including on the Advisory Panel on Clinical Effectiveness and Decision Science, Advisory Panel on Patient Engagement, and Advisory Panel on Rare Disease. Nominations are open through March 31, 2020. Click here to learn more, submit a nomination, or apply to be on an advisory panel.
4. Study: Step Therapy Can Lead to Higher OOP Costs for Crohn’s Disease Patients
A study by Avalere found that patients with Crohn’s disease who experience a negative outcome when required to step through a plan’s preferred product may face higher upfront out-of-pocket (OOP) costs and could increase costs to payers. Step therapy—also known as “fail first”—is a common form of utilization management that health plans use to control costs. Step therapy requires patients to try a preferred drug before gaining access to the drug prescribed by their provider. Click here to read more.
5. PCORI Proposed Principles for the Consideration of the Full Range of Outcomes Data and Webinar Series -- Comments Open through November 13, 2020
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. Click here to view the transcripts of the meeting.
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. The comment period is open through November 13, 2020. Click here to read the principles and to submit comments.
6. 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.
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's QALY-Based Study Topics: Hemophilia A, Sickle Cell Disease, 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.
9. Upcoming Events and Webinars
Bang for the Buck: How Health Care Innovation Affects Life Expectancy and Patient Well-Being
November 10, 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.
PCORI Advisory Panel on Healthcare Delivery and Disparities Research Winter 2020 Meeting
December 10, 2020
Click here for details.
10. Medical Journal Articles
Improving the Quality of Person-Centred Healthcare from the Patient Perspective: Development of Person-Centred Quality Indicators, click here to view.
Six Ways to Foster Community-Engaged Research During Times of Societal Crises, click here to view.
Improving Comparative Effectiveness Research of Complex Health Interventions: Standards from the Patient-Centered Outcomes Research Institute (PCORI), click here to view.
Improving Access and Quality of Health Care in the United States: Shared Goals Among Patient Advocates, click here to view.
Using Electronic Health Record Data to Identify Comparator Populations for Comparative Effectiveness Research, click here to view.
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.
11. AHRQ Effective Program Updates
White Paper: Roadmap for Narratively Describing Effects of Interventions in Systematic Reviews. Click here to view.
Systematic Review: Pharmacologic and Nonpharmacologic Treatments for Posttraumatic Stress Disorder. Click here to view.
Technical Brief: Prevention, Diagnosis, and Management of Opioids, Opioid Misuse, and Opioid Use Disorder in Older Adults. Click here to view.
Research Protocol: Living Systematic Review on Plant-Based Treatment for Chronic Pain. Click here to view.
Research Report: Testing a Machine Learning Tool for Facilitating Living Systematic Reviews of Chronic Pain Treatments. Click here to view.
OPEN FOR COMMENT THROUGH 11/23/2020: Safety of Vaccines Used for Routine Immunization in the United States: An Update. Click here to view.
Research Protocol: Malnutrition in Hospitalized Adults. 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.
No-Touch Modalities for Disinfecting Patient Rooms in Acute Care Settings: A Rapid Review. Click here to view.
Khrystal Davis, the mother of a child with spinal muscular atrophy, wrote in Inside Sources last week that a movement towards socialized medicine, exemplified by President Trump's executive order calling for "most-favored nation" drug pricing, will hurt children like hers. "COVID-19 has resurrected this un-American idea [the QALY] of rationing healthcare. States have sought to advance Crisis Standards of Care that allow them to deny care for people with disabilities and serious medical conditions. This logic is not dissimilar from the devaluation of life that underpins the QALY," she wrote. "Meanwhile, as states actively ration care, the Trump administration issued an executive order that ties the prices we pay for prescription drugs to prices in foreign, socialist countries where QALYs provide the rationale for restricting coverage and access. This is the same concept as a recent bill pushed by House Speaker Nancy Pelosi and progressive democrats.... I worry that due to the president’s executive order, I may not be able to protect my son, even by returning to my conservative roots in St. Louis." Click here to read the op-ed.
2. PhRMA Foundation Challenge Award Program Funding Opportunity
The PhRMA Foundation announced a new Challenge Award program soliciting research proposals to evaluate how value assessment can better serve diverse populations and address the drivers of health disparities. The foundation will award a total of $85,000 to researchers whose papers advance solutions to that question. Interested candidates should submit letters of intent to the Foundation by no later than December 1, 2020, to initiate the application process. Qualified applicants will be contacted no later than December 21, 2020, with a request to submit the full paper describing their response to the challenge question. To learn more about the application process, click here. For more details about the award, click here to view a brochure.
3. PCORI Seeks Nominations for Advisory Panels
PCORI is currently seeking nominations for appointments to its advisory panels. The advisory panels must include patients or patient advocates. PCORI's staff, board, and methodology committee take advisory panels' recommendations into account when making decisions and determinations. PIPC encourages patients and patient advocates to submit nominations to serve on PCORI's advisory panels, including on the Advisory Panel on Clinical Effectiveness and Decision Science, Advisory Panel on Patient Engagement, and Advisory Panel on Rare Disease. Nominations are open through March 31, 2020. Click here to learn more, submit a nomination, or apply to be on an advisory panel.
4. Study: Step Therapy Can Lead to Higher OOP Costs for Crohn’s Disease Patients
A study by Avalere found that patients with Crohn’s disease who experience a negative outcome when required to step through a plan’s preferred product may face higher upfront out-of-pocket (OOP) costs and could increase costs to payers. Step therapy—also known as “fail first”—is a common form of utilization management that health plans use to control costs. Step therapy requires patients to try a preferred drug before gaining access to the drug prescribed by their provider. Click here to read more.
5. PCORI Proposed Principles for the Consideration of the Full Range of Outcomes Data and Webinar Series -- Comments Open through November 13, 2020
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. Click here to view the transcripts of the meeting.
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. The comment period is open through November 13, 2020. Click here to read the principles and to submit comments.
6. 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.
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: Family self-funds revolutionary spinal muscular atrophy treatment as Health Canada continues to refuse to approve it. Drugs patients need may stop being provided through hospitals in order to cut costs.
- New Zealand: Man with melanoma self-funds treatment as Pharmac will not pay for it. Breast cancer advocate demands end to "unfair and outdated evaluation process" for new drugs. Type 2 diabetes patients await Pharmac decision on funding of critical new drugs. Indigenous Maori are not able to access critical diabetes treatments. Breakthrough drug that could give a child with cystic fibrosis a future is not funded by Pharmac. Pharmac CEO says that budget is why they let down New Zealanders.
- United Kingdom: After his twin died of the disease, a man with cystic fibrosis finally gains access to lifesaving drug.
8. ICER's QALY-Based Study Topics: Hemophilia A, Sickle Cell Disease, 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.
- 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: 11/20/2020: Final Evidence Report. Meeting 10/30/2020: New England CEPAC convened to deliberate and vote on evidence presented in ICER's report on treatments for hemophilia.
- Bladder Cancer: Evidence Report AVAILABLE. 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: Evidence Report AVAILABLE. 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. 11/13/2020: Evidence Report and Responses to Public Comments. 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: Research Protocol available. 12/17/2020: Model Analysis Plan.
- Service Dogs for PTSD: 11/30/2020: Final Assessment and Report.
- Alzheimer's Disease: Draft Scoping Document AVAILABLE, comments open through 11/10/2020. Meeting 5/7/2020: CTAF will convene virtually to deliberate and vote on evidence presented in ICER's report on Alzheimer's disease.
9. Upcoming Events and Webinars
Bang for the Buck: How Health Care Innovation Affects Life Expectancy and Patient Well-Being
November 10, 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.
PCORI Advisory Panel on Healthcare Delivery and Disparities Research Winter 2020 Meeting
December 10, 2020
Click here for details.
10. Medical Journal Articles
Improving the Quality of Person-Centred Healthcare from the Patient Perspective: Development of Person-Centred Quality Indicators, click here to view.
Six Ways to Foster Community-Engaged Research During Times of Societal Crises, click here to view.
Improving Comparative Effectiveness Research of Complex Health Interventions: Standards from the Patient-Centered Outcomes Research Institute (PCORI), click here to view.
Improving Access and Quality of Health Care in the United States: Shared Goals Among Patient Advocates, click here to view.
Using Electronic Health Record Data to Identify Comparator Populations for Comparative Effectiveness Research, click here to view.
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.
11. AHRQ Effective Program Updates
White Paper: Roadmap for Narratively Describing Effects of Interventions in Systematic Reviews. Click here to view.
Systematic Review: Pharmacologic and Nonpharmacologic Treatments for Posttraumatic Stress Disorder. Click here to view.
Technical Brief: Prevention, Diagnosis, and Management of Opioids, Opioid Misuse, and Opioid Use Disorder in Older Adults. Click here to view.
Research Protocol: Living Systematic Review on Plant-Based Treatment for Chronic Pain. Click here to view.
Research Report: Testing a Machine Learning Tool for Facilitating Living Systematic Reviews of Chronic Pain Treatments. Click here to view.
OPEN FOR COMMENT THROUGH 11/23/2020: Safety of Vaccines Used for Routine Immunization in the United States: An Update. Click here to view.
Research Protocol: Malnutrition in Hospitalized Adults. 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.
No-Touch Modalities for Disinfecting Patient Rooms in Acute Care Settings: A Rapid Review. Click here to view.
