In This Week’s Issue:
1. PIPC Submits Comment Letter to ICER on Draft Evidence Report for NASH, click here to read the letter.
2. Experts and Advocates Oppose Importing Discrimination Through Foreign Reference Pricing, see details below.
3. Cost Assessors Must Consider Novel Value Elements, click here to read the article.
4. Rethinking How We Measure a Drug's 'Value,' click here to read the article.
5. Over 50 Leading Patient Groups Join PIPC Letter on ICER’s COVID-19 Treatment Assessments, see details below.
6. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
7. 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, click here to provide patient input.
8. Upcoming Events and Webinars, see details below.
9. Medical Journal Articles, see details below.
10. AHRQ Effective Program Updates, see details below.
1. PIPC Submits Comment Letter to ICER on Draft Evidence Report for NASH, click here to read the letter.
2. Experts and Advocates Oppose Importing Discrimination Through Foreign Reference Pricing, see details below.
3. Cost Assessors Must Consider Novel Value Elements, click here to read the article.
4. Rethinking How We Measure a Drug's 'Value,' click here to read the article.
5. Over 50 Leading Patient Groups Join PIPC Letter on ICER’s COVID-19 Treatment Assessments, see details below.
6. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
7. 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, click here to provide patient input.
8. Upcoming Events and Webinars, see details below.
9. Medical Journal Articles, see details below.
10. AHRQ Effective Program Updates, see details below.
1. PIPC Submits Comment Letter to ICER on Draft Evidence Report for NASH
In a letter to the Institute for Clincial and Economic Review (ICER), Partnership to Improve Patient Care Chairman (PIPC) Chairman Tony Coelho provided feedback on ICER's draft evidence report for Non-alcoholic Steatohepatitis (NASH). Chairman Coelho criticized ICER's model for continuing to rely on the discriminatory quality-adjusted-life-years (QALY) metric, writing that it is an inappropriate metric to accurately show health gains for NASH patients. The model also makes inaccurate assumptions about liver transplant procedures. "ICER’s model exacerbates the shortcomings of the QALY by discounting the future health gains incorrectly," the letter states. "NASH is a complex condition, and it is important ICER holistically capture the complexity and the impact the disease has on individual patients and public health." Click here to read the letter.
2. Experts and Advocates Oppose Importing Discrimination Through Foreign Reference Pricing
Policymakers continue to propose foreign reference pricing as a tactic to lower drug prices within the United States. Though this policy may sound attractive on paper, experts and advocates worry that tis policy will hurt the nation’s most vulnerable causing restrictions and delays in access to needed treatments. The National Council on Disability made clear in a recent report that importing this type of QALY-based drug pricing that relies on prices set internationally would be contrary to United States civil rights law and disability policy.
3. Cost Assessors Must Consider Novel Value Elements
Writing in the Health Affairs blog, a group of researchers write that ICER's value assessment methodology is ill-suited to determining the value of drugs. Instead, they argue, the health system must consider additional elements of value, such as contextual considerations. "As scientists continue their efforts to develop new therapeutics and vaccines to treat and prevent COVID-19, other stakeholders—health technology assessment bodies, payers, policy makers, manufacturers, and health economists—have a responsibility to also ensure innovation in value assessment. The recent ICER analysis of remdesivir failed in this respect by not adequately addressing novel elements of value (even as contextual considerations) that are of particular relevance in the ongoing pandemic. While contentious debates surrounding the value and price of new therapies are likely to continue, the COVID-19 saga should make clear the importance of rethinking the existing paradigm and avoiding business as usual." Click here to read the article.
4. Rethinking How We Measure a Drug's 'Value'
Writing in STAT News, the Innovation and Value Initiative's (IVI) Patricia Deverka, Louis Garrison, and Samuel Nussbaum say that ICER should rethink how it conducts value assessments, focusing on its study relating to COVID-19. They highlight three primary issues with ICER's analysis of the antiviral Remdesivir: (1) significant uncertainty relating to infection prevalence and the effectiveness of Remdesivir, (2) a lack of transparency in ICER's models, and (3) omitting possible impacts on the health system capacity and health care personnel. Instead, they suggest the following principles for value assessment: (1) Consensus on model inputs, methods, and structures based on public input; (2) Flexibility to accommodate the information needs of stakeholders; (3) Transparency to allow real-time review and updating; and (4) Open-source development of models to enable widespread use and customization. Click here to read the article.
5. Over 50 Leading Patient Groups Join PIPC Letter on ICER’s COVID-19 Treatment Assessments
More than 50 leading groups representing patients and people with disabilities joined the Partnership to Improve Patient Care’s (PIPC) comment letter to the Institute for Clinical and Economic Review (ICER) regarding its assessment of remdesivir and other treatments for the novel coronavirus (COVID-19). The letter offers strong criticism of ICER’s use of flawed methodologies in its assessment, noting that ICER has chosen to completely ignore vast array of stakeholder feedback it has received over the last five years on its framework and processes. The groups reiterate that it’s imperative that discriminatory assessment tools like the quality-adjusted-life-years (QALY) metric are not used in determining the price of treatments, especially during public health emergencies such as COVID-19. “We have consistently raised the red flag that ICER’s value assessments are methodologically flawed and not fit for the purpose of making decisions related to coverage, reimbursement and incentive programs by policymakers and payers,” the letter states. “ The latest assessment from ICER validates our concerns.” Click here to read the letter. Click here to read PIPC's one-pager on the main issues with ICER's report.
6. 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.
7. 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
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. All of ICER’s current assessments have been put on hold for 2-3 months in response to COVID-19. We will continue to update below deadlines as we receive more information.
8. Upcoming Events and Webinars
PCORI Advisory Panel on Rare Disease Spring 2020 Meeting
June 22, 2020
Click here for details.
PCORI Board of Governors Meeting
June 23, 2020
Click here for details.
The Developing HTA Landscape in APAC – Insights and Lessons Learned
June 23, 2020
Click here for details.
PCORI Engagement Awardee Lunch and Learn: Virtual Engagement
June 24, 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.
9. Medical Journal Articles
The Impact of Drug Quality Ratings from Health Technology Assessments on the Adoption of New Drugs by Physicians in Germany, click here to view.
Developing a Patient- and Family-Centered Research Agenda for Hospital Medicine: The Improving Hospital Outcomes through Patient Engagement (i-HOPE) Study, click here to view.
Annals Clinical Decision Making: Communicating Risk and Engaging Patients in Shared Decision Making, click here to view.
Comparative Effectiveness Research in Critically Ill Patients: Risks Associated with Mischaracterising Usual Care, click here to view.
Unanticipated Therapeutic Value of the Patient-Centered Outcomes Research Institute (PCORI) Stakeholder Engagement Project for Homebound Older Adults, click here to view.
Impact of a Global Pandemic on Health Technology Assessment, click here to view.
How Are Incremental Cost-Effectiveness, Contextual Considerations, and Other Benefits Viewed in Health Technology Assessment Recommendations in the United States?, click here to view.
The Implementation of Health Technology Assessment Principles in Public Decisions Concerning Orphan Drugs, click here to view.
Decision-Making under Uncertainty: Comparing Regulatory and Health Technology Assessment Reviews of Medicines in the US and Europe, click here to view.
What Types of Real-World Evidence Studies Do U.S. Commercial Health Plans Cite in Their Specialty Drug Coverage Decisions?, click here to view.
Defining the Role of the Public in Health Technology Assessment (HTA) and HTA-Informed Decision-Making Processes, click here to view.
10. AHRQ Effective Program Updates
OPEN FOR COMMENT THROUGH JULY 13: Maternal and Fetal Effects of Mental Health Treatments in Pregnant and Breastfeeding Women: A Systematic Review of Pharmacological Interventions. Click here to view.
Research Protocol: Breast Reconstruction after Mastectomy. Click here to view.
Rapid Evidence Report: Masks for Prevention of COVID-19 in Community and Healthcare Settings: A Living Rapid Review. Click here to view.
OPEN FOR COMMENT THROUGH JULY 6: Systematic Review: Pharmacologic and Nonpharmacologic Treatments for Posttraumatic Stress Disorder: An Update of the PTSD Repository. Click here to view.
OPEN FOR COMMENT THROUGH JUNE 29: Systematic Review: Therapies for Clinically Localized Prostate Cancer. Click here to view.
OPEN FOR COMMENT THROUGH JUNE 29: Systematic Review: Management of Colonic Diverticulitis. Click here to view.
OPEN FOR COMMENT THROUGH JUNE 30: Systematic Review: Management of Primary Headaches in Pregnancy. Click here to view.
Systematic Review: Interventions for Substance Use Disorders in Adolescents. Click here to view.
White Paper: The Evidence Base for Telehealth: Reassurance in the Face of Rapid Expansion During the COVID-19 Pandemic. Click here to view.
White Paper: Standardized Library of Depression Outcome Measures. Click here to view.
Research Protocol: No-Touch Modalities for Disinfection of Hospital/Acute Care Settings: A Rapid Evidence Review. Click here to view.
Research Protocol: Masks for Prevention of COVID-19 in Healthcare and Community Settings. Click here to view.
Systematic Review: Labor Dystocia. Click here to view.
In a letter to the Institute for Clincial and Economic Review (ICER), Partnership to Improve Patient Care Chairman (PIPC) Chairman Tony Coelho provided feedback on ICER's draft evidence report for Non-alcoholic Steatohepatitis (NASH). Chairman Coelho criticized ICER's model for continuing to rely on the discriminatory quality-adjusted-life-years (QALY) metric, writing that it is an inappropriate metric to accurately show health gains for NASH patients. The model also makes inaccurate assumptions about liver transplant procedures. "ICER’s model exacerbates the shortcomings of the QALY by discounting the future health gains incorrectly," the letter states. "NASH is a complex condition, and it is important ICER holistically capture the complexity and the impact the disease has on individual patients and public health." Click here to read the letter.
2. Experts and Advocates Oppose Importing Discrimination Through Foreign Reference Pricing
Policymakers continue to propose foreign reference pricing as a tactic to lower drug prices within the United States. Though this policy may sound attractive on paper, experts and advocates worry that tis policy will hurt the nation’s most vulnerable causing restrictions and delays in access to needed treatments. The National Council on Disability made clear in a recent report that importing this type of QALY-based drug pricing that relies on prices set internationally would be contrary to United States civil rights law and disability policy.
3. Cost Assessors Must Consider Novel Value Elements
Writing in the Health Affairs blog, a group of researchers write that ICER's value assessment methodology is ill-suited to determining the value of drugs. Instead, they argue, the health system must consider additional elements of value, such as contextual considerations. "As scientists continue their efforts to develop new therapeutics and vaccines to treat and prevent COVID-19, other stakeholders—health technology assessment bodies, payers, policy makers, manufacturers, and health economists—have a responsibility to also ensure innovation in value assessment. The recent ICER analysis of remdesivir failed in this respect by not adequately addressing novel elements of value (even as contextual considerations) that are of particular relevance in the ongoing pandemic. While contentious debates surrounding the value and price of new therapies are likely to continue, the COVID-19 saga should make clear the importance of rethinking the existing paradigm and avoiding business as usual." Click here to read the article.
4. Rethinking How We Measure a Drug's 'Value'
Writing in STAT News, the Innovation and Value Initiative's (IVI) Patricia Deverka, Louis Garrison, and Samuel Nussbaum say that ICER should rethink how it conducts value assessments, focusing on its study relating to COVID-19. They highlight three primary issues with ICER's analysis of the antiviral Remdesivir: (1) significant uncertainty relating to infection prevalence and the effectiveness of Remdesivir, (2) a lack of transparency in ICER's models, and (3) omitting possible impacts on the health system capacity and health care personnel. Instead, they suggest the following principles for value assessment: (1) Consensus on model inputs, methods, and structures based on public input; (2) Flexibility to accommodate the information needs of stakeholders; (3) Transparency to allow real-time review and updating; and (4) Open-source development of models to enable widespread use and customization. Click here to read the article.
5. Over 50 Leading Patient Groups Join PIPC Letter on ICER’s COVID-19 Treatment Assessments
More than 50 leading groups representing patients and people with disabilities joined the Partnership to Improve Patient Care’s (PIPC) comment letter to the Institute for Clinical and Economic Review (ICER) regarding its assessment of remdesivir and other treatments for the novel coronavirus (COVID-19). The letter offers strong criticism of ICER’s use of flawed methodologies in its assessment, noting that ICER has chosen to completely ignore vast array of stakeholder feedback it has received over the last five years on its framework and processes. The groups reiterate that it’s imperative that discriminatory assessment tools like the quality-adjusted-life-years (QALY) metric are not used in determining the price of treatments, especially during public health emergencies such as COVID-19. “We have consistently raised the red flag that ICER’s value assessments are methodologically flawed and not fit for the purpose of making decisions related to coverage, reimbursement and incentive programs by policymakers and payers,” the letter states. “ The latest assessment from ICER validates our concerns.” Click here to read the letter. Click here to read PIPC's one-pager on the main issues with ICER's report.
6. 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: Australian government denying migraine patients access to CGRP inhibitors, creating a $35.7 drag on the economy.
- Canada: Pan-Canadian Pharmaceutical Alliance finally agrees to negotiations to cover cystic fibrosis drugs. Drug that can treat 90 percent of cystic fibrosis patients remains unavailable in Canada. While Canadian scientists work on promising COVID-19 drugs, they do not want to do clinical trials there without guarantees that an eventual product will be covered.
- United Kingdom: EMA may approve a breakthrough cystic fibrosis drug next month, increasing pressure on the NHS to cover it. Ten-year-old denied spinal muscular atrophy treatment for "taking one step too few." Click here to read more.
7. 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
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. All of ICER’s current assessments have been put on hold for 2-3 months in response to COVID-19. We will continue to update below deadlines as we receive more information.
- Ulcerative Colitis: Draft Evidence Report and Draft Voting Questions AVAILABLE. Comment period open through 7/29/2020. Meeting 9/24/2020: CTAF will convene to deliberate and vote on evidence presented in ICER's report on ulcerative colitis therapies.
- Cystic Fibrosis: Evidence Report and Response to Comments AVAILABLE. Meeting POSTPONED: CTAF will convene to deliberate and vote on evidence presented in ICER's report on treatments for cystic fibrosis.
- 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.
- Non-Alcoholic Steatohepatitis: 7/21/2020: Evidence Report and Responses to Comments. Meeting 8/13/2020: The Midwest CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for NASH.
- Hemophilia A: Research Protocol AVAILABLE. 7/1/2020: Model Analysis Plan.
- Bladder Cancer: Revised Scoping Document AVAILABLE. 7/14/2020: Research Protocol.
- Opioids: Digital Apps: Revised Scoping Document AVAILABLE. 7/14/2020: Research Protocol.
- Opioids: Supervised Injection Centers: Revised Scoping Document AVAILABLE. 7/21/2020: Research Protocol.
- High Cholesterol: Open input period through 6/30/2020.
- Anemia in Chronic Kidney Disease: Open input period through 7/15/2020.
- Unsupported Price Increase Assessment: Protocol Available. Public input through 7/17/2020.
8. Upcoming Events and Webinars
PCORI Advisory Panel on Rare Disease Spring 2020 Meeting
June 22, 2020
Click here for details.
PCORI Board of Governors Meeting
June 23, 2020
Click here for details.
The Developing HTA Landscape in APAC – Insights and Lessons Learned
June 23, 2020
Click here for details.
PCORI Engagement Awardee Lunch and Learn: Virtual Engagement
June 24, 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.
9. Medical Journal Articles
The Impact of Drug Quality Ratings from Health Technology Assessments on the Adoption of New Drugs by Physicians in Germany, click here to view.
Developing a Patient- and Family-Centered Research Agenda for Hospital Medicine: The Improving Hospital Outcomes through Patient Engagement (i-HOPE) Study, click here to view.
Annals Clinical Decision Making: Communicating Risk and Engaging Patients in Shared Decision Making, click here to view.
Comparative Effectiveness Research in Critically Ill Patients: Risks Associated with Mischaracterising Usual Care, click here to view.
Unanticipated Therapeutic Value of the Patient-Centered Outcomes Research Institute (PCORI) Stakeholder Engagement Project for Homebound Older Adults, click here to view.
Impact of a Global Pandemic on Health Technology Assessment, click here to view.
How Are Incremental Cost-Effectiveness, Contextual Considerations, and Other Benefits Viewed in Health Technology Assessment Recommendations in the United States?, click here to view.
The Implementation of Health Technology Assessment Principles in Public Decisions Concerning Orphan Drugs, click here to view.
Decision-Making under Uncertainty: Comparing Regulatory and Health Technology Assessment Reviews of Medicines in the US and Europe, click here to view.
What Types of Real-World Evidence Studies Do U.S. Commercial Health Plans Cite in Their Specialty Drug Coverage Decisions?, click here to view.
Defining the Role of the Public in Health Technology Assessment (HTA) and HTA-Informed Decision-Making Processes, click here to view.
10. AHRQ Effective Program Updates
OPEN FOR COMMENT THROUGH JULY 13: Maternal and Fetal Effects of Mental Health Treatments in Pregnant and Breastfeeding Women: A Systematic Review of Pharmacological Interventions. Click here to view.
Research Protocol: Breast Reconstruction after Mastectomy. Click here to view.
Rapid Evidence Report: Masks for Prevention of COVID-19 in Community and Healthcare Settings: A Living Rapid Review. Click here to view.
OPEN FOR COMMENT THROUGH JULY 6: Systematic Review: Pharmacologic and Nonpharmacologic Treatments for Posttraumatic Stress Disorder: An Update of the PTSD Repository. Click here to view.
OPEN FOR COMMENT THROUGH JUNE 29: Systematic Review: Therapies for Clinically Localized Prostate Cancer. Click here to view.
OPEN FOR COMMENT THROUGH JUNE 29: Systematic Review: Management of Colonic Diverticulitis. Click here to view.
OPEN FOR COMMENT THROUGH JUNE 30: Systematic Review: Management of Primary Headaches in Pregnancy. Click here to view.
Systematic Review: Interventions for Substance Use Disorders in Adolescents. Click here to view.
White Paper: The Evidence Base for Telehealth: Reassurance in the Face of Rapid Expansion During the COVID-19 Pandemic. Click here to view.
White Paper: Standardized Library of Depression Outcome Measures. Click here to view.
Research Protocol: No-Touch Modalities for Disinfection of Hospital/Acute Care Settings: A Rapid Evidence Review. Click here to view.
Research Protocol: Masks for Prevention of COVID-19 in Healthcare and Community Settings. Click here to view.
Systematic Review: Labor Dystocia. Click here to view.
