In This Week’s Issue:
1. Value Assessment Organizations Should Learn from the Experts on Patient Engagement, click here to read the blog post, the latest entry in PIPC's series The Data Mine.
2. Webinar on Protecting Patients with Cystic Fibrosis, click here for more information and to register.
3. Traditional Cost-Effectiveness Models Cannot Capture Medicines' Benefits, click here to read the article.
4. IVI: COVID-19 Forces a Reassessment of Measuring Value, click here to read the article.
5. Key Issues with ICER's Report on COVID-19, click here to read PIPC's one-pager.
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, 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. Value Assessment Organizations Should Learn from the Experts on Patient Engagement, click here to read the blog post, the latest entry in PIPC's series The Data Mine.
2. Webinar on Protecting Patients with Cystic Fibrosis, click here for more information and to register.
3. Traditional Cost-Effectiveness Models Cannot Capture Medicines' Benefits, click here to read the article.
4. IVI: COVID-19 Forces a Reassessment of Measuring Value, click here to read the article.
5. Key Issues with ICER's Report on COVID-19, click here to read PIPC's one-pager.
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, 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. Value Assessment Organizations Should Learn from the Experts on Patient Engagement
Dr. Steven Pearson, founder and president of the Institute for Clinical and Economic Review (ICER), recently wrote that no clear framework exists to engage patients in medical research. In A proposed framework for patient engagement throughout the broader research enterprise, published in the Journal of Comparative Effectiveness Research, Dr. Pearson acknowledges that patient engagement is an important component of medical research, but he fails to recognize years of accomplishments in the field. This misleading premise ignores rigorous patient-centered outcomes research and evolving development of evidence-based frameworks for patient engagement. Click here to read the blog post, the latest entry in PIPC's series The Data Mine.
2. Webinar on Protecting Patients with Cystic Fibrosis
On June 9, Cystic Fibrosis advocates Gunnar Esiason and Siri Vaeth will be featured on a webinar focusing on protecting patients with cystic fibrosis from discrimination by ICER and its QALY-based studies. The webinar will be hosted by the Pioneer Institute and feature the Institute’s Dr. William Smith and will be moderated by Mary Vought. Click here for more information and to register.
3. Traditional Cost-Effectiveness Models Cannot Capture Medicines' Benefits
Writing in the Health Affairs blog, Jamie Rubin Cahill says that one-size-fits-all value frameworks are inadequate and that the QALY is ill-suited to assessing medicines for rare diseases. "Cost-effectiveness analyses, which are often conducted to evaluate new medicines, use a standardized framework to inform health care decision makers," writes Ms. Cahill. However, a one-size-fits-all approach to evaluating cost-effectiveness is not adequate for every medicine....[T]here are unique attributes of many serious diseases and novel innovative medicines, that, when combined, lead to inherent biases when traditional modeling methodologies are used to calculate their cost-effectiveness." She also notes that even ICER recognizes that the QALY does not capture the benefits of rare disease treatments. Click here to read the article.
4. IVI: COVID-19 Forces a Reassessment of Measuring Value
The Innovation and Value Initiative (IVI) writes that the ongoing COVID-19 pandemic is forcing stakeholders to reassess how value can be measured. "Many have commented on the work of the Institute for Clinical and Economic Review (ICER) in conducting a first cost-effectiveness analysis of the product, with an eye toward informing its potential 'value-based price,'" it says in an article. "As companies tackle rapid investigations into therapies—including yet-unrealized vaccines and potential cures—we think it crucial to take a broader perspective in defining value....How 'value' is determined will have long-term consequences on the future investments in COVID-19 treatments. The unintended impact of narrow assessments could create disincentives for investment in novel treatments that could offer improvements or even a cure." Click here to read the article.
5. Key Issues with ICER's Report on COVID-19
The Institute for Clinical and Economic Review (ICER) recently released a model it intends to use to evaluate treatments for COVID-19, along with its results for remdesivir, the first drug shown to be efficacious against the virus. A thorough review of the model shows it to contain many concerning assumptions and to discriminate against older patients. In the height of this global crisis, it would be irresponsible, and put older Americans, patients, and people with disabilities as risk for policymakers to rely on ICER’s flawed model. Click here to read PIPC's one-pager on the issue.
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
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 Healthcare Delivery and Disparities Research Spring 2020 Meeting
June 11, 2020
Click here for details.
Building Capacity for Patient-Centered Research
June 19, 2020
Click here for details.
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.
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
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 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.
OPEN FOR COMMENT THROUGH JUNE 12: Technical Brief: Prevention, Diagnosis, and Management of Opioids, Opioid Misuse and Opioid Use Disorder in Older Adults. 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.
Dr. Steven Pearson, founder and president of the Institute for Clinical and Economic Review (ICER), recently wrote that no clear framework exists to engage patients in medical research. In A proposed framework for patient engagement throughout the broader research enterprise, published in the Journal of Comparative Effectiveness Research, Dr. Pearson acknowledges that patient engagement is an important component of medical research, but he fails to recognize years of accomplishments in the field. This misleading premise ignores rigorous patient-centered outcomes research and evolving development of evidence-based frameworks for patient engagement. Click here to read the blog post, the latest entry in PIPC's series The Data Mine.
2. Webinar on Protecting Patients with Cystic Fibrosis
On June 9, Cystic Fibrosis advocates Gunnar Esiason and Siri Vaeth will be featured on a webinar focusing on protecting patients with cystic fibrosis from discrimination by ICER and its QALY-based studies. The webinar will be hosted by the Pioneer Institute and feature the Institute’s Dr. William Smith and will be moderated by Mary Vought. Click here for more information and to register.
3. Traditional Cost-Effectiveness Models Cannot Capture Medicines' Benefits
Writing in the Health Affairs blog, Jamie Rubin Cahill says that one-size-fits-all value frameworks are inadequate and that the QALY is ill-suited to assessing medicines for rare diseases. "Cost-effectiveness analyses, which are often conducted to evaluate new medicines, use a standardized framework to inform health care decision makers," writes Ms. Cahill. However, a one-size-fits-all approach to evaluating cost-effectiveness is not adequate for every medicine....[T]here are unique attributes of many serious diseases and novel innovative medicines, that, when combined, lead to inherent biases when traditional modeling methodologies are used to calculate their cost-effectiveness." She also notes that even ICER recognizes that the QALY does not capture the benefits of rare disease treatments. Click here to read the article.
4. IVI: COVID-19 Forces a Reassessment of Measuring Value
The Innovation and Value Initiative (IVI) writes that the ongoing COVID-19 pandemic is forcing stakeholders to reassess how value can be measured. "Many have commented on the work of the Institute for Clinical and Economic Review (ICER) in conducting a first cost-effectiveness analysis of the product, with an eye toward informing its potential 'value-based price,'" it says in an article. "As companies tackle rapid investigations into therapies—including yet-unrealized vaccines and potential cures—we think it crucial to take a broader perspective in defining value....How 'value' is determined will have long-term consequences on the future investments in COVID-19 treatments. The unintended impact of narrow assessments could create disincentives for investment in novel treatments that could offer improvements or even a cure." Click here to read the article.
5. Key Issues with ICER's Report on COVID-19
The Institute for Clinical and Economic Review (ICER) recently released a model it intends to use to evaluate treatments for COVID-19, along with its results for remdesivir, the first drug shown to be efficacious against the virus. A thorough review of the model shows it to contain many concerning assumptions and to discriminate against older patients. In the height of this global crisis, it would be irresponsible, and put older Americans, patients, and people with disabilities as risk for policymakers to rely on ICER’s flawed model. Click here to read PIPC's one-pager on the issue.
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.
- Canada: Petition gets thousands of signatures to stop new rules that reduce patient access to new medications. Lawn signs honor families affected by cystic fibrosis and seek to stop rules reducing access to medication. A mother and her daughter, who lives with cystic fibrosis, fight for access to treatments.
- New Zealand: Woman with bowel cancer fights to raise awareness about Pharmac not funding lifesaving treatments.
- United Kingdom: Amid COVID-19 crisis, cystic fibrosis patients call for access to lifesaving treatments. Four-year-old with cystic fibrosis raises awareness for the disease.
7. ICER's QALY-Based Study Topics: Hemophilia A, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, Bladder Cancer, Opioid Treatments
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: MARCH 2020: DEADLINES PUSHED BACK 2-3 MONTHS. Draft Evidence Report and Voting Questions AVAILABLE, Comment Period OPEN through 6/24/2020. 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: MARCH 2020: REVIEW PAUSED 2-3 MONTHS. Revised Scoping Document available. 6/15/2020: Research Protocol. Meeting 11/5/2020: New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for hemophilia.
- Bladder Cancer: 6/12/2020: Revised Scoping Document
- Opioids: Digital Apps: 6/12/2020: Revised Scoping Document
- Opioids: Supervised Injection Centers: Draft Scoping Document AVAILABLE, Comment period open through TOMORROW, 6/9/2020.
8. Upcoming Events and Webinars
PCORI Advisory Panel on Healthcare Delivery and Disparities Research Spring 2020 Meeting
June 11, 2020
Click here for details.
Building Capacity for Patient-Centered Research
June 19, 2020
Click here for details.
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.
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
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 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.
OPEN FOR COMMENT THROUGH JUNE 12: Technical Brief: Prevention, Diagnosis, and Management of Opioids, Opioid Misuse and Opioid Use Disorder in Older Adults. 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.
