In This Week’s Issue:
1. Gunnar Esiason: Listen to Patients, Not Discriminatory Cost-Effectiveness Models, click here to read the article.
2. Value Our Health Sickle Cell Disease Webinar, see details below.
3. California Embracing “Most-Favored Nation” Concept Relying on International Drug Prices Based on QALYs and Similar Metrics, see details below.
4. Recap of the 10th Annual PIPC Forum, click here to see more.
5. WSJ Editorial: Where You Want to Get Cancer, click here to read the article.
6. Congratulations PCORI on a 10-Year Reauthorization!, click here to view the full press release.
7. Video: Partnerships May Lead to Better Engagement, click here to watch the video.
8. FH Call to Action, see details below.
9. PIPC Joins Leading Patient Groups in Comment Letter on TennCare II Demonstration, click here to read the letter.
10. PIPC Submits Feedback on 'Cures 2.0' Legislation, click here to read the letter.
11. Alliance for Aging Research Letter on Current Drug Proposals, click here to read the letter.
12. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
13. ICER's QALY-Based Study Topics: Hemophilia A, Beta Thalassemia, Acute Migraine, Arthritis, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, click here to provide patient input.
14. Upcoming Events and Webinars, see details below.
15. Medical Journal Articles, see details below.
16. AHRQ Effective Program Updates, see details below.
1. Gunnar Esiason: Listen to Patients, Not Discriminatory Cost-Effectiveness Models, click here to read the article.
2. Value Our Health Sickle Cell Disease Webinar, see details below.
3. California Embracing “Most-Favored Nation” Concept Relying on International Drug Prices Based on QALYs and Similar Metrics, see details below.
4. Recap of the 10th Annual PIPC Forum, click here to see more.
5. WSJ Editorial: Where You Want to Get Cancer, click here to read the article.
6. Congratulations PCORI on a 10-Year Reauthorization!, click here to view the full press release.
7. Video: Partnerships May Lead to Better Engagement, click here to watch the video.
8. FH Call to Action, see details below.
9. PIPC Joins Leading Patient Groups in Comment Letter on TennCare II Demonstration, click here to read the letter.
10. PIPC Submits Feedback on 'Cures 2.0' Legislation, click here to read the letter.
11. Alliance for Aging Research Letter on Current Drug Proposals, click here to read the letter.
12. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
13. ICER's QALY-Based Study Topics: Hemophilia A, Beta Thalassemia, Acute Migraine, Arthritis, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, click here to provide patient input.
14. Upcoming Events and Webinars, see details below.
15. Medical Journal Articles, see details below.
16. AHRQ Effective Program Updates, see details below.
1. Gunnar Esiason: Listen to Patients, Not Discriminatory Cost-Effectiveness Models
In an op-ed published in The Hill, Gunnar Esiason, a patient living with cystic fibrosis writes that patients like him should not be denied access to life-changing medication because an economic model says that their life is too expensive to save. Mr. Esiason is now taking the new cystic fibrosis drug Trikafta, which he says has changed his life. "QALY is not adequately able to quantify what happened in my own life — my journey from near end-stage illness and no hope for a future to correctly managed CF and entrance into an elite graduate program," he wrote. "I certainly would like to know why cost-effectiveness is being used against me. Would the analysts at ICER prefer I return to a state of recurrent hospitalizations, traumatic medical procedures, bouts of hemoptysis (coughing up blood), and the fast track towards end-stage illness and disability income? Frankly, I consider the talk of cost-effectiveness to be not only discriminatory but also pure ableism." Click here to read the article.
2. Value Our Health Sickle Cell Disease Webinar
Join Value Our Health and leading Sickle Cell Disease Organizations for a webinar on January 16, 2020 at 2pm. The webinar will provide information about the metric used to assess the value of health care treatments called the quality-adjusted life year or “QALY” as well as the primary organization conducting QALY-based value assessments called the Institute for Clinical Economic Review (ICER). Because ICER will assess the value of new sickle cell disease treatments in March, 2020, it is important for all stakeholders to understand QALYs, ICER and how those value assessments may be used by public and private payers in their decisions related to drug coverage and formularies. Learn more here, and RSVP to [email protected].
3. California Embracing “Most-Favored Nation” Concept Relying on International Drug Prices Based on QALYs and Similar Metrics
California’s Administration will propose to establish a single market for drug pricing within the state for all purchasers—Medi-Cal, California Public Employees’ Retirement System, Covered California, private insurers, self-insured employers, and others— in which California would invoke a most-favored-nation clause requiring manufacturers to offer prices at or below the price offered to "any other state, nation, or global purchaser.” In doing so, California would be referencing prices in other countries that use quality-adjusted life years (QALYs) and similar metrics to determine the cost effectiveness and coverage of drugs. As stated by the National Council on Disability, "In this report, NCD found sufficient evidence of the discriminatory effects of QALYs to warrant concern, including concerns raised by bioethicists, patient rights groups, and disability rights advocates about the limited access to lifesaving medications for chronic illnesses in countries where QALYs are frequently used. In addition, QALY-based programs have been found to violate the Americans with Disabilities Act.” Click here to view the California plan. Click here to view the NCD report calling on policymakers to "refrain from pursuing means of reducing Medicare and Medicaid prescription drug costs that attempt to model US pricing after the pricing in other countries, which may heavily rely on QALYs and often deny people with disabilities access to needed care.”
4. Recap of the 10th Annual PIPC Forum
PIPC held its 10th annual PIPC Forum on December 16, 2019. The Forum featured leading patient and disability advocates and technical health policy experts discussing the topic of international reference pricing. The presenters expressed concerns about discrimination and detrimental impacts on innovation should the United States move in the direction of using international reference pricing or cost-effectiveness analyses based on the QALY to address the cost of prescription drugs. Read a complete summary and view a video of the 10th Annual PIPC Forum here.
5. WSJ Editorial: Where You Want to Get Cancer
The Wall Street Journal published an editorial about recent decreases in the cancer death rate and remarkable advances in survival rates for many types of cancer. The editorial board noted that discriminatory policies in other countries mean that their cancer death rates are higher than those in the U.S. as patients cannot access lifesaving medication. "[G]overnment rationing and price controls on drugs are one major reason that countries with socialized medicine like the United Kingdom have lower cancer survival rates than the U.S. The age-adjusted cancer mortality rate is about 20% higher in the U.K and 10% higher in Canada and France than in the U.S. Survival rates for hard-to-treat cancers are also higher in the U.S. than in most countries with nationalized health systems," they wrote. Click here to read the article.
6. Congratulations PCORI on a 10-Year Reauthorization!
PIPC and members of Friends of PCORI Reauthorization applauded Congress for extending funding for the Patient-Centered Outcomes Research Institute (PCORI) for an additional ten years. PIPC Chairman and Friends of PCORI Reauthorization co-chair Tony Coelho stated, "I am pleased that Congress responded to the strong support of 200 plus stakeholder organizations for a long-term reauthorization of PCORI’s patient-centered mission. The next 10 years of PCORI is an opportunity to drive an efficient and informed health system that is truly patient-centered and responsive to the individual characteristics, needs and priorities of patients and people with disabilities. Going forward, as Chairman of the Partnership to Improve Patient Care, I believe the increased representation of payers on PCORI’s Board and the language permitting consideration of cost makes it even more important for Congress to act on the National Council on Disability’s recommendations to strengthen existing Medicare safeguards against government use of flawed cost-effectiveness standards such as quality-adjusted life years. In addition, we plan to continue to work with PCORI to amplify the voices of patients and people with disabilities in determining their research priorities and agenda to ensure balanced representation.” The Honorable Dr. Phil Gingrey, co-chair of Friends of PCORI Reauthorization, stated, "I was pleased to co-chair the bipartisan Friends of PCORI Reauthorization to ensure that the great work of PCORI continued for another decade. As a former Member of Congress and physician, I applaud the continued investment in PCORI as it is the only research organization dedicated to funding studies that compare care approaches to determine what works best, for whom, and under which circumstances. Importantly, PCORI’s work highlights the importance of shared decision making, which encourages patients to more actively participate in their care decisions, increasing the chance that they will follow through and be satisfied with the outcomes.” Click here to view the full press release.
7. Video: Partnerships May Lead to Better Engagement
In a new video from PCORI, Giselle Corbie-Smith shares the value of creating partnerships between different stakeholders, like patients, community partners, and researchers, to facilitate better engagement in healthcare research. Click here to watch the video.
8. FH Call to Action
The Familial Hypercholesterolemia (FH) Foundation, alongside the World Health Foundation, published a global call to action for the condition. "Familial hypercholesterolemia represents an unprecedented opportunity for prevention of heart disease around the world," said Katherine Wilemon, Founder and CEO of the FH Foundation. "Our aim initiating this Call to Action is to highlight that we have had the scientific understanding, and effective therapies, to stop inherited coronary heart disease, but have allowed generations of families to go uncared for. It is time to address Familial hypercholesterolemia as a public health priority." Click here to read the paper. Click here to read the press release.
9. PIPC Joins Leading Patient Groups in Comment Letter on TennCare II Demonstration
Twenty groups representing patients and people with disabilities submitted a comment letter to the Centers for Medicare & Medicaid Services (CMS) in response to Tennessee’s proposed amendment to the TennCare Demonstration, TennCare II Demonstration, Amendment 42. The groups expressed concern that adopting a closed formulary opens the door to evaluating the “value” of a drug using cost-effectiveness analyses based on the discriminatory quality-adjusted life year, which would lead to limited access to needed treatments for patients and people with disabilities. "Our goal is for TennCare to be centered on the needs, outcomes, and priorities of patients and people with disabilities; therefore, we oppose opening the door in Tennessee to limited formularies and the use of cost-effectiveness analyses based on the QALY and similar metrics, which would lead to discrimination and restricted access to care," the letter states. Click here to read the letter.
10. PIPC Submits Feedback on 'Cures 2.0' Legislation
In a letter to Reps. Diana DeGette (D-CO) and Fred Upton (R-MI), the Partnership to Improve Patient Care offered feedback on the next iteration of the 21st Century Cures Act — “Cures 2.0.” PIPC Chairman Tony Coelho encouraged the lawmakers to consider that role that the Patient-Centered Outcomes Research Institute and high quality shared decision making can play in supporting high-value, personalized health care decision making in the legislation. He also urged consideration of the recent report issued by the National Council on Disability calling on policymakers to avoid use of the discriminatory quality-adjusted life years (QALY) metric. “We look forward to working with you to ensure that Cures 2.0 is centered on patients and people with disabilities and supports the goal of innovative treatments reaching those who need them most.,” wrote Chairman Coelho. “We are committed to working with you to advance a personalized, informed health system that works to ensure patients access treatments that work for them, and do not fail them.” Click here to read the letter.
11. Alliance for Aging Research Letter on Current Drug Proposals
In a letter to congressional leaders, the Alliance for Aging Research (AAR) explained which policies currently under consideration would help seniors and which would ultimately serve to hinder access to quality care. In particular, AAR expressed its support for an out-of-pocket cap in Medicare Part D and "smoothing" to help seniors afford costly medications that could reach the catastrophic threshold on just one fill. The Alliance told Congress that the use of an international pricing index (IPI), reliance on QALYs to make coverage determinations, and ICER's QALY-based analyses are unacceptable. " While price competition with other countries seems reasonable on its face, in practice it would have a disproportionately negative effect on companies that have newer or more innovative products on the market, often for what are the most challenging diseases. The IPI would also effectively endorse the use here in the U.S. of discriminatory cost-effectiveness standards used by foreign governments. Many of the referenced countries, such as the U.K., Canada, and Greece, make drug reimbursement and coverage decisions based on cost-effectiveness assessments tied to the QALY. These QALY assessments assign a financial value to the patients for whom a given treatment is intended. If the group is sicker, older, and/or disabled, the value is less. When applied to health care decisionmaking, the results can mean that some patients, people with disabilities, veterans, and seniors are deemed “too expensive” to receive care," AAR wrote. Click here to read the letter.
12. 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.
13. ICER's QALY-Based Study Topics: Hemophilia A, Beta Thalassemia, Acute Migraine, Arthritis, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis
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. Please note the following upcoming formal ICER deadlines per their website:
14. Upcoming Events and Webinars
Webinar: Navigating Low-Value Care: A Roadmap for Stakeholders
January 15, 2020
Click here for details.
HMPF Advocacy Forum: ICER Acute Migraine Treatment Review
January 22, 2020
Click here for details.
PCORI Cycle 1 2020 Improving Methods Applicant Town Hall
January 22, 2020
Click here for details.
Real World Evidence and Data: A Tufts Study of 30 Pharma Companies
January 23, 2020
Click here for details.
PCORI Cycle 1 2020 Broad PFA Applicant Town Hall
January 23, 2020
Click here for details.
Patient Registries, Real World Evidence and HEOR
January 27-28, 2020
Click here for details.
PCORI Board of Governors Meeting
January 28, 2020
Click here for details.
PCORI Advisory Panel on Patient Engagement Winter 2020 Meeting
January 31, 2020
Click here for details.
PCORI Board of Governors Meeting
February 25, 2020
Click here for details.
FT US Pharma and Biotech Summit 2020
May 14, 2020
Click here for details.
15. Medical Journal Articles
Who Assigns Value in Value-Based Insurance Design?, click here to view.
Does the Institute for Clinical and Economic Review Revise Its Findings in Response to Industry Comments?, click here to view.
The Patient-Centered Outcomes Research Network Antibiotics and Childhood Growth Study: Implementing Patient Data Linkage, click here to view.
Viewpoint: Adding Patient-Reported Outcomes to Medicare's Oncology Value-Based Payment Model, click here to view.
Beyond Involvement: Multiple Methods and Purposes of Shared Decision Making, click here to view.
Behavioral Economic Insights to Improve Medication Adherence in Adults with Chronic Conditions: A Scoping Review, click here to view.
The Need for Clinical Judgement in the Application of Evidence-Based Medicine, click here to view.
Building Capacity in Evidence-Based Medicine in Low-Income and Middle-Income Countries: Problems and Potential Solutions, click here to view.
A Review and Synthesis of Frameworks for Engagement in Health Research to Identify Concepts of Knowledge User Engagement, click here to view.
Characteristics of Public Comments Submitted to State Health Technology Assessment Programs in Oregon and Washington, click here to view.
16. AHRQ Effective Program Updates
Research Protocol: Treatments for Acute Episodic Migraine, click here to view.
Research Protocol: Mixed Methods Review: Integrating Palliative Care With Chronic Disease Management in Ambulatory Care, click here to view.
Online Training for SRDR+ Now Available at the Evidence Synthesis Academy, click here to read more.
OPEN FOR COMMENT THROUGH 1/17/2020: Key Questions: Prehabilitation and Rehabilitation for Major Joint Replacement Surgery, click here to view.
Research Protocol: Treatments for Acute Pain: A Systematic Review, click here to view.
Research Report: Registries for Evaluating Patient Outcomes: A User's Guide, click here to view.
Research Protocol: Management of High-Need, High-Cost Patients: A Realist and Systematic Review, click here to view.
Systematic Review: Achieving Health Equity in Preventive Services, click here to view.
Research Protocol: Strategies for Patient, Family, and Caregiver Engagement, click here to view.
Research Report: Translation of a C. difficile Treatment Clinical Pathway Into Machine-Readable Clinical Decision Support Artifacts Prototyped for Electronic Health Record Integration, click here to view.
Research Protocol: Management of Primary Headache During Pregnancy, click here to view.
In an op-ed published in The Hill, Gunnar Esiason, a patient living with cystic fibrosis writes that patients like him should not be denied access to life-changing medication because an economic model says that their life is too expensive to save. Mr. Esiason is now taking the new cystic fibrosis drug Trikafta, which he says has changed his life. "QALY is not adequately able to quantify what happened in my own life — my journey from near end-stage illness and no hope for a future to correctly managed CF and entrance into an elite graduate program," he wrote. "I certainly would like to know why cost-effectiveness is being used against me. Would the analysts at ICER prefer I return to a state of recurrent hospitalizations, traumatic medical procedures, bouts of hemoptysis (coughing up blood), and the fast track towards end-stage illness and disability income? Frankly, I consider the talk of cost-effectiveness to be not only discriminatory but also pure ableism." Click here to read the article.
2. Value Our Health Sickle Cell Disease Webinar
Join Value Our Health and leading Sickle Cell Disease Organizations for a webinar on January 16, 2020 at 2pm. The webinar will provide information about the metric used to assess the value of health care treatments called the quality-adjusted life year or “QALY” as well as the primary organization conducting QALY-based value assessments called the Institute for Clinical Economic Review (ICER). Because ICER will assess the value of new sickle cell disease treatments in March, 2020, it is important for all stakeholders to understand QALYs, ICER and how those value assessments may be used by public and private payers in their decisions related to drug coverage and formularies. Learn more here, and RSVP to [email protected].
3. California Embracing “Most-Favored Nation” Concept Relying on International Drug Prices Based on QALYs and Similar Metrics
California’s Administration will propose to establish a single market for drug pricing within the state for all purchasers—Medi-Cal, California Public Employees’ Retirement System, Covered California, private insurers, self-insured employers, and others— in which California would invoke a most-favored-nation clause requiring manufacturers to offer prices at or below the price offered to "any other state, nation, or global purchaser.” In doing so, California would be referencing prices in other countries that use quality-adjusted life years (QALYs) and similar metrics to determine the cost effectiveness and coverage of drugs. As stated by the National Council on Disability, "In this report, NCD found sufficient evidence of the discriminatory effects of QALYs to warrant concern, including concerns raised by bioethicists, patient rights groups, and disability rights advocates about the limited access to lifesaving medications for chronic illnesses in countries where QALYs are frequently used. In addition, QALY-based programs have been found to violate the Americans with Disabilities Act.” Click here to view the California plan. Click here to view the NCD report calling on policymakers to "refrain from pursuing means of reducing Medicare and Medicaid prescription drug costs that attempt to model US pricing after the pricing in other countries, which may heavily rely on QALYs and often deny people with disabilities access to needed care.”
4. Recap of the 10th Annual PIPC Forum
PIPC held its 10th annual PIPC Forum on December 16, 2019. The Forum featured leading patient and disability advocates and technical health policy experts discussing the topic of international reference pricing. The presenters expressed concerns about discrimination and detrimental impacts on innovation should the United States move in the direction of using international reference pricing or cost-effectiveness analyses based on the QALY to address the cost of prescription drugs. Read a complete summary and view a video of the 10th Annual PIPC Forum here.
5. WSJ Editorial: Where You Want to Get Cancer
The Wall Street Journal published an editorial about recent decreases in the cancer death rate and remarkable advances in survival rates for many types of cancer. The editorial board noted that discriminatory policies in other countries mean that their cancer death rates are higher than those in the U.S. as patients cannot access lifesaving medication. "[G]overnment rationing and price controls on drugs are one major reason that countries with socialized medicine like the United Kingdom have lower cancer survival rates than the U.S. The age-adjusted cancer mortality rate is about 20% higher in the U.K and 10% higher in Canada and France than in the U.S. Survival rates for hard-to-treat cancers are also higher in the U.S. than in most countries with nationalized health systems," they wrote. Click here to read the article.
6. Congratulations PCORI on a 10-Year Reauthorization!
PIPC and members of Friends of PCORI Reauthorization applauded Congress for extending funding for the Patient-Centered Outcomes Research Institute (PCORI) for an additional ten years. PIPC Chairman and Friends of PCORI Reauthorization co-chair Tony Coelho stated, "I am pleased that Congress responded to the strong support of 200 plus stakeholder organizations for a long-term reauthorization of PCORI’s patient-centered mission. The next 10 years of PCORI is an opportunity to drive an efficient and informed health system that is truly patient-centered and responsive to the individual characteristics, needs and priorities of patients and people with disabilities. Going forward, as Chairman of the Partnership to Improve Patient Care, I believe the increased representation of payers on PCORI’s Board and the language permitting consideration of cost makes it even more important for Congress to act on the National Council on Disability’s recommendations to strengthen existing Medicare safeguards against government use of flawed cost-effectiveness standards such as quality-adjusted life years. In addition, we plan to continue to work with PCORI to amplify the voices of patients and people with disabilities in determining their research priorities and agenda to ensure balanced representation.” The Honorable Dr. Phil Gingrey, co-chair of Friends of PCORI Reauthorization, stated, "I was pleased to co-chair the bipartisan Friends of PCORI Reauthorization to ensure that the great work of PCORI continued for another decade. As a former Member of Congress and physician, I applaud the continued investment in PCORI as it is the only research organization dedicated to funding studies that compare care approaches to determine what works best, for whom, and under which circumstances. Importantly, PCORI’s work highlights the importance of shared decision making, which encourages patients to more actively participate in their care decisions, increasing the chance that they will follow through and be satisfied with the outcomes.” Click here to view the full press release.
7. Video: Partnerships May Lead to Better Engagement
In a new video from PCORI, Giselle Corbie-Smith shares the value of creating partnerships between different stakeholders, like patients, community partners, and researchers, to facilitate better engagement in healthcare research. Click here to watch the video.
8. FH Call to Action
The Familial Hypercholesterolemia (FH) Foundation, alongside the World Health Foundation, published a global call to action for the condition. "Familial hypercholesterolemia represents an unprecedented opportunity for prevention of heart disease around the world," said Katherine Wilemon, Founder and CEO of the FH Foundation. "Our aim initiating this Call to Action is to highlight that we have had the scientific understanding, and effective therapies, to stop inherited coronary heart disease, but have allowed generations of families to go uncared for. It is time to address Familial hypercholesterolemia as a public health priority." Click here to read the paper. Click here to read the press release.
9. PIPC Joins Leading Patient Groups in Comment Letter on TennCare II Demonstration
Twenty groups representing patients and people with disabilities submitted a comment letter to the Centers for Medicare & Medicaid Services (CMS) in response to Tennessee’s proposed amendment to the TennCare Demonstration, TennCare II Demonstration, Amendment 42. The groups expressed concern that adopting a closed formulary opens the door to evaluating the “value” of a drug using cost-effectiveness analyses based on the discriminatory quality-adjusted life year, which would lead to limited access to needed treatments for patients and people with disabilities. "Our goal is for TennCare to be centered on the needs, outcomes, and priorities of patients and people with disabilities; therefore, we oppose opening the door in Tennessee to limited formularies and the use of cost-effectiveness analyses based on the QALY and similar metrics, which would lead to discrimination and restricted access to care," the letter states. Click here to read the letter.
10. PIPC Submits Feedback on 'Cures 2.0' Legislation
In a letter to Reps. Diana DeGette (D-CO) and Fred Upton (R-MI), the Partnership to Improve Patient Care offered feedback on the next iteration of the 21st Century Cures Act — “Cures 2.0.” PIPC Chairman Tony Coelho encouraged the lawmakers to consider that role that the Patient-Centered Outcomes Research Institute and high quality shared decision making can play in supporting high-value, personalized health care decision making in the legislation. He also urged consideration of the recent report issued by the National Council on Disability calling on policymakers to avoid use of the discriminatory quality-adjusted life years (QALY) metric. “We look forward to working with you to ensure that Cures 2.0 is centered on patients and people with disabilities and supports the goal of innovative treatments reaching those who need them most.,” wrote Chairman Coelho. “We are committed to working with you to advance a personalized, informed health system that works to ensure patients access treatments that work for them, and do not fail them.” Click here to read the letter.
11. Alliance for Aging Research Letter on Current Drug Proposals
In a letter to congressional leaders, the Alliance for Aging Research (AAR) explained which policies currently under consideration would help seniors and which would ultimately serve to hinder access to quality care. In particular, AAR expressed its support for an out-of-pocket cap in Medicare Part D and "smoothing" to help seniors afford costly medications that could reach the catastrophic threshold on just one fill. The Alliance told Congress that the use of an international pricing index (IPI), reliance on QALYs to make coverage determinations, and ICER's QALY-based analyses are unacceptable. " While price competition with other countries seems reasonable on its face, in practice it would have a disproportionately negative effect on companies that have newer or more innovative products on the market, often for what are the most challenging diseases. The IPI would also effectively endorse the use here in the U.S. of discriminatory cost-effectiveness standards used by foreign governments. Many of the referenced countries, such as the U.K., Canada, and Greece, make drug reimbursement and coverage decisions based on cost-effectiveness assessments tied to the QALY. These QALY assessments assign a financial value to the patients for whom a given treatment is intended. If the group is sicker, older, and/or disabled, the value is less. When applied to health care decisionmaking, the results can mean that some patients, people with disabilities, veterans, and seniors are deemed “too expensive” to receive care," AAR wrote. Click here to read the letter.
12. 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: With the treatment not covered by the government, Toronto parents turn to a drug dose lottery for their child with spinal muscular atrophy as a last resort.
- New Zealand: Parents of a child with cystic fibrosis raise awareness of Pharmac's failure to provide access to lifesaving drugs. Click here and here to read more. With Pharmac refusing to fund lifesaving cancer drugs, one patient forced to resort to crowdfunding to be able to live and spend more time with his family. Following forced epilepsy medication switch, one dad died days before Christmas. Click here to read another story of a New Zealand man who died after Pharmac switched his epilepsy drug. Following a fifth death due to Pharmac's forcing epilepsy patients to switch medications, there are renewed calls for an inquiry. Click here to read more.
- United Kingdom: The number of cancer patients treated in target time frames by NHS is at a record low.
13. ICER's QALY-Based Study Topics: Hemophilia A, Beta Thalassemia, Acute Migraine, Arthritis, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis
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. Please note the following upcoming formal ICER deadlines per their website:
- Ulcerative Colitis: Research Protocol available. 2/3/2020: Model Analysis Plan.
- Cystic Fibrosis: Model Analysis Plan available. 2/20/2020: Draft Evidence Report and Draft Voting Questions. Meeting 4/30/2020: CTAF will convene to deliberate and vote on evidence presented in ICER's report on treatments for cystic fibrosis.
- Sickle Cell Disease: Model Analysis Plan available. 1/22/2020: Draft Evidence Report and Draft Voting Questions. Meeting 3/26/2020: New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for sickle cell disease.
- Arthritis: Final Evidence Report and Meeting Summary AVAILABLE.
- Acute Migraine: Revised Voting Questions, Evidence Report, and Responses to Public Comments AVAILABLE. Meeting 1/23/2020: Midwest CEPAC to review ICER's assessment of acute migraine treatments.
- 2020 Value Assessment Framework: 1/31/2020: Final Framework Adaptations.
- Non-Alcoholic Steatohepatitis: Revised Scoping Document Available. 1/15/2020: Research Protocol.
- Beta Thalassemia: Draft Scoping Document AVAILABLE, comment period OPEN through 1/27/2020
- Hemophilia A: Open Input Period through 1/22/2020. 1/24/2020: Draft Scoping Document.
14. Upcoming Events and Webinars
Webinar: Navigating Low-Value Care: A Roadmap for Stakeholders
January 15, 2020
Click here for details.
HMPF Advocacy Forum: ICER Acute Migraine Treatment Review
January 22, 2020
Click here for details.
PCORI Cycle 1 2020 Improving Methods Applicant Town Hall
January 22, 2020
Click here for details.
Real World Evidence and Data: A Tufts Study of 30 Pharma Companies
January 23, 2020
Click here for details.
PCORI Cycle 1 2020 Broad PFA Applicant Town Hall
January 23, 2020
Click here for details.
Patient Registries, Real World Evidence and HEOR
January 27-28, 2020
Click here for details.
PCORI Board of Governors Meeting
January 28, 2020
Click here for details.
PCORI Advisory Panel on Patient Engagement Winter 2020 Meeting
January 31, 2020
Click here for details.
PCORI Board of Governors Meeting
February 25, 2020
Click here for details.
FT US Pharma and Biotech Summit 2020
May 14, 2020
Click here for details.
15. Medical Journal Articles
Who Assigns Value in Value-Based Insurance Design?, click here to view.
Does the Institute for Clinical and Economic Review Revise Its Findings in Response to Industry Comments?, click here to view.
The Patient-Centered Outcomes Research Network Antibiotics and Childhood Growth Study: Implementing Patient Data Linkage, click here to view.
Viewpoint: Adding Patient-Reported Outcomes to Medicare's Oncology Value-Based Payment Model, click here to view.
Beyond Involvement: Multiple Methods and Purposes of Shared Decision Making, click here to view.
Behavioral Economic Insights to Improve Medication Adherence in Adults with Chronic Conditions: A Scoping Review, click here to view.
The Need for Clinical Judgement in the Application of Evidence-Based Medicine, click here to view.
Building Capacity in Evidence-Based Medicine in Low-Income and Middle-Income Countries: Problems and Potential Solutions, click here to view.
A Review and Synthesis of Frameworks for Engagement in Health Research to Identify Concepts of Knowledge User Engagement, click here to view.
Characteristics of Public Comments Submitted to State Health Technology Assessment Programs in Oregon and Washington, click here to view.
16. AHRQ Effective Program Updates
Research Protocol: Treatments for Acute Episodic Migraine, click here to view.
Research Protocol: Mixed Methods Review: Integrating Palliative Care With Chronic Disease Management in Ambulatory Care, click here to view.
Online Training for SRDR+ Now Available at the Evidence Synthesis Academy, click here to read more.
OPEN FOR COMMENT THROUGH 1/17/2020: Key Questions: Prehabilitation and Rehabilitation for Major Joint Replacement Surgery, click here to view.
Research Protocol: Treatments for Acute Pain: A Systematic Review, click here to view.
Research Report: Registries for Evaluating Patient Outcomes: A User's Guide, click here to view.
Research Protocol: Management of High-Need, High-Cost Patients: A Realist and Systematic Review, click here to view.
Systematic Review: Achieving Health Equity in Preventive Services, click here to view.
Research Protocol: Strategies for Patient, Family, and Caregiver Engagement, click here to view.
Research Report: Translation of a C. difficile Treatment Clinical Pathway Into Machine-Readable Clinical Decision Support Artifacts Prototyped for Electronic Health Record Integration, click here to view.
Research Protocol: Management of Primary Headache During Pregnancy, click here to view.
