In This Week’s Issue:
1. Congratulations PCORI on a 10-Year Reauthorization!, click here to view the full press release.
2. PIPC Joins Leading Patient Groups in Comment Letter on TennCare II Demonstration, click here to read the letter.
3. PIPC Submits Feedback on 'Cures 2.0' Legislation, click here to read the letter.
4. Alliance for Aging Research Letter on Current Drug Proposals, click here to read the letter.
5. PCORI December Board of Governors Meeting, see details below.
6. PIPC Submits Comment Letter to ICER on Draft Evidence Report for Acute Migraines, click here to read the letter
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: Beta Thalassemia, Acute Migraine, Arthritis, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, 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. Congratulations PCORI on a 10-Year Reauthorization!, click here to view the full press release.
2. PIPC Joins Leading Patient Groups in Comment Letter on TennCare II Demonstration, click here to read the letter.
3. PIPC Submits Feedback on 'Cures 2.0' Legislation, click here to read the letter.
4. Alliance for Aging Research Letter on Current Drug Proposals, click here to read the letter.
5. PCORI December Board of Governors Meeting, see details below.
6. PIPC Submits Comment Letter to ICER on Draft Evidence Report for Acute Migraines, click here to read the letter
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: Beta Thalassemia, Acute Migraine, Arthritis, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, 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. 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.
2. 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.
3. 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.
4. 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.
5. PCORI December Board of Governors Meeting
On December 9, PCORI held a Board of Governors meeting during which board members received a presentation on what PCORI has learned about patient engagement. Board members were also given an update on PCORI's work regarding cancer. Click here to see the full agenda and other meeting materials. Click here to read a transcript of the meeting.
6. PIPC Submits Comment Letter to ICER on Draft Evidence Report for Acute Migraines
In a letter to the Institute for Clinical and Economic Review (ICER), Partnership to Improve Patient Care (PIPC) Chairman Tony Coelho provided feedback on ICER's draft evidence report for acute migraines. Chairman Coelho criticized the report for making an "oversimplified assumption" that there are no mortality effects in migraine treatment, citing that a number of people with more severe types of migraine have higher rates of all-cause mortality in both men and women. He also reiterated PIPC's concern with ICER's use of the QALY, saying that it is not an appropriate methodology for use in assessments where the patient population is heterogeneous. "ICER continues to overlook outcomes that matter to patients in favor of overly simplistic QALY-based models," wrote Chairman Coelho. "We urge ICER to be more thoughtful in its model construction and take seriously the feedback from patients and clinicians who are experts in migraine attacks." Click here to read the letter.
7. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage?
Other countries are often referenced as examples of how the use of QALYs or similar cost-based thresholds impact access to care.
8. ICER's QALY-Based Study Topics: 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:
9. Upcoming Events and Webinars
EveryLife Foundation’s A Rare Affair Fundraiser at the JP Morgan Healthcare Conference
January 12, 2020
Click here for details.
Webinar: Navigating Low-Value Care: A Roadmap for Stakeholders
January 15, 2020
Click here for details.
PCORI Cycle 1 2020 Improving Methods Applicant Town Hall
January 22, 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.
10. Medical Journal Articles
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.
What Is Next for Patient Preferences in Health Technology Assessment? A Systematic Review of the Challenges, click here to view.
Commentary: One Size Does Not Always Fit All in Value Assessment, click here to view.
11. AHRQ Effective Program Updates
OPEN FOR COMMENT THROUGH 1/6/2020: Key Questions: Breast Reconstruction After Mastectomy, click here to view.
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.
Research Report: Assessing the Accuracy of Machine-Assisted Abstract Screening With DistillerAI: A User Study, click here to view.
Research Report: Performance and Usability of Machine Learning for Screening in Systematic Reviews: A Comparative Evaluation of Three Tools, click here to view.
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.
2. 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.
3. 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.
4. 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.
5. PCORI December Board of Governors Meeting
On December 9, PCORI held a Board of Governors meeting during which board members received a presentation on what PCORI has learned about patient engagement. Board members were also given an update on PCORI's work regarding cancer. Click here to see the full agenda and other meeting materials. Click here to read a transcript of the meeting.
6. PIPC Submits Comment Letter to ICER on Draft Evidence Report for Acute Migraines
In a letter to the Institute for Clinical and Economic Review (ICER), Partnership to Improve Patient Care (PIPC) Chairman Tony Coelho provided feedback on ICER's draft evidence report for acute migraines. Chairman Coelho criticized the report for making an "oversimplified assumption" that there are no mortality effects in migraine treatment, citing that a number of people with more severe types of migraine have higher rates of all-cause mortality in both men and women. He also reiterated PIPC's concern with ICER's use of the QALY, saying that it is not an appropriate methodology for use in assessments where the patient population is heterogeneous. "ICER continues to overlook outcomes that matter to patients in favor of overly simplistic QALY-based models," wrote Chairman Coelho. "We urge ICER to be more thoughtful in its model construction and take seriously the feedback from patients and clinicians who are experts in migraine attacks." Click here to read the letter.
7. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage?
Other countries are often referenced as examples of how the use of QALYs or similar cost-based thresholds impact access to care.
- Canada: Family fights for province to cover cystic fibrosis drugs. One province won't cover a cystic fibrosis drug for a toddler, citing cost effectiveness. Patients say they cannot wait for access to lifesaving cystic fibrosis drugs. A parent writes that it is incomprehensible that drugs exist that could help her child yet the government will not cover them. Province pulls funding for drug that helps Crohn's Disease and arthritis patients, leading doctors to warn of adverse effects.
- New Zealand: Epilepsy group says that Pharmac must stop switching patients to a generic following deaths. Five deaths have been linked to the switching practice. Pharmac knew of deaths linked to switching for weeks but stayed silent.
- United Kingdom: Patient forced to raise tens of thousands of pounds to cover lifesaving drug after her application to NHS was rejected three times.
8. ICER's QALY-Based Study Topics: 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: Evidence Presentation AVAILABLE. 1/9/2020: Final Evidence Report and Meeting Summary.
- Acute Migraine: Draft Evidence Report and Draft Voting Questions available. 1/8/2020: Revised Voting Questions, Evidence Report, and Responses to Public Comments. 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
9. Upcoming Events and Webinars
EveryLife Foundation’s A Rare Affair Fundraiser at the JP Morgan Healthcare Conference
January 12, 2020
Click here for details.
Webinar: Navigating Low-Value Care: A Roadmap for Stakeholders
January 15, 2020
Click here for details.
PCORI Cycle 1 2020 Improving Methods Applicant Town Hall
January 22, 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.
10. Medical Journal Articles
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.
What Is Next for Patient Preferences in Health Technology Assessment? A Systematic Review of the Challenges, click here to view.
Commentary: One Size Does Not Always Fit All in Value Assessment, click here to view.
11. AHRQ Effective Program Updates
OPEN FOR COMMENT THROUGH 1/6/2020: Key Questions: Breast Reconstruction After Mastectomy, click here to view.
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.
Research Report: Assessing the Accuracy of Machine-Assisted Abstract Screening With DistillerAI: A User Study, click here to view.
Research Report: Performance and Usability of Machine Learning for Screening in Systematic Reviews: A Comparative Evaluation of Three Tools, click here to view.
