In This Week’s Issue:
1. Please Join PIPC For Our 10th Annual Forum!, click here to view the invitation.
2. National Council on Disability Report on QALYs, click here to read the report.
3. EveryLife Foundation Briefing with the Rare Disease Caucus, click here to learn more and register.
4. International Reference Pricing Would Lead to Fewer New Medicines For People with Disabilities, Report Finds, click here to see the report.
5. PCORI Reauthorization Discussions Advance, see details below.
6. House Begins 21st Century Cures 2.0 Effort, see details below.
7. PIPC Urges Policymakers to Protect Patients and People with Disabilities, see below for details.
8. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
9. ICER's QALY-Based Study Topics: Acute Migraine, Type 2 Diabetes, Arthritis, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, click here to provide patient input.
10. Upcoming Events and Webinars, see details below.
11. Medical Journal Articles, see details below.
12. AHRQ Effective Program Updates, see details below.
1. Please Join PIPC For Our 10th Annual Forum!, click here to view the invitation.
2. National Council on Disability Report on QALYs, click here to read the report.
3. EveryLife Foundation Briefing with the Rare Disease Caucus, click here to learn more and register.
4. International Reference Pricing Would Lead to Fewer New Medicines For People with Disabilities, Report Finds, click here to see the report.
5. PCORI Reauthorization Discussions Advance, see details below.
6. House Begins 21st Century Cures 2.0 Effort, see details below.
7. PIPC Urges Policymakers to Protect Patients and People with Disabilities, see below for details.
8. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
9. ICER's QALY-Based Study Topics: Acute Migraine, Type 2 Diabetes, Arthritis, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, click here to provide patient input.
10. Upcoming Events and Webinars, see details below.
11. Medical Journal Articles, see details below.
12. AHRQ Effective Program Updates, see details below.
1. Please Join PIPC For Our 10th Annual Forum!
The Partnership to Improve Patient Care (PIPC) will hold its 10th Annual Forum on December 16, 2019 from 12:00 - 2:00 p.m. at the Reserve Officers Association in Washington, DC. We will be joined by Ana Torres-Davis who will share an overview of the National Council on disability’s important and timely recent report: “Quality Adjusted Life Years and the Devaluation of Life with a Disability.” We will also learn about international healthcare systems from real patients, caregivers, and technical policy experts. RSVP to [email protected]. Click here to view the invitation.
2. National Council on Disability Report on QALYs
The National Council on Disability issued a new report assessing the discriminatory effects of using QALYs to determine treatment coverage. "In an effort to lower their healthcare costs, public and private health insurance providers have utilized the Quality Adjusted Life Year (QALY) to determine the cost-effectiveness of medications and treatment," the Council wrote. "QALYs place a lower value on treatments which extend the lives of people with chronic illnesses and disabilities. 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." In addition, the report calls on Congress to ban the use of QALYs in Medicare and Medicaid and recommended that the administration refrain from modeling US pricing after the pricing in other countries, calling on the administration specifically to rescind its proposed rule to use an international pricing index in Medicare Part B. Click here to read the report.
3. EveryLife Foundation Briefing with the Rare Disease Caucus
The EveryLife Foundation for Rare Diseases for The Rare Disease Caucus is holding a briefing hosted in partnership with the Rare Disease Congressional Caucus. The briefing will share important information and research on the economic burden of rare diseases. It will be held on December 4th at 11:30 am in Rayburn House Office Building, Room 2075. Click here to learn more and register.
4. International Reference Pricing Would Lead to Fewer New Medicines For People with Disabilities
Vital Transformations modeled the impact of a proposal to institute international reference pricing on the biotechnology sector and found that revenue reductions would particularly hurt small, innovative firms that are developing the cures of tomorrow. "Targeted therapies, medicines requiring long-term outcomes, and medicines with lower revenue potential are among the types most likely to be affected by H.R.3’s drastic change to economic incentives. This includes areas where the science is toughest, such as in Alzheimer’s and ALS," the authors wrote. Click here to see the report.
5. PCORI Reauthorization Discussions Advance
Over the summer, the House Energy and Commerce and the primary jurisdiction House Ways and Means Committees marked up legislation to reauthorize the Patient-Centered Outcomes Research Institute (PCORI), for 3 years and for 7 years with limited changes to its underlying priorities, respectively. While PCORI reauthorization was included in legislation to extend various health programs until November 21, 2019, the PCOR trust fund was extended without extending the funding for it. Most recently, Senators Bill Cassidy (R-LA), Mark Warner (D-VA), Shelley Moore Capito (R-WV) and Chris Van Hollen (D-MD) introduced a bill reauthorizing PCORI with full funding for 10 years that proposed additional policy changes for PCORI. PIPC provided comments on the discussion draft opposing added payer seats on the PCORI Board Governors. Friends of PCORI Reauthorization, a coalition of 184 organizations, has consistently communicated to both House and Senate leaders support for PCORI to remain patient-centered, and specifically support for a 10 year, fully funded reauthorization that continues to direct PCORI to fund comparative clinical effectiveness research. Click here to view PIPC’s comments or click here for information about Friends of PCORI Reauthorization.
6. House Begins 21st Century Cures 2.0 Effort
The lead authors of 21st Century Cures are soliciting feedback on a second iteration of the legislation, which they say will focus on issues such as digital health, coverage of innovative treatments, the use of real-world evidence (RWE), and improving education for caregivers. The lawmakers vision of so-called Cures 2.0 legislation is laid out in a new request for information (RFI) from Reps. Diana DeGette (D-CO) and Fred Upton (R-MI), who are soliciting feedback by December 16 on ideas that should be considered as part of a redux of their landmark bipartisan initiative. While the Department of Health and Human Services (HHS) is still fulfilling provisions of the original law, enacted in 2016, Rep. Upton recently indicated that at least part of the focus of Cures 2.0 will be on “how to make the law work better.”
The bipartisan pair of Reps. DeGette and Upton launched a public campaign for the 21st Century Cures Act five years ago with a similar request for ideas and support and spent two years crafting the bill, which was enacted in December 2016. The legislation, signed by President Barack Obama in the final weeks of his presidency, was aimed at speeding research and therapies for patients. It bolstered aid to the NIH, gave FDA new powers and responsibilities, and required more information sharing. Click here to view the call to action issued to kick off the Cures 2.0 effort.
7. PIPC Urges Policymakers to Protect Patients and People with Disabilities
PIPC Chairman Tony Coelho expressed concern about policies placing hard-fought existing patient protections under serious threat, especially those relying on policy decisions made in other countries that we know establish prices based on discriminatory cost-effectiveness standards (such as Quality-Adjusted Life Years or QALYs) and similar average metrics. PIPC supports policies to improve care and affordability for patients, adopt and extend safeguards in current statute that prohibit application of discriminatory cost-effectiveness thresholds in Medicare (whether done overtly by Medicare or covertly via “comparative effectiveness analysis” or reference to foreign countries’ use of these thresholds) and codify criteria for patient-centeredness called for in CMMI’s statute. Click here to view prior stakeholder letter on referencing International pricing. Click here to view information on Value Our Health.
8. 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.
9. ICER's QALY-Based Study Topics: Acute Migraine, Type 2 Diabetes, 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:
10. Upcoming Events and Webinars
The Patient's Stake in the Next Generation of Value-Based Arrangements
December 4, 2019
Click here for details.
PCORI Stakeholder Workshop on Maternal Morbidity and Mortality
December 5, 2019
Click here for details.
Advisory Panel on Healthcare Delivery and Disparities Research Winter 2019 Meeting
December 6, 2019
Click here for details.
Webinar: Providing Quality Benefits for Employees with Cardiovascular Disease and Other Chronic Conditions
December 10, 2019
Click here for details.
Webinar: Request for Proposals – Addressing Barriers to Medication Access
December 12, 2019
Click here for details.
PCORI Stakeholder Workshop on Suicide Prevention
December 18, 2019
Click here for details.
EveryLife Foundation’s A Rare Affair fundraiser at the JP Morgan Healthcare Conference
January 12, 2020
Click here for details.
Patient Registries, Real World Evidence and HEOR
January 27-28, 2020
Click here for details.
FT US Pharma and Biotech Summit 2020
May 14, 2020
Click here for details.
11. Medical Journal Articles
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.
Engaging the Patient: Patient-Centered Research, click here to view.
Little Consistency In Evidence Cited By Commercial Plans For Specialty Drug Coverage, click here to view.
Ten Ways to Optimise Evidence-Based Policy, click here to view.
Mapping of Health Technology Assessment in China: Situation Analysis and International Comparison, click here to view.
Reflections on the ISPOR Special Task Force on U.S. Value Frameworks: Implications of a Health Economics Approach for Managed Care Pharmacy, click here to view.
12. AHRQ Effective Program Updates
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.
OPEN FOR COMMENT THROUGH 12/20/2019: Key Questions: Prehospital Airway Management: A Systematic Review, 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.
Research Report: Facilitating the Implementation of EPC Reports in Learning Health Systems Engaged in Quality Initiatives: an EPC Pilot Project on ADHD, click here to view.
Research Protocol: Interventions for Dyspnea in Patients with Advanced Cancer, click here to view.
Technical Brief: Treatment for Acute Pain: An Evidence Map, click here to view.
Research Report: Pilot To Promote Entry of Structured Data Into the Systematic Review Data Repository (SRDR), click here to view.
The Partnership to Improve Patient Care (PIPC) will hold its 10th Annual Forum on December 16, 2019 from 12:00 - 2:00 p.m. at the Reserve Officers Association in Washington, DC. We will be joined by Ana Torres-Davis who will share an overview of the National Council on disability’s important and timely recent report: “Quality Adjusted Life Years and the Devaluation of Life with a Disability.” We will also learn about international healthcare systems from real patients, caregivers, and technical policy experts. RSVP to [email protected]. Click here to view the invitation.
2. National Council on Disability Report on QALYs
The National Council on Disability issued a new report assessing the discriminatory effects of using QALYs to determine treatment coverage. "In an effort to lower their healthcare costs, public and private health insurance providers have utilized the Quality Adjusted Life Year (QALY) to determine the cost-effectiveness of medications and treatment," the Council wrote. "QALYs place a lower value on treatments which extend the lives of people with chronic illnesses and disabilities. 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." In addition, the report calls on Congress to ban the use of QALYs in Medicare and Medicaid and recommended that the administration refrain from modeling US pricing after the pricing in other countries, calling on the administration specifically to rescind its proposed rule to use an international pricing index in Medicare Part B. Click here to read the report.
3. EveryLife Foundation Briefing with the Rare Disease Caucus
The EveryLife Foundation for Rare Diseases for The Rare Disease Caucus is holding a briefing hosted in partnership with the Rare Disease Congressional Caucus. The briefing will share important information and research on the economic burden of rare diseases. It will be held on December 4th at 11:30 am in Rayburn House Office Building, Room 2075. Click here to learn more and register.
4. International Reference Pricing Would Lead to Fewer New Medicines For People with Disabilities
Vital Transformations modeled the impact of a proposal to institute international reference pricing on the biotechnology sector and found that revenue reductions would particularly hurt small, innovative firms that are developing the cures of tomorrow. "Targeted therapies, medicines requiring long-term outcomes, and medicines with lower revenue potential are among the types most likely to be affected by H.R.3’s drastic change to economic incentives. This includes areas where the science is toughest, such as in Alzheimer’s and ALS," the authors wrote. Click here to see the report.
5. PCORI Reauthorization Discussions Advance
Over the summer, the House Energy and Commerce and the primary jurisdiction House Ways and Means Committees marked up legislation to reauthorize the Patient-Centered Outcomes Research Institute (PCORI), for 3 years and for 7 years with limited changes to its underlying priorities, respectively. While PCORI reauthorization was included in legislation to extend various health programs until November 21, 2019, the PCOR trust fund was extended without extending the funding for it. Most recently, Senators Bill Cassidy (R-LA), Mark Warner (D-VA), Shelley Moore Capito (R-WV) and Chris Van Hollen (D-MD) introduced a bill reauthorizing PCORI with full funding for 10 years that proposed additional policy changes for PCORI. PIPC provided comments on the discussion draft opposing added payer seats on the PCORI Board Governors. Friends of PCORI Reauthorization, a coalition of 184 organizations, has consistently communicated to both House and Senate leaders support for PCORI to remain patient-centered, and specifically support for a 10 year, fully funded reauthorization that continues to direct PCORI to fund comparative clinical effectiveness research. Click here to view PIPC’s comments or click here for information about Friends of PCORI Reauthorization.
6. House Begins 21st Century Cures 2.0 Effort
The lead authors of 21st Century Cures are soliciting feedback on a second iteration of the legislation, which they say will focus on issues such as digital health, coverage of innovative treatments, the use of real-world evidence (RWE), and improving education for caregivers. The lawmakers vision of so-called Cures 2.0 legislation is laid out in a new request for information (RFI) from Reps. Diana DeGette (D-CO) and Fred Upton (R-MI), who are soliciting feedback by December 16 on ideas that should be considered as part of a redux of their landmark bipartisan initiative. While the Department of Health and Human Services (HHS) is still fulfilling provisions of the original law, enacted in 2016, Rep. Upton recently indicated that at least part of the focus of Cures 2.0 will be on “how to make the law work better.”
The bipartisan pair of Reps. DeGette and Upton launched a public campaign for the 21st Century Cures Act five years ago with a similar request for ideas and support and spent two years crafting the bill, which was enacted in December 2016. The legislation, signed by President Barack Obama in the final weeks of his presidency, was aimed at speeding research and therapies for patients. It bolstered aid to the NIH, gave FDA new powers and responsibilities, and required more information sharing. Click here to view the call to action issued to kick off the Cures 2.0 effort.
7. PIPC Urges Policymakers to Protect Patients and People with Disabilities
PIPC Chairman Tony Coelho expressed concern about policies placing hard-fought existing patient protections under serious threat, especially those relying on policy decisions made in other countries that we know establish prices based on discriminatory cost-effectiveness standards (such as Quality-Adjusted Life Years or QALYs) and similar average metrics. PIPC supports policies to improve care and affordability for patients, adopt and extend safeguards in current statute that prohibit application of discriminatory cost-effectiveness thresholds in Medicare (whether done overtly by Medicare or covertly via “comparative effectiveness analysis” or reference to foreign countries’ use of these thresholds) and codify criteria for patient-centeredness called for in CMMI’s statute. Click here to view prior stakeholder letter on referencing International pricing. Click here to view information on Value Our Health.
8. 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: Health Canada will not negotiate on cystic fibrosis drugs, leaving patients in the lurch. Forced switching to alternative drugs may hurt Canadian patients.
- New Zealand: New Zealand women are dying as they await coverage of a cancer drug. Pharmac has been assessing a lifesaving treatment for six years, leaving patients behind. Patient imports a cancer drug from Bangladesh because Pharmac will not cover it. Patients call for an inquiry over Pharmac's switching epilepsy drug coverage. Victims of epilepsy drug switch say they should be compensated. Pharmac violated patient rights during its epilepsy drug switch.
- United Kingdom: NICE delaying approval of less-toxic, lifesaving cancer drug.
9. ICER's QALY-Based Study Topics: Acute Migraine, Type 2 Diabetes, 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: Revised Scoping Document available. 12/11/2019: Research Protocol.
- Cystic Fibrosis: Revised Scoping Document available. 12/5/2019: Research Protocol.
- 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 Report, Revised Voting Questions, and Responses to Public Comments AVAILABLE. Meeting 12/9/2019: CTAF to an update to its 2017 rheumatoid arthritis assessment.
- Type 2 Diabetes: 12/9/2019: Final Evidence Report and Meeting Summary. Meeting 11/14/2019: The New England CEPAC convened to deliberate on ICER's review of oral semaglutide for the treatment of type 2 diabetes.
- Acute Migraine: Draft Evidence Report and Draft Voting Questions AVAILABLE, Comment Period OPEN through 12/6/2019. 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.
10. Upcoming Events and Webinars
The Patient's Stake in the Next Generation of Value-Based Arrangements
December 4, 2019
Click here for details.
PCORI Stakeholder Workshop on Maternal Morbidity and Mortality
December 5, 2019
Click here for details.
Advisory Panel on Healthcare Delivery and Disparities Research Winter 2019 Meeting
December 6, 2019
Click here for details.
Webinar: Providing Quality Benefits for Employees with Cardiovascular Disease and Other Chronic Conditions
December 10, 2019
Click here for details.
Webinar: Request for Proposals – Addressing Barriers to Medication Access
December 12, 2019
Click here for details.
PCORI Stakeholder Workshop on Suicide Prevention
December 18, 2019
Click here for details.
EveryLife Foundation’s A Rare Affair fundraiser at the JP Morgan Healthcare Conference
January 12, 2020
Click here for details.
Patient Registries, Real World Evidence and HEOR
January 27-28, 2020
Click here for details.
FT US Pharma and Biotech Summit 2020
May 14, 2020
Click here for details.
11. Medical Journal Articles
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.
Engaging the Patient: Patient-Centered Research, click here to view.
Little Consistency In Evidence Cited By Commercial Plans For Specialty Drug Coverage, click here to view.
Ten Ways to Optimise Evidence-Based Policy, click here to view.
Mapping of Health Technology Assessment in China: Situation Analysis and International Comparison, click here to view.
Reflections on the ISPOR Special Task Force on U.S. Value Frameworks: Implications of a Health Economics Approach for Managed Care Pharmacy, click here to view.
12. AHRQ Effective Program Updates
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.
OPEN FOR COMMENT THROUGH 12/20/2019: Key Questions: Prehospital Airway Management: A Systematic Review, 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.
Research Report: Facilitating the Implementation of EPC Reports in Learning Health Systems Engaged in Quality Initiatives: an EPC Pilot Project on ADHD, click here to view.
Research Protocol: Interventions for Dyspnea in Patients with Advanced Cancer, click here to view.
Technical Brief: Treatment for Acute Pain: An Evidence Map, click here to view.
Research Report: Pilot To Promote Entry of Structured Data Into the Systematic Review Data Repository (SRDR), click here to view.
