In This Week’s Issue:
1. PCORI Reauthorization Discussions Advance, see details below.
2. Video: Risk of Modeling Other Countries’ Drug Pricing: How a Canadian Child is Forced to Get Sicker to Access Treatment, click here to watch the video.
3. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
4. PIPC Urges House to Protect Patients and People with Disabilities in Title 1 of New House Bill, see below for details.
5. Video: Role of PCORI Patient Reviewers, click here to view.
6. EveryLife Foundation Briefing with the Rare Disease Caucus, click here to learn more and register.
7. Washington Post: New Cystic Fibrosis Drug Could Turn Deadly Disease into Manageable Condition, click here to read the article.
8. ICER Studies: Acute Migraine, Type 2 Diabetes, 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. PCORI Reauthorization Discussions Advance, see details below.
2. Video: Risk of Modeling Other Countries’ Drug Pricing: How a Canadian Child is Forced to Get Sicker to Access Treatment, click here to watch the video.
3. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
4. PIPC Urges House to Protect Patients and People with Disabilities in Title 1 of New House Bill, see below for details.
5. Video: Role of PCORI Patient Reviewers, click here to view.
6. EveryLife Foundation Briefing with the Rare Disease Caucus, click here to learn more and register.
7. Washington Post: New Cystic Fibrosis Drug Could Turn Deadly Disease into Manageable Condition, click here to read the article.
8. ICER Studies: Acute Migraine, Type 2 Diabetes, 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. PCORI Reauthorization Discussions Advance
Over the summer, the House Energy and Commerce and 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) shared a discussion draft of legislation reauthorizing PCORI with full funding for 10 years that proposed additional policy changes for PCORI, and invited stakeholders to provide input on those proposed policy changes. PIPC provided comments on the discussion draft highlighting support for an expert advisory panel for high-impact research, as well as a long-term, fully funded reauthorization that protects PCORI’s patient-centered mission and extends all of the existing funding mechanisms. PIPC also urged consideration of extending the protections in Section 1182 more broadly to cover Medicaid, the CMS Innovation Center, and other reforms that would apply value judgments to national policy decisions. PIPC opposes added payer seats on the PCORI Board Governors and an accompanying shift of the selection of the Methodology Committee to the Board of 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.
2. Video: Risk of Modeling Other Countries’ Drug Pricing: How a Canadian Child is Forced to Get Sicker to Access Treatment
Access to treatments is a constant struggle for patients in countries like Canada, where access is limited based on cost effectiveness or assessments of average clinical benefit. We are sharing a video developed in Canada with a mother, Sandy, and her daughter, Laura, who has cystic fibrosis. Sandy and her daughter Laura spoke honestly and freely about their experience with the Canadian health system. It is a heartbreaking story of the requirement to get sicker in order to access a new treatment she needs due to government utilization management for drugs, e.g. fail first policies. As our country considers modeling international drug pricing policies, it demonstrates the inherent risk for patient access to care. Click here to watch the video.
3. 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.
4. PIPC Urges House to Protect Patients and People with Disabilities in Title 1 of New House Bill
Legislation has been introduced in the House of Representatives which includes certain provisions concerning patients and people with disabilities in Title 1, particularly forcing patients and people with disabilities into “one-size-fits-all” decisions about their health. PIPC Chairman Tony Coelho expressed concern that Title 1 places hard-fought existing patient protections under serious threat. It explicitly relies 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 the bill. Click here to view prior stakeholder letter on referencing International pricing. Click here for our explainer of Title I of the bill. Click here to view information on Value Our Health.
5. Video: Role of PCORI Patient Reviewers
In this video, Paul McLean describes the work of a PCORI patient peer reviewer and what to look for in a final research report. Click here to view.
6. 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.
7. Washington Post: New Cystic Fibrosis Drug Could Turn Deadly Disease into Manageable Condition
The Washington Post reported about the approval of a new drug that can treat 90 percent of patients with cystic fibrosis. Trikafta, the new combination drug, had clinical trial data so persuasive that the Food and Drug Administration (FDA) approved it five months before its deadline. Sarah Carollo, a patient with cystic fibrosis, said that “as a person living with CF, my parents had been passing on to me this fear — we always had this constant fear of when the decline was going to happen, because we knew it was going to happen." But after starting the drug, the article states, "her doctors tested her lung function and were so stunned at the improvement that they had to check whether they were really looking at the results from the right patient. Earlier this month, Carollo ran a 5K race with another patient, Laurana Blackburn, who was also taking the drug through the clinical trial." Click here to read the article.
8. ICER Studies: 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:
9. Upcoming Events and Webinars
Personalized Medicine Conference
November 13-14, 2019
Click here for details.
Stay, Safe, Side: Seizure First Aid & Priorities for the Epilepsy Community
November 14, 2019
9:30 a.m.
Rayburn House Office Building, Room 2168
RSVP to [email protected].
Webinar: The National Patient-Centered Clinical Research Network (PCORnet)
November 14, 2019
Click here for details.
2019 AUCD Annual Meeting
November 17-20, 2019
Click here for details.
Advisory Panel on Healthcare Delivery and Disparities Research Winter 2019 Meeting
December 6, 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.
FT US Pharma and Biotech Summit 2020
May 14, 2020
Click here for details.
10. Medical Journal Articles
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.
Real-World Evidence: Research Reveals a Lack of Racial Diversity in Clinical Trials for Cancer Drugs, click here to view.
Feasibility of Using Real-World Data to Replicate Clinical Trial Evidence, click here to view.
Variation in US Private Health Plans' Coverage of Orphan Drugs, click here to view.
Incorporating Patient Perspectives and Transparency for Patient-Centered Value Assessment, click here to view.
Accounts from developers of generic health state utility instruments explain why they produce different QALYs: A qualitative study, click here to view.
The Shared Decision-Making Process in the Pharmacological Management of Depression, click here to view.
Training Patients to Review Scientific Reports for the Patient-centered Outcomes Research Institute: An Observational Study, click here to view.
Patient Reported Outcomes Have Arrived: A Practical Overview for Clinicians in Using Patient Reported Outcomes in Oncology, click here to view.
Prioritizing Evidence-Based Interventions for Dissemination and Implementation Investments: AHRQ's Model and Experience, click here to view.
11. AHRQ Effective Program Updates
OPEN FOR COMMENT THROUGH 11/26/2019: Systematic Review: Interventions for Substance Use Disorders in Adolescents: A Systematic Review, 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.
OPEN FOR COMMENT THROUGH 11/12/2019: Noninvasive Nonpharmacological Treatments for Chronic Pain: A Systematic Review Update, click here to view.
OPEN FOR COMMENT THROUGH 11/12/2019: Systematic Review: Opioid Treatments for Chronic Pain, click here to view.
OPEN FOR COMMENT THROUGH 11/12/2019: Systematic Review: Nonopioid Pharmacologic Treatments for Chronic Pain, click here to view.
Research Report: Pilot To Promote Entry of Structured Data Into the Systematic Review Data Repository (SRDR), click here to view.
Systematic Review: Pharmacologic and Nonpharmacologic Therapies in Adult Patients With Exacerbation of COPD, click here to view.
Research Protocol: Management of Colonic Diverticulitis, click here to view
Over the summer, the House Energy and Commerce and 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) shared a discussion draft of legislation reauthorizing PCORI with full funding for 10 years that proposed additional policy changes for PCORI, and invited stakeholders to provide input on those proposed policy changes. PIPC provided comments on the discussion draft highlighting support for an expert advisory panel for high-impact research, as well as a long-term, fully funded reauthorization that protects PCORI’s patient-centered mission and extends all of the existing funding mechanisms. PIPC also urged consideration of extending the protections in Section 1182 more broadly to cover Medicaid, the CMS Innovation Center, and other reforms that would apply value judgments to national policy decisions. PIPC opposes added payer seats on the PCORI Board Governors and an accompanying shift of the selection of the Methodology Committee to the Board of 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.
2. Video: Risk of Modeling Other Countries’ Drug Pricing: How a Canadian Child is Forced to Get Sicker to Access Treatment
Access to treatments is a constant struggle for patients in countries like Canada, where access is limited based on cost effectiveness or assessments of average clinical benefit. We are sharing a video developed in Canada with a mother, Sandy, and her daughter, Laura, who has cystic fibrosis. Sandy and her daughter Laura spoke honestly and freely about their experience with the Canadian health system. It is a heartbreaking story of the requirement to get sicker in order to access a new treatment she needs due to government utilization management for drugs, e.g. fail first policies. As our country considers modeling international drug pricing policies, it demonstrates the inherent risk for patient access to care. Click here to watch the video.
3. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage?
Other countries are often referenced as examples of how the use of QALYs or similar cost-based thresholds impact access to care.
- Australia: Lung cancer patient fundraising for trip to Mexico for treatment she cannot receive in Australia. MS patient has to go to Russia to access treatment she cannot get in Australia. Australian government cuts coverage of critical migraine drug.
- Canada: British Columbia patients fight for access to lifesaving SMA drug.
- New Zealand: Pharmac hurts patient safety by changing epilepsy drug coverage. The change may have led to a child's death.
- United Kingdom: Cancer survivor fighting to get NHS to cover "miracle" cancer drug. Hundreds of people have needlessly died since CF drug has been licensed but not covered. Still no access to CF drug for patients in Northern Ireland.
4. PIPC Urges House to Protect Patients and People with Disabilities in Title 1 of New House Bill
Legislation has been introduced in the House of Representatives which includes certain provisions concerning patients and people with disabilities in Title 1, particularly forcing patients and people with disabilities into “one-size-fits-all” decisions about their health. PIPC Chairman Tony Coelho expressed concern that Title 1 places hard-fought existing patient protections under serious threat. It explicitly relies 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 the bill. Click here to view prior stakeholder letter on referencing International pricing. Click here for our explainer of Title I of the bill. Click here to view information on Value Our Health.
5. Video: Role of PCORI Patient Reviewers
In this video, Paul McLean describes the work of a PCORI patient peer reviewer and what to look for in a final research report. Click here to view.
6. 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.
7. Washington Post: New Cystic Fibrosis Drug Could Turn Deadly Disease into Manageable Condition
The Washington Post reported about the approval of a new drug that can treat 90 percent of patients with cystic fibrosis. Trikafta, the new combination drug, had clinical trial data so persuasive that the Food and Drug Administration (FDA) approved it five months before its deadline. Sarah Carollo, a patient with cystic fibrosis, said that “as a person living with CF, my parents had been passing on to me this fear — we always had this constant fear of when the decline was going to happen, because we knew it was going to happen." But after starting the drug, the article states, "her doctors tested her lung function and were so stunned at the improvement that they had to check whether they were really looking at the results from the right patient. Earlier this month, Carollo ran a 5K race with another patient, Laurana Blackburn, who was also taking the drug through the clinical trial." Click here to read the article.
8. ICER Studies: 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: Revised Scoping Document available. 11/5/2019: Research Protocol. 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: Revised Draft Evidence Report and Voting Questions available. Comment period OPEN through 11/8/2019. Meeting 11/19/2019: CTAF to an update to its 2017 rheumatoid arthritis assessment.
- Type 2 Diabetes: Evidence Report and Voting Questions available. Meeting 11/14/2019: The New England CEPAC will convene to deliberate on ICER's review of oral semaglutide for the treatment of type 2 diabetes.
- Acute Migraine: Model Analysis Plan available. 11/7/2019: Draft Evidence Report. Meeting 1/23/2020: Midwest CEPAC to review ICER's assessment of acute migraine treatments
- Valuing A Cure Project: 11/15/2019: Revised White Paper.
- 2020 Value Assessment Framework: 12/18/2019: Final Framework Adaptations.
- Non-Alcoholic Steatohepatitis: Draft Scoping Document available. Comment period OPEN through 11/20/2019.
9. Upcoming Events and Webinars
Personalized Medicine Conference
November 13-14, 2019
Click here for details.
Stay, Safe, Side: Seizure First Aid & Priorities for the Epilepsy Community
November 14, 2019
9:30 a.m.
Rayburn House Office Building, Room 2168
RSVP to [email protected].
Webinar: The National Patient-Centered Clinical Research Network (PCORnet)
November 14, 2019
Click here for details.
2019 AUCD Annual Meeting
November 17-20, 2019
Click here for details.
Advisory Panel on Healthcare Delivery and Disparities Research Winter 2019 Meeting
December 6, 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.
FT US Pharma and Biotech Summit 2020
May 14, 2020
Click here for details.
10. Medical Journal Articles
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.
Real-World Evidence: Research Reveals a Lack of Racial Diversity in Clinical Trials for Cancer Drugs, click here to view.
Feasibility of Using Real-World Data to Replicate Clinical Trial Evidence, click here to view.
Variation in US Private Health Plans' Coverage of Orphan Drugs, click here to view.
Incorporating Patient Perspectives and Transparency for Patient-Centered Value Assessment, click here to view.
Accounts from developers of generic health state utility instruments explain why they produce different QALYs: A qualitative study, click here to view.
The Shared Decision-Making Process in the Pharmacological Management of Depression, click here to view.
Training Patients to Review Scientific Reports for the Patient-centered Outcomes Research Institute: An Observational Study, click here to view.
Patient Reported Outcomes Have Arrived: A Practical Overview for Clinicians in Using Patient Reported Outcomes in Oncology, click here to view.
Prioritizing Evidence-Based Interventions for Dissemination and Implementation Investments: AHRQ's Model and Experience, click here to view.
11. AHRQ Effective Program Updates
OPEN FOR COMMENT THROUGH 11/26/2019: Systematic Review: Interventions for Substance Use Disorders in Adolescents: A Systematic Review, 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.
OPEN FOR COMMENT THROUGH 11/12/2019: Noninvasive Nonpharmacological Treatments for Chronic Pain: A Systematic Review Update, click here to view.
OPEN FOR COMMENT THROUGH 11/12/2019: Systematic Review: Opioid Treatments for Chronic Pain, click here to view.
OPEN FOR COMMENT THROUGH 11/12/2019: Systematic Review: Nonopioid Pharmacologic Treatments for Chronic Pain, click here to view.
Research Report: Pilot To Promote Entry of Structured Data Into the Systematic Review Data Repository (SRDR), click here to view.
Systematic Review: Pharmacologic and Nonpharmacologic Therapies in Adult Patients With Exacerbation of COPD, click here to view.
Research Protocol: Management of Colonic Diverticulitis, click here to view
