In This Week’s Issue:
1. PCORI Reauthorization Discussions Advance
2. PCORI Videos on Patient-Centered Research Advancing Quality Care, see details below.
3. PIPC, Leading Patient Groups Submit Joint Letter to ICER on Draft 2020 Value Framework, click here to read the letter.
4. Advancing the Use of Patient-Generated Data, click here to read the article.
5. Liver Cancer Hill Briefing, see below for details.
6. EveryLife Foundation Briefing with the Rare Disease Caucus, click here to learn more and register.
7. 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.
8. PIPC Urges House to Protect Patients and People with Disabilities in Title 1 of New House Bill, see below for details.
9. Report on the Value Assessment Landscape in the U.S., click here to read the report.
10. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
11. ICER Studies: Acute Migraine, Type 2 Diabetes, Arthritis, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, click here to provide patient input.
12. Upcoming Events and Webinars, see details below.
13. Medical Journal Articles, see details below.
14. AHRQ Effective Program Updates, see details below.
1. PCORI Reauthorization Discussions Advance
2. PCORI Videos on Patient-Centered Research Advancing Quality Care, see details below.
3. PIPC, Leading Patient Groups Submit Joint Letter to ICER on Draft 2020 Value Framework, click here to read the letter.
4. Advancing the Use of Patient-Generated Data, click here to read the article.
5. Liver Cancer Hill Briefing, see below for details.
6. EveryLife Foundation Briefing with the Rare Disease Caucus, click here to learn more and register.
7. 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.
8. PIPC Urges House to Protect Patients and People with Disabilities in Title 1 of New House Bill, see below for details.
9. Report on the Value Assessment Landscape in the U.S., click here to read the report.
10. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
11. ICER Studies: Acute Migraine, Type 2 Diabetes, Arthritis, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, click here to provide patient input.
12. Upcoming Events and Webinars, see details below.
13. Medical Journal Articles, see details below.
14. 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. Over the next week, it is anticipated that stakeholders will provide feedback on the implications of these proposed policies for PCORI’s patient-centered mission. 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. Feel free to reach out to [email protected] for more information or click here for information about Friends of PCORI Reauthorization.
2. PCORI Videos on Patient-Centered Research Advancing Quality Care
Anand Parekh, MD, chief medical advisor at the Bipartisan Policy Center, explains how patient-centered outcomes research is producing evidence that helps policy makers and providers advance value-based health care. Click here to watch the video. He also explains that patient-centered outcomes research provides the best evidence for improving health outcomes. Click here to watch the video.
3. PIPC, Leading Patient Groups Submit Joint Letter to ICER on Draft 2020 Value Framework
On Friday, Oct. 18, the Partnership to Improve Patient Care joined over 50 leading patient advocacy groups in submitting a joint comment letter to the Institute for Clinical and Economic Review (ICER) on its draft 2020 Value Assessment Framework. The letter criticized ICER for defending its use of the QALY, saying that ICER’s framework ignores ethical principles that enjoy broad support among the general public. Instead of relying on metrics that treat patients as averages, the letter encouraged ICER to abandon it’s use of discriminatory value metrics and develop mechanisms that incorporate robust patient and clinician feedback. “Above all, we urge ICER to put patients and people with disabilities at the center of all of your assessments,” the letter states. “ICER’s value assessments do not promote affordability for patients, but instead give payers justification to create barriers to treatment coverage that benefit their own bottom line.” Click here to read the letter.
4. Advancing the Use of Patient-Generated Data
Patient-generated data can help providers and patients make decisions that are tailored to each individual situation, however, a number of operational, regulatory, and behavioral obstacles remain. "PGHD has the potential to significantly support individuals with chronic conditions who may require regular and ongoing monitoring. It can help providers and patients detect emergent issues or trends. Certainly, patients have much more on their plate than the constant collection or documentation of their health-related issues," writes Andrea Maresca in Morning Consult. "The adoption of PGHD as a regular component of the health care landscape hinges on the engagement of patients and providers on the front end. Their respective receptivity and concerns at this early stage will reveal the speed with which CMS can move forward." Click here to read the article.
5. Liver Cancer Hill Briefing
Global Liver Institute, American Gastroenterological Association, Hepatitis B Foundation, Hep B United, AAPCHO, NVHR, and NASTAD are hosting a Congressional briefing titled, "Liver Cancer Drivers and Disparities" on Thursday, October 31 from 10:30 a.m. - 12:00 p.m. Learn from our nation’s leading clinicians and patient advocates about the steps needed to combat the most rapidly rising cancer since 1980, and how response strategies must begin with modernizing the systems in place to better meet the needs of impacted populations. The briefing will be held in room 121 of the Cannon House Office Building. Please RSVP to [email protected] if you are interested in attending.
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. 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.
8. PIPC Urges House to Protect Patients and People with Disabilities in Title 1 of New House Bill
Legislation has been marked up in 3 House committees, the House Committees on Energy & Commerce, Education & Labor and Ways and Means, which includes certain provisions concerning patients and people with disabilities. Title 1 of the bill takes an approach that is entrenched in controversial comparative clinical and cost effectiveness standards that force patients and people with disabilities into “one-size-fits-all” decisions about their health. It 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.
9. Report on the Value Assessment Landscape in the U.S.
The National Pharmaceutical Council released a report on the value assessment entities in the U.S. and how they differ. The report explores the target audiences of the different entities as well as what sorts of services and conditions they address. It finds that many of the value assessors suffer from a lack of transparency and accountability, as well as failing to incorporate real-world evidence, relying instead primarily on clinical trials. Click here to read the report.
10. 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.
11. ICER Studies: Acute Migraine, Type 2 Diabetes, Arthritis, Cardiovascular Disease, 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:
12. Upcoming Events and Webinars
Personalized Medicine Conference
November 13-14, 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.
13. Medical Journal Articles
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.
Observational Studies Have a Critical Role to Play in Cancer Comparative Effectiveness Research, click here to view.
High-Quality Evidence to Inform Clinical Practice, click here to view.
14. AHRQ Effective Program Updates
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.
Research Protocol: Therapies for Clinically Localized Prostate Cancer, click here to view.
Research Report: Web Interactive Presentation of EPC Reports: A Foray Into Interactive Reports, 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. Over the next week, it is anticipated that stakeholders will provide feedback on the implications of these proposed policies for PCORI’s patient-centered mission. 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. Feel free to reach out to [email protected] for more information or click here for information about Friends of PCORI Reauthorization.
2. PCORI Videos on Patient-Centered Research Advancing Quality Care
Anand Parekh, MD, chief medical advisor at the Bipartisan Policy Center, explains how patient-centered outcomes research is producing evidence that helps policy makers and providers advance value-based health care. Click here to watch the video. He also explains that patient-centered outcomes research provides the best evidence for improving health outcomes. Click here to watch the video.
3. PIPC, Leading Patient Groups Submit Joint Letter to ICER on Draft 2020 Value Framework
On Friday, Oct. 18, the Partnership to Improve Patient Care joined over 50 leading patient advocacy groups in submitting a joint comment letter to the Institute for Clinical and Economic Review (ICER) on its draft 2020 Value Assessment Framework. The letter criticized ICER for defending its use of the QALY, saying that ICER’s framework ignores ethical principles that enjoy broad support among the general public. Instead of relying on metrics that treat patients as averages, the letter encouraged ICER to abandon it’s use of discriminatory value metrics and develop mechanisms that incorporate robust patient and clinician feedback. “Above all, we urge ICER to put patients and people with disabilities at the center of all of your assessments,” the letter states. “ICER’s value assessments do not promote affordability for patients, but instead give payers justification to create barriers to treatment coverage that benefit their own bottom line.” Click here to read the letter.
4. Advancing the Use of Patient-Generated Data
Patient-generated data can help providers and patients make decisions that are tailored to each individual situation, however, a number of operational, regulatory, and behavioral obstacles remain. "PGHD has the potential to significantly support individuals with chronic conditions who may require regular and ongoing monitoring. It can help providers and patients detect emergent issues or trends. Certainly, patients have much more on their plate than the constant collection or documentation of their health-related issues," writes Andrea Maresca in Morning Consult. "The adoption of PGHD as a regular component of the health care landscape hinges on the engagement of patients and providers on the front end. Their respective receptivity and concerns at this early stage will reveal the speed with which CMS can move forward." Click here to read the article.
5. Liver Cancer Hill Briefing
Global Liver Institute, American Gastroenterological Association, Hepatitis B Foundation, Hep B United, AAPCHO, NVHR, and NASTAD are hosting a Congressional briefing titled, "Liver Cancer Drivers and Disparities" on Thursday, October 31 from 10:30 a.m. - 12:00 p.m. Learn from our nation’s leading clinicians and patient advocates about the steps needed to combat the most rapidly rising cancer since 1980, and how response strategies must begin with modernizing the systems in place to better meet the needs of impacted populations. The briefing will be held in room 121 of the Cannon House Office Building. Please RSVP to [email protected] if you are interested in attending.
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. 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.
8. PIPC Urges House to Protect Patients and People with Disabilities in Title 1 of New House Bill
Legislation has been marked up in 3 House committees, the House Committees on Energy & Commerce, Education & Labor and Ways and Means, which includes certain provisions concerning patients and people with disabilities. Title 1 of the bill takes an approach that is entrenched in controversial comparative clinical and cost effectiveness standards that force patients and people with disabilities into “one-size-fits-all” decisions about their health. It 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.
9. Report on the Value Assessment Landscape in the U.S.
The National Pharmaceutical Council released a report on the value assessment entities in the U.S. and how they differ. The report explores the target audiences of the different entities as well as what sorts of services and conditions they address. It finds that many of the value assessors suffer from a lack of transparency and accountability, as well as failing to incorporate real-world evidence, relying instead primarily on clinical trials. Click here to read the report.
10. 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: The mother of a girl with cystic fibrosis calls for a strategy for tackling rare diseases.
- New Zealand: Some MS patients continue to be denied medication due to not meeting Pharmac's criteria. Click here for more. Breast and lung cancer patients call for a doubling of Pharmac's budget to increase access.
- United Kingdom: Cystic fibrosis patient calls for the government to fund a drug that will greatly help her. Family of a patient with cystic fibrosis moves to Scotland to access a lifesaving drug and another family says they will have to move soon if NHS does not start to fund a critical drug.
11. ICER Studies: Acute Migraine, Type 2 Diabetes, Arthritis, Cardiovascular Disease, 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: Draft Scoping Document available. 11/4/2019: Updated Scoping Document.
- Cystic Fibrosis: Draft Scoping Document available. Comment period OPEN through TODAY, 10/21/2019. 10/29/2019: Revised Scoping Document.
- 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: 10/11/2019: Revised Draft Evidence Report and Voting Questions. Comment period OPEN through 11/8/2019. Meeting 11/19/2019: CTAF to an update to its 2017 rheumatoid arthritis assessment.
- Type 2 Diabetes: 11/1/2019: Voting Questions and Evidence Report. 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: 10/30/2019: Draft Scoping Document.
12. Upcoming Events and Webinars
Personalized Medicine Conference
November 13-14, 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.
13. Medical Journal Articles
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.
Observational Studies Have a Critical Role to Play in Cancer Comparative Effectiveness Research, click here to view.
High-Quality Evidence to Inform Clinical Practice, click here to view.
14. AHRQ Effective Program Updates
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.
Research Protocol: Therapies for Clinically Localized Prostate Cancer, click here to view.
Research Report: Web Interactive Presentation of EPC Reports: A Foray Into Interactive Reports, click here to view.
