In This Week’s Issue:
1. New York: Final Budget Being Analyzed for Discrimination Implications, see below for details.
2. FDA Releases New Framework for Incorporating Real-World Evidence, click here for the full announcement.
3. AAFP: Identifying Priorities Is First Step to Better Outcomes, click here to read the article.
4. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
5. ICER Studies: Cardiovascular Disease, SMA, Depression, MS, Duchenne Muscular Dystrophy, Peanut Allergy, click here to provide patient input.
6. Request for Comment: Definition of Health Technology Assessment, see below for details.
7. BMJ Opinion: All Evidence is Real-World Evidence, click here to read the article.
8. Submit 2019 Fly-ins, Advocacy Days, and Conferences to Democratic Caucus, click here to submit your events.
9. Upcoming Events and Webinars, see details below.
10. Medical Journal Articles, see details below.
11. AHRQ Effective Program Updates, see details below.
1. New York: Final Budget Being Analyzed for Discrimination Implications, see below for details.
2. FDA Releases New Framework for Incorporating Real-World Evidence, click here for the full announcement.
3. AAFP: Identifying Priorities Is First Step to Better Outcomes, click here to read the article.
4. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
5. ICER Studies: Cardiovascular Disease, SMA, Depression, MS, Duchenne Muscular Dystrophy, Peanut Allergy, click here to provide patient input.
6. Request for Comment: Definition of Health Technology Assessment, see below for details.
7. BMJ Opinion: All Evidence is Real-World Evidence, click here to read the article.
8. Submit 2019 Fly-ins, Advocacy Days, and Conferences to Democratic Caucus, click here to submit your events.
9. Upcoming Events and Webinars, see details below.
10. Medical Journal Articles, see details below.
11. AHRQ Effective Program Updates, see details below.
1. New York: Final Budget Being Analyzed for Discrimination Implications
In March, the New York Executive and Assembly included provisions in their proposed budgets that would allow for the use of QALY-based value assessments to determine access and coverage, such as the assessments conducted by the Institute for Clinical Economic Review (ICER). While the New York Senate proposed budget incorporated provisions to address the concerns of advocates by banning use of discriminatory, biased value assessments that are not transparent, the Senate’s budget language related to discrimination was not incorporated into the final budget. Please note advocates are still reviewing the language and will provide an update shortly on any improvements that may have been made to the original Executive budget language. We appreciate the hard work of advocates to speak out against measures that discriminate against seniors and individuals with chronic conditions and disabilities. As background, click here to view the joint letter sent to New York legislators opposing the Executive Budget provision. We will share more information on its implications related to discrimination, bias and transparency soon. Follow the PIPC Blog for more information once an analysis is complete!
2. FDA Releases New Framework for Incorporating Real-World Evidence
The Food and Drug Administration released a new framework that will facilitate incorporation of real-world evidence into its work, intending to have information about patient outcomes influence clinical decision-making. "RWD collected from a variety of sources offer new opportunities to generate evidence and better understand clinical outcomes. These data may be derived from a diverse array of sources, such as electronic health records (EHR), medical claims, product and disease registries, laboratory test results and even cutting-edge technology paired with consumer mobile devices. These data are being used to develop information and real-world evidence (RWE) that can better inform regulatory decisions. Because they include data covering the experience of physicians and patients with the actual use of new treatments in practice, and not just in research studies, the collective evaluation of these data sources has the potential to inform clinical decision making by patients and providers, develop new hypotheses for further testing of new products to drive continued innovation and inform us about the performance of medical products," wrote FDA Commissioner Scott Gottlieb. Click here for the full announcement.
3. AAFP: Identifying Priorities Is First Step to Better Outcomes
Helping patients identify their priorities is central to patient-centered care and ensuring that patients and providers use their time properly, according to a study by the American Academy of Family Physicians (AAFP). They used a tool dubbed a "Visit Planner" to help patients plan out their visits while in the waiting room. "Patients in the intervention arm were given a tablet in the office waiting room on which the Visit Planner was loaded. When launched, the planner began with an introductory video about the tool and the importance of patients communicating their concerns to physicians early in the visit. Users were prompted to choose their top one or two priorities from a list of six categories with space for free-form entries. Patients also were asked to express how they would stay involved with their care -- either during or after the visits -- via activities such as note taking, physician questions and visiting a patient portal....Findings showed that patients who used the planner reported 'significantly greater visit preparation and discussion of their priorities' when compared to patients in the control group," wrote the AAFP's Sheri Porter. Click here to read the article.
4. 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.
5. ICER Studies: Cardiovascular Disease, SMA, Depression, MS, Duchenne Muscular Dystrophy, Peanut Allergy
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:
6. Request for Comment: Definition of Health Technology Assessment
Dr. Brian O’Rourke of the International Network of Agencies for Health Technology Assessment (INAHTA) and Dr. Wija Oortwijn of Health Technology Assessment International (HTAi) are co-chairing an international joint task group that is proposing an updated definition of Health Technology Assessment (HTA) that consolidates and simplifies the various definitions in existence and which reflects the current and emerging realities of HTA. Along with the co-chairs from INAHTA and HTAi, the joint task group includes representatives from WHO, EUnetHTA, HTAsiaLink, RedETSA, the English Editorial Board of the HTA Glossary, and ISPOR. The group has followed a consensus-building process to develop the proposed updated definition and they are now inviting input from the HTA community to build broad consensus on the updated definition. Click here for more information.
7. BMJ Opinion: All Evidence is Real-World Evidence
A piece in the British Medical Journal (BMJ) argues that all evidence, including evidence from randomized controlled trials, ought to be considered as real-world evidence. "Glorifying real world data by (indirectly) undermining other research and evidence from RCTs tarnishes pragmatic trials or large international trials that mitigate concerns about indirectness without sacrificing principles to reduce risk of bias....[T]he phrase “real world evidence” waters down what we should be focusing on. Trustworthy recommendations should be based on an evaluation of the evidence and an assessment of how reliably the evidence supports all factors that determine a recommendation or decision. These factors include the importance of a health problem, the balance between health benefits and harms, values that people attach to outcomes, resource use, equity, acceptability, and feasibility," wrote Holger Schunemann. Click here to read the article.
8. Submit 2019 Fly-ins, Advocacy Days, and Conferences to Democratic Caucus
In the interest of amplified patient and stakeholder engagement, your organization may be interested that the House Majority Leader’s office is compiling a list of fly-ins, advocacy days, and conferences that will be taking place throughout the year. This information will be shared with all House Democratic offices and used for a variety of purposes including scheduling and messaging. Feel free to share any events you have planned. If we hear of similar efforts by the Minority Leader, we hope to share that as well. Please submit your events here.
9. Upcoming Events and Webinars
Cycle 1 2019 Limited PCORI Funding Announcement: Partnerships To Conduct Research (PaCR) within PCORnet
April 3, 2019
Click here for details.
Webinar: MCDA - How to Guide Through the Application of Different Methods for Assessment and Prioritization of Rare Disease Health Technologies
April 4, 2019
Click here for details.
Comparative Effectiveness Research: Recent Applications and Future Investments
April 9, 2019
Click here for details.
Big Data, RWD and RWE: What’s the Difference and How is it Changing the Research Landscape?
April 15, 2019
Click here for details.
PCORI Board of Governors Meeting
April 16, 2019
Click here for details.
17th Annual Eyeforpharma Philadelphia
April 16-17, 2019
Click here for details.
Cycle 2 2019 Improving Methods Applicant Town Hall
May 9, 2019
Click here for details.
PCORI Board of Governors Meeting
May 13, 2019
Click here for details.
Advisory Panel on Clinical Trials Spring 2019 Meeting
May 15, 2019
Click here for details.
Advisory Panel on Healthcare Delivery and Disparities Research Spring 2019 Meeting
May 16, 2019
Click here for details.
2019 NEC Symposium
June 2 - 5, 2019
Click here for details.
A New Path Forward for Using Real World Evidence in Randomized Clinical Trials
June 23, 2019
Click here for details.
2019 PCORI Annual Meeting
September 18-20, 2019
Click here for details.
10. Medical Journal Articles
Why and How to Use Patient-Oriented Research to Promote Translational Research, click here to view.
The Evolution of European HTA and Access to Innovative Medicines, click here to view.
Advocating for New Patient-Centered Tools for Value-Based Treatment Choices in Oncology, click here to view.
Toward a Strategy to Involve Patients in Health Technology Assessment in Spain, click here to view.
Patient Engagement In Research: Early Findings From The Patient-Centered Outcomes Research Institute, click hereto view.
Quality Decision Making in Health Technology Assessment: Issues Facing Companies and Agencies, click here to view.
Letter: Multi-Method Patient-Engagement Approach: A Case Example from a PCORI-Funded Training Project, clickhere to view.
Perspectives of Patients in Identifying Their Values-Based Health Priorities, click here to view.
How Patient's Access to Evidence-Based Medicine Provides Them with a New Hope "The 45 Min—An Insightful Round," click here to view.
Improving Patient-Reported Measures in Oncology, click here to view.
Moving Forward from Drug-Centred to Patient-Centred Research, click here to view.
11. AHRQ Effective Program Updates
Systematic Review: Adverse Effects of Pharmacological Treatments of Major Depression in Older Adults, click hereto view.
Comment Period: Management of Primary Headaches in Pregnancy, click here to view.
Comment Period: Antipsychotics for the Prevention and Treatment of Delirium, click here to view.
Nonopioid Pharmacologic Treatments for Chronic Pain, click here to view.
Systematic Review Update: Noninvasive Nonpharmacologic Treatments for Chronic Pain, click here to view.
Treatment of Acute Pain: An Evidence Map, click here to view.
Development of Harmonized Outcome Measures for Use in Patient Registries and Clinical Practice: Methods and Lessons Learned, click here to view.
Standardized Library of Depression Outcome Measures, click here to view.
Patient Navigation Models for Lung Cancer, click here to view.
Stroke Prevention in Patients With Atrial Fibrillation: A Systematic Review Update, click here to view.
Long-term Drug Therapy and Drug Holidays for Osteoporosis Fracture Prevention: A Systematic Review, click here to view.
In March, the New York Executive and Assembly included provisions in their proposed budgets that would allow for the use of QALY-based value assessments to determine access and coverage, such as the assessments conducted by the Institute for Clinical Economic Review (ICER). While the New York Senate proposed budget incorporated provisions to address the concerns of advocates by banning use of discriminatory, biased value assessments that are not transparent, the Senate’s budget language related to discrimination was not incorporated into the final budget. Please note advocates are still reviewing the language and will provide an update shortly on any improvements that may have been made to the original Executive budget language. We appreciate the hard work of advocates to speak out against measures that discriminate against seniors and individuals with chronic conditions and disabilities. As background, click here to view the joint letter sent to New York legislators opposing the Executive Budget provision. We will share more information on its implications related to discrimination, bias and transparency soon. Follow the PIPC Blog for more information once an analysis is complete!
2. FDA Releases New Framework for Incorporating Real-World Evidence
The Food and Drug Administration released a new framework that will facilitate incorporation of real-world evidence into its work, intending to have information about patient outcomes influence clinical decision-making. "RWD collected from a variety of sources offer new opportunities to generate evidence and better understand clinical outcomes. These data may be derived from a diverse array of sources, such as electronic health records (EHR), medical claims, product and disease registries, laboratory test results and even cutting-edge technology paired with consumer mobile devices. These data are being used to develop information and real-world evidence (RWE) that can better inform regulatory decisions. Because they include data covering the experience of physicians and patients with the actual use of new treatments in practice, and not just in research studies, the collective evaluation of these data sources has the potential to inform clinical decision making by patients and providers, develop new hypotheses for further testing of new products to drive continued innovation and inform us about the performance of medical products," wrote FDA Commissioner Scott Gottlieb. Click here for the full announcement.
3. AAFP: Identifying Priorities Is First Step to Better Outcomes
Helping patients identify their priorities is central to patient-centered care and ensuring that patients and providers use their time properly, according to a study by the American Academy of Family Physicians (AAFP). They used a tool dubbed a "Visit Planner" to help patients plan out their visits while in the waiting room. "Patients in the intervention arm were given a tablet in the office waiting room on which the Visit Planner was loaded. When launched, the planner began with an introductory video about the tool and the importance of patients communicating their concerns to physicians early in the visit. Users were prompted to choose their top one or two priorities from a list of six categories with space for free-form entries. Patients also were asked to express how they would stay involved with their care -- either during or after the visits -- via activities such as note taking, physician questions and visiting a patient portal....Findings showed that patients who used the planner reported 'significantly greater visit preparation and discussion of their priorities' when compared to patients in the control group," wrote the AAFP's Sheri Porter. Click here to read the article.
4. 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.
- New Zealand: Breast cancer patients are not receiving access to to life-saving medications. As a result of widespread criticism, PHARMAC is set to review its practices of breast cancer drug funding. Click here and here to read more. Patients in New Zealand also face barriers to access for treatment of spinal muscular atrophy. Click here to read more.
- Canada: Discriminatory cost effectiveness measures are blocking access to care for patients with spinal muscular atrophy patients. Click here and here to read more. Click here to view the story of a child with thyroid cancer forced to come to the U.S. for care, and here for an article about high costs of Parkinson's and MS treatments. Click here for a story about a patient with cystic fibrosis whose province does not cover the drug she needs.
- Netherlands: Cost effectiveness measures are blocking access to cystic fibrosis treatments for children. It also remains unavailable for adults because the government has failed to reach an agreement. Click here to read more.
- United Kingdom: English families are moving to Scotland and Ireland in an attempt to access MS drugs not covered by the NHS. Activists are fighting for access to treatments for cystic fibrosis and cancer. Click here, here, and here for articles related to cystic fibrosis. Click here and here to read about the fight for access to cancer drugs in Scotland. Click here for an article related to MS. Click here for more information on the NHS drug approval process.
5. ICER Studies: Cardiovascular Disease, SMA, Depression, MS, Duchenne Muscular Dystrophy, Peanut Allergy
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:
- Arthritis: Open Input Period through 4/8/2019. Meeting 10/31/2019: CTAF to an update to its 2017 rheumatoid arthritis assessment.
- Cardiovascular Disease : Comment period OPEN on Draft Scoping Document through 4/5/2019. 9/26/2019 Meeting: Midwest CEPAC to deliberate and vote on ICER's report on evidence presented in ICER's report on additive CVD therapies.
- Spinal Muscular Atrophy: 4/3/2019: Final Evidence Report and Meeting Summary.
- Depression: Comment period on Draft Evidence Report OPEN through 4/17/2019. Meeting 5/23/2019: Midwest CEPAC to review evidence on esketamine as a therapy for treatment-resistant depression.
- Multiple Sclerosis: Public Comments OPEN through April 10, 2019 on the Draft Analysis Report. Meeting 5/23/2019: Midwest CEPAC to review ICER's assessment of siponimod (Novartis) for secondary progressive multiple sclerosis.
- Peanut Allergy: Draft Evidence Report: 4/9/2019. Meeting 6/11/2019: CTAF to review ICER's assessment of treatments for peanut allergy.
- Duchenne Muscular Dystrophy: Research protocol available. Meeting 7/25/2019: New England CEPAC to deliberate and vote on evidence presented in ICER's report on treatments for Duchenne muscular dystrophy.
- Unsupported Price Increase Assessment: Open Input Period through 4/19/2019.
6. Request for Comment: Definition of Health Technology Assessment
Dr. Brian O’Rourke of the International Network of Agencies for Health Technology Assessment (INAHTA) and Dr. Wija Oortwijn of Health Technology Assessment International (HTAi) are co-chairing an international joint task group that is proposing an updated definition of Health Technology Assessment (HTA) that consolidates and simplifies the various definitions in existence and which reflects the current and emerging realities of HTA. Along with the co-chairs from INAHTA and HTAi, the joint task group includes representatives from WHO, EUnetHTA, HTAsiaLink, RedETSA, the English Editorial Board of the HTA Glossary, and ISPOR. The group has followed a consensus-building process to develop the proposed updated definition and they are now inviting input from the HTA community to build broad consensus on the updated definition. Click here for more information.
7. BMJ Opinion: All Evidence is Real-World Evidence
A piece in the British Medical Journal (BMJ) argues that all evidence, including evidence from randomized controlled trials, ought to be considered as real-world evidence. "Glorifying real world data by (indirectly) undermining other research and evidence from RCTs tarnishes pragmatic trials or large international trials that mitigate concerns about indirectness without sacrificing principles to reduce risk of bias....[T]he phrase “real world evidence” waters down what we should be focusing on. Trustworthy recommendations should be based on an evaluation of the evidence and an assessment of how reliably the evidence supports all factors that determine a recommendation or decision. These factors include the importance of a health problem, the balance between health benefits and harms, values that people attach to outcomes, resource use, equity, acceptability, and feasibility," wrote Holger Schunemann. Click here to read the article.
8. Submit 2019 Fly-ins, Advocacy Days, and Conferences to Democratic Caucus
In the interest of amplified patient and stakeholder engagement, your organization may be interested that the House Majority Leader’s office is compiling a list of fly-ins, advocacy days, and conferences that will be taking place throughout the year. This information will be shared with all House Democratic offices and used for a variety of purposes including scheduling and messaging. Feel free to share any events you have planned. If we hear of similar efforts by the Minority Leader, we hope to share that as well. Please submit your events here.
9. Upcoming Events and Webinars
Cycle 1 2019 Limited PCORI Funding Announcement: Partnerships To Conduct Research (PaCR) within PCORnet
April 3, 2019
Click here for details.
Webinar: MCDA - How to Guide Through the Application of Different Methods for Assessment and Prioritization of Rare Disease Health Technologies
April 4, 2019
Click here for details.
Comparative Effectiveness Research: Recent Applications and Future Investments
April 9, 2019
Click here for details.
Big Data, RWD and RWE: What’s the Difference and How is it Changing the Research Landscape?
April 15, 2019
Click here for details.
PCORI Board of Governors Meeting
April 16, 2019
Click here for details.
17th Annual Eyeforpharma Philadelphia
April 16-17, 2019
Click here for details.
Cycle 2 2019 Improving Methods Applicant Town Hall
May 9, 2019
Click here for details.
PCORI Board of Governors Meeting
May 13, 2019
Click here for details.
Advisory Panel on Clinical Trials Spring 2019 Meeting
May 15, 2019
Click here for details.
Advisory Panel on Healthcare Delivery and Disparities Research Spring 2019 Meeting
May 16, 2019
Click here for details.
2019 NEC Symposium
June 2 - 5, 2019
Click here for details.
A New Path Forward for Using Real World Evidence in Randomized Clinical Trials
June 23, 2019
Click here for details.
2019 PCORI Annual Meeting
September 18-20, 2019
Click here for details.
10. Medical Journal Articles
Why and How to Use Patient-Oriented Research to Promote Translational Research, click here to view.
The Evolution of European HTA and Access to Innovative Medicines, click here to view.
Advocating for New Patient-Centered Tools for Value-Based Treatment Choices in Oncology, click here to view.
Toward a Strategy to Involve Patients in Health Technology Assessment in Spain, click here to view.
Patient Engagement In Research: Early Findings From The Patient-Centered Outcomes Research Institute, click hereto view.
Quality Decision Making in Health Technology Assessment: Issues Facing Companies and Agencies, click here to view.
Letter: Multi-Method Patient-Engagement Approach: A Case Example from a PCORI-Funded Training Project, clickhere to view.
Perspectives of Patients in Identifying Their Values-Based Health Priorities, click here to view.
How Patient's Access to Evidence-Based Medicine Provides Them with a New Hope "The 45 Min—An Insightful Round," click here to view.
Improving Patient-Reported Measures in Oncology, click here to view.
Moving Forward from Drug-Centred to Patient-Centred Research, click here to view.
11. AHRQ Effective Program Updates
Systematic Review: Adverse Effects of Pharmacological Treatments of Major Depression in Older Adults, click hereto view.
Comment Period: Management of Primary Headaches in Pregnancy, click here to view.
Comment Period: Antipsychotics for the Prevention and Treatment of Delirium, click here to view.
Nonopioid Pharmacologic Treatments for Chronic Pain, click here to view.
Systematic Review Update: Noninvasive Nonpharmacologic Treatments for Chronic Pain, click here to view.
Treatment of Acute Pain: An Evidence Map, click here to view.
Development of Harmonized Outcome Measures for Use in Patient Registries and Clinical Practice: Methods and Lessons Learned, click here to view.
Standardized Library of Depression Outcome Measures, click here to view.
Patient Navigation Models for Lung Cancer, click here to view.
Stroke Prevention in Patients With Atrial Fibrillation: A Systematic Review Update, click here to view.
Long-term Drug Therapy and Drug Holidays for Osteoporosis Fracture Prevention: A Systematic Review, click here to view.
