In This Week’s Issue:
1. Health Affairs: To Get More Bang For Your Health-Care Buck, Invest In Innovation, click here to read the blog
2. PAVE Blog: Putting Our Guard Down — Engaging Multiple Stakeholders To Define Value In Healthcare, click here to read the blog.
3. PMC Webinar: Moving Beyond Population Averages — Patient Principles for a Personalized Medicine Research Agenda, see details below.
4. An Update From the Friends of PCORI Reauthorization Coalition, see details below.
5. The PCORI Blog: Making Research More Patient Centered, One Application at a Time, click here to read the blog.
6. AcademyHealth Blog: Engagement Science — Factors Fueling the Need for Better Defined Engagement, click here to read the blog.
7. PIPC: Don't Discriminate on Care, click here to view.
8. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? Click here to view.
9. Upcoming ICER Studies: Angiodema, Asthma, Opioid Use Disorder, MS, Peanut Allergy, SMA, Depression, 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. Health Affairs: To Get More Bang For Your Health-Care Buck, Invest In Innovation, click here to read the blog
2. PAVE Blog: Putting Our Guard Down — Engaging Multiple Stakeholders To Define Value In Healthcare, click here to read the blog.
3. PMC Webinar: Moving Beyond Population Averages — Patient Principles for a Personalized Medicine Research Agenda, see details below.
4. An Update From the Friends of PCORI Reauthorization Coalition, see details below.
5. The PCORI Blog: Making Research More Patient Centered, One Application at a Time, click here to read the blog.
6. AcademyHealth Blog: Engagement Science — Factors Fueling the Need for Better Defined Engagement, click here to read the blog.
7. PIPC: Don't Discriminate on Care, click here to view.
8. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? Click here to view.
9. Upcoming ICER Studies: Angiodema, Asthma, Opioid Use Disorder, MS, Peanut Allergy, SMA, Depression, 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. Health Affairs: To Get More Bang For Your Health-Care Buck, Invest In Innovation
In a post for Health Affairs Blog, Tanisha Carino writes that PCORI has done a great job of integrating patient perspectives into its decision-making processes. “Not all foundations have the resources of MJFF; these groups need new sources of financial support. One might be the Patient-Centered Outcomes Research Institute (PCORI), a nongovernmental organization created by the Affordable Care Act to study health outcomes relevant to patients and caregivers. As such, PCORI has gone to great lengths to integrate patient perspectives into its priorities and decision-making processes. PCORI—due for reauthorization by Congress in 2019—has the potential to help disease foundations develop their research infrastructures and be strong partners in the biomedical ecosystem.” Click here to read the blog.
2. PAVE Blog: Putting Our Guard Down — Engaging Multiple Stakeholders To Define Value In Healthcare
In a post for Health Affairs Blog, members of the recently formed Patient-Driven Values in Healthcare Evaluation (PAVE) Center of Excellence discuss their patient engagement goals moving forward. “The organization aims to partner with a broad range of patients and other stakeholders to develop a research agenda, expand existing educational programs to increase the capacity for engagement among patient and research communities, advance methods that capture the voice of the patient in value assessment, and disseminate findings so that messages will reach groups within the patient and research communities. Rather than start from a guarded position, skeptical that some stakeholders are less concerned about individual patients than others, we assume that all parties are engaging in good faith.” Click here to read the blog.
3. PMC Webinar: Moving Beyond Population Averages — Patient Principles for a Personalized Medicine Research Agenda
The Personalized Medicine Coalition (PMC) is convening patients, patient advocates, caregivers, health care professionals and academic researchers from different disease areas to participate in a series of webinar discussions that will inform a patient-centered research agenda for personalized medicine. These discussions will help develop principles for how to make the research agenda for personalized medicine most helpful to patients. During the second web forum, which will be held Thursday, January 31, 12 p.m. – 1:30 p.m. ET, guest speakers who have led similar projects to establish patient-centered research agendas will share their models for developing research agendas with patient input and lessons learned. To register, click here. You can also find more information about the project here.
4. An Update from the Friends of PCORI Reauthorization Coalition
Organizations supporting PCORI’s reauthorization gathered on January 15, 2019 with Senate staff to discuss PCORI reauthorization. We urge those engaged in advocating for PCORI reauthorization to include PCORI reauthorization in your messages for Hill visits and advocacy days among your members to help educate Congressional members and staff on the great work PCORI is doing. To facilitate your advocacy, we are working on a centralized website that will include advocacy materials and link to PCORI’s state-by-state materials (currently accessible on PIPC’s website) and backgrounders. Please click here to confirm that your organization wants to be listed as a member of Friends of PCORI Reauthorization. Please reach out to [email protected] with any questions or concerns.
5. The PCORI Blog: Making Research More Patient Centered, One Application at a Time
Ashlee Horn, Roycelynn Mentor-Marcel and Laura Forsythe write that patient engagement plays a key role in PCORI-funded projects in a post for The PCORI Blog. “...We've published two papers that demonstrate how patients and other stakeholders contribute to merit review. We used analyses of review scores, reviewer surveys, and focus groups to study our process and how it affected both review outcomes and reviewer experiences. Unsurprisingly, we found that technical merit—whether a project is scientifically sound—is important to all types of reviewers and is the main factor driving which projects we fund.” Click here to read the blog.
6. AcademyHealth Blog: Engagement Science — Factors Fueling the Need for Better Defined Engagement
In a post for AcademyHealth Blog, Holly Peay discusses the need for more patient engagement in medical research. “...Practiced and studied approaches to patient engagement have gained traction and recognition across the field. Funders play a pivotal role in accelerating adoption of these practices; they increasingly encourage, or require, applicants to utilize an ‘engaged’ methodology for studies – or even to conduct a formal evaluation of engagement activities. Targeted funding is often made available through explicit allocations (e.g. PCORI Engagement Awards), delineated requirements for proposal elements, or incentivized engagement through increased likelihood of funding. PCORI, AHRQ, and RWJF are leaders in this space, with funders like the NIH and FDA quickly following their lead.” Click here to read the blog.
7. PIPC: Don't Discriminate on Care
Patients and people with disabilities face major threats as private insurers and government programs look to cut costs using quality-adjusted-life-years (QALYs) and other assessments that discriminate against patients, people with disabilities and seniors. Recently, new threats have emerged, including:
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. In New Zealand, two Tauranga woman, each living with a terminal diagnosis, are calling for an independent inquiry into the Government agency that decides what medicines do and do not get funded, as they fight for better access to "game-changing" life-prolonging drugs in New Zealand. Click here to view. A Scottish patient is looking to leave the country to come to America for treatment of her cancer because it is not considered cost effective for her to receive the care in Scotland. Click here to view. In Canada, click here to view the story of a child with thyroid cancer forced to come to the U.S. for care, and here to learn about patients seeking medications for heart pain being denied access. Spinal muscular atrophy advocates are similarly petitioning Health Canada to expand access to treatment. Click here to view the article. Similarly, in the United Kingdom, activists fight for access to treatments for cystic fibrosis and cancer. Click here and here for articles related to cystic fibrosis, and here for an article related to limited access to treatments for lung cancer. Click here for an article related to MS. Click here for more information on the NHS drug approval process. The Wall Street Journal’s Editorial Board recently opined on "why the U.S. shouldn’t put the world’s most innovative drug market at the mercy of what Greece is willing to pay for a cancer treatment.” Click here to view.
9. Upcoming ICER Studies: Angiodema, Asthma, Opioid Use Disorder, MS, Peanut Allergy, SMA, Depression
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:
Spinal Muscular Atrophy: 12/20/2018-1/31/19 Open Input on draft report!! 3/7/19 Public meeting.
Depression: Revised scoping document available on treatment-resistant depression. 1/14/19 Research Protocol. Also note interventions of interest for 2019 review: Esketamine (Janssen)
Multiple Sclerosis: Revised scoping document available on treatment for secondary progressive multiple sclerosis. 1/14/19 Research Protocol.
Peanut Allergy: Stakeholder list available. Revised scoping document on 12/20/2018. 4/9/19 Draft Evidence Report.
Duchenne Muscular Dystrophy: Open input period 12/20-1/8/2019!!
10. Upcoming Events and Webinars
PCORI Board of Governors Meeting
January 29, 2019
Click here for details.
Patient Registries and Real-World Evidence Summit J
January 30, 2019
Click here for details.
Webinar: Let's Get Real: How Will the FDA Framework for Real-World Evidence Impact You?
January 30, 2019
Click here for details.
Patient Registries and Real-World Evidence Summit J
January 30-31, 2019
Click here for details.''
Webinar: How Real-World Evidence Is Playing Out In The Real World
February 14, 2019
Click here for details.
PCORI Board of Governors Meeting
February 26, 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.
12. Medical Journal Articles
Patient-Community Perspectives on Real-World Evidence: Enhancing Engagement, Understanding, and Trust, click here to view.
A Narrative Review of Data Collection and Analysis Guidelines for Comparative Effectiveness Research in Chronic Pain Using Patient-Reported Outcomes and Electronic Health Records, click here to view.
A Patient-Centered Approach to Comparative Effectiveness Research Focused on Older Adults: Lessons From the Patient-Centered Outcomes Research Institute, click here to view.
Editorial: Evaluating Patient and Public Involvement in Research, click here to view.
Developing a Patient-Centered Outcome for Targeting Early Childhood Obesity Across Multiple Stakeholders, click here to view.
Cancer Patient Perspectives on the Use of Clinical Pathways and Shared Decision-Making in Cancer Care, click here to view.
In Proportion: Approaches for Displaying Patient-reported Outcome Research Study Results as Percentages Responding to Treatment, click here to view.
If Patients Are the True North, Patient-Centeredness Should Guide Research, click here to view.
Understanding and Improving Value Frameworks With Real-World Patient Outcomes, click here to view.
Multi-Method Patient-Engagement Approach: A Case Example from a PCORI-Funded Training Project, click here to view.
Comparative Effectiveness and Safety of Bariatric Procedures for Weight Loss: A PCORnet Cohort Study, click here to view.
Oh, the Places We'll Go: Patient-Reported Outcomes and Electronic Health Records, click here to view.
Putting Patients at the Centre of Healthcare: Progress and Challenges for Health Technology Assessments, click here to view.
13. AHRQ Effective Program Updates
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.
Addressing Social Isolation to Improve the Health of Older Adults: A Rapid Review, click here to view.
Labor Dystocia, click here to view.
Nonsurgical Treatments for Urinary Incontinence in Women: A Systematic Review Update, click here to view.
Randomized Trial of a Patient-Centered Decision Aid for Promoting Informed Decisions about Lung Cancer Screening: Implementation of a PCORI Study Protocol and Lessons Learned, click here to view.
Collaboration Is Key to Accelerating Diagnostics Access to Optimize Benefits of Precision Medicines, click here to view.
Telehealth for Acute and Chronic Care Consultations, click here to view.
Library of Patient-Centered Outcomes Research Resources, click here to view.
In a post for Health Affairs Blog, Tanisha Carino writes that PCORI has done a great job of integrating patient perspectives into its decision-making processes. “Not all foundations have the resources of MJFF; these groups need new sources of financial support. One might be the Patient-Centered Outcomes Research Institute (PCORI), a nongovernmental organization created by the Affordable Care Act to study health outcomes relevant to patients and caregivers. As such, PCORI has gone to great lengths to integrate patient perspectives into its priorities and decision-making processes. PCORI—due for reauthorization by Congress in 2019—has the potential to help disease foundations develop their research infrastructures and be strong partners in the biomedical ecosystem.” Click here to read the blog.
2. PAVE Blog: Putting Our Guard Down — Engaging Multiple Stakeholders To Define Value In Healthcare
In a post for Health Affairs Blog, members of the recently formed Patient-Driven Values in Healthcare Evaluation (PAVE) Center of Excellence discuss their patient engagement goals moving forward. “The organization aims to partner with a broad range of patients and other stakeholders to develop a research agenda, expand existing educational programs to increase the capacity for engagement among patient and research communities, advance methods that capture the voice of the patient in value assessment, and disseminate findings so that messages will reach groups within the patient and research communities. Rather than start from a guarded position, skeptical that some stakeholders are less concerned about individual patients than others, we assume that all parties are engaging in good faith.” Click here to read the blog.
3. PMC Webinar: Moving Beyond Population Averages — Patient Principles for a Personalized Medicine Research Agenda
The Personalized Medicine Coalition (PMC) is convening patients, patient advocates, caregivers, health care professionals and academic researchers from different disease areas to participate in a series of webinar discussions that will inform a patient-centered research agenda for personalized medicine. These discussions will help develop principles for how to make the research agenda for personalized medicine most helpful to patients. During the second web forum, which will be held Thursday, January 31, 12 p.m. – 1:30 p.m. ET, guest speakers who have led similar projects to establish patient-centered research agendas will share their models for developing research agendas with patient input and lessons learned. To register, click here. You can also find more information about the project here.
4. An Update from the Friends of PCORI Reauthorization Coalition
Organizations supporting PCORI’s reauthorization gathered on January 15, 2019 with Senate staff to discuss PCORI reauthorization. We urge those engaged in advocating for PCORI reauthorization to include PCORI reauthorization in your messages for Hill visits and advocacy days among your members to help educate Congressional members and staff on the great work PCORI is doing. To facilitate your advocacy, we are working on a centralized website that will include advocacy materials and link to PCORI’s state-by-state materials (currently accessible on PIPC’s website) and backgrounders. Please click here to confirm that your organization wants to be listed as a member of Friends of PCORI Reauthorization. Please reach out to [email protected] with any questions or concerns.
5. The PCORI Blog: Making Research More Patient Centered, One Application at a Time
Ashlee Horn, Roycelynn Mentor-Marcel and Laura Forsythe write that patient engagement plays a key role in PCORI-funded projects in a post for The PCORI Blog. “...We've published two papers that demonstrate how patients and other stakeholders contribute to merit review. We used analyses of review scores, reviewer surveys, and focus groups to study our process and how it affected both review outcomes and reviewer experiences. Unsurprisingly, we found that technical merit—whether a project is scientifically sound—is important to all types of reviewers and is the main factor driving which projects we fund.” Click here to read the blog.
6. AcademyHealth Blog: Engagement Science — Factors Fueling the Need for Better Defined Engagement
In a post for AcademyHealth Blog, Holly Peay discusses the need for more patient engagement in medical research. “...Practiced and studied approaches to patient engagement have gained traction and recognition across the field. Funders play a pivotal role in accelerating adoption of these practices; they increasingly encourage, or require, applicants to utilize an ‘engaged’ methodology for studies – or even to conduct a formal evaluation of engagement activities. Targeted funding is often made available through explicit allocations (e.g. PCORI Engagement Awards), delineated requirements for proposal elements, or incentivized engagement through increased likelihood of funding. PCORI, AHRQ, and RWJF are leaders in this space, with funders like the NIH and FDA quickly following their lead.” Click here to read the blog.
7. PIPC: Don't Discriminate on Care
Patients and people with disabilities face major threats as private insurers and government programs look to cut costs using quality-adjusted-life-years (QALYs) and other assessments that discriminate against patients, people with disabilities and seniors. Recently, new threats have emerged, including:
- A pharmacy benefit manager’s new plan to limit access to prescription drugs using a rigid cost-per-QALY threshold, similar to that used by the United Kingdom’s National Health Service;
- The use of cost-per-QALY thresholds in New York’s Medicaid program;
- A new Medicare proposal that would set reimbursement for physician-administered medicines based off of decisions made in countries that use WALYs and cost effectiveness thresholds to set national coverage.
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. In New Zealand, two Tauranga woman, each living with a terminal diagnosis, are calling for an independent inquiry into the Government agency that decides what medicines do and do not get funded, as they fight for better access to "game-changing" life-prolonging drugs in New Zealand. Click here to view. A Scottish patient is looking to leave the country to come to America for treatment of her cancer because it is not considered cost effective for her to receive the care in Scotland. Click here to view. In Canada, click here to view the story of a child with thyroid cancer forced to come to the U.S. for care, and here to learn about patients seeking medications for heart pain being denied access. Spinal muscular atrophy advocates are similarly petitioning Health Canada to expand access to treatment. Click here to view the article. Similarly, in the United Kingdom, activists fight for access to treatments for cystic fibrosis and cancer. Click here and here for articles related to cystic fibrosis, and here for an article related to limited access to treatments for lung cancer. Click here for an article related to MS. Click here for more information on the NHS drug approval process. The Wall Street Journal’s Editorial Board recently opined on "why the U.S. shouldn’t put the world’s most innovative drug market at the mercy of what Greece is willing to pay for a cancer treatment.” Click here to view.
9. Upcoming ICER Studies: Angiodema, Asthma, Opioid Use Disorder, MS, Peanut Allergy, SMA, Depression
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:
Spinal Muscular Atrophy: 12/20/2018-1/31/19 Open Input on draft report!! 3/7/19 Public meeting.
Depression: Revised scoping document available on treatment-resistant depression. 1/14/19 Research Protocol. Also note interventions of interest for 2019 review: Esketamine (Janssen)
Multiple Sclerosis: Revised scoping document available on treatment for secondary progressive multiple sclerosis. 1/14/19 Research Protocol.
Peanut Allergy: Stakeholder list available. Revised scoping document on 12/20/2018. 4/9/19 Draft Evidence Report.
Duchenne Muscular Dystrophy: Open input period 12/20-1/8/2019!!
10. Upcoming Events and Webinars
PCORI Board of Governors Meeting
January 29, 2019
Click here for details.
Patient Registries and Real-World Evidence Summit J
January 30, 2019
Click here for details.
Webinar: Let's Get Real: How Will the FDA Framework for Real-World Evidence Impact You?
January 30, 2019
Click here for details.
Patient Registries and Real-World Evidence Summit J
January 30-31, 2019
Click here for details.''
Webinar: How Real-World Evidence Is Playing Out In The Real World
February 14, 2019
Click here for details.
PCORI Board of Governors Meeting
February 26, 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.
12. Medical Journal Articles
Patient-Community Perspectives on Real-World Evidence: Enhancing Engagement, Understanding, and Trust, click here to view.
A Narrative Review of Data Collection and Analysis Guidelines for Comparative Effectiveness Research in Chronic Pain Using Patient-Reported Outcomes and Electronic Health Records, click here to view.
A Patient-Centered Approach to Comparative Effectiveness Research Focused on Older Adults: Lessons From the Patient-Centered Outcomes Research Institute, click here to view.
Editorial: Evaluating Patient and Public Involvement in Research, click here to view.
Developing a Patient-Centered Outcome for Targeting Early Childhood Obesity Across Multiple Stakeholders, click here to view.
Cancer Patient Perspectives on the Use of Clinical Pathways and Shared Decision-Making in Cancer Care, click here to view.
In Proportion: Approaches for Displaying Patient-reported Outcome Research Study Results as Percentages Responding to Treatment, click here to view.
If Patients Are the True North, Patient-Centeredness Should Guide Research, click here to view.
Understanding and Improving Value Frameworks With Real-World Patient Outcomes, click here to view.
Multi-Method Patient-Engagement Approach: A Case Example from a PCORI-Funded Training Project, click here to view.
Comparative Effectiveness and Safety of Bariatric Procedures for Weight Loss: A PCORnet Cohort Study, click here to view.
Oh, the Places We'll Go: Patient-Reported Outcomes and Electronic Health Records, click here to view.
Putting Patients at the Centre of Healthcare: Progress and Challenges for Health Technology Assessments, click here to view.
13. AHRQ Effective Program Updates
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.
Addressing Social Isolation to Improve the Health of Older Adults: A Rapid Review, click here to view.
Labor Dystocia, click here to view.
Nonsurgical Treatments for Urinary Incontinence in Women: A Systematic Review Update, click here to view.
Randomized Trial of a Patient-Centered Decision Aid for Promoting Informed Decisions about Lung Cancer Screening: Implementation of a PCORI Study Protocol and Lessons Learned, click here to view.
Collaboration Is Key to Accelerating Diagnostics Access to Optimize Benefits of Precision Medicines, click here to view.
Telehealth for Acute and Chronic Care Consultations, click here to view.
Library of Patient-Centered Outcomes Research Resources, click here to view.
