
1. PIPC Comments on ICER's Multiple Sclerosis Study, click here to read the letter.
2. PIPC Chairman Tony Coehlo: New York Budget is a Call to Action, click here to read the article.
3. PIPC Comments on IVI's Open-Source Value Platform Model, click here to read the letter.
4. Alliance for Health Policy Briefing on Comparative Effectiveness Research, click here for more information and to register.
5. Congressional Staff Preview PCORI Reauthorization at Bipartisan Policy Center Event, click here to read the article and click here to watch a webcast of the event.
6. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
7. ICER Studies: Type 2 Diabetes, Arthritis, Cardiovascular Disease, Depression, MS, Duchenne Muscular Dystrophy, Peanut Allergy, click here to provide patient input.
8. AUCD: Submit a Proposal for 2019 Conference! Click here for more information.
9. Request for Comment: Definition of Health Technology Assessment, see below for details.
10. PCORI: Getting Useful Evidence to Stakeholders Faster, click here to read the blog post.
11. AHRQ Awards Physician $1.1M Grant for Clinical Decision Support, click here to read the article.
12. Submit 2019 Fly-ins, Advocacy Days, and Conferences to Democratic Caucus, click here to submit your events.
13. Upcoming Events and Webinars, see details below.
14. Medical Journal Articles, see details below.
15. AHRQ Effective Program Updates, see details below.
In a letter to the Institute for Clinical and Economic Review (ICER), Partnership to Improve Patient Care (PIPC) Chairman Tony Coelho offered feedback on ICER's draft evidence report on a treatment for Secondary Progressive Multiple Sclerosis (SPMS). The letter aligns with the National Multiple Sclerosis Society's position that ICER should discontinue the current review because ICER’s scope of its draft evidence report is no longer sufficient based on recent approvals by the FDA. Additionally, PIPC is concerned that ICER’s model includes data from a study that uses “negative utilities” which implies ICER is assuming there are health states worse than death. Also, once again, ICER fails to capture patient and caregiver preferences, and relies on faulty outdated data. "ICER has once again missed the mark by showing callous disregard for patients," wrote Chairman Coelho. "Instead of working to engage with MS patients and taking their preferences and needs into consideration in evaluating a treatment designed for MS patients, ICER instead has chosen to rely on dated studies and mechanisms that are widely considered flawed." Click here to read the letter.
2. PIPC Chairman Tony Coehlo: New York Budget is a Call to Action
In spite of advocates' efforts to educate lawmakers on how QALYs discriminate against people with disabilities and apply arbitrary notions of "value" to individuals' lives, the New York State budget included language that would allow for QALY-based value assessments. "Going forward, PIPC will work harder to amplify the voices of advocates calling on policymakers to value our health. It will take your leadership, your guidance and your time to fight back against emerging threats at the state and federal level to restrict access to care based on discriminatory value assessments from groups like ICER. We have to ask, “Value to whom?” Because when value assessments based on averages are applied to coverage decisions, we get caught in the middle without access to care. We know different people respond differently to the same treatments. We know one size does not fit all. And we are going to fight back against anyone that tries to say that value is a one-size-fits-all proposition. I hope you will join PIPC in this fight by sharing your experiences with us and letting us know how we can support your work to convince policymakers to value our health," wrote PIPC Chairman Tony Coehlo. Click here to read the article.
3. PIPC Comments on IVI's Open-Source Value Platform Model
The Partnership to Improve Patient Care (PIPC) submitted a comment letter to the Innovation and Value Initiative (IVI) on their first oncology-specific Open-Source Value Platform (OSVP) model focused on non-small cell lung cancer (NSCLC). PIPC Chairman Tony Coelho complimented the detail and comprehensiveness of the IVI's model, noting the tremendous value that open-source models offer. However, Chairman Coelho strongly encouraged IVI not to utilize the flawed quality-adjusted-life-year metric in its multi-criteria decision analysis (MCDA) component. "As QALYs are assigned by both quality as well as quantity of life, an incremental QALY assessment would prioritize providing treatment to a non-disabled population with a longer theoretical life expectancy, and otherwise perfect health, over a population with a disability or chronic condition," wrote Chairman Coelho. "It is our hope that IVI chooses to be innovative in moving beyond the QALY." Click here to read the letter.
4. Alliance for Health Policy Briefing on Comparative Effectiveness Research
On Wednesday, April 17, the Alliance for Health Policy will hold a briefing for Congressional staff and other interested parties on the purposes and perspectives surrounding comparative effectiveness research. Panelists will include Alfiee Breland-Noble, Ph.D. of Georgetown Medical Center, John Bulger of Geisinger Health Plan, Eleanor Perfetto, Ph.D. of the National Health Council, Sean Tunis, M.D. of the Center for Medical Technology Policy, and Gail Wilensky, Ph.D. of Project HOPE. The briefing, which is open to the public, will be held from 12:00 p.m. to 1:30 p.m. on April 17 in room G-50 of the Dirksen Senate Office Building. Click here for more information and to register.
5. Congressional Staff Preview PCORI Reauthorization at Bipartisan Policy Center Event
At an event on the applications of comparative effectiveness research and needed future investments in it, Congressional staff discussed the efforts to reauthorize PCORI. "Comparative effectiveness research, which essentially studies two different health interventions to figure out what works best, generally has bipartisan support once congressional members and staffers understand the program....Lawmakers are in the early stages of taking up a reauthorization bill and are currently soliciting feedback from stakeholders, Alyssa Penna, a health policy adviser for Sen. Chris Van Hollen (D-Md.), said during the event. Van Hollen, along with Sens. Mark Warner (D-Va.) and Bill Cassidy (R-La.), is leading the reauthorization bill in the Senate," wrote Bloomberg News. Click here to read the article and click here to watch a webcast of the event.
6. 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: PHARMAC faces criticism over a lack of transparency and timeliness. Click here for more. 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 to read more. PHARMAC has claimed to cover a breast cancer treatment that it does not cover. Patients in New Zealand also face barriers to access for treatment of spinal muscular atrophy. Click here to read more. New Zealand advocates unfavorably compare its cancer drug coverage to other countries.
- 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 in an attempt to access needed cystic fibrosis drugs. The elderly are going blind as the NHS ignores advice to end cataract surgery rationing. Epilepsy Society calls for review of UK supply chain following "steep rise" in access issues. 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. Click here for an article about the fight for access to spinal muscular atrophy drugs.
- France: Patients are alarmed as a multiple sclerosis drug will no longer be covered.
7. ICER Studies: Type 2 Diabetes, Arthritis, Cardiovascular Disease, 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: Public Comment period OPEN through 5/1/2019 on Draft Scoping Document. Meeting 10/31/2019: CTAF to an update to its 2017 rheumatoid arthritis assessment.
- Type 2 Diabetes: Open Input Period through 4/29/2019. Draft Scoping Document 5/2/2019. 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.
- Cardiovascular Disease : 4/15/2019: Revised scoping document. 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.
- 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: Evidence Report 5/2/2019. Meeting 5/23/2019: Midwest CEPAC to review ICER's assessment of siponimod (Novartis) for secondary progressive multiple sclerosis.
- Peanut Allergy: Draft Evidence Report available. Public Comments OPEN through May 8, 2019. Meeting 6/11/2019: CTAF to review ICER's assessment of treatments for peanut allergy.
- Duchenne Muscular Dystrophy: Model Analysis Plan 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.
8. AUCD: Submit a Proposal for 2019 Conference!
The AUCD Conference welcomes proposals to highlight the work of the disability community. Over 200 different presentations will be accepted to the conference in several different types of sessions, formatted for maximum visibility and interaction with attendees. Proposals are encouraged from students, self-advocates, family leaders, clinicians, and researchers in over 25 topics. Click here for more information.
9. 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. The deadline to comment is April 30. Click here for more information.
10. PCORI: Getting Useful Evidence to Stakeholders Faster
PCORI has posted a new round of systematic evidence reviews on induction of labor, radiation therapy for brain metastases, and dyspnea in patients with advanced cancer. "Our published review updates, conducted in partnership with the Agency for Healthcare Research and Quality, focus on the effectiveness of drug and behavioral treatments for posttraumatic stress disorder; the effectiveness of drug therapies for patients who have had rheumatoid arthritis for a year or less; the benefits and harms of drug and nondrug treatments for urinary incontinence, the uncontrollable loss of urine; and the evaluation of evidence on stroke prevention in patients who have atrial fibrillation, an irregular heartbeat," PCORI wrote in a blog. Click here to read the blog post.
11. AHRQ Awards Physician $1.1M Grant for Clinical Decision Support
AHRQ has awarded $1.1 million to a physician for his work furthering clinical decision support tools. "A professor at the Feinstein Institute for Medical Research, [Thomas] McGinn serves as senior vice president and deputy physician-in-chief at Northwell Health. He worked with a team of clinical researchers at the Feinstein Institute’s Center for Health Innovations and Outcomes Research (CHIOR) to study, test, and distribute clinical decision support in EHR systems....CHOIR is working to develop a clinical decision support system based on validated clinical prediction rules for detecting blood clots in the lungs and bleeding risk in hospitalized patients. CHOIR will assess the system’s ability to affect care in emergency medicine and inpatient care settings as part of the research project," wrote EHR Intelligence. Click here to read the article.
12. 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.
13. Upcoming Events and Webinars
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.
Alliance for Health Policy Briefing: Right Care, Right Patient, Right Time: The Role of Comparative Effectiveness Research
April 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.
2019 AUCD Annual Meeting
November 17-20, 2019
Click here for details.
14. Medical Journal Articles
Addressing Health System Values in Health Technology Assessment: The Use of Evidence-Informed Deliberative Processes, click here to view.
Launch of AHRQ's Systematic Review Data Repository (SRDR)+, click here to view.
Knowledge and Use of Evidence-Based Medicine in Daily Practice by Health Professionals: A Cross-Sectional Survey, click here to view.
Quantifying Preferences in Drug Benefit-Risk Decisions, click here to view.
Raising the Impact of Real World Evidence, click here to view.
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.
15. AHRQ Effective Program Updates
Systematic Review: Can Physical Activity Improve the Health of Wheelchair Users?, click here to view.
Comment Period: Registries for Evaluating Patient Outcomes: A User's Guide: Fourth Edition, click here to view.
Treatment of Acute Pain: An Evidence Map, click here to view.
Systematic Review Pharmacologic and Non-pharmacologic Therapies in Adult Patients with Acute Exacerbation of COPD, click here to view.
Integrating Palliative Care With Chronic Disease Management in Ambulatory Care, click here to view.
Systematic Review: Adverse Effects of Pharmacological Treatments of Major Depression in Older Adults, click here to view.
Comment Period: Management of Primary Headaches in Pregnancy, 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.