1. PIPC Comments on ICER's Treatment-Resistant Depression Study, click here to read the letter.
2. PIPC Chairman Tony Coehlo Headlines Headache and Migraine Policy Forum Panel, click here to read more.
3. IHP Article: Patient Advocates are Concerned About Comparative Effectiveness Data Being Used to Deny Access, click here to read the story.
4. PIPC Comments on ICER's Multiple Sclerosis Study, click here to read the letter.
5. Washington Examiner: HHS Secretary Alex Azar Doesn't Want Drug Access to Become an Equation, click here to read the article.
6. IFPA: Who Controls ICER?, click here to read the fact sheet.
7. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
8. ICER Studies: Type 2 Diabetes, Arthritis, Cardiovascular Disease, Depression, MS, Duchenne Muscular Dystrophy, Peanut Allergy, click here to provide patient input.
9. AUCD: Submit a Proposal for 2019 Conference! Click here for more information.
10. Request for Comment: Definition of Health Technology Assessment, see below for detail.
11. PCORI Releases New Evidence Maps, click here to view PCORI's new evidence maps and click here to read more.
12. Alliance for Health Policy Holds Capitol Hill Briefing, click here to read more.
13. Submit 2019 Fly-ins, Advocacy Days, and Conferences to Democratic Caucus, click here to submit your events.
14. Upcoming Events and Webinars, see details below.
15. Medical Journal Articles, see details below.
16. 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 provided feedback on ICER's draft evidence report for Treatment-Resistant Depression (TRD). The letter offers criticism of ICER's model for inaccurately accounting for the cost burden of TRD, as well as ICER's misleading estimates on mortality rates associated with the disease. Chairman Coelho encouraged ICER to abandon discriminatory value assessment metrics such as the Quality-Adjusted-Life-Year (QALY), and instead focus on outcomes that truly matter to patients. "As the National Alliance on Mental Illness (NAMI) highlighted in its November comment letter to ICER, individuals with treatment resistant depression (TRD) are in desperate need of treatments that offer fast, effective relief," wrote Chairman Coelho. "The ICER model fails to capture the value of the treatment’s immediate impact. For patients, the ability to quickly get back to work and their families is invaluable." Click here to read the letter.
2. PIPC Chairman Tony Coehlo Headlines Headache and Migraine Policy Forum Panel
The Headache and Migraine Policy Forum held a panel discussion on Capitol Hill featuring patients, advocates, and policy experts to explore how people living with migraine diseases can sustain employment. "PIPC Chairman Tony Coehlo delivered the event’s keynote address. 'You’re all covered by the [Americans with Disabilities Act],' Mr. Coelho announced to a crowded room of headache advocates. 'I know,' he emphasized, 'because I wrote it.' The former representative from California has dealt with epilepsy throughout his life, spurring his leadership on disability issues. He authored the landmark Americans with Disabilities Act while serving in Congress and today chairs the advocacy group Partnership to Improve Patient Care. Mr. Coelho shared his past struggles and encouraged the advocates present to use their own stories to spur members," wrote the Headache and Migraine Policy Forum in their summary of the event. Click here to read more.
3. IHP Article: Patient Advocates are Concerned About Comparative Effectiveness Data Being Used to Deny Access
PIPC Executive Director Sara van Geertruyden was quoted in an Inside Health Policy article saying that comparative effectiveness research can help get the right treatments to the right patients, but should not be used to limit patient access. "Comparative effectiveness improves decision making by making sure the right patient gets the right treatment at the right time. But when you are talking about allowing coverage decisions based on averages to drive health care decision-making for individual patients, you allow payers and governments to drive one-size-fits-all decisions for patients," she told Inside Health Policy. Click here to read the story.
4. PIPC Comments on ICER's Multiple Sclerosis Study
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.
5. Washington Examiner: HHS Secretary Alex Azar Doesn't Want Drug Access to Become an Equation
An article for the Washington Examiner quotes Health and Human Services (HHS) Secretary Alex Azar as opposing QALYs in federal programs, saying that instead patients determine value. "Azar was explicit in his opposition to the use of QALYs in federal programs. Azar said he had a 'special responsibility' to protect patient access to drugs in these public programs and does not 'buy into the idea that QALYs' represent an 'objectively identifiable notion of value.' Azar insisted that the market determines value, not studies by health economists....Azar’s comments on the use of QALYs point to a major cultural difference between the U.S. and the rest of the developed world. In Europe, patients are regularly denied access to innovative, sometimes life-saving treatments based upon the recommendations of actuaries and bean-counters. European citizens are inured to a system in which government economists make these decisions," wrote William Smith in the Washington Examiner. Click here to read the article.
6. IFPA: Who Controls ICER?
The Institute for Patient Access (IFPA) published a fact sheet about ICER, its methods, its supporters, and how it harms patients. "Patients, in consultation with their doctors, should make their own medical decisions. But with increasing frequency, an outside organization known as the Institute for Clinical and Economic Review is influencing what treatment options are available for consideration. Today, patients’ access to innovative drugs, life-saving devices and diagnostic testing often rides on this third-party’s value assessment....patients and providers have remarked that ICER does not adequately incorporate the intangible benefits of good health. These include quality-of-life factors like being physically able to perform an activity or attend a function, as well as having the emotional wellbeing to engage with family and friends, at work or in community activities," IFPA wrote. Click here to read the fact sheet.
7. 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: After an inquiry into PHARMAC was blocked, breast cancer advocates are speaking out. Click here, here, and here to learn 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 and here to read more. New Zealand advocates unfavorably compare its cancer drug coverage to other countries. A treatment for CLN2 Batten's disease is not covered in New Zealand despite being considered a lifesaving drug in Australia.
- Canada: Discriminatory cost effectiveness measures are blocking access to care for patients with spinal muscular atrophy patients. Click 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 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.
8. 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 : 5/20/2019: Research Protocol. 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: 5/9/2019: Evidence Report. Meeting 5/23/2019: Midwest CEPAC to review evidence on esketamine as a therapy for treatment-resistant depression.
- Multiple Sclerosis: 5/2/2019: Evidence 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 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: 10/8/2019: Final Report.
9. 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.
10. 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.
11. PCORI Releases New Evidence Maps
PCORI's Evidence Synthesis Initiative provides overviews of research findings to patients, clinicians, policy makers, and others who need that information to make the critical healthcare decisions they face daily. As part of the initiative, PCORI released new Evidence Maps, entitled Treatments for Fatigue in Multiple Sclerosis and The Impact of mHealth for Self-Management of Chronic Disease on Patient-Centered Outcomes. These evidence maps follow systematic reviews on induction of labor, radiation therapy for brain metastases, and dyspnea in patients with advanced cancer. PCORI continues to solicit input as it considers future topics for review. Click here to view PCORI's new evidence maps and click here to read more.
12. Alliance for Health Policy Holds Hill Briefing
The Alliance for Health Policy held a briefing this week for Congressional staff and other stakeholders focusing on how comparative effectiveness research can promote effective patient care. "The evidence needs not only to tell which treatment works best, but what works best for which patients. Having patients involved as partners in the research is important to achieving that. Patients need to be involved as study participants or through the data collection but need to be collaborating with researchers from the design of the study all the way through analysis of the results. Treatments and tests are rarely, if ever, “always” or “never” effective, so it is important that research investigates the nuances of effects on different individuals....Important steps to making this happen include focusing on conditions rather than interventions and starting with high-cost conditions that have great variation in treatments. While double-blind randomized control trials (RCTs) are often thought of in research as the “gold standard” we need to rethink this. RCTs introduce their own biases and limitations; all data has limitations. We need to understand these limitations and work to triangulate research methods to account for this," the Patient-Centered Primary Care Collaborative wrote in a post about the event. Click here to read the article.
13. 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.
14. Upcoming Events and Webinars
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.
15. Medical Journal Articles
How Well Does the Patient-Centered Outcomes Research Institute Fund Primary Care and Comparative Effectiveness Research?, click here to view.
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.
16. AHRQ Effective Program Updates
White Paper: Standardized Library of Asthma Outcome Measures, click here to view.
Research Protocol: Characteristics of Existing Asthma Self-Management Education Packages, click here to view.
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.