
1. PCORI Advocacy 101: The Politics and Policy of PCORI Reauthorization, click here for a complete recording of the event and the slides used in the presentation.
2. Gottlieb: As FDA Advances RWE Use, Groups Other Than ICER Needed, click here to read more.
3. To Improve Health Care, Focus on Clinical Measures and Listen to Patients, click here to read more.
4. Now Is the Time to Develop Patient-Centered Quality Measures, click here to read the article.
5. Proposed Changes to ICER Framework Available for Comment, click here to view the changes and to comment and see below for more.
6. ICER Announces Possible 2020 Assessments, click here to see the list of possible assessments.
7. AAR Seeks Recruits for Annual Senior Patient and Family Caregivers Network Training, click here to learn more and to apply for the training.
8. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
9. ICER Studies: Acute Migraine, Type 2 Diabetes, Arthritis, Cardiovascular Disease, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, 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
On September 13, 2019 the Partnership to Improve Patient Care (PIPC) held an 'Advocacy 101' webinar to help explain the Institute’s pending reauthorization and offer suggestions on how stakeholders can support patient-centered research. Topics covered in the webinar include a background related to PCORI’s creation, the legislative process that we anticipate for reauthorization in 2019, and appropriate tactics for outreach to legislators to ensure they understand the personal impact that PCORI’s work has on patients and other stakeholders. Click here for a complete recording of the event and the slides used in the presentation.
2. Gottlieb: As FDA Advances RWE Use, Groups Other Than ICER Needed
As the Food and Drug Administration (FDA) develops and promotes a model for real-world evidence (RWE) use in post-market decisions, groups other than ICER will be needed to analyze therapies. "If we do end up relying on third-party expert assessments to inform some of the decisions that get made, it’s not going to be ICER,” Gottlieb said on September 11 at a panel on payment reform for breakthrough innovations hosted by the Duke-Margolis Center for Health Policy. "You’re going to need to have a competitive market for groups like that, that are seen as truly impartial, independent, and we don’t have that right now." Click here to read more.
3. To Improve Health Care, Focus on Clinical Measures and Listen to Patients
A study led by Academy Health found that to increase "value" in health care, practitioners should take a big-picture view and incorporate patients' own experiences into decision-making. The review "shows that most efforts to decrease low-value care have based their measurement of success on how much they reduced the overall use of certain tests and treatments. Far fewer looked at whether these efforts actually ensured that patients got more appropriate care and avoided unintended negative consequences," writes the University of Michigan's Kara Gavin. "In general, the team says, researchers and evaluators should incorporate more clinically meaningful and patient-centered measures into studies to provide a more comprehensive understanding of the impact of these interventions." Click here to read more.
4. Now Is the Time to Develop Patient-Centered Quality Measures
Writing for Health Affairs, a team of health policy experts say that the time is now for developing and implementing patient-centered outcomes measures. Agencies are focused on implementing President Trump's health care executive order, which focused in part on quality. "A confluence of enabling factors has set the stage for the immediate development and adoption of patient-centered outcomes measures. The executive order requires the development of a quality roadmap within 180 days of issue. At the same time, there is an opportunity to build on ongoing efforts to define populations for whom providers will be held accountable," the experts say. Click here to read the article.
5. Proposed Changes to ICER Framework Available for Comment
ICER has released its proposed changes to its value assessment framework for 2020. Public comment is open on the proposed changes through October 18, 2019. Click here to view the changes and to comment. Click here to view a comment letter signed by over 30 stakeholders. PIPC looks forward to working again with stakeholders to comment on this new opportunity. Click here and here to view videos about ICER's methodology.
6. ICER Announces Possible 2020 Assessments
The Institute for Clinical and Economic Research (ICER) published a list of possible assessments for 2020. The possible assessments include drugs to treat cystic fibrosis, breast cancer, multiple sclerosis, and HIV. ICER's QALY-based studies are not transparent, use discriminatory cost-effectiveness measures, and ignore patient preferences and input. Click here to see the list of possible assessments.
7. AAR Seeks Recruits for Annual Senior Patient and Family Caregivers Network Training
Alliance for Aging Research’s Senior Patient and Family Caregiver Network (SP&FCN) is seeking advocates to participate in a research-advocacy training program designed to empower senior patients and their family caregivers to engage in patient-centered outcomes research (PCOR). The training will be held November 19-21, 2019 in Dallas, TX, and reimbursements for a hotel stay and travel expenses are available. In particular, the Alliance is looking for patients or caregivers of patients with Alzheimer’s disease, sarcopenia, atrial fibrillation, chronic pain, age-related macular degeneration, or heart valve disease. Click here to learn more and to apply for the training.
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.
- Australia: While blood cancer rates are worse than previously thought in Australia, patients are having trouble accessing needed medications.
- New Zealand: Cancer patients are dying as they await a decision on treatment coverage. Child forced to go to Australia for treatment as Pharmac fails to cover a spinal muscular atrophy therapy. Patient says that government's failure to cover a lifesaving gene therapy could be a "death sentence" for future patients. Pharmac refuses to fund lifesaving cancer treatment.
- Canada: Duchenne muscular dystrophy patients raise awareness of a lack of approved treatments in Canada.
- United Kingdom: Of two sisters with cystic fibrosis, only one is receiving critical treatment. NHS refuses to fund "extremely successful" cancer drug. Father dying without needed cancer drug as his son asks him when he will get better.
9. 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: Open Input Period through 9/24/2019. 9/27/2019: Draft Scoping Document.
- Cystic Fibrosis: Open Input Period through 9/25/2019. 9/30/2019: Draft Scoping Document.
- Sickle Cell Disease: Draft Scoping Document and Stakeholder List available, comment period OPEN through 9/20/2019. 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: Model Analysis Plan available. 9/24/2019: Draft Evidence Report. Meeting 11/19/2019: CTAF to an update to its 2017 rheumatoid arthritis assessment.
- Type 2 Diabetes: Draft Evidence Report and Voting Questions available. Public Comment period OPEN through 10/8/2019. 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 : Response to comments, evidence report, and revised voting questions AVAILABLE. 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.
- Unsupported Price Increase Assessment: 10/8/2019: Final Report.
- Acute Migraine: Research Protocol available. 9/24/2019: Model Analysis Plan. Meeting 1/23/2020: Midwest CEPAC to review ICER's assessment of acute migraine treatments
- Valuing A Cure Project: White Paper AVAILABLE. Meeting 9/17/2019: Invited stakeholders to discuss single or short-term transformative therapies.
- 2020 Value Assessment Framework: Proposed changes AVAILABLE. Comment period open through 10/18/2019.
10. Upcoming Events and Webinars
Health Spending: Moving from Theory to Action
September 11, 2019
Click here for details.
PCORI Improving Methods Applicant Town Hall
September 12, 2019
Click here for details.
ISPOR Latin America
September 12-14, 2019
Click here for details.
Advisory Panel on Rare Disease Fall 2019 Meeting
September 16, 2019
Click here for details.
PCORI Advisory Panel on Patient Engagement Fall 2019 Meeting
September 17, 2019
Click here for details.
ICER's Valuing Cures Summit
September 17, 2019
Click here for details.
IMPACCT: Real World Evidence
September 18-19, 2019
Click here for details.
2019 PCORI Annual Meeting
September 18-20, 2019
Click here for details.
CMS Public Stakeholder Meeting: Resources to Reduce Opioid Misuse
September 20, 2019
Click here for details.
Cycle 3 2019 Broad PFA Applicant Town Hall
September 25, 2019
Click here for details
FT Pharma Pricing and Value Summit 2019
September 26, 2019
Click here for details.
Data Capacity for Patient-centered Outcomes Research for People with Multiple Chronic Conditions
October 2-3, 2019
Click here for details.
ISPOR Summit 2019 on Building Trust in RWE – The Role of Study Registration
October 11, 2019
Click here for details.
2019 AUCD Annual Meeting
November 17-20, 2019
Click here for details.
11. Medical Journal Articles
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.
An Ethical Analysis of Coverage With Evidence Development, click here to view.
How Different Is Research Done by the Patient-Centered Outcomes Research Institute, and What Difference Does it Make?, click here to view.
The Importance of Measuring the Impact of Patient-Oriented Research, click here to view.
Building Evidence and Measuring Clinical Outcomes for Genomic Medicine, click here to view.
Evidence-Based Medicine: A Data-Driven Approach to Lean Healthcare Operations, click here to view.
Patient Registries: An Underused Resource for Medicines Evaluation: Operational Proposals for Increasing the Use of Patient Registries in Regulatory Assessments, click here to view.
Commonly Used Definitions in Real-World Studies May Underestimate the Prevalence of Renal Disease Among Nonvalvular Atrial Fibrillation Patients, click here to view.
Factors Associated with Evidence-Based Decision-Making Among Patients and Providers, click here to view.
12. AHRQ Effective Program Updates
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.
Systematic Review: Antipsychotics for the Prevention and Treatment of Delirium, click here to view.
Systematic Review: Comparative Effectiveness of Analgesics To Reduce Acute Pain in the Prehospital Setting, click here to view.
Blog: Innovations to Foster Evidence-based Care in Learning Health Systems: Ideas from the AHRQ Evidence-based Practice Centers, click here to view.
Research Protocol: Prevention, Diagnosis, and Management of Opioids, Opioid Misuse and Opioid Use Disorder in Older Adults, click here to view.
AHRQ EPC Pilot Projects Summary: Improving Health Systems' Access to High Quality Evidence, click here to view.
Research Protocol: Care Interventions for People With Dementia (PWD) and Their Caregivers, click here to view.
Research Protocol: Impact of Community Health Worker Certification on Workforce and Service Delivery for Asthma and Other Selected Chronic Diseases, click here to view.
AHRQ EPC Program Helps Health Systems Use Evidence, click here to view.