1. AAPD, NCD, and NCIL Caution About the Discriminatory Implications of the QALY and Foreign Reference Pricing, click here to view the webinar.
2. Proposed 'Most Favored Nations' Order Would Import Discriminatory Value Assessments, see details below.
3. New Patient-Centered Research Agenda for Personalized Medicine, click here to read the report.
4. National Academy of Medicine Framework for COVID-19 Vaccine Allocation, see details below.
5. Canadian Cystic Fibrosis Advocate Cautions Americans Against Importing Foreign Drug Pricing Policies, click here to read the blog post.
6. Opinion: Global Pharmaceutical Models Would Destroy U.S. Innovation, click here to read the op-ed.
7. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
8. PCORI Annual Meeting September 16-17, click here to learn more and to register.
9. ICER's QALY-Based Study Topics: Hemophilia A, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, Bladder Cancer, Opioid Treatments, High Cholesterol, Anemia in Chronic Kidney Disease, Lupus Nephritis, 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.
The American Association of People with Disabilities (AAPD) released a webinar featuring an interview between Maria Town, the President/CEO of AAPD, and Ana Torres Davis, Senior Attorney for the National Council on Disability (NCD), as well as a presentation by Kelly Buckland, Executive Director of the National Council on Independent Living (NCIL), about dangers of quality-adjusted life years (QALYs) for discrimination against people with disabilities and their importation from foreign countries into Medicare. The webinar focused on the National Council on Disability’s 2019 report that recommended that the administration rescind a proposal to use an international pricing index in Medicare and strongly advocated against President Trump’s proposed Executive Order due to its implications for importing discrimination and restricted access to care for people with disabilities. Click here to view the webinar.
2. Proposed 'Most Favored Nations' Order Would Import Discriminatory Value Assessments
The administration is preparing to release an Executive Order importing QALY-based prices to Medicare, hurting patients, and reducing access. PIPC Chairman Tony Coelho has long been fighting against these changes, writing in February that a similar proposal would "would do little to reduce out-of-pocket costs and would harm people with disabilities and serious chronic conditions." Click here to read the blog post.
The National Council on Disability (NCD) released a statement after the signing of the recent Executive Order calling out the harms of using an international pricing index -- which relies on the use of discriminatory measures like QALYs. Click here to read the statement.
Chairman Coelho also reiterated his concern in an op-ed in The Hill, writing that with the signing of an Executive order importing QALY-based prices to Medicare, the administration is subordinating the health of individuals with disabilities, older adults, and patients with chronic diseases to election-year politics and cost concerns. "In the last week, we watched President Trump panic at his sinking poll numbers and decide to issue a slew of executive orders — more to create talking points for his campaign about how he was “lowering drug prices” and with little consideration for the practical reality that the policies do nothing to provide relief to Medicare beneficiaries with their out-of-pocket costs," Chairman Coelho writes. "The most troubling is the one not yet released, which the president has described as imposing deep price cuts on physician-administered treatments in Medicare by importing prices set by foreign governments based on the 'patient unfriendly' quality-adjusted life year (QALY) metric." Click here to read the article.
3. New Patient-Centered Research Agenda for Personalized Medicine
The Personalized Medicine Coalition published a new patient-centered research agenda, in which education and access issues emerge as patients' top priorities for personalized medicine. By focusing research on questions in these areas, the agenda creates a path toward a new era of patient-centered personalized medicine. Reflecting on their encounters with the health care system and their perspectives on personalized medicine, the participants in the project focused largely on their desire for research that could improve the quality and quantity of interactions between patients and providers of health care services, with an emphasis on the importance of education and access. Click here to read the report.
4. National Academy of Medicine Framework for COVID-19 Vaccine Allocation
The National Academy of Medicine will invite public comments starting September 1 on a discussion draft of the Preliminary Framework for Equitable Allocation of COVID-19 Vaccine, a study commissioned by NIH and CDC. The study will recommend priorities to inform allocaiton of a limited initial supply of a vaccine. The National Academy of Medicine seeks to take into account factors such as risk associated with health status and social determinants of health. The body will hold a public listening session on September 2 from 12 to 5 p.m. Eastern. Click here to register. Click here to learn more about the comment period.
5. Canadian Cystic Fibrosis Advocate Cautions Americans Against Importing Foreign Drug Pricing Policies
Sandy Stevens shares her personal story of struggling to access needed medication for her fourteen year old daughter, Laura, who lives with Cystic Fibrosis. Sandy strongly cautions Americans against implementing policies that would model Canada’s drug pricing scheme, including the most recent “most favored nations” Executive Order. She describes the persistent challenges Laura faced in Canada in attempting to access drugs prescribed to her by her physicians, including being forced to become so sick she was on a lung transplant list before qualifying for the prescribed treatment. Click here to read the blog post.
6. Opinion: Global Pharmaceutical Models Would Destroy U.S. Innovation
In an op-ed for the Dallas Morning News, Tom Kowalski of the Texas Healthcare and Bioscience Institute in Austin writes about the detrimental impact that international reference pricing would have on U.S. pharmaceutical innovation. "As scientists race for treatments and vaccines to stamp out the spread of COVID-19, American pharmaceutical companies are helping lead the way, with groundbreaking, rapidly developing research that has allowed us to hold out collective hope for an end to this pandemic," he writes. "Yet, while the government has wisely stepped up its partnership with the private sector, investing resources and loosening regulations that would hamper our ability to rush toward a cure, it is on the precipice of making a devastating mistake that could bring innovation to a devastating halt." Click here to read the op-ed.
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.
- Australia: After government refuses to subsidize a lifesaving prostate cancer drug, the manufacturer makes it free.
- Canada: Life-saving cystic fibrosis drug remains unavailable in Canada. The drug was found to be "life-changing" in a Dalhousie University study.
- New Zealand: New Zealand man fights for access to a treatment that is available to his brothers living elsewhere. Pioneering health researcher who fought for access to cystic fibrosis treatments dies.
- United Kingdom: Nurse in desperate fight for cancer treatment given just months to live. Click here to read more.
8. PCORI Annual Meeting September 16-17
Make plans now to attend PCORI's sixth Annual Meeting, Accelerating Impact on Care and Patient-Centered Outcomes, being held virtually September 16-17. The meeting will highlight results from several PCORI-funded studies and explore how we can shorten the time from research to improved health for patients. During the meeting, attendees will:
- Learn about our efforts to disseminate research findings
- Network with others
- Examine PCORI’s emphasis on engagement
- Explore participation in developing our research agenda
- Listen to powerful, inspiring keynote speakers
Click here to learn more and to register.
9. ICER's QALY-Based Study Topics: Hemophilia A, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, Bladder Cancer, Opioid Treatments, High Cholesterol, Anemia in Chronic Kidney Disease, Lupus Nephritis
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.
- Ulcerative Colitis: Draft Evidence Report and Draft Voting Questions AVAILABLE. 9/2/2020: Revised Voting Questions and Evidence Report. Meeting 9/24/2020: CTAF will convene to deliberate and vote on evidence presented in ICER's report on ulcerative colitis therapies.
- Cystic Fibrosis: Meeting 8/27/2020: CTAF convened to deliberate and vote on evidence presented in ICER's report on treatments for cystic fibrosis. 9/23/2020: Final Evidence Report.
- Sickle Cell Disease: Evidence Report and Responses to Comments AVAILABLE. Meeting POSTPONED: New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for sickle cell disease.
- Hemophilia A: Draft Evidence Report AVAILABLE. Comment period OPEN through 9/23/2020.
- Bladder Cancer: Model Analysis Plan available. 9/17/2020: Draft Evidence Report.
- Opioids: Digital Apps: Model Analysis Plan available. 9/17/2020: Draft Evidence Report.
- Opioids: Supervised Injection Centers: Model Analysis Plan available. 9/24/2020: Draft Evidence Report.
- High Cholesterol: Revised Scoping Document available. 9/8/2020: Research Protocol.
- Anemia in Chronic Kidney Disease: Research Protocol available. 10/14/2020: Model Analysis Plan.
- Unsupported Price Increase Assessment: 1/8/2021: Final Assessment and Report.
- Lupus Nephritis: Draft Scoping Document AVAILABLE, Comment Period OPEN through 9/21/2020, Meeting 3/26/2021: New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for lupus nephritis.
- Service Dogs for PTSD: Open Input Period through 10/2/2020.
10. Upcoming Events and Webinars
National Academy of Medicine Listening Session on COVID-19 Vaccine Allocation Priorities
September 2, 2020
Click here for details.
PCORI Cycle 3 2020 Improving Methods Applicant Town Hall
September 8, 2020
Click here for details.
2020 PCORI Annual Meeting (A Virtual Event): Accelerating Impact on Care and Patient Outcomes
September 16-17, 2020
Click here for details.
STAT News: Getting Real World Data about Covid-19
September 17, 2020
Click here for details.
PCORI: New Evidence to Inform Decisions
October 16, 2020
Click here for details.
11. Medical Journal Articles
Potential Impact of Missing Outcome Data on Treatment Effects in Systematic Reviews: Imputation Study, click here to view.
Advancing Community-Engaged Research: Increasing Trustworthiness Within Community-Academic Partnerships, click here to view.
Patient and Other Stakeholder Engagement in Patient-Centered Outcomes Research Institute Funded Studies of Patients with Kidney Diseases, click here to view.
Patient-Reported Outcomes: Central to the Management of COVID-19, click here to view.
How to Include Patient-Reported Outcome Measures in Clinical Trials, click here to view.
Evaluation of the Use of Cancer Registry Data for Comparative Effectiveness Research, click here to view.
Outcome-Based Payment Schemes: What Outcomes Do Patients with Cancer Value?, click here to view.
Engaging Patients and Stakeholders in Preresearch: Findings from the Pipeline to Proposal Awards Initiative, click here to view.
Why Clinical Trials May Not Help Patients Make Treatment Decisions: Results from Focus Group Discussions with 22 Patients, click here to view.
Defining Patient Engagement in Research: Results of a Systematic Review and Analysis: Report of the ISPOR Patient-Centered Special Interest Group, click here to view.
12. AHRQ Effective Program Updates
OPEN FOR COMMENT THROUGH 9/16/2020: Key Questions: Transitions of Care from Pediatric to Adult Services for Children with Special Healthcare Needs. Click here to view.
Technical Brief: Strategies for Patient, Family, and Caregiver Engagement. Click here to view.
Research Protocol: Interventions To Decrease Hospital Length of Stay. Click here to view.
Rapid Evidence Product: Retention Strategies for Medications for Addiction Treatment in Adults With Opioid Use Disorder. Click here to view.
OPEN FOR COMMENT THROUGH 9/8/2020: Key Questions: Models of Care that Include Primary Care for Adult Survivors of Childhood Cancer. Click here to view.
OPEN FOR COMMENT THROUGH 9/28/2020: Systematic Review: Cervical Ripening in the Outpatient Setting. Click here to view.
OPEN FOR COMMENT THROUGH 9/14/2020: Systematic Review: Radiation Therapy for Brain Metastases. Click here to view.
OPEN FOR COMMENT THROUGH 9/18/2020: Systematic Review: Integrating Palliative Care in Ambulatory Care of Non-Cancer Serious Chronic Illness. Click here to view.
Systematic Review: Care Interventions for People Living With Dementia and Their Caregivers. Click here to view.
Research Report: A Prospective Comparison of Evidence Synthesis Search Strategies Developed With and Without Text-Mining Tools. Click here to view.