1. PIPC Comments on ICER's Cardiovascular Disease Study, click here to read the letter.
2. Proposed Changes to ICER Framework Available for Comment, click here to view the changes and to comment and see below for more.
3. RetireSafe: IPI Would Hurt American Seniors, click here to view the article.
4. National Health Council Training on Value Assessment and Patient Voice Rubric, see below for more.
5. CARE About Fibroids White Paper and Upcoming Patient Tool, see below for more.
6. Maisie's Army Mobilizes to Help Toddlers Access Lifesaving SMA Therapy, click here to read the article.
7. ICER Announces Possible 2020 Assessments, click here to see the list of possible assessments.
8. AAR Seeks Recruits for Annual Senior Patient and Family Caregivers Network Training, click here to learn more and to apply for the training.
9. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
10. ICER Studies: Acute Migraine, Type 2 Diabetes, Arthritis, Cardiovascular Disease, Duchenne Muscular Dystrophy, Sickle Cell Disease, click here to provide patient input.
11. Upcoming Events and Webinars, see details below.
12. Medical Journal Articles, see details below.
13. 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 on treatments for Cardiovascular disease (CVD). Chairman Coelho argued that there are several key flaws with ICER's report, noting that their model uses inappropriate data for the population being targeted with CVD medications. He also criticized ICER for using an "artificially narrow" definition of a major adverse cardiovascular event. "ICER continues to use a flawed methodology, ignoring real-world data and quality of life outcomes that matter to patients in favor of data that easily crosswalks into the discriminatory QALY metric," wrote Chairman Coelho. "We urge ICER to reconsider both its data sources and its concerning theory that health care must be rationed to achieve savings and efficiency in our health care system." Click here to read the letter.
PIPC also signed onto a comment letter alongside members of the Value and Access Steering Committee addressing ICER's CVD study. Click here to read the letter, written by the National Forum for Heart Disease and Stroke Prevention.
2. 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 its 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.
3. RetireSafe: IPI Would Hurt American Seniors
Mark Gibbons, President of RetireSafe, an advocacy organization that works on behalf of older Americans, published an op-ed arguing that the Trump Administration's proposed international pricing index (IPI) for prescription drugs in Medicare Part B would lower seniors' standard of care. "Medicare must not be subjected to these unnecessary changes — changes that would silence the voices of mature Americans nationwide and stunt progress in value-based care that allows our health care system to stay innovative and progress forward. Beneficiaries and their health care teams know what works for them. Overly broad, one-size-fits-all drug pricing proposals that model themselves after those in countries with socialized health care systems are not the answer to the issue of rising drug costs. Beneficiaries must be able to continue accessing whichever medications they need, without middlemen interfering in this process and discounting their needs," he wrote. Click here to view the article.
4. National Health Council Training on Value Assessment and Patient Voice Rubric
On September 6, the National Health Council (NHC) will hold a training on value assessments for members of the patient community and health policymakers. The PCORI-funded training will address value assessment and its importance to the changing health care landscape. The patient community and how it can participate in value assessments will be a major area of focus. It is open to participants of all experience levels and will incorporate both a beginner and intermediate track. Click here for more information and to register.
NHC also recently released its Rubric to Capture the Patient Voice, a tool for the patient community and other stakeholders can use to evaluate attributes of patient-centeredness and to guide them on meaningful patient engagement. NHC is soliciting feedback from patients and other health care stakeholders. Click here to see the Rubric and to comment.
5. CARE About Fibroids White Paper and Upcoming Patient Tool
A new white paper from CARE About Fibroids details the expanding menu of treatment options for uterine fibroids, ranging from over-the-counter analgesics to major invasive surgical interventions. It also talks about the symptoms associated with fibroids and provides a look at what is "normal" and what is not. This comprehensive overview — written for a lay audience — provides readers with an in-depth look of the various symptoms associated with fibroids. As new therapies are developed, patients have greater choices as they work with their doctors to find appropriate treatments for them. Click here to read the white paper.
Responsum Health and the Duke Clinical Research Institute (DCRI), a leading academic research organization, announced an agreement to collaborate to improve the quality of life for patients with uterine fibroids. They are joined in this effort by The White Dress Project and CARE About Fibroids. The announcement follows Fibroids Awareness Month, recognized in July. Under the agreement, Responsum will develop and promote a unique, uterine fibroids patient-centered information portal, similar to its work in other therapeutic areas. This new platform will serve the dual goals of providing comprehensive, understandable, and trustworthy information while helping to connect a nationwide community of patients. Click here to read more.
6. Maisie's Army Mobilizes to Help Toddlers Access Lifesaving SMA Therapy
A grassroots group of over 700 volunteers called Maisie's Army has been raising awareness of coverage of a new gene therapy to treat SMA and to pressure payers to allow beneficiaries to access the drug. Following the group's victory for its namesake patient, it said it would not stop at one and would fight for other families as well. Maisie's Army has now helped a number of toddlers across the country access the lifesaving SMA treatment. Click here to read more.
7. 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.
8. 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.
9. 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: New Zealand ranks last in OECD for medicine access. Cancer patients are unsurprised by report on report detailing Pharmac's shortcomings. Lung cancer patient urges others not to ignore subtle symptoms. SMA drug's not being funded when patient was younger means that she is deteriorating at faster rate now.
- Canada: Glioblastoma patient pays out-of-pocket for clinical trial for treatment that was approved in U.S. Ontario criticizes Ottawa's new drug pricing plan.
- United Kingdom: Patients call Scotland's decision to not fund cystic fibrosis drug "not acceptable." Click here to read more about the decision.
10. ICER Studies: Acute Migraine, Type 2 Diabetes, Arthritis, Cardiovascular Disease, Duchenne Muscular Dystrophy, Sickle Cell Disease
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:
- Sickle Cell Disease: 8/30/2019: Draft Scoping Document. 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. 9/18/2019: Draft Evidence Report. Meeting 11/19/2019: CTAF to an update to its 2017 rheumatoid arthritis assessment.
- Type 2 Diabetes: Model Analysis Plan available. 9/11/2019: Draft Evidence Report. 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 : 9/12/2019: Response to comments, evidence report, and revised voting questions. 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: Revised Scoping Document available. 9/6/2019: Research Protocol. Meeting 1/23/2020: Midwest CEPAC to review ICER's assessment of acute migraine treatments
- Valuing A Cure Project: White Paper AVAILABLE. Comment period open 8/6/2019-9/6/2019. Meeting 9/17/2019: Invited stakeholders to discuss single or short-term transformative therapies.
11. Upcoming Events and Webinars
ICER’s Value Framework and Patient Feedback: Q&A with Steve Pearson
September 4, 2019
Click here for details.
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.
Advisory Panel on Rare Disease Fall 2019 Meeting
September 16, 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.
FT Pharma Pricing and Value Summit 2019
September 26, 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.
12. Medical Journal Articles
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.
Rethinking Bias and Truth in Evidence-Based Medicine, click here to view.
13. AHRQ Effective Program Updates
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.
Technical Brief: Pharmacological and Nonpharmacological Treatments for Post-Traumatic Stress Disorder, click here to view.
Systematic Review: Management of Infertility, click here to view.
Key Questions: Cervical Ripening in the Outpatient Setting, click here to view.
Key Questions: Radiation Therapy for Brain Metastases, click here to view.
Protocol: A Rapid Evidence Review of Retention Strategies for Medications for Addiction Treatment (MAT) in Adults with Opioid Use Disorder, click here to view.
Systematic Review: Long-Term Drug Therapy and Drug Holidays for Osteoporosis Fracture Prevention, click here to view.