1. Webinar: Value Assessments – the Impact on People with Rare Diseases, see below for details.
2. Value Our Health Op-Ed: Stop Discriminatory Value Assessments, click here to read the op-ed.
3. Veterans Groups in California Comment on ICER’s 2020 Framework, click here to read the American Legion letter and click here to read the joint letter.
4. Alliance for Aging Research: Video on ICER’s Methodology, click here to see the video.
5. PIPC Patient Blog: CancerCare Response to 'Mouse That Roared,' click here to read the blog.
6. NHC Rubric to Capture the Patient Voice, click here to learn more and see the rubric.
7. ICER Announces Possible 2020 Assessments, click here to see the list of possible assessments.
8. University of Maryland Welcomes Patients, Researchers to PATIENTS Day, click here to read more.
9. AAR Seeks Recruits for Annual Senior Patient and Family Caregivers Network Training, click here to learn more and to apply for the training.
10. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
11. ICER Studies: Type 2 Diabetes, Arthritis, Cardiovascular Disease, Duchenne Muscular Dystrophy click here to provide patient input.
12. Upcoming Events and Webinars, see details below.
13. Medical Journal Articles, see details below.
14. AHRQ Effective Program Updates, see details below.
The Partnership to Improve Patient Care (PIPC) and Everylife Foundation for Rare Diseases invites people with rare diseases, as well as their families and providers to join our webinar on July 17 from 1:30-3 PM EST to learn about value assessments, their potential to limit access and innovation, and related public policy threats at the federal and state level.
We are in a very promising time for medical research and development in which true disease altering gene therapies are right around the corner. These therapies have the potential to provide life-altering, and, in some cases, curative treatments for rare disease, which up until recently had no hope for treatment. As these treatments are being developed, organizations like ICER are rushing to determine their value or “cost-effectiveness.” Metrics for measuring the “cost effectiveness” of treatments often relies on discriminatory methods, like the quality-adjusted-life-year (QALY), which values the lives of people with disabilities and serious chronic conditions as worth less than those of non-disabled people. This concerning way of measuring value consistently devalues drugs for rare disease, which can lead to limitations on patient access and a disincentive for investment in rare disease research and development. While no patient is average, average value metrics are increasingly at the center of state and federal discussions about drug pricing, and advocates may be unfamiliar with how to engage with a complicated but important issue area.
Disability and patient advocates have long fought against the QALY, achieving a prohibition against its use in Medicare within the Affordable Care Act. Previous administrations have ruled that using QALYs to allocate healthcare resources may constitute a violation of the Americans with Disabilities Act. But over the last few years, proposals to use average metrics such as QALYs have become increasingly common, threatening access to lifesaving medications for people with disabilities and those with rare diseases.
This event will arm advocates with the information they need to advocate against policies that will limit innovation and access for rare disease patients. We hope you will join us in advocating for policymakers to Value Our Health! Please RSVP to [email protected].
2. Value Our Health Op-Ed: Stop Discriminatory Value Assessments
Elizabeth Franklin, Executive Director of Cancer Support Community's Cancer Policy Institute, wrote in an op-ed for Morning Consult that patients are concerned with the QALY's implications, calling the measure “ethically inappropriate” and “immoral.” "As a representative of the patient advocacy community, we believe that patients make informed decisions that are right for them. In a partnership with their loved ones and their providers, patients make careful choices regarding treatments that fit into their lives and personal value framework. Discriminatory measures such as the QALY should be banned in the United States. Organizations determining value must be completely transparent and incorporate patient, survivor and caregiver feedback into assessments. Individuals living with chronic diseases and disabilities have a right to know how decisions that could have irreparable impact on their lives are being made and must have their voices heard," she wrote. Click here to read the op-ed.
3. Veterans Groups in California Comment on ICER’s 2020 Framework
Veterans groups in California submitted comment letters on ICER's 2020 assessment framework, highlighting their opposition to the use of discriminatory measures such as the QALY. California's American Legion wrote "We have significant concerns that...protections against the use of a cost-per-QALY value assessment do not exist for our members. It is profoundly unfair and offensive to those who have served this country to allow for this kind of discrimination in the veterans health system." ICER's frameworks are particularly important to veterans as the Veterans Affairs Administration agreed to work with ICER to integrate the group's reports into its formulary. A group of veterans organizations composed of AMVETS, the American GI Forum, the California Association of County Veterans Service Officers, NGAC, and the Women Veterans Alliance penned a similar letter to ICER. Click here to read the American Legion letter and click here to read the joint letter.
4. Alliance for Aging Research: Video on ICER’s Methodology
The Alliance for Aging Research recently released a video explaining the discriminatory implications of the methodology for assessment treatment value used by the Institute for Clinical Economic Review (ICER) for seniors and people with disabilities. Click here to see the video.
5. PIPC Patient Blog: CancerCare Response to 'Mouse That Roared'
In a PIPC blog, patient advocate Carole Florman responds to a Boston Globe article that characterizes ICER as a "mouse that roared," noting that ICER's methodologies are fatally flawed and discriminate against people with disabilities. "ICER continues to rely on a one-size-fits all approach that does not account for differences among patients. Not every drug or treatment works for every patient, but what ICER may judge to be a “low value” treatment may be the only one that can save a particular patient’s life. My guess is that to that patient and their family, such treatment is seen as very high value indeed. Those of us advocating for patients simply want the patient’s voice included and there are models for doing so," she wrote. Click here to read the blog.
6. NHC Rubric to Capture the Patient Voice
There is finally broad consensus in the US and globally that patients should be engaged in all aspects of health, from research, to developing a new treatment, to care delivery, and in policy. However, there remains confusion about and misuse of what it means to be patient-centered and how to achieve meaningful patient engagement. To this end, the National Health Council (NHC) has created the NHC Rubric to Capture the Patient Voice: A Guide to Incorporating the Patient Voice into the Health Ecosystem. Potential uses might include, but are not limited to, patient engagement in health care policy, quality-measure development, shared decision-making, value assessment, product development, etc. Click here to learn more and see the rubric.
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. University of Maryland Welcomes Patients, Researchers to PATIENTS Day
In late May, the University of Maryland Baltimore held its third PATIENTS day, bringing together patients, researchers, and other members of the community invested in patient-centered care. The Patient-Centered Involvement in Evaluating the Effectiveness of Treatments (PATIENTS) Program partners with patients and health care providers to answer questions about the best treatment options to improve health and quality of life. "The patients who have served as advisors on studies supported by The PATIENTS Program have helped researchers reframe their research questions from very technical language to language that resonates with real people and real situations, improving the quality of those studies,” said Dr. Natalie Eddington, Dean of the University of Maryland Baltimore's School of Pharmacy. Click here to read more.
9. 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.
10. 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: Government restrictions result in patients taking outdated drugs. It can take five years for Australians to access lifesaving medicine.
- New Zealand: NZ Myeloma patients lack access to needed medicine. Breast cancer advocates say that human life is not prioritized by PHARMAC. Click here to read more.
- Canada: Lifesaving SMA drugs continue to not be covered. Patient sues Canadian government over not covering a needed drug, leaving him needing dialysis permanently.
- United Kingdom: NHS continues to deny coverage of Batten Disease drugs. SMA sufferers in the UK are still denied lifesaving treatments.
11. ICER Studies: Acute Migraine, Type 2 Diabetes, Arthritis, Cardiovascular Disease, 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: Research Protocol available. 8/5/2019: Model Analysis Plan. Meeting 11/19/2019: CTAF to an update to its 2017 rheumatoid arthritis assessment.
- Type 2 Diabetes: Research Protocol Available. 7/29/2019: Model Analysis Plan. 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 : Model Analysis Plan available. 7/24/2019: Draft Evidence Report. 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.
- Peanut Allergy: Final Evidence Report and Meeting Summary available.
- Duchenne Muscular Dystrophy: Evidence Report, Revised Voting Questions, and Response to Comments 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.
- Acute Migraine: Comments OPEN on Draft Scoping Document, Stakeholder List through 7/19/2019. Meeting 1/23/2020: Midwest CEPAC to review ICER's assessment of acute migraine treatments
- Valuing A Cure Project: White Paper available 8/6/2019. Comment period open 8/6/2019-9/3/2019.
12. Upcoming Events and Webinars
PCORI Board of Governors Meeting
July 23, 2019
Click here for details.
Webinar: VBID X: A New Plan Option for the Individual Health Insurance Market
July 24, 2019
Click here for details.
NVHR Hepatitis C Patient Summit
July 29-30, 2019
Click here for details.
Webinar: Rethinking Drug Reimbursement: Value Communications in a Post-Rebate World
July 31, 2019
Click here for details.
PCORI Board of Governors Meeting
August 20, 2019
Click here for details.
Advisory Panel on Rare Disease Fall 2019 Meeting
September 16, 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.
2019 AUCD Annual Meeting
November 17-20, 2019
Click here for details.
13. Medical Journal Articles
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.
Ethical Challenges Related to Patient Involvement in Health Technology Assessment, click here to view.
Value-Based Insurance Design: Current Evidence and Future Directions, click here to view.
Novel Approaches to Value Assessment Beyond the Cost-Effectiveness Framework, click here to view.
Navigating Joint HTA, Procurement, and Fair Pricing: Evidence-Based Insights and Practical Recommendations - A Meeting Report from ISPOR Regional Conference in Warsaw, 2019, click here to view.
Health Technology Assessment as Part of a Broader Process for Priority Setting and Resource Allocation, click here to view.
Improving Quality Measure Maintenance: Navigating the Complexities of Evolving Evidence, click here to view.
A Tool for Empirical Equipoise Assessment in Multigroup Comparative Effectiveness Research, click here to view.
As Health Technology Assessment Evolves So Must its Approach to Patient Involvement, click here to view.
14. AHRQ Effective Program Updates
Systematic Review: Achieving Health Equity in Preventative Services, 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.
Systematic Review: Treatment of Depression in Children, 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: Diagnosis and Treatment of Clinical Alzheimer's-type Dementia (CATD), click here to view.
Systematic Review: Comparative Effectiveness of Analgesics to Reduce Acute Pain in the Prehospital Setting, click here to view.
Systematic Review: Long-Term Drug Therapy and Drug Holidays for Osteoporosis Fracture Prevention, click here to view.
Systematic Review: Telehealth for Acute and Chronic Care Consultations, click here to view.