1. Webinar: Value Assessments – the Impact on People with Rare Diseases, see details below.
2. Value Our Health Capitol Hill Briefing Recap, click here to read more.
3. PIPC Submits Comment Letter to ICER Outlining Concerns with Draft Evidence Report on DMD, click here to read the letter
4. PIPC, Patient and Disability Groups Highlight Concerns with ICER’s Value Assessment Framework, click here to read the letter.
5. The Perils of QALYs Event Recap, click here to read more.
6. AcademyHealth: Supporting Patient-Centered Research, click here to read the blog.
7. Research!America Event: Improvements to Rural Health Care through Patient-Centered Research, see details below.
8. IVI Submits Comment Letter to ICER on 2020 Value Assessment Framework, click here to read the letter.
9. Consortium for Citizens with Disabilities Opposes Use of QALYs and Discriminatory Benefit Design, click here to view the full recommendations.
10. PIPC Chairman Coelho and Doctors Caucus Co-Founder Dr. Phil Gingrey on PCORI, click here to read the article.
11. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
12. ICER Studies: Type 2 Diabetes, Arthritis, Cardiovascular Disease, Duchenne Muscular Dystrophy click here to provide patient input.
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.
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@example.com.
2. Value Our Health Capitol Hill Briefing Recap
On June 20, 2019, the Partnership to Improve Patient Care hosted two Capitol Hill briefings to introduce Value Our Health, a group of patient and disability organizations that have come together to champion patient-centric value assessments. The panelists shared their concern about current value assessments, like those conducted by the Institute for Clinical and Economic Review (ICER), because the metrics they use, like the quality-adjusted life year (QALY) and equal value life-year gained (evLYG), treat patients as averages. As policymakers entertain policies to lower healthcare costs, Value Our Health hopes they will not allow patients to become collateral damage of a one-size-fits-all health system by misusing discriminatory forms of value assessment. Click here to read more.
3. PIPC Submits Comment Letter to ICER Outlining Concerns with Draft Evidence Report on DMD
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 Duchenne Muscular Dystrophy (DMD). Chairman Coelho offered criticism of ICER’s model for oversimplifying the disease, noting that ICER continues to overlook outcomes that matter to patients and caregivers. He also pointed out that ICER continues to use the flawed and discriminatory quality-adjusted-life-year (QALY) metric in its review. “ICER continues to overlook outcomes that matter to patients, families and caregivers in their haste to provide reports to payers,” wrote Chairman Coelho. “We encourage ICER to be more strategic and focus on producing complete and thoughtful analysis using high quality data incorporating a range of outcomes important to patients instead of rushing to complete reports that do not have appropriate scientific rigor." Click here to read the letter.
4. PIPC, Patient and Disability Groups Highlight Concerns with ICER’s Value Assessment Framework
In a letter to the Institute for Clinical Economic Review (ICER), over 30 patient and disability groups joined the Partnership to Improve Patient Care (PIPC) in outlining concerns about ICER’s 2020 Value Assessment Framework. The letter calls on ICER to abandon its use of the quality-adjusted-life-years (QALY) metric, as well as other metrics that discriminate against patients and people with disabilities. PIPC also emphasized that ICER must develop novel measures of value to account for patient differences and priorities, as well as models that are open-source, transparent, and available to all patients and researchers. “Above all, we urge ICER to put patients and people with disabilities at the center of all of your assessments,” the letter states. “While we share your interest in lowering healthcare spending and addressing affordability, we do not believe that generating value assessments in a manner that leads to restricted access and discrimination is a necessary tactic or ethical strategy for achieving these goals.” Click here to read the letter.
5. The Perils of QALYs Event Recap
People with disabilities are caught in the middle of the fight over health care costs in America, says PIPC Chairman Tony Coelho, simply because they are the most vulnerable. Speaking at the Coelho Center at Loyola Law School Los Angeles, he told a crowd of disability advocates that unless they stand and fight, interests that scapegoat the wellbeing of people with disabilities will win. At the center of this fight is a measure — the quality-adjusted life year, or QALY. Chairman Coelho was joined by Ari Ne’eman, Collin Killick, and PIPC’s Executive Director, Sara van Geertruyden, to say that the use of QALYs distorts and misrepresents how patients value their own lives, and it can lead to insurers and the government to deny care to people who would benefit from it. Click here to read more.
6. AcademyHealth: Supporting Patient-Centered Research
In a post for the AcademyHealth blog, Terri Finkel, M.D., Ph.D. touched on comments that AcademyHealth submitted supporting reauthorization of the Patient-Centered Outcomes Research Institute (PCORI). "The power of PCORI-supported networks like PEDSnet is enabling researchers to share data and ask questions about chronic disease that can be answered only by studying millions of people. Imagine finding a way to prevent a million cases a year of new-onset asthma; in fact, earlier this year, Nemours and our PEDSnet partners published a study leveraging information from close to 20 million medical encounters with over half a million children to demonstrate that obesity is the only preventable risk factor for asthma. In addition, in children, many health conditions – muscular dystrophy, for example – are uncommon or rare, and 'Big Data' are needed to answer important questions about care and cure of a rare disease. A shared data resource like the 6.2 million patient records available through PEDSnet enables us to conduct studies that would not be feasible for any one institution alone." Click here to read the blog.
7. Research!America Event: Improvements to Rural Health Care through Patient-Centered Research
Join Research!America and partners for a Capitol Hill lunch briefing on Tuesday, June 26, 2019 to explore advances in patient-centered research to improve delivery of care in rural areas. Panelists will share what makes health care delivery unique in rural areas and how patient-centered/community-based programs can lower the risk of heart disease and address other public health challenges. Click here for more information and click here to RSVP.
8. IVI Submits Comment Letter to ICER on 2020 Value Assessment Framework
The Innovation and Value Initiative recently submitted a comment letter to ICER offering recommendations for its value assessment framework for 2020. "Patient preferences for treatment attributes such as mode and frequency of administration should also be explicitly addressed. In addition, capturing heterogeneity in preferences may be of interest. To support this expansion to include non-clinical factors, ICER should seek partners in the patient and research communities. For example, a small-scale study with patients could be used to generate preliminary data on the impacts of changes in clinical measures or side effects on caregivers, which could then be linked to individual therapies’ relative effects to compare caregiver impact across therapies. Such a study could be conducted in partnership with an existing patient group or research institution." Click here to read the letter.
9. Consortium for Citizens with Disabilities Opposes Use of QALYs and Discriminatory Benefit Design
The Consortium for Citizens with Disabilities (CCD) is the largest coalition of national organizations working together to advocate for federal public policy that ensures the self-determination, independence, empowerment, integration and inclusion of children and adults with disabilities in all aspects of society. Prescription drugs are an essential aspect of daily life for many people with disabilities and chronic conditions. Among its recommendations to ensure access to treatment that is affordable, accessible, easy to navigate, and based on a physician-directed and person-centered treatment plan determined by the individual and their health care providers, CCD has stated a clear opposition to the use of Quality Adjusted Life Years (QALYs), a discriminatory measure based on the idea that disabled lives are less valuable and less worth living than non-disabled lives, and related measures. CCD supports an explicit ban on the use of QALYs in new health care legislation, opposes discriminatory benefit designs and calls on policymakers not to use access to care as leverage in negotiations. Click here to view the full recommendations.
10. PIPC Chairman Coelho and Doctors Caucus Co-Founder Dr. Phil Gingrey on PCORI
PIPC Chairman Tony Coelho and Doctors Caucus co-founder Dr. Phil Gingrey both served as health care advocates in Congress, and now they have written an op-ed together in Modern Healthcare about the successes of PCORI and the importance of continuing its work. "If we're to continue to make progress on improving patient care and outcomes, clinicians and patients must have access to more reliable, useful information to help determine which healthcare options are best for them. And in order to ensure that evidence is relevant and accessible to them, patients need a seat at the table in developing it....PCORI has lived up to its name and its mandate to drive patient-centeredness. We both feel strongly that the funding that supports its work should be reauthorized before it expires at the end of fiscal 2019, so the valuable investment in its efforts to date can produce even more useful evidence for healthcare decisionmakers well into the future," the duo wrote. Click here to read the article.
11. 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: Australians' lives have a price -- about $50,000 per QALY. PBS is making a woman pay $4,500 per month out of pocket for a breast cancer drug because she does not meet PBS's criteria.
- New Zealand: Breast cancer advocates say that human life is not prioritized by PHARMAC. Click here to read more. Advocates also call for greater funding for breast cancer drugs by PHARMAC. Lung cancer patients say similar things. Patients are fighting to have PHARMAC cover spinal muscular atrophy drug Spinraza, including through legal action.
- Canada: Patients call on Ontario to cover "miracle drugs." An Ontario woman living with CF advocates for greater access to lifesaving drugs.
- United Kingdom: Patients continue to fight for access to cystic fibrosis drugs. Click here and here to read more. NHS has doubled its cataract treatment rationing, ignoring evidence that surgery is effective. Click here to read more.
12. ICER Studies: Acute Migraine, 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: Research Protocol available. 7/10/2019: Model Analysis Plan. Meeting 10/31/2019: CTAF to an update to its 2017 rheumatoid arthritis assessment.
- Type 2 Diabetes: 7/8/2019: Research Protocol. 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.
- Depression: Final Evidence Report and Meeting Summary available.
- Multiple Sclerosis: Final Evidence Report and Meeting Summary available.
- Peanut Allergy: Evidence Presentation and Report available. 7/3/2019: Final Evidence Report.
- Duchenne Muscular Dystrophy: 7/11/2019: Evidence Report, Revised Voting Questions, and Response to Comments. 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: Open Input Period through 6/25/2019. 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.
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
Improvements to Rural Health Care Through Patient-Centered Research
June 26, 2019
Click here for details.
PCORI Advisory Panel on Patient Engagement Summer 2019 Meeting
June 27-28, 2019
Click here for details.
Leveraging Randomized Clinical Trials to Generate Real-World Evidence for Regulatory Purposes
July 11-12, 2019
Click here for details.
PCORI Board of Governors Meeting
July 23, 2019
Click here for details.
NVHR Hepatitis C Patient Summit
July 29-30, 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.
15. Medical Journal Articles
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.
Evaluation of Value-Based Insurance Design for Primary Care, click here to view.
Leveraging Patient/Community Partnerships to Disseminate Patient Centered Outcomes Research in Geriatrics, click here to view.
Enabling Individualised Health in Learning Healthcare Systems, click here to view.
Analysis of Sponsor Hearings on Health Technology Assessment Decision Making, click here to view.
16. 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.