1. Please Join PIPC For Our 10th Annual Forum!, click here to view the invitation.
2. Tennessee 1115 Waiver Application Would Reference “Cost Effectiveness," see details below.
3. National Council on Disability Report on QALYs, click here to read the report.
4. PCORI Reauthorization Discussions Advance, see details below.
5. Medical Groups Push to Save PCORI, click here to read the article.
6. IVI: Future Value Assessments Must Include Patient-Defined Factors of Value, click here to read the article.
7. House Begins 21st Century Cures 2.0 Effort, see details below.
8. PIPC Urges Policymakers to Protect Patients and People with Disabilities, see below for details.
9. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
10. ICER's QALY-Based Study Topics: Beta Thalassemia, Acute Migraine, Type 2 Diabetes, Arthritis, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, 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.
The Partnership to Improve Patient Care (PIPC) will hold its 10th Annual Forum on December 16, 2019 from 12:00 - 2:00 p.m. at the Reserve Officers Association in Washington, DC. We will be joined by Ana Torres-Davis who will share an overview of the National Council on disability’s important and timely recent report: “Quality Adjusted Life Years and the Devaluation of Life with a Disability.” We will also learn about international healthcare systems from real patients, caregivers, and technical policy experts. RSVP to email@example.com. Click here to view the invitation.
2. Tennessee 1115 Waiver Application Would Reference “Cost Effectiveness”
Instead of requiring TennCare to cover any drug that is in the federal Medicaid drug rebate program, the state is asking CMS to allow it to adopt a commercial-style closed formulary with at least one drug available per therapeutic class. It also wants the ability to exclude new drugs coming to market through the FDA’s accelerated approval pathway until market prices are consistent or sufficient data exist regarding the "cost-effectiveness" of the drug. On page 15 of its waiver application, TennCare states, "The state proposes that it have the flexibility under this demonstration to adopt a commercial-style closed formulary with at least one drug available per therapeutic class...The state proposes that it have flexibility to exclude these new drugs from its formulary until market prices are consistent with prudent fiscal administration or the state determines that sufficient data exist regarding the cost effectiveness of the drug...In selecting drugs available in each therapeutic class, the state will ensure that the selected drugs meet the clinical needs of the vast majority of members and that they are cost effective.” The waiver application does not preclude the state from determining "cost effectiveness" of drugs using existing value assessment practices relying on discriminatory quality-adjusted life year (QALY) metrics. As stated by PIPC Steering member, Sue Peschin, with the Alliance for Aging Research in the Tennessean, discrimination has no place in American prescription drug coverage. Comments are due to CMS by December 27, 2019. Click here to view the link to comment. Click here to view the application. Click here to view Sue Peschin’s opinion in the Tennessean. Click here to learn more about the discriminatory implications of QALYs.
3. National Council on Disability Report on QALYs
The National Council on Disability issued a new report assessing the discriminatory effects of using QALYs to determine treatment coverage. "In an effort to lower their healthcare costs, public and private health insurance providers have utilized the Quality Adjusted Life Year (QALY) to determine the cost-effectiveness of medications and treatment," the Council wrote. "QALYs place a lower value on treatments which extend the lives of people with chronic illnesses and disabilities. In this report, NCD found sufficient evidence of the discriminatory effects of QALYs to warrant concern, including concerns raised by bioethicists, patient rights groups, and disability rights advocates about the limited access to lifesaving medications for chronic illnesses in countries where QALYs are frequently used. In addition, QALY-based programs have been found to violate the Americans with Disabilities Act." In addition, the report calls on Congress to ban the use of QALYs in Medicare and Medicaid and recommended that the administration refrain from modeling US pricing after the pricing in other countries, calling on the administration specifically to rescind its proposed rule to use an international pricing index in Medicare Part B. Click here to read the report.
4. PCORI Reauthorization Discussions Advance
Over the summer, the House Energy and Commerce and the primary jurisdiction House Ways and Means Committees marked up legislation to reauthorize the Patient-Centered Outcomes Research Institute (PCORI), for 3 years and for 7 years with limited changes to its underlying priorities, respectively. While PCORI reauthorization was included in legislation to extend various health programs until November 21, 2019, the PCOR trust fund was extended without extending the funding for it. Most recently, the Senate Finance Committee Chair and Ranking Member introduced a bill reauthorizing PCORI, including strongly supported provisions that would reauthorize PCORI with mostly full funding (eliminating the Medicare Trust Fund Transfer and increasing mandatory appropriations) for 10 years, as well as add a new positive provision that would add intellectual and development disabilities to PCORI’s national priorities. PIPC had provided comments on an earlier Senate discussion draft opposing added payer seats on the PCORI Board Governors. We also shared concerns about requiring collection of additional economics “outcomes data,” that may be better expressed report language, not statute, so as to ensure its compliance with the patient protections in Section 1182. We look forward to continued conversations with Senate leaders about those provisions. Friends of PCORI Reauthorization, a coalition of 184 organizations, has consistently communicated to both House and Senate leaders support for PCORI to remain patient-centered, and specifically support for a 10 year, fully funded reauthorization that continues to direct PCORI to fund comparative clinical effectiveness research. Click here to view PIPC’s comments or click here for information about Friends of PCORI Reauthorization. Click here to view the new Senate bill.
5. Medical Groups Push to Save PCORI
As PCORI's authorization nears its end date, a number of health care professionals are rushing to keep the agency and its patient-centered research afloat. "Supporters say it’s obvious that PCORI should continue. With annual incoming funds averaging about $480 million since 2014, the operation has paid for more than 700 projects, more than half completed by now, that are already influencing health care. 'We’re starting to see results that really matter,' says Christine Goertz, a health services researcher at Duke University in Durham, North Carolina, who chairs PCORI’s governing board," Science Magazine wrote. Click here to read the article.
6. IVI: Future Value Assessments Must Include Patient-Defined Factors of Value
Writing in Morning Consult, the Innovation and Value Initiative's (IVI) Jennifer Bright says that as researchers think of how to assess care, patient preferences and values must be incorporated. "Patient perspectives on value matter. When included in assessment, the value of a therapy is broadened beyond just unit cost of an intervention, to include considerations such as impact on caregivers and family and ability to work. Incorporating these value factors into modeling is time consuming and complex. But complexity should not be a barrier or a rationale for omission. Instead, the research community must continue to scientifically test and apply methods to demonstrate the additive importance of incorporating patient perspectives," she said. Click here to read the article.
7. House Begins 21st Century Cures 2.0 Effort
The lead authors of 21st Century Cures are soliciting feedback on a second iteration of the legislation, which they say will focus on issues such as digital health, coverage of innovative treatments, the use of real-world evidence (RWE), and improving education for caregivers. The lawmakers vision of so-called Cures 2.0 legislation is laid out in a new request for information (RFI) from Reps. Diana DeGette (D-CO) and Fred Upton (R-MI), who are soliciting feedback by December 16 on ideas that should be considered as part of a redux of their landmark bipartisan initiative. While the Department of Health and Human Services (HHS) is still fulfilling provisions of the original law, enacted in 2016, Rep. Upton recently indicated that at least part of the focus of Cures 2.0 will be on “how to make the law work better.”
The bipartisan pair of Reps. DeGette and Upton launched a public campaign for the 21st Century Cures Act five years ago with a similar request for ideas and support and spent two years crafting the bill, which was enacted in December 2016. The legislation, signed by President Barack Obama in the final weeks of his presidency, was aimed at speeding research and therapies for patients. It bolstered aid to the NIH, gave FDA new powers and responsibilities, and required more information sharing. Click here to view the call to action issued to kick off the Cures 2.0 effort.
8. PIPC Urges Policymakers to Protect Patients and People with Disabilities
PIPC Chairman Tony Coelho expressed concern about policies placing hard-fought existing patient protections under serious threat, especially those relying on policy decisions made in other countries that we know establish prices based on discriminatory cost-effectiveness standards (such as Quality-Adjusted Life Years or QALYs) and similar average metrics. PIPC supports policies to improve care and affordability for patients, adopt and extend safeguards in current statute that prohibit application of discriminatory cost-effectiveness thresholds in Medicare (whether done overtly by Medicare or covertly via “comparative effectiveness analysis” or reference to foreign countries’ use of these thresholds) and codify criteria for patient-centeredness called for in CMMI’s statute. Click here to view prior stakeholder letter on referencing International pricing. Click here to view information on Value Our Health.
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.
- Canada: Canadian mother of children with cystic fibrosis: "instead of getting up and going to school in the morning, we're heading to the hospital." Ontario family pleads for equal access to cystic fibrosis treatments. Canadian family has had to resort to crowdfunding to get a lifesaving spinal muscular atrophy drug as the government refuses to fund it.
- New Zealand: New Zealand man cannot access a cancer drug that has only been approved by Pharmac for one type of cancer although it is effective for others.
- United Kingdom: Even after approval, UK families face waits to access Orkambi. NHS will not cover "life-changing" migraine drug. Scottish woman says NHS stopped her son's doctor from providing lifesaving medicine. NHS will ration 34 everyday tests and treatments.
10. ICER's QALY-Based Study Topics: Beta Thalassemia, Acute Migraine, Type 2 Diabetes, Arthritis, 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: Revised Scoping Document available. 12/11/2019: Research Protocol.
- Cystic Fibrosis: Research Protocol available. 12/16/2019: Model Analysis Plan.
- Sickle Cell Disease: Model Analysis Plan available. 1/22/2020: Draft Evidence Report and Draft Voting Questions. 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: Evidence Presentation AVAILABLE. Meeting 12/9/2019: CTAF to an update to its 2017 rheumatoid arthritis assessment. 1/9/2020: Final Evidence Report and Meeting Summary.
- Type 2 Diabetes: Final Evidence Report and Meeting Summary AVAILABLE.
- Acute Migraine: Draft Evidence Report and Draft Voting Questions available. 1/8/2020: Revised Voting Questions, Evidence Report, and Responses to Public Comments. Meeting 1/23/2020: Midwest CEPAC to review ICER's assessment of acute migraine treatments.
- 2020 Value Assessment Framework: 1/31/2020: Final Framework Adaptations.
- Non-Alcoholic Steatohepatitis: Revised Scoping Document Available. 1/15/2020: Research Protocol.
- Beta Thalassemia: Open Input Period through 12/20/2019. 1/6/2020: Draft Scoping Document.
11. Upcoming Events and Webinars
PCORI Board of Governors Meeting
December 9, 2019
Click here for details.
Webinar: Providing Quality Benefits for Employees with Cardiovascular Disease and Other Chronic Conditions
December 10, 2019
Click here for details.
Webinar: Request for Proposals – Addressing Barriers to Medication Access
December 12, 2019
Click here for details.
PCORI Stakeholder Workshop on Suicide Prevention
December 18, 2019
Click here for details.
EveryLife Foundation’s A Rare Affair Fundraiser at the JP Morgan Healthcare Conference
January 12, 2020
Click here for details.
Patient Registries, Real World Evidence and HEOR
January 27-28, 2020
Click here for details.
FT US Pharma and Biotech Summit 2020
May 14, 2020
Click here for details.
12. Medical Journal Articles
Beyond Involvement: Multiple Methods and Purposes of Shared Decision Making, click here to view.
Behavioral Economic Insights to Improve Medication Adherence in Adults with Chronic Conditions: A Scoping Review, click here to view.
The Need for Clinical Judgement in the Application of Evidence-Based Medicine, click here to view.
Building Capacity in Evidence-Based Medicine in Low-Income and Middle-Income Countries: Problems and Potential Solutions, click here to view.
A Review and Synthesis of Frameworks for Engagement in Health Research to Identify Concepts of Knowledge User Engagement, click here to view.
Characteristics of Public Comments Submitted to State Health Technology Assessment Programs in Oregon and Washington, click here to view.
What Is Next for Patient Preferences in Health Technology Assessment? A Systematic Review of the Challenges, click here to view.
Commentary: One Size Does Not Always Fit All in Value Assessment, click here to view.
Engaging the Patient: Patient-Centered Research, click here to view.
Little Consistency In Evidence Cited By Commercial Plans For Specialty Drug Coverage, click here to view.
13. AHRQ Effective Program Updates
OPEN FOR COMMENT THROUGH 12/24/2019: Technical Brief: Characteristics of Existing Asthma Self-Management Education Packages, click here to view.
Research Report: Registries for Evaluating Patient Outcomes: A User's Guide, click here to view.
Research Protocol: Management of High-Need, High-Cost Patients: A Realist and Systematic Review, click here to view.
Systematic Review: Achieving Health Equity in Preventive Services, click here to view.
Research Protocol: Strategies for Patient, Family, and Caregiver Engagement, click here to view.
Research Report: Translation of a C. difficile Treatment Clinical Pathway Into Machine-Readable Clinical Decision Support Artifacts Prototyped for Electronic Health Record Integration, click here to view.
OPEN FOR COMMENT THROUGH 12/20/2019: Key Questions: Prehospital Airway Management: A Systematic Review, click here to view.
Research Protocol: Management of Primary Headache During Pregnancy, click here to view.
Research Report: Assessing the Accuracy of Machine-Assisted Abstract Screening With DistillerAI: A User Study, click here to view.
Research Report: Performance and Usability of Machine Learning for Screening in Systematic Reviews: A Comparative Evaluation of Three Tools, click here to view.