1. Please Join PIPC TODAY For Our 10th Annual Forum!, click here to view the invitation.
2. Patient Advocacy Groups Submit Letter Urging Massachusetts Health Policy Commission to Reject QALY-Based Value Assessments, click here to read the letter.
3. PIPC Submits Comment Letter to ICER on Draft Evidence Report for Acute Migraines, click here to read the letter
4. Expanding the Use of Multi-Criteria Decision Analysis, click here to read the article.
5. IVI Issue Brief on ICER's Analysis of JAK Inhibitors, see details below.
6. Needs of Older Patients Must Be Considered in Value Assessments, click here to read the interview.
7. One Patient's Path Towards Advocacy, click here to read the article.
8. Tennessee 1115 Waiver Application Would Reference “Cost Effectiveness," see details below.
9. PCORI Reauthorization Discussions Advance, see details below.
10. Input Due TODAY -- House Begins 21st Century Cures 2.0 Effort, see details below.
11. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
12. ICER's QALY-Based Study Topics: Beta Thalassemia, Acute Migraine, Arthritis, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, click here to provide patient input.
13. Upcoming Events and Webinars, see details below.
14. Medical Journal Articles, see details below.
15. AHRQ Effective Program Updates, see details below
The Partnership to Improve Patient Care (PIPC) will hold its 10th Annual Forum TODAY, 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. Patient Advocacy Groups Submit Letter Urging Massachusetts Health Policy Commission to Reject QALY-Based Value Assessments
Groups representing patients and people with disabilities submitted written testimony to the Commonwealth of Massachusetts Executive Office of Health and Human Services and Health Policy Commission (HPC) related to their consideration of the “value” of medications as a part of the oversight and administration of prescription drug pricing. The groups expressed concern about the proposed regulations identifying “value” by referencing prices paid by other countries; the seriousness and prevalence of the disease or condition that is treated by the drug; and analyses by third parties, which could include the Institute for Clinical and Economic Review (ICER). The groups warned that evaluating “value” in this manner would allow for QALY-based cost-effectiveness analyses that discriminate against people with disabilities and chronic conditions and urged Massachusetts not to codify a regulation that could lead to restricted access to care. Read the entire letter here.
3. PIPC Submits Comment Letter to ICER on Draft Evidence Report for Acute Migraines
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 for acute migraines. Chairman Coelho criticized the report for making an "oversimplified assumption" that there are no mortality effects in migraine treatment, citing that a number of people with more severe types of migraine have higher rates of all-cause mortality in both men and women. He also reiterated PIPC's concern with ICER's use of the QALY, saying that it is not an appropriate methodology for use in assessments where the patient population is heterogeneous. "ICER continues to overlook outcomes that matter to patients in favor of overly simplistic QALY-based models," wrote Chairman Coelho. "We urge ICER to be more thoughtful in its model construction and take seriously the feedback from patients and clinicians who are experts in migraine attacks." Click here to read the letter.
4. Expanding the Use of Multi-Criteria Decision Analysis
In the American Journal of Managed Care, Dr. Charles Phelps writes that cost-effectiveness analysis as it exists is incomplete and excludes multiple important dimensions. From the article: "Expanding the understanding and measurement of value beyond CEA requires a systems analysis perspective. Control of complex systems requires the ability to measure and combine information in new ways, a task for which MCDA is ideally suited. Getting “there” requires systematic accumulation of data that do not currently exist. Ultimately, MCDA use will expand with improved usability and familiarity. People resist new ideas even when old ones are insufficient." Click here to read the article.
5. IVI Issue Brief on ICER's Analysis of JAK Inhibitors
The Innovation and Value Initiative (IVI) released an issue brief on the shortcomings of ICER's recent study and why it is necessary to move beyond traditional, ICER-style, QALY-based value assessment and to more complex, patient-centered tools. "In the context of chronic illnesses like RA, decision-makers can often reliably predict that a patient will progress through a series of treatment lines as therapies fail or disease progresses. To support decisions that account for that broader context, value assessment must allow for the comparison of treatment strategies, rather than individual therapies. They must also account for differences in treatment effect and outcomes across patient populations," said the IVI's Mark Linthicum. Click here to read the press release. Click here to read the issue brief.
6. Needs of Older Patients Must Be Considered in Value Assessments
NPC's President spoke with Sue Peschin, the President and CEO of the Alliance for Aging Research, about the importance of including the perspectives and needs of older patients in any value assessment effort. "QALYs don’t adjust for quality of life issues based on age, disability or severity of illness. There is no allowance for heterogeneity in the patient population. It puzzles me how that’s helpful from a payer perspective. No insured population is homogenous, so these assessments are limited and not reflective of the populations served," she said. Click here to read the interview.
7. One Patient's Path Towards Advocacy
Hep Magazine published a profile of Donna Cryer, the founder and CEO of the Global Liver Institute (GLI), whose personal struggles with liver disease led her to fight on the behalf of patients like herself. From the article: "[D]espite the burdens she carries, she doesn’t regret that her personal health challenges have determined her career. 'It’s a blessing to have found such a clear purpose,' she says, 'and know I’m able to have a meaningful impact on so many people’s lives.'” Click here to read the article.
8. 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.
9. 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.
10. Input Due TODAY -- 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 TODAY, 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.
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.
- Canada: Canada's bureaucratic approval system impedes cystic fibrosis patients' access to life-changing drugs. Toddler denied a drug that would treat a virus that may kill him.
- New Zealand: Patient advocates demand a doubling of Pharmac's budget to fund access to lifesaving treatments. Inquiry launched after deaths of epilepsy patients whose medicines were switched by Pharmac. Pharmac tries to downplay the side effects of a drug linked to four deaths of epilepsy patients.
12. ICER's QALY-Based Study Topics: Beta Thalassemia, Acute Migraine, 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: Research Protocol available. 2/3/2020: Model Analysis Plan.
- Cystic Fibrosis: Model Analysis Plan available. 2/20/2020: Draft Evidence Report and Draft Voting Questions. Meeting 4/30/2020: CTAF will convene to deliberate and vote on evidence presented in ICER's report on treatments for cystic fibrosis.
- 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. 1/9/2020: Final Evidence Report and Meeting Summary.
- 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.
13. Upcoming Events and Webinars
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.
PCORI Advisory Panel on Patient Engagement Winter 2020 Meeting
January 31, 2020
Click here for details.
Patient Registries, Real World Evidence and HEOR
January 27-28, 2020
Click here for details.
PCORI Board of Governors Meeting
February 25, 2020
Click here for details.
FT US Pharma and Biotech Summit 2020
May 14, 2020
Click here for details.
14. 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.
15. 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.