1. Over 60 Leading Patient and Disability Groups Join PIPC in Letter to New ICER VP for Patient Engagement, click here to read the letter.
2. ICER’s Cost Model Is Not Only Wrong It’s Also Dangerous, click here to read the article.
3. Gunnar Esiason: A Flawed Economic Model Should Not Be Used to Deny Access to a Miracle Medication, click here to read the op-ed.
4. IVI Announces Patient Advisory Council, click here to read more.
5. In Letter to PCORI, PIPC Offers Input on Next 10 Years, click here to read the letter.
6. ICER Report Adds Insult to Injury for Cystic Fibrosis Patients, click here to read the blog.
7. APPLICATION DEADLINE FRIDAY: PCORI Engagement Awards to Address COVID-19, click here to learn more.
8. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
9. ICER's QALY-Based Study Topics: Hemophilia A, Acute Migraine, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, Bladder Cancer, Opioid Treatments, click here to provide patient input.
10. Upcoming Events and Webinars, see details below.
11. Medical Journal Articles, see details below.
12. AHRQ Effective Program Updates, see details below.
More than 60 leading patient advocacy and disability groups signed onto the Partnership to Improve Patient Care’s (PIPC) outreach letter to the Institute for Clinical and Economic Review’s (ICER) new Vice President for Patient Engagement Yvette Venable. The undersigned groups applauded the creation of this new position, and look forward to working with Ms. Venable to ensure that ICER develops and implements a robust patient engagement framework. “As organizations that have worked across the spectrum of health care – including life sciences innovation, payer decision-making and value assessment – with missions to improve the quality of life for patients and people with disabilities, we hope that you will engage with us and take advantage of our real-world patient engagement experiences and lessons learned,” the letter states. “Do not hesitate to reach out to us and to the experts in this field described above as you consider patient engagement frameworks that may be fit for use within ICER.” Click here to read the letter.
2. ICER’s Cost Model Is Not Only Wrong It’s Also Dangerous
Writing in Forbes, the Pacific Research Institute's Wayne Winegarden says that ICER's model for the antiviral Remdesivir, which may help in treating COVID-19, is "precisely wrong." "Should policymakers listen to ICER, they will be punishing the private companies and individuals who are stepping up to help solve this pandemic," he writes. "The basic logic of incentives tells us that fewer people will do what is right if you punish them for doing the right thing. Essentially, this is what ICER’s cost model does." Click here to read the article.
3. Gunnar Esiason: A Flawed Economic Model Should Not Be Used to Deny Access to a Miracle Medication
ICER's QALY-based models should not be used to deny lifesaving drugs to patients like him, writes Gunnar Esiason, who is living with cystic fibrosis. "The medication that has turned my life around has, however, come under the suspicious eye of a watchdog group called the Institute for Clinical and Economic Review," he writes. "ICER uses a discriminatory cost-effectiveness model called Quality-Adjusted Life Years to determine the value of different medications. QALY, which ICER calls the 'gold standard' to determine cost-effectiveness, is a utility analysis that evaluates the price of a particular drug against its efficacy in improving a person’s life, or according to the model, the utility value recovered by the drug. It is discriminatory because it does not value years of life equally for people who are terminally ill compared with people who are healthy." Click here to read the op-ed.
4. IVI Announces Patient Advisory Council
The Innovation and Value Initiative (IVI) announced the members of its Patient Advisory Council. The Council advises IVI's Board of Directors and was chartered to ensure that IVI is patient-focused and transparent. The members are Alan Balch of the Patient Advocate Foundation, Donna Cryer of the Global Liver Institute, Anna Hyde of the Arthritis Foundation, Annie Kennedy of the EveryLife Foundation for Rare Diseases, Eleanor Perfetto of the National Health Council, Jeanne Regnante of LUNGevity, and Leslie Ritter of The National Multiple Sclerosis Society. Click here to read more.
5. In Letter to PCORI, PIPC Offers Input on Next 10 Years
In a letter to the Patient-Centered Outcomes Research Institute (PCORI), Partnership to Improve Patient Care (PIPC) Chairman Tony Coelho offered input on the next 10 years of PCORI. Chairman Coelho framed PIPC’s recommendations around three core principles: relevance, timeliness, and trust. Specifically, he encouraged PCORI to: (1) create a national agenda for research priorities; (2) expand collection of and access to patient-centered outcomes; (3) advance use of patient-centered outcomes in value assessment; and (4) promote patient-centered methodologies. “This is a crucial moment to build on PCORI’s success for another 10 years,” wrote Chairman Coelho. “Success, we believe, will entail effectively connecting PCORI and its research strengths to the current, pressing needs of our health care system and the patients and caregivers it serves. Click here to read the letter.
6. ICER Report Adds Insult to Injury for Cystic Fibrosis Patients
In the latest PIPC Patient Blog, Siri Vaeth writes that ICER's evaluation of breakthrough cystic fibrosis (CF) drugs does not value the lives and wellbeing of people living with CF, such as her daughter. "ICER’s reports are based on a discriminatory metric, the Quality-Adjusted Life Year, which quite simply measures the lives of people with disabilities and chronic illnesses, like my daughter, as less valuable than those of “healthy” people," she writes. "ICER’s report found that Trikafta had clear clinical benefit. Indeed, they gave Trikafta an 'A,' their highest rating for comparative clinical effectiveness....Without question, this drug is a breakthrough. For many patients it can turn CF from a definitive death sentence into a chronic life-threatening disease. Despite this clear clinical benefit, ICER also found that the drug is 'not worth the cost,' even if it were a curative therapy. Put bluntly, ICER believes that my daughter is not worth treating." Click here to read the blog.
7. APPLICATION DEADLINE FRIDAY: PCORI Engagement Awards to Address COVID-19
PCORI has committed existing Eugene Washington PCORI Engagement Awards funding to a special opportunity: Supporting Engagement in and Dissemination of PCOR/CER in the Context of COVID-19.
This funding will support projects that help communities increase their ability to participate across all phases of PCOR/CER while responding to societal changes resulting from the COVID-19 pandemic. PCORI seeks to fund organizations and community groups to actively disseminate PCORI-funded research results during the pandemic. PCORI invites applicants to explore virtual or distanced methods of engagement, and to generate new questions and evidence needs across stakeholder groups.
The application deadline for awards is May 22, 2020, and projects could start July 1 or August 1. Click here to learn more.
8. 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 finally funds breast cancer drug, but it may come too late for one grandmother.
- Canada: Advocates call for government to work to fund cystic fibrosis drug. Members of Parliament call for government to fund cystic fibrosis drug.
- New Zealand: Government put cost concerns over patient's lives with decision on breast cancer drug. PHARMAC gets new money, but it is unlikely to be spent on things like a critical breast cancer drug. The same is true for lung cancer sufferers.
- United Kingdom: COVID restrictions drive NHS to provide at-home treatments. Click here to read more. Local footballer pays for critical treatment for a four-year-old with a rare cancer.
- Ireland: Woman says treatment that she had to fund herself has helped combat her disease.
9. ICER's QALY-Based Study Topics: Hemophilia A, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, Bladder Cancer, Opioid Treatments
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. All of ICER’s current assessments have been put on hold for 2-3 months in response to COVID-19. We will continue to update below deadlines as we receive more information.
- Ulcerative Colitis: Model Analysis Plan available. 5/26/2020: Draft Evidence Report and Draft Voting Questions. Meeting 9/24/2020: CTAF will convene to deliberate and vote on evidence presented in ICER's report on ulcerative colitis therapies.
- Cystic Fibrosis: Evidence Report and Response to Comments AVAILABLE. Meeting POSTPONED: CTAF will convene to deliberate and vote on evidence presented in ICER's report on treatments for cystic fibrosis.
- Sickle Cell Disease: Evidence Report and Responses to Comments AVAILABLE. Meeting POSTPONED: New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for sickle cell disease.
- Non-Alcoholic Steatohepatitis: MARCH 2020: DEADLINES PUSHED BACK 2-3 MONTHS. Draft Evidence Report and Voting Questions AVAILABLE, Comment Period OPEN through 6/24/2020. Meeting 8/13/2020: The Midwest CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for NASH.
- Hemophilia A: MARCH 2020: REVIEW PAUSED 2-3 MONTHS. Revised Scoping Document available. 6/15/2020: Research Protocol. Meeting 11/5/2020: New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for hemophilia.
- Bladder Cancer: MARCH 2020: REVIEW PAUSED 2-3 MONTHS. Draft Scoping Document available, comment period open through 6/1/2020.
- Opioids: Digital Apps: MARCH 2020: REVIEW PAUSED 2-3 MONTHS. Draft Scoping Document available, Comment Period open through 6/1/2020.
- Opioids: Supervised Injection Centers: 5/19/2020: Patient Input Period Begins
10. Upcoming Events and Webinars
PCORI Observational Analyses of Second-Line Pharmacological Agents in Type 2 Diabetes (Cycle 2 2020) Applicant Town Hall
May 20, 2020
Click here for details.
PCORI Advisory Panel on Patient Engagement Spring 2020 Meeting
June 4-5, 2020
Click here for details.
PCORI Board of Governors Meeting
June 23, 2020
Click here for details.
11. Medical Journal Articles
Comparative Effectiveness Research in Critically Ill Patients: Risks Associated with Mischaracterising Usual Care, click here to view.
Unanticipated Therapeutic Value of the Patient-Centered Outcomes Research Institute (PCORI) Stakeholder Engagement Project for Homebound Older Adults, click here to view.
Impact of a Global Pandemic on Health Technology Assessment, click here to view.
How Are Incremental Cost-Effectiveness, Contextual Considerations, and Other Benefits Viewed in Health Technology Assessment Recommendations in the United States?, click here to view.
The Implementation of Health Technology Assessment Principles in Public Decisions Concerning Orphan Drugs, click here to view.
Decision-Making under Uncertainty: Comparing Regulatory and Health Technology Assessment Reviews of Medicines in the US and Europe, click here to view.
What Types of Real-World Evidence Studies Do U.S. Commercial Health Plans Cite in Their Specialty Drug Coverage Decisions?, click here to view.
Defining the Role of the Public in Health Technology Assessment (HTA) and HTA-Informed Decision-Making Processes, click here to view.
When Guidelines Recommend Shared Decision-Making, click here to view.
Upholding Trust in Therapeutic Trials and Evidence-Based Medicine: Need for Full Disclosure of Data, Crowdsourcing Data Analysis and Independent Review?, click here to view.
12. AHRQ Effective Program Updates
White Paper: The Evidence Base for Telehealth: Reassurance in the Face of Rapid Expansion During the COVID-19 Pandemic. Click here to view.
White Paper: Standardized Library of Depression Outcome Measures. Click here to view.
OPEN FOR COMMENT THROUGH JUNE 12: Technical Brief: Prevention, Diagnosis, and Management of Opioids, Opioid Misuse and Opioid Use Disorder in Older Adults. Click here to view.
Research Protocol: No-Touch Modalities for Disinfection of Hospital/Acute Care Settings: A Rapid Evidence Review. Click here to view.
Research Protocol: Masks for Prevention of COVID-19 in Healthcare and Community Settings. Click here to view.
Systematic Review: Labor Dystocia. Click here to view.
Systematic Review: Diagnosis and Treatment of Clinical Alzheimer’s-Type Dementia. Click here to view.
Technical Brief: Characteristics of Existing Asthma Self-Management Education Packages. Click here to view.
Systematic Review: Opioid Treatments for Chronic Pain. Click here to view.
Systematic Review: Nonopioid Pharmacologic Treatments for Chronic Pain. Click here to view.