1. Gunnar Esiason: A Flawed Economic Model Should Not Be Used to Deny Access to a Miracle Medication, click here to read the op-ed.
2. IVI Announces Patient Advisory Council, click here to read more.
3. In Letter to PCORI, PIPC Offers Input on Next 10 Years, click here to read the letter.
4. ICER Report Adds Insult to Injury for Cystic Fibrosis Patients, click here to read the blog.
5. Ruderman Foundation White Paper: Fair Resource Allocation During the COVID-19 Pandemic, click here to read the paper.
6. LA Times Editorial: Take Disability, QALYs Out of Equation for COVID-19 Care, click here to read the editorial.
7. PCORI Engagement Awards to Address COVID-19, click here to learn more.
8. PIPC, Other Disability and Patient Organizations Call for Further Guidance on COVID-19 Patient Non-Discrimination, click here to read the letter.
9. DUE WEDNESDAY: Request for Nominations for the Board of Governors of the Patient-Centered Outcomes Research Institute, click here to read the notice and to submit a nomination.
10. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
11. 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.
12. Upcoming Events and Webinars, see details below.
13. Medical Journal Articles, see details below.
14. AHRQ Effective Program Updates, see details below.
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.
2. 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.
3. 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.
4. 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.
5. Ruderman Foundation White Paper: Fair Resource Allocation During the COVID-19 Pandemic
The Ruderman Foundation published a white paper exploring how medical resources are allocated in a time of shortage, like the current COVID-19 pandemic. The paper finds that individuals with disabilities are particularly vulnerable to discrimination in health care rationing due to extreme resource scarcity. It also notes that ethically acceptable triage models must include safeguards for individuals with disabilities and that prioritizing the needs of patients with COVID-19 over others who are critically ill due to other illnesses or trauma is arbitrary and unjust. Furthermore, the Foundation calls for the designation “allied healthcare providers” to include experienced family members and paraprofessional caregivers of persons with disability. Click here to read the paper.
6. LA Times Editorial: Take Disability, QALYs Out of Equation for COVID-19 Care
The LA Times editorial board called for taking disability, age, and other factors out of decisions on who gets COVID-19 care in the situation where there is not enough to go around. "California is one of many states prepared to assign patients their place in line based on 'life years' — an actuarial determination of how much longer the patient is likely to survive after recovery, whether it be from COVID-19, a grievous injury (from an earthquake, perhaps) or another life-threatening condition," the editorial board wrote. "Other states use quality adjusted life years — 'QALY' — that modify the supposed value of the predicted life span by adding possible debilitating conditions to the equation. Some quality-of-life determinations consider whether the coronavirus patient has a preexisting illness or 'comorbidity' factor such as obesity, or a physical or intellectual disability. State guidelines in Washington recommend transferring patients with 'loss of reserves of energy, physical ability, cognition and general health' to outpatient or palliative care. Going a step further, some economists advocate prioritizing those who, on recovery, will contribute the most economic or intellectual value to the nation. Happily, no state has adopted such a policy — at least not explicitly." Click here to read the editorial.
7. 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. PIPC, Other Disability and Patient Organizations Call for Further Guidance on COVID-19 Patient Non-Discrimination
Over 400 aging, disability, and patient organizations sent a letter urging the Department of Health and Human Services (HHS) to issue further guidance addressing the application of federal non-discrimination laws to plans to ration scarce health care resources during the COVID-19 pandemic.
While HHS has previously issued a bulletin on this topic, advocates say additional guidance is necessary. In a letter to Health and Human Services Secretary Alex Azar and Office for Civil Rights (OCR) Director Roger Severino, the coalition of organizations, led by the American Association of People with Disabilities, the Bazelon Center for Mental Health Law, the Center for Public Representation, and the Disability Rights, Education and Defense Fund called upon the HHS Office of Civil Rights to provide guidance clarifying, among other things, that federal law prohibits states and health care providers from making treatment allocation decisions based on assumptions that an individual’s disability or age will lessen the possibility of survival beyond the short-term or require the use of greater treatment resources. Click here to read the letter.
AARP wrote a letter to OCR Director Roger Severino thanking him for OCR's recent call for Alabama to "abandon guidelines calling for blunt age and disability cutoffs for their Crisis Standards of Care." Categorically excluding people with disabilities or seniors from care violates federal law, the letter said, and it is important that health care providers not discriminate in that way. Click here to read the letter.
To track nondiscrimination and civil rights activities related to COVID-19, click here to view information on the Center for Public Representation website.
9. DUE WEDNESDAY: Request for Nominations for the Board of Governors of the Patient-Centered Outcomes Research Institute
The Government Accountability Office (GAO) is seeking nominations for PCORI's Board of Governors. Among several categories, GAO is looking to add a patient advocate to the Board of Governors. Letters of nomination should be submitted no later than THIS WEDNESDAY, May 13, 2020. Click here to read the notice and to submit a nomination.
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.
- New Zealand: Government says that drug that could extend the life of a teen with spinal muscular atrophy is too expensive. New Zealand family moves to Australia to get access to spinal muscular atrophy drug. Pharmac's withholding of a cancer drug causes 30 deaths in a week.
11. 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. 5/15/2020: 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: Open input period, no end date specified.
12. Upcoming Events and Webinars
Limited Competition PFA: Conducting Rare Disease Research using PCORnet® (Cycle 2 2020) Applicant Town Hall
May 12, 2020
Click here for details.
FT US Pharma and Biotech Summit 2020
May 14, 2020
Click here for details.
Advancing Value-Based Care
May 14, 2020
Click here for details.
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.
13. Medical Journal Articles
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.
Physician and Patient Adjustment to Reference Pricing for Drugs, click here to view.
Patient-Centered Outcomes Research and the Injured Patient: A Summary of Application, click here to view.
14. AHRQ Effective Program Updates
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.
Systematic Review: Noninvasive Nonpharmacologic Treatments for Chronic Pain. Click here to view.
Systematic Review: Care Interventions for People With Dementia (PWD) and Their Caregivers. Click here to view.
OPEN FOR COMMENT THROUGH MAY 4: Systematic Review: Interventions for Dyspnea in Patients with Advanced Cancer. Click here to view.
Research Protocol: Safety of Vaccines Used for Routine Immunization in the United States. Click here to view.
Systematic Review: Treatment of Depression in Children and Adolescents. Click here to view.
Research Report: Integrating Health System Data With Systematic Reviews. Click here to view