In This Week’s Issue:
1. Value Our Health: What is a Quality Adjusted Life Year, or QALY? Click here for a video on how QALYs put patients’ access at risk.
2. SWHR Migraine Toolkit, click here to view the toolkit.
3. Bloomberg: International Pricing Index Policy Expected in September, Opposed by Many Advocates, click here to view a letter from advocates and here for international case studies. Click here to view the Bloomberg article.
4. Aging Advocate: Discrimination has No Place in American Prescription Drug Coverage, click here to read the article.
5. RetireSafe: IPI Would Damage the Health of American Seniors, click here to read the article.
6. NHC Rubric on Capturing the Patient Voice -- Comment Opportunity, click here to see the Rubric and to comment.
7. CMS to Cover CAR-T Therapy for Patients Nationwide, click here to read the article.
8. ICER Announces Possible 2020 Assessments, click here to see the list of possible assessments.
9. AAR Seeks Recruits for Annual Senior Patient and Family Caregivers Network Training, click here to learn more and to apply for the training.
10. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
11. ICER Studies: Type 2 Diabetes, Arthritis, Cardiovascular Disease, Duchenne Muscular Dystrophy 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.
1. Value Our Health: What is a Quality Adjusted Life Year, or QALY? Click here for a video on how QALYs put patients’ access at risk.
2. SWHR Migraine Toolkit, click here to view the toolkit.
3. Bloomberg: International Pricing Index Policy Expected in September, Opposed by Many Advocates, click here to view a letter from advocates and here for international case studies. Click here to view the Bloomberg article.
4. Aging Advocate: Discrimination has No Place in American Prescription Drug Coverage, click here to read the article.
5. RetireSafe: IPI Would Damage the Health of American Seniors, click here to read the article.
6. NHC Rubric on Capturing the Patient Voice -- Comment Opportunity, click here to see the Rubric and to comment.
7. CMS to Cover CAR-T Therapy for Patients Nationwide, click here to read the article.
8. ICER Announces Possible 2020 Assessments, click here to see the list of possible assessments.
9. AAR Seeks Recruits for Annual Senior Patient and Family Caregivers Network Training, click here to learn more and to apply for the training.
10. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
11. ICER Studies: Type 2 Diabetes, Arthritis, Cardiovascular Disease, Duchenne Muscular Dystrophy 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.
1. Value Our Health: What is a Quality Adjusted Life Year, or QALY?
The use of flawed, discriminatory value assessments could threaten access to care for patients with chronic illnesses and people with disabilities. These assessments place a value on the life of a human based on their health status and assume every patient will respond the same way to treatments. Stay informed about the potential harms of clinical and cost-effectiveness analyses and their reliance on the quality-adjusted-life-year (QALY) by visiting the Value Our Health website: valueourhealth.org and be sure to check out this video on how QALYs put patients’ access at risk here.
2. SWHR Migraine Toolkit
The Society for Women’s Health Research (SWHR) recently released the Migraine Patient Toolkit: A Guide to Your Care,an informative resource for both people with migraine and healthcare providers, vetted by both medical experts and people with migraine. The toolkit is designed to facilitate productive conversations between patients and health care providers so people with migraine can get the best possible outcomes in their care. Healthcare providers can provide the toolkit to their patients to promote open dialogue about migraine. SWHR wants to ensure the Migraine Patient Toolkit reaches as many people as possible —including people with migraine and their caregivers, health care providers, employers, etc. Click here to view the toolkit.
3. Bloomberg: International Pricing Index Policy Expected in September, Opposed by Many Advocates
According to a senior White House official and reported by Bloomberg News, President Trump will give a major health policy speech in September, including an international pricing index (IPI) for purchasing certain drugs under the Medicare. The White House official gave no indication if the plan would resemble one outlined in an advance notice of proposed rulemaking (ANPRM) from October 2018 that targeted Part B drugs or if it would be more expansive. Under the system proposed last year, CMS would construct an index of Part B drug prices based on those in a basket of other wealthy countries, many of which use quality-adjusted life years (QALYs) and similar metrics based on averages and often resulting in coverage delays or non-coverage in other countries. Advocates sent a letter to CMS strongly opposing the initial proposed policy on December 31, 2018 for two reasons. First, by referencing the policies underpinning coverage and reimbursement of foreign governments, it effectively endorses the use of discriminatory cost-effectiveness standards used by foreign governments here in the U.S. Second, the proposal would be implemented through a large scale, mandatory “demonstration” that effectively forces almost half of doctors (and their Medicare patients) in the U.S. into a radical change in policy with unknown, and potentially very serious, effects on their patients without necessary safeguards to ensure their basic protections. Click here to view the letter from advocates and here for international case studies. Click here to view the Bloomberg article.
4. Aging Advocate: Discrimination has No Place in American Prescription Drug Coverage
Writing in the Tennessean, Alliance for Aging Research President and CEO Sue Peschin exposes ICER's discriminatory and arbitrary methodology for its value assessments. "In practice, this macabre cost-benefit analysis has alarming results. In the 1960s, one of the first applications of the QALY system was in the United Kingdom’s National Health Services, which rations health care to British citizens. This rationing system has since been fraught with controversy, facing accusations of mismanagement and leaving thousands of people without timely access to treatment. In any other industry, a QALY-like system that punishes the most vulnerable would rightly be viewed as an outrage," she writes. Click here to read the article.
5. RetireSafe: IPI Would Damage the Health of American Seniors
Mark Gibbons, president of RetireSafe, a group that advocates for the well-being of older Americans, writes that the administration's proposal to link U.S. drug markets to prices abroad would hurt American seniors. "Overly broad, one-size-fits-all drug pricing proposals that model themselves after those in countries with socialized health care systems are not the answer to the issue of rising drug costs. Beneficiaries must be able to continue accessing whichever medications they need, without middlemen interfering in this process and discounting their needs," he writes. Click here to read the article.
6. NHC Rubric on Capturing the Patient Voice -- Comment Opportunity
The National Health Council (NHC) recently released its Rubric to Capture the Patient Voice, a tool for the patient community and other stakeholders can use to evaluate attributes of patient-centeredness and to guide them on meaningful patient engagement. NHC is soliciting feedback from patients and other health care stakeholders. "We envision that the Rubric will be broadly useful across a range of activities, including research; medical-product development, including biopharmaceuticals, devices, and diagnostics; health care delivery and shared decision making; value assessment; real-world data analyses; evidence generation; scientific exchange and communication; and health policy. This Rubric can serve as a general blueprint in a wide range of circumstances where patient-centricity is desired," NHC writes, and solicits feedback on experiences implementing the Rubric. Click here to see the Rubric and to comment.
7. CMS to Cover CAR-T Therapy for Patients Nationwide
Reversing its previous position of covering CAR-T therapy with an evidence development requirement, CMS announced that it would cover the treatment for all eligible patients. "CMS decided not to proceed with a model called Coverage With Evidence With Development (CED), which critics feared could overburden providers, causing some to opt out of offering CAR T-cell treatment to Medicare patients," reported AJMC. "As we move forward with these new, life-saving treatments we look forward to continued collaboration with the agency to find ways to more effectively bring these treatments to Medicare beneficiaries in need," said Joseph Alvarnas, MD, editor-in-chief of Evidence-Based Oncology. Click here to read the article.
8. ICER Announces Possible 2020 Assessments
The Institute for Clinical and Economic Research (ICER) published a list of possible assessments for 2020. The possible assessments include drugs to treat cystic fibrosis, breast cancer, multiple sclerosis, and HIV. ICER's QALY-based studies are not transparent, use discriminatory cost-effectiveness measures, and ignore patient preferences and input. Click here to see the list of possible assessments.
9. AAR Seeks Recruits for Annual Senior Patient and Family Caregivers Network Training
Alliance for Aging Research’s Senior Patient and Family Caregiver Network (SP&FCN) is seeking advocates to participate in a research-advocacy training program designed to empower senior patients and their family caregivers to engage in patient-centered outcomes research (PCOR). The training will be held November 19-21, 2019 in Dallas, TX, and reimbursements for a hotel stay and travel expenses are available. In particular, the Alliance is looking for patients or caregivers of patients with Alzheimer’s disease, sarcopenia, atrial fibrillation, chronic pain, age-related macular degeneration, or heart valve disease. Click here to learn more and to apply for the training.
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.
11. ICER Studies: Acute Migraine, Type 2 Diabetes, Arthritis, Cardiovascular Disease, Duchenne Muscular Dystrophy, Sickle Cell Disease
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:
12. Upcoming Events and Webinars
Webinar: Real World Evidence and Data: A Tufts Study of 30 Pharma Companies
August 15, 2019
Click here for details.
PCORI Board of Governors Meeting
August 20, 2019
Click here for details.
Webinar: How Frequently Should Cancer Survivors be Screened for Disease Recurrence? Is More Always Better?
August 22, 2019
Click here for details.
Health Spending: Moving from Theory to Action
September 11, 2019
Click here for details.
PCORI Improving Methods Applicant Town Hall
September 12, 2019
Click here for details.
Advisory Panel on Rare Disease Fall 2019 Meeting
September 16, 2019
Click here for details.
IMPACCT: Real World Evidence
September 18-19, 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.
ISPOR Summit 2019 on Building Trust in RWE – The Role of Study Registration
October 11, 2019
Click here for details.
2019 AUCD Annual Meeting
November 17-20, 2019
Click here for details.
13. Medical Journal Articles
The Importance of Measuring the Impact of Patient-Oriented Research, click here to view.
Building Evidence and Measuring Clinical Outcomes for Genomic Medicine, click here to view.
Evidence-Based Medicine: A Data-Driven Approach to Lean Healthcare Operations, click here to view.
Patient Registries: An Underused Resource for Medicines Evaluation: Operational Proposals for Increasing the Use of Patient Registries in Regulatory Assessments, click here to view.
Commonly Used Definitions in Real-World Studies May Underestimate the Prevalence of Renal Disease Among Nonvalvular Atrial Fibrillation Patients, click here to view.
Factors Associated with Evidence-Based Decision-Making Among Patients and Providers, click here to view.
Rethinking Bias and Truth in Evidence-Based Medicine, click here to view.
Ethical Challenges Related to Patient Involvement in Health Technology Assessment, click here to view.
Value-Based Insurance Design: Current Evidence and Future Directions, click here to view.
Novel Approaches to Value Assessment Beyond the Cost-Effectiveness Framework, click here to view.
14. AHRQ Effective Program Updates
AHRQ EPC Pilot Projects Summary: Improving Health Systems' Access to High Quality Evidence, click here to view.
Research Protocol: Care Interventions for People With Dementia (PWD) and Their Caregivers, 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.
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: Long-Term Drug Therapy and Drug Holidays for Osteoporosis Fracture Prevention, click here to view.
The use of flawed, discriminatory value assessments could threaten access to care for patients with chronic illnesses and people with disabilities. These assessments place a value on the life of a human based on their health status and assume every patient will respond the same way to treatments. Stay informed about the potential harms of clinical and cost-effectiveness analyses and their reliance on the quality-adjusted-life-year (QALY) by visiting the Value Our Health website: valueourhealth.org and be sure to check out this video on how QALYs put patients’ access at risk here.
2. SWHR Migraine Toolkit
The Society for Women’s Health Research (SWHR) recently released the Migraine Patient Toolkit: A Guide to Your Care,an informative resource for both people with migraine and healthcare providers, vetted by both medical experts and people with migraine. The toolkit is designed to facilitate productive conversations between patients and health care providers so people with migraine can get the best possible outcomes in their care. Healthcare providers can provide the toolkit to their patients to promote open dialogue about migraine. SWHR wants to ensure the Migraine Patient Toolkit reaches as many people as possible —including people with migraine and their caregivers, health care providers, employers, etc. Click here to view the toolkit.
3. Bloomberg: International Pricing Index Policy Expected in September, Opposed by Many Advocates
According to a senior White House official and reported by Bloomberg News, President Trump will give a major health policy speech in September, including an international pricing index (IPI) for purchasing certain drugs under the Medicare. The White House official gave no indication if the plan would resemble one outlined in an advance notice of proposed rulemaking (ANPRM) from October 2018 that targeted Part B drugs or if it would be more expansive. Under the system proposed last year, CMS would construct an index of Part B drug prices based on those in a basket of other wealthy countries, many of which use quality-adjusted life years (QALYs) and similar metrics based on averages and often resulting in coverage delays or non-coverage in other countries. Advocates sent a letter to CMS strongly opposing the initial proposed policy on December 31, 2018 for two reasons. First, by referencing the policies underpinning coverage and reimbursement of foreign governments, it effectively endorses the use of discriminatory cost-effectiveness standards used by foreign governments here in the U.S. Second, the proposal would be implemented through a large scale, mandatory “demonstration” that effectively forces almost half of doctors (and their Medicare patients) in the U.S. into a radical change in policy with unknown, and potentially very serious, effects on their patients without necessary safeguards to ensure their basic protections. Click here to view the letter from advocates and here for international case studies. Click here to view the Bloomberg article.
4. Aging Advocate: Discrimination has No Place in American Prescription Drug Coverage
Writing in the Tennessean, Alliance for Aging Research President and CEO Sue Peschin exposes ICER's discriminatory and arbitrary methodology for its value assessments. "In practice, this macabre cost-benefit analysis has alarming results. In the 1960s, one of the first applications of the QALY system was in the United Kingdom’s National Health Services, which rations health care to British citizens. This rationing system has since been fraught with controversy, facing accusations of mismanagement and leaving thousands of people without timely access to treatment. In any other industry, a QALY-like system that punishes the most vulnerable would rightly be viewed as an outrage," she writes. Click here to read the article.
5. RetireSafe: IPI Would Damage the Health of American Seniors
Mark Gibbons, president of RetireSafe, a group that advocates for the well-being of older Americans, writes that the administration's proposal to link U.S. drug markets to prices abroad would hurt American seniors. "Overly broad, one-size-fits-all drug pricing proposals that model themselves after those in countries with socialized health care systems are not the answer to the issue of rising drug costs. Beneficiaries must be able to continue accessing whichever medications they need, without middlemen interfering in this process and discounting their needs," he writes. Click here to read the article.
6. NHC Rubric on Capturing the Patient Voice -- Comment Opportunity
The National Health Council (NHC) recently released its Rubric to Capture the Patient Voice, a tool for the patient community and other stakeholders can use to evaluate attributes of patient-centeredness and to guide them on meaningful patient engagement. NHC is soliciting feedback from patients and other health care stakeholders. "We envision that the Rubric will be broadly useful across a range of activities, including research; medical-product development, including biopharmaceuticals, devices, and diagnostics; health care delivery and shared decision making; value assessment; real-world data analyses; evidence generation; scientific exchange and communication; and health policy. This Rubric can serve as a general blueprint in a wide range of circumstances where patient-centricity is desired," NHC writes, and solicits feedback on experiences implementing the Rubric. Click here to see the Rubric and to comment.
7. CMS to Cover CAR-T Therapy for Patients Nationwide
Reversing its previous position of covering CAR-T therapy with an evidence development requirement, CMS announced that it would cover the treatment for all eligible patients. "CMS decided not to proceed with a model called Coverage With Evidence With Development (CED), which critics feared could overburden providers, causing some to opt out of offering CAR T-cell treatment to Medicare patients," reported AJMC. "As we move forward with these new, life-saving treatments we look forward to continued collaboration with the agency to find ways to more effectively bring these treatments to Medicare beneficiaries in need," said Joseph Alvarnas, MD, editor-in-chief of Evidence-Based Oncology. Click here to read the article.
8. ICER Announces Possible 2020 Assessments
The Institute for Clinical and Economic Research (ICER) published a list of possible assessments for 2020. The possible assessments include drugs to treat cystic fibrosis, breast cancer, multiple sclerosis, and HIV. ICER's QALY-based studies are not transparent, use discriminatory cost-effectiveness measures, and ignore patient preferences and input. Click here to see the list of possible assessments.
9. AAR Seeks Recruits for Annual Senior Patient and Family Caregivers Network Training
Alliance for Aging Research’s Senior Patient and Family Caregiver Network (SP&FCN) is seeking advocates to participate in a research-advocacy training program designed to empower senior patients and their family caregivers to engage in patient-centered outcomes research (PCOR). The training will be held November 19-21, 2019 in Dallas, TX, and reimbursements for a hotel stay and travel expenses are available. In particular, the Alliance is looking for patients or caregivers of patients with Alzheimer’s disease, sarcopenia, atrial fibrillation, chronic pain, age-related macular degeneration, or heart valve disease. Click here to learn more and to apply for the training.
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.
- Australia: Patients travel to U.S., spend hundreds of thousands of dollars on treatment they do not qualify for in Australia. Australians fight for treatment for blood cancer patients.
- New Zealand: Lung cancer patients fight for a review of Pharmac and better coverage of drugs. Pharmac won't fund needed treatment for infantile hypophosphotasia. Patients head to Mexico to access MS treatment.
- Canada: Patients are drawing attention to the government's lack of coverage of cystic fibrosis drugs. Lifesaving SMA drugs continue to not be covered.
- United Kingdom: Patients needing lifesaving cancer drugs may not have time to negotiate with NHS. Following patient pressure, NICE reconsidering making lifesaving Batten Disease drug available.
11. ICER Studies: Acute Migraine, Type 2 Diabetes, Arthritis, Cardiovascular Disease, Duchenne Muscular Dystrophy, Sickle Cell Disease
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:
- Sickle Cell Disease: 8/30/2019: Draft Scoping Document. 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: Model Analysis Plan available. 9/18/2019: Draft Evidence Report. Meeting 11/19/2019: CTAF to an update to its 2017 rheumatoid arthritis assessment.
- Type 2 Diabetes: Model Analysis Plan available. 9/11/2019: Draft Evidence Report. 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 : Draft Evidence Report and Draft Voting Questions available and OPEN for public comment through 8/20/2019. 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.
- Duchenne Muscular Dystrophy: Evidence Presentation available. Meeting 7/25/2019: New England CEPAC deliberated and voted on evidence presented in ICER's report on treatments for Duchenne muscular dystrophy. 8/15/2019: Final Evidence Report and Meeting Summary.
- Unsupported Price Increase Assessment: 10/8/2019: Final Report.
- Acute Migraine: Revised Scoping Document available. 9/6/2019: Research Protocol. Meeting 1/23/2020: Midwest CEPAC to review ICER's assessment of acute migraine treatments
- Valuing A Cure Project: White Paper AVAILABLE. Comment period open 8/6/2019-9/6/2019. Meeting 9/17/2019: Invited stakeholders to discuss single or short-term transformative therapies.
12. Upcoming Events and Webinars
Webinar: Real World Evidence and Data: A Tufts Study of 30 Pharma Companies
August 15, 2019
Click here for details.
PCORI Board of Governors Meeting
August 20, 2019
Click here for details.
Webinar: How Frequently Should Cancer Survivors be Screened for Disease Recurrence? Is More Always Better?
August 22, 2019
Click here for details.
Health Spending: Moving from Theory to Action
September 11, 2019
Click here for details.
PCORI Improving Methods Applicant Town Hall
September 12, 2019
Click here for details.
Advisory Panel on Rare Disease Fall 2019 Meeting
September 16, 2019
Click here for details.
IMPACCT: Real World Evidence
September 18-19, 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.
ISPOR Summit 2019 on Building Trust in RWE – The Role of Study Registration
October 11, 2019
Click here for details.
2019 AUCD Annual Meeting
November 17-20, 2019
Click here for details.
13. Medical Journal Articles
The Importance of Measuring the Impact of Patient-Oriented Research, click here to view.
Building Evidence and Measuring Clinical Outcomes for Genomic Medicine, click here to view.
Evidence-Based Medicine: A Data-Driven Approach to Lean Healthcare Operations, click here to view.
Patient Registries: An Underused Resource for Medicines Evaluation: Operational Proposals for Increasing the Use of Patient Registries in Regulatory Assessments, click here to view.
Commonly Used Definitions in Real-World Studies May Underestimate the Prevalence of Renal Disease Among Nonvalvular Atrial Fibrillation Patients, click here to view.
Factors Associated with Evidence-Based Decision-Making Among Patients and Providers, click here to view.
Rethinking Bias and Truth in Evidence-Based Medicine, click here to view.
Ethical Challenges Related to Patient Involvement in Health Technology Assessment, click here to view.
Value-Based Insurance Design: Current Evidence and Future Directions, click here to view.
Novel Approaches to Value Assessment Beyond the Cost-Effectiveness Framework, click here to view.
14. AHRQ Effective Program Updates
AHRQ EPC Pilot Projects Summary: Improving Health Systems' Access to High Quality Evidence, click here to view.
Research Protocol: Care Interventions for People With Dementia (PWD) and Their Caregivers, 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.
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: Long-Term Drug Therapy and Drug Holidays for Osteoporosis Fracture Prevention, click here to view.
