In This Week’s Issue:
1. Value Assessments – the Impact on People with Rare Diseases, click here to learn more and watch the webinar.
2. Webinar: What's Ahead for Comparative Effective Research? Click here to learn more and RSVP.
3. The Evidence Base Interview: Quarreling Over the QALY, click here to read the full interview.
4. Value Our Health Op-Ed: Stop Discriminatory Value Assessments, click here to read the op-ed.
5. Veterans Groups in California Comment on ICER’s 2020 Framework, click here to read the American Legion letter and click here to read the joint letter.
6. NHC Rubric to Capture the Patient Voice, click here to learn more and see the rubric.
7. AJMC: Precision Medicines Need Precision Patient Assistance Programs, 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 Assessments – the Impact on People with Rare Diseases, click here to learn more and watch the webinar.
2. Webinar: What's Ahead for Comparative Effective Research? Click here to learn more and RSVP.
3. The Evidence Base Interview: Quarreling Over the QALY, click here to read the full interview.
4. Value Our Health Op-Ed: Stop Discriminatory Value Assessments, click here to read the op-ed.
5. Veterans Groups in California Comment on ICER’s 2020 Framework, click here to read the American Legion letter and click here to read the joint letter.
6. NHC Rubric to Capture the Patient Voice, click here to learn more and see the rubric.
7. AJMC: Precision Medicines Need Precision Patient Assistance Programs, 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 Assessments – the Impact on People with Rare Diseases
On July 17, the Partnership to Improve Patient Care (PIPC) and Everylife Foundation for Rare Diseases held a webinar about value assessments, their potential to limit access and innovation for rare disease treatments. The webinar featured Sara van Geertruyden, Executive Director, Partnership to Improve Patient Care; Ari Ne’eman, Disability Advocate, Partnership to Improve Patient Care; Siri Vaeth, Executive Director, Cystic Fibrosis Research, Inc.; and Christina Hartman, Senior Director of Policy and Advocacy, Everylife Foundation for Rare Diseases. The panelists highlighted their concern about the value assessments — like the quality-adjusted life year (QALY) and equal value life-year gained (evLYG) – because the metrics they use treat patients as averages. They noted that cost-effectiveness assessments are particularly concerning when it comes to treatments developed for rare diseases, as they have the potential to impact both patient access and disincentivize investment in rare disease research and development. Click here to learn more and watch the webinar.
2. Webinar: What's Ahead for Comparative Effective Research?
On July 24, the Alliance for Health Policy will hold a webinar on the future of comparative effectiveness research and the legislation that will shape it. Participants will outline the various PCORI reauthorization bills and amendments that have been discussed by House and Senate committees to date. Our panel of experts will also explore some of the critical questions facing congressional staff and discuss potential next steps in the legislative process. Click here to learn more and RSVP.
3. The Evidence Base Interview: Quarreling Over the QALY
The National Health Council's Eleanor Perfetto gave an interview based on her ISPOR presentation on the QALY and its misapplications and shortcomings. "Outcomes from clinical trials, for example, which have often been incorporated into the QALY calculation, may not be the outcomes that patients care about. Therefore, we are potentially making an access decision based on an incorrect endpoint. I see this in my work; decision makers, who have access to this information, might rely on it, but they might also be motivated to restrict patient access to save themselves money. It could be used for convenience, to back a money-saving decision. This is what we want to avoid. You may ask: what is the problem? Is it the methodology of calculating the QALY, or the way the QALY is implemented? Which is the problem? I believe it is both. The methodology is problematic because we know that there are inherent limitations to it, and that some poor or flawed data is often incorporated into calculating it. The implementation of the QALY is also an issue, as some may use it to make an access decision without correct context," she wrote. Click here to read the full interview.
4. Value Our Health Op-Ed: Stop Discriminatory Value Assessments
Elizabeth Franklin, Executive Director of Cancer Support Community's Cancer Policy Institute, wrote in an op-ed for Morning Consult that patients are concerned with the QALY's implications, calling the measure “ethically inappropriate” and “immoral.” "As a representative of the patient advocacy community, we believe that patients make informed decisions that are right for them. In a partnership with their loved ones and their providers, patients make careful choices regarding treatments that fit into their lives and personal value framework. Discriminatory measures such as the QALY should be banned in the United States. Organizations determining value must be completely transparent and incorporate patient, survivor and caregiver feedback into assessments. Individuals living with chronic diseases and disabilities have a right to know how decisions that could have irreparable impact on their lives are being made and must have their voices heard," she wrote. Click here to read the op-ed.
5. Veterans Groups in California Comment on ICER’s 2020 Framework
Veterans groups in California submitted comment letters on ICER's 2020 assessment framework, highlighting their opposition to the use of discriminatory measures such as the QALY. California's American Legion wrote "We have significant concerns that...protections against the use of a cost-per-QALY value assessment do not exist for our members. It is profoundly unfair and offensive to those who have served this country to allow for this kind of discrimination in the veterans health system." ICER's frameworks are particularly important to veterans as the Veterans Affairs Administration agreed to work with ICER to integrate the group's reports into its formulary. A group of veterans organizations composed of AMVETS, the American GI Forum, the California Association of County Veterans Service Officers, NGAC, and the Women Veterans Alliance penned a similar letter to ICER. Click here to read the American Legion letter and click here to read the joint letter.
6. NHC Rubric to Capture the Patient Voice
There is finally broad consensus in the US and globally that patients should be engaged in all aspects of health, from research, to developing a new treatment, to care delivery, and in policy. However, there remains confusion about and misuse of what it means to be patient-centered and how to achieve meaningful patient engagement. To this end, the National Health Council (NHC) has created the NHC Rubric to Capture the Patient Voice: A Guide to Incorporating the Patient Voice into the Health Ecosystem. Potential uses might include, but are not limited to, patient engagement in health care policy, quality-measure development, shared decision-making, value assessment, product development, etc. Click here to learn more and see the rubric.
7. AJMC: Precision Medicines Need Precision Patient Assistance Programs
There is a need for patient assistance programs that truly target those in need with the treatments that will help them, write two health care experts for the American Journal of Managed Care. "There is a robust evidence base showing that individuals who are subject to high levels of cost sharing use less of both high- and low-value care in similar proportions. The higher the cost sharing, the greater the corresponding reduction in service use. Not surprisingly, cost-related underuse of evidence-based services disproportionally impacts poorer Americans and those with chronic conditions," they wrote. 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, Peanut Allergy
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
PCORI Board of Governors Meeting
July 23, 2019
Click here for details.
Webinar: VBID X: A New Plan Option for the Individual Health Insurance Market
July 24, 2019
Click here for details.
NVHR Hepatitis C Patient Summit
July 29-30, 2019
Click here for details.
Webinar: Rethinking Drug Reimbursement: Value Communications in a Post-Rebate World
July 31, 2019
Click here for details.
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.
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.
2019 AUCD Annual Meeting
November 17-20, 2019
Click here for details.
13. Medical Journal Articles
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.
Navigating Joint HTA, Procurement, and Fair Pricing: Evidence-Based Insights and Practical Recommendations - A Meeting Report from ISPOR Regional Conference in Warsaw, 2019, click here to view.
Health Technology Assessment as Part of a Broader Process for Priority Setting and Resource Allocation, click here to view.
14. AHRQ Effective Program Updates
Systematic Review: Achieving Health Equity in Preventative Services, 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.
Systematic Review: Treatment of Depression in Children, 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: Diagnosis and Treatment of Clinical Alzheimer's-type Dementia (CATD), click here to view.
Systematic Review: Comparative Effectiveness of Analgesics to Reduce Acute Pain in the Prehospital Setting, click here to view.
Systematic Review: Long-Term Drug Therapy and Drug Holidays for Osteoporosis Fracture Prevention, click here to view.
Systematic Review: Telehealth for Acute and Chronic Care Consultations, click here to view.
On July 17, the Partnership to Improve Patient Care (PIPC) and Everylife Foundation for Rare Diseases held a webinar about value assessments, their potential to limit access and innovation for rare disease treatments. The webinar featured Sara van Geertruyden, Executive Director, Partnership to Improve Patient Care; Ari Ne’eman, Disability Advocate, Partnership to Improve Patient Care; Siri Vaeth, Executive Director, Cystic Fibrosis Research, Inc.; and Christina Hartman, Senior Director of Policy and Advocacy, Everylife Foundation for Rare Diseases. The panelists highlighted their concern about the value assessments — like the quality-adjusted life year (QALY) and equal value life-year gained (evLYG) – because the metrics they use treat patients as averages. They noted that cost-effectiveness assessments are particularly concerning when it comes to treatments developed for rare diseases, as they have the potential to impact both patient access and disincentivize investment in rare disease research and development. Click here to learn more and watch the webinar.
2. Webinar: What's Ahead for Comparative Effective Research?
On July 24, the Alliance for Health Policy will hold a webinar on the future of comparative effectiveness research and the legislation that will shape it. Participants will outline the various PCORI reauthorization bills and amendments that have been discussed by House and Senate committees to date. Our panel of experts will also explore some of the critical questions facing congressional staff and discuss potential next steps in the legislative process. Click here to learn more and RSVP.
3. The Evidence Base Interview: Quarreling Over the QALY
The National Health Council's Eleanor Perfetto gave an interview based on her ISPOR presentation on the QALY and its misapplications and shortcomings. "Outcomes from clinical trials, for example, which have often been incorporated into the QALY calculation, may not be the outcomes that patients care about. Therefore, we are potentially making an access decision based on an incorrect endpoint. I see this in my work; decision makers, who have access to this information, might rely on it, but they might also be motivated to restrict patient access to save themselves money. It could be used for convenience, to back a money-saving decision. This is what we want to avoid. You may ask: what is the problem? Is it the methodology of calculating the QALY, or the way the QALY is implemented? Which is the problem? I believe it is both. The methodology is problematic because we know that there are inherent limitations to it, and that some poor or flawed data is often incorporated into calculating it. The implementation of the QALY is also an issue, as some may use it to make an access decision without correct context," she wrote. Click here to read the full interview.
4. Value Our Health Op-Ed: Stop Discriminatory Value Assessments
Elizabeth Franklin, Executive Director of Cancer Support Community's Cancer Policy Institute, wrote in an op-ed for Morning Consult that patients are concerned with the QALY's implications, calling the measure “ethically inappropriate” and “immoral.” "As a representative of the patient advocacy community, we believe that patients make informed decisions that are right for them. In a partnership with their loved ones and their providers, patients make careful choices regarding treatments that fit into their lives and personal value framework. Discriminatory measures such as the QALY should be banned in the United States. Organizations determining value must be completely transparent and incorporate patient, survivor and caregiver feedback into assessments. Individuals living with chronic diseases and disabilities have a right to know how decisions that could have irreparable impact on their lives are being made and must have their voices heard," she wrote. Click here to read the op-ed.
5. Veterans Groups in California Comment on ICER’s 2020 Framework
Veterans groups in California submitted comment letters on ICER's 2020 assessment framework, highlighting their opposition to the use of discriminatory measures such as the QALY. California's American Legion wrote "We have significant concerns that...protections against the use of a cost-per-QALY value assessment do not exist for our members. It is profoundly unfair and offensive to those who have served this country to allow for this kind of discrimination in the veterans health system." ICER's frameworks are particularly important to veterans as the Veterans Affairs Administration agreed to work with ICER to integrate the group's reports into its formulary. A group of veterans organizations composed of AMVETS, the American GI Forum, the California Association of County Veterans Service Officers, NGAC, and the Women Veterans Alliance penned a similar letter to ICER. Click here to read the American Legion letter and click here to read the joint letter.
6. NHC Rubric to Capture the Patient Voice
There is finally broad consensus in the US and globally that patients should be engaged in all aspects of health, from research, to developing a new treatment, to care delivery, and in policy. However, there remains confusion about and misuse of what it means to be patient-centered and how to achieve meaningful patient engagement. To this end, the National Health Council (NHC) has created the NHC Rubric to Capture the Patient Voice: A Guide to Incorporating the Patient Voice into the Health Ecosystem. Potential uses might include, but are not limited to, patient engagement in health care policy, quality-measure development, shared decision-making, value assessment, product development, etc. Click here to learn more and see the rubric.
7. AJMC: Precision Medicines Need Precision Patient Assistance Programs
There is a need for patient assistance programs that truly target those in need with the treatments that will help them, write two health care experts for the American Journal of Managed Care. "There is a robust evidence base showing that individuals who are subject to high levels of cost sharing use less of both high- and low-value care in similar proportions. The higher the cost sharing, the greater the corresponding reduction in service use. Not surprisingly, cost-related underuse of evidence-based services disproportionally impacts poorer Americans and those with chronic conditions," they wrote. 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: Children with cystic fibrosis have lifesaving drugs rejected by the government for being too expensive. Australians are dying due to non-coverage of vaccines.
- New Zealand: Breast cancer advocates call for review of drug funding problems. Newspaper criticizes government's "hardline" approach on funding needed drugs.
- 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 continue to fight for access to cystic fibrosis drugs. Click here, here, here, and here for more.
11. ICER Studies: Acute Migraine, Type 2 Diabetes, Arthritis, Cardiovascular Disease, Duchenne Muscular Dystrophy, Peanut Allergy
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:
- Arthritis: Research Protocol available. 8/5/2019: Model Analysis Plan. Meeting 11/19/2019: CTAF to an update to its 2017 rheumatoid arthritis assessment.
- Type 2 Diabetes: Research Protocol Available. 7/29/2019: Model Analysis Plan. 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 : Model Analysis Plan available. 7/24/2019: Draft Evidence Report. 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: Meeting agenda available. Meeting 7/25/2019: New England CEPAC to deliberate and vote on evidence presented in ICER's report on treatments for Duchenne muscular dystrophy.
- Unsupported Price Increase Assessment: 10/8/2019: Final Report.
- Acute Migraine: 7/29/2019: Revised Scoping Document. Meeting 1/23/2020: Midwest CEPAC to review ICER's assessment of acute migraine treatments
- Valuing A Cure Project: White Paper available 8/6/2019. Comment period open 8/6/2019-9/3/2019.
12. Upcoming Events and Webinars
PCORI Board of Governors Meeting
July 23, 2019
Click here for details.
Webinar: VBID X: A New Plan Option for the Individual Health Insurance Market
July 24, 2019
Click here for details.
NVHR Hepatitis C Patient Summit
July 29-30, 2019
Click here for details.
Webinar: Rethinking Drug Reimbursement: Value Communications in a Post-Rebate World
July 31, 2019
Click here for details.
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.
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.
2019 AUCD Annual Meeting
November 17-20, 2019
Click here for details.
13. Medical Journal Articles
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.
Navigating Joint HTA, Procurement, and Fair Pricing: Evidence-Based Insights and Practical Recommendations - A Meeting Report from ISPOR Regional Conference in Warsaw, 2019, click here to view.
Health Technology Assessment as Part of a Broader Process for Priority Setting and Resource Allocation, click here to view.
14. AHRQ Effective Program Updates
Systematic Review: Achieving Health Equity in Preventative Services, 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.
Systematic Review: Treatment of Depression in Children, 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: Diagnosis and Treatment of Clinical Alzheimer's-type Dementia (CATD), click here to view.
Systematic Review: Comparative Effectiveness of Analgesics to Reduce Acute Pain in the Prehospital Setting, click here to view.
Systematic Review: Long-Term Drug Therapy and Drug Holidays for Osteoporosis Fracture Prevention, click here to view.
Systematic Review: Telehealth for Acute and Chronic Care Consultations, click here to view.
