In This Week’s Issue:
1. Pioneer Institute: ICER's Methods Are Likely Illegal under the ADA, click here to read the report.
2. Massachusetts Finalizes Value Assessment Regulations, see details below.
3. ICER's Oral Semaglutide Model Exists in an Imaginary World, click here to read the article.
4. Patients and People with Disabilities Remain Concerned about Importing QALYs From Other Countries, see details below.
5. Administration’s Medicaid Initiative Would Invite Closed Formularies, Implicate Use of QALYs, see details below.
6. A Trusting Relationship is Crucial to Bridge the Gap Between Quality Metrics and Real Life, click here to read the article (subscription needed).
7. ICER Releases Revised Value Assessment Framework for 2020, see details below.
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, Beta Thalassemia, Acute Migraine, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, 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.
1. Pioneer Institute: ICER's Methods Are Likely Illegal under the ADA, click here to read the report.
2. Massachusetts Finalizes Value Assessment Regulations, see details below.
3. ICER's Oral Semaglutide Model Exists in an Imaginary World, click here to read the article.
4. Patients and People with Disabilities Remain Concerned about Importing QALYs From Other Countries, see details below.
5. Administration’s Medicaid Initiative Would Invite Closed Formularies, Implicate Use of QALYs, see details below.
6. A Trusting Relationship is Crucial to Bridge the Gap Between Quality Metrics and Real Life, click here to read the article (subscription needed).
7. ICER Releases Revised Value Assessment Framework for 2020, see details below.
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, Beta Thalassemia, Acute Migraine, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, 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.
1. Pioneer Institute: ICER's Methods Are Likely Illegal under the ADA
A new report from the Pioneer Institute found that ICER's methods for value assessment are likely illegal under the Americans with Disabilities Act (ADA) if applied to government programs because they may decrease the availability of effective treatments for people living with a disability and increase the risk of institutionalization of certain people living with a mental disability. Pioneer Institute’s report predicts that a legal challenge to any adoption of ICER’s QALY methodology by state Medicaid systems on ADA grounds seems almost certain. Click here to read the report.
2. Massachusetts Finalizes Value Assessment Regulations
The Massachusetts Health Policy Commission finalized its regulations on how they will evaluate the “value” of medications as a part of the oversight and administration of prescription drug pricing. Advocates sent letters to the Commission and met in person with the commission to advocate for an explicit ban on the use of quality-adjusted life years (QALYs) in the regulation. The Commission ultimately did not include any provisions that would ban or even restrict or limit the use of QALY-based value assessments such as those conducted by the Institute for Clinical and Economic Review (ICER). And they opened the door to international reference pricing from countries that use QALYs. The final regulation did include new language on transparency and public input that may be useful in allowing patients and people with disabilities to make their voices heard in this process. But it was very disappointing that the Commission did nothing to acknowledge that QALYs discriminate, despite significant engagement.
In response to the Commission’s final regulation, PIPC joined patient organizations in Massachusetts in sending out a press statement. We stated, "Massachusetts has always been a leader in both health care and inclusion, and this regulation does not honor that legacy.” We also shared the National Council on Disability report on QALYs and the comment letter that advocates sent to the HPC.
3. ICER's Oral Semaglutide Model Exists in an Imaginary World
Writing in Innovations in Pharmacy, a University of Minnesota researcher writes that ICER's Oral Semaglutide model has little semblance to reality. "While this model differs from previous ICER models, the result is still a framework that constructs a so-called evidence base that fails the demarcation test. It is best described as pseudoscience. The model creates, by assumption, an imaginary world. The claims made for oral semaglutide by ICER should not be taken seriously by health care decision makers. The purpose of this commentary is to point to the limitations of the model with particular reference to the utility metrics employed, the resulting claims for quality adjusted life years (QALYs) and consequent recommendations for price discounting and affordability," he writes. Click here to read the article.
4. Patients and People with Disabilities Remain Concerned about Importing QALYs From Other Countries
PIPC Chairman Tony Coelho called on advocates to urge the Administration to follow the recommendations of the National Council on Disability opposing policies that would reference health care prices and policies established in other countries using discriminatory QALYs and similar summary metrics. Chairman Coelho stated, "Is the life of a person with a disability or chronic condition less valuable than the life of a person without one? There is renewed interest among lawmakers to tackle healthcare costs, specifically, the rising cost of prescription drugs. However, the Administration’s proposed International Pricing Index (IPI) model, an initiative proposed in 2018 that would arbitrarily base prices of American medications off the government set prices of medications in foreign countries, is deeply flawed. Yet, we may see some version of it reintroduced soon.” Click here to view the PIPC Chairman Blog. Click here to view a letter from advocates opposing an International Pricing Index. Click here for more information from experts.
5. Administration’s Medicaid Initiative Would Invite Closed Formularies, Implicate Use of QALYs
On January 30, 2020, the administration announced the Healthy Adult Opportunity (HAO) initiative allowing states to carry out demonstrations under section 1115(a)(2) of the Social Security Act (the Act) to provide "cost-effective" coverage using "flexible benefit designs.” Among its provisions, State Medicaid Directors were invited to "adopt a closed formulary in line with Essential Health Benefit (EHB) requirements (with special protections for individuals with HIV and behavioral health conditions)…This means that the open formulary requirements in section 1927 that apply to state plan drug coverage do not apply.” PIPC remains concerned that states will respond to increased opportunities to limit formularies and restrict access to therapies by referencing “cost effectiveness” studies that discriminate against patients and people with disabilities by using quality-adjusted life years (QALYs) and similar summary metrics. Click here to view the letter to State Medicaid Directors. Click here to view stakeholder comments opposing Tennessee’s waiver application. Click here to view a legislative template for states to advance patient-centered research in decision-making.
6. A Trusting Relationship is Crucial to Bridge the Gap Between Quality Metrics and Real Life
Doctors and patients need to have good, trusting relationships to bridge the gap between impersonal quality measures and reality, writes Dr. Jennifer DeVoe. "On the surface, the American College of Cardiology/American Heart Association guidelines for treating hypertension seemed straightforward....I did not have a good answer when she asked me, 'Dr DeVoe, given my history and tendency to experience side effects with every new medication we try, is this one going to be any different?' And where was the evidence-based guideline to answer her questions about whether spending money to buy this medication was more important than buying the healthy foods we had also recommended?" Patient-centered care and a longstanding doctor-patient relationship can begin to answer those questions. Click here to read the article (subscription needed).
7. ICER Releases Revised Value Assessment Framework for 2020
On Jan. 31, the Institute for Clinical and Economic Review (ICER) released its final revisions to its value assessment framework. The document describes changes in ICER’s process, including creating a formal process for incorporating new evidence that emerges after the assessment, standardizing cost-effectiveness thresholds across reviews, and increasing the incorporation of real-world evidence.
While the revised framework seeks to formalize a patient engagement program, the program falls short in committing to meaningful incorporation of patient perspectives and preferences into ICER’s reviews. ICER commits to work with patient groups throughout the process, but it does little to ensure that the patient voice is heard. The framework also makes it clear that economic analysis still maintains primacy at ICER and would be counted as more valuable than patient input.
In response to a request for feedback, stakeholders submitted comments on ICER’s draft 2020 value assessment framework last summer, but ICER failed to incorporate their suggestions in several important ways. First and foremost, ICER doubles down on its embrace of the discriminatory QALY measure and its cousin, the equal value of life years gained (evLYG) measure. Stakeholders also called on ICER to meaningfully incorporate patient preferences, but ICER instead opted to ceremonially consider patient input and not commit to patient-centered decision making.
The group did adopt a process to use real-world evidence (RWE) to update assessments of treatments that were approved under an accelerated pathway after the treatment has been on the market for two years. ICER does not, however, commit to using RWE in its base models, choosing to continue to conduct rushed assessments before appropriate data are available.
Unfortunately, ICER’s updated framework does not take a meaningful turn towards patient-centeredness. Under these revised guidelines, ICER will continue to use discriminatory measures to argue for reduced access to life-changing treatments. Click here to view the final value assessment framework. Click here to view PIPC's comments on the framework. Click here to read PIPC's Data Mine blog and see a table comparing PIPC's comments to ICER's final product.
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.
9. ICER's QALY-Based Study Topics: Hemophilia A, Beta Thalassemia, Acute Migraine, 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:
10. Upcoming Events and Webinars
PCORI Board of Governors Meeting
February 25, 2020
Click here for details.
PCORI Board of Governors Meeting
March 2, 2020
Click here for details.
Webinar: Pharmaceutical Contracting: Focus on Value-Based Contracting
March 3, 2020
Click here for details.
Market Access USA at eyeforpharma Philadelphia
April 15, 2020
Click here for details.
FT US Pharma and Biotech Summit 2020
May 14, 2020
Click here for details.
11. Medical Journal Articles
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.
Does the Use of Health Technology Assessment Have an Impact on the Utilisation of Health Care Resources? Evidence from Two European Countries, click here to view.
Patients, Clinicians and Researchers Working Together to Improve Cardiovascular Health: A Qualitative Study of Barriers and Priorities for Patient-Oriented Research, click here to view.
HTA Around the World: Broadening Our Understanding of Cross-Country Differences, click here to view.
Early Returns From the Era of Precision Medicine, click here to view.
Who Assigns Value in Value-Based Insurance Design?, click here to view.
Does the Institute for Clinical and Economic Review Revise Its Findings in Response to Industry Comments?, click here to view.
The Patient-Centered Outcomes Research Network Antibiotics and Childhood Growth Study: Implementing Patient Data Linkage, click here to view.
Viewpoint: Adding Patient-Reported Outcomes to Medicare's Oncology Value-Based Payment Model, click here to view.
12. AHRQ Effective Program Updates
OPEN FOR COMMENT THROUGH MARCH 2: Technical Brief: Strategies for Patient, Family, and Caregiver Engagement. Click here to view.
Research Protocol: Pharmacologic and Nonpharmacologic Treatments of Posttraumatic Stress Disorder, click here to view.
2019 Year in Review—Accomplishments From the Evidence-based Practice Center Program, click here to view.
Research Protocol: Radiation Therapy for Brain Metastases: A Systematic Review, click here to view.
Research Protocol: Cervical Ripening in the Outpatient Setting, click here to view.
Research Protocol: Treatments for Acute Episodic Migraine, click here to view.
Research Protocol: Mixed Methods Review: Integrating Palliative Care With Chronic Disease Management in Ambulatory Care, click here to view.
Online Training for SRDR+ Now Available at the Evidence Synthesis Academy, click here to read more.
Research Protocol: Treatments for Acute Pain: A Systematic Review, click here to view.
Research Report: Registries for Evaluating Patient Outcomes: A User's Guide, click here to view.
A new report from the Pioneer Institute found that ICER's methods for value assessment are likely illegal under the Americans with Disabilities Act (ADA) if applied to government programs because they may decrease the availability of effective treatments for people living with a disability and increase the risk of institutionalization of certain people living with a mental disability. Pioneer Institute’s report predicts that a legal challenge to any adoption of ICER’s QALY methodology by state Medicaid systems on ADA grounds seems almost certain. Click here to read the report.
2. Massachusetts Finalizes Value Assessment Regulations
The Massachusetts Health Policy Commission finalized its regulations on how they will evaluate the “value” of medications as a part of the oversight and administration of prescription drug pricing. Advocates sent letters to the Commission and met in person with the commission to advocate for an explicit ban on the use of quality-adjusted life years (QALYs) in the regulation. The Commission ultimately did not include any provisions that would ban or even restrict or limit the use of QALY-based value assessments such as those conducted by the Institute for Clinical and Economic Review (ICER). And they opened the door to international reference pricing from countries that use QALYs. The final regulation did include new language on transparency and public input that may be useful in allowing patients and people with disabilities to make their voices heard in this process. But it was very disappointing that the Commission did nothing to acknowledge that QALYs discriminate, despite significant engagement.
In response to the Commission’s final regulation, PIPC joined patient organizations in Massachusetts in sending out a press statement. We stated, "Massachusetts has always been a leader in both health care and inclusion, and this regulation does not honor that legacy.” We also shared the National Council on Disability report on QALYs and the comment letter that advocates sent to the HPC.
3. ICER's Oral Semaglutide Model Exists in an Imaginary World
Writing in Innovations in Pharmacy, a University of Minnesota researcher writes that ICER's Oral Semaglutide model has little semblance to reality. "While this model differs from previous ICER models, the result is still a framework that constructs a so-called evidence base that fails the demarcation test. It is best described as pseudoscience. The model creates, by assumption, an imaginary world. The claims made for oral semaglutide by ICER should not be taken seriously by health care decision makers. The purpose of this commentary is to point to the limitations of the model with particular reference to the utility metrics employed, the resulting claims for quality adjusted life years (QALYs) and consequent recommendations for price discounting and affordability," he writes. Click here to read the article.
4. Patients and People with Disabilities Remain Concerned about Importing QALYs From Other Countries
PIPC Chairman Tony Coelho called on advocates to urge the Administration to follow the recommendations of the National Council on Disability opposing policies that would reference health care prices and policies established in other countries using discriminatory QALYs and similar summary metrics. Chairman Coelho stated, "Is the life of a person with a disability or chronic condition less valuable than the life of a person without one? There is renewed interest among lawmakers to tackle healthcare costs, specifically, the rising cost of prescription drugs. However, the Administration’s proposed International Pricing Index (IPI) model, an initiative proposed in 2018 that would arbitrarily base prices of American medications off the government set prices of medications in foreign countries, is deeply flawed. Yet, we may see some version of it reintroduced soon.” Click here to view the PIPC Chairman Blog. Click here to view a letter from advocates opposing an International Pricing Index. Click here for more information from experts.
5. Administration’s Medicaid Initiative Would Invite Closed Formularies, Implicate Use of QALYs
On January 30, 2020, the administration announced the Healthy Adult Opportunity (HAO) initiative allowing states to carry out demonstrations under section 1115(a)(2) of the Social Security Act (the Act) to provide "cost-effective" coverage using "flexible benefit designs.” Among its provisions, State Medicaid Directors were invited to "adopt a closed formulary in line with Essential Health Benefit (EHB) requirements (with special protections for individuals with HIV and behavioral health conditions)…This means that the open formulary requirements in section 1927 that apply to state plan drug coverage do not apply.” PIPC remains concerned that states will respond to increased opportunities to limit formularies and restrict access to therapies by referencing “cost effectiveness” studies that discriminate against patients and people with disabilities by using quality-adjusted life years (QALYs) and similar summary metrics. Click here to view the letter to State Medicaid Directors. Click here to view stakeholder comments opposing Tennessee’s waiver application. Click here to view a legislative template for states to advance patient-centered research in decision-making.
6. A Trusting Relationship is Crucial to Bridge the Gap Between Quality Metrics and Real Life
Doctors and patients need to have good, trusting relationships to bridge the gap between impersonal quality measures and reality, writes Dr. Jennifer DeVoe. "On the surface, the American College of Cardiology/American Heart Association guidelines for treating hypertension seemed straightforward....I did not have a good answer when she asked me, 'Dr DeVoe, given my history and tendency to experience side effects with every new medication we try, is this one going to be any different?' And where was the evidence-based guideline to answer her questions about whether spending money to buy this medication was more important than buying the healthy foods we had also recommended?" Patient-centered care and a longstanding doctor-patient relationship can begin to answer those questions. Click here to read the article (subscription needed).
7. ICER Releases Revised Value Assessment Framework for 2020
On Jan. 31, the Institute for Clinical and Economic Review (ICER) released its final revisions to its value assessment framework. The document describes changes in ICER’s process, including creating a formal process for incorporating new evidence that emerges after the assessment, standardizing cost-effectiveness thresholds across reviews, and increasing the incorporation of real-world evidence.
While the revised framework seeks to formalize a patient engagement program, the program falls short in committing to meaningful incorporation of patient perspectives and preferences into ICER’s reviews. ICER commits to work with patient groups throughout the process, but it does little to ensure that the patient voice is heard. The framework also makes it clear that economic analysis still maintains primacy at ICER and would be counted as more valuable than patient input.
In response to a request for feedback, stakeholders submitted comments on ICER’s draft 2020 value assessment framework last summer, but ICER failed to incorporate their suggestions in several important ways. First and foremost, ICER doubles down on its embrace of the discriminatory QALY measure and its cousin, the equal value of life years gained (evLYG) measure. Stakeholders also called on ICER to meaningfully incorporate patient preferences, but ICER instead opted to ceremonially consider patient input and not commit to patient-centered decision making.
The group did adopt a process to use real-world evidence (RWE) to update assessments of treatments that were approved under an accelerated pathway after the treatment has been on the market for two years. ICER does not, however, commit to using RWE in its base models, choosing to continue to conduct rushed assessments before appropriate data are available.
Unfortunately, ICER’s updated framework does not take a meaningful turn towards patient-centeredness. Under these revised guidelines, ICER will continue to use discriminatory measures to argue for reduced access to life-changing treatments. Click here to view the final value assessment framework. Click here to view PIPC's comments on the framework. Click here to read PIPC's Data Mine blog and see a table comparing PIPC's comments to ICER's final product.
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: Multiple Sclerosis patient's condition is worsening and she wants to be the first Australian to use a life-changing new drug. Parents of child with spinal muscular atrophy desperate to access gene therapy for their daughter before she turns two. Blood cancer is killing Australians, and access to treatment is critical.
- Canada: Crohn's patient struggles with forced transition to different medicine. Family of cystic fibrosis patient worries that without access to a new drug, she may not have much time left.
- New Zealand: Patients forced to pay taxes on medications that are denied to them by the government.
- United Kingdom: Young mother needs access to cystic fibrosis medications to continue to care for her children. NHS will not provide patients with adequate care so they resort to crowdfunding.
9. ICER's QALY-Based Study Topics: Hemophilia A, Beta Thalassemia, Acute Migraine, 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: Model Analysis Plan available. 4/1/2020: Draft Evidence Report and Draft Voting Questions. Meeting 6/30/2020: CTAF will convene to deliberate and vote on evidence presented in ICER's report on ulcerative colitis therapies.
- 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: Draft Evidence Report and Draft Voting Questions AVAILABLE, comment period open though 2/20/2020. 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.
- Acute Migraine: Midwest CEPAC met 1/23/2020 to review ICER's assessment of acute migraine treatments. 2/25/2020: Final Evidence Report.
- Non-Alcoholic Steatohepatitis: Model analysis plan available. 3/19/2020: Draft Evidence Report.
- Beta Thalassemia: Revised Scoping Document available. 3/11/2020: Research Protocol.
- Hemophilia A: Draft Scoping Document available. 2/25/2020: Revised Scoping Document. Meeting 8/3/2020: New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for hemophilia.
10. Upcoming Events and Webinars
PCORI Board of Governors Meeting
February 25, 2020
Click here for details.
PCORI Board of Governors Meeting
March 2, 2020
Click here for details.
Webinar: Pharmaceutical Contracting: Focus on Value-Based Contracting
March 3, 2020
Click here for details.
Market Access USA at eyeforpharma Philadelphia
April 15, 2020
Click here for details.
FT US Pharma and Biotech Summit 2020
May 14, 2020
Click here for details.
11. Medical Journal Articles
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.
Does the Use of Health Technology Assessment Have an Impact on the Utilisation of Health Care Resources? Evidence from Two European Countries, click here to view.
Patients, Clinicians and Researchers Working Together to Improve Cardiovascular Health: A Qualitative Study of Barriers and Priorities for Patient-Oriented Research, click here to view.
HTA Around the World: Broadening Our Understanding of Cross-Country Differences, click here to view.
Early Returns From the Era of Precision Medicine, click here to view.
Who Assigns Value in Value-Based Insurance Design?, click here to view.
Does the Institute for Clinical and Economic Review Revise Its Findings in Response to Industry Comments?, click here to view.
The Patient-Centered Outcomes Research Network Antibiotics and Childhood Growth Study: Implementing Patient Data Linkage, click here to view.
Viewpoint: Adding Patient-Reported Outcomes to Medicare's Oncology Value-Based Payment Model, click here to view.
12. AHRQ Effective Program Updates
OPEN FOR COMMENT THROUGH MARCH 2: Technical Brief: Strategies for Patient, Family, and Caregiver Engagement. Click here to view.
Research Protocol: Pharmacologic and Nonpharmacologic Treatments of Posttraumatic Stress Disorder, click here to view.
2019 Year in Review—Accomplishments From the Evidence-based Practice Center Program, click here to view.
Research Protocol: Radiation Therapy for Brain Metastases: A Systematic Review, click here to view.
Research Protocol: Cervical Ripening in the Outpatient Setting, click here to view.
Research Protocol: Treatments for Acute Episodic Migraine, click here to view.
Research Protocol: Mixed Methods Review: Integrating Palliative Care With Chronic Disease Management in Ambulatory Care, click here to view.
Online Training for SRDR+ Now Available at the Evidence Synthesis Academy, click here to read more.
Research Protocol: Treatments for Acute Pain: A Systematic Review, click here to view.
Research Report: Registries for Evaluating Patient Outcomes: A User's Guide, click here to view.
