In This Week’s Issue:
1. Headache and Migraine Patients Speak Out on ICER's Acute Migraine Report, see details below.
2. Washington State Senate Votes to Give Prescription Drug Affordability Board Power to Use Cost Thresholds Without Patient Representation, see details below.
3. Save the Date: Sickle Cell Patient Advocacy Summit, see details below.
4. A Conversation About Cystic Fibrosis, Caregiving and Innovation, click here to read the interview.
5. PCORI Advisory Panel Head on Continued Dedication to Rare Disease Research, click here to read the blog post.
6. ICER Releases Cystic Fibrosis Draft Evidence Report: Learn More, see details below.
7. PIPC Submits Comment Letter to ICER on Draft Evidence Report for Sickle Cell Disease, click here to read the letter.
8. Pioneer Institute: ICER's Methods Are Likely Illegal under the ADA, click here to read the report.
9. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
10. ICER's QALY-Based Study Topics: Hemophilia A, Beta Thalassemia, Acute Migraine, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, Bladder Cancer, Opioid Treatments, click here to provide patient input.
11. Upcoming Events and Webinars, see details below.
12. Medical Journal Articles, see details below.
13. AHRQ Effective Program Updates, see details below.
1. Headache and Migraine Patients Speak Out on ICER's Acute Migraine Report, see details below.
2. Washington State Senate Votes to Give Prescription Drug Affordability Board Power to Use Cost Thresholds Without Patient Representation, see details below.
3. Save the Date: Sickle Cell Patient Advocacy Summit, see details below.
4. A Conversation About Cystic Fibrosis, Caregiving and Innovation, click here to read the interview.
5. PCORI Advisory Panel Head on Continued Dedication to Rare Disease Research, click here to read the blog post.
6. ICER Releases Cystic Fibrosis Draft Evidence Report: Learn More, see details below.
7. PIPC Submits Comment Letter to ICER on Draft Evidence Report for Sickle Cell Disease, click here to read the letter.
8. Pioneer Institute: ICER's Methods Are Likely Illegal under the ADA, click here to read the report.
9. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
10. ICER's QALY-Based Study Topics: Hemophilia A, Beta Thalassemia, Acute Migraine, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, Bladder Cancer, Opioid Treatments, click here to provide patient input.
11. Upcoming Events and Webinars, see details below.
12. Medical Journal Articles, see details below.
13. AHRQ Effective Program Updates, see details below.
1. Headache and Migraine Patients Speak Out on ICER's Acute Migraine Report
In response to ICER's revision to its acute migraine report reversing course on its determination that certain migraine drugs were not cost effective, migraine advocates continued to point out that ICER undervalues drugs' benefits and does not address the real economic costs of migraines, not to mention the patient perspective. The Headache and Migraine Policy Forum's Lindsay Vedinieks was quoted in multiple publications calling for ICER to revise its report taking into account societal factors to better reflect the actual impact that new medications can have on the lives of migraine patients. "Waiting is painful for migraine patients," she said, adding that there is an issue whenever an individual with migraine issues has to wait for care. Click here to read the Politico article. Click here to read the Inside Health Policy article. Click here to read the Bloomberg Law article.
2. Washington State Senate Votes to Give Prescription Drug Affordability Board Power to Use Cost Thresholds Without Patient Representation
On February 18, 2020, the Washington State Senate passed SBB 6088, a bill establishing the prescription drug affordability board to identify prescription drugs priced above a certain threshold, with authority to conduct cost reviews of drugs and set upper payment limits for state purchasers. The legislation does not bar the Board from utilizing discriminatory quality-adjusted life years (QALYs) as the metric for determining whether treatments exceed certain thresholds. The Board also does not include representation from patients or people with disabilities, only "five members who have expertise in health care economics or clinical medicine appointed by the governor.” The legislation heads toward state House consideration this week, highlighting the continued challenge that patient and disability advocates face in combatting discrimination in state Medicaid programs. Click here to view the bill summary. Click here to view value assessment principles supported by patients and people with disabilities. Click here to learn more about state activities related to QALYs and here to view a state legislative template supported by Value Our Health.
3. Save the Date: Sickle Cell Patient Advocacy Summit
Leading sickle cell disease groups will be hosting a patient advocacy summit highlighting the patient perspective on value of new sickle cell disease treatments. The event will be on March 25 from 5-8pm in Boston, Massachusetts at the Museum of African American History. It will also be livestreamed. More details to follow.
4. A Conversation About Cystic Fibrosis, Caregiving and Innovation
NPC's E.V.I.dently Today blog published an interview with Lisa Yourman, an advocate and mother of two adult children with cystic fibrosis. "I am humbled by the innovation of new medication because it has been such a game changer for CF patients and their families. All of the medications and the new medical devices have extended their lives and their quality of life. They have benefited from having mucus thinners, inhaled antibiotics that go directly into the lungs, and respiratory vests that keep those infections at bay. For Sarah, the new insulins, continuous glucose monitors and insulin pumps have helped her tremendously in managing her diabetes. Keeping blood sugars under control also helps prevent lung infections. The newest innovations in medicines are changing the underlying nature of CF itself. Today, Sarah is working full time, enjoying life, and planning her wedding. Without these innovations, our lives would be very different. There is great hope now in the CF community because of the life-changing research and development," she said in the interview. Click here to read the interview.
5. PCORI Advisory Panel Head on Continued Dedication to Rare Disease Research
Dr. Scott Berns, chair of PCORI's Advisory Panel on Rare Disease, wrote this week about PCORI's continued dedication to researching rare diseases. "I think there’s an opportunity for the panel to help galvanize the rare disease field. Personally, I have a two-year opportunity as Chair to connect the dots from my personal and career experiences with the dots at PCORI. As someone who has been personally affected by rare disease and now also works to improve systems of care for those living with sickle cell disease, I am intimately aware of the need for urgent action and increased national momentum," he wrote. Click here to read the blog post.
6. ICER Releases Cystic Fibrosis Draft Evidence Report: Learn More
On February 20, 2020, the Institute for Clinical Economic Review (ICER) released its draft evidence report for cystic fibrosis treatments, concluding that "CFTR modulator therapies plus best supportive care substantially improve patient health outcomes compared to best supportive care” but exceeded "commonly used cost-effectiveness thresholds.” Stakeholders are able to comment until March 18, 2020. To assist advocates, Cystic Fibrosis Research, Inc. invited Ari Ne’eman to conduct a webinar explaining the quality-adjusted life year methodology that is being used by ICER to determine the “cost effectiveness” of new treatments for cystic fibrosis. Click here to view the webinar. Click here to view the draft evidence report.
7. PIPC Submits Comment Letter to ICER on Draft Evidence Report for Sickle Cell Disease
In a letter to the Institute for Clinical and Economic Review (ICER), Partnership to Improve Patient Care (PIPC) Chairman Tony Coelho offered feedback on ICER’s draft evidence report for Sickle Cell Disease (SCD) treatments. Chairman Coelho expressed concerns with ICER’s use of the QALY metric to assess SCD treatments, saying that the QALY is a particularly inappropriate method for evaluating any value accrued from interventions aimed at its alleviation. The letter also criticizes ICER for choosing to use claims data instead of listening to input from patient and clinician stakeholders. “ICER risks doing the SCD community a disservice by using the QALY, a metric highly inappropriate for the assessment of SCD treatments, and by failing to listen to the community about factors like standard of care and outcomes that matter to patients that should be meaningfully incorporated into its model,” wrote Chairman Coelho. Click here to read the letter.
8. 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.
9. 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.
10. ICER's QALY-Based Study Topics: Hemophilia A, Beta Thalassemia, Acute Migraine, 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. Please note the following upcoming formal ICER deadlines per their website:
11. Upcoming Events and Webinars
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.
PCORI Board of Governors Meeting
March 24, 2020
Click here for details.
Webinar: Adding Depth to Observational Research through Data Linkage
April 8, 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.
12. Medical Journal Articles
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.
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.
13. AHRQ Effective Program Updates
EPC Program Concludes Series on Improving Health Systems Use of Evidence Reviews. Click here to view.
Research Protocol: Prehospital Airway Management: A Systematic Review. Click here to view.
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.
In response to ICER's revision to its acute migraine report reversing course on its determination that certain migraine drugs were not cost effective, migraine advocates continued to point out that ICER undervalues drugs' benefits and does not address the real economic costs of migraines, not to mention the patient perspective. The Headache and Migraine Policy Forum's Lindsay Vedinieks was quoted in multiple publications calling for ICER to revise its report taking into account societal factors to better reflect the actual impact that new medications can have on the lives of migraine patients. "Waiting is painful for migraine patients," she said, adding that there is an issue whenever an individual with migraine issues has to wait for care. Click here to read the Politico article. Click here to read the Inside Health Policy article. Click here to read the Bloomberg Law article.
2. Washington State Senate Votes to Give Prescription Drug Affordability Board Power to Use Cost Thresholds Without Patient Representation
On February 18, 2020, the Washington State Senate passed SBB 6088, a bill establishing the prescription drug affordability board to identify prescription drugs priced above a certain threshold, with authority to conduct cost reviews of drugs and set upper payment limits for state purchasers. The legislation does not bar the Board from utilizing discriminatory quality-adjusted life years (QALYs) as the metric for determining whether treatments exceed certain thresholds. The Board also does not include representation from patients or people with disabilities, only "five members who have expertise in health care economics or clinical medicine appointed by the governor.” The legislation heads toward state House consideration this week, highlighting the continued challenge that patient and disability advocates face in combatting discrimination in state Medicaid programs. Click here to view the bill summary. Click here to view value assessment principles supported by patients and people with disabilities. Click here to learn more about state activities related to QALYs and here to view a state legislative template supported by Value Our Health.
3. Save the Date: Sickle Cell Patient Advocacy Summit
Leading sickle cell disease groups will be hosting a patient advocacy summit highlighting the patient perspective on value of new sickle cell disease treatments. The event will be on March 25 from 5-8pm in Boston, Massachusetts at the Museum of African American History. It will also be livestreamed. More details to follow.
4. A Conversation About Cystic Fibrosis, Caregiving and Innovation
NPC's E.V.I.dently Today blog published an interview with Lisa Yourman, an advocate and mother of two adult children with cystic fibrosis. "I am humbled by the innovation of new medication because it has been such a game changer for CF patients and their families. All of the medications and the new medical devices have extended their lives and their quality of life. They have benefited from having mucus thinners, inhaled antibiotics that go directly into the lungs, and respiratory vests that keep those infections at bay. For Sarah, the new insulins, continuous glucose monitors and insulin pumps have helped her tremendously in managing her diabetes. Keeping blood sugars under control also helps prevent lung infections. The newest innovations in medicines are changing the underlying nature of CF itself. Today, Sarah is working full time, enjoying life, and planning her wedding. Without these innovations, our lives would be very different. There is great hope now in the CF community because of the life-changing research and development," she said in the interview. Click here to read the interview.
5. PCORI Advisory Panel Head on Continued Dedication to Rare Disease Research
Dr. Scott Berns, chair of PCORI's Advisory Panel on Rare Disease, wrote this week about PCORI's continued dedication to researching rare diseases. "I think there’s an opportunity for the panel to help galvanize the rare disease field. Personally, I have a two-year opportunity as Chair to connect the dots from my personal and career experiences with the dots at PCORI. As someone who has been personally affected by rare disease and now also works to improve systems of care for those living with sickle cell disease, I am intimately aware of the need for urgent action and increased national momentum," he wrote. Click here to read the blog post.
6. ICER Releases Cystic Fibrosis Draft Evidence Report: Learn More
On February 20, 2020, the Institute for Clinical Economic Review (ICER) released its draft evidence report for cystic fibrosis treatments, concluding that "CFTR modulator therapies plus best supportive care substantially improve patient health outcomes compared to best supportive care” but exceeded "commonly used cost-effectiveness thresholds.” Stakeholders are able to comment until March 18, 2020. To assist advocates, Cystic Fibrosis Research, Inc. invited Ari Ne’eman to conduct a webinar explaining the quality-adjusted life year methodology that is being used by ICER to determine the “cost effectiveness” of new treatments for cystic fibrosis. Click here to view the webinar. Click here to view the draft evidence report.
7. PIPC Submits Comment Letter to ICER on Draft Evidence Report for Sickle Cell Disease
In a letter to the Institute for Clinical and Economic Review (ICER), Partnership to Improve Patient Care (PIPC) Chairman Tony Coelho offered feedback on ICER’s draft evidence report for Sickle Cell Disease (SCD) treatments. Chairman Coelho expressed concerns with ICER’s use of the QALY metric to assess SCD treatments, saying that the QALY is a particularly inappropriate method for evaluating any value accrued from interventions aimed at its alleviation. The letter also criticizes ICER for choosing to use claims data instead of listening to input from patient and clinician stakeholders. “ICER risks doing the SCD community a disservice by using the QALY, a metric highly inappropriate for the assessment of SCD treatments, and by failing to listen to the community about factors like standard of care and outcomes that matter to patients that should be meaningfully incorporated into its model,” wrote Chairman Coelho. Click here to read the letter.
8. 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.
9. 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.
- Canada: Death of cystic fibrosis patient shines light on Canada's drug access issues. Access to arthritis medication in Canada largely depends on where you live.
- New Zealand: Unfunded drug is working for one patient's brain tumor. Man who needed an unfunded drug for his blood cancer dies after long fight with the disease.
- United Kingdom: Child dies while fundraising to get brain cancer treatment in the U.S. -- a treatment that is not available in the UK. Mother of child with cystic fibrosis says that NICE's decisions on a cystic fibrosis drug will come too late for her son to benefit, even if NICE decides to fund it. Woman with cancer launches appeal for an unfunded "last option" treatment.
10. ICER's QALY-Based Study Topics: Hemophilia A, Beta Thalassemia, Acute Migraine, 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. 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: Draft Evidence Report and Draft Voting Questions AVAILABLE, comment period OPEN through 3/18/2020. 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: 3/12/2020: Evidence Report and Responses to Comments. 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: Final Evidence Report and Meeting Summary Available.
- Non-Alcoholic Steatohepatitis: Model analysis plan available. 3/19/2020: Draft Evidence Report. Meeting 5/28/2020: The Midwest CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for NASH.
- Beta Thalassemia: Revised Scoping Document available. 3/11/2020: Research Protocol.
- Hemophilia A: Revised Scoping Document available. 3/30/2020: Research Protocol. Meeting 8/3/2020: New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for hemophilia.
- Bladder Cancer: Open input period through 3/10/2020. 3/13/2020: Draft Scoping Document.
- Opioids: Digital Apps: Open input period through 3/10/2020. 3/13/2020: Draft Scoping Document.
- Opioids: Supervised Injection Centers: Open input period, no end date specified.
11. Upcoming Events and Webinars
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.
PCORI Board of Governors Meeting
March 24, 2020
Click here for details.
Webinar: Adding Depth to Observational Research through Data Linkage
April 8, 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.
12. Medical Journal Articles
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.
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.
13. AHRQ Effective Program Updates
EPC Program Concludes Series on Improving Health Systems Use of Evidence Reviews. Click here to view.
Research Protocol: Prehospital Airway Management: A Systematic Review. Click here to view.
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.
