In This Week’s Issue:
1. PIPC Patient Blog: Migraine Patients Deserve Access to Needed Treatments, click here to read the blog.
2. Briefing: The Need for Safeguards at CMMI, see details below.
3. Save the Date: Sickle Cell Patient Advocacy Summit, see details below.
4. Headache and Migraine Patients Speak Out on ICER's Acute Migraine Report, see details below.
5. Washington State Senate Votes to Give Prescription Drug Affordability Board Power to Use Cost Thresholds Without Patient Representation, see details below.
6. A Conversation About Cystic Fibrosis, Caregiving and Innovation, click here to read the interview.
7. PCORI Advisory Panel Head on Continued Dedication to Rare Disease Research, click here to read the blog post.
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, Acute Migraine, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, Bladder Cancer, Opioid Treatments, 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. PIPC Patient Blog: Migraine Patients Deserve Access to Needed Treatments, click here to read the blog.
2. Briefing: The Need for Safeguards at CMMI, see details below.
3. Save the Date: Sickle Cell Patient Advocacy Summit, see details below.
4. Headache and Migraine Patients Speak Out on ICER's Acute Migraine Report, see details below.
5. Washington State Senate Votes to Give Prescription Drug Affordability Board Power to Use Cost Thresholds Without Patient Representation, see details below.
6. A Conversation About Cystic Fibrosis, Caregiving and Innovation, click here to read the interview.
7. PCORI Advisory Panel Head on Continued Dedication to Rare Disease Research, click here to read the blog post.
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, Acute Migraine, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, Bladder Cancer, Opioid Treatments, 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. PIPC Patient Blog: Migraine Patients Deserve Access to Needed Treatments
Jamie Sanders, who lives with intractable migraine, penned a PIPC patient blog about her experience and the importance of access to treatments for patients like her. "Patients like me should immediately access new treatments. To ensure this happens, it is vital that doctors’ assessments be widely accepted in determining which patients have failed or can’t use older medicines. Migraine disease is an incredibly personal journey and the clinician-patient relationship should be primary, not the academics over at ICER or their recommendations for prior authorization, in making treatment decisions," she wrote. Click here to read the blog.
2. Briefing: The Need for Safeguards at CMMI
The Healthcare Leadership Council will hold a briefing on Thursday, March 12 on Capitol Hill to discuss the importance of guardrails at the Center for Medicare and Medicaid Innovation to protect patients. They will discuss the “Strengthening Innovation in Medicare and Medicaid Act,” (H.R. 5741), legislation that would make changes to CMMI to improve transparency and ensure CMMI operates as originally intended. H.R. 5741 provides safeguards for CMMI to ensure CMMI demonstrations are patient-focused and benefit Medicare and Medicaid beneficiaries. PIPC Executive Director Sara van Geertruyden will participate on a panel discussing the issue.
The briefing will be held on Thursday, March 12, 2020 at 2:30 p.m. in the Longworth House Office Building in Washington, D.C. Please RSVP to [email protected] if you would like to attend.
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. 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.
5. 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.
6. 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.
7. 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.
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, 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:
10. 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.
Webinar: Communicating Real-World Evidence in the Medical Literature
March 18, 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.
Advancing Value-Based Care
May 14, 2020
Click here for details.
11. 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.
12. 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.
Jamie Sanders, who lives with intractable migraine, penned a PIPC patient blog about her experience and the importance of access to treatments for patients like her. "Patients like me should immediately access new treatments. To ensure this happens, it is vital that doctors’ assessments be widely accepted in determining which patients have failed or can’t use older medicines. Migraine disease is an incredibly personal journey and the clinician-patient relationship should be primary, not the academics over at ICER or their recommendations for prior authorization, in making treatment decisions," she wrote. Click here to read the blog.
2. Briefing: The Need for Safeguards at CMMI
The Healthcare Leadership Council will hold a briefing on Thursday, March 12 on Capitol Hill to discuss the importance of guardrails at the Center for Medicare and Medicaid Innovation to protect patients. They will discuss the “Strengthening Innovation in Medicare and Medicaid Act,” (H.R. 5741), legislation that would make changes to CMMI to improve transparency and ensure CMMI operates as originally intended. H.R. 5741 provides safeguards for CMMI to ensure CMMI demonstrations are patient-focused and benefit Medicare and Medicaid beneficiaries. PIPC Executive Director Sara van Geertruyden will participate on a panel discussing the issue.
The briefing will be held on Thursday, March 12, 2020 at 2:30 p.m. in the Longworth House Office Building in Washington, D.C. Please RSVP to [email protected] if you would like to attend.
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. 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.
5. 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.
6. 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.
7. 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.
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: Parents fight for access to gene therapy for their daughter with SMA, while the government delays on approval. Hemophilia patient receives gene therapy for the disease that killed his brothers through a clinical trial. Eczema sufferers call for drug to be covered by government. Click here to read more. Irish man with cystic fibrosis denied re-entry to Australia, and his father believes it is because he may be expensive to the government now that Orkambi is covered in the country. Click here and here to read more.
- Canada: Man says that government's "games" on drug prices cost his daughter his life. "CF warrior" continues to fight for Health Canada to cover orphan drugs. Woman who lost her sister to CF is fighting to get the government to allow access to lifesaving drug. Parents of kids with rare diseases feel let down by government.
- New Zealand: Auckland man fights for wife's cancer drug to be administered through public system. MP treated by MS drug that was only recently approved by government. Pharmac should not deny medicines simply because they benefit a small number of people.
- United Kingdom: NHS refuses to make an 11 year old drug available to patients.
9. ICER's QALY-Based Study Topics: Hemophilia A, 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.
- 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: INDEFINITELY POSTPONED.
- 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.
10. 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.
Webinar: Communicating Real-World Evidence in the Medical Literature
March 18, 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.
Advancing Value-Based Care
May 14, 2020
Click here for details.
11. 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.
12. 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.
