1. Video: Risk of Modeling Other Countries’ Drug Pricing: How a Canadian Child is Forced to Get Sicker to Access Treatment, click here to watch the video.
2. ICER’s Concern for Patients: Where’s the Beef?, click here to read the op-ed.
3. PIPC Urges House to Protect Patients and People with Disabilities in Title 1 of New House Bill, see below for details.
4. Proposed Changes to ICER Framework Available for Comment, click here to view the changes and to comment and see below for more.
5. PIPC Comments on ICER's Type 2 Diabetes Draft Evidence Report, click here to read the letter.
6. Liver Cancer Hill Briefing, see below for details.
7. New PQA Patient Engagement in Quality Measurement Rubric, click here to read more and to view the rubric.
8. How ICER Discriminates Against People with Rare Diseases, click here to read the article.
9. PCORI News and Updates, see details below.
10. Cancer Support Community is Hosting a Summit on Utilization Management, click here to learn more and register.
11. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
12. ICER Studies: Acute Migraine, Type 2 Diabetes, Arthritis, Cardiovascular Disease, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, click here to provide patient input.
13. Upcoming Events and Webinars, see details below.
14. Medical Journal Articles, see details below.
15. AHRQ Effective Program Updates, see details below.
Access to treatments is a constant struggle for patients in countries like Canada, where access is limited based on cost effectiveness or assessments of average clinical benefit. We are sharing a video developed in Canada with a mother, Sandy, and her daughter, Laura, who has cystic fibrosis. Sandy and her daughter Laura spoke honestly and freely about their experience with the Canadian health system. It is a heartbreaking story of the requirement to get sicker in order to access a new treatment she needs due to government utilization management for drugs, e.g. fail first policies. As our country considers modeling international drug pricing policies, it demonstrates the inherent risk for patient access to care. Click here to watch the video.
2. ICER’s Concern for Patients: Where’s the Beef?
CancerCare published an op-ed in STAT News about how ICER gives lip service to patient experiences without truly incorporating patient preferences into its analyses. "We all want to pay for value. No one — no patient, no caregiver, no clinician — wants to pay more for a treatment than the treatment is worth. The real questions are who is deciding that worth and are they basing it on their own values or the values of those for whom the treatment is intended. ICER describes itself as the 'nation’s drug pricing watchdog.' But it seems to us that it is protecting the insurance companies that pay for treatments. If the organization truly wants to become the watchdog for the nation — which includes the people who use health care — it needs to research and quantify the value of treatments as they relate to the people who need them," wrote CancerCare's Patricia Goldsmith and Carole Florman. Click here to read the op-ed.
3. PIPC Urges House to Protect Patients and People with Disabilities in Title 1 of New House Bill
Legislation has been introduced in the House of Representatives which includes certain provisions concerning patients and people with disabilities in Title 1. This section of the bill takes an approach that is entrenched in controversial comparative clinical and cost effectiveness standards that force patients and people with disabilities into “one-size-fits-all” decisions about their health. PIPC Chairman Tony Coelho expressed concern that these provisions place hard-fought existing patient protections under serious threat. First, it authorizes national determinations of value to be based on comparative or cost effectiveness standards that ignore differences in patient needs and discriminate against vulnerable subgroups of patients and people with disabilities, opening the door to reliance on assessments like those conducted by the Institute for Clinical and Economic Review (ICER). Second, it relies on policy decisions made in other countries that we know establish prices based on discriminatory cost-effectiveness standards (such as Quality-Adjusted Life Years or QALYs) and similar average metrics. Third, it lacks any reference to existing statutory safeguards against misuse of QALYs and similar average metrics. PIPC supports policies to improve care and affordability for patients, adopt and extend safeguards in current statute that prohibit application of discriminatory cost-effectiveness thresholds in Medicare (whether done overtly by Medicare or covertly via “comparative effectiveness analysis” or reference to foreign countries’ use of these thresholds) and codify criteria for patient-centeredness called for in CMMI’s statute. Click here to view the bill. Click here to view prior stakeholder letter on referencing International pricing. Click here for our explainer of Title I of the bill. Click here to view information on Value Our Health.
4. Proposed Changes to ICER Framework Available for Comment
ICER has released its proposed changes to its value assessment framework for 2020. Public comment is open on the proposed changes through October 18, 2019. Click here to view the changes and to comment. Click here to view the comment letter signed by over 30 stakeholders during the initial open comment period. Click here and here to view videos about ICER's methodology. Please contact firstname.lastname@example.org to review and sign on to comments on the updated ICER draft 2020 framework developed in collaboration with patient organizations.
5. PIPC Comments on ICER's Type 2 Diabetes Draft Evidence Report
In a letter to the Institute for Clinical and Economic Review (ICER), Partnership to Improve Patient Care (PIPC) Chairman Tony Coelho provided feedback on ICER's draft evidence report for Type 2 Diabetes Treatments. Chairman Coelho criticized ICER for omitting quality of life data deemed valuable by patients, pointing out that the institute continues to rely on faulty data in its assessments. The letter also condemns ICER for ignoring the heterogeneity of Type 2 Diabetes Patients in its report. “ICER continues to use a flawed methodology, ignoring the reality of heterogeneous patient populations and quality of life outcomes that matter to patients in favor of data that easily crosswalks into the discriminatory QALY metric,” wrote Chairman Coelho. “We urge ICER to consider alternative methodologies that will foster improved health care decisions for individual patients.” Click here to read the letter.
6. Liver Cancer Hill Briefing
Global Liver Institute, American Gastroenterological Association, Hepatitis B Foundation, Hep B United, AAPCHO, NVHR, and NASTAD are hosting a Congressional briefing titled, "Liver Cancer Drivers and Disparities" on Thursday, October 31 from 10:30 a.m. - 12:00 p.m. Learn from our nation’s leading clinicians and patient advocates about the steps needed to combat the most rapidly rising cancer since 1980, and how response strategies must begin with modernizing the systems in place to better meet the needs of impacted populations. The briefing will be held in room 121 of the Cannon House Office Building. Please RSVP to email@example.com if you are interested in attending.
7. New PQA Patient Engagement in Quality Measurement Rubric
The Pharmacy Quality Alliance (PQA) released a new rubric to help incorporate the patient perspective throughout the quality measure life cycle. It describes the levels of engagement in quality measurement across patient-centered principles and provides recommended activities to improve patient engagement. "The quality measurement experts, patient advocates, caregivers and patients who created this tool have made a tremendous contribution to our national effort to transform our health care system into a patient-centered enterprise," said Laura Cranston, PQA's CEO. Click here to read more and to view the rubric.
8. How ICER Discriminates Against People with Rare Diseases
ICER's value assessment methodology inherently discriminates against people with rare diseases, writes Randy Rutta, board chairman of the Partnership to Fight Chronic Disease and former president and CEO of Easterseals, in the San Francisco Examiner. "If health plans follow ICER’s recommendations and limit or stop covering rare disease medicines, drug companies will be under pressure to stop developing those treatments. After all, it’d make no sense to spend billions creating a medicine if health plans won’t pay for it and patients won’t receive it. Scientists are currently developing more than 560 medicines to treat rare diseases. Many include potential cures for rare genetic diseases that disable and kill. That research will very likely come to a screeching halt — causing patients to lose access to lifesaving drugs — if insurers fail to recognize the faults in ICER’s reasoning and continue to adopt the group’s increasingly disputed recommendations," he writes. Click here to read the article.
9. PCORI News and Updates
- New Executive Director: PCORI announced that Dr. Josie Briggs will step into the Executive Director role in an interim capacity effective November 1. Dr. Briggs is currently the Editor in Chief of the Journal of the American Society of Nephrology, the leading journal in kidney care. Her career includes nearly 20 years in leadership at the NIH, including key positions with NIH's Common Fund Health Systems Research Collaboratory and its Precision Medicine Initiative. Dr. Briggs while serve while a search committee works to find a permanent Executive Director.
- PCORI Engagement Awards: PCORI is accepting Letters of Inquiry for two Engagement Award special project funding announcements. Up to $1.6 million in funding will be awarded in two categories: Accelerating the Adoption of Tools and Resources, and Community Convening around Patient-Centered Outcomes Research. Click here to learn more.
- Advisory Panel Openings: PCORI is seeking nominations and applications for new members of its Advisory Panel on Rare Disease. Members help guide PCORI's work in patient-centered research in rare diseases and engagement with the rare disease research community. Click here to learn more and apply or submit a nomination.
10. Cancer Support Community Event on Utilization Management
The Cancer Support Community is holding a summit to discuss the future of utilization management strategies and practices. The event, which will be held on October 23, 2019 in Washington, D.C., will feature panel discussions, presentations, and patient perspectives on the trends, policies, and values that will shape utilization management going forward. Click here to learn more and register.
11. 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: Australian mother lobbies government for access to a broader range of preventative vaccines.
- New Zealand: Pharmac decides to fund a handful of new cancer and MS treatments, but the use criteria are 'inhumanely strict.' MS patients feel abandoned by Pharmac's coverage decisions. Lung cancer patient pushes for greater access to medicines.
- United Kingdom: 5-year-old cancer patient sees very positive results from treatment that NHS would not cover. A number of individuals waiting for access to PrEP contract HIV.
12. ICER Studies: Acute Migraine, Type 2 Diabetes, Arthritis, Cardiovascular Disease, 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: Draft Scoping Document available, comment period OPEN through 10/18/2019.
- Cystic Fibrosis: Draft Scoping Document available. Comment period OPEN through 10/21/2019. 10/29/2019: Revised Scoping Document.
- Sickle Cell Disease: Revised Scoping Document available. 11/5/2019: Research Protocol. Meeting 3/26/2020: New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for sickle cell disease.
- Arthritis: 10/11/2019: Revised Draft Evidence Report and Voting Questions. Comment period OPEN through 11/8/2019. Meeting 11/19/2019: CTAF to an update to its 2017 rheumatoid arthritis assessment.
- Type 2 Diabetes: 10/31/2019: Voting Questions and Evidence Report. Meeting 11/14/2019: The New England CEPAC will convene to deliberate on ICER's review of oral semaglutide for the treatment of type 2 diabetes.
- Cardiovascular Disease : 10/17/2019: Final Evidence Report and Meeting Summary. Midwest CEPAC met 9/26/2019 to deliberate and vote on ICER's report on evidence presented in ICER's report on additive CVD therapies. 10/17/2019: Final Evidence Report and Meeting Summary.
- Unsupported Price Increase Assessment: Final Report available.
- Acute Migraine: Model Analysis Plan available. 11/7/2019: Draft Evidence Report. Meeting 1/23/2020: Midwest CEPAC to review ICER's assessment of acute migraine treatments
- Valuing A Cure Project: 11/15/2019: Revised White Paper.
- 2020 Value Assessment Framework: Proposed changes AVAILABLE. Comment period open through 10/18/2019.
- Non-Alcoholic Steatohepatitis: Open Input Period through 10/25/2019.
13. Upcoming Events and Webinars
Webinar: The Roadmap To A Value Model That Supports Breakthrough Innovation
October 18, 2019
Click here for details.
Cancer Support Community Utilization Management Summit
October 23, 2019
Click here for details.
Webinar: Applying Real-World Evidence to Regulatory and Drug Development Challenges
October 25, 2019
Click here for details.
Personalized Medicine Conference
November 13-14, 2019
Click here for details.
2019 AUCD Annual Meeting
November 17-20, 2019
Click here for details.
FT US Pharma and Biotech Summit 2020
May 14, 2020
Click here for details.
14. Medical Journal Articles
Feasibility of Using Real-World Data to Replicate Clinical Trial Evidence, click here to view.
Variation in US Private Health Plans' Coverage of Orphan Drugs, click here to view.
Incorporating Patient Perspectives and Transparency for Patient-Centered Value Assessment, click here to view.
Accounts from developers of generic health state utility instruments explain why they produce different QALYs: A qualitative study, click here to view.
The Shared Decision-Making Process in the Pharmacological Management of Depression, click here to view.
Training Patients to Review Scientific Reports for the Patient-centered Outcomes Research Institute: An Observational Study, click here to view.
Patient Reported Outcomes Have Arrived: A Practical Overview for Clinicians in Using Patient Reported Outcomes in Oncology, click here to view.
Prioritizing Evidence-Based Interventions for Dissemination and Implementation Investments: AHRQ's Model and Experience, click here to view.
Observational Studies Have a Critical Role to Play in Cancer Comparative Effectiveness Research, click here to view.
High-Quality Evidence to Inform Clinical Practice, click here to view.
An Ethical Analysis of Coverage With Evidence Development, click here to view.
15. AHRQ Effective Program Updates
Research Protocol: Management of Colonic Diverticulitis, click here to view.
Research Protocol: Therapies for Clinically Localized Prostate Cancer, click here to view.
Research Report: Web Interactive Presentation of EPC Reports: A Foray Into Interactive Reports, click here to view.
Systematic Review: Antipsychotics for the Prevention and Treatment of Delirium, click here to view.
Systematic Review: Comparative Effectiveness of Analgesics To Reduce Acute Pain in the Prehospital Setting, click here to view.
Blog: Innovations to Foster Evidence-based Care in Learning Health Systems: Ideas from the AHRQ Evidence-based Practice Centers, click here to view.
Research Protocol: Prevention, Diagnosis, and Management of Opioids, Opioid Misuse and Opioid Use Disorder in Older Adults, click here to view.
AHRQ EPC Pilot Projects Summary: Improving Health Systems' Access to High Quality Evidence, click here to view.
Research Protocol: Care Interventions for People With Dementia (PWD) and Their Caregivers, click here to view.
Research Protocol: Impact of Community Health Worker Certification on Workforce and Service Delivery for Asthma and Other Selected Chronic Diseases, click here to view.
AHRQ EPC Program Helps Health Systems Use Evidence, click here to view.