CER Daily Newsfeed®
Articles
It’s Time to Get Health Care Value Assessment Right (4/15, Jennifer Bright and Richard H. Chapman, STAT) comments “...An important step forward in value assessment is the release of principles for capturing the full range of patient-centered outcomes data by the Patient-Centered Outcomes Research...These new principles call for identifying outcomes that are important to patients and caregivers, as well as to other stakeholders; collecting data such as the potential burden and economic impact of interventions if they are appropriate and relevant to a study’s clinical aims; and conducting certain types of economic analyses as part of research studies.” Full
Providers Call for Another Chance to Sign up for Direct Contracting Model (4/15, Robert King, Fierce Healthcare) reports “...The National Association of Accountable Care Organizations was also worried about the future of the Next Generation ACO model, which calls on providers to take on a greater degree of financial risk than ACOs in the Medicare Shared Services Program...Because of the decision to end a second application period, the ACOs in Next Gen will not have the ability to participate in a higher-risk model and instead must go into the MSSP. ‘Next Generation ACOs need clarification on what happens to their participation in high-risk, accountable care models,’ said Clif Gaus, president and CEO of NAACOS...” Full
Alexion's Pricey Ultomiris Wins Backing from England's Cost Watchdogs Thanks to Confidential Discount (4/15, Angus Liu, Fierce Pharma) reports “Alexion has added a nice piece to Ultomiris’ growth potential ahead of its planned merger with AstraZeneca, scoring backing from England’s drug cost watchdog in a key indication. The C5 inhibitor now has full coverage on the National Health Service for the treatment of paroxysmal nocturnal hemoglobinuria, thanks to a recommendation from the National Institute for Health and Care Excellence. It can now be used in patients with high disease activity who have remained stable on the drug’s predecessor Soliris for at least six months.” Full
Requests for Comment
NPC Comments on Medicare Program; Medicare Coverage of Innovative Technology (MCIT) and Definition of “Reasonable and Necessary” April 16, 2021 National Pharmaceutical Council
Events
Reminder: Financial Times US Pharma and Biotech Summit May 5 - 6, 2021 National Pharmaceutical Council
Articles
Real-World HCRU, Costs Associated With Ibrutinib vs Bendamustine Plus Rituximab in CLL (4/14, Marta Rybczynski, Journal of Clinical Pathways) reports “...Due to higher rates of inpatient admissions, longer lengths of stays, and more ER visits for ibrutinib vs bendamustine plus rituximab, Debra Irwin, IBM Watson Health, Cambridge, MA, and colleagues suggest that more real-world effectiveness research needs to be conducted. ‘Given the differences in costs, it is important to further examine the impact these healthcare expenditures have on the cost-effectiveness of [ibrutinib monotherapy] first line treatment,’ concluded Dr Irwin and colleagues.” Full
Hospices See Rising Volume, Billing Challenges in Medicare Advantage (4/13, Holly Vossel, Hospice News) reports “Value-based care has opened up a different world to hospices beginning this year. More than 90 days into the value-based insurance design (VBID) demonstration, hospices are seeing rising patient volumes while expanding service offerings and adapting to new billing processes.” Full
UK: NICE Publishes New Guidance on Chronic Pain Management (4/14, Lucy Parsons, PharmaTimes) reports “...The guidance, published by NICE last week, has placed emphasis on shared decision making by recommending patients be put at the centre of their care. NICE recommends that a care and support plan be developed for people suffering with chronic pain, which should be based on the effects of pain on day-to-day activities, as well as individual’s preferences, abilities and goals.” Full
Press Releases
Children's Hospital Los Angeles Receives $6.1M to Study Treatment of Youth Anxiety (4/14, Children's Hospital Los Angeles Press Release) “Funding for the study comes from the Patient-Centered Outcomes Research Institute, an independent, nonprofit organization authorized by Congress in 2010 to fund research that will provide patients, their caregivers and clinicians with the evidence-based information needed to make better-informed health care decisions. In collaboration with some of the nation's leading experts in youth anxiety, Dr. Peterson will compare treatment strategies. ‘There hasn't yet been a head-to-head comparison for treatment strategies in real-world settings,’ says Dr. Peterson. ‘We are excited to be able to lay this foundation and finally answer these questions.’” Full
Better Therapeutics Launches Real World Evidence Study of Prescription Digital Therapeutic for Type 2 Diabetes (4/14, Better Therapeutics Press Release) “Better Therapeutics, Inc...today announced the commencement of a real-world evidence study with Steward Health Care to evaluate the long-term effectiveness and cost of care impact associated with the use of BT-001 for the treatment of type 2 diabetes. BT-001 is an investigational, prescription digital therapeutic that delivers a novel form of cognitive behavioral therapy to patients with uncontrolled type 2 diabetes.” Full
Articles
Accelerated Approval, Real World Evidence Pilot From ICER, Aetion Picks HAE As First Therapeutic Area (4/12, Sarah Karlin-Smith, Pink Sheet) reports “...The Institute for Clinical and Economic Review picked three hereditary angioedema treatments for the first of its pilot program with Aetion to generate new real world evidence on prescription drugs approved by the US Food and Drug Administration under accelerated approval so that it can better understand the value of the products.” Paid Subscription Required
Research Institute Taps into RWE to Assess Angioedema Therapies (4/13, Jenni Spinner, Outsourcing Pharma) reports “...Carolyn Magill, CEO of Aetion, said the collaboration will help further ICER's commendable mission to continually look for ways that life sciences professionals can make better use of RWE. ‘Pilots like this will help inform efforts of HTA organizations around the world in their quest to evaluate how and when to base their decisions on RWE while advancing how we as a health care industry—and a society—determine value?,’ Magill said.” Full
Press Releases
ICER Publishes Research Protocol for Assessing the Unsupported Price Increases on Prescription Drugs That Occurred in 2020 (4/12, ICER Press Release) “The Institute for Clinical and Economic Review today posted a Protocol outlining how we will conduct our next analysis of ‘Unsupported Price Increases’ (UPI) among prescription drugs within the US market. This initiative will examine the most significant drug-price increases that occurred in 2020 and determine whether new clinical evidence exists that could be used to support those increases. Previously, ICER has published UPI reports for increases that occurred during the two-year period of 2017-2018, and for 2019.” Full
Reports
Unsupported Price Increases Occurring in 2020 April 12, 2021 ICER
Events
Advisory Panel on Healthcare Delivery and Disparities Research Spring 2021 Meeting May 6, 2021 11:00AM - 4:30PM EDT PCORI
Articles
Bringing Real World Evidence into the Care Setting (4/9, Ken Tarkoff, Managed Healthcare Executive) comments “...Ultimately, RWE has the potential to bridge the divide between evidence and practice due to the power of where the insight is generated and its applicability to the real world.Rather than an amalgam of patients treated in a wide variety of modalities (i.e., claims), newer forms of RWE are developed from the record of the care decisions documented by a specific set of providers, for their particular patient populations. The insights include the full patient journey, not just the initial diagnosis and first treatments (i.e., registries), which allows them to be used to understand ongoing patient care choices and outcomes. Data supporting this newer RWE typically updates daily, allowing insights to be reflective of real-time care decisions, rather than care that was received months ago. And finally, these insights are also fully identified under the provider’s technology umbrella, allowing providers to bring insights back directly to patients currently undergoing treatment.” Full
COA Panel Says Confidence Rests on Real-World Data (4/9, Tony Hagen, The Center for Biosimilars) reports “...[University of Chicago Medicine] still wants to see more real-world evidence that shows biosimilars can stand the test of time outside of clinical trials performed in limited numbers of patients for the purposes of FDA approval, she said. [Sonia T. Oskouei, PharmD, BCMAS, DPLA] agreed. ‘The conversation has definitely evolved with a desire for more real world evidence and studies around biosimilar to biosimilar switching. With 5 trastuzumab biosimilars on the market now for example, and more in the pipeline, this dynamic is becoming something that providers must navigate.’” Full
Though Not In Skinny Budget, Drug Pricing Expected In Biden's Full Budget (4/9, John Wilkerson, InsideHealthPolicy) reports “...It is unclear whether Democrats will combine President Joe Biden's $2 trillion-plus physical infrastructure proposal with his health care proposal, which is expected to include drug pricing offsets, or whether they will tackle the infrastructure and health care proposals separately.” Paid Subscription Required
Press Releases
ICER to Use Aetion Observational RWE to Update Value Assessment of Treatments for Hereditary Angioedema (4/12, ICER Press Release) “...ICER's updated assessment will leverage observational RWE analyzed by Aetion, as part of the ongoing collaboration between the two organizations, and consistent with ICER's commitment to expand use of RWE to complement other sources of information used in its value assessments. Aetion researchers will use Optum's de-identified Clinformatics® Data Mart, Commercial and Medicare Advantage claims database, to generate the RWE. In addition to this specific pilot to use RWE to update ICER assessments completed more than 24 months ago, Aetion has analyzed RWE to support ICER's earlier assessment of treatments for sickle cell disease.” Full
Articles
John Robinson Recruits a Small Band of Array Vets to Kickstart Discovery at Cogent; Syntimmune Co-Founder Takes Charge at Arch Oncology (4/9, Alex Hoffman, et al., Endpoints News) reports “...The search is over for a new leader at the National Pharmaceutical Council, with John O’Brien taking on the roles of president and CEO at the organization effective June 1. Robert Dubois had held those titles on an interim basis when Dan Leonard stepped aside last September after a 12-year run, and Dubois will ease back into his duties as EVP and CSO. O’Brien was senior advisor to former HHS Secretary Alex Azar and deputy assistant secretary of planning and evaluation (health policy). He’s also been VP of public policy for CareFirst BlueCross BlueShield.” Full
To Sustain The Value Movement, Make Next Generation ACOs A Permanent Option (4/9, Jeff Micklos, et al., Health Affairs Blog) comments “...The road to advanced value-based payment arrangements requires many checkpoints along the way. Next Gen ACOs serve as a valuable destination on the journey to full risk, and the model evaluation should be revisited to ensure a proper focus and analysis in support of making the program a permanent option for Medicare providers. The Biden administration should prioritize model expansion when reviewing the Next Gen ACO demonstration with the objective of improving the model over discarding it.” Full
Real-World Evidence Supports Efficacy of Palbociclib Plus Letrozole in HR+/HER2– Breast Cancer (4/9, Audrey Sternberg, Cancer Network) reports “...‘Real-world evidence is woven into the fabric of how we innovate and advance care for patients with breast cancer, supporting our randomized clinical trials,’ Chris Boshoff, MD, PhD, Chief Development Officer, Oncology, Pfizer Global Product Development, said...‘With more than 6 years of patient experience, a positive benefit-risk profile, strong clinical data, and robust real-world data, the totality of evidence solidifies the role of [palbociclib] plus endocrine therapy as a treatment for patients with [hormone receptor–positive, HER2-negative] metastatic breast cancer.’” Full
UK: NICE 'No' for Opdivo/Yervoy Lung Cancer Combo (4/9, Selina McKee, PharmaTimes) reports “...In an appraisal consultation document, NICE notes that clinical trial evidence suggests people who have the Opdivo/Yervoy regimen live longer than those who have platinum-doublet chemotherapy. However, in the economic model it is uncertain how long the effect lasts, and whether people having it live longer depending on their PD-L1 status and the type of NSCLC they have, it said.” Full
Journals
Comparative Effectiveness of Multiple Different First-Line Treatment Regimens for Helicobacter pylori Infection: A Network Meta-Analysis Theodore Rokkas, et al. April 8, 2021, Gastroenterology Gastroenterology
Articles
Unprecedented Development of Covid-19 Vaccines, Treatments Points to Biopharma’s Future (4/8, Andrew Bolt and Sonal Shah, STAT Plus) comments “...Real-world evidence is becoming increasingly valuable as the health care system gains experience with Covid-19. The Covid-19 Evidence Accelerator, launched by the Reagan-Udall Foundation in collaboration with the Friends of Cancer Research, has become a venue for major data organizations, government and academic researchers, and health systems to collectively use real-world evidence to help answer questions related to the management of Covid-19. The collaboration is also running repeat analyses to compare results using different techniques and data sources.” Paid Subscription Required
Press Releases
ICER to Assess Treatment for Hypertrophic Cardiomyopathy (4/8, ICER Press Release) “The Institute for Clinical and Economic Review (ICER) announced today that it will assess the comparative clinical effectiveness and value of mavacamten (MyoKardia and Bristol-Myers Squibb) for the treatment of hypertrophic cardiomyopathy. An FDA decision on mavacamten is expected in mid to late 2021.” Full
ADAPTABLE, A PCORnet® Demonstration Project, Offers A Template For Future Pragmatic Studies (4/7, PCORnet Press Release) “Two manuscripts recently published in peer-reviewed journals offer researchers a glimpse into the opportunities and challenges for novel pragmatic trials like Aspirin Dosing: A Patient-Centric Trial Assessing Benefits and Long-term Effectiveness (ADAPTABLE), a demonstration project of PCORnet®, the National Patient-Centered Clinical Research Network. The publications highlight separate, but equally important, topics of interest in pragmatic research: maximizing clinician engagement and navigating institutional review board challenges.” Full
Xcenda’s FormularyDecisions Launches Resource Hub for Biosimilar Products (4/8, AmerisourceBergen Press Release) “...‘The biosimilar category holds incredible promise, and the market for these products is on an upward trajectory. But in order to reach the full potential of these medicines, it’s critical to provide payers with access to in-depth product information that can help them assess value and make reimbursement decisions,’ said Sean McGowan, senior director of Biosimilars at AmerisourceBergen. ‘At AmerisourceBergen, we are working with all stakeholders—from biosimilar manufacturers to payers and providers—to facilitate more robust communication and education to help reduce barriers that restrict product success and patient access.’” Full
Journals
Reducing Low-Value Care and Improving Health Care Value Allison H. Oakes, PhD; Thomas R. Radomski, MD, MS April 8, 2021, JAMA JAMA