CER Daily Newsfeed®
Articles
Video: The Importance of CER for Insurers (1/18, PCORI) “Janet McCauley says comparative effectiveness research can provide insurers with evidence about how treatments perform in real-world settings, informing decisions about clinical questions and the core benefits insurers offer.” View Video
ICER's ‘Unsupported Price Increase' Report Due This Fall (1/17, Cathy Kelly, Pink Sheet) reports “...In the report, the group proposes to review data on up to 13 drugs with list price increases of double the rate of the medical Consumer Price Index over a two-year period. ICER will review changes in the evidence base for these drugs, and report on whether it finds potential evidentiary support for price increases...‘These UPI reports are not intended to determine whether a price increase for a drug is fully justified by new clinical evidence or meets an ICER value-based price benchmark,’ ICER explained. ‘Instead, we will focus the analysis on whether or not substantial new evidence exists that could justify its price increase.’” Paid Subscription Required
ICER to Review Price Hikes to Prescription Drugs (1/18, The Center for Biosimilars) reports “...These drugs will be identified through research into the 100 drugs with the largest dollar sales in the United States and analysis of wholesale acquisition costs to these drugs over the past 24 months. ICER will determine which drugs have had a WAC increase that is twice the rate of the medical consumer price index or higher. Among that group of products, ICER will obtain net price information and determine changes over the prior 24 months and then rank the 10 drugs whose net price increases have had the largest impact on US healthcare spending over the past 2 years.” Full
UK: NICE OK for Bravtovi Skin Cancer Drug (1/18, Anna Smith, PharmaTimes) reports “The National Institute for Health and Care Excellence has recommended Pierre Fabre's Bravtovi (encorafenib) and Mektovi (binimetinib) combination as an option for some people with melanoma. This combination therapy will be available to adults with BRAF V600 mutation-positive melanoma, that cannot be removed surgically or that has spread to nearby organs.” Full
UK: Venetoclax Gets NICE Approval for Chronic Lymphocytic Leukaemia (1/18, Anna Smith, PharmaTimes) reports “The National Institute for Health and Care Excellence has recommended AbbVie's Venclyxto (venetoclax) to benefit thousands of adults with chronic lymphocytic leukaemia. The new treatment Venclyxto has been recommended with Rituxan (rituximab) for adults with relapsed or refractory chronic lymphocytic leukaemia.” Full
Press Releases
ICER to Assess Whether the Most Significant Prescription Drug Price Increases Are Supported by New Clinical Evidence (1/17, ICER Press Release) “[ICER] today posted a draft protocol describing a new annual analysis — an ICER ‘Unsupported Price Increase’ report — that will analyze significant prescription drug increases and determine whether or not new clinical evidence exists that could be used to support those increases. Public comment is being sought to inform a final version of the analytic protocol. Once finalized, the protocol will guide the development of the first of these annual reports, currently scheduled for October 2019.” Full
CMS Announces New Model to Lower Drug Prices in Medicare Part D and Transformative Updates to Existing Model for Medicare Advantage (1/18, CMS Press Release) “...The model for Medicare Advantage plans is an update to the Medicare Advantage Value-Based Insurance Design or ‘VBID’ model that CMS first launched in 2017. The model for Part D plans is called the Part D Payment Modernization model. These are both voluntary models to advance innovation – meaning certain Medicare Advantage and Part D plans can choose to participate, and patients can choose to enroll in participating plans. The models will be closely monitored, and if they clear certain thresholds for impact on quality, costs, and access to benefits, the models can be expanded in scope.” Full
Monoclonal Antibody Pembrolizumab Prolongs Survival in Patients with Squamous Cell Carcinoma (1/18, IQWiG Press Release) “...In an early benefit assessment, the German Institute for Quality and Efficiency in Health Care has now examined whether in these patients with PD-L1 expressing tumors, the monoclonal antibody pembrolizumab offers an added benefit over individual treatment of physician's choice. According to the findings, there is a hint of an added benefit, which is non-quantifiable, however.” Full
Journals
The Health Information Technology Special Issue: New Real-World Evidence and Practical Lessons Mary E. Reed, DrPH January 18, 2019, The American Journal of Managed Care AJMC
Reports
White Paper: Breaking Through the Hype of Real-World Evidence January 2019 Milliman
Unsupported Price Increase Assessment: Draft Protocol January 17, 2019 ICER
Events
Webinar: How Real-World Evidence Is Playing Out In The Real World February 14, 2019 11:00AM - 12:00PM EST Informa Pharma Intelligence
Articles
The U.S. Should Assess the Economic Value of Drugs Rather than Leave it up to Other Countries (1/17, William V. Padula, STAT News) comments “...It is important that such assessments of the economic value of drugs come from an independent but publicly funded body, like the Federal Reserve Board or the National Transportation Safety Board, to ensure transparency and impartiality. It should not be part of Medicare itself, but rather produce recommendations that could be used by Medicare, as well as by other stakeholders in drug pricing such as the Department of Veterans Affairs, state health programs, and commercial payers.” Full
Ocrelizumab Shows Superiority to or Comparability with Other Multiple Sclerosis Treatments (1/17, Erica Slaughter, MS, MD Magazine) reports “Efficacy of ocrelizumab, a humanized anti-CD20 monoclonal antibody, was found to be superior to or comparable with 14 other disease-modifying therapies currently approved by the European Medicines Agency forrelapsing multiple sclerosis in 7 network meta analyses. Ocrelizumab also demonstrated a similar safety profile to each of the DMTs in the analysis.” Full
Patient and Medical Groups Blast Trump Administration Drug Pricing Proposal in New Ad Campaign (1/17, Ike Swetlitz, STAT News) reports “...Carl Schmid, [The AIDS Institute’s] deputy executive director, explained that it's dangerous to require prior authorization because that may make it harder for a patient to get their drugs quickly. And step therapy can be harmful because it might force patients to take drugs that aren't appropriate for their specific type of HIV.” Full
Healthcare Data Are Plentiful Now. Why Isn't it Easier for Researchers to Use It? (1/17, Eli Richman, Fierce Healthcare) reports “Data from electronic health records was supposed to revolutionize research. But U.S. regulations around healthcare data are holding the market back, according to a recent study published in the Journal of the American Medical Informatics Association. Lack of interoperability between EHRs is only part of the problem, the study said. U.S. regulatory standards also aren't set up to enable evidence-based medicine and evidence-generating medicine data.” Full
A 21st-Century Lexicon of Value (1/17, Peter Pitts, Morning Consult) comments “...Value-based health care isn't about harmonization of decision-making; it's about harmonization of design and process. This is crucial as other HTA ‘harmonization’ efforts are heralding themselves as a quasi-nice ‘one-size-fits-all’ approach. ‘Value’ should be a constant, and political entities should make decisions based on constants — but decisions can be different based on different national needs, priorities and biases.” Full
UK: NICE Approval for Verzenios in Metastatic Breast Cancer (1/17, Anna Smith, PharmaTimes) reports “The National Institute for Health and Care Excellence has recommended Eli Lilly's Verzenios (abemaciclib) in combination with an aromatase inhibitor as treatment option for locally advanced or metastatic breast cancer.” Full
Journals
Systematic Review and Network Meta-Analysis Comparing Ocrelizumab with Other Treatments for Relapsing Multiple Sclerosis Rachael McCool, et al. January 2, 2019, Multiple Sclerosis and Related Disorders Multiple Sclerosis and Related Disorders
Financial Interests of Patient Organisations Contributing to Technology Assessment at England's National Institute for Health and Care Excellence: Policy Review Kate L Mandeville, et al. January 16, 2019, BMJ BMJ
Reimagining the Research-Practice Relationship: Policy Recommendations for Informatics-Enabled Evidence-Generation Across the US Health System Peter J Embi, et al. January 16, 2019, JAMIA Open JAMIA Open
Articles
Putting Patients' Goals And Needs At The Center Of Care: How Can We Do Better? (1/15, Tara Oakman and Lauren Gerlach, Health Affairs Blog) comments “...In recent years, for example, [the Robert Wood Johnson Foundation] has invested in research to understand what consumers value in health care, supported state and local consumer health advocacy organizations, developed principles of what measurement in health care could look like if it were truly patient centered, and supported research to better understand the information that people, especially low-income populations, are seeking in making decisions related to their coverage and care...Here, we highlight a few strategies discussed at the meeting that may be of particular interest to those in health philanthropy.” Full
ICYMI: New Study Reinforces Dangers of Proposed IPI Model (1/15, Nicole Longo, PhRMA: The Catalyst) comments “...According to IHS Markit, Americans diagnosed with locally advanced or metastatic NSCLC between 2006 and 2017 would have lost half of their survival gains if the United States adopted health technology assessment frameworks like those used by foreign governments to determine access to care – meaning half of the life years they gained from new treatments...Study after study underscores that the administration should stop this harmful proposal and instead pursue reforms that strengthen market competition and the move toward value-based care, which can control costs while supporting better outcomes for patients.” Full
67% of MSSP ACOs May Be High-Revenue Under Pathways to Success (1/16, Jacqueline LaPointe, RevCycle Intelligence) reports “Over two-thirds of Medicare Shared Savings Program accountable care organizations would qualify as high-revenue organizations under the new Pathways to Success initiative, a recent Leavitt Partners analysis uncovered.” Full
BMS Gets New OK for Opdivo/Yervoy in Kidney Cancer (1/16, Phil Taylor, PharmaPhorum) reports “Bristol-Myers Squibb has claimed a new approval for its immuno-oncology combination of Opdivo and Yervoy, getting a green light from the European Medicines Agency for its use in renal cell carcinoma, the most common form of kidney cancer.” Full
UK: NICE Endorsement Means More Donor Livers Could Be Used for Transplantation (1/16, Anna Smith, PharmaTimes) reports “...NICE has issued final guidance which recommends the perfusion machine, potentially increasing the number of organs viable for transplant, saving more lives and reducing liver transplant waiting lists. The procedure will be used under special arrangements as more data is gathered into its efficacy, although no safety concerns have been identified.” Full
Canada: Gene Therapy Drug Kymriah Should Be Publicly Covered If Manufacturer Drops Price, Report Recommends (1/15, Kelly Grant, The Globe and Mail) reports “A groundbreaking gene therapy for cancer could cost the Canadian health-care system more than $400-million over three years, according to a new report that recommends the drug be publicly covered, provided the manufacturer drops the price...The new report, released Tuesday, comes from an expert panel convened by the Canadian Agency for Drugs and Technologies in Health...” Full
Press Releases
OM1 Rheumatoid Arthritis Registry Reaches More Than 100,000 Patients Prospectively Followed with Deep Clinical Data (1/16, OM1 Press Release) “...The OM1 RA registry is a continually updating linked database of more than 100,000 patients prospectively followed with deep clinical, claims, patient reported, laboratory and other data, such as measures of disease activity and treatment response. The registry provides unique insights into patient journeys, comparative effectiveness, provider behavior and competitive intelligence that is not possible with existing smaller and less complete registries and datasets.” Full
Journals
Willingness to Pay for Medical Treatments in Chronic Diseases: A Multicountry Survey of Patients and Physicians Etienne Audureau, et al. January 16, 2019, Journal of Comparative Effectiveness Research Journal of Comparative Effectiveness Research
Reports
Tisagenlecleucel (Kymriah) for Pediatric Acute Lymphoblastic Leukemia and Diffuse Large B-Cell Lymphoma January 15, 2019 CADTH
Events
Webinar: Let’s Get Real: How Will the FDA Framework for Real-World Evidence Impact You? January 30, 2019 1:00 - 2:00PM ET National Pharmaceutical Council
Articles
Copay Coupons Help (A Little) with CV Drug Adherence but Not Outcomes: Study (1/15, Eric Sagonowsky, Fierce Pharma) reports “...The study looked at more than 11,000 patients who'd suffered a heart attack and found that the group who received coupons had a 3.3% increase in medication persistence after 1 year versus their counterparts, while they had no benefit in health outcomes. At the same time, the coupons also seemed to make doctors more comfortable prescribing guideline-preferred options that carry higher costs, according to the study, which was published in the Journal of the American Medical Association.” Full
Here's Why Many Prescription Drugs in the US Cost So Much-And It's Not Innovation or Improvement (1/14, Yoni Blumberg, CNBC) reports “...In the past, pharmaceutical companies have attributed high prices to innovation, arguing that new and improved drugs are naturally more expensive. But a new study published in the journal Health Affairs complicates that idea. It suggests that, largely, costs have gone up because companies are raising the price of drugs that are already available.” Full
UK: SMC OK's Four Meds for NHS Use, Including Roche's Perjeta (1/15, Anna Smith, PharmaTimes) reports “...The drug, already approved by NICE, will be made available to HER2-positive metastatic breast cancer patients, also known as stage IV or secondary breast cancer...Pfizer's Xeljanz (tofacitinib) was recommended for the treatment of psoriatic arthritis, a painful and debilitating condition which causes red scaly patches on the skin and inflammation of the joints. MSD's Steglatro (ertugliflozin) was accepted for the treatment of type II diabetes as part of a package of treatment which includes diet and exercise to improve glycaemic control. Novo Nordisk's Ozempic (semaglutide) was also endorsed for the treatment of type II diabetes alongside a diet and exercise regime.” Full
UK's NICE Under Fire For New HTA Fees (1/15, Francesca Bruce, Pink Sheet) reports “...Fees for health technology appraisals conducted by NICE, the National Institute for Health and Care Excellence in the UK, could be bigger than the revenues that some medicines with smaller indications bring in, Paul Catchpole, director of value and access at the Association of the British Pharmaceutical Industry has warned.” Paid Subscription Required
Journals
The Contribution Of New Product Entry Versus Existing Product Inflation In The Rising Costs Of Drugs Inmaculada Hernandez, et al. January 2019, Health Affairs Health Affairs
A Cost-Utility Analysis of Dulaglutide Versus Insulin Glargine as Third-Line Therapy for Type 2 Diabetes in Canada Richard F Pollock, et al. January 15, 2019, Journal of Comparative Effectiveness Research Journal of Comparative Effectiveness Research
Gelatin Tannate Versus Other Antidiarrheal Medication in Children with Acute Gastroenteritis: A Retrospective, Observational Study Elena D Serban & Mihai Manolache January 15, 2019, Journal of Comparative Effectiveness Research Journal of Comparative Effectiveness Research
Cost-Effectiveness of Buprenorphine–Naloxone Versus Extended-Release Naltrexone to Prevent Opioid Relapse Sean M. Murphy, PhD, et al. January 15, 2019, Annals of Internal Medicine Annals of Internal Medicine
Testing Novel Payment and Delivery Approaches Through the Veterans Health Administration's New Center for Innovation Steven D. Pizer, PhD, et al. January 15, 2019, Annals of Internal Medicine Annals of Internal Medicine
Articles
How Costs for 7 Chronic Conditions Changed in 20 Years (1/11, Morgan Haefner, Becker's Hospital Review) reports “...For the study, researchers with RTI Health Solutions in Durham, N.C., and the National Pharmaceutical Council examined whether medical intervention spending on seven chronic conditions during the past two decades has been a good investment. The study authors analyzed national-level data from the CDC, the Global Burden of Disease Study and the Medical Expenditure Panel Survey...‘The study had two key findings: First, for some conditions, dollars spent on medical care can be a source of high value creation, and such investment should continue,’ the authors wrote. ‘Second, there is significant variability in value across diseases, which highlights the need for disease-specific spending approaches.’” Full
Mayo Clinic, Wake Forest Baptist Health Join Clinical Research Network (1/14, Greg Slabodkin, Health Data Management) reports “...The healthcare organizations are the latest to join the Mid-South Clinical Data Research Network, founded in 2014 with funding from [PCORI], authorized by Congress to fund research aimed at improving the health of entire populations.In welcoming the two new member organizations—Mayo Clinic, with locations in Arizona, Florida and Minnesota, and Wake Forest Baptist Health, an affiliate of Wake Forest University, located in Winston-Salem, N.C.—the clinical research network will now be called the Stakeholder Technology and Research Clinical Research Network.” Full
The Future Of Real World Data In Clinical Trials (1/11, Clinical Informatics News) reports “Real-world evidence is unarguably rich, but it's also noisy and can be difficult to parse. But those challenges are worth overcoming says Cathy Critchlow...Critchlow is vice president and head of Amgen's Center for Observational Research...On behalf of Clinical Informatics News, Marina Filshtinsky spoke with Critchlow about how real-world evidence is changing clinical research, where it's gaining ground, and what work is left to be done.” Full
A QALY Is A QALY Is A QALY, Or Is It? (1/14, Joshua Cohen, Forbes.com) comments “...Besides the arbitrariness of the numerator in cost-per-QALY thresholds, a central question facing ICER and CVS Caremark is whether a drug's value should depend on not only a measure such as QALYs, but also on which disease or population is being treated, which implies that a QALY may not always have the same weight. In other words, should ICER invoke higher (i.e., more lenient) cost-per-QALY gained cost-effectiveness thresholds in some areas (say, cancer or rare diseases, or young patients) than others and, if so, on what basis?” Full
Oversight Launches Major Drug Pricing Investigation (1/14, Nathaniel Weixel, The Hill) reports “...[Elijah Cummings (D-Md.)] sent letters to a dozen different companies seeking detailed information and documents about how the companies price their medications. He is billing it as one of the broadest drug pricing investigations in decades...‘The goals of this investigation are to determine why drug companies are increasing prices so dramatically, how drug companies are using the proceeds, and what steps can be taken to reduce prescription drug prices,’ he said.” Full
Press Releases
ICER Posts Draft Scoping Document for the Assessment of Treatments for Duchenne Muscular Dystrophy (1/11, ICER Press Release) “...ICER's assessment will focus on eteplirsen (Exondys 51™, Sarepta Therapeutics), golodirsen (Sarepta Therapeutics), and deflazacort (Emflaza®, PTC Therapeutics). Eteplirsen was approved by the FDA in September of 2016, deflazacort was approved in February of 2017, and golodirsen has an anticipated FDA approval decision expected in the middle of 2019.” Full
Journals
Health Economics and Outcomes Research (HEOR) Knowledge Needs Assessment for Latin America Hughes VS, et al. January 8, 2019, Value in Health Regional Issues PubMed
Reports
DMD: Draft Scoping Document January 11, 2019 ICER
Articles
Unproven Medical Treatments Cost Us Lives and Money. Let Research Tell Us What Works. (1/11, Ezekiel J. Emanuel and Justin E. Bekelman, USA Today) comments “...[I]nsurers should pay for unproven treatments only when received through studies. Specifically, while the intervention is being tested, insurers and government programs should set the price of new treatments equal to the current standard of care, a model known as reference pricing. The price should be reset only if research ultimately shows better (or worse) outcomes. This approach promotes both access and innovation. The University of Pennsylvania already does this for proton therapy and has arranged with some insurers to review cases within 24 hours.” Full
FDA to Beef Up Sentinel Postmarket Safety System (1/11, Susan Kelly, BioPharma DIVE) reports “...In a statement announcing the agency's strategy for expanding Sentinel, FDA Commissioner Scott Gottlieb and Gerald Dal Pan, director of the agency's Office of Surveillance and Epidemiology in the Center for Drug Evaluation and Research, said greater use of real world evidence can improve patient outcomes and lower the cost of medical product development...In addition to developing approaches to identify unexpected safety concerns, the agency is working with the National Institutes of Health Collaboratory Distributed Research Network and the Patient Centered Outcomes Research Network to develop the Sentinel data infrastructure for other public health purposes.” Full
NAACOS and ACO Leaders Express Distress Over Just-Announced MSSP Application Deadline (1/11, Mark Hagland, Healthcare Informatics) reports “...NAACOS stated that ‘[CMS] late Wednesday announced applications to participate in the new Pathways to Success accountable care organization program will be due February 19, two months after the agency published a nearly 267-page rule overhauling the Medicare Shared Savings Program. In response, the National Association of ACOs is calling on CMS to give ACOs till later in March to understand the complex changes and determine participation options with affiliated doctors, hospitals, and other providers before committing to high-stakes decisions.’” Full
Ireland: Updated Guidelines to Improve HTAs Accuracy (1/11, Valerie Ryan, Irish Medical Times) reports “...Dr Conor Teljeur, HIQA's Chief Scientist, said: ‘A considerable proportion of the health budget each year is invested in new technologies and programmes. Funding decisions should be based on sound evidence, and there should be reassurance that those decisions will deliver care that is both patient-focused and achieves value for money. Health technology assessment provides a framework for a comprehensive evaluation of the evidence. A critical element is how clinical outcomes are evaluated in a HTA, and these guidelines show how this can be achieved,’ he added.” Full
Journals
Measuring the Value of MRI: Comparative Effectiveness & Outcomes Research Stella K. Kang MD, MSc January 10, 2019, Journal of Magnetic Resonance Imaging Wiley
Do Interventions That Address Patient Cost-Sharing Improve Adherence to Prescription Drugs? A Systematic Review of Recently Published Studies Sensharma A, Yabroff KR. January 10, 2019, Expert Review of PharmacoEconomics and Outcomes Research PubMed
Requests for Comment
Tools and Technologies for Registry Interoperability January 11 - February 8, 2019 AHRQ