CER Daily Newsfeed®
Articles
Will ICER's Response to Attacks on the QALY Quiet the Critics? (12/18, Joshua T. Cohen, PhD, Daniel A. Ollendorf, PhD and Peter J. Neumann, ScD, Center for the Evaluation of Value and Risk in Health) comments “...QALYs have limitations as we have noted elsewhere, but they account for key outcome attributes, including length and quality of life, and they provide payers and decision makers a way to assess tradeoffs between clinical benefits and resource use (by comparing cost-effectiveness ratios to a benchmark or to each other). ISPOR's recent Special Task Force on Value Frameworks recommends[9] additional research on methods to augment the QALY, and CEVR will shortly be embarking on such an effort. However, while that research continues, we see ICER's approach -– i.e., using QALYs as an input to a broad discussion of clinical benefit, context, and value to all stakeholders in the health care system – as reasonable.” Full
Report Assesses Degree of Alignment Between ASCO, ESMO Oncology Frameworks (12/17, Allison Inserro, The American Journal of Managed Care) reports “A joint assessment comparing the results of value frameworks created by the American Society of Clinical Oncology and the European Society for Medical Oncology found that the frameworks produce comparable measures of the clinical benefits of new cancer therapies in roughly two-thirds of more than 100 drug comparisons examined. The findings were published Monday in ASCO's Journal of Clinical Oncology.” Full
Novartis Braces for a Battle over the Cost Effectiveness of a Gene Therapy (12/17, Ed Silverman, STAT Plus) reports “...The drug maker held a media briefing on Monday to discuss a draft report due later this week from the Institute for Clinical and Economic Review on Zolgensma, which was developed to treat spinal muscular atrophy, a rare and inherited neurodegenerative disease that in its most fatal form causes children to die by the time they turn 2 years old. Trials showed a single dose corrected a defective gene.” Paid Subscription Required
Peer Exchange: The Efficacy and Safety of CDK4/6 Inhibitors (12/18, The American Journal of Managed Care) “A comparative evaluation of the safety and efficacy of 3 cyclin-dependent kinases 4 and 6 (CDK4/6) inhibitors: ribociclib, palbociclib, and abemaciclib.” View Video
To Advance Public and Patient Engagement in Research, We Must Demonstrate Its Value (12/17, Kristin L. Carman, PhD, MA and Laura Forsythe, PhD, MPH, The PCORI Blog) comments “...[W]e were thrilled to be among an international set of authors of a recent editorial in The BMJ, one of the few major professional journals to involve patients in its peer-review process. As the editorial's title clearly indicates, if we're serious about public and patient engagement, we need to be serious about its evaluation...This editorial responds to an ever-growing body of evidence that the approach we are taking is shifting the broader landscape of health research, and the care it informs, to be more patient centered. It is part of a package of recent articles in The BMJ, including one research paper, two commentaries, and our editorial.” Full
Training the EHR to Speak When a Child Can't (12/18, Mohana Ravindranath, Politico) reports “...With initial funding from the Patient-Centered Outcomes Research Institute, Berger is designing a system in the hospital's Cerner EHR that could search a patient's record for indicators of abuse, such as past injuries or a history of unexplained symptoms. Her longer-term goal is a predictive algorithm that pulls public data sets — school records, prior abuse reports or a parent's criminal history — to identify children likely to suffer abuse in the future.” Full
2018 in Review: Governments Start Taking Access Seriously (12/18, George Underwood, PharmaPhorum) comments “...As the UK continued to fiercely debate its future relationship with the EU, the European Commission set out proposals this year for more HTA collaboration and harmonisation between the Union's member states.It would in effect make bodies such as Germany's Institute for Quality and Efficiency in Healthcare and the French Economic Committee for Health Products redundant, replacing divergent Health Technology Assessment systems across Europe.” Full
Press Releases
TriNetX Real-World Evidence Platform Validates Outcomes of Randomized Clinical Trials (12/18, TriNetX Press Release) “...‘The results showed that this type of analysis using real-world data could replicate the results coming from much more complex and costly studies,’ said Manfred Paul Stapff, MD, PhD, Chief Medical Officer of TriNetX who led the study. ‘The study validates our methods and the quality of data in our network. It also shows that TriNetX is perfectly positioned to take advantage of the FDA's recently announced framework evaluating ways to incorporate greater use of real-world evidence in drug development.’” Full
Journals
Threats to the Value of Health Technology Assessment: Qualitative Evidence from Canada and Poland Wranik WD, et al. December 7, 2018, Health Policy PubMed
Comparative Assessment of Clinical Benefit Using the ESMO-Magnitude of Clinical Benefit Scale Version 1.1 and the ASCO Value Framework Net Health Benefit Score Nathan I. Cherny, et al. December 17, 2018, Journal of Clinical Oncology Journal of Clinical Oncology
Is the National Institute for Health and Care Excellence (NICE) in England More "Innovation-Friendly" than the Federal Joint Committee (G-BA) in Germany? Schaefer R, Schlander M December 17, 2018, Expert Review of Pharmacoeconomics & Outcomes Research PubMed
Cost-Effectiveness of Buprenorphine–Naloxone Versus Extended-Release Naltrexone to Prevent Opioid Relapse Sean M. Murphy, PhD, et al. December 18, 2018, Annals of Internal Medicine Annals of Internal Medicine
Establishing the Effectiveness of Procedural Interventions: The Limited Role of Randomized Trials Christopher J. D. Wallis, MD, PhD, et al. December 18, 2018, JAMA JAMA
Interventions to Improve Medication Adherence: A Review Vinay Kini, MD, MSHP; P. Michael Ho, MD, PhD December 18, 2018, JAMA JAMA
Observational Studies of Treatment Effectiveness: Worthwhile or Worthless? Sharma M, Nazareth I, Petersen I December 18, 2018, Clinical Epidemiology Dove Press
Articles
In Value Assessment, Additional Voices Bring Much-Needed Alternative Perspectives (12/17, National Pharmaceutical Council: E.V.I.dently Today) comments “...The National Pharmaceutical Council has been keeping a close eye on value assessment frameworks for several years, engaging with developers on ways to improve their methodologies, involve patient organizations and use a broad range of evidence-based considerations, as outlined in our Guiding Practices for Patient-Centered Value Assessment. As noted in our Guiding Practices, there is no single answer to a value assessment. Just as a one-size-fits-all approach doesn't work for treating individual patients, one framework alone won't be able to address all health care questions or end-user needs. Alternative approaches to value assessment frameworks brought by groups like IVI bring important new perspectives to the discussion on value in health care.” Full
A Cost-Saving Medical Revolution If We Can Finance It (12/17, Wayne Winegarden, Forbes.com) comments “...In response to these high costs, ICER attempts to define whether a new therapy will be cost-effective in order to justify which therapies should be covered, and which should not. Such an approach is bound to under-value the benefits that these new gene therapies will create. Further, a focus on arbitrary cost thresholds, by definition, encourages greater restrictions on patients' access to these revolutionary gene therapies. If you are the patient whose devastating disease can be cured by a new gene therapy, then the purpose of having insurance was to enable access to these medicines.” Full
Thinking Big: Low-Value Care Beyond Choosing Wisely Lists (12/17, Jose Benuzillo, M.A., M.S. and Lucy A. Savitz, Ph.D., M.B.A., AcademyHealth Blog) comments “...The problem of overuse, unnecessary care, or low-value care is increasingly a focus of the national health policy conversation. Estimates suggest that as much as one-third of health care spending in the United States is of low-value or wasteful. Many factors contribute to the problem, including increased patient demand, information asymmetry, perverse financial incentives for providers, and a culture of ‘more is better than less.’ Despite our growing knowledge, defining and measuring low-value care remains challenging and has led to important questions about the care we deliver.” Full
Redefining Quality Through a Patient-Centered Approach (12/14, Anne Zink, MD, KevinMD.com) comments “...[W]e need to redefine the term "quality" so it aligns more closely with the latest evidence-based medicine. Quality may mean giving the right antibiotics in a timely fashion to a patient with sepsis, but may also mean not giving antibiotics at all because the patient's sore throat is likely from a virus.” Full
Docs Don't Routinely Report Patient Symptoms in EHRs (12/17, Greg Slabodkin, Health Data Management) reports “...The study, funded by the Patient-Centered Outcomes Research Institute, revealed that patients want to tell their physicians about their symptoms and would be willing to do so through a computerized patient reporting system. However, researchers reveal that patients interviewed for the study were reluctant to report symptoms if they perceive busy clinicians will not use that information to improve care.” Full
Video: Dr Robert Daly Explains the Importance of Updating Pathways With New Data (12/16, The American Journal of Managed Care) “As new breakthrough therapies are approved, clinical pathways have to be kept updated in order for patients to get the best treatments, said Robert Daly, MD, MBA, medical oncologist, Memorial Sloan Kettering Cancer Center.” View Video
Press Releases
Results of a Comparative Effectiveness Claims Analysis from a Large Commercial Payer, Presented at the AHA Scientific Sessions 2018, Reinforce the Clinical Utility of the Corus® CAD Blood Test in the Outpatient Assessment of Obstructive CAD (12/17, CardioDX, Inc. Press Release) “...The analysis found that commercial patients with symptoms suggestive of obstructive coronary artery disease and who received the Corus CAD blood test had significantly lower rates of downstream healthcare resource utilization – lower rates of invasive coronary angiography procedures, revascularization procedures, CAD-related visits¥ and hospitalizations, and noninvasive cardiac testing - without increased adverse cardiac event rates as compared to traditional cardiac testing at 6-month follow-up.” Full
PAREXEL Recognized with Awards for Innovation and Expertise in Real-World Evidence (12/17, PAREXEL Press Release) “...‘These recognitions for PAREXEL's Real-World Evidence capabilities are particularly significant in light of the recent FDA framework issued that highlights the growing importance of RWE to the industry,’ said Michelle Hoiseth, Chief Data Officer of PAREXEL. ‘The framework indicates a commitment to expanding the regulatory model to include RWE approaches, and PAREXEL's award-winning applications are strong examples of solutions to leverage RWE and help move healthcare forward.’” Full
Journals
Health Technology Assessment and Health Care Utilisation in the Management of Early Prostate Cancer De Abreu Lourenco R, Williams S. December 11, 2018, European Urology Focus PubMed
Use of Decision Modelling in Economic Evaluations of Diagnostic Tests: An Appraisal and Review of Health Technology Assessments in the UK Yang Y, et al. December 14, 2018, PharmacoEconomics PubMed
Challenges of Non–Intention-to-Treat Analyses David L. DeMets, PhD, Thomas Cook, PhD December 17, 2018, JAMA JAMA
Reports
Guiding Practices for Patient-Centered Value Assessment December 2018 National Pharmaceutical Council
Three Essays in Health Economics: Towards Alternative Payment Models for High-Value, High-Cost Medical Treatments 2018 RAND Corporation
Articles
CAR T-Cell Therapy Cost-Effective Compared with past Treatments (12/14, HemOnc Today) reports “...‘I think CAR T therapy clearly was significantly better in terms of incremental qualities when compared with both other treatments for hematologic cancers and treatments for nonhematologic cancers,’ James Baumgardner, PhD, senior research economist at Precision Health Economics, said during his presentation. ‘In terms of trends, the introduction of CAR T therapy represents a major break of the trend of declining incremental effectiveness while showing similar cost-effectiveness to other innovations.’” Full
Amgen, Magellan Rx Partner to Improve Patient Care for Chronic, Difficult-to-Treat Conditions (12/14, Jaime Rosenberg, The American Journal of Managed Care) reports “...‘We're collaborating to evaluate the impact of interventions in patients with migraine or osteoporosis,’ said Steve Michurski, senior vice president of Trade Relations at Magellan Rx, in a statement. ‘We have a unique testing opportunity that can offer real-world evidence. The relationship combines our collective capabilities and resources to create value and insights for patients and providers across healthcare systems.’” Full
ACO Trade Group Says Medicare Program Saved $859M in 2016 (12/13, Les Masterson, Healthcare DIVE) reports "...The Medicare Shared Savings Program, the largest value-based accountable care organization initiative, saved $859 million in 2016, according to an analysis from Dobson | DaVanzo & Associates for the National Association of ACOs...The program, which includes 561 ACOs and 10.5 million patients, saved more than $660 million to the Medicare Trust Fund between 2013 and 2016 after accounting for bonuses paid to ACOs for meeting spending and quality targets." Full
Big Data Pushing Democratization of Healthcare, but Barriers Remain (12/14, Meg Bryant, Healthcare DIVE) reports “...Among the biggest hurdles are interoperability and lack of standards for EHR data preparation. ‘The biggest problem is that our data are not prepared in a way that allows us to even make sense of it,’ Amy Abernethy, chief medical officer, chief scientific officer & SVP oncology at Flatiron Health, told Stanford Medicine.” Full
Take the Lead to Deploy Emerging Technologies for Improved Outcomes (12/14, Brad Wilson, Healthcare Informatics) comments “...[H]istorically, one of the biggest barriers to value-based models has been providers' and payers' possessiveness of their own data. There is a good business reason for that possessiveness: competitive advantage. The different parts of the healthcare system do not want competitors to use shared data to steal business. But the guarding of data drives healthcare costs higher and, more importantly, makes delivering better, more personalized healthcare more difficult.” Full
Three Key Dynamics to Understand for Canadian Market Access (12/14, Sandra Anderson, PharmExec) comments “...Manufacturers entering the Canadian marketplace will need to develop plans to collect, interpret, and distribute data constantly throughout a product's life-cycle. For example, payers are increasingly seeking information that showcases a product's outcomes and cost-effectiveness outside the controlled environment of a clinical trial. Meaning, manufacturers will now need to develop data sets that go beyond Phase 2 and Phase 3 investigations and include ongoing, real-world evidence.” Full
Press Releases
ICER to Publish Upcoming Assessment on Treatments for Duchenne Muscular Dystrophy (12/13, ICER Press Release) “...ICER's assessment will focus on eteplirsen (Exondys 51™, Sarepta Therapeutics) and golodirsen (Sarepta Therapeutics). Eteplirsen was approved by the FDA in September of 2016, and golodirsen has an anticipated FDA approval decision expected in the middle of 2019.” Full
Events
NPC @ Paying for Cures: Ensuring Patient Access and System Sustainability February 12, 2019 Washington, D.C. National Pharmaceutical Council
Articles
Personalized Medicine in a World of Value Frameworks (12/2018, Robert W. Dubois, MD, PhD, Journal of Clinical Pathways) comments “...In order to truly realize the potential of value frameworks in personalized medicine, framework developers need to incorporate subgroup assessments from clinical trials as well as relevant post-trial analyses. They also need to both consider and incorporate patient preferences. Preferences are the most challenging to handle, but groups like IVI have shown that it can be done. Other organizations working on their own frameworks should incorporate similar patient-centric and personalized medicine approaches.” Full
Quality Measurement as a Tool for a Patient-Centered Approach to Assessing Value (12/2018, Tom Valuck, MD, JD and Russ Montgomery, PhD, MHS, Journal of Clinical Pathways) comments “...Measures can encourage wider use of shared decision-making. As Dr Dubois noted, shared decision-making is a way for clinicians to partner with patients to thoroughly consider the treatment options, the pros and cons of each option, and the potential outcomes so patients can make informed care decisions in concert with their clinicians. Ultimately, shared decision-making is a form of patient value assessment at the point of care for selecting the right care option for the patient.” Full
Personalized Medicine in a World of Value Frameworks (12/13, National Pharmaceutical Council: E.V.I.dently Today) comments “...Value is much broader than cost, explains National Pharmaceutical Council Chief Science Officer and Executive Vice President Robert W. Dubois, MD, PhD, in a commentary published in the Journal of Clinical Pathways. ‘Personalized medicine and driving for value are key directions in health care today,’ Dr. Dubois writes. ‘They can support one another; in fact, narrowing a medicine's use to when it is most effective is a wise use of resources.’” Full
NHC Publishes White Paper on Overcoming Barriers to Amplify the Patient Voice in Value Assessment (12/12, Dr. Elisabeth Oehrlein, National Pharmaceutical Council: E.V.I.dently Today) comments “...[P]atient groups and VA bodies agreed that the ultimate goal of patient-centered VA is for patients to have access to treatments they need at prices they can afford. Patient-centered VA exists when patients have been engaged, heard, understood, and respected throughout the entire process, and their input is incorporated and guides decision-making. Participants developed 17 recommendations to enhance patient centricity in VA. Recommendations include examples of recommended actions for improving engagement and examples of recommended actions for collecting and leveraging patient-provided data.” Full
J&J Says its Psoriasis Drug Superior to Novartis Treatment in Study (12/12, Reuters) reports “...After 48 weeks of therapy, 84.5 percent of the 1,048 participants treated with Tremfya showed 90 percent improvement in disease symptoms, as measured by the Psoriasis Area Severity Index, compared with 70 percent on Novartis's Cosentyx, J&J said.” Full
Value-Based Pricing And Reimbursement: Still More Promise Than Reality (12/13, Joshua Cohen, Forbes.com) comments “...Today, approximately 25 branded drugs are subject to some form of value- or outcomes-based contracting in the U.S. But, most contracts are ‘limited in applicability to disease states with more standardized protocols and dominated by drug therapies with single indications - notably osteoporosis, diabetes and hepatitis C.’ In sum, there appear to be quite a number of operational barriers, which include achieving consensus on what constitutes value, the need for extensive outcomes data collection, and the administrative costs of managing value-based pricing programs over extended periods of time.” Full
Measures of Physician Quality Don't Measure Up (12/13, Peter Ubel, Forbes.com) comments “...Panelists were asked to evaluate the validity of quality measures being used by either Medicare, the National Committee for Quality Assurance, or the National Quality Forum...The experts reviewed each measure and determined whether it was valid, uncertain, or invalid. They looked at 86 quality measures that Medicare will use as part of its MIPS program (an acronym for Merit-based Incentive Payment System). Barely more than a third of these measures were valid. The measures from NCQA and NQF came out better, with 60% and half being judged valid, respectively. But that still leaves a huge number of quality measures that are either uncertain or out and out invalid.” Full
When It Comes to The Big Debate on ACOs, What Is "Big Enough" Savings? (12/12, Mark Hagland, Healthcare Informatics) comments "...[CMS] Administrator Verma and her fellow senior federal healthcare officials seriously risk cratering the MSSP program, the core federal ACO program, if they push too hard on the downside-risk issue. On the other hand, if they don't push hard enough, the progress made so far could simply be dwarfed, in the broader scheme of things, by the current acceleration of overall U.S. healthcare spending inflation." Full
Press Releases
Janssen Signs Results-Based Contract with Oklahoma Health Care Authority (OHCA) for its Long-Acting Injectable Schizophrenia Medicines (12/13, Janssen Press Release) “...Under this arrangement focused on enabling the use of INVEGA SUSTENNA® (paliperidone palmitate) and INVEGA TRINZA® (paliperidone palmitate) earlier in treatment of adults with schizophrenia, OHCA aims to improve care among patients with schizophrenia and reduce expenses for medical services...A comparative effectiveness study found that the long-acting treatment INVEGA SUSTENNA® demonstrated superiority versus a group of seven commonly prescribed oral treatments in delaying time to relapse.” Full
ICER Describes for Patients and Policymakers Why the QALY Is Considered the Best Way to Reward the Care that Improves Patients' Lives (12/12, ICER Press Release) “...To address this concern of discrimination directly, and ensure that all stakeholders can engage with cost-effectiveness in confidence that it provides analyses that value a year of life for all patients exactly the same, ICER's future reports will prominently feature a calculation of the Equal Value of Life Years Gained (evLYG). The evLYG is not as flexible as the QALY in capturing benefits to quality of life but does measure any gains in length of life exactly the same across all conditions, regardless of age, severity of illness, or level of disability.” Full
National Science Foundation Announces Award on Pragmatic Clinical Trials (12/13, Verantos Press Release) “...As regulatory bodies increasingly look to real world evidence to augment the standard of care, pharmaceutical companies are exploring how to run advanced studies to understand real world impact of therapies on important clinical outcomes. Outcomes, such as symptoms, signs, and disease-free survival, are unavailable or inaccurate in claims and EHR structured data. This award will deploy advanced semantic technologies to extract these hidden outcomes and enable valid and relevant RWE.” Full
Nautilus Medical Announces Full Suite of Radiology Software Tools at No Cost to Providers to Lower Healthcare Cost in light of Patient Centered Outcomes Research Institute (PCORI) (12/13, Nautilus Medical Press Release) “...The policy advances PCORI's commitment to improve healthcare decision making based upon research. Sometimes getting the data together or having access to it can be problematic between the many disparate systems. Nautilus Medical is helping to change that.” Full
Nemours Researchers Tap PEDSnet to Glean New Insights on Link between Childhood Obesity and Asthma (12/12, PCORnet Press Release) “In a recent Pediatrics paper, the nonprofit pediatric health system Nemours credited PEDSnet for increasing the speed and scope of a study of obesity and asthma in children. PEDSnet, a partner network of PCORnet, the National Patient-Centered Clinical Research Network, offered the Nemours research team access to deidentified data from the medical records of more than 500,000 children. The study's results suggest obesity increases children's risk of developing asthma, a finding that many researchers have long suspected, but lacked the hard data from a study of this size and scope to prove.” Full
Journals
Being Overweight or Obese and the Development of Asthma Jason E. Lang, et al. December 2018, Pediatrics Pediatrics
Heterogeneity in the Respiratory Symptoms of Patients with Mild-to-Moderate COPD Johnson KM, et al. December 13, 2018, International Journal of Chronic Obstructive Pulmonary Disease Dove Press
Reports
A Dialogue on Patient-Centered Value Assessment: Overcoming Barriers to Amplify the Patient Voice December 2018 National Health Council
The QALY: Rewarding the Care: That Most Improves Patients’ Lives December 2018 ICER
Articles
Taking Tradeoffs To The Consumer With An Employee-Designed Health Benefit Package (12/10, Going Below the Surface) comments “...‘What we have not done very well in our country is to incentivize and equip consumers with information about value and about implications about different choices they may be making. In many cases, the insurance product is opaque and hard for them to understand,’ said Len M. Nichols, PhD...That's what makes a new case study from Janet McNichol, SPHR, CAE, human resources director, American Speech-Hearing-Language Association, along with Nichols, so interesting. She was interested in engaging employees in the design of a health benefit for her 291-employee organization and set up a structured game to allow all employees to weigh the different tradeoffs for themselves and for the organization as a whole.” Full
VBCs: Are They Working? (12/12, Adam Chapman, Eye for Pharma) reports “...A recent survey of 120 payers conducted by the ORC International and commissioned by Change Healthcare, found that payers reported an average saving in medical costs of 5.6% from VBC strategies with an accompanying 77% improvement in care quality, provider relationships and patient engagement. Yet, the same report highlighted ambivalence; more than half of payers reported dissatisfaction with current analytics, automation and reporting. On balance, Dan Leonard, President and CEO of the National Pharmaceutical Council, is optimistic. ‘We hear from payers and manufacturers that the impact is either neutral to positive in terms of improving patient access to treatments. There are very few negative reports out there,’ he says.” Full
Real-World Health Data Push Brings Challenges And Opportunities (12/12, Morris Panner, Forbes.com) comments “...The available data varies in quality. For example, medical records harbor mistakes. Information sits in electronic silos that cannot talk to each other, showing the need for innovative ways to gather data from far-flung sites for analysis elsewhere. It also requires efforts to get data from patients and patient advocacy groups. Merging data from multiple sources poses another challenge. Experts will need to establish standards for what counts as high-quality RWE. That may increase demand for epidemiologists, who were studying health outcomes in real communities before it became fashionable.” Full
Video: Dr Brian Koffman Explains How PROs Can Provide Insight Into CAR T Benefits (12/12, The American Journal of Managed Care) “While patient-reported outcomes don't provide the whole story, they can provide insight into how chimeric antigen receptor (CAR) T-cell therapy changes a patient's life, said Brian Koffman, MDCM, DCFP, DABFM, MS Ed, medical director, CLL Society.” View Video
U.K. Cost Watchdog NICE Issues Preliminary ‘No' on Alnylam's Onpattro and Ionis' Tegsedi (12/12, Arlene Weintraub, Fierce Pharma) reports “...NICE issued preliminary guidance on Wednesday stating neither drug is cost-effective and therefore shouldn't be routinely used by England's National Health Service. The agency is taking comments until Jan. 9 and plans to meet again in February to re-evaluate the drugs.” Full
Beneluxa: The Future of European Market Access? (12/12, Andrew McConaghie, PMLiVE) reports “...Marcel van Raaij is Director, Pharmaceutical Affairs & Medical Technology at the Netherland's ministry for health, and is one of the leaders of Beneluxa. He makes it quite clear that one of the main rationales for starting the alliance was the sense that pharmaceutical companies had an unfair advantage over national health systems and HTA organisations – what economists call ‘information asymmetry.’ ‘The pharmaceutical companies know every detail about their product and the reimbursement system in each country, while at the same time two countries in Europe often wouldn't know anything about the situation in their neighbouring countries.’” Full
Press Releases
Non-Invasive MRI Scan for Prostate Cancer Recommended by NICE (12/12, NICE Press Release) “...Multi-parametric magnetic resonance imaging works by creating detailed images of the prostate that enable clinicians to better detect suspected prostate cancer...In draft guidelines published today, NICE has recommended that mpMRI scanning should be offered as a first-line investigation for people with suspected clinically localised prostate cancer. This will not be replacing standard first-line tests such as the PSA test and people who are not going to have radical treatment should n--ot be routinely offered the scan.” Full
Journals
Systematic Reviews of Clinical Practice Guidelines: A Methodological Guide Johnston A, et al. December 5, 2018, Journal of Clinical Epidemiology PubMed
Cochrane Reviews and Dermatological Trials Outcome Concordance; Why Core Outcome Sets Could Make Trial Results More Usable Schmitt J, et al. December 5, 2018, The Journal of Investigative Dermatology PubMed
Value-Based Pricing in Latin America: How Far Away Are We? Mejía A, et al. December 6, 2018, Value in Health Regional Issues PubMed
Requests for Comment
Call for Feedback: Dual Antiplatelet Therapy Following Percutaneous Coronary Intervention: Clinical and Economic Impact of Standard Versus Extended Duration December 10 - 24, 2018 CADTH
Articles
FDA Maps Strategy to Broaden Use of Real-World Evidence (12/10, Jill Wechsler, Pharmaceutical Executive) reports “...Despite ongoing skepticism that RWE provides comparably valid and objective data as from randomized clinical trials, the development of clear methods and standards for collecting and using this information, ‘may encourage others to accept high quality studies’ in this area, commented Jennifer Graff, Vice President of the National Pharmaceutical Council. ‘There is so much quality data that is available,’ she noted, ‘let's not waste it.’” Full
Securing a Winning Strategy for Real-World Evidence (12/10, Julian Upton, Pharmaceutical Executive) reports “...[T]he winners and losers in pharma's quest to embed meaningful RWE strategies will be determined by their preparedness to make these dramatic organizational shifts, requiring trust-building and change management, along with investments in data sources, partnerships, and technology. According to ZS's Ye and Abhay Jha, principal and R&D technology lead, it drills down to establishing a cross-functional operating model that considers how different teams leverage data, with a clear action plan to measure ROI, leadership support, and the appropriate level of technology to support a data-driven model.” Full
RWE: Answering Questions in Rare Disease (12/10, Julian Upton, Pharmaceutical Executive) reports “...[Alnylam’s Sonalee Agarwal:] The biggest challenge in answering research questions in rare diseases is that we need to find sufficient number of patients and the right data sources to answer the critical questions. On the technical and the statistical side, we have the tools for this, and if not, we collaborate with people who do. We use a variety of data sources, including patient surveys, claims data, electronic medical records, and observational studies. It can be difficult because claims databases, for example, give us the volume, but not the granularity of data that we need. EMRs gives us more of the information we're looking for, but not the volume.” Full
Many Diabetics Needlessly Test Blood Sugar at Home (12/10, Lisa Rapaport, Reuters) reports “...More than half of the people testing their blood sugar at home didn't need to do this, accounting for 14 percent of the total study population, researchers reported in JAMA Internal Medicine. ‘Many type 2 diabetes patients not using insulin or other medications at risk of rapid changes in blood sugar levels are testing far more often then they need to be,’ said lead study author Dr. Kevin Platt of the University of Michigan in Ann Arbor. ‘This needless behavior causes unnecessary pokes, worry, and costs,’ Platt said by email. ‘More is not always better when it comes to medical care.’” Full
At UPMC, Turbo-Charging Quality Improvement Efforts through Data Analytics (12/11, Mark Hagland, Healthcare Informatics) reports “...[UPMC’s Dr. Oscar Marroquin] and his colleagues have been busy harnessing the power of creating and nurturing purpose-specific teams focused intensively on the management of data to power performance improvement, particularly in the clinical area...Below are excerpts from the interview for that cover story that Healthcare Informatics Editor-in-Chief Mark Hagland conducted with Dr. Marroquin this summer.” Full
I've Been Forced to Go Through ‘Step Therapy.' Don't Make Medicare Part B Participants Do That (12/11, Elizabeth Krempley, STAT News) comments “...Step therapy is a practice that requires patients to first try and ‘fail’ an insurer's preferred treatment before being able to access the therapy their own doctors have prescribed. It's essentially an insurer's way of saying, ‘We know more about drug therapy than your doctor and we need to protect you from his or her prescribing the most expensive drug first.’ The insurers' agents who make these decisions, by the way, are not always doctors.” Full
Revamped Cancer Drugs Fund Means More Positive Recommendations, Says NICE (12/11, Francesca Bruce, Pink Sheet) reports “...‘Drugs deemed promising but lacking sufficient clinical evidence are funded by the CDF for up to two years before undergoing a full NICE assessment, ample time for the marketing company to gather real-world clinical and economic data. This move has enabled earlier access to oncology therapies that gain accelerated EMA approval based on promising early-phase data, without head-to-head comparator trials,’ [Datamonitor Healthcare analyst Louisa Joseph] told the Pink Sheet.” Paid Subscription Required
Better Together When Buying Medicines? Taking Stock of the BeNeLuxA Initiative (12/11, Leela Barnham, PMLiVE) comments “...Simple economics would predict that the bigger the buyer, the better the deal. But that relies on successfully concluding the deal. So far, BeNeLuxA has only been successful in completing formal joint negotiations when buying Biogen's Spinraza (nusinersen). An earlier BeNeLuxA pilot for Vertex's Orkambi (lumacaftor/ ivacaftor) failed to lead to a deal in both 2016 and 2017.” Full
Press Releases
PCORI Board Approves $20 Million to Support Research on Improving Mental Health Care, Preventing Obesity in Young Children (12/11, PCORI Press Release) “...The newly approved awards include: $12.7 million for a study by researchers in Chapel Hill, N.C., Chicago, and Toronto, Canada, of the effectiveness of different strategies to treat anxiety and depression in expectant and new mothers...$7 million for a Vanderbilt University Medical Center study comparing different approaches to preventing obesity in young children.” Full
Journals
Intensive Intervention to Improve Outcomes for Patients With COPD Seppo T. Rinne, MD, PhD, et al. December 11, 2018, JAMA JAMA
Effect of a Program Combining Transitional Care and Long-Term Self-Management Support on Outcomes of Hospitalized Patients With Chronic Obstructive Pulmonary Disease: A Randomized Clinical Trial Hanan Aboumatar, MD, MPH, et al. December 11, 2018, JAMA JAMA
Requests for Comment
Dual Antiplatelet Therapy Following Percutaneous Coronary Intervention: Clinical and Economic Impact of Standard Versus Extended Duration December 10 - 24, 2018 CADTH
Management of Diverticulitis December 11, 2018 - January 8, 2019 AHRQ