CER Daily Newsfeed®
Articles
What the Rise of Real-World Evidence Means for the Pharmaceutical Industry: A Closer Look (September/October 2020, Value & Outcomes Spotlight) reports “...VOS: Do you foresee real-world evidence driving greater levels of collaboration between stakeholders (healthcare providers, payers, and policy makers)? [Jennifer Graff, PharmD, Vice President, Comparative-Effectiveness Research at the National Pharmaceutical Council:] Absolutely. Collaboration will extend beyond the traditional stakeholders, providers, payers, and policy makers. Activated patient groups are eager to contribute data if they have clarity around privacy and ownership of data and offer opportunities for supplemental real-world evidence endpoints. I also expect we will see more collaboration across the biopharmaceutical and vaccine industries as more pragmatic and adaptive study designs are initiated to ensure more efficient trials and adapt to new treatment combinations.” Full
Video: STAT+ Conversations: Beyond Real-World Data and COVID-19 (10/20, Casey Ross, STAT) “...This week, STAT's national technology correspondent, Casey Ross, is joined by two industry leaders to discuss the application of real-world data in confronting the Covid-19 pandemic. They'll cover how real-world data are already being applied, and their potential shortcomings as the race for effective vaccines or treatments continues. They will also discuss the unlikely alliances that have formed across institutions to help share these data, and how these collaborative efforts will hopefully have far-reaching impacts beyond the pandemic.” View Video
ICER: Infliximab Is Highest in Cost-Effectiveness for UC (10/19, Skylar Jeremias, The Center for Biosimilars) reports “...‘Prices are generally too high, even for the significant clinical benefits obtained, but the advent of biosimilars for infliximab has led to dramatic price decreases for this one agent and its biosimilars, displaying the potential cost savings that biosimilars may be able to deliver more broadly in the [United States],’ Pamela Brandt, MD, ICER's chief scientific officer, said in a statement.” Full
Avalere: Step Therapy May Exacerbate Patient, Payer Costs (10/19, Tony Hagen, The Center for Biosimilars) reports “...Step therapy policies, which require patents to fail on a preferred therapy before switching to an agent doctors may consider more appropriate, could end up costing payers more money and shouldering patients with high, up-front out-of-pocket (OOP) costs, according to an Avalere Health analysis of patients receiving treatment for Crohn disease, paid for by Bristol Myers Squibb.” Full
Biden Looks to Germany for Answers on How to Tackle High U.S. Drug Prices: Analyst (10/20, Eric Sagonowsky, Fierce Pharma) reports “...Under Biden’s plan, the government would set up a group to assess ‘fair’ values of drugs, like the Institute for Clinical and Economic Review already does in the U.S. Other governments have their own drug value assessment agencies, but ICER is not a U.S. government group. Biden made the proposal Sunday night, Bernstein analyst Ronny Gal wrote in a note to clients.” Full
What About the German Negotiation Model? Biden Steers Drug Pricing Debate to a Showdown (10/20, Amber Tong, Endpoints) reports “...The approach would give an independent group the power to estimate the ‘fair value’ for any given therapy, Bernstein analyst Ronny Gal noted, and would cover both newly introduced and existing drugs. While critical details are still missing, Gal reckoned that under this proposal, pharma companies can likely still charge higher prices than they could under the international pricing index, a yet-to-be-implemented Trump policy that industry execs have blasted as ‘radical’ and ‘horrible.’” Full
German Insurers Want End To Free Pricing In Amnog Review (10/20, Francesca Bruce, Pink Sheet) reports “...German medicine prices are ‘out of control’ and need to be reigned in, according to a senior official at Germany’s biggest statutory health insurer. Speaking to the Pink Sheet, Evert Jan Van Lente, director of EU affairs at AOK, suggested that ending the initial 12 months of free pricing for new pharmaceuticals and more transparency on public investments in R&D would be a good start.” Paid Subscription Required
Reports
Step Therapy Can Lead to Higher OOP Costs for Crohn’s Disease Patients October 15, 2020 Avalere
Articles
How Real Human Experiences Can Power a Healthier Future (10/19, Maria Raad, PharmaPhorum) comments “...The emergence of big data, smartphone adoption and cloud storage technologies means that information can be captured in real-time, and then aggregated and analysed to develop new insights. Collaborations such as HONEUR (Haematology Outcomes Network in Europe) – a partnership of universities, hospitals and institutions across Europe – can help with this by bringing together multiple stakeholders to analyse real-world data (RWD), quickly and at scale, from as many sources as possible. By answering research questions in real time, partners can extract real-world evidence (RWE) that informs their conclusions and accelerates their work.” Full
Journals
Network Meta-Analysis Results Against a Fictional Treatment of Average Performance: Treatment Effects and Ranking Metric Adriani Nikolakopoulou, et al. October 17, 2020, Research Synthesis Methods Wiley
Efficacy of Immunotherapy with PD-1 Inhibitor in Colorectal Cancer: A Meta-Analysis Shengqi He, et al. October 19, 2020, Journal of Comparative Effectiveness Research Journal of Comparative Effectiveness Research
Reports
Moneyball in Medicare: Heterogeneous Treatment Effects October 2020 National Bureau of Economic Research
Articles
Biden Hints At What Could Be Model For Drug Pricing Compromise (10/16, John Wilkerson, Inside Health Policy) reports “...In response to a question about drug prices, Biden said he backs Medicare negotiation of drug prices. ‘Number one, the cost of healthcare, as it relates to drugs, we're going to make sure that Medicare is going to be able to negotiate drug prices with drug companies,’ Biden said, according to the press pool report. Then Biden floated the idea of creating a variation of the German model, which includes collective insurer negotiation with arbitration, based on comparative research, as a backstop.” Paid Subscription Required
Real-World Data Support Use of Polatuzumab Vedotin for Heavily Pretreated Patients with R/R DLBCL (10/16, Jared Kaltwasser, Targeted Oncology) reports “A new study has confirmed the efficacy and safety of polatuzumab vedotin (Polivy) for the treatment of patients with relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL) in real-world settings. The study affirms earlier evidence that was obtained in more tightly controlled clinical trial settings. The research was published in the journal Leukemia & Lymphoma.” Full
UK: Novartis' MS Therapy Mayzent Approved by NICE (10/16, Lucy Parsons, PMLiVE) reports “Novartis' oral drug Mayzent has been recommended by the UK's National Institute for Health and Care Excellence for the treatment of secondary progressive multiple sclerosis. Mayzent (siponimod), a sphinogosine 1-phosphate receptor modulator, is the first oral disease-modifying treatment recommended by both NICE and the Scottish Medicines Consortium for SPMS with active disease.” Full
Press Releases
ICER Publishes Evidence Report on Therapies for Hemophilia A (10/16, ICER Press Release) “The Institute for Clinical and Economic Review today released an Evidence Report assessing the comparative clinical effectiveness and value of valoctocogene roxaparvovec (Roctavian™, BioMarin Pharmaceutical) and emicizumab (Hemlibra®, Genentech) for the treatment of hemophilia A.” Full
ICER Publishes Final Report and Policy Recommendations for Targeted Immune Modulator Therapies for Ulcerative Colitis (10/16, ICER Press Release) “...All agents in this review had evidence clearly demonstrating their superiority to placebo. The evidence was quite limited in helping to distinguish among the different treatments, but in one head-to-head trial, supported by indirect network meta-analysis, vedolizumab was found to produce greater rates of clinical response and remission over adalimumab, the market leader, in both biologic-naïve and biologic-experienced patients. All the other TIMs were found to produce net health benefits at least comparable to adalimumab, with no clear differences among them.” Full
Study Reveals Most Effective Drugs for Common Type of Neuropathic Pain (10/15, University of Missouri-Columbia) “...‘There was no clearly superior performing drug in the study,’ Barohn said. ‘However, of the four medications, nortriptyline and duloxetine performed better when efficacy and dropouts were both considered. Therefore, we recommend that either nortriptyline or duloxetine be considered before the other medications we tested.’” Full
Journals
Outcome of Relapsed/Refractory Diffuse Large B-Cell Lymphoma Patients Treated with Polatuzumab Vedotin-Based Therapy: Real-Life Experience Yafit Segman, et al. September 27, 2020, Leukemia & Lymphoma PubMed
Reports
Hemophilia A Update: Evidence Report October 16, 2020 ICER
Ulcerative Colitis: Final Evidence Report & Meeting Summary October 16, 2020 ICER
Articles
HTA Criteria Used to Evaluate Diagnostics (10/14, Jason Shafrin, Healthcare Economist) comments “When evaluating a new diagnostic, HTA agencies must assess two separate issues: analytical and clinical validity. Analytical validity basically indicates whether the test works; is it able to accurately predicts the presence or absence of a particular biomarker of interest. Clinical validity is whether the test matters in clinical practice. It could be the case that a test perfectly predicts a biomarker, but the presence or absence of the biomarker would not have a big impact on the treatment recommended by a physician. Two papers in Value in Health evaluate the criteria health technology assessment bodies use to evaluate new diagnostic tests.” Full
Health Systems Offer Lots of Data, but What Do Payors Want to Know? (10/14, David Wild, Pharmacy Technology Report) reports “Although health-system specialty pharmacies have access to an abundance of clinical and dispensing data, according to one expert who spoke during a session at the 2020 NASP Annual Meeting & Expo Virtual Experience, some pharmacies in outcomes-based contracts continue to ask payors the same question: What outcomes do they want to know about? That's the ‘million-dollar question,’ Bryan Schuessler, PharmD, MS, the director of home infusion and specialty pharmacy at Saint Luke's Health System in Kansas City, Mo., said...‘What is it that people really want to see on that payor line? We're not sure from payor to payor what's valuable at this point.’” Full
England's HTA Body To Revamp Product Selection Processes (10/15, Ian Schofield, Pink Sheet) reports “...The National Institute for Health and Care Excellence, which evaluates the cost effectiveness of medicines and other health technologies for use on the National Health Service in England, is proposing to improve the way it identifies and selects those products for the development of guidance. Under the proposals, which are out for consultation until 19 November, the criteria for selecting medicines, devices and diagnostics would be clarified, particularly in cases where costs and impacts were expected to be ‘significantly cost incurring or cost saving,’ or the likely costs and impacts for the NHS were uncertain. The plans are part of NICE's wider review of the methods and processes it uses to develop guidance. “ Paid Subscription Required
UK: NICE Recommends Novartis' Mayzent on the Heels of SMC Approval (10/15, Lucy Parsons, PharmaTimes) reports “...‘The decision by both NICE and the SMC represents a significant step forward in ensuring people living with SPMS with active disease in the UK have access to treatment. We are working closely with the NHS to ensure eligible patients can start benefiting from Mayzent as soon as possible,’ said Chinmay Bhatt, managing director UK, Ireland & Nordic for Novartis Pharmaceuticals.” Full
Press Releases
UAMS Researcher Awarded $500,000 to Study Quality of Digital Care (10/15, Spencer Watson, University of Arkansas for Medical Sciences Press Release) “The University of Arkansas for Medical Sciences has been awarded $500,000 by the Patient-Centered Outcomes Research Institute to study the quality of digital health visits for older patients. The funding is a supplemental award to a $4 million study funded last year to directly compare digital delivery of health care to standard in-person visits for patients with heart failure.” Full
NICE Draft Guidance Recommends Siponimod for Treating Secondary Progressive Multiple Sclerosis (10/15, NICE Press Release) “NICE has today issued draft guidance which now recommends siponimod (also called Mayzent and made by Novartis) for treating secondary progressive multiple sclerosis (MS). Siponimod, which is taken orally, is licensed to treat secondary progressive MS in adults with evidence of active disease (where people have relapses or features of inflammatory activity that show up on imaging).” Full
Articles
GBTS Town Hall Sparks A Serious Dialogue About Health Care Spending (10/13, Going Below the Surface) comments “...Dr. Robert Dubois, Interim President and CEO and Chief Science Officer of the National Pharmaceutical Council, reminded attendees that concerns about health care spending in the United States are nothing new. In 1960, health spending accounted for about five percent of GDP. It doubled by the late 1980s and rose to nearly 15 percent at the start of this century. It's currently at 18 percent and is projected to hit 20 percent by 2028, Dr. Dubois noted...A serious dialogue on spending requires us to go below the surface, noted Dr. Dubois, as the participants pondered a serious question: are we spending too much on health care?” Full
The Value of Value-Based Care (10/14, Aaron R. Wells, PhD, Medical Economics) comments “...[B]y attributing program performance across multiple programs members are engaged in allows the plan to ensure duplication of value does not occur, and, concomitantly, value is accurately assigned to the contracted entity. Lastly, and most importantly, for patients who are likely to never know how the care they receive translates into shared savings or penalties for their providers, the benefits of value-based care will be realized by improved health outcomes, lower out of pocket health expenditures, and more time living outside of the health care system.” Full
Study: Enhanced Oncology Clinic Reimbursement Increases Prescriptions of Evidence-Based Drugs (10/14, Aislinn Antrim, Pharmacy Times) reports “...‘We know that prescribing evidence-based cancer drugs is high-quality care and increases both the length and quality of life for patients with cancer,’ said co-lead author Justin E. Bekelman, MD, in a press release. ‘And yet, changing prescribing patterns has been and remains a big challenge. Based on what we found here, paying oncology practices to prescribe evidence-based drugs can serve as a valuable tactic to improve the quality of cancer care.’” Full
Journals
Developing AHRQ's Feasibility Assessment Criteria for Wide-Scale Implementation of Patient-Centered Outcomes Research Findings Alaina Kay Fournier Ph.D., et al. October 13, 2020, Journal of General Internal Medicine Springer
Findings and Lessons Learnt from Early Termination of a Pragmatic Comparative Effectiveness Trial of Video Consultations in Home-Based Palliative Care Huong Q. Nguyen, et al. October 13, 2020, BMJ Supportive & Palliative Care BMJ Supportive & Palliative Care
Pharmaceutical Pricing Benchmarks: Governmental Versus Private Sector Fatema Turkistani & Aseel Bin Sawad October 14, 2020, Journal of Comparative Effectiveness Research Journal of Comparative Effectiveness Research
Articles
Real-World Evidence Uptake Is "Silver Lining" Of COVID-19 Response, USFDA's Abernethy Says (10/12, Kate Rawson, Pink Sheet) reports “...For real-world data technologies to go forward post-COVID, [FDA principal deputy commissioner Amy Abernethy] said, three criteria will need to be met: Sponsors, who design, pay for and execute the trials ‘need to be willing to incorporate these technologies,’; Regulators need to have the ‘confidence to review datasets and provide feedback on what does and does not work,’ and; Clinical sites and patients need to ensure that RWD fits ‘in the way that they conduct clinical trials and they know how to use the technologies.’” Paid Subscription Required
A Pay-for-Performance Program Increases Evidence-Based Prescribing of Cancer Drugs (10/13, Lisa Kuhns, Journal of Clinical Pathways) reports “...‘We know that prescribing evidence-based cancer drugs is high-quality care and increases both the length and quality of life for patients with cancer,’ said Justin Bekelman, MD, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA. ‘And yet, changing prescribing patterns has been and remains a big challenge. Based on what we found here, paying oncology practices to prescribe evidence-based drugs can serve as a valuable tactic to improve the quality of cancer care.’” Full
PCORI and RAND Project to Evaluate Measures of Patient and Stakeholder Engagement (10/13, The PCORI Blog) comments “...The project team is engaging these groups—just as on any PCORI-funded project—to understand what they believe is important to measure and why. We are also asking stakeholders to tell us what attributes are most important for evaluating the quality and usefulness of a measure. For instance, is it that the measure was developed with patient input, or that it has been thoroughly tested? RAND will search and analyze peer-reviewed and grey literature—materials and research produced by organizations outside of traditional commercial or academic publishing—in health research and in related research fields from domestic and international settings.” Full
Health Leaders Question How, Not If, Value-Based Care Should Be Implemented (10/13, Mallory Hackett, Healthcare Finance News) reports “...Whether value-based care is delivered through a health system, accountable care organization or through a group of private practices, what matters most is the fact that patients are getting the best care for their money, Bryony Winn, the chief strategy officer at Anthem and the moderator of the debate said. ‘This is a material conversation,’ she said. ‘I think we've shifted past saying “Should we do value-based care?” And now the materiality is on how. And what you got from this is that there are real choices in how to change the cost and quality trajectory of the care we deliver here.’” Full
UK: Evidence for HTA: Innovative Methods for Challenging Times (10/13, Dalia Dawoud, NICE Blog) comments “Dalia Dawoud, Scientific Adviser at NICE Science, Evidence and Analytics Directorate explains NICE's role as co-host of HTAi 2021 in the UK, her part in planning the scientific programme and how the meeting will encourage debate and discussion around the latest advances in defining, generating and using evidence in healthcare decision making.” Full
Journals
Health Outcomes and Cost-Effectiveness of Diversion Programs for Low-Level Drug Offenders: A Model-Based Analysis Cora L. Bernard, et al. October 13, 2020, PLOS Medicine PLOS Medicine