CER Daily Newsfeed®
Articles
Broadening the Value Assessment Paradigm: Including the Value of Hope and the Value of Insurance (10/2019, Sean McElligott, MS and Ira Klein, MD, MBA, FACP, Journal of Clinical Pathways) comments “...The advent of social media and collection of people's ‘likes’ has created a way to recommend consumer goods based on revealed personal preferences. In many ways, as medical science advances to improve the lives of patients through better treatments and cures, so should the science of valuation. This will create a more accurate way to value and inform decision-making around treatment pathways and appropriate coverage for existing therapies and cures.” Full
Contemplating the Future of Oncology Pathways (10/2019, Gordon Kuntz, Journal of Clinical Pathways) comments “...Many outside observers think that pathway development is an exact science, simply weighting easily quantified factors to determine the best treatment for a given patient. Instead, it is a matter of weighing and debating the relative merits of heterogenous trials with endpoints that may defy direct comparison. Head-to-head drug trials would help to resolve these difficulties but are unlikely to happen due to lack of sponsorship for these types of trials. Post-market surveillance, developed with real-world data from pathways users, may help provide these types of comparative studies.” Full
J&J's Xarelto, Amarin's Vascepa Are Cost-Effective, Not Budget Friendly — ICER (10/18, Natalie Grover, Endpoints News) reports “...J&J said it had expected ICER to find Xarelto cost-effective in its new indication. ‘However, we do not endorse the use of cost-per-QALY or cost-per-life-year-gained analysis as the sole or primary basis of decision making,’ the company said in a statement to Endpoints News.” Full
UK: NICE ‘OK' for Bayer's Xarelto (10/18, Anna Smith, PharmaTimes) reports “The National Institute for Health and Care Excellence has published the final appraisal determination for Xarelto (rivaroxaban). The approval is for the indication of preventing atherothrombotic events in adult patients with coronary artery disease or symptomatic peripheral artery disease who are at high risk of ischaemic events, and is approved to be administered at a dose of 2.5 mg twice daily (BID) combined with aspirin (75-100 mg) once daily.” Full
Requests for Comment
NPC Comments on Proposed Changes for ICER 2020 Value Assessment Framework October 18, 2019 National Pharmaceutical Council
Amgen Comments on ICER’s Proposed Changes to the 2020 Value Assessment Framework October 18, 2019 Amgen
Articles
J&J, Amarin Cardiovascular Drugs Are Cost-Effective, ICER Says—But There's a Catch (10/17, Kyle Blankenship, Fierce Pharma) reports “[ICER] unveiled its final evidence report on J&J's Xarelto and Amarin's Vascepa, finding both drugs hit the group's cost-effectiveness standards when used as add-on treatments for CV disease...Citing a survey from a physician meeting, ICER said doctors considered using Xarelto in 30% or more of eligible U.S. patients. But by the group's calculations, based on annual costs, only 6% of those patients could be treated before busting payers' budgets...Vascepa would be even more popular, ICER said.” Full
COAs – Build New or Adapt Old? (10/17, Aimee Lee Russell, National Health Council Blog) comments “...If you do find a tool that measures the concept you are interested in studying, be sure that it is patient centered, or one that matters to patients. If the tool does not capture a patient-centered measure, it will need some modification. If there are no older, existing tools that fit what you are looking to study, you might consider building a new [clinical outcome assessment], that incorporates patient-centered methods and endpoints. Know that the process will take time and resources, but the end result will be a measure that captures the patient perspective.” Full
Italy: Added Benefit Will Be Key To Reimbursement (10/17, Francesca Bruce, Pink Sheet) reports “...To secure reimbursement in Italy, pharmaceutical companies will have to demonstrate that their medicines offer additional therapeutic value over treatments already on the market, according to a draft decree updating pricing and reimbursement regulations. The draft decree formally shifts the focus of reimbursement decision-making from cost-effectiveness to additional therapeutic value, according to Roberto Cursano and Riccardo Ovidi, who are respectively partner and associate at the law firm Baker McKenzie's Italy Pharmaceutical and Healthcare Department.” Paid Subscription Required
Press Releases
ICER Issues Final Report and Policy Recommendations Regarding Additive Treatments for Cardiovascular Disease (10/17, ICER Press Release) “The Institute for Clinical and Economic Review today released a Final Evidence Report and Report-at-a-Glance assessing the comparative clinical effectiveness and long-term value for money of rivaroxaban (Xarelto®, Janssen Pharmaceuticals), and icosapent ethyl (Vascepa®, Amarin Pharma). ICER's report on these additive treatments for cardiovascular disease was reviewed at the September 2019 public meeting of the Midwest Comparative Effectiveness Public Advisory Council, one of ICER's three independent evidence appraisal committees.” Full
ASP Framework Points the Way Forward For Building National, Multi-Disciplinary Key Stakeholder Collaboration In Precision Medicine (10/17, American Society of Pharmacovigilance Press Release) “American Society of Pharmacovigilance directors today presented to key stakeholders in the field of pharmacogenomics, a detailed framework for building a national, public-private multi-disciplinary stakeholder collaboration to accelerate the development of precision medicine practices as a standard of care. The Standardizing Laboratory Practices in Pharmacogenomics Initiative seeks to unite patients, providers, industry and government entities, among others, to align efforts in optimizing pharmacogenomics and to enable the highest levels of patient medication safety.” Full
Journals
Real-World Evidence: Research Reveals a Lack of Racial Diversity in Clinical Trials for Cancer Drugs Matthew Gavidia October 2019, Evidence-Based Oncology AJMC
Comparative Effectiveness of Dual Antiplatelet Therapy with Aspirin and Clopidogrel vs Aspirin Monotherapy in Acute, Nonminor Stroke: A Nationwide, Multicenter Registry-Based Study Kim JT, Park MS, Choi KH, et al. October 15, 2019, Stroke Stroke
Changes In The Equity Of US Health Care Financing In The Period 2005–16 Paul D. Jacobs and Thomas M. Selden October 16, 2019, Health Affairs Health Affairs
Reports
CVD: Final Evidence Report October 17, 2019 ICER
Articles
US FDA's Temple On Real-World Evidence: 'I Find The Whole Thing Very Frustrating' (10/15, Michael Cipriano, Pink Sheet) reports “...[FDA] officials have been enthusiastic, but cautious, about the prospects of using real-world evidence to support new claims of efficacy. But Robert Temple, deputy director for clinical science in the Center for Drug Evaluation and Research, offered an especially pessimistic assessment of the concept. ‘I find the whole thing very frustrating,’ Temple said during a 7 October FDA panel discussion at the Biopharma Congress. Temple specifically pointed to the desire of industry to use epidemiological studies to obtain new efficacy claims.” Paid Subscription Required
RWE to Supplement RCTs: FDA Projects Continue (10/15, Zachary Brennan, Regulatory Focus) reports “...‘I don't think the optimal scenario is to obviate the need for clinical trials,’ [Andrew Norden, chief medical officer of COTA,] said, noting there ‘may be a small subset’ of trials for which that's possible, but, ‘Where this gets more rational and exciting is in the scenarios unfolding in oncology with a novel drug coming out that targets a novel mutation or an unmet need and patients don't want to be randomized to standard of care or placebo… the question is can we use RWD to see if we can do better with the data from these single-arm trials.’” Full
Op-Ed: A Troubling Method of Curbing Health Care Costs Is Gaining Ground (10/16, Debbie Hart, NJ Spotlight) comments “...If ICER reviews are adopted more widely, it could deny patients access to new and life-saving drugs and bring medical innovation — in New Jersey and elsewhere — grinding to a halt. It would allow ICER, rather than physicians, to make treatment decisions for patients. And when patients can't access the treatments they need, it can lead to complications, increased doctor visits, increased costs, trips to the emergency room, and even death.” Full
Some Say a Nonprofit Research Group Unfairly Influences Drug Prices (10/16, Liz Seegert, Next Avenue) reports “...[N]ot everyone agrees with the way ICER determines drug prices. The Alliance for Aging Research says ICER's calculations discriminate against older adults. AAR...recently launched an information campaign claiming that because of the way ICER determines drug costs, ‘sick and older patients are more likely to be denied access to medications that could help improve their condition or quality of life.’” Full
Press Releases
New Physiatry Clinical Data Registry to Measure and Improve Outcomes, Effectiveness, and Value (10/16, ArborMetrix Press Release) “...‘Our registry provides an infrastructure for the specialty to define and prove its value to key stakeholders in the health care system, including hospital administrators and payers. The evidence of our work will be critical in supporting our role in medicine and advancing our vision for the specialty,’ said Peter C. Esselman, M.D., FAAPMR, President, AAPM&R. ‘Data from the registry will help us learn from each other what are the best treatments for our patients that result in optimal patient-centered outcomes.’” Full
Articles
RWE: Comparators, Therapeutic Area May Be Key For Trial Replication (10/14, Sue Sutter, Pink Sheet) reports “...The ability to use observational study data to replicate randomized controlled trial results may depend upon the comparators studied in the underlying trials as well as the therapeutic area, according to the head of the [FDA’s] real-world evidence demonstration project program. ‘Comparators matter,’ David Martin, associate director for RWE analytics in the Office of Medical Policy, said in describing early learnings from trial replication projects. ‘It may be easier to replicate trials where the trial itself compared one agent to another,’ as opposed to add-on trials in which the comparator is usual care plus placebo, he said.” Paid Subscription Required
Leading AstraZeneca's Return to Growth in Europe (10/15, PMLiVE) reports “...‘For more than a year we have been developing a range of models that will address the different criteria that payers have. Having said that, in essence all healthcare systems want the same thing: data that proves your product produces the desired clinical outcomes – not only from clinical trial data, but also in a real-world setting.’ [AstraZeneca’s Iskra Reic] added: ‘Being able to show that real-world benefit, in a specific population in a specific market, makes a huge difference in those negotiations.’” Full
McKesson and Aetion to Combine RWE Platform, EHR Data (10/15, Melissa Fassbender, Outsourcing Pharma) reports “...As per the agreement, the companies will combine Aetion's real world evidence platform with data from McKesson's iKnowMedSM oncology electronic health record system. Initially accessible to the researchers at Brigham and Women's Hospital leading the [FDA] demonstration project, RCT DUPLICATE, the solutions will be available for commercial use, soon, an Aetion spokesperson told us.” Full
Video: Dr Richard Snyder on the Impact of Novel, Costly Drugs on Prevalence of Value-Based Agreements (10/15, The American Journal of Managed Care) “Richard Snyder, MD, executive vice president of Facilitated Health Networks and chief medical officer at Independence Blue Cross, discusses how the increasing share of novel, costly therapies will impact the prevalence of value-based agreements.” View Video
PCOM Receives $5.5 Million for Opioid Study (10/14, The Philadelphia Tribune) reports “Philadelphia College of Osteopathic Medicine was awarded a $5.5 million contract from [PCORI] to study the effectiveness of psychosocial treatments for individuals receiving medication assisted treatment for opioid use disorder at four federally qualified health centers. The study will examine the effectiveness of two psychosocial treatments — cognitive behavioral therapy and peer recovery support — individually and in combination.” Full
New Tool To Help Evaluate Quality of RWD (10/15, Francesca Bruce, Pink Sheet) reports “...Registries are being increasingly used in HTA and regulatory assessments and interest in the use of observational data to complement experimental data is growing, says EUnetHTA's vision paper explaining the tool. ‘This project aims to support best practice in the collection, use and re-use of real world data, and explore options to support sustainable multi-stakeholder collaboration,’ it says.” Paid Subscription Required
Press Releases
Dr. Josephine Briggs to Keynote PhRMA Foundation-National Health Council Conference on New Approaches to Value Assessment (10/15, PhRMA Foundation Press Release) “...‘The conference will showcase cutting-edge research underway to develop new approaches to value assessment that better incorporate the patient voice,’ said Eileen Cannon, President of the PhRMA Foundation. ‘The emerging research we'll highlight at the conference fills in the many missing pieces in current assessment methodologies. The PhRMA Foundation is excited to be a part of this innovative exploration of value, and to convene stakeholders to discuss how better defining value can impact the entire health care system.’” Full
Journals
Editorial: Toward Evidence-Based Policy Making to Reduce Wasteful Health Care Spending Karen E. Joynt Maddox, MD, MPH; Mark B. McClellan, MD, PhD October 7, 2019, JAMA JAMA
Articles
Empagliflozin Beats Pair of DPP-4s in Cost-Effectiveness for Patients With Type 2 Diabetes, CVD (10/14, Mary Caffrey, The American Journal of Managed Care) reports “Empagliflozin, the sodium glucose co-transporter 2 inhibitor sold as Jardiance, was more effective for patients with type 2 diabetes and established cardiovascular disease than either of 2 well-known drugs from a competitor class, when used with standard of care, according to an analysis just published in Diabetes Therapy.” Full
Researchers Find 15% of Trials Could Be Replicated Using Real World Data (10/11, Michael Mezher, Endpoints News) reports “...‘This finding suggests the potential for real-world evidence to complement clinical trials, both by examining the concordance between randomized experiments and observational studies and by comparing the generalizability of the trial population with the real-world population of interest,’ the authors write. However, the authors caution that for new products, RWE is unlikely to serve as a replacement for RCTs and point out that many of the trials they looked at could not be replicated because the data necessary to do so ‘are unlikely to appear in an EHR in structured form if at all.’” Full
Accounting for Hope: Using ‘Mean Survival Gain' to Price New Cancer Drugs (10/14, Alice Chen and Dana Goldman, STAT) comments “...The United Kingdom's National Institute for Health and Care Excellence relies predominately on mean survival gains to appraise new drug technologies. The need to account for longer-term survival has also been recognized by institutions such as the American Society of Clinical Oncology and the Institute for Clinical and Economic Review. Promising therapies await more and more people with cancer. Striking a balance between affordability and future innovation will require policies that, among other considerations, accurately relate a drug's price to its total value. We need to take into account the mountaintop view, not just the price per square foot.” Full
Validating Value: Two Key Elements for Leaders to Confidently Take on Risk (10/14, Modern Healthcare) comments “...Value-based agreements require significant payer-provider collaboration, and a major element behind such collaboration is access to data. Providers have access to rich, detailed EHR data that predict future medical needs, but often lack data from the wider breadth of providers a patient has seen, which is often found in claims data held by payers. Claims data also contains financial metrics that offer insight into costs. Both parties need to consider what they don't know and agree to share as much information as possible to bridge those gaps and ensure a successful partnership.” Paid Subscription Required
House Drug Bill Would Save $345 Billion, Reshape Pharma Industry (10/11, Drew Armstrong, Bloomberg) reports "...The bill, H.R. 3...would direct Medicare, the U.S. government health program for the elderly and disabled, to set pharmaceutical prices based on an index of what other developed nations pay for top-selling drugs. For products not sold abroad, the government would set their price at a discount to what U.S. purchasers paid...Drugmakers would raise their prices in other countries to compensate for lower prices in the U.S. For new drugs, pharmaceutical companies would likely introduce them at higher prices to try and offset discounts. And there would be a slight decrease in the number of new drugs developed, the CBO projected." Full
CBO: $345B In Savings From H.R. 3 Comes At Cost Of Fewer New Drugs (10/11, John Wilkerson, InsideHealthPolicy.com) reports “The Congressional Budget Office estimates that House Democrats' bill to negotiating drug prices would save taxpayers $345 billion in six years, but that savings would come at the cost of 8 to 15 fewer drugs being invented over a decade -- FDA approves about 30 new drugs annually. CBO's preliminary estimate of H.R. 3 is that brand drug makers revenues would be reduced by half a trillion dollars to $1 trillion.” Paid Subscription Required
Journals
Cost-Effectiveness Analysis of Empagliflozin in Comparison to Sitagliptin and Saxagliptin Based on Cardiovascular Outcome Trials in Patients with Type 2 Diabetes and Established Cardiovascular Disease Ramos M, et al. October 10, 2019, Diabetes Therapy PubMed
Articles
ICER Says AbbVie's Rinvoq for RA May Be Cost-Effective but Still Questions the Value of JAK Class (10/11, Arlene Weintraub, Fierce Pharma) reports “...Instead of analyzing the costs of the medicines over a lifetime, ICER changed the time horizon of treatment to one year. And the new assessment calculated long-term cost-effectiveness of Rinvoq based on an assumed net price that matches the current net price of Humira and that reflects the average discount of the other two JAK inhibitors. Those changes were enough to bring the cost burden of Rinvoq below ICER's threshold for cost-effectiveness...” Full
Who is Driving the HTA Bus? And Where is it Headed? (10/10, Jeffrey J. Lemay, et al., PharmExec) comments “...By their actions, federal agencies are focused on conducting CER within subpopulations and generating RWE through observational trials and pragmatic randomized clinical trials. Regulatory changes may facilitate an increase in value-based contracting, which would increase demand for outcomes benchmarks to enable such contracts. Finally, an increase in data availability and future standards for RWE, could mean that payers will expect [pharmaceutical value assessment] organizations to update their findings with RWE at some point after a drug is launched. For-profit companies and not-for-profit organizations that have access to real world data or the technology for sophisticated analysis will evolve stronger capabilities to play a key role in PVA.” Full
Real-World Evidence Can Better Inform Diabetes, Cardiometabolic Care (10/10, Regina Schaffer, Healio Endocrine Today) reports “...Randomized controlled trials remain the gold standard for the initial assessment of efficacy and safety for any therapy, but real-world evidence can answer some questions that are difficult or impossible to answer with a randomized controlled study design, Lawrence Blonde, MD, FACP, MACE...said during a presentation. Real-world data — information on patient characteristics, care and outcomes derived from electronic health records, claims databases and patient registries — can inform patient care and outcomes research, safety surveillance, research on health care systems and well-controlled effectiveness studies, Blonde said.” Full
Study Finds Real-World Evidence Replicates less than One-Fifth of Clinical Trials (10/11, Alaric Dearment, MedCity News) reports “...‘I think there are lots of ways that RWE will be used for medical product evaluation, but for these data to be a suitable replacement, work is still needed to enhance the data that are available,’ one of the researchers, Yale professor of medicine Dr. Joseph Ross, wrote in an email. ‘Working with current real-world data sources, I believe large, safety-oriented evaluations are useful and feasible. But I think more needs to be done to better discern product effectiveness using real-world data.’” Full
Replicating RCTs With Real-World Data Is Unlikely for Most Trials (10/11, Laura Joszt, The American Journal of Managed Care) reports “...While RWE better reflects patient demographics, comorbidities, adherence, and use of concurrent treatment in actual clinical environments, ‘enthusiasm for using retrospective RWE to complement the evidence generated by RCTs may still need to be tempered by feasibility concerns because it is unclear whether it is reasonable to expect that observational data can be used to address the same clinical questions being answered by traditional clinical trials,’ the authors explained.” Full
Are Value-Based Models Helping or Hindering Care Delivery for Primary Care Providers? (10/10, Jaime Rosenberg, The American Journal of Managed Care) reports “...No matter what value-based models providers are participating in, whether it be the 2 under the Primary Cares Initiative or under ACOs, physicians are at risk; however, the financial implications differ based on the model, said [Roji Health Intelligence CEO Theresa Hush]. While Direct Contracting, ACOs, and Medicare Advantage models all leave providers responsible for their costs of care, the extent to which they are responsible may differ. For example, Direct Contracting offers partial and global capitation. Meanwhile, under Primary Care First, there are rewards and penalties for hospital utilization.” Full
3 Reforms Aim to Reduce Administrative Burden of Value-Based Care (10/11, Laura Dorr, Managed Healthcare Executive) reports “...[The American Medical Group Association] suggested CMS scale down the amount of quality measures for value-based care providers. ‘Using a standard set of value measures will help reduce the variation in the measures that are reported and help eliminate unnecessary confusion and administrative burdens,’ AMGA wrote in their comment letter to CMS. AMGA suggested that CMS use a set of 14 process and outcomes quality measures approved in 2018 by the association's Board of Directors.” Full
Press Releases
ICER Releases Updated Draft Evidence Report on Rheumatoid Arthritis Therapies (10/11, ICER Press Release) “...After initially publishing an earlier version of this Draft Evidence Report on September 26, 2019, ICER's internal reviewers identified the need to reevaluate some of the assumptions and calculations in the report to better align our economic modeling with how patients transition between these therapies in the real world. In our updated model, patients that are failed by first-line treatment now transition to a market basket of targeted immune modulators, instead of palliative care. Also, because clinical differentiation between these therapies is uncertain over time, we will now analyze their cost-effectiveness in the first year of use, instead of over a patient's lifetime.” Full
Another Treatment Option for Ovarian Cancer Approved for the Cancer Drugs Fund (10/11, NICE Press Release) “...Rucaparib can now be offered to women with relapsed ovarian, fallopian tube or peritoneal cancer, that has responded to platinum-based chemotherapy. Taken as a tablet, twice daily, it slows the progression of cancer by preventing cancer cells repairing so slowing down the tumour's growth. Around 1,350 people in England could benefit from this new treatment which will be available immediately through the CDF.” Full
Journals
Trends in High- and Low-Value Cardiovascular Diagnostic Testing in Fee-for-Service Medicare, 2000-2016 Vinay Kini, MD, MSHP, et al. October 11, 2019, JAMA Network Open JAMA Network Open
Reports
RA Update: Draft Evidence Report October 11, 2019 ICER