CER Daily Newsfeed®

Articles

Roche Official: Outcomes-Based Pricing Could Spur Precision Dosing (8/15, Beth Wang, InsideHealthPolicy.com) reports “...Experts say that a cultural change in industry is needed to get drug makers to focus more on precision dosing for drugs, and that both FDA and payers could help push industry in that direction. FDA could actively encourage individualized dosing in clinical trials rather than a one-size-fits-all approach. And a move to outcomes-based pricing could spur drug makers to conduct such trials so they that don't lose money if some patients are later found to do well on lower doses.” Paid Subscription Required

Frontiers In Real-World Evidence: US FDA Partners With Syapse To Explore Sources Beyond Electronic Records (8/15, Derrick Gingery, Pink Sheet) reports “...‘The key question for any collaboration involving real-world evidence is for us to have a better understanding of treatment patterns, dose adjustments, and outcomes outside of the traditional clinical trial space,’ the [FDA] told the Pink Sheet. ‘We approve drugs based on clinical trials, but our work does not end there. We want to learn from what occurs outside of clinical trials in the post-approval setting.’” Paid Subscription Required

Mercy Launches Real-World Evidence Network for Advanced Analytics (8/16, Jessica Kent, Health IT Analytics) reports “...The Real-World Evidence Insights Network will leverage the large amount of health data generated by health systems each year. MTS will coordinate the pooling of data between health systems by creating a consortium of providers, leading to more advanced analytics. In partnership with tech company SAP and hospitals around the country, MTS will build on its current repository of de-identified patient data from EHRs and other sources to establish a database of real-world evidence.” Full

Comparing Effectiveness of Agents for MSSA Bacteremia (8/16, J. Drew Zimmer, PharmD, BCPS, Contagion Live) reports “...The study helps antimicrobial stewardship efforts by not only providing more evidence of the effectiveness of narrow spectrum agents, but by indicating that broad spectrum beta lactams, in this case piperacillin/tazobactam, may not be as effective. More data is needed showing efficacy or even superiority of broader spectrum ß-lactams, prior to recommending or justifying their use over the narrow spectrum agents. Although efficacy of fluoroquinolones for MSSA bacteremia was shown, their usage should not be considered first line.” Full

ICER Blasts FDA, PTC and Sarepta for High Prices on DMD Drugs Emflaza, Exondys 51 (8/16, Amber Tong, Endpoints News) reports “...10 out of 17 members on its advisory council agreed that deflazacort confers a better health benefit than prednisone, as patients taking the drug appears to have better motor function and experience less weight gain...However, they argue that the drug's price tag is unjustified, with the majority agreeing the longtime value for money is low...The panelists were less impressed by eteplirsen's results, voting 16 to 1 that there is not adequate evidence to claim treatment with the drug is superior to corticosteroid and supportive care alone.” Full

Video: DIA 2019: Advancing the Use of Real World Evidence for Regulatory Decision Making (8/16, DIA) “Barbara Lopez Kunz, Global Chief Executive of DIA, and Nicole Mahoney, Senior Director of Regulatory Policy at Flatiron Health, discuss appropriate regulatory use cases for the information generated by Electronic Health Records, the promises this information holds, and the challenges that need to be overcome.” View Video

The Relevance of Patient Perspectives to Value (8/16, PMLiVE) reports “...Regulators and health technology assessment agencies are taking increasing account of patient insights in their assessments of new treatments. This is particularly true where treatments are associated with considerable toxicity, or incremental survival benefit. Patient insights also are needed to justify treatment claims for improving symptoms or quality of life.” Full

Press Releases

ICER Issues Final Report and Policy Recommendations Regarding Treatments for Duchenne Muscular Dystrophy (8/15, 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 value of the corticosteroid deflazacort (Emflaza®, PTC Therapeutics), and two exon-skipping therapies — eteplirsen (Exondys 51™, Sarepta Therapeutics) and golodirsen (Sarepta Therapeutics).” Full

Journals

Will Increasing Primary Care Spending Alone Save Money? Zirui Song, MD, PhD; Suhas Gondi, BA August 15, 2019, JAMA JAMA

Reports

DMD: Final Evidence Report August 15, 2019 ICER

Events

Symposium on Generating Support and Demand for Health Data Sharing, Linkage, and Use August 23, 2019 National Academy of Sciences Building National Academy of Medicine

ICER’s Value Framework and Patient Feedback: Q&A with Steve Pearson September 4, 2019 2:00 - 3:00PM EDT ICER

Articles

High Health Spending In The U.S. Isn't Being Driven By Low Social Spending (8/15, Going Below the Surface) comments “The United States has the highest per-capita rate of health care spending in the world, and the country's status as an international outlier has been linked to a perceived under-investment in social programs, such as employment, housing and education, which, in turn, has been theorized to harm health outcomes and drives health spending. But a new study in Health Affairs – part of the National Pharmaceutical Council- and Anthem, Inc.-sponsored ‘Considering Health Spending’ series – calls into question the idea that increased social spending will pay for itself in lower health care bills.” Full

The U.S. Doesn't Spend Less on Social Care than Other Developed Countries (8/15, Caitlin Owens, Axios) comments “...The big picture: Some policymakers and researchers say that the underinvestment in social services has led to a less healthy population, and point to the low U.S. ratio of social-to-health spending. Between the lines: Instead, our spending on social services as a percentage of GDP is actually greater than the average of OECD countries, and there's a positive correlation between a country's spending on social services and its spending on health care.” Full

Outcomes-Based Pricing: Can It Facilitate The Study Of Precision Dosing? (8/14, Michael Cipriano, Pink Sheet) reports “...‘Outcomes-based pricing, my goodness,’ [Richard Peck, global head or clinical pharmacology at Roche,] said. ‘If there's outcomes-based pricing, people like my colleagues are really going to want to know, we only want to dose the right patients, and we really need to give you the right dose because we don't want you to not to have efficacy because we didn't give you enough. And we don't want you not to have efficacy because we gave you too much and you have side effects and stop taking the drug. And so that's an enormous incentive.’” Paid Subscription Required

The Real-World Evidence Project at the FDA Adds Syapse to its List of Partners in a Push past Trial Boundaries (8/15, John Carroll, Endpoints News) reports “...‘Advancing a deeper understanding of real-world endpoints and analytical methodologies is critical to assuring that all stakeholders can have confidence in the quality of evidence produced and accelerating the use of RWE in regulatory decision-making,’ noted company founder and president Jonathan Hirsch in a prepared statement. ‘The network of health systems that Syapse represents offers a unique opportunity to learn from oncology patient journeys and populations not well represented in traditional clinical trials.’” Full

Real-World Data in the Era of Precision Medicine for Lymphoid Malignancies (8/15, Caleb Rans, PharmD, Hematology Advisor) reports “Until recently, treatment strategies for lymphoma have been largely based on cell morphology. At present, standardized treatment approaches are common, despite ongoing advances in precision medicine for lymphoid malignancies. As the field of precision medicine continues to advance, the utility of real-world data to support the development of improved therapies for lymphoma is becoming increasingly evident.” Full

Press Releases

Leaders of NIH's All of Us Research Program Recap Progress and Next Steps (8/14, National Institutes of Health Press Release) “The All of Us Research Program at the National Institutes of Health has made strong progress in its efforts to advance precision medicine, according to program leadership in a forthcoming paper in the New England Journal of Medicine.” Full

Journals

The Relationship Between Health Spending And Social Spending In High-Income Countries: How Does The US Compare? Irene Papanicolas, et al. August 14, 2019, Health Affairs Health Affairs

Doctors Should Treat Impetigo with Antiseptics Not Antibiotics, Says NICE Elisabeth Mahase August 15, 2019, BMJ BMJ

Cost Effectiveness of Interventions to Improve Adherence to Statin Therapy in ASCVD Patients in the United States Armstrong SO, Little RA August 15, 2019, Patient Preference and Adherence Patient Preference and Adherence

Ataluren Use in Patients with Nonsense Mutation Duchenne Muscular Dystrophy: Patient Demographics and Characteristics from the STRIDE Registry Francesco Muntoni, et al. August 15, 2019, The Journal of Comparative Effectiveness Research The Journal of Comparative Effectiveness Research

Articles

FDA Cancer Office Taps Syapse for Real-World Evidence Development (8/14, Conor Hale, Fierce Biotech) reports “...‘Real-world evidence from well-designed studies meeting appropriate data quality standards can help to inform decision-making and provide information regarding the impact of new therapies in real-world patient populations, particularly those not represented in clinical trials,’ [FDA associate director for oncology regulatory science and informatics Sean Khozin] added. ‘This is especially critical in precision medicine, where understanding all of the factors that may drive safety and response is both imperative and difficult to capture at scale using traditional clinical trials.’” Full

ICER Reviews Duchenne: Exondys 51 Lacks Net Health Benefit; Emflaza Fairs Better (8/13, Pink Sheet) reports “...[ICER President Steve Pearson] was forced to walk a difficult line during the meeting – asking tough questions on long-term value to elicit a well-rounded discussion while expressing appropriate sympathy to the parents at the table. ‘Value is a tricky word, because to a patient or a family, value is intrinsic in the benefits – or even the hope – that they can get from a new treatment,’ he said. ‘Votes here are not intended to say that patients aren't important. This is a hard-edged look,’ he acknowledged, noting that even an ‘extraordinarily successful treatment can still be overpriced.’” Paid Subscription Required

Video: Dr Jeroen Jansen Discusses Value-Assessment Questions Posed by Curative Treatments (8/14, The American Journal of Managed Care) “With the increasing prevalence of more expensive, potentially curative therapies, there comes the question of can we still afford expensive interventions that are that valuable, explained Jeroen Jansen, PhD, lead scientific advisor, Open-Source Value Project, Innovation and Value Initiative.” View Video

COTA Collaboration: Helping FDA Figure Out What's Possible and What's Not in Embrace of Real-World Evidence (8/13, Mary Caffrey, Evidence-Based Oncology) reports “...In an interview with Evidence-Based Oncology, [COTA chief medical officer Andrew Norden, MD, MPH, MBA] emphasized that COTA is not looking to eliminate RCTs. But by unlocking the secrets of the data sets in EHRs, real-world data can ‘extend’ the findings of the RCT, as Norden puts it, and offer researchers, clinicians, and the FDA insight into how drugs work in populations that don't find their way into clinical trials.” Full

A Patient-Centered Model of Congenital Thrombotic Thrombocytopenic Purpura Symptoms and Effects (8/14, Sonali Gattani, Hematology Advisor) reports “In order to get an in-depth understanding of patient experience, researchers developed a conceptual model of congenital thrombocytopenic purpura (TTP) that described its symptoms, effects, and disease burden. They reported their findings in The Patient — Patient-Centered Outcomes Research.” Full

Journals

Patient Experience with Congenital (Hereditary) Thrombotic Thrombocytopenic Purpura: A Conceptual Framework of Symptoms and Impacts Abiola O. Oladapo, et al. July 30, 2019, The Patient - Patient-Centered Outcomes Research Springer

Barriers and Facilitators to Conduct High-Quality, Large-Scale Safety and Comparative Effectiveness Research: The Biologics and Biosimilars Collective Intelligence Consortium Experience Lockhart CM, et al. August 11, 2019, Pharmacoepidemiology and Drug Safety PubMed

Articles

Best Ideas Aren't Always (or Often) From Washington (8/13, National Pharmaceutical Council: E.V.I.dently Today) comments “In his latest commentary for Chain Drug Review, National Pharmaceutical Council President and CEO Dan Leonard explores the health care concerns and ideas for improvement discussed at recent regional town halls hosted by the Healthcare Leadership Council and NPC.” Full

Best Ideas Aren't Always (Or Often) from Washington (8/12, Dan Leonard, Chain Drug Review) comments “...Earlier this spring, the National Pharmaceutical Council partnered with the Healthcare Leadership Council to hold town hall meetings in Raleigh, N.C., and Nashville. We asked health care leaders in those communities what really matters to them...One of the key themes that emerged from these town hall conversations is that, while the national conversation has been intensely focused on health care spending, it's more important to focus on getting maximum value for the dollars spent. In fact, sometimes it makes sense to spend more in certain areas, especially when the expenditure serves a public good. This is true for preventive care.” Full

New Research Questions FDA's Reliance on and Inability to Enforce Postmarketing Requirements (8/13, Zachary Brennan, Regulatory Focus) reports “...And as FDA continues its push toward further using real-world evidence, one current FDA official told [Matthew Herder, director of the Health Law Institute at Dalhousie University]: ‘You just let patients take them, and somehow all of these massive insurance databases will cough up an answer at the end of a year or two about how well the products work and how safe they are. We're not really anywhere near that degree of sophistication in our ability to analyze those big datasets, but there's a huge amount of pressure to push off lots of data collection there without a lot of methodology to do it.’” Full

Datavant, PCORnet to Build Real-World Data Network Spanning 60M Patients (8/13, Conor Hale, Fierce Biotech) reports “...[PCORnet’s] hundreds of data sets will be able to employ Datavant's token-based ecosystem, which aims to preserve the privacy of patients and study participants while also allowing researchers to share real-world data. Datavant and the [People-Centered Research Foundation] say that unlocking access and merging the findings could create one of the world's richest sources of patient outcomes research.” Full

Journals

Assessing Heterogeneity of Treatment Effect Analyses in Health-Related Cluster Randomized Trials: A Systematic Review Monique Anderson Starks, et al. August 12, 2019, PLOS One PLOS One

Assessing Value in Health Care: Using an Interpretive Classification System to Understand Existing Practices Based on a Systematic Review Brayan V. Seixas, et al. August 13, 2019, BMC Health Services Research BMC Health Services Research

Comparative Effectiveness of a Human Fibroblast-Derived Dermal Substitute and a Viable Cryopreserved Placental Membrane for the Treatment of Diabetic Foot Ulcers Michael L Sabolinski and John V Capotorto August 13, 2019, Journal of Comparative Effectiveness Research Journal of Comparative Effectiveness Research

The Potential Impact of CAR T-Cell Treatment Delays on Society Julia Thornton Snider, PhD, et al. August 13, 2019, The American Journal of Managed Care AJMC

Articles

Podcast: Non-Binding Guidance: Real-World Evidence in Drug Development and FDA Submissions (8/12, Ropes & Gray) reports “...In this episode, Ropes & Gray lawyers Kellie Combs and Sarah Blankstein discuss FDA's current thinking on real-world evidence and how industry has been using real-world evidence studies in FDA submissions, as well as with payors and in product promotion. The speakers highlight key takeaways from FDA's framework for real-world evidence, issued in December 2018, including the types of data sources and study designs that FDA considers to be ‘real-world,’ and the Agency's approach to assessing real-world evidence intended to support a determination of effectiveness.” Full

Video: Payer Perspective and Value of Treatment of SMA (8/12, The American Journal of Managed Care) “Surya Singh, MD, provides a payer perspective on the value of treatment for patients with SMA.” View Video

People Show Greater Interest to Know Current Local Medical Research (8/12, News Medical) reports “...‘People are interested and keen to know more, and it's vitally important to stimulate interest in evidence-based medicine. Otherwise, we risk losing funding for medical research in the long run. Other options already exist, in Facebook groups and elsewhere, where academic medicine is seen as something undesirable that people want to avoid. If that attitude is furthertakes rooted in society in earnest, we're in danger of getting into a situation where tax revenues might be spent where evidence is lacking, and then there'd be less money for evidence-based medicine. And that's a crucial issue,’ [University of Gothenburg’s Ronny Gunnarsson] concludes.” Full

Skilled Nursing Wants Bigger Piece of ACO Pie. A New Model That Shows Paying Provides for Upgrades — and Outcomes — Could Pay Off (8/11, Kimberly Marselas, McKnight's Long-Term Care News) reports “Paying subsidies to offset the cost of making patient care more efficient could boost participation in the Medicare Shared Savings Plan and save Medicare up to 40%. Those are the findings of a new data-driven model that examines why more providers aren't benefiting from related accountable care organizations, which covered one-sixth of all Medicare enrollees in 2017. They found one major obstacle was the cost of investing in improvements such as information technology needed for care coordination or quality improvements meant to target readmission rates.” Full

Another No For Vertex As Scotland Rejects Offer For Orkambi And Symkevi (8/12, Neena Brizmohun, Pink Sheet) reports “...The SMC announced today that it could not accept Orkambi (lumacaftor/ivacaftor) or another CF drug made by Vertex, Symkevi (tezacaftor/ivacaftor), because the company had failed to justify the price it was asking. The consortium said it came to its decision despite strong evidence from patient groups about the potential benefits of both medicines on patients' symptoms, reducing the need for hospital admissions and the overall burden of care.” Paid Subscription Required

Journals

Evidence Based Medicine V; How to Use in Clinical Practice Magdalena Fossum, et al. August 9, 2019, Journal of Pediatric Urology Journal of Pediatric Urology

Cost Effectiveness of Interval Cytoreductive Surgery With Hyperthermic Intraperitoneal Chemotherapy in Stage III Ovarian Cancer on the Basis of a Randomized Phase III Trial Simone N. Koole, MD, et al. August 10, 2019, Journal of Clinical Oncology Journal of Clinical Oncology

Articles

The Evolution of Real-World Studies (8/9, Nick Taylor, PMLiVE) comments “...The growing role of RWE in the US is supported by the 21st Century Cures Act, which led the FDA to create a framework for incorporating RWD into regulatory decisions, commit to publish guidance on the specifics and create the MyStudies app to support collection of results in the real world. Yet, the industry is pressing the FDA to go faster. In feedback on the framework, the American Society of Clinical Oncology raised concerns that the FDA is ‘overly conservative’, while others including the Biotechnology Innovation Organization pushed for the agency to clearly state that RWE can support new drug approvals when two or more RCTs are not necessary or feasible.” Full

CAM Therapies Evaluated for the Treatment of Clinical Depression (8/9, Diana Ernst, RPh, Monthly Prescribing Reference) reports “...To better understand the safety and efficacy of CAM in the treatment of clinical depression, study authors searched various databases for randomized clinical trials assessing CAM as monotherapy or in combination with other treatments. Studies were included if they evaluated the effect of CAM on depression severity, response, remission, and relapse, in addition to adverse events; the GRADE method was used to assess the quality of evidence.” Full

UK's NICE Not Convinced By Cannabis-Based Medicines (8/9, Ian Schofield, Pink Sheet) reports “...NICE, the health technology assessment body for England and Wales, has issued a new draft guideline that recommends against using cannabis-based medicines to treat chronic pain because they are of only ‘modest’ efficacy in this indication. It says that GW Pharmaceuticals' Sativex (cannabidiol spray) should not be used for treating spasticity in multiple sclerosis because it is not cost effective. This is in line with NICE's earlier advice and also takes account of a new economic model developed specifically for this guidance.” Paid Subscription Required

Press Releases

ICER to Assess Treatments for Sickle Cell Disease (8/9, ICER Press Release) “The Institute for Clinical and Economic Review announced today that it plans to assess the comparative clinical effectiveness and value of crizanlizumab (Novartis) and voxelotor (Global Blood Therapeutics) for the treatment of sickle cell disease. Crizanlizumab, a P-selectin inhibitor, is currently under FDA priority review with an anticipated decision expected in the first half of 2020. Global Blood Therapeutics is planning to initiate and complete a rolling NDA for voxelotor, an HbS polymerization inhibitor, before the end of 2019.” Full

Journals

Comparative Effectiveness of First-Line Nab-Paclitaxel Versus Paclitaxel Monotherapy in Triple-Negative Breast Cancer Patricia Luhn August 9, 2019, Journal of Comparative Effectiveness Research Journal of Comparative Effectiveness Research

Events

ISPOR Summit 2019 on Building Trust in RWE – The Role of Study Registration October 11, 2019 Baltimore Marriott Inner Harbor at Camden Yards 110 South Eutaw Street Baltimore, MD National Pharmaceutical Council