CER Daily Newsfeed®
Articles
Supporting Patients in Peer Review Can Improve the Relevance of Research (1/15, Kelly J. Vander Ley, PhD, The BMJ Opinion) comments “...Given that patient review programmes are still in their infancy, the utility of review comments provided by patient peer reviewers has yet been fully established. Still, a growing body of evidence suggests patient comments help authors improve quality of the research manuscripts. Since the inception of the [PCORI] Peer Review Program in 2016, 175 patient, caregiver, or patient advocate reviewers have provided insights and recommendations on over 280 PCORI-funded research reports. From our experience managing peer review for PCORI, we have seen firsthand how patient reviews improve the quality of the research reports.” Full
Lagging COVID Antibody Uptake Continues To Worry US Government (1/14, Sarah Karlin-Smith, Pink Sheet) reports “...The reasons for the limited uptake are ‘multifactorial,’ said Janet Woodcock, Warp Speed’s therapeutic lead and FDA’s principal medical advisor to the commissioner. ‘We don’t have the kind of evidence people are used to,’ she said noting the medicines were developed in about nine months. Woodcock said that additional randomized controlled trial data, along with real-world evidence should eventually add to the current knowledge base but envisioned there might always be some gaps.” Paid Subscription Required
UK: NICE Backs GSK’s Zejula in Advanced Ovarian Cancer (1/15, Lucy Parsons, PharmaTimes) reports “The UK’s National Institute for Health and Care Excellence (NICE) has recommended the use of GlaxoSmithKline’s Zejula (niraparib) on the NHS for women with newly diagnosed advanced ovarian cancer...’GSK is delighted that all eligible women in England with advanced ovarian cancer will soon be able to potentially benefit from Zejula on the NHS,’ said Marc Clausse, head of the UK Oncology Business Unit, GSK.” Full
UK: Speaking with One Language: How Using a Common Data Model Might Speed up Decision Making (1/15, Seamus Kent and Pall Jonsson, NICE Blog) comments “...Common data models can significantly increase the speed with which evidence can be generated without compromising rigour and while enhancing transparency. This could help us ensure patients get faster access to innovative treatments in areas of unmet need. NICE will continue to work within the EHDEN project post Brexit to help realise these potential benefits for patients in the UK and elsewhere.” Full
Journals
Comparative Efficacy of Dabrafenib + Trametinib Versus Treatment Options for Metastatic Melanoma in First-Line Settings Jing Wu, et al. January 15, 2021, Journal of Comparative Effectiveness Research Journal of Comparative Effectiveness Research
Events
PCORI Board of Governors Meeting February 9, 2021 9:00AM - 5:00PM EST PCORI
Articles
Secukinumab Associated With Greater PASI 2 or Lower Achievement vs Ustekinumab in Psoriasis (1/13, Brandon May, Dermatology Advisor) reports “Treatment with secukinumab was associated with a greater likelihood of achieving a PASI of 2 or lower after 12 months compared with treatment with ustekinumab in patients with psoriasis, according to study data published in JAMA Dermatology.” Full
Journals
Building a Better Clinician Value-Based Payment Program in Medicare Kenton J. Johnston, PhD; Jason M. Hockenberry, PhD; Karen E. Joynt Maddox, MD, MPH December 18, 2020, JAMA JAMA
Health Technology Assessment With Diminishing Returns to Health: The Generalized Risk-Adjusted Cost-Effectiveness (GRACE) Approach Darius N. Lakdawalla, Charles E. Phelps, PhD January 12, 2021, Value in Health Value in Health
Articles
ICER Pricing Report: Creating a Dialogue or Just Playing for Headlines? (1/12, National Pharmaceutical Council: E.V.I.dently Today) comments “...In its new report, ICER again ignored relevant economic information – the kind used by payers in their decision-making – to paint an unflattering and inaccurate picture of industry. Health researchers have found that over time, some medicines reduced health system spending, such as through their route of administration or a reduced need for doctor visits. This type of economic information, viewed as important by most health care payers, was not considered...Going forward, we need to ask critical questions and have a broader dialogue before citing flawed reports like ICER's. Flawed information can lead to flawed policy decisions, and that won't benefit patients or society. This report may get headlines -- it won't, however, create a productive dialogue.” Full
The Prices of 7 Drugs Were Hiked Without Proof of New Benefits, Costing the U.S. $1.2 Billion in 2019 (1/12, Ed Silverman, STAT Plus) reports “...[T]he National Pharmaceutical Council criticized the [ICER] report and cited an analysis that found net prices overall declined by 2.2% over the past five years. ‘Given those declines in prices, the Institute for Clinical and Economic Review's second report on drug pricing – focusing on a hand-picked, narrow group of medicines rather than the overall landscape – is an unnecessary and misleading exercise. ICER used incomplete evidence and inadequate analyses, creating a flawed report that should not be relied on by any policy- or decision-maker interested in developing sound, evidence-based drug policy.’” Paid Subscription Required
Copay Assistance Programs Help Patients but Confuse Them Too (1/13, Joyce Frieden, Medpage Today) reports “...Insurers are notified when their patients use copay coupons, and many of them use the data to compile ‘copay accumulator’ records for enrollees. Often, insurers won't allow the money paid by drugmakers to count toward the patient's deductible or out-of-pocket costs. These rules can be confusing for patients, who may be unaware of the practice. ‘They think they've met the total amount and then they're surprised when the plan says, “We've accumulated all those coupons and they don't count for out-of-pocket costs,”’ Jennifer Graff, vice president for policy research at the National Pharmaceutical Council, said...” Full
External Beam RT Associated With Highest Financial Burden for Prostate Cancer (1/14, Journal of Clinical Pathways) reports “Among contemporary treatment modalities for patients with prostate cancer, external beam radiotherapy was associated with the highest financial burden, according to a recent study published in Journal of Urology...‘Prospective studies that directly measure out-of-pocket and indirect costs and account more thoroughly for baseline socioeconomic differences are warranted in order to identify those most at risk,’ they added.” Full
Industry, FDA Begin to Hammer Out PDUFA VII Details (1/13, Kari Oakes, Regulatory Focus) reports “...The use of real-world evidence and real-world data (RWE/RWD) is addressed both by PDUFA VI and the 21st Century Cures Act. “These commitments included initiating appropriate activities to address key issues in the use of RWE for regulatory decision-making purposes and publishing draft guidance on how RWE can contribute to the assessment of safety and effectiveness in regulatory submissions,” according to the meeting minutes. A point made by FDA during this portion of the meeting is that real-world data sources can augment, but not supplant, clinical trials during the regulatory decision-making process.” Full
Next Generation ACOs Saved Medicare $558M in 2019 (1/14, Jacqueline LaPointe, RevCycle Intelligence) reports “...So far, the data shows that Next Generation ACOs reduced Medicare spending on beneficiaries attributed to the providers while maintaining an average quality score of 93.7 percent out of a perfect score of 100, the National Association of ACOs reported. Most Next Generation ACOs earned shared savings payments as a result of spending reductions...‘For every year of the program, Next Gen ACOs yielded savings for Medicare money while also showing an improvement in quality. Very few programs CMS has developed over the years can say that,’ said Clif Gaus, ScD, president and CEO of NAACOS.” Full
Journals
Network Meta-Analysis of Nivolumab plus Ipilimumab in the Second-line Setting for Advanced Hepatocellular Carcinoma Neehar D Parikh, et al. January 14, 2021, Journal of Comparative Effectiveness Research Journal of Comparative Effectiveness Research
Events
PCORI 2021 and Beyond: Opportunities for Funding and Involvement in Patient-Centered Research February 2, 2021 2:00 - 2:30PM EST PCORI
Articles
ICER Pricing Report: Creating a Dialogue or Just Playing for Headlines? (1/12, National Pharmaceutical Council: E.V.I.dently Today) comments “...In its new report, ICER again ignored relevant economic information – the kind used by payers in their decision-making – to paint an unflattering and inaccurate picture of industry. Health researchers have found that over time, some medicines reduced health system spending, such as through their route of administration or a reduced need for doctor visits. This type of economic information, viewed as important by most health care payers, was not considered...Going forward, we need to ask critical questions and have a broader dialogue before citing flawed reports like ICER's. Flawed information can lead to flawed policy decisions, and that won't benefit patients or society. This report may get headlines -- it won't, however, create a productive dialogue.” Full
The Prices of 7 Drugs Were Hiked Without Proof of New Benefits, Costing the U.S. $1.2 Billion in 2019 (1/12, Ed Silverman, STAT Plus) reports “...[T]he National Pharmaceutical Council criticized the [ICER] report and cited an analysis that found net prices overall declined by 2.2% over the past five years. ‘Given those declines in prices, the Institute for Clinical and Economic Review's second report on drug pricing – focusing on a hand-picked, narrow group of medicines rather than the overall landscape – is an unnecessary and misleading exercise. ICER used incomplete evidence and inadequate analyses, creating a flawed report that should not be relied on by any policy- or decision-maker interested in developing sound, evidence-based drug policy.’” Paid Subscription Required
FDA Lays out 2021 Regulatory Science Areas of Focus (1/11, Kari Oakes, Regulatory Focus) reports “...The final data-centric area of focus is the increasing importance of real-world evidence in the conduct of clinical trials and postmarketing review. Following the establishment of the RWE program under the 21st Century Cures Act, ‘FDA is committed to exploring the use of RWE in regulatory decision-making, including its ability to provide fit-for-purpose clinically meaningful information about the safety and effectiveness of medical products,’ according to the [Focus Areas of Regulatory Science] report.” Full
Press Releases
ICER Identifies Most Significant 2019 US Drug-Price Hikes — Even After Rebates — That Were Not Supported by New Clinical Evidence (1/12, ICER Press Release) “...Among the top drugs with price increases in 2019 that had substantial effects on US spending, ICER determined that seven of 10 lacked adequate new evidence to demonstrate a substantial clinical benefit that was not yet previously known. The 2019 unsupported price increases on these seven treatments, even after pharmaceutical rebates and other concessions, cost the US health system an additional $1.2 billion beyond what would have been spent if their net prices had remained flat.” Full
Scientists Receive $3.4 Million to Study Care Options for Patients with Chest Pain Due to Anxiety (1/11, Regenstrief Institute Press Release) “Indiana University School of Medicine and Regenstrief Institute research scientists have received a $3.4 million award from the Patient-Centered Outcomes Research Institute to investigate the best care options for the 40 percent of people who go to the hospital emergency department with chest pain that is determined to be caused by anxiety rather than a cardiac event.” Full
Journals
STaRT-RWE: Structured Template for Planning and Reporting on the Implementation of Real World Evidence Studies Shirley V Wang, et al. January 12, 2021, BMJ BMJ
Choosing Important Health Outcomes for Comparative Effectiveness Research: 6th Annual Update to a Systematic Review of Core Outcome Sets for Research Elizabeth Gargon, Sarah L. Gorst , Karen Matvienko-Sikar, Paula R. Williamson January 12, 2021, PLOS One PLOS One
Articles
Forging a Path Toward a More Stable and Patient-Centric Health System in 2021 (1/6, National Pharmaceutical Council: E.V.I.dently Today) comments “In his commentary for Chain Drug Review, NPC Interim President and Chief Executive Officer Robert Dubois, MD, PhD, explores the steps we can take to improve our current health care system in the wake of the COVID-19 pandemic. To do this successfully, Dr. Dubois says that we need to focus on three key elements: appropriately managing health care spending, addressing patients' concerns about costs and access to treatments, and providing incentives for sustained innovation.” Full
Pharmacy Outlook: Robert Dubois, NPC (1/8, Robert Dubois, Chain Drug Review) comments “...The second requirement needed to address the challenges of 2021 will be making sure that the patient is front and center in any systemic changes that are considered. There will almost certainly be more of a focus on health technology assessments, making it critical that we understand what is measured when we measure ‘value’ — what's valuable to an insurance company may not match what's valuable to the patient. NPC has already published guiding practices on the use of value assessment, which can offer an excellent road map to whether a given approach measures what is truly important, especially for patients.” Full
What's In Your RWE Evaluation Toolbox? (1/6, National Pharmaceutical Council: E.V.I.dently Today) comments “...‘Although RWE has been growing, citations of these studies in formulary and coverage decisions remain infrequent,’ said Jennifer Graff, PharmD, vice president of policy research at the National Pharmaceutical Council, and a co-author of the article along with authors from the University of Washington. ‘In a survey of health plan pharmacy and medical directors, most believe RWE can help make more informed decisions, but few feel adequately prepared to evaluate or use RWE. Over 50% of surveyed health plan pharmacy and medical directors noted a pivotal barrier to RWE use was their personal or their organization's lack of experience interpreting RWE studies,’ she said.” Full
EMA Will Use Panalgo Software for Real-World Data Analytics (1/8, Phil Taylor, PharmaPhorum) reports “...Panalgo has won a contract to supply the European Medicines Agency with its IHD data analytics platform, pledging to streamline its public health efforts. IHD – or Instant Health Data – will be used by the EMA to carry out data analyses and examine medicinal product utilisation, answer questions about safety and efficacy, and understand how treatments perform in real-world settings, said the Boston, US-based company.” Full
Press Releases
C-Path PRO Consortium Measure Used to Support an FDA-Approved Label Claim (1/7, Critical Path Institute Press Release) “...‘The multi-stakeholder collaboration within the Critical Path Institute's PRO Consortium, inclusive of sponsors, patients, measurement experts and FDA has been critical in being able to incorporate the patient's voice into a development program and product labeling. This is a huge win for patients to understand the impact of treatment on symptoms that matter to them and inform their treatment decision-making for this burdensome condition,’ stated Robyn T. Carson, MPH, Vice President, Patient-Centered Outcomes Research, at AbbVie and co-chair of the PRO Consortium's IBS Working Group.” Full
ICER Publishes Final Evidence Report and Policy Recommendations for Supervised Injection Facilities (1/8, 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 supervised injection facilities, as a means to reduce harm among people who inject drugs. This assessment is another facet of ICER’s work to deliver independent evaluations of the clinical evidence related to a wide-range of potential interventions addressing the opioid crisis, including abuse-deterrent opioid formulations, medication-assisted treatment for opioid use disorder, non-drug interventions for chronic lower back pain, and digital health therapeutics for OUD.” Full
NICE Is Gearing up for the Evaluation of Further Digital Health Technologies as Pilot Project Produces First Result (1/8, NICE Press Release) “...The guidance is published and it is official. NICE has successfully completed the first assessment using the digital health technologies guidance development pilot project. The Zio XT service, which is used for detecting abnormal heart rhythms, is recommended as an option while further data is collected to address evidence gaps about the benefit for patients and the NHS.” Full
Journals
Online Tools to Synthesize Real-World Evidence of Comparative Effectiveness Research to Enhance Formulary Decision Making Shuxian Chen , MA, Jennifer Graff , PharmD, Sophia Yun , PharmD, Brennan Beal , PharmD, Jamie T Ta , PharmD, MS, Aasthaa Bansal , PhD, Joshua J Carlson , MPH, PhD, David L Veenstra , PharmD, PhD, Anirban Basu , PhD, Beth Devine , PhD, PharmD, MBA January 2021, Journal of Managed Care & Specialty Pharmacy JMCP
Articles
With US Senate Wins In Georgia, Democrats Gain Path For Rx Price Reform, But It May Be Lower Priority (1/6, Cathy Kelly, Pink Sheet) reports “...Launched in 2011, the German approach is based on a centralized assessment of manufacturer dossiers and other information on new drugs, a determination of how much added benefit they offer, and negotiation on a national price...If a drug is shown to offer added value, the national organization representing insurers negotiates with manufacturers on a price. If no value is demonstrated, the drug’s price is capped at what is being charged for comparable drugs. The pharma industry believes that criteria for demonstrating value in the German system is too restrictive. But it may not look so bad compared to HR 3.” Paid Subscription Required
Industry Collaboration Aims to Advance the Development of Synthetic Arms in Clinical Trials (1/7, Scientific Computing World) reports “Phesi, a data-driven provider of AI-powered clinical development analytics products and solutions, has announced it has entered into an exclusive strategic collaboration with clinical AI technology company, Sensyne Health...Together, the companies will provide an enhanced offering of clinical trial and real-world data predictive analytics, enabling life sciences organisations to quickly and safely determine the feasibility of replacing or supplementing a comparator or placebo arm with a synthetic arm.” Full
UK: NICE to Reject Bristol Myers' Opdivo in Head and Neck Cancer after 3 Years of Restricted Coverage (1/7, Angus Liu, Fierce Pharma) reports “Three years after offering Bristol Myers Squibb’s Opdivo limited coverage in head and neck cancer, England’s cost watchdog is poised to back away entirely. After reviewing new evidence, the National Institute for Health and Care Excellence concluded that Opdivo’s not worthy of coverage for recurrent or metastatic squamous cell carcinoma of the head and neck after platinum-based chemotherapy, according to draft guidance published Thursday.” Full
Journals
Practices of Decision Making in Priority Setting and Resource Allocation: A Scoping Review and Narrative Synthesis of Existing Frameworks Brayan V. Seixas, François Dionne & Craig Mitton January 7, 20201, Health Economics Review Health Economics Review