CER Daily Newsfeed®
Articles
Making Real World Evidence Fit for Purpose (1/22, George Underwood, PharmaPhorum) reports “...‘We see a growing need to contextualise clinical trial data, particularly for niche conditions where it is not feasible and sometimes not ethical to randomise patients,’ says Nancy Dreyer, chief scientific officer at IQVIA and co-author of the report, explaining the impetus behind the study. ‘Currently there are no standards to determine when RWE is “regulatory grade”… There are also questions about the qualifications and purity of motives of those who conduct the data extraction, assembly and analysis.’” Full
Why Collaboration is Vital to Value-Based Arrangements (1/22, LillyPad) comments “...The evolution to VBAs that are more patient focused will require increased collaboration between payers, health systems, patients, and industry, said Eric Klein, Lilly's senior director, oncology, global patient outcomes and real-world evidence. ‘The value-based arrangements we are implementing in the market are already helping all of us learn how to effectively implement these agreements and measure value in the real world,’ Klein said. ‘Lilly is committed to moving toward a health care model based on value and not purely volume, and we'll continue to share learnings with patient advocates and payers to further enhance those positive steps forward.’” Full
What the Latest ACO Numbers Mean for the Future of the MSSP (1/22, Jacqueline LaPointe, RevCycle Intelligence) reports “...‘In the CMS blog they cited 53 new ACOs in 2020 and we cite 35,’ Allison Brennan, senior vice president of government affairs at the National Association of ACOs, recently told RevCycleIntelligence in an interview. The 18 remaining ACO applications are not truly ‘new’ to the program, Brennan continued. These ACOs were in the MSSP before and are now reentering the program despite their new start date of Jan. 1, 2020. Some of these organizations have even participated in two performance periods in the past.” Full
England's NICE Future Proofs Approach To Using Data (1/22, Francesca Bruce, Pink Sheet) reports “...NICEhas published a finalized statement of intent on how it proposes to broaden its use of data when developing guidance and advice. The updated statement expands the breadth of data sources that NICE will consider to include patient reported outcomes and genomic data...NICE told the Pink Sheet that it ‘will be working on methods and processes documentation this year. We do not currently have a release date as this is contingent on a number of factors.’” Paid Subscription Required
UK: NICE Shuts Out AstraZeneca's Tagrisso—and Dings J&J's Stelara, Too (1/22, Carly Helfand, Fierce Pharma) reports “It's final: England won't be covering AstraZeneca's quick-selling Tagrisso in previously untreated non-small cell lung cancer patients with EGFR mutations...AstraZeneca isn't the only Big Pharma player to run into trouble with NICE this week. Monday, the body released draft guidance rejecting Johnson & Johnson's Stelara in ulcerative colitis...” Full
Journals
Strengthening Pharma's Contract with Society: The Value of Trusted Partnerships Between Pharma and Healthcare Facilitated by Real-World Data Michael J Seewald, et al. January 22, 2020, Journal of Comparative Effectiveness Research Journal of Comparative Effectiveness Research
Events
Webinar: AMCP Format v4.1: New Guidance on Evidence Requirements for Unapproved Products and Unapproved Uses January 23, 2020 2:00 - 3:00PM ET Academy of Managed Care Pharmacy
Articles
Women Matter in Value Assessments (1/21, National Pharmaceutical Council: E.V.I.dently Today) comments “...On January 23 in Chicago, ICER will hold a public meeting to discuss treatments for acute migraine, a debilitating condition that is three times more common in women than men and is the second leading cause of global disability burden. [The Society for Women's Health Research’s] Sarah Wells Kocsis, MBA, vice president of public policy, will participate in the meeting to share SWHR's concerns and perspectives on ICER's assessment process and evidence report findings. The National Pharmaceutical Council caught up with Ms. Wells Kocsis to learn more about SWHR's approach to value assessment and what they hope ICER will focus on during the January 23 meeting.” Full
GBTS Forum Webinar: How Do We Define And Navigate Low-Value Care? (1/20, Going Below the Surface) comments “...While estimates on low-value care use and spending allow us to understand the scope of the problem, identifying where these issues occur and at what cost can better inform de-implementation priorities and strategies. That means stakeholders need new guidance or tools – as well as the awareness of the tools that do exist – to prioritize which areas of low-value care to tackle. Additionally, stakeholders must continue to ask the tough questions about the ‘never,’ ‘rarely’ and ‘never repeated’ services. The Going Below The Surface Forum is working on a roadmap to navigate these issues and better arm stakeholders to address low-value care at a macro level, which will be rolled out in early spring.” Full
Value Analysis of JAK Inhibitors for RA Hampered by Limited Data (1/17, Jake Remaly, MDedge Internal Medicine) reports “Adequate evidence shows that adding a Janus kinase (JAK) inhibitor to conventional disease-modifying antirheumatic drug therapy provides a net health benefit for patients with rheumatoid arthritis, compared with conventional drugs alone, according to a report by an independent research institute. But the long-term economic value of JAK inhibitors for rheumatoid arthritis is less clear, the report by the Institute for Clinical and Economic Review indicates.” Full
Pfizer's Big Data Exec on Pharma's ‘Arms Race' to Partner with Companies like Fitbit, 23AndMe, and Others (1/21, Megan Thielking, STAT Plus) reports “...[Pfizer’s Christopher Boone:] We don't necessarily generate real-world data. We generate clinical trial data and evidence, but we don't generate real-world evidence right now. So we have to rely on our partnerships in order to get access to that data and conduct analyses. So the arms race is to solidify what I like to refer to as the data supply chain. Everyone wants to shore up their real-world data supply chain to ensure they have an adequate supply of data in order to demonstrate the value and the effectiveness of our medicines.” Full
Podcast: Patient-Centered Pathways in the Community: Utilizing RWE, AI Tools (1/21, Journal of Clinical Pathways) reports “Ray Page, DO, PhD, FACOI, president and director of research, The Center for Cancer and Blood Disorders (Fort Worth, TX), explains the inherent patient-centered nature of clinical pathways at his institution as well as the ability of real-world evidence and artificial intelligence tools to further personalize care.” Full
Journals
ASH 2019: Real-World Evidence and Cost of Care Maggie L. Shaw, Mary Caffrey January 2020, Evidence-Based Oncology AJMC
Envisioning a Better U.S. Health Care System for All: Health Care Delivery and Payment System Reforms Shari M. Erickson, MPH, et al. January 21, 2020, Annals of Internal Medicine Annals of Internal Medicine
"Original Sin" and U.S. Health Care Reform David Meltzer, MD, PhD January 21, 2020, Annals of Internal Medicine Annals of Internal Medicine
Crizotinib Versus Chemotherapy: A Real-World Cost–Effectiveness Study in China Meijuan Huang, et al. January 21, 2020, Journal of Comparative Effectiveness Research Journal of Comparative Effectiveness Research
Cost–Effectiveness Analysis of Ocriplasmin Versus Watchful Waiting for Treatment of Symptomatic Vitreomacular Adhesion in the US Arshad M Khanani, et al. January 21, 2020, Journal of Comparative Effectiveness Research Journal of Comparative Effectiveness Research
Articles
Overheard at J.P. Morgan: Perspectives on EQRx (1/17, ICER Blog) comments “One of the more interesting announcements from this week's JP Morgan Healthcare Conference was the launch of a new company that intends to create me-too products at prices up to 66% lower than the existing branded drugs. The company, EQRx, will be led by Alexis Borisy, a serial biotech entrepreneur...[W]e are hopeful that EQRx is successful, and that they are able to help the entire industry focus more on aligning a drug's price with how well it improves patients' lives.” Full
Press Releases
NICE Encourages Further Data Collection on ‘Game Changing' Histology Independent Cancer Drugs (1/17, NICE Press Release) “Larotrectinib (also called Vitrakvi and made by Bayer), a new treatment for a range of cancers, can't be recommended for use in the NHS because at its current price, it doesn't have the potential to be cost-effective. Another histology independent treatment, entrectinib (made by Roche), which NICE is also appraising could become the first histology independent treatment to be available to patients, provided it receives its marketing authorisation.” Full
Journals
Use of Patient Preference Studies in HTA Decision Making: A NICE Perspective Bouvy JC, et al. January 16, 2020, The Patient PubMed
Implementing Evidence-Informed Deliberative Processes in Health Technology Assessment: A Low Income Country Perspective Kapiriri L, Baltussen R, Oortwijn W. January 16, 2020, International Journal of Technology Assessment in Healthcare PubMed
Health Technology Assessment in Japan: A Work in Progress Kamae I, et al. January 16, 2020, Journal of Medical Economics PubMed
Proton Pump Inhibitors vs Histamine-2 Receptor Blockers for Stress Ulcer Prophylaxis in Critically Ill Patients: Issues of Interpretability in Pragmatic Trials Todd W. Rice, MD, MSc, et al. January 17, 2020, JAMA JAMA
Biomedical Conferences' Author Instructions Rarely Mention Guidelines for Reporting Abstracts of Trials and Systematic Reviews Lenko Saric, et al. January 17, 2020, Journal of Comparative Effectiveness Research Journal of Comparative Effectiveness Research
Reports
Cost Differences for Oncology Medicines Based on Site of Treatment January 16, 2020 Employee Benefit Research Institute
Articles
Fewer Interventions after Sleeve Gastrectomy Vs. Roux-en-Y, Real-World Data Show (1/15, Andrew D. Bowser, MDedge Internal Medicine) reports “Sleeve gastrectomy was associated with significantly fewer postsurgical interventions and operations than was Roux-en-Y gastric bypass over longer-term follow-up in a recent cohort study based on real-world clinical data, according to investigators...The benefit of sleeve gastrectomy seemed to be most pronounced in patients with lower body mass index and those with fewer comorbidities...” Full
Germany's IQWiG Finds First Tumor-Agnostic Drug Provides No Added Benefit (1/16, Zachary Brennan, Regulatory Focus) reports “Germany's Institute for Quality and Efficiency in Health Care (IQWiG) this week said that Bayer's Vitrakvi (larotrectinib), approved in the EU last summer, has not been proven to provide an added benefit. Vitrakvi was the first medicine to be approved in the EU as a treatment for tumors with a specific mutation, regardless of where in the body the tumor originated. But the treatment's safety and efficacy were evaluated in three single-arm trials, and IQWiG took issue with the lack of comparator arms in the trials.” Full
Press Releases
Breast Cancer Patients to Have Further NICE-Approved Drug Combination Option on Cancer Drugs Fund (1/16, NICE Press Release) “Another potentially life-extending drug combination for some people with advanced breast cancer will now be available on the Cancer Drugs Fund following its approval by NICE in final guidance published today. The guidance recommends palbociclib (also called Ibrance and made by Pfizer) given with fulvestrant for people with a type of advanced breast cancer called hormone receptor-positive, human epidermal growth factor receptor 2 (HER2)-negative breast cancer who have already had endocrine therapy.” Full
Journals
Evidence-Based Medicine Self-Assessment, Knowledge, and Integration into Daily Practice: A Survey among Romanian Physicians and Comparison Between Trainees and Specialists Roxana-Denisa Capras, et al. January 16, 2020, BMC Medical Education Springer
Articles
National Heart, Lung, and Blood Institute Cardiologist Nakela Cook, M.D., to Lead PCORI (1/15, David Raths, Healthcare Innovation) reports “...Cook...comes to PCORI from her role as a Senior Scientific Officer and Chief of Staff for the National Heart, Lung, and Blood Institute...She will assume her new position April 15, taking over leadership of PCORI from Interim Executive Director Josephine P. Briggs, M.D...‘PCORI's mission fully reflects my personal and professional values and commitment to promote excellence in health and healthcare in a way that is patient-centered and engages all parties to help us achieve shared goals,’ she said in a statement.” Full
US FDA's Decade In Review: From Biosimilars To Real-World Evidence (1/15, Brenda Sandburg, Pink Sheet) reports “...FDA has used real-world evidence – the data about the usage and potential benefits or risks of a drug that are derived from sources other than traditional clinical trials – to evaluate the postmarket safety of products, and in some instances has accepted RWE to support drug development approvals. But the agency is going further under the framework, establishing demonstration projects and increasing engagement with stakeholders.” Paid Subscription Required
CVS, Cigna Execs Tell JPM20 They're Ready for a Contentious Election Year (1/15, Rebecca Pifer, Healthcare Dive) reports “...[T]here's more to do to bring value-based care to the PBM model, CVS executives said Tuesday. ‘We do think there's an opportunity for PBMs to evolve on this front,’ [CVS CFO Eva Boratto] said. Within its payer business, roughly 65% of commercial and Medicare contracts are value-based, but in the PBM ‘you're essentially at the starting line.’ CVS plans to start with its condition-specific management programs, called Transform Care, which is outcomes-based, and combine that with its retail locations, including HealthHUBs, to move toward more integrated, value-based care.” Full
UK: NICE Rejects Keytruda, Asks for More Info (1/15, Anna Smith, PharmaTimes) reports “The National Institute for Health and Care Excellence has published a new draft guidance not recommending MSD's cancer blockbuster Keytruda (pembrolizumab) for untreated metastatic or unresectable recurrent head and neck squamous cell carcinoma. The organisation is calling for an urgent reassessment of the evidence for the drug's benefit, and has asked for more clinical and cost-effectiveness evidence from MSD.” Full
Press Releases
Patient-Centered Outcomes Research Institute Names Nakela Cook as New Executive Director (1/15, PCORI Press Release) “...‘On behalf of PCORI's Board and staff, and the health care community we serve, I am thrilled to welcome Dr. Cook as our new Executive Director,’ [PCORI Board of Governors Chairperson Christine Goertz, DC, PhD] said. ‘Her deep experience in research, in managing a large and complex research enterprise, and her strong commitment to patient-centered research and clinical practice make her exactly the right person to lead PCORI at this time. She will be an excellent steward of the vital work we will do in the years ahead, as we partner with patients, clinicians, payers, employers and other stakeholders to improve the health and health care of the people of this country.’” Full
Articles
Value Assessment Frameworks: Is There One "Best" Model? (1/10, Dan Leonard, STAT) comments “...Our findings support the idea that there is no one ‘best’ framework. They have different purposes, methods, inputs and outputs. Some have a narrower scope, focusing only on certain types of treatments; some are intended for insurers to use; and others are meant as shared decision-making tools to be used between patients and their physicians. Instead of looking to one framework to inform health care decisions, decision-makers should leverage multiple approaches in measuring value, thus providing a more robust understanding of a treatment and its implications for patient health.” Full
Looking Back and Looking Ahead (1/13, Josephine P. Briggs, MD, The PCORI Blog) comments “....The urgency that underlies our mandate remains; the need for rigorous patient-centered comparative clinical effectiveness research is as great as ever. We need better ways to move the results of that work into practice as quickly as possible and to provide stakeholders across the healthcare system with information that can better inform clinical decisions. In 2020 we will focus on building on our accomplishments and moving quickly, with the ongoing guidance of our stakeholder communities, to show that the confidence they and Congress have expressed in us is well placed.” Full
Oncologists Rely on Pharma Reps for Information (1/13, Thomas Sullivan, Policy & Medicine) reports “...According to the study, more than two-thirds of oncologists (71%) said that pharmaceutical sales representatives ‘play an important role in helping me learn about new drug products.’...The survey also asked about the preferred type of content oncologists want from pharmaceutical companies, beyond clinical and safety trial data. The top answer was patient outcome studies based on real-world evidence (24% of respondents), followed by comparative effectiveness studies (20%).” Full
Updated Guidance on Ulcerative Colitis Management (1/14, Physician's Weekly) reports “...[Millie D. Long, MD, MPH] notes a lack of randomized controlled trials (RCTs) focused on comparative effectiveness of medications, with studies mostly comparing medications with placebo. ‘This makes it difficult to make recommendations surrounding positioning of medications,’ she adds. ‘However, the large RCT UC Success demonstrated that infliximab combined with azathioprine was superior to either medication alone. In the future, we hope to see more comparative effectiveness studies that will help us surrounding questions of positioning therapies.’” Full
How CVS Health Is Addressing the Cost of Gene Therapies (1/13, Paige Minemyer, Fierce Healthcare) reports “...Gene therapy products are a perfect place to roll out a value-based contract, as manufacturers really believe in them and want to grow their use, Troy Brennan, M.D., executive vice president and chief medical officer at CVS Health, told FierceHealthcare. ‘They lend themselves to value-based contracting better than almost any other kind of therapy,’ Brennan said.” Full
JPM20: Why Humana Is Focusing Investments on Growing Clinical Services (1/13, Paige Minemyer, Fierce Healthcare) reports “...[Humana CEO Bruce Broussard] said the insurer is in the testing phase with such benefits and how customers use them, as offering them will likely either raise the member's cost or ‘cannibalize’ another potential benefit. Humana has deployed a number of pilots in different markets, including benefits targeting the social determinants of health and a value-based insurance design product, and it will use lessons from those as it moves forward, he said. ‘I think the industry is just learning and spending a bit of time to understand it,’ Broussard said.” Full
US FDA's Precision Medicine Efforts Turn Towards Adverse Events (1/13, M. Nielsen Hobbs, Pink Sheet) reports “...Principal Deputy Commissioner Amy Abernathy announced the project, a PrecisionFDA Data Challenge titled ‘Detecting Adverse Event Anomalies Using FDA Open Data,’ on 13 January. ‘This FDA Open Data Challenge encourages the development of computational algorithms for automatic detection of drug adverse event anomalies,’ the agency explained. ‘Participants will develop computational algorithms to identify adverse event anomalies in open FDA adverse event data.’” Paid Subscription Required
UK: NICE Backs Medtronic's AF Monitor in Post-Stroke Patients (1/14, Richard Staines, PharmaPhorum) reports “England's NHS could prevent hundreds of strokes of unknown cause with a new implantable monitor that transmits data to doctors remotely, which is recommended for funding following draft guidance from NICE. The Reveal LINQ monitor developed by Medtronic identifies more people who have atrial fibrillation after a cryptogenic stroke – with no identified cause – or transient ischaemic attack.” Full
Journals
Early Returns From the Era of Precision Medicine David M. Cutler, PhD January 14, 2020, JAMA JAMA
Cost-Effectiveness Analysis of Ezetimibe as the Add-on Treatment to Moderate-Dose Rosuvastatin versus High-Dose Rosuvastatin in the Secondary Prevention of Cardiovascular Diseases in China: A Markov Model Analysis Yang H, et al. January 14, 2020, Drug Design, Development and Therapy Dove Press