CER Daily Newsfeed®
Articles
Up-Front Transparency and Pass-Through Model Key to Unlocking Confusion Behind Pharmacy Benefit Managers (7/17, Brent Eberle, SSM Health Blog) comments “...Traditional PBMs make money through spread pricing – i.e., when pharmacies are paid one price and plan sponsors are charged a higher price...[T]his can lead traditional PBMs to promote drugs that have higher rebates, creating an incentive for manufacturers to price products higher and deeply rebate the products back to the PBMs. As a result, plans sponsors and their members pay more. This might be why more than two-thirds of employers in a 2017 survey conducted by the National Pharmaceutical Council said they'd welcome an alternative to rebate-driven approaches to managing pharmacy costs.” Full
Precision Medicines Need Precision Patient Assistance Programs (7/17, A. Mark Fendrick, MD and Jason D. Buxbaum, MHSA, The American Journal of Managed Care) reports “...There is a robust evidence base showing that individuals who are subject to high levels of cost sharing use less of both high- and low-value care in similar proportions. The higher the cost sharing, the greater the corresponding reduction in service use. Not surprisingly, cost-related underuse of evidence-based services disproportionally impacts poorer Americans and those with chronic conditions.” Full
US FDA-Funded Heart Failure Study To Focus On Patient-Centric Outcomes (7/16, Brenda Sandburg, Pink Sheet) reports “...Heart failure drugs do not require favorable effect on survival or hospitalization for approval, US FDA says; correlation between physical function and physiology biomarkers and clinical endpoints to be measured in upcoming clinical trial examining feasibility of capturing patient-centric data.” Paid Subscription Required
Telemedicine Takes Focus in Study of HCV Treatment in Methadone Centers (7/17, Healio) reports “With $7 million from the Patient-Centered Outcomes Research Institute, seven opioid treatment programs announced entry into an ongoing study of telemedicine for treatment of hepatitis C...The ongoing study follows promising results from a pilot study, published in Clinical Infectious Diseases, in which Talal and colleagues assessed the efficacy of HCV management via telemedicine in an opioid treatment program in 62 patients.” Full
UK: Novartis' Kisqali, Trailing Lilly's Verzenio, Nabs Limited NICE Backing in Breast Cancer (7/17, Angus Liu, Fierce Pharma) reports “...In a typical change of heart at the National Institute for Health and Care Excellence, the drug cost watchdog backed Kisqali, in tandem with AstraZeneca's Faslodex, for previously treated HR+/HER2- breast cancer—after Novartis offered ‘an improved patient access scheme,’ the agency said Wednesday. Rather than a full nod, Kisqali's reimbursement on NHS is available only through the Cancer Drugs Fund, which requires a patient's application. That's the same limitation NICE gave Verzenio in its own recommendation this April.” Full
Press Releases
Health Insurance Idea Born at U-M Could Help Millions of Americans Spend less for the Care They Need Most (7/17, University of Michigan News Release) “...Today, the U.S. Department of the Treasury gave health insurers more flexibility to cover the cost of certain medications and tests for people with common chronic conditions who are enrolled in many high-deductible health plans. The rule change came about in part because of research and over a decade of policy engagement by U-M professor A. Mark Fendrick and colleagues at the U-M Center for Value-Based Insurance Design.” Full
Multi-Stakeholder Coalition Applauds the Release of Updated U.S. Treasury Guidance to Ensure Access to Critical Services and Drugs for the Chronically Ill (7/17, Smarter Health Care Coalition Press Release) “...The Coalition has long supported and called for updated guidance to expand the preventive service safe harbor and commends the Secretary for swiftly issuing the guidance following the Administration's recent Executive Order to expand the safe harbor. The U.S. Treasury guidance aligns with our mission to advance value-based insurance design (V-BID), and we believe this provision will improve the utilization of high-value health care services and have a significant impact on the chronic disease epidemic facing our nation.” Full
At-Home Support Helps Stroke Patients Adjust After Hospital Stay (7/17, Michigan State University News Release) “Michigan State University researchers have found that many stroke patients feel unprepared when discharged from the hospital. Their caregivers feel the same. But when a home-based support network using social work case managers and online resources is put into place, quality of life and confidence in managing one's health improve, according to a new study...The study was funded by the Patient-Centered Outcomes Research Institute...” Full
Journals
Comparative Effectiveness of Treatments for High-Risk Prostate Cancer Patients Jayadevappa R, et al. July 5, 2019, Urologic Oncology PubMed
Articles
Op-Ed: Ask Whether a Drug Works Before Worrying about What it Costs (7/16, Steven Woloshin, Los Angeles Times) comments “...A more ambitious solution would be for the HHS to have the FDA commission an app to compare data across drugs (and ideally, non-drug options) used for the same purpose. Unfortunately, such comparative effectiveness data are rarely available, since the FDA does not require new drugs to be tested against those already on the market. If the goal is to promote wiser use of prescription drugs, this needs to change.” Full
Gilead and Novartis Seek to Expand What FDA Considers as Real-World Data and Real-World Evidence (7/15, Zachary Brennan, Regulatory Focus) reports “...‘Study designs using observational data are complex and should be explained in detail, particularly matching and follow-up procedures,’ Gilead says, and also calls on FDA to include examples of observational studies, such as cohort studies, case control studies and cross-sectional studies...‘We believe the guidance should expand the scope of RWE for regulatory purposes,’ [Novartis] says. ‘Patient reported outcomes and quality of life are often collected and become an important part of the RWE for regulatory and reimbursement purposes.’” Full
Precision Medicine for Cancer Care Costs a Fortune but Could Save Money in the Long Run (7/16, Michael Caligiuri, Newsweek) comments “...What makes next-generation genetic testing so appealing is that it brings us closer to the holy grail of medicine: getting better care without spending more money. There is no worse outcome—for the patient, for the physician, for the health care system—than the delivery of a drug that provides nothing but side effects and expenses.” Full
NICE Overcomes UK Reimbursement Doubts Over Forxiga, US FDA Says No To Approval (7/15, Neena Brizmohun, Pink Sheet) reports “...NICE has recommended that AstraZeneca's Forxiga (dapagliflozin) be used on the National Health Service in England and Wales as an adjunct to insulin in patients with type 1 diabetes...The recommendation follows initial doubts NICE has had over the product. When the HTA body's independent appraisal committee met to make its earlier provisional decision in April, ‘it was minded not to recommend dapagliflozin and asked for further clarification and analyses from the company,’ NICE explained.” Paid Subscription Required
Journals
Patient Registries: An Underused Resource for Medicines Evaluation : Operational Proposals for Increasing the Use of Patient Registries in Regulatory Assessments McGettigan P, et al. July 13, 2019, Drug Safety PubMed
Articles
Video: Dr Devin Incerti: Use Value Assessments to Maximize the Well-Being of a Population (7/14, The American Journal of Managed Care) “If you set social benefits equal to social costs, you will be able to maximize the well-being of a population, and that's what value assessment allows for, explained Devin Incerti, PhD, lead economist, Innovation and Value Initiative.” View Video
V-BID X: Creating A Value-Based Insurance Design Plan For The Exchange Market (7/15, Haley Richardson, Michael Budros, Michael E. Chernew, A. Mark Fendrick, Health Affairs Blog) comments “...V-BID X designs explicitly recognize that it is possible to increase accessibility to services deemed to improve patient-centered outcomes without increasing total health care costs. By abandoning blunt cost-sharing strategies and using a more clinically nuanced approach, V-BID X plans incentivize consumers to use more of the services that improve their health and less of those that don't. In recognizing that not all health care services produce that same amount of benefit to patients, V-BID X designs are more likely to better meet the financial and medical needs of their beneficiaries.” Full
Amgen on the Role of RWE in a Product Evidence Generation Plan (7/15, Hanson Wade, PharmaPhorum) reports “...Cathy Critchlow, Vice President, Center for Observational Research at Amgen, will be joining the 7th annual IMPACCT: Real World Evidence forum this September, in Boston, to discuss the actual role of RWE in defining and establishing a comprehensive product evidence generation plan, and how this is being done at Amgen. Cathy will address what are the key questions and challenges across the product life cycle that can be addressed through RWE, as well as share their experience in creating the necessary synergies across the organization to leverage RWE in its full strength.” Full
Role of Real-World Evidence in Clinical Trials Needs Definition, Industry and FDA Agree (7/15, Brandon May and James Miessler, CenterWatch) reports “...Novartis and Gilead believe the agency's draft guidance on identifying drug submissions that use RWE is too limited in scopeNovartis stressed that the guidance needs to clearly distinguish between RWD and RWE and asked the agency to clarify how RWE can be applied in single-arm trials that lack external/historical arms. Gilead also recommended that the guidance be expanded. The company said the agency should include sNDAs and sBLAs in its list of submissions and allow lab data linked to medical claims or electronic medical records as a source of RWD.” Full
Video: Leveraging Randomized Clinical Trials to Generate RWE for Regulatory Purposes - Day 1 (7/15, Duke Margolis) View Video
Podcast: A Small Nonprofit Becomes Powerful Drug Price Watchdog (7/15, Carey Goldberg, WBUR) reports “The independent Institute for Clinical and Economic Review uses a cost-benefit analysis to figure out fair drug prices. It's been called ‘Big Pharma's biggest threat.’ Carey Goldberg (@goldbergcarey) of WBUR reports.” Full
AbbVie's Rising Immunology Star Skyrizi Scores Nod from England's Cost Watchdog (7/15, Kyle Blankenship, Fierce Pharma) reports “...The National Institute for Health and Care Excellence recommended Skyrizi as a second-line treatment for severe plaque psoriasis in adults who've failed on conventional treatments such as ciclosporin, methoxotrate and phototherapy. In its final appraisal, NICE said second-line Skyrizi was more effective for plaque psoriasis than Humira and Johnson & Johnson's Il-12/23 med Stelara and similar in health benefits to J&J's Tremfya, a head-to-head IL-23 competitor.” Full
Journals
Evidence-Based Medicine: A Data-Driven Approach to Lean Healthcare Operations John T. Langell July 12, 2019, International Journal of Healthcare Management International Journal of Healthcare Management
Redefining the Physician's Role in Cost-Conscious Care: The Potential Role of the Electronic Health Record Brian J. Miller, MD, MBA, MPH; Jennifer M. Slota; Jesse M. Ehrenfeld, MD, MPH July 12, 2019, JAMA JAMA
Real-World Evidence of Glycemic Control Among Patients with Type 2 Diabetes Mellitus in India: The TIGHT Study Surendra S Borgharkar, Soma S Das July 14, 2019, BMJ Open Diabetes Research & Care BMJ Open Diabetes Research & Care
Reports
Evaluation of Medical Technologies with Uncertain Benefits July 2019 National Bureau of Economic Research
Events
Webinar: Real World Evidence and Data: A Tufts Study of 30 Pharma Companies August 15, 2019 1:30 - 3:00PM EDT FDANews
IMPACCT: Real World Evidence September 18 - 19, 2019 Boston, MA IMPACCT RWE
FT Pharma Pricing and Value Summit 2019 September 26, 2019 9:00AM ET Stewart Hotel New York 371 7th Avenue New York, NY Patch
Articles
Friends of Cancer Research Launches Next Phase in Real-World Evidence Initiative (7/11, The ASCO Post) comments “...The initial Friends' RWE pilot project demonstrated that different datasets could be used to extract several real-world endpoints in a relatively consistent manner. In order to further characterize the role real-world endpoints may play in measuring treatment effect size, pilot project 2.0 will examine the ability of different real-world endpoints to detect treatment effectiveness in real-world patient populations. Additionally, data from this pilot project will help inform a framework containing key data elements, endpoint definitions, and algorithms that could be applied to other datasets representing different therapeutic areas and disease types.” Full
ICER Update on Duchenne Drugs Pushes up Price Estimates for Exondys 51, Emflaza Ahead of July Panel Review (7/12, Natalie Grover, Endpoints News) reports “...ICER evaluated the efficacy, safety and cost-effectiveness of four treatments in its evidence report. It looked at the steroids deflazacort (sold as Emflaza) and prednisone — as well as the exon-skipping drugs: the approved eteplirsen (marketed as Exondys 51) and the experimental golodirsen (both come from Sarepta, and each treatment is designed to treat a different subset of DMD patients). As it concluded in its draft report in May, ICER reiterated that the evidence supporting each of the four treatments is lacking, and their impact on patients unclear.” Full
True Innovation or Just Later to Market? German Agency Questions Benefits of New Drugs (7/12, Angus Liu, Fierce Pharma) reports “...Even of the 89 drugs that showed at least some additional benefit, 37 delivered advances in only part of the approved patient population, IQWiG said...What caused the huge gap? IQWiG blamed the current drug development and approval process and the illusion that newer is better. ‘Presumably, this is because regulators still allow placebo-controlled studies even though health technology assessment bodies have long recommended active-controlled trials, which provide more useful information,’ it said.” Full
Video: Dr Jason Mitchell Details the Importance of Data in Value-Based Care (7/11, The American Journal of Managed Care) “Data is critical to all value-based care, said Jason Mitchell, MD, chief medical and clinical transformation officer, Presbyterian Healthcare Services.” View Video
UK: NICE Recommends Risankizumab to Treat Severe Psoriasis (7/12, Victoria Rees, European Pharmaceutical Review) reports “...The National Institute for Health and Care Excellence has issued a final draft guidance to recommend risankizumab (ABBV-066) for treating plaque psoriasis. Risankizumab, produced by AbbVie, is an anti-IL-23 antibody. The drug can be used to treat severe plaque psoriasis when there has been no response from other systemic treatments, including ciclosporin, methotrexate and phototherapy.” Full
Press Releases
ICER Publishes Evidence Report on Treatments for Duchenne Muscular Dystrophy (7/11, ICER Press Release) “The Institute for Clinical and Economic Review today released an Evidence Report assessing the comparative clinical effectiveness and value of two exon-skipping therapies to treat Duchenne muscular dystrophy — eteplirsen (Exondys 51™, Sarepta Therapeutics) and golodirsen (Sarepta Therapeutics) — as well as deflazacort (Emflaza®, PTC Therapeutics), a corticosteroid.” Full
NICE Recommends Innovative Treatment for Type 1 Diabetes (7/12, NICE Press Release) “...NICE has today issued draft final guidance on dapagliflozin with insulin for treating type 1 diabetes. Around eight per cent of people with diabetes in the UK have type 1 diabetes. It's a serious lifelong condition which is caused when the level of glucose (sugar) in the person's blood is too high because their body cannot produce enough insulin. It is not caused by someone's diet or lifestyle and its origin is unknown. Daily injections of insulin are needed to keep a person's blood glucose levels under control.” Full
Journals
Rethinking Bias and Truth in EBM Sietse Wieringa, et al. July 12, 2019, BMJ Evidence-Based Medicine BMJ Evidence-Based Medicine
Requests for Comment
Call for Patient Input and Clinical Experts: Stelara July 12 - September 3, 2019 CADTH
Reports
DMD: Evidence Report July 11, 2019 ICER
Articles
Can Real-World Evidence Be Used To Remove LPAD's Limited Population Designation? (7/10, Michael Cipriano, Pink Sheet) reports “...Can real-world evidence be sufficient to support a supplemental application for lifting the restrictions of the 21st Century Cures Act's limited population antibacterial drug pathway? Or would a clinical trial be required? These are among the many questions industry has raised about LPAD as the US Food and Drug Administration readies for a 12 July public meeting on the subject.” Paid Subscription Required
Trump Calls For Artificial Kidney Development In Executive Order (7/11, David Roza, InsideHealthPolicy) reports “...HHS says the information FDA collects also will be used by device makers, patients, providers, payers and other researchers to inform the development of new treatments. ‘The patient preferences survey will be an important example of how patient engagement can contribute to building infrastructure for expanded patient-centered outcomes research and how patient input can be used in FDA's review processes,’ HHS says.” Paid Subscription Required
UF Health Joins Research Network Developing a Data Repository on Infectious Diseases (7/11, Florida Trend) reports “With funding from a five-year, $6.7 million National Institutes of Health grant, researchers at UF Health have joined the University of Pittsburgh Graduate School of Public Health and other academic centers to form a collaborative research network focused on improving global knowledge of infectious diseases.” Full
The National Health Council Announces the Release of a Rubric to Capture the Patient Voice (7/11, Silke Schoch, National Health Council Blog) comments “...The purpose of the NHC Rubric to Capture the Patient Voice (Rubric) is to provide a tool the patient community or any other health care stakeholder can use to evaluate attributes of patient centeredness and to guide them on meaningful patient engagement throughout any activity they might undertake. It is an all-purpose tool to assist users across the health ecosystem. Potential uses might include, but are not limited to, patient engagement in health care policy, quality-measure development, shared decision-making, value assessment, product development, etc.” Full
UK: NICE Recommends gammaCore for Cluster Headaches (7/11, Anna Smith, PharmaTimes) reports “In a new draft guidance, The National Institute for Health and Care Excellence has recommended that using gammaCore in addition to standard care (sumatriptan, zolmitriptan and oxygen) could save the NHS £450 per patient in the first year compared to standard care alone.” Full
Press Releases
No Evidence of Added Benefit for Most New Drugs, Say Researchers (7/10, BMJ Press Release) “...Between 2011 and 2017, IQWiG assessed 216 drugs entering the German market following regulatory approval, they explain. Almost all of these drugs were approved by the European Medicines Agency for use throughout Europe. Yet only 54 (25%) were judged to have a considerable or major added benefit. In 35 (16%), the added benefit was either minor or could not be quantified. And for 125 drugs (58%), the available evidence did not prove an added benefit over standard care in the approved patient population.” Full
Aimmune Statement on Institute for Clinical and Economic Review (ICER) Final Report on AR101 for Peanut Allergy (7/11, Aimmune Therapeutics Press Release) “Aimmune Therapeutics...cautions stakeholders against drawing conclusions from the Institute for Clinical and Economic Review's final report on emerging desensitization treatments for peanut allergy, released yesterday. In its review, ICER fails to incorporate available data on both long-term outcomes and quality-of-life. ICER's analysis and the public meeting discussion discounted patient and community perspectives on the physical, social and psychological benefits of desensitization.” Full
ACO Model Delivers Improved Health Care to Rural Areas and $48M Savings to Medicare (7/11, Abt Associates Press Release) “...Financing and inadequate capital for information technology have been cited by rural providers as significant infrastructure obstacles to ACO participation. Under [the CMS ACO Investment Model], up-front and ongoing payments to ACOs enabled them to overcome those initial operational barriers. CMS recoups the payments from the ACOs' subsequent savings. ACOs also receive bonuses from CMS if their savings exceed the payments they've received from CMS and their quality of care is robust.” Full
Journals
New Drugs: Where Did We Go Wrong and What Can We Do Better? Beate Wieseler, et al. July 10, 2019, BMJ BMJ
Press Releases
ICER Lists Potential Topics for Assessments in 2020 (7/9, ICER Press Release) “...The list is drawn from ICER horizon scans of new and emerging therapies, conversations with stakeholders, and suggestions submitted by the public. ICER's topic selection criteria – including the projected timing and likelihood of FDA approval, and whether a review of the evidence would suggest specific actions for payers, physicians, patients, and policymakers to improve clinical practice – are applied to produce this preliminary list of potential topics.” Full
ICER's Final Report on Peanut Allergy Treatments: Clinical Evidence Does Not Yet Demonstrate That Long-Term Benefits of Desensitization Outweigh Short-Term Risks (7/10, ICER Press Release) “...The report evaluates Viaskin Peanut (DBV Technologies) and AR101 (Aimmune Therapeutics), as well as non-commercialized oral immunotherapy...ICER's evaluation of long-term cost-effectiveness found that the fair value-based price benchmark range would be $4,800-7,200 per year for AR101 and $3,000-$4,500 per year for Viaskin Peanut.” Full
Journals
Cost-Effectiveness Analysis of Abiraterone Acetate vs Docetaxel in the Management of Metastatic Castration-Sensitive Prostate Cancer: Hong Kong's Perspective Chiang CL, et al. July 4, 2019, Prostate Cancer & Prostatic Diseases Prostate Cancer & Prostatic Diseases
Engaging Parent–Child Dyad and Healthcare Provider Stakeholders in a Patient-Centered Comparative Effectiveness Study Briana A Woods-Jaeger, et al. July 10, 2019, Journal of Comparative Effectiveness Research Journal of Comparative Effectiveness Research
Factors Associated with Evidence-Based Decision-Making Among Patients and Providers Nyna Williams & Rachel Kogan July 10, 2019, Journal of Comparative Effectiveness Research Journal of Comparative Effectiveness Research
Reports
Peanut Allergy: Final Evidence Report and Meeting Summary July 10, 2019 ICER
Events
Webinar Video: What’s in a Number? It Matters in Health Policy Conversations AcademyHealth