CER Daily Newsfeed®
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and FDA Agree (7/15, Brandon May and James Miessler, CenterWatch) reports “...Novartis and Gilead believe the agency's draft guidance on identifying drug submissions that use RWE is too limited in scopeNovartis stressed that the guidance needs to clearly distinguish between RWD and RWE and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
Articles
A Market-Oriented Framework For Reforming Medicare Part B Drug Payment (7/9, Joseph R. Antos and James C. Capretta, Health Affairs Blog) comments “...Clinical assessments of therapies can be controversial. They necessarily involve judgments and assumptions, as well as hard data, and it is difficult to assemble a process that all parties view as neutral and fair. Nonetheless, better and more broadly disseminated clinical assessments can add value to the pricing environment in the US, especially in situations that involve a clinically important therapy where no effective alternative is available to patients and purchasers. In those circumstances, the only way to assess value is by looking at the actual clinical benefits for patients.” Full
FDA Leverages Private Sector Technology-Based Tools In Its Embrace Of Real World Evidence (7/9, John Osborn, Forbes.com) comments “...The FDA's new working arrangements offer the promise of meaningful access. Flatiron and COTA have partnered with the FDA's Information Exchange and Data Transformation Program to conceptualize and launch a program to better understand which cancer treatments work well, and which regimens are less effective. Related to this, the agency's Oncology Center of Excellence is working with several cancer research groups in an effort to identify which patients and tumor types are more likely to respond to immunotherapy.” Full
Cigna Ventures Leads $23M Funding Round in Precision Medicine Company GNS Healthcare (7/9, Heather Landi, Fierce Healthcare) reports “Cigna Ventures joins other big names in health insurance and biotech backing GNS Healthcare, a technology company that uses machine learning to provide precision medicine...GNS uses machine learning to effectively match the appropriate therapeutics, procedures and treatment intervention to the right patient at the right time. Cigna Ventures plans to tap GNS' technology and solutions to further advance whole-person health for Cigna's customers and clients, the company said.” Full
Despite Growth, Uptake of Downside Risk in ACO Contracts Remains Low (7/9, Jaime Rosenberg, The American Journal of Managed Care) reports “While the number of accountable care organization contracts with downside risk is growing, the majority of ACOs remain in upside-only risk contracts, according to a new study. In 2012, 28% of accountable care organizations had a contract with downside risk. That rate increased modestly to 33% in 2018. However, according to study researchers, the number of ACOs has increased approximately 5-fold during the time period, which could mean that the number of ACO downside risk contracts also grew significantly.” Full
UK Plans Upfront Payment Scheme To Spur Antimicrobial Development (7/9, Ian Schofield, Pink Sheet) reports “...The UK government is planning to test a new scheme aimed at fostering the development of new antimicrobials whereby companies will be paid upfront for new drugs on the basis of their expected value to the national health service, rather than on the volume of products actually used on the NHS. The trial, involving what the government describes as "the world's first 'subscription' style" payment scheme, is intended to help tackle the growing problem of antimicrobial resistance and will be developed and run by the health technology assessment body NICE, NHS England and NHS Improvement.” Paid Subscription Required
UK: Alnylam's Onpattro Back in the Game Against Ionis, Thanks to NICE U-Turn (7/9, Angus Liu, Fierce Pharma) reports “...[NICE] now recommends Onpattro to treat the ultrarare disease hereditary transthyretin-mediated amyloidosis in adults with damage to peripheral nerves. NICE's drug evaluation experts still think Onpattro's cost-effectiveness falls short of their usual threshold for a highly specialized drug. However, the committee reversed last year's ‘no’ vote after considering the benefits of halting—and potentially reversing—the condition, and the benefits that could offer patients for the rest of their lives.” Full
Journals
ACO Contracts With Downside Financial Risk Growing, But Still In The Minority Kristen A. Peck, et al. July 2019, Health Affairs Health Affairs
Comparative Effectiveness of Certolizumab Pegol, Abatacept and Biosimilar Infliximab in Patients with Rheumatoid Arthritis Treated in Routine Care. Observational Data from the Danish DANBIO Registry Emulating a Randomized Trial Kathrine L. Grøn, et al. July 3, 2019, Arthritis & Rheumatology Wiley
Articles
Editorial: Sound, Fury and Prescription Drugs (7/6, The New York Times) comments “...In the United States, any drug is allowed to enter the market as long as the [FDA] decides it is safe and effective. And once it enters the market, drug makers are allowed to charge whatever they want for it. That's not how it works in the rest of the world...For example, Britain will cover a new medication only if the benefits it provides are high relative to its price...The United States government could begin to put a similar system into effect if it directed [HHS] to evaluate and rank medications based on their cost-effectiveness. The nonprofit Institute for Clinical and Economic Review already does this independently and has had success in getting some drug companies to lower some of their prices.” Full
Value Our Health: Stop Discriminatory Value Assessments (7/8, Elizabeth Franklin, Morning Consult) comments “...Within many of these value assessments, the quality-adjusted life year is the measure used to calculate value...The measure has inherent ethical and operational concerns, particularly for people living with chronic conditions and disabilities. The lives of people in ‘less-than-perfect health’ are valued less than those ‘in perfect health.’ It is impossible to make broad brush value assessments for all people when we make judgments about diverse individuals that discriminate and penalize.” Full
Europe Urged to Adapt Faster to Gene Therapy Challenges (7/8, Andrew McConaghie, PMLiVE) reports “...Europe's leading nations should commit to a rapid overhaul of health technology assessment and bold initiatives such as new pan-European real world evidence frameworks, in order to nurture the nascent cell and gene therapy revolution in the region. That is the conclusion of a new report from The Alliance for Regenerative Medicine...The experts recommended faster uptake of new payment models like conditional reimbursement, pay-for-performance, and annuity-based payments...” Full
UK: AZ to Appeal NICE Rejection for Lung Cancer Drug (7/8, Anna Smith, PharmaTimes) reports “...The company's CEO, Pascal Soriot, said that they are ‘Very disappointed with this decision and will appeal. The UK has the second worst lung cancer survival outcomes in Europe and patients need new innovative treatments.’...He went further, saying that it's time for a ‘Comprehensive review of how NICE values innovative medicines. This is needed to ensure British patients can access the latest medical advances, to support the ambitions of the NHS Long Term Plan to improve outcomes for people with cancer, and to help deliver Government's ambition that the UK continues to be a globally leading center for life sciences.’” Full
Journals
A New Method to Determine the Optimal Willingness to Pay in Cost-Effectiveness Analysis Phelps CE July 2019, Value in Health PubMed
Health Technology Assessment (HTA) of Optoelectronic Biosensors for Oncology by Analytic Hierarchy Process (AHP) and Likert Scale Improta G, et al. July 5, 2019, BMC Medical Research Methodology PubMed
Reports
Getting Ready for Advanced Therapy Medicinal Products (ATMPs) in Europe July 2019 Alliance for Regenerative Medicine
Events
Webinar: VBID X: A New Plan Option for the Individual Health Insurance Market July 24, 2019 1:00 - 2:00PM EST V-BID Center
Webinar: Rethinking Drug Reimbursement: Value Communications in a Post-Rebate World July 31, 2019 1:00 - 2:00PM EDT W2O