CER Daily Newsfeed®
Articles
Towards Patient Centricity: GSK (9/20, Amanda Barrell, PharmaPhorum) reports “...Since the pioneering GSK-backed Salford Lung Study, which collected RWE from more than 2,800 people with COPD and 2,233-plus with asthma, was published in 2017, things have started to change in this area. ‘There are signs that regulators are becoming increasingly open to understanding how RWE can better inform a broader range of regulatory decisions,’ explains [GSK's Dr. Karen Mullen].” Full
How Data Analysis Can Outpace the Lab to Answer Clinically Pressing Questions (9/20, Ulf Staginnus, Ipsen) comments “...The almost parallel development timeline of new treatments in aHCC resulted in a number of second line (2L) trials being initiated at approximately the same time and with placebo-controlled design. Ipsen recognized the lack of clarity for healthcare professionals in sequencing the new treatments that this resulted in, as they were short of insight into the comparative effectiveness of these new treatment approaches...So, Ipsen turned to a matching-adjusted indirect comparison or MAIC. As a commonly accepted statistical method to compare treatments when head-to-head studies are not available, this approach can unlock the insight in existing randomized controlled trials.” Full
PCORI Grant Allows Community Health Worker Program to Scale Up (9/20, David Raths, Healthcare Innovation) reports “As social determinants of health take on more importance for health systems, community health workers are playing a key role. During the annual meeting of the Patient-Centered Outcomes Research Institute on Sept. 19, leaders of Penn Medicine's IMPaCT (Individualized Management for Patient-Centered Targets) program discussed their efforts to scale their program nationally. IMPact hires and trains trusted neighborhood residents to become community health workers.” Full
Press Releases
National Quality Forum Releases Practical Resource to Advancing Patient-Centered Renal Care (9/20, National Quality Forum Press Release) “The National Quality Forum recently published its latest NQP Playbook™: Supporting Shared Decision Making for Individuals with Chronic Kidney Disease and End-Stage Renal Disease – a practical resource with guidance and tools that healthcare systems, providers, and patient advocacy organizations can utilize to improve the quality of care through shared decision making in chronic kidney disease and end-stage renal disease.” Full
NICE Calls for Further Research on Management Options for Tinnitus (9/20, NICE Press Release) “...Tinnitus is sometimes associated with problems with concentrating, listening or sleeping and for some people it can have a significant impact on their mental wellbeing. To address these symptoms, the draft guideline advises healthcare professionals to develop a management plan with the patient to explore different approaches that may help.” Full
Journals
Flawed Evidence Underpins Approval of New Cancer Drugs Barbara Mintzes, Agnes Vitry September 18, 2019, BMJ BMJ
Articles
Accelerating The Shift Toward Person-Generated Real-World Evidence (9/19, Tanisha Carino and Mark B. McClellan, Health Affairs Blog) comments “...FasterCures and the Duke-Robert J. Margolis Center for Health Policy convened a workshop in the Fall of 2018 with key representatives of the stakeholder community to better understand 1) the types of real-world data that patients themselves are generating and how it is being collected, organized, and evaluated by patient organizations and others; 2) use cases for applying person-generated data to serve the objectives of a broad range of stakeholders in the R&D ecosystem; and 3) action items that could support a business case for investing in person-generated data assets and their integration into the broader data ecosystem.” Full
Real-World Evidence In Clinical Research: We're Not In Kansas Anymore (9/19, Alethea Wieland, Clinical Leader) comments “...Part one of this series is intended to give readers a glimpse into what RWE is today, with its potential to utilize staggering volumes of data, digital health technologies,1 and mobile applications. Part two will further describe the challenges and opportunities for life sciences companies, highlight recent examples of RWE, and discuss how this newer way of thinking will transform the way we design and conduct clinical research or evaluate value-based decisions for the better.” Full
IQVIA Study: Real World Data Can Augment Clinical Trial Findings for New Drugs (9/19, Audrey McCamy-Miller, WRAL TechWire) reports “...The study shows the benefit of real world data to support decision-making by regulatory agencies and healthcare payers, IQVIA said in a press release. ‘These findings demonstrate that real-world end points are generally consistent with each other and with outcomes observed in randomized clinical trials, which substantiates the potential validity of real-world data to support regulatory and payer decision making,’ the conclusion of the study notes.” Full
Pelosi Says Key Component of Drug Pricing Bill Not Open to Negotiations (9/19, Andrew Siddons, Roll Call) reports “...According to a summary document released Thursday, HHS would be ordered to negotiate prices for ‘as many drugs as possible each year,’ but would have to negotiate for a minimum of 25...The negotiated price would not be able to exceed the ceiling set by the international reference price. Once a drug has a generic competitor, its price would no longer be limited by the negotiation program. HHS would be directed to prioritize drugs based on the greatest potential savings to taxpayers, and consider their research costs and whether the drug provides value relative to alternate treatment options.” Full
Understanding The House Democrats' Drug Pricing Package (9/19, Rachel Sachs, Health Affairs) comments “...[T]he released package of course recognizes the importance of not just innovation, but innovation that provides value for patients. First, R&D costs and the value of a drug are key elements of the negotiation provisions of the package. As such, Democrats can argue that their proposal ensures that industry can recoup its R&D costs while also negotiating for fair prices that provide greater returns for significant innovation. The question for Democrats is not just whether innovation is occurring -- it is also about the nature of that innovation and its value to patients.” Full
Press Releases
Minorities More Likely to Have Diabetes at Lower Weights (9/19, Kaiser Permanente Press Release) “Being overweight or obese is commonly associated with diabetes, but a new Kaiser Permanente study finds the connection differs widely by race or ethnicity. Members of racial and ethnic minority groups were much more likely to have diabetes or prediabetes at lower weights — even at normal or below-normal body mass index, according to research published in Diabetes Care. The large analysis included more than 4.9 million people of diverse backgrounds and geographies who were part of the nationwide Patient Outcomes Research to Advance Learning network.” Full
Journals
Health Technology Assessment of Drugs in Ireland: An Analysis of Timelines Connolly E, et al. September 17, 2019, PharmacoEconomics PubMed
Less Is More in Critical Care Is Supported by Evidence-Based Medicine Catherine L. Auriemma, et al. September 18, 2019, Intensive Care Medicine Springer
Reports
White Paper: Improving Transparency in Non-Interventional Research for Hypothesis Testing—WHY, WHAT, and HOW: Considerations from The Real-World Evidence Transparency Initiative September 18, 2019 ISPOR
Events
ISPOR Summit 2019 -Building Trust in Real-World Evidence: The Role of Study Registration October 11, 2019 8:30AM - 3:45PM ET Baltimore Marriott Inner Harbor at Camden Yards 110 South Eutaw Street Baltimore, MD 21201 Duke Margolis Center for Health Policy
Articles
Value-Based Contracts: Medicaid Best Price Concerns Could Be Eased With CMS Guidance (9/17, Cathy Kelly, Pink Sheet) reports “...The issue of best price is one of the most frequently cited regulatory obstacles to value-based arrangements such as outcomes-based contracts. The concern is that if a drug's price is heavily discounted under such a contract, it could trigger a new Medicaid best price that must be offered to every state. CMS could issue guidance stating that value-based contracts should be included in the definition of ‘bundled sales’ for the purpose of calculating the average manufacturer price for covered outpatient drugs in the Medicaid rebate program, the paper suggests.” Paid Subscription Required
No Simple Formula for Determining the Value of Cures (9/18, Mari Serebrow, BioWorld) comments “...Outcomes-based contracting has been touted as a go-to solution for [single or short-term transformative therapies]. But the absence of long-term clinical data can make such contracts challenging. Who defines the value of such therapies? And what's the cost if a drug fails? Should the drug company just refund the cost of the drug? Should it also have to cover the hospital care involved in delivering the therapy? Or should it cover the cost of care for the remainder of the patient's life?” Full
Video: Dr Scott Gottlieb: Paying Over Time for Expensive Therapies (9/18, The American Journal of Managed Care) “Providing access to complex therapies through the Medicare market can benefit patients who are disproportionately ignored by socioeconomic status, said Scott Gottlieb, MD, former FDA commissioner (2017-2019).” View Video
UK: NICE Recommends Takhzyro (9/18, Anna Smith, PharmaTimes) reports “[NICE] has recommended the use of Takeda's Takhzyro (lanadelumab) on the NHS...The recommendation was made based partly on data from the HELP-03 Study, in which Takhzyro reduced the mean number of monthly HAE attacks by 87% compared with placebo when administered at 300 mg every two weeks and 73% compared with placebo when administered at 300 mg every four weeks.” Full
UK Politicians Want NICE To Use Conditional Reimbursement (9/18, Francesca Bruce, Pink Sheet) reports “...NICE, the health technology appraisal institute for England and Wales, should consider the greater use of conditional reimbursement and more flexibility when appraising medicines for severe disease and unmet needs, says a new report from the UK's All-Party Parliamentary Group on Access to Medicines and Medical Devices. The report, published on September 13, sets out what the group thinks should be included in NICE's methods review.” Paid Subscription Required
Press Releases
PCORI Welcomes Christine Goertz as Next Chairperson and Sharon Levine as Next Vice Chairperson of Board of Governors (9/17, PCORI Press Release) “...‘We are delighted with the GAO's appointments and I look forward to continuing to work closely with Dr. Goertz and Dr. Levine in their new leadership roles,’ said PCORI Executive Director Joe Selby, MD, MPH. ‘Their complementary expertise and long histories with PCORI will serve us and our Board very well in continuing to pursue our mission of helping people make better-informed healthcare decisions through patient-centered research.’” Full
IQVIA Collaborates with Cancer Researchers to Advance Use of Real World Evidence (9/18, IQVIA Press Release) “...‘While randomized clinical trials are the gold standard to evaluate whether a medical treatment can work, they don't determine if treatment works for diverse patients outside the clinical setting and within real world situations,’ said Nancy Dreyer, chief scientific officer, IQVIA Real World Solutions, and an author of the study report. ‘IQVIA is excited to be part of this initiative. This important study demonstrates the value of real world evidence to measure and quantify the comparative benefits and risks of various medical products.’” Full
Verantos Announces Publication of a Real-World Evidence Collaborative Study with Amgen (9/18, Verantos Press Release) “...‘This highlights the importance of routinely measuring accuracy, including sensitivity and specificity, in EHR-based studies intended for regulatory use,’ says Dan Riskin, M.D., FACS, founder and CEO of Verantos, and senior author on the study. ‘These are the types of findings that should generate important discussion in the healthcare community as we determine what regulatory-grade RWE means.’” Full
Journals
Current Value-Based Care Models Need Greater Emphasis on Specialty Care David Roer, MD, et al. September 18, 2019, The American Journal of Accountable Care AJMC
Reports
Clarifying Medicaid Best Price Regulations In the Context of Value-Based Payment Arrangements September 2019 Duke Margolis Center for Health Policy
NICE Methods Review: A Report of the All-Party Parliamentary Group on Access to Medicines and Medical Devices September 2019 The All-Party Parliamentary Group on Access to Medicines and Medical Devices
Events
Implementing New PCOR Evidence into Practice: AHRQ’s Experience and Lessons Learned September 19, 2019 9:00 - 10:30AM EDT PCORI
Patient Perspective: Learnings and Strategies for Partnership in Research September 19, 2019 9:00 - 10:30AM EDT PCORI
Beyond Opioids: Improving Chronic Pain Management Using Evidence-Based Nonpharmacological Treatment September 19, 2019 9:00 - 10:30AM EDT PCORI
A Patient-Centered Outcomes Research Approach to Prevention September 19, 2019 3:00 - 4:30PM EDT PCORI
Articles
Moving The Health Spending Conversation Forward: From The Bottom-Up To The Top-Down (9/16, Going Below the Surface) comments “The second Health Affairs and National Pharmaceutical Council event, ‘Health Spending: Moving from Theory to Action,’ convened more than 350 health policy experts in Washington, D.C., on Sept. 11 to explore novel solutions to rising U.S. health spending...The overarching questions raised by speakers centered around a question of health care philosophy: Should the country prioritize ‘bottom-up’ strategies that identify and reduce the use of low-value services at the provider or patient level, or should more emphasis fall to ‘top-down’ approaches that leverage a regulatory or budgetary approach to better allocate health care resources?” Full
Back to School with the NHC: Patient Community Training on Value Assessment (9/17, Aimee Lee Russell, National Health Council Blog) comments “...On Friday, September 6, the National Health Council convened a training for patients and patient organizations to learn about value assessment with an emphasis on inclusion of the patient voice. To start the day, participants learned about the origins of value frameworks in the United States. Next, Dr. Joey Mattingly provided a primer on important terms for understanding value assessment.” Full
Another Day, Another Gottlieb Board Seat: this Time It's Real-World Evidence Startup Aetion (9/17, Eric Sagonowsky, Fierce Pharma) reports “...At Aetion, he'll advise the startup as it works to promote its evidence platform, a tool to assess safety, efficacy and value of medical treatments. In a statement, Gottlieb said he's ‘inspired by the opportunity to join the company's efforts to bring RWE to the forefront of drug research and development.’ Aetion CEO Carolyn Magill said he's an ‘ideal partner in our work to help define RWE's role in the development, delivery and payment of treatment.’” Full
The Future of Healthcare: Let's Add "Evidence-Generating Medicine" to "Evidence-Based Medicine." with Dr. Graham Hughes of Sutherland Healthcare (9/17, Christina D. Warner, MBA, Thrive Global) comments “...[Sutherland Healthcare Chief Executive Dr. Graham Hughes:] Let's add ‘evidence-generating medicine’ to ‘evidence-based medicine.’ The goal should be to let the data guide on what works most effectively in specific populations of patients. This so called, real-world evidence will allow us to better understand which therapies work most effectively, taking into account the specific social circumstances, individual preferences, past history and genetic makeup of individuals.” Full
Assessing the Value of New Medicines (9/17, Oliver Leatham, European Pharmaceutical Manufacturer) comments “...[S]ponsors and payers need to work closely together to identify what constitutes good practice and then determine how best to predict, measure and evaluate performance. Moving from large, late-phase clinical trials to resourceful, real-world reimbursement approaches will help to ensure that payer and health authority budgets are used wisely, reward innovation, and deliver the most positive impact possible on patients' quality of life.” Full
UK: Three Areas Where NICE's Collaboration with CMAC Can Make a Difference (9/17, Heidi Livingstone, NICE Blog) comments “...The NICE/CMAC partnership has identified three areas where we can make a difference: Increasing patient involvement in the assessment of new medicines. Reviewing how NICE judges the value of new medicines. Using evidence from NHS care, as well as clinical trials, in NICE's decision-making.” Full
UK: NICE, PHE Publish Antimicrobial Prescribing Guidance on Pneumonia (9/16, Anna Smith, PharmaTimes) reports “...The organisation says that the two guidelines set out an antimicrobial prescribing strategy that aims to optimise antibiotic use and reduce antibiotic resistance. The guidance also states that both types of pneumonia can be life-threatening. New recommendations have been added to highlight that antibiotic treatment should be started as soon as possible - no more than four hours after diagnosis and one hour if sepsis is suspected.” Full
Press Releases
GAO Names Chair and Vice Chair to PCORI Governing Board (9/17, U.S. Government Accountability Office Press Release) “...Christine Goertz, DC, PhD, was named to a three-year term as chair and Sharon Levine, MD, to a three-year term as vice chair...‘These two individuals bring strong leadership and an impressive breadth of research and clinical experience that should prove invaluable to the PCORI Governing Board as it brings patient-centered research to the forefront in health-care decision making,’ [GAO Comptroller General Gene L. Dodaro] said. ‘I am very pleased to name such capable individuals to these top positions.’” Full
HTA in the European Network: Osteoporosis Screening Without Proof of Benefit (9/17, IQWiG Press Release) “...For the first time, researchers from the Institute for Quality and Efficiency in Health Care were in charge of preparing a EUnetHTA report. According to the conclusion, the benefit of screening for osteoporosis in the general population has not yet been proven. The eight studies available do not prove that screening prevents fractures.” Full
Journals
Effect of Sacubitril-Valsartan vs Enalapril on Aortic Stiffness in Patients With Heart Failure and Reduced Ejection Fraction: A Randomized Clinical Trial Akshay S. Desai, MD, MPH, et al. September 17, 2019, JAMA JAMA
Articles
Announcing New Special Engagement Award Funding Opportunities, Evaluating Reporting Tool (9/16, Lia Hotchkiss, MPH, The PCORI Blog) comments “...These new special project funding announcements are: Engagement Award: Accelerating the Adoption of Tools and Resources. This provides support for projects that give organizations and community groups the opportunity to scale up or adopt meaningful engagement tools and processes with more communities, stakeholders, and patients with the intent to further build capacity/skills for PCOR/CER. Engagement Award: Community Convening Around Patient-Centered Outcomes Research. This provides funding to organizations and community groups to hold multistakeholder convenings that include patients, researchers, clinicians, purchasers, payers, health system leaders, and other stakeholders.” Full
Video: Dr Daniel Kantor on the Real-World Value of MS Disease Registries (9/16, The American Journal of Managed Care) “Since the introduction of disease-modifying therapies for multiple sclerosis (MS), evidence from disease registries has shown how the therapies work in the real world and reduce the effect of MS on people's lives, according to Daniel Kantor, MD, president of Kantor Neurology.” View Video
Real World Evidence in Health Care Improvement (9/13, Elizabeth Zimmermann, Mayo Clinic: Advancing the Science) comments “...Recently OptumLabs Chief Scientific Officer William Crown, Ph.D., connected with research collaborator David Kent, M.D., director of the Predictive Analytics and Comparative Effectiveness Center at Tufts Medical Center and the Clinical and Translational Science Graduate Program at Tufts University, to share perspectives on the use of real world evidence based on their extensive experience working with data from both clinical trials and real-world data from the OptumLabs Data Warehouse.” Full
Why Use Real-World Evidence for Clinical Trials? (9/15, Jason Shafrin, Healthcare Economist) comments “...As reported in a Bipartisan Policy Center's report: ‘Clinical trials can take as long as seven years and have cost $1.5 billion of the more than $2 billion spent on drug development.’ Thus, if real-world evidence could in part replace part of the clinical trial process, it could bring down drug development cost, reduce prices and increase incentives for life science R&D investment. An alternative approach is to use more real-world settings with pragmatic trials.” Full
Wasteful Drug Spending Contributes to High Prescription Costs (9/15, Alison Rodriguez, The American Journal of Managed Care) reports “...‘Large self-insured employers and other healthcare plan sponsors are concerned about rising prescription drug costs. Formularies developed on their behalf by intermediaries like pharmacy benefit managers and health plans can ensure drug safety and support negotiating with manufacturers,’ explained [Lauren Vela, MBA]. ‘But intermediaries can profit from these negotiations, creating financial incentives to include high-price drugs even if they offer little clinical value.’” Full
Scottish HTA OKs Kymriah For Lymphoma After Novartis Drops Price (9/16, Neena Brizmohun, Pink Sheet) reports “...The SMC said last week that it was able to accept Kymriah for [diffuse large B-cell non-Hodgkin lymphoma] after Novartis offered a confidential discount to the cost of the CAR T-cell therapy. The health technology assessment body added that its decision was also influenced by its ability to apply extra flexibility because Kymriah is a medicine for a very rare, end of life condition.” Paid Subscription Required
Journals
Sample Size Estimation for Randomised Controlled Trials with Repeated Assessment of Patient-Reported Outcomes: What Correlation Between Baseline and Follow-Up Outcomes Should We Assume? Walters SJ, et al. September 13, 2019, Trials PubMed
Prioritizing Evidence-Based Interventions for Dissemination and Implementation Investments: AHRQ's Model and Experience Huppert JS, et al. October 2019, Medical Care PubMed
Articles
Issues and Considerations of Promoting Cost-Effective Care (9/12, Matthew Gavidia, The American Journal of Managed Care) reports “During a panel on decisions at the point of purchase, Robert Dubois, MD, PhD, chief science officer and executive vice president of the National Pharmaceutical Council, moderated a discussion that answered questions regarding the use of cost-effectiveness by payers and the considerations surrounding the topic. Decisions at the point of purchase, also known as the bottom-up approach, revolve around the issue of low-value care, which promotes unnecessary or inefficient healthcare services.” Full
Employers Urged to Get off Sidelines to Trim Healthcare Costs (9/11, Rebecca Pifer, Healthcare DIVE) reports “Employers need to move off the sidelines and play a more aggressive role in helping curb ever-rising healthcare costs, according to a broad spectrum of stakeholders. ‘If 'leave us alone' is the industry mantra, there is no hope but political solutions. And political solutions will be like performing surgery with a sledgehammer,’ Reed Tuckson, former chief of medical affairs at UnitedHealth Group, said Wednesday at a Health Affairs event in Washington, D.C.” Full
Real World Evidence, Digital Partnerships and the Future of Pharma (9/13, George Underwood, PharmaPhorum) reports “...[Jennifer Wong, senior director, RWE strategy & alliances, global medical affairs, AstraZeneca:] RWE is a necessary complement to support traditional clinical trial designs, especially because so much of what we are looking to understand and assess about patients can only be generated and evaluated in the real-world setting. As the industry embraces RWE, it is great to have the strong leadership and vocal support from FDA in openly encouraging the industry to explore how best to utilize and rely on RWE going forward.” Full
The New Payer Dogma: US FDA Approved Drugs Have 'Less Evidence' (9/12, Michael McCaughan, Pink Sheet) reports “...Just because insurers tell each other that the FDA requires less evidence than it used to doesn't make it true. But the message doesn't have to be true to be a critical threat to the FDA and the drug industry. If everyone starts to believe that hugely expensive new therapies are being approved without clear evidence of efficacy, the current drug pricing debate is only going to turn darker and more damaging for anyone who cares about innovation.” Paid Subscription Required
Review Highlights Need for More Holistic Measures Around Low-Value Care (9/13, Lauren Adams, M.A., AcademyHealth Blog) comments “A review commissioned by AcademyHealth and published in the Journal of General Internal Medicine reveals those trying to reduce low-value care should expand their considerations to include clinically-relevant measures.” Full
NAACOS: ACO Participation Dip Under New Rules Should Worry CMS (9/12, Chelsea Cirruzzo, InsideHealthPolicy.com) reports “...In a recent Health Affairs blog published on Wednesday, NAACOS' Clifton Gaus, president and CEO, Allison Brennan, senior vice president of government affairs, and David Pittman, health policy and communications advisor, call on CMS to combat the drop by encouraging participation in voluntary, total-cost-of-care payment models, including ACOs...According to Gaus, Brennan and Pittman, it takes three years for an ACO to generate savings. They express concern that forcing providers to take on risk before three years will cause ACOs to drop out of the program.” Paid Subscription Required
Journals
Measures Used to Assess the Impact of Interventions to Reduce Low-Value Care: A Systematic Review Jennifer K. Maratt, et al. September 2019, Journal of General Internal Medicine Springer
Guidance for a Causal Comparative Effectiveness Analysis Using ‘Big Real World' Evidence: When to Start Statin Treatment Felicitas Kuehne, et al. September 12, 2019, Journal of Comparative Effectiveness Research Journal of Comparative Effectiveness Research
Comparative Effectiveness of Operative Versus Nonoperative Treatment for Rotator Cuff Tears: A Propensity Score Analysis From the ROW Cohort Show all authors Nitin B. Jain, MD, MSPH, et al. September 13, 2019, The American Journal of Sports Medicine Sage
Participation in an Innovative Patient Support Program Reduces Prescription Abandonment for Adalimumab-Treated Patients in a Commercial Population Brixner D, et al. September 13, 2019, Patient Preference and Adherence Dove Press