CER Daily Newsfeed®

Articles

Six Ways to Make Real-World Evidence Methods More Transparent (5/20, National Pharmaceutical Council: E.V.I.dently Today) comments “...Both NPC and AcademyHealth recognized that by making RWE research methods more transparent, they could improve efforts to reproduce studies, disclose key study methods and reduce skepticism about the ultimate results. However, there is little agreement in the scientific community about how to achieve these goals. Through individual interviews and a panel discussion with a broad group of researchers, they developed six actionable recommendations to enhance research methods transparency and credibility.” Full

Is Our Health Care Spending Worth It? (5/20, Austin Frakt, The New York Times) comments “...Another Health Affairs study focused on seven prevalent chronic conditions. The conditions examined - breast and lung cancer, cerebrovascular disease, chronic obstructive pulmonary disease, diabetes, H.I.V./AIDS and ischemic heart disease - are collectively responsible for most of the mortality and serious disease in the population. According to the study, which compared changes in cost and length and quality of life from 1995 to 2015 for people with those conditions, the health system provides good value.” Full

Differentiating Health Care Costs from Health Care Value (5/20, Wayne Winegarden, Forbes.com) comments “...The coming gene therapies will revolutionize health care, but to fully benefit from these treatments our current approach to financing health care must change. The growing trend to use cost-effectiveness as a benchmark will limit access and discourage continued innovation. The best way to encourage the development of 21st-century medical treatments is to effectively apply traditional insurance principles to the health care market.” Full

ICER Seeks to Make Drug Price Research More Accessible (5/20, Ned Pagliarulo, Biopharma DIVE) reports “...ICER is developing two tools that it hopes will help payers and drugmakers more easily incorporate the group's work. The first will compile ICER's public evidence reports into a searchable and sortable database, with the aim of helping others develop templates to ‘guide pharmaceutical pricing strategies, payer benefit design, and the deliberations of Pharmacy & Therapeutics Committees,’ ICER said. ICER is also developing an ‘interactive modeler’ that will allow outside groups to tailor models around patient sub-populations or different cost structures, for example.” Full

Real-World Operations: NESTcc Director on Building a Business and the Future of Device Evaluation (5/20, Maria Rachal, Medtech DIVE) reports “...A system designed to better investigate medical devices' performance once they're on the market is finally getting off the ground. That [RWE] initiative has been formally in the works for about three years through the National Evaluation System for health Technology's coordinating center, better known as NESTcc...Convincing device makers to get on board with the research hub falls partly on the shoulders of health economist and researcher Rachael Fleurence, who joined NEST's coordinating center as executive director in 2017. She previously helped develop [PCORI’s] comparative effectiveness clinical research platform.” Full

Preserving Quality of Life in Face of Cognitive Impairment (5/17, Judy George, MedPage Today) reports “...‘Most research to date in this area has focused on the cognitive performance outcome itself or functional ability,’ [Melanie Chandler, PhD] told MedPage Today. But the first part of this Patient-Centered Outcomes Research Institute study asked a sample of patients with mild cognitive impairment and their partners to rank which outcomes were most important in an intervention trial. ‘The top four responses, from a possible 13 choices, were quality of life, mood, self-efficacy, and memory-related activities of daily living,’ she said.” Full

Press Releases

ICER Plans Cloud-Based Tools to Accelerate the Use of Value Assessments in the US Health System (5/20, ICER Press Release) “...‘Increasingly, ICER's independent and rigorous value assessments are informing the critical decisions pharmaceutical companies and US payers make around a prescription drug's price, reimbursement, and coverage,’ said Steven D. Pearson, MD, MSc, President of ICER. ‘Our Evidence Compendium and Interactive Modeler will significantly expand and expedite all stakeholders' ability to access key details across a wide collection of past ICER assessments, and to tailor modeling inputs & assumptions based on a specific patient population or existing cost structure.” Full

Corrona Expands Its Real-World Evidence Registry Collaboration for Patients with Multiple Sclerosis (5/20, Corrona Press Release) “...‘The comprehensive, high-quality observational data generated by the registry will bridge the evidence gap in MS research and provide the real-world context needed to make informed treatment decisions,’ said Dr. Bruce Bebo, Executive Vice President, Research of the National MS Society.” Full

Value in Health Goes Back to the Future to Celebrate Its Past (5/20, ISPOR Press Release) “Value in Health, the official journal of ISPOR—the professional society for health economics and outcomes research, announced today the publication of a special 20th anniversary issue that reflects on how ‘value’ has been defined, measured, and debated in the journal for the past two decades. To mark the occasion, the Editors commissioned articles that dealt with topics that have been widely discussed in the journal over the past 20 years, but for which there is an exciting future agenda in the field of HEOR.” Full

National Quality Forum Taps Healthcare Industry Experts to Drive Value Through the Next Generation of New Task Force (5/17, National Quality Forum Press Release) “...The Task Force has established a core committee led by co-chairs NQF President and CEO Shantanu Agrawal, MD, MPhil and Founding NQF President and CEO Kenneth W. Kizer, MD who will oversee five subcommittees designed to develop cross-cutting, actionable recommendations to serve as a blueprint to drive high-value care and outcomes through the next generation of quality. A final report with the Task Force's recommendations will be released later this year...” Full

Articles

Look Behind the Lecture: Payer Consideration of Patient Input (5/8, The Evidence Base) reports “...[James Chambers, Associate Professor, Tufts Medical Center:] I think that RWE offers important opportunities to collect PRO data and evaluate the impact of interventions on patients. While PRO data from randomized controlled trials is useful, PRO data from RWE is particularly valuable information that has the potential to improve clinical practice. A challenge is to ensure that we are collecting the right data: this is where input from patients is invaluable!” Full

House Passes Legislation Aiming to Shore Up Health Law and Lower Drug Costs (5/16, Abby Goodnough, The New York Times) reports “...The drug-pricing bills passed Thursday would take aim at the tactics that drug makers use to protect their monopolies and market share, which help keep drug prices high. One would seek to ban ‘pay-for-delay’ deals, in which brand-name manufacturers pay makers of generic pharmaceuticals to delay bringing a lower-priced drug to market. Another would loosen the rules on the exclusive six-month sales period that a generic drug maker gets when it is the first to market after a brand-name drug loses its patent protection.” Full

CMS Scales Back Plan to Allow Step Therapy for Part D Protected Classes (5/16, Paige Minemyer, Fierce Healthcare) reports “The Centers for Medicare & Medicaid Services finalized a scaled-back version of a proposed rule that will allow Medicare Part D plan sponsors greater leeway to use prior authorization and step therapy for drugs in protected classes. The final rule will allow Part D plans to use these utilization management tools for members starting new therapies and in all classes except antiretroviral drugs, codifying existing policy.” Full

UK: NICE Grants Celgene Long-Awaited Thumbs-Up for Revlimid in Untreated Myeloma (5/17, Arlene Weintraub, Fierce Pharma) reports “...On Friday—a full four years after the European Commission approved Revlimid for the first-line treatment of some adults with multiple myeloma—the National Institute for Health and Care Excellence recommended that the drug be covered by England's National Health Service in that setting. The agency sanctioned its use in combination with the steroid dexamethasone for patients who are not eligible for stem cell transplants and who cannot take the drug thalidomide.” Full

Journals

Using Health Technology Assessment for Better Healthcare Decisions Tucker S, et al. May 15, 2019, Healthcare Management Forum PubMed

Requests for Comment

Call for Patient Input and Clinical Experts: Vascepa May 17 - July 9, 2019 CADTH

Events

PCORI Board of Governors Meeting June 18, 2019 12:00 - 1:30PM EDT PCORI

Advisory Panel on Patient Engagement Summer 2019 Meeting June 27 - 28, 2019 PCORI

Articles

'How Could We Fail So Miserably?': Real-World Evidence Offers Sobering Reality (5/16, H. Jack West, MD, Medscape) comments “...How could we fail so miserably? One third of patients who qualify for well-established targeted therapies, and two thirds of those with less common but still evidence-based options highlighted by the [National Comprehensive Cancer Network], are not receiving the benefit of some of our most effective treatments for advanced [non–small cell lung cancer].” Full

EMA Undertook 14 RWE Studies In 2018 To Complement Company Data (5/16, Vibha Sharm, Pink Sheet) reports “...Pharmacoepidemiologists and statisticians at the European Medicines Agency used databases of electronic healthcare records to support decision-making by its scientific committees using real-world evidence...For all RWE studies, the EMA explained that it relied on two databases of electronic health records, namely the IQVIA Medical Research Data-UK (IMRD-UK, formerly the THIN database), and the IQVIA Medical Research Data-France and Germany (IMRD-France and Germany, formerly IMS-France and Germany).” Paid Subscription Required

UK's NICE Price Discounts ‘Pass Risk To Patients' (5/16, Francesca Bruce, Pink Sheet) reports “...Some of the issues that NICE faces when conducting appraisals could be solved through better evidence generation on what patients actually want and need, more input from academia, and a ‘more front loaded’ NICE system, said Eric Lowe, an independent healthcare consultant and former CEO of Myeloma UK...Lowe says that there is little room for more ‘sticking plaster policies’ such as the criteria that allow NICE to consider higher cost effectiveness thresholds for end-of-life drugs, and the Cancer Drugs Fund.” Paid Subscription Required

UK: Misguided Pricing Tactics Delay Access To Medicines, Warns NHS England (5/15, Francesca Bruce, Pink Sheet) reports “...Drugs should be priced according to their clinical value, not based on ideas about what companies think the NHS is already spending, [Blake Dark, commercial medicines director at NHS England,] said. Added to that, value propositions must be clear and transparent. ‘You would be surprised how complicated some of the propositions are that come across our desk. Complexity does not mean speed, let's be clear.’” Paid Subscription Required

Journals

Sertraline and Mirtazapine Versus Placebo in Subgroups of Depression in Dementia: Findings From the HTA-SADD Randomized Controlled Trial Zuidersma M, et al. April 6, 2019, The American Journal of Geriatric Psychiatry PubMed

Articles

With New Cancer Drugs, the Challenge Is Finding Where Cost, Value Meet (5/15, Alaric Dearment, MedCity News) reports “...In the last two decades, numerous new drugs have come to market and turned some cancers that used to be death sentences into manageable or even curable diseases. But one significant hurdle remains: They're often incredibly expensive. So the question that faces payers, physicians and ultimately patients is how to balance the high cost of new cancer therapies – particularly cutting-edge ones like cell therapies – with the value they add to the healthcare system. That will be the topic of a panel discussion at the upcoming MedCity CONVERGE conference, ‘Value and Cost of Novel Cancer Treatments,’ taking place June 19 in Philadelphia.” Full

Want Better Patient Outcomes? Track Physician Adherence to Evidence-Based Medicine (5/15, Christopher Cheney, HealthLeaders Media) reports “...LaGrangeville, New York–based Health Quest and Western Connecticut Health Network...have been using EHRs to set alerts, to manage order sets called ‘power plans,’ and to give physicians access to the most recent guidelines, best practices, and journal articles...‘We're finding that our increased compliance scores with evidence-based practice are correlating with improved patient outcomes and financial outcomes, which is encouraging. It gives validity to the initiative,’ says Kelly Philiba, ND, physician informaticist at Health Quest.” Full

Reducing Churn to Increase Value in Health Care: Solutions for Payers, Providers, and Policymakers (5/15, Niko Lehman-White and Saeed Aminzadeh, The Health Care Blog) comments “...The value-based contracts that have proliferated in this country over the past decade and which now account for about half of the money spent on healthcare allow these wellness investments to make good financial sense in addition to benefiting patient health. However, a phenomenon in health coverage in the US is increasing costs, destabilizing care continuity and holding back the potential of value-based care. It prevents us from making the long-term investments we desperately need.” Full

Finding Clinical Assertions in the World of Real-World Evidence (5/15, Maggie Lynch, Outsourcing Pharma) reports “...Dan Riskin, founder of Verantos, a digital contract research organization, tells Outsourcing-Pharma that RWE has its own place in the clinical and regulatory world, but it's use still has room for improvement. To Riskin, there is more than one type of RWE and in its variances, there are additional aspects of applications. He said that the most commonly seen type of RWE is one that used to find patterns. However, the use of RWE has greater bandwidth, according to Riskin, and can be used to take the place of control arms in clinical trials, bolster regulatory filings, and give a deeper look into the lives of patients.” Full

Scotland: SMC Follows NICE And Backs Lilly's Verzenios In Advanced Breast Cancer (5/15, Ian Schofield, Pink Sheet) reports “...The SMC has given the all clear to funding for Lilly's Verzenios for breast cancer, Richter's Reagila for schizophrenia, and MSD's Keytruda for melanoma. However, it rejected Alliance's Xonvea for sickness in pregnancy because the evidence on benefits and cost effectiveness did not stack up.” Paid Subscription Required

Lithuania's New Assessment Procedure for Reimbursable Medicines: Will There Be Any Positive Changes? (5/15, The Pharma Letter) reports “Lithuanian attorney Ruta Pumputiene provides an Expert View on amendments to the Lithuanian Pharmaceutical Law and the subsequent changes to the reimbursable medicines system...These changes will provide the basis for the introduction of an innovative medicines' evaluation mechanism, the so-called health technology assessment, which puts the focus not only on the price but also on the medicine's value in terms of its price and other direct costs.” Paid Subscription Required

Press Releases

NICE Recommends First Ever Treatment for Children with Rare Muscle-Wasting Condition (5/15, NICE Press Release) “Children with the rare genetic disorder spinal muscular atrophy (SMA) can now be treated with nusinersen after NICE today recommended it following agreement of a deal between the company, Biogen, and NHS England. Nusinersen (also called Spinraza) is the first treatment that targets the underlying cause of SMA.” Full

Journals

Health Technology Assessment and Its Use in Drug Policy in Malaysia Shafie AA, et al. May 9, 2019, Value in Health Regional Issues PubMed

Leveraging Patient/Community Partnerships to Disseminate Patient Centered Outcomes Research in Geriatrics Lindquist LA, et al. May 11, 2019, Geriatrics PubMed

Comparative Effectiveness of Antifungal Agents in Patients with Hematopoietic Stem Cell Transplantation: A Systematic Review and Network Meta-Analysis Su HC, et al. May 15, 2019, Infection and Drug Resistance Dove Press

Articles

FDA Approval for Ibrance in Men with Breast Cancer Sets Precedent for Use of Real-World Evidence (5/14, Pharmaceutical Technology) comments “...Due to the small target patient population, leading to the difficulty in conducting a large-scale clinical trial, and the established efficacy of Ibrance in women, the extension of Ibrance into the treatment of men provided a perfect opportunity for the applicability of RWE. Using data from clinical registries, Pfizer was able to demonstrate that the efficacy and safety profile in men was consistent with women treated with Ibrance.” Full

4 Approaches To Data Access Strategy — And How To Apply Them Throughout Drug Development (5/14, Jamie Cartland and Philip Winkworth, Clinical Leader) comments “...Real-world evidence from routine clinical care is increasingly being used in regulatory settings. Regulatory bodies are becoming more comfortable with innovative applications such as external and synthetic control arms to supplement placebo or current standard of care arms in randomized clinical trials.” Full

Utilizing an Integrated, MultiChannel Approach Can Improve Access for Oncology Products (5/13, PharmExec) comments “...Payers are paying increasing attention to that kind of real-world evidence of both safety and effectiveness, she continued. When biopharma companies are building their value story, real world evidence plays an important role. Real-world evidence can also inform commercialization strategies. The distributor has a direct view on treatment and utilization patterns nationwide, where a new product is taking off, where it is lagging behind existing treatments.” Full

Press Releases

AJMC® Study Finds Removing Cost Sharing in Primary Care Reduces Cost, Emergency Visits (5/14, The American Journal of Managed Care Press Release) “When a large national employer removed cost sharing for primary care visits, medical utilization fell, as did monthly costs, driven mostly by reduced emergency department visits. The findings on this value-based insurance design model, coming as the nation grapples with rising healthcare spending, putting pressure on payers, employers, and consumers, are reported in the May issue of The American Journal of Managed Care.

Health Informatics Program Director Selected as Patient Centered Outcomes Research Partnership Scholar (5/13, Idaho State University Press Release) “Idaho State University's Velma L. Payne has recently been accepted to participate in the Patient Centered Outcomes Research Partnership, a highly sought after opportunity offered by the University of Washington. According to their website, the PCORP is a training program designed to prepare scientists, clinicians, and health care managers for conducting research on patient-centered outcomes.” Full

Canada To Open Up Priority Review To More New Drugs (5/14, Vibha Sharma, Pink Sheet) reports “...Using more information about drugs in real-world settings to build up Health Canada's understanding of their benefit-risk profile and to facilitate the approval of drugs for rare diseases...Working with provinces and territories through the Canadian Agency for Drugs and Technologies in Health and the Institut national d'excellence en santé et en services sociaux to align reviews and share information to streamline processes.” Paid Subscription Required

Journals

Cost-Effectiveness of DPP-4 Inhibitor and SGLT2 Inhibitor Combination Therapy for Type 2 Diabetes Manjiri Pawaskar, PhD, et al. May 14, 2019, The American Journal of Managed Care AJMC

Potentially Inappropriate Prescribing Among Older Persons: A Meta-Analysis of Observational Studies Tau Ming Liew, MRCPsych, et al. May/June 2019, Annals of Family Medicine Annals of Family Medicine

Articles

Getting Clinical On New Drug Launches (5/13, Bruce Booth, Forbes.com) reports “...Features of the clinical trials in the regulatory approval packages: Randomized controlled trials clearly remain the gold standard for approval: nearly 90% of drugs included RCTs in their regulatory packages. Trials with active control arms are more common today, and were included in the regulatory filings of nearly half of these new drugs (46%), versus only 20% a few years ago. This highlights the increasing importance of comparative effectiveness in disease indications where existing standards of care are in place.” Full

Novartis Pitches Discounts on Pricey Gene Therapy for Deadly Muscle Disorder (5/10, Deena Beasley and John Miller, Reuters) reports “Novartis AG is offering price discounts in negotiations with U.S. health insurers on its gene therapy for spinal muscular atrophy...Negotiations with payers are in advanced stages, but final pricing will only be set after the drug's approval. Novartis has offered payment alternatives, such as reimbursement in installments over three to five years, or providing discounts if a patient does not see specific benefits from the therapy.” Full

HTA Frameworks for RA Therapies Need Improvement, Researchers Say (5/13, Kelly Davio, The Center for Biosimilars) reports “...According to the paper, which appeared in Plos One, current health technology assessments for RA rely on disease activity measures that are not aligned to guideline-recommended target measures of remission, and they may even be biased in favor of certain therapies insofar as they privilege measures like C-reactive protein reduction (an outcome particularly achieved with interleukin-6 inhibitors).” Full

States Bring Price Fixing Suit Against Generic Drug Makers (5/11, Steve LeBlanc and Martin Crutsinger, Associated Press) reports "...Connecticut Attorney General William Tong, a Democrat, said investigators obtained evidence implicating 20 firms. ‘We have hard evidence that shows the generic drug industry perpetrated a multibillion dollar fraud on the American people,’ Tong said. ‘We have emails, text messages, telephone records and former company insiders that we believe will prove a multi-year conspiracy to fix prices and divide market share for huge numbers of generic drugs.’" Full

Journals

Analysis of Sponsor Hearings on Health Technology Assessment Decision Making Flowers M, Lybrand S, Wonder M. May 10, 2019, Australian Health Review PubMed

Enabling Individualised Health in Learning Healthcare Systems Shannon Wongvibulsin May 11, 2019, BMJ Evidence-Based Medicine BMJ Evidence-Based Medicine

A Cost-Effectiveness Evaluation of the Originator Follitropin Alpha Compared to the Biosimilars for Assisted Reproduction in Germany Xue W, et al. May 13, 2019, International Journal of Women's Health International Journal of Women's Health

Events

Getting Real: The Changing Tide on Real-World Evidence in Drug Development June 5, 2019 Philadelphia Marriott Fierce Biotech