CER Daily Newsfeed

The Comparative Effectiveness Research Daily Newsfeed®, known for short as the CER Daily Newsfeed®, offers the latest news, research and related information on comparative effectiveness research, real-world data and evidence, value assessment and other important health care topics. 

News from Tuesday, September 26, 2023


NPC Evaluates ICER’s Revised 2023 Value Assessment Framework: A Missed Opportunity for Patients and the Field

(9/25, National Pharmaceutical Council: E.V.I.dently Today) comments “...Consistent with NPC’s comments, ICER did not lower its threshold range when stating its opinion on the value of treatments. However, ICER continues to threaten to put its thumb on the pricing scale of innovative treatments. Doing so signals a narrowly focused interpretation of a treatment’s value and can do more harm than good to patient access. ICER documented enhanced approaches to patient engagement. However, it remains to be seen whether these efforts will lead to more patient-focused inputs being incorporated into ICER’s analyses and results vs. being another box-checking exercise.” Full


ICER Finalizes Its Approach to Value Assessment. Industry Remains Dissatisfied

(9/26, Brian Reid, Cost Curve) comments “...The topline conclusion is that ICER’s approach, at its core, isn’t going to change that much...Industry had hoped for better. ‘Look, value assessment is not a rule, it is a tool comprised of both science and opinion,’ NPC’s John O’Brien said via email. ‘We're very concerned about the areas that weren't addressed because they have the potential to harm patients and do a disservice to those who've dedicated their lives to the scientific application of value methods.’” Full


Practice Points Developed for Treatment of Outpatient COVID-19

(9/26, HealthDay News) reports “...‘More research evaluating the efficacy, effectiveness, and comparative effectiveness, as well as harms, of pharmacologic and biologic treatments of COVID-19 in the outpatient setting is needed, particularly in the context of changing dominant severe acute respiratory syndrome coronavirus 2 variants and subvariants,’ the authors write.” Full


Advancing Value-Based Care: AMA, AHIP and NAACOS Team up to Launch New Playbook

(9/26, Dan Cook, Benefits Pro) reports “...[A] collaboration of three major organizations has launched a campaign to address the barriers that have so far clipped VBC’s wings. AHIP, the American Medical Association (AMA), and the National Association of ACOs (NAACOS) unveiled their barrier-busting strategy recently with the release of a playbook designed to work VBC adopters over and under the obstacles. The playbook–The Future of Value-Based Care and Payment–went public with the support of a half-hour webinar devoted to the first phase of implementation: data sharing.” Full

Press Releases

ICER Releases Draft Evidence Report on Treatment for Pulmonary Arterial Hypertension

(9/25, ICER Press Release) “The Institute for Clinical and Economic Review today released a Draft Evidence Report assessing the comparative clinical effectiveness and value of sotatercept (Merck & Co) for pulmonary arterial hypertension...On October 5, 2023, as part of ICER’s Early Insights Webinar Series, ICER’s Chief Medical Officer, David Rind, MD will present the initial findings of this draft report.” Full


Medicare Advantage and Medicare Prescription Drug Programs to Remain Stable in 2024

(9/26, CMS Press Release) “...[M]ore than 1,500 Medicare Advantage plans will participate in the CMS Innovation Center’s Medicare Advantage Value-Based Insurance Design (VBID) Model in 2024, which will test the effect of offering person-centered innovative benefits that are critical to meeting health care needs and improving health equity to a projected 8.7 million people. The VBID Model expands access to additional supplemental benefits that can address a wide range of needs, such as food and nutrition benefits. The VBID Model offers plans the flexibility to target these benefits to people with chronic conditions or low incomes.” Full


COTA Announces New Data Platform to Accelerate Cancer Research and Advance Generative AI Models for Cancer Care

(9/26, COTA Press Release) “...‘Real-world data that tells a more complete story of a patient has accelerated our understanding of cancer and the development of lifesaving and life-extending therapies,’ said Miruna Sasu, CEO of COTA. ‘Generative AI has even more potential to advance treatment options for millions of people if we can build trusted large language models for oncology. That means first curating and validating data at a fundamentally different pace and scale. That’s what we are doing with Vista.’” Full


NICE Draft Guidance Recognises Potential of New Targeted Treatment for Type of Advanced Breast Cancer

(9/26, NICE Press Release) “NICE has today published draft guidance for public consultation that does not recommend trastuzumab deruxtecan for treating advanced HER2-low breast cancer in adults. NICE’s independent appraisal committee has asked the company for more information ahead of its next meeting.” Full


Comparative Effectiveness and Safety of Drug Therapy for Chronic Urticaria: A Network Meta-Analysis and Risk-Benefit Assessment

Tian Qian, et al.

September 25, 2023, Expert Opinion on Drug Safety



The Future of Sustainable Value-Based Payment: Voluntary Best Practices to Advance Data Sharing




Sotatercept for Pulmonary Arterial Hypertension

September 25, 2023



Trustworthy Artificial Intelligence (TAI) for Patient-Centered Outcomes Research (PCOR)

September 26, 2023

Office of the Assistant Secretary for Planning and Evaluation

News from Monday, September 25, 2023


FDA’s Califf: Expect to See More RWE-Based Regulatory Decisions

(9/22, Ferdous Al-Faruque, Regulatory Focus) reports “...[FDA commissioner Robert Califf] said that using RWE to expand product indications has been a sound theory, but it has taken a long time to make it a reality. ‘I'm optimistic we're going to make a real run at it in this upcoming year, across the whole federal government, not just within the FDA,’ said Califf. ‘I believe that we're moving very rapidly now to a place where more and more of the data we use for evidence is going to come from the so-called real-world clinical environment. After all, the reason we do a randomized trial is to take a sample of people and reach a causal inference that allows us to extrapolate to the population,’ he added.” Full


Finding The Sweet Spot For Real-World Evidence: Aetion Stresses Early Feasibility Assessment

(9/24, Bridget Silverman, Pink Sheet) reports “...Robust natural history data sets with well-chosen outcomes are fundamental in Aetion’s regulatory feasibility tool for real-world evidence, built through analysis of US FDA case studies and guidance.” Subscription Required


Editorial: Here's How Medicare Should Negotiate Drug Prices

(9/22, Bloomberg) comments “...[Other countries including Australia and the UK] regularly use cost-effectiveness assessments and, unsurprisingly, their prescription-drug spending is a fraction the US’s. Unfortunately, the process for Medicare won’t be so straightforward. With QALYs banned, it will need to use other (less tested) methodologies. Even so, effective alternatives exist, including some that have been developed to minimize the disparities that disability advocates cite. Medicare should embrace these metrics and be transparent with the public about its pricing methods.” Subscription Required


Congressman Yakym Introduces Legislation to Bring Greater Awareness to Lifesaving Medical Devices

(9/22, Kathy Bottorff, WTCA 106.1) reports “This week, Congressman Yakym (IN-02) introduced H.R. 5458, the ‘Baby Observation Act’ or ‘BO’s Act.’...‘BO’s Act’ requires the Patient-Centered Outcomes Research Institute to study the use of home cardiorespiratory monitors (medical devices that track the respiratory effort and heart rate of a sleeping infant) to reduce and prevent sleep-related sudden infant death syndrome or sudden unexpected infant death.” Full


How Can Predictive Analytics Help ACOs Boost Value-Based Care Delivery?

(9/25, Shania Kennedy, Health IT Analytics) reports “...In the past, ACOs have relied on Medicare data and risk adjustment factor scores to gain insights into their patient populations, but these are limited in that they cannot capture all the complexities associated with a patient and her health. Digital twins—digital or virtual representations of people, objects, processes, or systems designed to help simulate a potential outcome—represent a ‘third wave’ of analysis, [David Clain, chief product officer at the Health Data Analytics Institute,] posited, tying de-identified data from these and other sources to an ACO population.” Full


Gene Therapy HTA: How Do The European, Australian And Canadian Systems Shape Up?

(9/25, Eliza Slawther, Pink Sheet) reports “...A report comparing the health technology assessment methods used in nine European countries, Australia and Canada found that England has the most favorable reimbursement landscape for gene therapies – but outlined several areas for improvement across the board.” Subscription Required

Press Releases

ICER Finalizes 2023 Updates to Value Assessment Framework

(9/25, ICER Press Release) “The Institute for Clinical and Economic Review today published an updated Value Assessment Framework document outlining the principles, methodology, and deliberation procedures that will guide future ICER assessments. In an upcoming webinar on September 28th, President Steven Pearson, MD, MSc, and President-Elect Sarah Emond, MPP, will summarize the philosophy behind ICER’s approach to value assessment, as well as several key updates that will be implemented in the near term.” Full


Evidence into Practice: A National Cohort Study of NICE-Recommended Oncological Drug Therapy Utilisation among Women Diagnosed with Invasive Breast Cancer in England

Melissa Ruth Gannon, et al.

September 23, 2023, British Journal of Cancer

British Journal of Cancer


Comparison of the Efficacy and Safety Between Rivaroxaban and Dabigatran in the Treatment of Acute Portal Vein Thrombosis in Cirrhosis

Haonan Zhou, et al.

September 25, 2023, BMC Gastroenterology

BMC Gastroenterology


Value Assessment Framework

September 25, 2023



Webinar: Updates to ICER’s Value Assessment Framework

September 28, 2023

11:00AM ET


News from Friday, September 22, 2023


Industry-Funded Study: CBO Underestimated IRA’s Impact On Drug R&D

(9/22, Gabrielle Wanneh, InsideHealthPolicy) reports “A policy brief recently released by the University of Chicago and partially funded by drug maker Gilead suggests that Medicare’s new drug price negotiation authority could lead to a reduction in research & development that translates to 79 fewer small molecule drugs developed and 116 million life years lost over the next two decades.” Subscription Required


Rx Price Controls Mean Hard Choices for Investors

(9/21, John Stanford, RealClearHealth) comments “...Small molecule drugs currently make up 75% of all FDA-approved medications, including some modern blockbusters. Several of the first medicines subject to government price controls are small-molecule drugs that have had significant impacts on disease -- including one that helps lower blood-sugar levels for patients with type 2 diabetes and another that inhibits B-cell proliferation to slow the spread of blood cancers. It's fair to ask whether their developers would have proceeded with these medicines if they had known that their products would be among the first subject to price controls.” Full


After FDA Rejection, Lilly's Ulcerative Colitis Drug Omvoh Picks up Support from England's NICE

(9/22, Eric Sagonowsky, Fierce Pharma) reports “...Friday, England’s National Institute for Health and Care Excellence recommended the drug for the treatment of patients with moderate to severe active ulcerative colitis who can’t tolerate—or haven’t responded to—certain other treatments. The NICE blessing sets up the drug, known in the U.K. as Omvoh, to be the first IL-23p19 inhibitor to be used in NHS England, Lilly said in a release.” Full


Understanding the Impacts of OS-PCORTF Projects on Data Capacity: An Interim Qualitative Assessment

September 22, 2023

Office of the Assistant Secretary for Planning and Evaluation


Advisory Panel on Clinical Trials Fall 2023 Meeting

October 19 - 20, 2023


News from Thursday, September 21, 2023


Panel: Alternative Funding for Pharmaceuticals Brings Numerous Risks

(9/21, Karen Blum, Pharmacy Practice News) reports “...Alternative funding models may pose significant risks to individual patient access and the health system, said Mr. Einodshofer and co-panelists Corey Belken, PharmD, a national account manager for Genentech, and John Michael O’Brien, PharmD, MPH, the president and CEO of the National Pharmaceutical Council, a health policy research organization. Disadvantages include the following: They may interfere with patients receiving timely treatment since delays in care could lead to disease progression or a decline in health status; Patients with complex health needs may not have access to the right level of specialty support through the alternative funding vendor.” Full


How Patient Apps Can Help Unlock The Benefits Of Real-World Data

(9/21, Kal Patel M.D., MBA, Forbes.com) comments “...Many pharma companies look to companion apps or patient trackers to initiate patients onto new treatments and ensure that they’re adherent and compliant with those regimens...That initial RWD can offer some valuable insights immediately to help optimize digital tools and reveal patient journey pain points. And RWE holds even greater potential, both for improving patients’ lives and pharma’s bottom line. But to gain this untapped potential, brand and RWE teams must not be kept in separate silos—that prevents developing the necessary holistic approach that can drive value and insights for both teams.” Full


A Powerful Methodology to Guide Health Decision-Making

(9/21, Harvard T.H. Chan School of Public Health) reports “...[O]bservational studies from the past decade or so suggested that patients who took statins, which are drugs used to treat heart disease, had around half the risk of cancer. However, randomized trials showed that there was no such effect, suggesting that statins should not be used for cancer prevention. To resolve the discrepancy, Dickerman and her colleagues analyzed a U.K. national database of electronic health records and found that using causal inference methods would have resulted in the correct conclusion found in the randomized trials. ‘This was an important case study to show how you can run into major bias simply by applying the wrong analytic methods to observational data,’ she said. ‘It also proposed a better way forward that may serve as a model for investigators who use electronic health records for cancer comparative effectiveness research.’” Full

Press Releases

Truveta and Mathematica Partner to Advance Public Health and Health Equity with Real-World Data

(9/21, Truveta Press Release) “...‘Truveta’s mission is saving lives with data. With this partnership with Mathematica, we will be able to advance medical research in many ways, informing important public health conversations, advancing health equity, and addressing maternal health,’ said Ryan Ahern, MD, MPH, chief medical officer and co-founder, Truveta. ‘By combining Truveta’s complete, timely, and clean EHR data that is representative of the diversity of the United States with Mathematica’s experience in public health and policy, we can advance healthcare for Americans.’” Full


Comparative Value of Dapagliflozin vs Empagliflozin in Patients with Heart Failure and Preserved Ejection Fraction: A Cost-Effectiveness Analysis

Amey Rane, BPharm, MS, et al.

September 2023, Journal of Managed Care & Specialty Pharmacy



Comparative Effectiveness of Umeclidinium/Vilanterol versus Indacaterol/Glycopyrronium on Moderate-to-Severe Exacerbations in Patients with Chronic Obstructive Pulmonary Disease in Clinical Practice in England

Gema Requena, et al.

September 15, 2023, International Journal of Chronic Obstructive Pulmonary Disease



Sources of Low-Value Care Received by Medicare Beneficiaries and Associated Spending Within US Health Systems

Emma D. Chant, PhD; Maia Crawford, MS; Ching-Wen Wendy Yang, MSPH; et al

September 20, 2023, JAMA Network Open

JAMA Network Open


Comparative Efficacy of Venetoclax-based Combination Therapies and Other Therapies in Treatment Naïve Patients with Acute Myeloid Leukemia (AML) Ineligible for Intensive Chemotherapy: A Network Meta-Analysis

Xue Li, PhD, et al.

September 20, 2023, Value in Health

Value in Health

News from Wednesday, September 20, 2023


Two Pediatric Sinusitis Antibiotics Found Equally Effective

(9/29, Chris Dall, MA, CIDRAP) reports “The results of a large observational study in children show no difference in treatment failure rates between the two most commonly prescribed antibiotics for pediatric acute sinusitis, researchers reported today in JAMA. Overall, treatment failure for sinus infections was rare in children treated with amoxicillin or amoxicillin-clavulanate. But the latter was associated with more adverse events...” Full


Value-Based Payments Are Making it Harder to See Your Primary Care Doctor on Short Notice

(9/20, Jeffrey Millstein, STAT) comments “...Physician leaders must push for value-based incentives that better align with patient needs and goals, and allow primary care clinicians the autonomy to balance routine and acute/subacute visits to meet practice demands. For instance, limiting yearly comprehensive visits to patients in the highest risk subgroups should meet the metric threshold.” Full


Shutdown Threat Clouds Start of Biden Drug-Pricing Negotiations

(9/20, Alex Ruoff, Bloomberg Law) reports “...A potential government shutdown at the end of the month risks complicating the Biden administration's top health priorities, just as drugmakers are supposed to report data for pricing negotiations and states are pushing people off Medicaid rolls...A shutdown would bump up against an Oct. 2 deadline for some drugmakers to report a slew of data to Medicare as part of how the agency will eventually determine new prices for medicines selected for negotiation.” Full

Press Releases

PCORnet® Leaders Hit The Road To Share How The Network Works And Why Researchers Should Use It To Power Their Next Study

(9/20, PCORnet Press Release) “Why and how should you use PCORnet®? That’s the question PCORnet leaders sought to answer when they recently presented at an adjunct event for AcademyHealth’s 2023 Annual Research Meeting (ARM) and at a half-day virtual convening held by NORC in collaboration with PCORI. Both events were a chance to showcase the uniqueness of PCORnet, which was developed in 2014 with funding from PCORI as a national resource for all.” Full


Addressing Missing Data in the Estimation of Time-Varying Treatments in Comparative Effectiveness Research

Juan Segura-Buisan, Clemence Leyrat, Manuel Gomes

September 19, 2023, Statistics in Medicine



Treatment Failure and Adverse Events After Amoxicillin-Clavulanate vs Amoxicillin for Pediatric Acute Sinusitis

Timothy J. Savage, MD, MPH, MSc, et al.

September 19, 2023, JAMA



Network Meta-Analysis of Adjuvant Treatments for Patients with Hepatocellular Carcinoma after Curative Resection

Yanyan Ye, et al.

September 20, 2023, BMC Gastroenterology

BMC Gastroenterology


Challenges of Calculating Cost-Effectiveness Thresholds – Authors' Reply

Andres Pichon-Riviere, et al.

October 2023, The Lancet Global Health

The Lancet Global Health

News from Tuesday, September 19, 2023


Real-World Data Or Studies Of Competitor Drugs Can Serve As Confirmatory Evidence For US FDA

(9/18, Brenda Sandburg, Pink Sheet) reports “...Draft guidance describes seven types of confirmatory evidence that can be used with one adequate and well-controlled clinical study to demonstrate substantial evidence of effectiveness, including evidence from expanded access, real-world data, and studies of other drugs in the same class.” Subscription Required


FDA's Single-Trial Guidance Calls for the Good, Bad and Ugly Data to Support Effectiveness

(9/19, Annalee Armstrong, Fierce Biotech) reports “...To show confirmatory evidence, drug sponsors have plenty of options and they may not have to reinvent the wheel to find it. The FDA provided some examples...Options include efficacy evidence from a previously submitted or approved indication for a drug; preclinical data from an established animal mode; evidence from therapies in the same pharmacological class; natural history data; real-world data or evidence; and data from an expanded access program.” Full


Paxlovid and Lagevrio Benefit COVID Outpatients in Omicron Era

(9/19, Diana Swift, Medscape) reports “...‘The practice points only address [whether] treatments work compared to placebo, no treatment, or usual care,’ cautioned Linda L. Humphrey, MD, MPH, MACP, chair of the ACP's Population Health and Medical Science Committee and a professor of medicine at Oregon Health and Science University VA Portland Health Care System. The ACP continues to monitor the evidence. ‘Once enough evidence has emerged, it will be possible to compare treatments to each other. Until that time we are unable to determine if there is an advantage to using one treatment over another.’” Subscription Required


Dr Ryan Haumschild Discusses Payer, Provider Perspectives on Prior Authorization in Rare Diseases

(9/19, Pearl Steinzor, The American Journal of Managed Care) reports “...[Ryan Haumschild, PharmD, MS, MBA:] As we're evaluating rare diseases, we need to look at the value they bring. We need to look at value not just on acquisition cost, we need to look at it from total cost of care perspective or quality of life perspective, or even looking at the ICER and the quality to say, ‘Does this meet the minimum threshold?’ And if it doesn't, we need to have those open conversations with providers. Maybe there might be a sub-population that may benefit the most, but not all populations are going to benefit. And be very clear about that.” Full

Press Releases

Cristina Murray-Krezan Will Chair the PCORI Clinical Trials Advisory Panel

(9/19, University of Pittsburgh) “Cristina Murray-Krezan, associate professor of medicine and clinical and translational science at Pitt, was selected by the Patient-Centered Outcomes Research Institute (PCORI) board of governors as chair of the organization’s Clinical Trials Advisory Panel. The panel advises those conducting research through the PCORI Methodology Committee on the selection, research design, implementation, and technical issues of clinical trials for patient-centered outcomes. Murray-Krezan has served on the committee for three years and served as co-chair in 2022-23.” Full


Curta Selected as Partner to the Peterson Health Technology Institute (PHTI) to Provide Independent Health Technology Assessment Evaluations of Innovative Digital Health Technologies

(9/19, Curta Press Release) “...As a leader in the field of health technology assessment, Curta was selected to bring learnings from the from decades of experience in comparative and economic assessment to the evaluation of digital health technologies. ‘Due to Curta's unique and deep understanding of HEOR and HTA in the US, we make the perfect partner to PHTI for this important and precedent-establishing program to evaluate digital health technologies under the ICER-PHTI Assessment Framework for Digital Health Technologies.’ –Lisa Bloudek, PharmD, MS, CEO, Curta.” Full

News from Monday, September 18, 2023


Is ICER's New Digital Health Value Assessment Framework Relevant as Technology Pivots to OTC?

(9/15, Kimberly Westrich, LinkedIn) comments “...Last week, No Patient Left Behind published a white paper titled, ‘Getting the Math Right When Measuring the Value of New Medicines,’ which found that traditional cost-effectiveness analysis methods often omit sources of social value that, when incorporated, move the needle on cost-effectiveness estimates. The authors reviewed ICER assessments for 20 medications, only 8 of which were found to be cost-effective using ICER’s traditional CEA methods. However, after applying generalized cost-effectiveness analysis (GCEA) to account for 2 additional dimensions of value, including diminishing returns to health improvement and dynamic pricing, the authors found that at least 17 of the 20 assessed medications offered good value for money from a societal standpoint.” Full


Blackstone's $250M Bet Beats Xarelto in Bleeding Trial, Making Case for Anthos' Ex-Novartis Asset

(9/18, Nick Paul Taylor, Fierce Biotech) reports “...The data show Anthos Therapeutics’ abelacimab significantly cut bleeding compared to Bayer and Johnson & Johnson’s Xarelto, adding to evidence that the biotech's candidate can treat thrombosis without affecting hemostasis.” Full


EMA Waves Goodbye To HTA Network As Collaboration Framework Enters New Phase

(9/18, Eliza Slawther, Pink Sheet) reports “...A report published by the European Medicines Agency highlights milestones reached under its collaboration with EUnetHTA over recent years and outlines areas for further development once the initiative changes hands under the new HTA Regulation.” Subscription Required

Press Releases

Cochrane Partners with International Coalition to Advance Global Health Equity on World Evidence-Based Healthcare Day

(9/18, Cochrane Press Release) “...The 2023 campaign is a call to action to take concrete steps towards equity-centred evidence-informed decision making, drawing attention to the need to enhance collaboration, information sharing and networking to increase access to research evidence, and to move towards integrated systems that prioritise equity, inclusivity and multisectoral participation of all actors.” Full


Tolerability and Comparative Effectiveness of TNF-, IL-17-, and IL-23(p19) Inhibitors in Psoriatic Arthritis: A Target Trial Emulation Study

Zara R Stisen, et al.

September 15, 2023, Rheumatology



Comparative Effectiveness of Aspirin Dosing in Cardiovascular Disease and Diabetes Mellitus: A Subgroup Analysis of the ADAPTABLE Trial

Dennis I Narcisse, et al.

September 15, 2023, Diabetes Care



Comparison of Pharmacological Therapies in Relapse Rates in Patients With Relapsing-Remitting Multiple Sclerosis

Indu Etta, et al.

September 18, 2023, Cureus


News from Friday, September 15, 2023


Real-World Data in a Post-Pandemic World

(9/15, Surani Fernando, Medical Marketing & Media) reports “...As for the FDA, when asked via email about source-related challenges, the spokesperson responded: ‘In regards to real-world data sources, we find issues related to data reliability and clinical relevance, a need for linkage to other data sources, missing or “mistimed” data and insufficient capture of endpoints. For non-randomized study designs, issues include the threat of residual confounding, problems with index date (“zero time”) and use of an inappropriate comparator.’” Full


Three Outstanding Questions About CMS’s Ambitious New AHEAD Model

(9/14, Troyen A. Brennan, Health Affairs Forefront) comments “...As a hospital executive, your key strategy, perhaps your only strategy, has been to increase in size, gain leverage with insurers, bargain for better fee-for-service rates, and do more procedures. Working under a prospective budget blocks that strategy. So one might ask about Pennsylvania, would centers like UPMC in the west, and Jefferson or University of Pennsylvania in the East, be ready to abandon the fee-for-service revenue generation program for one based solely on value-based care that improves population management. I wish that were true, but it seems doubtful.” Full


Americans Overwhelmingly Support Medicare Drug Negotiations, But Biden Sees Little Political Boost

(9/15, Seung Min Kim and Linley Sanders, Associated Press) reports “...Three-quarters of Americans, or 76%, favor allowing the federal health care program for the elderly to negotiate prices for certain prescription drugs. That includes strong majorities of Democrats (86%) and Republicans (66%), according to a new poll from The Associated Press-NORC Center for Public Affairs Research. About one in five Americans are neutral on the issue, while 6% outright oppose it. But the same poll shows Biden's approval rating, at 40%, is about where it's been for the last year.” Full

Press Releases

More People Could Be Offered Genetic Testing for Genes Linked to Ovarian Cancer in New Draft NICE Guideline for Public Consultation

(9/15, NICE News Release) “A new draft NICE guideline for inherited ovarian cancer published today (15 September 2023), recommends more people are tested for genes linked to ovarian cancer...The guideline aims to raise greater awareness and increase the availability of genetic testing, allowing people to take preventive measures such as surgery, meaning fewer people will go on to develop ovarian cancer.” Full


Towards a Permanent Collaboration Framework for EMA and Health Technology Assessment Bodies

(9/15, European Medicines Agency Press Release) “...‘EMA’s collaboration with EUnetHTA began in 2010 as a project to test out whether early engagement between regulators and HTA bodies could bring tangible benefits for patient access to medicines,’ said Michael Berntgen, Head of Scientific Evidence Generation Department at EMA. ‘Together, we were able to help medicine developers enhance clinical research and become more efficient in generating the evidence relevant for both regulatory authorities and HTA bodies.’” Full


The Cost–Effectiveness of Pegcetacoplan in Complement Treatment-Naïve Adults with Paroxysmal Nocturnal Hemoglobinuria in the USA

Jesse Fishman, et al.

September 2023, Journal of Comparative Effectiveness Research

Journal of Comparative Effectiveness Research


Comparative Effectiveness of Umeclidinium/Vilanterol versus Indacaterol/Glycopyrronium on Moderate-to-Severe Exacerbations in Patients with Chronic Obstructive Pulmonary Disease in Clinical Practice in England

Requena G, et al.

September 15, 2023, International Journal of Chronic Obstructive Pulmonary Disease

International Journal of Chronic Obstructive Pulmonary Disease


A Real-World Comparison of Apixaban and Rivaroxaban in Obese and Morbidly Obese Patients With Nonvalvular Atrial Fibrillation

Kevin T Burnham, et al.

September 15, 2023, Journal of Pharmacy Practice



Viewpoint: The Problem of Limited-Supply Agreements for Medicare Price Negotiation

Sarah M. E. Gabriele, LLM, MBE; William B. Feldman, MD, DPhil, MPH

September 15, 2023, JAMA


News from Thursday, September 14, 2023


US FDA Commissioner Pushes Real-World Evidence To Guide Use Of Alzheimer’s, Obesity Drugs

(9/13, Kate Rawson, Pink Sheet) reports “...Califf has been particularly interested in using real-world evidence in the post-approval setting, which he described as a ‘system in shambles.’ He reiterated that ‘we're not talking about the fundamental structure of drug and device approval. I think that works pretty well. We are talking about what happens after that, where FDA is only part of the system.’ Califf reiterated his view that other countries have a longer life expectancy and a better quality of life (he mentioned Singapore, specifically), ‘because they have organized approaches to information.’ Israel uses real-time electronic health data, he noted, and the UK National Health Service enables a streamlined approach in which clinical trials are integrated directly into clinical care, ‘in other words, real-word evidence.’” Subscription Required


Pushing Both Sides of the Drug Pricing Aisle

(9/14, Asher Mullard, Nature Reviews Drug Discovery: An Audience With) reports “Few topics are as controversial in healthcare as drug pricing. As prices edge ever higher, budgets are stretched ever thinner. And while some patients and clinicians clamor for new and better therapeutics, others question the value of the drugs that are reaching the market. The Institute for Clinical and Economic Review...has leaned into this tension, developing systematic and transparent methods to assess the cost-effectiveness of new treatments. With ICER’s founding President Steve Pearson now stepping down, President-elect Sarah Emond is ready to take up the medical mantle.” Subscription Required


England's NICE Backs Pfizer's Vydura as Treatment for Acute Migraines

(9/13, Kevin Dunleavy, Fierce Pharma) reports “...This time, NICE still stopped short of recommending Vydura for primary use. According to the draft guidance, rimegepant is endorsed for adults who have had unsuccessful results with at least two triptan medicines. Vydura also is suggested for use by those who can't take triptans and had unsuccessful results with nonsteroidal anti-inflammatory drugs (NSAIDs) or the beta blocker paracetamol. ‘NICE understands that rimegepant could eventually be used in primary care but should initially be started in secondary care,’ the regulator said...” Full

Press Releases

ICER Publishes Evidence Report on Gene Therapy for Metachromatic Leukodystrophy

(9/14, ICER Press Release) “The Institute for Clinical and Economic Review today posted its revised Evidence Report assessing the comparative clinical effectiveness and value of atidarsagene autotemcel (“arsa-cel”, Orchard Therapeutics) for metachromatic leukodystrophy (MLD).” Full


Researchers Will Use PCORnet® Resources To Explore Whether Cancer Screening Practices Change After Weight Loss Surgery

(9/13, PCORnet Press Release) “...Can weight loss associated with bariatric surgery reduce the risk of some cancers? Maybe, according to a recent meta-analysis of several key studies, which found that people with obesity who had bariatric surgery had a substantially lower risk of developing five types of solid tumors. But it remains unclear if the factors driving the risk reductions might be physical, behavioral, or both. Now, a new study funded by the National Cancer Institute will use PCORnet resources to understand if one of those factors is a change in adherence to recommended preventative cancer screenings after bariatric surgery.” Full


Atidarsagene Autotemcel for Metachromatic Leukodystrophy

September 14, 2023


News from Wednesday, September 13, 2023


Osimertinib Plus Chemotherapy Demonstrated Greater PFS Benefit in Patients With EGFRm Advanced NSCLC Than Osimertinib Alone

(9/12, Justina Petrullo, The American Journal of Managed Care) reports “Osimertinib with the addition of chemotherapy showed a statistically significant and clinically meaningful progression-free survival (PFS) benefit compared with only osimertinib in patients with epidermal growth factor receptor–mutated (EGFRm) advanced non–small cell lung cancer (NSCLC) in the FLAURA2 study, according to results presented at the International Association for the Study of Lung Cancer 2023 World Conference on Lung Cancer.” Full


A Framework For Prioritizing Pharmaceutical Supply Chain Interventions

(9/13, Marta E. Wosinska, et al., Health Affairs Forefront) comments “...To assess criticality is to address the following question: Without which pharmaceuticals will individual patients suffer great harm? The answer to this question will necessarily consider the severity of the condition the medicine treats and the comparative effectiveness of the drug relative to alternatives. In the criticality assessment, it is important to consider heterogeneity of treatment effect and the drug’s importance to specific vulnerable subgroups that face challenges in accessing needed medical care.” Full


Pharma Cries Procedural Foul in Next Front of Drug Price War

(9/13, Ian Lopez, Bloomberg Law) reports “...AstraZeneca's complaint in particular has gotten the attention of legal experts. The company argues that the HHS overrides the statutory definition of Qualifying Single Source Drug, for which there's no biosimilar or generic competition, and essentially lumps together separately approved products and justifies making them eligible for negotiations...‘They're claiming an excess of statutory authority,’ and ‘they have a point,’ said Robert Charrow, who served as HHS general counsel in the Trump administration.” Subscription Required


America’s New Drug-Pricing Rules Have Perverse Consequences

(8/30, The Economist) comments "…According to research published in 2021, once their spending on research and development is treated as an investment rather than an expense, pharma firms are not making outsize returns compared with the average firm in the S&P 500. If they doubt that they will make a sufficient profit on their investments, they will spend less on finding new drugs. Sure enough, studies suggest that falling revenues hit research and development spending hard. This is typical of the unintended and undesirable effects from price regulation. Lower prices are popular with patients today and mean less of a drain on the public purse in the near term. But if they discourage investment in new medicines, that will be to the detriment of patients and society tomorrow.” Subscription Required


Pharma Industry Urges HTA Bodies To Look Beyond Survival Data For Cancer Drugs

(9/13, Eliza Slawther, Pink Sheet) reports “...A new report by industry association EFPIA says that health technology assessment bodies often rely too heavily on overall survival data when assessing the value of novel cancer drugs, and should take into account other clinical and patient-reported outcome measures where appropriate.” Subscription Required

Press Releases

Mental, Maternal and Heart Health Headline PCORI’s 2023 Annual Meeting October 4-5

(9/13, PCORI Press Release) “...Two full days of dynamic sessions will tackle some of the most newsworthy health issues and greatest concerns among health care stakeholders, such as: Improving perinatal mental health. Using social determinants of health data to advance health equity. Improving mental health for people with intellectual and developmental disabilities. Creating pathways to optimal heart health for women. Addressing racism and discrimination in care delivery. Leveraging telehealth in the post-pandemic environment.” Full


Improving the Understanding, Acceptance and Use of Oncology–Relevant Endpoints in HTA Body / Payer Decision-Making

(9/12, EFPIA Press Release) “In clinical trials, overall survival (OS), defined as the time from randomisation (assignment of clinical trial participants to treatment groups) until death from any cause, remains a robust measure of the clinical benefit of cancer medicines. Its objectivity, suitability for comparing treatment regimens and relevance to patients have made it the preferred measure of clinical efficacy in regulatory and HTA body / payer decision-making. However, reliance on OS data in regulatory and HTA body / payer decisions has its limitations.” Full


A Clinical Pathway to Well-Being: Putting Patient Priorities at the Center of Care

Neil Wagle, MD, MBA, Claire Campbell, MD

September 2023, The American Journal of Accountable Care



Improving the Understanding, Acceptance and Use of Oncology–Relevant Endpoints in HTA Body / Payer Decision-Making

September 2023

European Federation of Pharmaceutical Industries and Associations