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 Friday, December 1, 2023


Without Comprehensive PBM Reform, Patient Health Outcomes Will Suffer

(12/1, Liz Helms, RealClearHealth) comments “...By putting medications on formulary tiers too costly for many patients, denying the value of copay assistance towards patient cost-sharing requirements, or making patients try and fail on other medications first, PBM practices disrupt the process patients use to manage their health. I have heard directly from physicians that, in some cases, it has taken three to four months for their patient to access a medicine that works for them.” Full


‘Vast Amount Of Valuable Input’ Leads Australia To Extend HTA Review

(12/1, Ian Schofield, Pink Sheet) reports “...A second public consultation on the review of the HTA system will be held in January to February 2024 after more than 100 stakeholder responses were received during the first consultation earlier this year.” Subscription Required


Japan's CEA Scheme: How It Works And Impact So Far

(11/30, Lisa Takagi, Pink Sheet) reports “...Japan's Economic Effectiveness Price Cut Scheme, a system of cost effectiveness assessment for drug reimbursement under the country's national health insurance program, has now entered its fourth year with a new list of drugs coming under evaluation...In this analysis, the Pink Sheet takes a look at how the scheme has worked so far, its latest outcomes and the list of the drugs undergoing evaluation as of November 2023.” Subscription Required

Press Releases

Arman Oganisian and Roee Gutman Approved for ~$1 Million for Study on Bayesian Machine Learning for Causal Inference in EHR data with Missing Covariates

(11/30, Brown University School of Public Health News Release) “A research team at the Brown University Center for Statistical Science has been approved for a $920,991 funding award by the Patient-Centered Outcomes Research Institute for a methodology study on Bayesian Machine Learning for Causal Inference in EHR data with Missing Covariates.” Full


American College of Medical Toxicology and the American Academy of Clinical Toxicology Position Statement: Nalmefene Should Not Replace Naloxone as the Primary Opioid Antidote at this Time

Andrew I Stolbach, et al.

December 1, 2023, Clinical Toxicology


News from Thursday, November 30, 2023


Building Effective Real World Evidence Studies to Support New Indications for Established Products

(11/30, Dr. Samy Suissa and Dr. Meg Richards, Pharmaceutical Executive) comments “...To craft effective RWE studies in the future, researchers must steer away from the outdated ways of conducting observational studies, and instead begin utilizing emulated randomized trial approaches using real-world observational data. For example, the 2015 treatment guidelines for idiopathic pulmonary fibrosis recommended the use of proton pump inhibitors based on observational studies impacted by immortal time bias. A team at McGill recently conducted an observational study of this question using a prevalent new-user design that imitates a randomized controlled trial.” Full


Atropos Health forms Life Science Advisory Board with Pharma Industry Experts

(11/30, Liza Laws, BioPharma Reporter) reports “A Life Sciences Advisory Board has been launched by Atropos Health, a company that translates data into personalized real-world insights across healthcare. The life sciences advisory board indicates a continued focus on regulatory complaint and transparent methods for rapid and responsible acceleration of innovation for life science partners on the Evidence Network. The board will consist of executives from the world's preeminent pharma companies including Moderna, Novartis among others.” Full


Big Pharma is 'Reeling' Over the Inflation Reduction Act. Now a Philly-Area Drugmaker is Fighting Back.

(11/30, Andrew Seidman, The Philadelphia Inquirer) reports “...[Merck] filed a lawsuit in June alleging the Inflation Reduction Act's drug price negotiation program for Medicare amounts to extortion and violates the U.S. Constitution...Analysts say drugmakers are hoping to expand existing exemptions to the law. Pascal Soriot, CEO of AstraZeneca...pointed to a provision of the law that exempts some drugs that treat rare diseases. He suggested it was too narrow.” Subscription Required


UK: MSD & Kyowa Question NICE’s Processes After English Funding Rejected For Rare Disease Drugs

(11/30, Eliza Slawther, Pink Sheet) reports “...MSD and Kyowa Kirin have separately criticized England’s HTA body, NICE, for assessing their respective rare disease medicines Welireg and Crysvita using its standard appraisal pathway. Both medicines were provisionally rejected for reimbursement.” Subscription Required

Press Releases

New Study Finds 92% of New FDA Approved Medicines Have No Federally Funded Intellectual Property or Patents

(11/30, Vital Transformation Press Release) “New research released by Vital Transformation finds the pharmaceutical industry is the dominant source of innovation for funding new FDA approved medicines. By studying a cohort of 361 new FDA approved medicines and patents protecting the assets between 2011-2020, VT found that 92% of the medicines they researched were directly discovered by industry, and have no government interest statement, federally funded co-development, or federal partnership program associated with any patents core to the development of the medicine.” Full


More than $13M Awarded to Study Childhood Obesity Interventions in Rural and Minority Communities in Louisiana and Tennessee

(11/30, Pennington Biomedical Research Center Press Release) “Pennington Biomedical Research Center and Vanderbilt University Medical Center have received $13.8 million for five years of research funding from the Patient-Centered Outcomes Research Institute to study the ideal ‘dose’ of behavioral interventions to treat childhood obesity in rural and minority communities across Louisiana and Tennessee.” Full


Casey Taft, PhD, Awarded Funding to Prevent Intimate Partner Violence

(11/29, Boston University Chobanian & Avedisian School of Medicine News Release) “Casey Taft, PhD, professor of psychiatry, has been approved for a five-year, $2.8 million funding award from the Patient-Centered Outcomes Research Institute for his research study ‘A Randomized Controlled Trial to Evaluate a Trauma-Informed Partner Violence Intervention Program.’” Full


PCORI Grant Awards $2.1M To Downstate Health Sciences University and University Of Illinois Urban-Champaign For Inclusive Research Study

(11/29, Downstate Health Sciences University Press Release) “A research team at the School of Public Health at Downstate Health Sciences University led by Thomas I. Mackie, Ph.D., MPH, Chair of Health Policy and Management, has secured a $2.1M award from the Patient-Centered Outcomes Research Institute to conduct a study to advance the meaningful engagement of communities underrepresented in research partnerships. The study will be co-led by Karen Tabb, Ph.D., from the University of Illinois Urban-Champaign.” Full


March-In Rights Under the Bayh-Dole Act & NIH Contributions to Pharmaceutical Patents

November 30, 2023

Vital Transformation

News from Wednesday, November 29, 2023


What Pain Medications Work Best for Adolescents and Young Adults?

(11/28, Ellin Kavanagh, Children’s Hospital Los Angeles Blog) comments “...‘We are grateful to PCORI for taking a leadership role in funding multiple trials to develop research-based evidence about the effectiveness of using pain medication in adults,’ says [Lorraine Kelley-Quon, MD]. ‘But since it isn’t possible to extrapolate information gained from an adult study to a pediatric population, this is a truly unique study—done in adolescents and young adults—and will impact millions of families each year.’” Full


VUMC Receives $10 Million to Study Childhood Obesity Interventions in Rural and Minority Communities in Tennessee and Louisiana

(11/28, Christina Echegaray, VUMC Reporter) reports “Vanderbilt University Medical Center has received a $10 million, five-year research funding award from the Patient-Centered Outcomes Research Institute to study the ideal ‘dose’ of behavioral interventions to treat childhood obesity in rural and minority communities across Tennessee and Louisiana.” Full


CU Department of Medicine Cardiologist Lands $7 Million Funding Award for Nationwide Study on Improving Heart-Failure Treatment

(11/28, Mark Harden, University of Colorado School of Medicine) reports “...The PCORI award will fund what [Larry Allen, MD] calls a ‘compare and contrast’ study that will examine whether versions of EPIC-HF and PROMPT-HF, modified to fit into each of the five health systems’ clinical workflow, work better individually or in combination to improve treatment of heart-failure patients. The study will include a randomized trial involving about 200 cardiology providers and 2,000 patients.” Full


Sanacora, Wilkinson Receive $12.6M in Funding for Ketamine Study

(11/28, Yale School of Medicine) reports “Gerard Sanacora, MD, PhD, George D. and Esther S. Gross Professor of Psychiatry, and Samuel Wilkinson, MD, associate professor of psychiatry, have received $12.6 million in funding from the Patient-Centered Outcomes Research Institute for the study, ‘Comparative Effectiveness of Racemic Ketamine versus S-Ketamine (Spravato) for Depression.’ The project will compare two new treatments for treatment-resistant depression: racemic ketamine (IV ketamine), delivered intravenously, and esketamine (Spravato), delivered as a nasal spray.” Full


Harvard Scholars Call to Eliminate Orphan Drug Exemption in IRA to Save Medicare More

(11/28, Zachary Brennan, Endpoints News) reports “...‘Had the IRA been in effect during this period, the sole orphan exemption would have prevented Medicare from negotiating prices on otherwise eligible drugs with a total $1.1 to $3.0 billion in Medicare spending each year,’ wrote the authors from Harvard's business school, medical school and government school. But pharma execs like former Genentech CEO Alexander Hardy, who will soon take the helm at BioMarin, previously explained to Endpoints News how the orphan exemption is also currently affecting which indication to enter first.” Full


The Role of Payment and Financing in Achieving Health Equity

Brede H. Eschliman MPH, et al.

November 28, 2023, Health Services Research


News from Tuesday, November 28, 2023


Arriving At A Fair Price In The Medicare Drug Price Negotiation Program: Considerations From Other Countries

(11/28, John K. Lin, et al., Health Affairs Forefront) comments “...[W]hile we recognize this is outside the current scope of the IRA’s mandate to CMS, in the long-term, we strongly recommend that the United States establish a publicly funded and impartial health technology assessment body. Drug assessments can be complicated, requiring technical expertise across numerous disciplines. Drug negotiations can become political. Canada, France, and Germany have been successful in part because they each have developed fully independent technical bodies with the requisite scientific expertise and freedom to assess each drug without political influence, partiality, or an agenda of cutting costs.” Full


PCORI Funds Four Studies on Maternal Health Equity

(11/28, David Raths, Healthcare Innovation) reports “...Two studies will address maintaining a healthy maternal weight to prevent delivery or postpartum complications among primarily low-income patients in Arkansas and New York City, respectively...A third study to be undertaken in 10 counties in North Carolina will track the results of a multi-pronged program that includes home-based blood pressure monitoring, provider education and support for community health workers to reduce hypertensive disorders of pregnancy...” Full


PCORI Funds 4 New Large Studies Focused on Maternal Health

(11/28, Anastassia Gliadkovskaya, Fierce Healthcare) reports “...‘These funding awards mark an important advancement of PCORI’s longstanding leadership in engaging patients and those who care for them in all aspects of comparative clinical effectiveness research to ensure that results are relevant, useful and impactful,’ PCORI Deputy Executive Director for Patient-Centered Research Programs, Harv Feldman, M.D., said in the announcement. ‘We look forward to seeing the impact the studies’ findings will have for maternal health across the United States, particularly among populations that continue to disproportionately experience adverse outcomes.’” Full


Brolucizumab as Effective as Aflibercept in Macular Edema Treatment

(11/28, Julia Bonavitacola, The American Journal of Managed Care) reports “There were no clinically meaningful differences in visual outcomes when patients with diabetic macular edema (DME) were treated with either brolucizumab or aflibercept, according to a study published in JAMA Ophthalmology. Anatomic improvements were found in patients who had received brolucizumab.” Full


In the Wild, Lilly’s Mounjaro Holds Weight Loss Edge Over Novo’s Ozempic

(11/28, Marc Iskowitz, Medical Marketing & Media) reports “...A trial comparing tirzepatide to semaglutide in patients with overweight or obesity, but without diabetes, is underway though results aren’t expected until late next year. Added [VP of Truveta Research Nicky Stucky], a practicing physician, ‘We’ve been able to compare the head-to-head efficacy of these two important medications for weight loss in advance of randomized clinical trials. This study can help to inform patient care and outcomes today, not months from now.’” Full


The Future State of Value-Based Care

(11/27, NEJM Catalyst) reports “In his keynote address, the Chairman and Co-founder of the International Consortium for Health Outcomes Measurement describes the need for value-based care models over fee-for-service to improve health care quality, and the evolution mindset needed for health care leaders to shape care of the future that focuses on four levers central to adaptive systems. He stresses the importance of outcomes measurement and that tools already exist for clinicians and leaders to begin the journey toward value and outcomes that actually matter to patients.” Full


Medicare ACO Enrollment Did Not Improve Mental Healthcare Outcomes

(11/28, Victoria Bailey, Health Payer Intelligence) reports “Medicare beneficiaries newly enrolled in accountable care organizations did not see any improvements in their depression and anxiety symptoms, suggesting Medicare ACOs may need better incentives to improve mental healthcare, a study published in Health Affairs found.” Full

Press Releases

PCORI Approves $225 Million in Funding for Dozens of Health Research Studies and Related Projects

(11/28, PCORI Press Release) “...Four awards include support for large patient-centered CER studies in which community organizations and research institutions as coequal partners will tackle clinical and social factors that contribute to inequities in maternal morbidity and mortality...This latest round of PCORI funding awards also includes CER studies researching questions about how to optimally use brief interventions to prevent alcohol use among adolescents; ways to prevent delirium among hospitalized patients, a particular concern for older adults and their families; strategies to achieve better blood pressure control; and ways to enhance use of cancer screening and treatment of depression.” Full


PCORI Approves $80.5 Million for Health Research Using Novel Approaches to Tackle Social and Clinical Care Factors That Contribute to Maternal Health Inequities

(11/28, PCORI Press Release) “...‘The usual approaches to health research and health care have not sufficiently addressed the alarming and worsening national crisis of maternal death and severe illness,’ said PCORI Executive Director Nakela L. Cook, M.D., MPH. ‘Patient-centered comparative clinical effectiveness research that responds to the many challenges concurrently facing pregnant individuals and those who care for them has the capacity to answer questions about which combinations of approaches can best resolve some of these complex maternal health care challenges that have for too long defied solutions.’” Full


Ohio State Receives $14 Million to Study Optimal Aspirin Therapy in Pregnancy

(11/28, Ohio State University Wexner Medical Center Press Release) “A research team at The Ohio State University Wexner Medical Center and College of Medicine has been approved for a $14 million award from the Patient-Centered Outcomes Research Institute (PCORI) to study whether a higher daily dose of aspirin is more effective in decreasing the risk of dangerous blood pressure complications among some pregnant people.” Full


University of Colorado Department of Medicine Cardiologist Lands $7 Million Funding Award for Nationwide Study on Improving Heart-Failure Treatment

(11/28, University of Colorado Anschutz Medical Campus Press Release) “Larry Allen, MD, chief of the Division of Cardiology at the University of Colorado School of Medicine, will receive a funding award for a nationwide study that he hopes will lead to more heart-failure patients getting the life-saving medications they need. The $7 million in support, announced Nov. 28, is from the nonprofit Patient-Centered Outcomes Research Institute...” Full


Opioids vs. NSAIDS: Which Are Safest and Most Effective for Treating Pain Following Surgery?

(11/28, Children's Hospital Los Angeles Press Release) “...The CARES for Kids study (Comparing Analgesic Regimen Effectiveness and Safety After Surgery for Kids) will be the first prospective, randomized, controlled trial—considered the gold standard—comparing opioid and non-opioid pain medication following common surgical procedures in adolescents and young adults. The Patient-Centered Outcomes Research Institute has approved nearly $7 million of funding to support this study, which aims to generate this research-based evidence.” Full


ICER Releases Draft Evidence Report on Treatment for Schizophrenia

(11/28, ICER Press Release) “The Institute for Clinical and Economic Review today released a Draft Evidence Report assessing the comparative clinical effectiveness and value of xanomeline tartrate/trospium chloride (KarXT, Karuna Therapeutics) for the treatment of schizophrenia. This preliminary draft marks the midpoint of ICER’s eight-month process of assessing these treatments, and the findings within this document should not be interpreted to be ICER’s final conclusions.” Full


No Improvement In Mental Health Treatment Or Patient-Reported Outcomes At Medicare ACOs For Depression And Anxiety Disorders

Jason M. Hockenberry, et al.

November 2023, Health Affairs

Health Affairs


Efficacy and Safety of Brolucizumab for Diabetic Macular Edema: The KINGFISHER Randomized Clinical Trial

Rishi P. Singh, MD, et al.

November 16, 2023, JAMA Ophthalmology

JAMA Ophthalmology


Comparative Effectiveness of COVID-19 Bivalent Versus Monovalent mRNA Vaccines in the Early Stage of Bivalent Vaccination in Korea: October 2022 to January 2023

Ryu Kyung Kim, et al.

November 27, 2023, Journal of Korean Medical Science



Comparative Effectiveness of Supine-Avoidance versus Continuous Positive Airway Pressure for Treating Supine-Isolated Sleep Apnea

Matthew M Rahimi, et al.

November 28, 2023, Annals of the American Thoracic Society



Draft Evidence Report: KarXT for Schizophrenia

November 28, 2023


News from Monday, November 27, 2023


Network Meta-Analysis Validates the Use of Methotrexate in RA Compared With Other Treatments

(11/24, Kyle Munz, The American Journal of Managed Care) reports “Results from a meta-analysis reinforced the use of methotrexate as the primary reference conventional synthetic disease-modifying antirheumatic drug (csDMARD) in the treatment of rheumatoid arthritis (RA), according to a recent study published in JAMA Network Open.” Full


Novartis Urges Court to Hand It Quick Win in Drug Price Battle

(11/24, Nyah Phengsitthy, Bloomberg Law) reports “...Novartis urged the US District Court for the District of New Jersey to declare the Centers for Medicare & Medicaid Services' price-setting provisions unconstitutional and to enjoin the government from enforcing it against the company. ‘It recklessly gambles with public health and violates core tenets of our constitutional order, for no purpose other than to advance the government's preferred narrative and then shield the government from any resulting political accountability for its decisions. It must be struck down,’ the company wrote in its Nov. 22 motion.” Full


Senators Call for Investigation of Health Insurers' Role in Driving Up Drug Costs

(11/22, Joseph Walker, The Wall Street Journal) reports “...Sen. Elizabeth Warren (D., Mass.) and Sen. Mike Braun (R., Ind.) sent a letter on Wednesday to the [HHS OIG] requesting an investigation into the high drug prices and any role played by health insurers' shared ownership with the pharmacies that often fill the prescriptions. The letter cites a recent article in The Wall Street Journal that reported that big health insurers Cigna Group, CVS Health and UnitedHealth Group are paying multiples more for drugs such as cancer therapy Gleevec and multiple-sclerosis treatment Tecfidera than what manufacturers charge for generic versions.” Subscription Required


European Opportunities Loom For Value Added Medicines

(11/23, David Wallace, Pink Sheet) reports “...Conversation during the conference also highlighted the importance of the role of national payers in Europe. During a panel discussion, Zsuzsanna Petykó – a researcher at Semmelweis University’s center for health technology assessment in Hungary and a health economist at the Syreon Research Institute – suggested that this aspect was the ‘elephant in the room’ for value added medicines. She highlighted the need for the sector to move from a conversation around European-level regulation onto national-level pricing and reimbursement decisions, which panelists acknowledged were often inconsistent between different EU member states, creating uncertainty for developers.” Subscription Required

Press Releases

Largest Real-World Study Finds Tirzepatide (Mounjaro) up to Three Times More Effective than Semaglutide (Ozempic) for Weight Loss

(11/27, Truveta Press Release) “...Using Truveta Data, Truveta Research began with a patient population of over 40,000 patients with overweight or obesity initiating either semaglutide or tirzepatide between May 2022 and September 2023...The analysis found that patients taking tirzepatide were significantly more likely to achieve weight loss: Those taking tirzepatide were 1.8 times more likely than those taking semaglutide to achieve 5% weight loss, 2.6 times more likely than those taking semaglutide to achieve 10% weight loss, And three times more likely than those taking semaglutide to achieve 15% weight loss.” Full


Section 50 of the Inflation Reduction Act Drug Price Negotiation Program: Considerations for the Centers for Medicare & Medicaid Services, Manufacturers, and the Health Economics and Outcomes Research Community

John M. O’Brien, Jan Elias Hansen

December 2023, Value in Health

Value in Health


Comparative Effectiveness of Semaglutide and Tirzepatide for Weight Loss in Adults with Overweight and Obesity in the US: A Real-World Evidence Study

Patricia J Rodriguez, et al.

November 22, 2023, medRxiv



Heterogeneity of Outcomes in Randomized Controlled Trials on Implant Prosthodontic Therapy Is Hindering Comparative Effectiveness Research: Meta-Research Study

Ante Vardić, et al.

November 22, 2023, BMC Oral Health



Cost-Effectiveness Analysis of Dupilumab Versus Omalizumab, Mepolizumab and Benralizumab Added to the Standard of Care in Adults with Severe Asthma in Colombia

Abraham Ali, et al.

November 23, 2023, Expert Review of Pharmacoeconomics & Outcomes Research

Expert Review of Pharmacoeconomics & Outcomes Research


Integrating Participants as Partners in Research Governance and Operations: An Approach from the All of Us Research Program Engagement Core

Catherine Hammack-Aviran, et al.

November 27, 2023, BMJ Open

BMJ Open


Comparative Effectiveness of Etanercept Originator and Biosimilar for Treating Rheumatoid Arthritis: Implications for Cost-Savings

Claire T Deakin, et al.

November 27, 2023, Internal Medicine Journal



Ensuring Equity in Implementation of IRA Drug Price Negotiations

December 5, 2023

8:30AM - 12:30PM ET

Innovation and Value Initiative

News from Wednesday, November 22, 2023


Ophthalmology Can Set Pace for Regulatory-Grade Real-World Data

(11/22, Theodore Leng, MD, MS, Ophthalmology Times) comments “...Using traditional methods, the multicenter, double-masked, randomized, parallel group, active treatment–controlled VIEW 1/2 trials would have spent several months developing a site selection strategy, identifying eligible patient populations, engaging with clinicians, and aligning clinical trial resources in sites around the globe before the actual trial could even start. We were able to find roughly 3 times the number of eligible patients in a matter of days by comparing the VIEW 1/2 study cohorts with the real-world journeys of patients with nAMD captured in the IRIS Registry. Effectively, we were able to create a synthetic control arm that replicated the experience of the trial participants using external patient-level data.” Full


Budget Panel To CBO: Are You Updating Analysis Of IRA's Impact As New Data Emerge?

(11/21, Gabrielle Wanneh, InsideHealthPolicy) reports “...[The House Budget Committee’s] letter to CBO notes that several drug companies have already begun to announce pipeline shutdowns and canceled drug development programs for drugs under certain therapeutic areas. The lawmakers voice concern the IRA will lead to less innovative treatments being brought to market, meaning fewer available treatments for patients with rare diseases or other chronic conditions.” Subscription Required


Bernie Sanders Invites CEOs of J&J, Merck, Bristol Myers to Testify On Drug Pricing

(11/21, Lia DeGroot, Endpoints News) reports “...Senate Health Committee Chair Bernie Sanders (I-VT) and fellow Democrats on Tuesday announced that the CEOs of Johnson & Johnson, Merck and Bristol Myers Squibb have been asked to testify about why the US pays the highest prices in the world for prescription drugs.” Full


Democrats Summon CEOs of J&J, Merck and BMS for Senate Hearing On Drug Prices

(11/21, Angus Liu, Fierce Pharma) reports “...The meeting will focus on the ‘outrageously high cost of prescription drugs,’ according to a release. In their letters to the pharma CEOs, the 11 lawmakers asked why the U.S. is paying the highest drug prices in the world.” Full


Comparative Effectiveness of Biological Disease-Modifying Antirheumatic Drugs and Janus Kinase Inhibitor Monotherapy in Rheumatoid Arthritis

Akira Onishi, et al.

November 21, 2023, Rheumatology


News from Tuesday, November 21, 2023


Incorporating Information on Therapeutic Alternatives into the IRA Prescription Drug Negotiations

(11/21, Gerard F. Anderson, Richard G. Frank, and Benjamin Rome, Brookings) comments “...In some cases, there may be important differences in the effectiveness, safety, or mode of delivery of therapeutic alternatives. CMS will need to take account of these differences when developing their initial price proposals. To categorize these clinical differences, CMS could assign an ordinal rating of clinical benefit that qualitatively summarizes the totality of its review of existing evidence and any additional comparative effectiveness analyses performed as part of CMS review.” Full


How to Use PCORnet® in Your Research

(11/21, PCORI Blog) comments “...PCORnet now offers four new tools that can empower you to maximize every aspect of your research and uniquely position you to conduct patient-centered CER more efficiently. These tools will: Speed prep-to-research queries; Speed the contracting process for PCORI-funded studies; Support fast and easy search of crucial details about PCORnet-participating sites; Facilitate fast and consistent IRB applications in your PCORnet-leveraged study.” Full


England’s NICE Considers Challenges In Assessing Cost Effectiveness Of Alzheimer’s Drugs

(11/21, Neena Brizmohun, Pink Sheet) reports “...Health technology assessment institute NICE is scheduled to appraise Eisai/Biogen’s lecanemab and Eli Lilly’s donanemab next year.” Subscription Required


Hemgenix: European Countries Start Joint HTA Of Another Costly Gene Therapy

(11/21, Francesca Bruce, Pink Sheet) reports “...CSL’s Hemgenix is the latest expensive gene therapy to come under scrutiny from the BeNeLuxA cross-country coalition.” Subscription Required


Are Haemophilia Gene Therapies Truly “One and Done”?

(11/21, Irena Maragkou, Pharmaceutical Technology) reports “...[E]xperts say the most crucial limitation is that despite the steep price tags and marketing around the therapies being ‘one-time treatments,’ factor VIII expression levels after Roctavian were found to be temporary and declined over time. Regarding Hemgenix, as noted by an Institute for Clinical and Economic Review report, it is inconclusive whether the same long-term decline pattern in factor IX levels will be observed.” Full

Press Releases

‘The Flagship Infrastructure in Patient-Centered Outcomes Research’: PCORnet®’s Decade-Long Impact Lauded at PCORI Annual Meeting

(11/21, PCORnet Press Release) “...In her opening remarks, PCORI Executive Director Nakela Cook highlighted PCORnet as the flagship infrastructure for enhancing and accelerating patient-centered outcomes research, one of PCORI’s five national priorities for health. Via a partnership of clinical research networks representing major healthcare institutions across the U.S., the Network allows research teams to generate rich insights into 30 million patient encounters at more than 40 health systems. These data are deidentified to adhere to stringent patient privacy standards. ‘With PCORnet, every patient interaction becomes an opportunity to leverage data that’s collected during healthcare delivery to conduct comparative clinical effectiveness research and build a stronger evidence base,’ Cook said.” Full


ICER to Assess Treatment for Chronic Obstructive Pulmonary Disease

(11/21, ICER Press Release) “The Institute for Clinical and Economic Review announced today that it will assess the comparative clinical effectiveness and value of ensifentrine for the treatment of chronic obstructive pulmonary disease (COPD).” Full


Medicare Data Linkages for Conducting Patient-Centered Outcomes Research on Economic Outcomes

Derek S Brown, et al.

December 2023, Medical Care


News from Monday, November 20, 2023


QALY Alternatives Abound, but None Fully Addresses Discrimination Concerns

(11/17, Kimberly Westrich, LinkedIn) comments “On Tuesday, Expert Review of Pharmacoeconomics & Outcomes Research published a special report on innovative methods for value assessment...The authors concluded that 4 of the approaches (evLY, HYT, GRACE, and severity weighting) are aligned with a cost-effectiveness framework for informing Medicare price negotiation under the Inflation Reduction Act, but ‘none fully addresses all aspects of the stated concerns that QALYs may be used to unintentionally implement discrimination.’” Full


Cassidy Carries On With 340B Probe, Requests Data From Major CHCs

(11/17, Gabrielle Wanneh, InsideHealthPolicy) reports “...Senate health committee ranking Republican Bill Cassidy (LA) wants two of the largest community health center systems in the United States to provide information regarding the revenue they derive from the 340B drug discount program and how the revenue is being spent. The request is part of Cassidy's ongoing efforts to collect information from 340B covered entities to lay the foundation for potential legislation on the issue.” Subscription Required

Press Releases

NICE Gets Ready to Assess New Dementia Treatments

(11/20, NICE Press Release) “...While there are currently no licensed DMDTs in the UK, several are at varying stages of development and regulatory approval. Two of these, lecanemab, made by Eisai and Biogen and donanemab (made by Eli Lilly and Company), are scheduled to be appraised by NICE next year. Both treatments target mild cognitive impairment and mild dementia due to Alzheimer’s disease. Working with the NHS and patients, NICE have already begun work in readiness for the companies submitting their evidence.” Full


Reflections on Estimands for Patient-Reported Outcomes in Cancer Clinical Trials

Rachael Lawrance, et al.

November 19, 2023, Journal of Biopharmaceutical Statistics

Journal of Biopharmaceutical Statistics


Alternative Payment Models in Pregnancy to Improve Outcomes and Advance Equity

Jordan S. Stone, MD; Arina E. Chesnokova, MD, MPH; Sindhu K. Srinivas, MD, MSCE

November 20, 2023, JAMA



Leveraging Real-World Evidence and Observational Studies in Treating Multiple Sclerosis

Albert Aboseif, et al.

February 2024, Neurologic Clinics


News from Friday, November 17, 2023


Olaparib Does Not Improve OS vs Chemo in Recurrent Platinum-Sensitive Ovarian Cancer

(11/17, Courtney Flaherty, OncLive) reports “The use of olaparib (Lynparza) as a monotherapy or in combination with cediranib did not provide a statistically significance overall survival (OS) benefit compared with standard of care platinum-based chemotherapy in patients with recurrent, platinum-sensitive ovarian cancer, according to the final report of survival outcomes from the randomized phase 3 NRG-GY004 trial.” Full


We Need Better Data, Not Just New Drugs, To Reduce Disparities In Alzheimer’s Care

(11/17, Daniel A. Ollendorf, et al., Health Affairs Forefront) comments “...Making US data fit for equity-informed research in Alzheimer’s disease requires some relatively simple ingredients: interested researchers, engaged patients and caregivers, funding, and the political will to make changes in existing data collection vehicles. Many more diseases would also benefit from this kind of information. The time to change the data collection infrastructures was yesterday, but wouldn’t it be better to start today than to wait until tomorrow?” Full


FDA Oncology Center Signals ‘Tough’ Approach On Confirmatory Trials

(11/17, Jessica Karins, InsideHealthPolicy) reports “FDA plans to start cracking down on companies that do not complete timely confirmatory trials for products granted accelerated approvals and may start pulling drugs from the market, or denying accelerated approval to companies that do not already have a confirmatory trial underway, officials with the agency’s cancer drug center said this week ahead of a meeting on two drugs that have been delinquent in completing confirmatory trials for over nine years.” Subscription Required


Sen. Sanders Pushes NIH to Rein in Drug Prices

(11/17, Sydney Lupkin, NPR) reports “...Sanders tells NPR he plans to work with Bertagnolli, who was confirmed Nov. 7. He says something has to change at the NIH, which spends billions of dollars each year on biomedical research that lays the foundation for lucrative profits for the drug industry...‘NIH shares concerns about high drug prices and the burden they place on patients and their families,’ NIH spokesperson Renate Myles wrote in an email to NPR. ‘As stated during her Senate confirmation hearing, Dr. Bertagnolli has expressed her commitment to ensuring that the benefits of NIH-funded research are affordable and available to all the Americans.’” Full


PhRMA's Narasimhan Asks Japan To Reform Pricing Policies

(11/17, Lisa Takagi, Pink Sheet) reports “...During a recent visit to the country for the group's annual PhRMA Week meetings with government officials, chairman Vasant Narasimhan reiterated PhRMA's three main policy requests: to maintain drug prices during the patent period; to improve the price-setting policy framework to broadly assess a product's value; and to free up funding for innovation by reducing spending on non-innovative drugs and promoting the use of generics and biosimilars.” Subscription Required

Press Releases

NICE Draft Updated Guideline Recommends More Treatment Choices for Menopause Symptoms

(11/17, NICE Press Release) “New evidence shows that cognitive behavioural therapy (CBT) can help reduce menopause symptoms including hot flushes and night sweats, depressive symptoms and problems sleeping NICE has said in its draft updated guideline on menopause published today.” Full


Multiply Robust Causal Inference of the Restricted Mean Survival Time Difference

Di Shu, et al.

November 15, 2023, Statistical Methods in Medical Research


News from Thursday, November 16, 2023


Three Takeaways from CMS's Patient-Focused Listening Sessions: Toward Improved Patient Engagement

(11/16, Julie Patterson, PharmD, PhD, Tyler D. Wagner, PharmD, PhD and Jon Campbell, MS, PhD, National Pharmaceutical Council) comments “...As we move into the next phases of CMS’s evaluation process, several areas of significant and impactful uncertainty remain, including how the patient, patient advocate, and clinician input the Agency heard through the patient listening sessions will inform and influence CMS’s evaluation and price determination. It remains critical that CMS be transparent in the ways in which patient input informs and influences each stage of the evaluation process. In future sessions, we call upon CMS to continue to evolve their opportunities for patient engagement to align with evidence-based practices for obtaining purposeful and representative information.” Full


Proposals on PBMs and Medical Devices Advanced by House Subcommittee

(11/16, Noah Tong, Fierce Healthcare) reports “...‘NAACOS is encouraged that the House Energy and Commerce Health Subcommittee unanimously advanced legislation today that provides a short-term extension of Medicare’s value-based care incentives,’ said Clif Gaus, president and CEO, in a statement. ‘Eligibility to earn this critical support expires at the end of the year. We’re pleased to see lawmakers recognize the important role these incentives play in improving our health care system and providing better patient care at lower costs.’” Full


Vertex, CRISPR Gain 'Historic' Nod in UK for Exa-Cel. But Will Cost Watchdogs Embrace the Gene-Editing Therapy?

(11/16, Kevin Dunleavy, Fierce Pharma) reports “...Without revealing what it will charge for exa-cel—which has been dubbed Casgevy in Europe—Vertex said it is ‘already working closely with national health authorities to secure access for eligible patients as quickly as possible.’...Thursday’s U.K. authorization—which endorses Casgevy’s safety, quality and effectiveness—is to treat patients 12 and older.” Full

Press Releases

iConquerMS™ Patient-Powered Research Network Welcomes Multiple Sclerosis Caregivers to Learn, Guide, and Participate in Research about Those Caring for People Living with MS

(11/16, Accelerated Cure Project for Multiple Sclerosis Press Release) “...Participants will have an opportunity to contribute information through surveys, share ideas for research questions, and connect with opportunities to play an active role in shaping and conducting research focused on MS caregivers. In addition, the caregiver focused initiative provides much needed information and a circle of support to help MS caregivers.” Full


ASTRO Issues Patient-Centered Clinical Guideline on Partial Breast Irradiation for Early-Stage Invasive Breast Cancer and DCIS

(11/15, American Society for Radiation Oncology Press Release) “...For the guideline, experts in breast cancer compiled evidence—including several recently published large, randomized clinical trials—to provide guidance on which patients can benefit from partial breast radiation, as well as best practices to deliver the treatment effectively.” Full


Comparative Effectiveness of Multiple Different Treatment Regimens for Nonalcoholic Fatty Liver Disease with Type 2 Diabetes Mellitus: A Systematic Review and Bayesian Network Meta-Analysis of Randomised Controlled Trials

Manjun Deng, et al.

November 16, 2023, BMC Medicine

BMC Medicine