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 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

News from Tuesday, September 12, 2023


Beneficiaries with Cancer Spend Less Out-of-Pocket in Medicare Advantage

(9/12, Victoria Bailey, Health Payer Intelligence) reports “Medicare Advantage beneficiaries with cancer experienced lower out-of-pocket costs and had access to more benefits than those in Medicare fee-for-service (FFS), research conducted by ATI Advisory and commissioned by the Better Medicare Alliance found...Specifically, those with Medicare Advantage spent $3,996, while those with FFS spent $6,091, for a difference of $2,095.” Full


The Evolution of Precision Medicine in Pharmacy

(9/11, Rayn Oswalt, Pharmacy Times) comments “...[P]recision medicine holds immense promise in revolutionizing the field of pharmacy and transforming patient care. By leveraging patient-specific genetic, environmental, and lifestyle information, pharmacists can tailor drug selection, dosing, and treatment monitoring to optimize outcomes and minimize [adverse effects]. As technology and research progress, the future prospects for precision medicine are bright, with expanded genetic markers, increased availability of pharmacogenomic testing, and personalized treatments for rare diseases on the horizon. However, challenges related to data privacy, cost, and patient awareness must be addressed to realize the full potential of precision medicine.” Full


Japan Takes New Steps To Build On Real-World Data Foundations

(9/11, Lisa Takagi, Pink Sheet) reports “...As Japan widens its window for the use of real-world data to support drug development though revision of the Next Generation Medical Infrastructure Act, the pharma industry is hoping for more regular inclusion in regulatory filings. A dedicated group within the PMDA shares its views on how the area is developing and practical measures being taken to drive RWD adoption.” Subscription Required

Press Releases

PCORI's Board of Governors Approves 34 New Members to Multi-Stakeholder Advisory Panels

(9/12, PCORI Press Release) “...‘Members of our advisory panels bring perspectives vital to PCORI’s mission, and we eagerly anticipate the contributions that this new and distinguished set of colleagues brings to our work,’ said Harv Feldman, M.D., MSCE, PCORI’s deputy executive director for patient-centered research programs. ‘The wide range of stakeholder voices represented within these panels will greatly benefit our efforts to achieve the ambitious goal that PCORI has set forth: to fund comparative clinical effectiveness research to improve patients’ health and clinical outcomes.’” Full


Peterson Health Technology Institute Releases New Digital Health Assessment Framework

(9/12, Peterson Health Technology Institute Press Release) “...The ICER-PHTI Assessment Framework for Digital Health Technologies was developed in partnership with the Institute for Clinical and Economic Research...PHTI’s independent assessments will evaluate the performance of health technologies designed to replace or augment traditional care delivery, including digital therapeutics, chronic care management apps, remote patient monitoring, and administrative technologies, many of which include artificial intelligence. The assessment framework will prioritize products’ clinical benefits and economic impact, as well as their effects on health equity, privacy, and security.” Full


Walgreens and Pearl Health Announce Strategic Partnership to Advance Value-Based Care Delivery

(9/12, Walgreens Press Release) “...Pearl Health offers technology and insight solutions that empower clinical teams to provide holistic, personalized treatment necessary for value-based, quality-focused care. Walgreens will provide complementary services, including prescription fulfillment, medication adherence, immunizations, care gap closure and diagnostic testing. Walgreens will also work with providers to help patients discharged from the hospital transition to home.” Full


Improving Food and Drug Administration–Centers for Medicare and Medicaid Services Coordination for Drugs Granted Accelerated Approval

Peter J. Neumann, Elliott Crummer, James D. Chambers, Sean R. Tunis

August 31, 2023, The Milbank Quarterly

The Milbank Quarterly


A Centralized EHR-Based Model for the Recruitment of Rural and Lower Socioeconomic Participants in Pragmatic Trials: A Secondary Analysis of the Diuretic Comparison Project

Cynthia Hau, et al.

September 5, 2023, JAMA Network Open



Use of SGLT2i and ARNi in Patients with Atrial Fibrillation and Heart Failure in 2021-2022: An Analysis of Real-World Data

Alvaro Alonso, et al.

September 10, 2023, medRxiv



The Stiff Joint: Comparative Evaluation of Monotherapy and Combination Therapy With Urate Lowering Agents in Managing Acute Gout

Okelue E. Okobi, et al.

September 12, 2023, Cureus



Real-World Data: Applications and Relevance to Cancer Clinical Trials

Andrew J Gross, et al.

October 2023, Seminars in Radiation Oncology



Medicare Advantage Has Unique Ability to Meet Needs of Cancer Patients and Survivors

September 7, 2023

Better Medicare Alliance

News from Monday, September 11, 2023


CMS Clarifies Factors It Will Consider In Negotiating Drug Prices

(9/8, Gabrielle Wanneh, InsideHealthPolicy) reports “...Relating to evidence available on therapeutic alternatives to the selected drugs, CMS will also consider: The extent to which the selected drug represents a therapeutic advance compared to existing therapeutic alternatives; The costs of the therapeutic alternatives; The FDA-approved prescribing information available for selected drugs and their therapeutic alternatives; The comparative effectiveness of selected drugs, including the impact for specific populations, such as individuals with disabilities, the elderly, the terminally ill, children, and other populations.” Subscription Required


Pharmaceutical Innovativeness Is Highly Variable, As CMS Will Soon Find Out When It Assesses Drugs It Selected For Negotiation

(9/9, Joshua Cohen, Forbes.com) comments “...The key question when conducting comparative effectiveness evaluations is whether the pharmaceuticals being analyzed are clinically better than existing standards of care. And if so, then HTA must probe whether the incremental benefit is worth the price premium? As was said above, among the top-selling prescription drugs sold to Medicare most were not considered high value products.” Full


Big Pharma's Battle With the Biden Administration Could Have Legs

(9/9, David Wainer, The Wall Street Journal) reports “...[Carmel Shachar, assistant clinical professor at Harvard Law,] ultimately thinks the law has a good chance of surviving, but she notes that in an era where courts have shown growing skepticism toward administrative agency action anything is possible. She says the industry argument that the government is unfairly taking their property, which is protected by patents, will be looked at seriously by some courts.” Subscription Required


Real-World Data Can Include EUA Observations, US FDA Final Guidance Says

(9/10, Bridget Silverman, Pink Sheet) reports “...Agency clarifies expectations for non-interventional studies providing RWD to support regulatory decisions while continuing to emphasis familiar themes around early interaction and stringent data practices...The considerations guidance focuses on non-interventional clinical study designs, which are not subject to IND regulations; other RWE-using trial designs, like external controls, have been highlighted in their own guidance documents. "There weren't huge changes" between the draft and final versions, Aetion head of scientific strategy Ulka Campbell observed in an interview with the Pink Sheet.” Subscription Required


Comparative Effectiveness of Alternative Treatment Approaches to Secondary Hyperparathyroidism in Patients Receiving Maintenance Hemodialysis: An Observational Trial Emulation

Alyssa Platt, et al.

September 5, 2023, American Journal of Kidney Diseases



Estimated Savings from Using Added Therapeutic Benefit and Therapeutic Reference Pricing in United States Medicare Drug Price Negotiations

Michael J DiStefano, et al.

September 7, 2023, Value in Health



Treatment Patterns and Clinical Outcomes in Patients with Rheumatoid Arthritis Initiating Etanercept, Adalimumab, or Janus Kinase Inhibitor as First-Line Therapy: Results from the Real-World CorEvitas RA Registry

Dimitrios A Pappas, et al.

September 9, 2023, Arthritis Research & Therapy


News from Friday, September 8, 2023


Uncertainties Still Loom Over IRA Negotiations

(9/7, Angela Maas, AIS Health) reports “...‘The decisions around the key question of which therapeutic alternatives will be selected as comparators, how clinical superiority and clinical net benefits are demonstrated and how CMS plans to assign a price value to that difference in this first year will come too late for the manufacturers selected today and might come too late for the manufacturers selected for the next year of negotiation as well,’ [NPC president and CEO John O’Brien, Pharm.D.] maintains. ‘Manufacturers currently face uncertainty about what evidence should be developed and submitted, whether decisions explained in the concise justifications during the first year of negotiation under the program will be the same decisions made in future years and whether the people making the decisions next year will be the same. How this list was put together is a great question for the White House and HHS,’ he tells AIS Health. ‘The many questions people have about today’s announcement are a reminder of the lack of transparency and tremendous uncertainty surrounding the process.’” Full


Biden's Price Control Scheme Harms Patients, Compromises Free Market

(9/7, Tom Price, RealClearHealth) comments “...To preserve the pipeline of new innovative treatments, therapies, and vaccines, drug companies must have the opportunity to recoup investment rather than being forced to sell products at below market rates. Unlike Uncle Sam, businesses don't have the option to continuously spend beyond their means and take on trillions of dollars in debt. The Biden administration doesn't seem to grasp this concept. The White House's price control scheme-approved by his allies in Congress-is moving forward at full steam ahead. As a result, the free-market incentive to create new medicine will fade-compromising the health of future Americans.” Full


The Reality of Drug Pricing Negotiations Isn't a Slam Dunk for Patients

(9/7, Kenneth E. Thorpe, RealClearHealth) comments “ ...Congressional Budget Office prepared an estimate projecting changes in utilization rates for a similar drug price negotiation program that preceded the IRA. To achieve expected savings under the program, the score estimated reduced utilization for some medications of more than 25 percent. This disruption to access has no medical justification. Price negotiations will also have a chilling effect on drug research - and thereby diminish the number of new medicines available to patients in the years and decades to come. One recent study from Vital Transformation estimates that over the next decade, the IRA's drug price negotiations will result in 139 fewer medicines being developed.” Full


Video: Jeffrey Sparks, MD, MMSc: Treatment Strategies for Rheumatoid Arthritis

(9/7, HCP Live) “...[T]he number of choices has also made it more challenging to select the most suitable therapy. Additionally, most of the trials leading to drug approvals have compared the new drugs with placebos, with a limited number of head-to-head trials. In his presentation, he discusses these head-to-head trials to provide evidence of efficacy. Further, he explores real-world evidence, examining comparative effectiveness studies that assess different drug classes against each other.” View Video


NAACOS: Medicare Payment Incentives Favor Clinicians in Fee-For-Service

(9/8, Victoria Bailey, RevCycle Intelligence) reports “...According to the NAACOS blog post, MACRA’s incentives for Payment Year 2026 and Performance Year 2024 will favor clinicians who do not participate in advanced APMs and remain in the Merit-Based Incentive Payment System (MIPS). In 2026, MIPS clinicians will receive a 0.25 percent conversion factor update and can receive additional positive payment adjustments in MIPS. The average MIPS adjustment is typically 3 percent, meaning the total potential payment adjustment would be 3.25 percent. Meanwhile, clinicians in advanced APMs will only receive a 0.75 percent conversion factor update. Incentives will not favor clinicians in advanced APMs again until 2032.” Full


Lilly's Diabetes Drug Mounjaro Approved by UK Watchdog

(9/8, Reuters) reports “Eli Lilly's diabetes drug Mounjaro has gained the backing of Britain's healthcare cost-effectiveness watchdog, which said it would be a good option for patients with poorly controlled type 2 diabetes. The National Institute for Health and Care Excellence said in draft final guidance that it estimates some 180,000 people could benefit from the new treatment.” Full

Press Releases

Medical Care Publishes Article Collection on Integrating Evidence-Based Programs into Clinical Practice

(9/8, Wolters Kluwer Health Press Release) “As part of its partnership with the Patient-Centered Outcomes Research Institute, Medical Care has published its first PCORI-sponsored article collection, which provides specific information about the costs that healthcare systems can expect to incur in promoting the uptake of specific evidence-based programs. Medical Care, the official journal of the Medical Care Section of the American Public Health Association, is published in the Lippincott portfolio by Wolters Kluwer.” Full


Comparative Effectiveness and Duration of Protection of ChAdOx1, CoronaVac, BNT162b2, mRNA-1273, and Ad26.COV2.S COVID-19 Vaccines for Symptomatic and Hospitalized Mu, Delta, and Omicron: A Test-Negative Case-Control Study

Angel Paternina-Caicedo, et al.

September 5, 2023, Vaccine



The Price of Practice Change: Assessing the Cost of Integrating Research Findings Into Clinical Practice

Lehman, Valerie E.R.; Siegel, Joanna E.; Chiang, Ethan N.

October 2023, Medical Care

Medical Care


A Cost Analysis of Rethink the Strip: De-implementing a Low-value Practice in Primary Care

Spees, Lisa P.; Young, Laura A.; Rees, Jennifer; et al.

October 2023, Medical Care

Medical Care


Webinar: Updates to ICER’s Value Assessment Framework

September 28, 2023

11:00AM ET



Advancing Precision Oncology Research through Real-World Evidence (RWE): Current Landscape and Future Challenges in the European Context

October 17, 2023