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 Monday, March 18, 2024

Articles

Value Viewpoint: March 16, 2024

(3/16, Kimberly Westrich, LinkedIn) comments “In a new Value in Health article, Joshua T. Cohen made a strong case for replacing the three percent discount rate typically used in health technology assessments with a rate ranging from 1.5 to just over 2 percent. Why does this matter in practice? As Cohen explains: ‘Using a lower discount rate will improve projected cost-effectiveness for therapies with long-term (e.g., lifetime) benefits and increase the impact of accounting for long-term drug price dynamics, including reduced prices attending loss of market exclusivity.’” Full

 

New Study Reveals Key Differences in Parkinson’s Disease Progression Using Real-World Data

(3/18, Tarun Sai Lomte, News Medical) reports “...The study analyzed PD progression across real-world and research populations and illustrated systematic differences and directional biases between them. Research populations were diagnosed and started on levodopa and other medicines much earlier, with slower changes in clinical scales. By contrast, PD diagnosis and treatment initiation were much later in real-world populations; they also had accelerated changes in clinical scales. Overall, the findings offer detailed insights into PD progression in different populations.” Full

 

The White House Wants to Double Dip on Drug Rebates

(3/15, Tomas Philipson, RealClearHealth) comments "...The White House premised its march-in proposal on the idea that patients and insurers overpay for medicines that benefited from basic research investments. But the government already enjoys the best of both worlds -- robust economic growth from innovation, tax revenues from those innovations, a healthier populace, and preferential regulations that give it discounted prices." Full

 

Australia To Give Consumers More Formal Role In HTA Processes

(3/18, Ian Schofield, Pink Sheet) reports “...The health department says consumers and patients should be able to initiate health technology assessments because many potentially helpful and lifesaving medicines might never be assessed for funding due to ‘a lack of commercial incentive for pharmaceutical companies.’” Subscription Required

Press Releases

The National Eczema Association Approved for $250,000 Engagement Award from PCORI

(3/18, National Eczema Association Press Release) “...Working with academic institutions, researchers and patient communities, NEA will recruit a diverse group of eczema stakeholders to form six regional research hubs that will help identify priority topics for future patient-centered PCOR and comparative clinical effectiveness research (CER) on eczema. NEA will develop regional engagement frameworks highlighting patient partnerships to guide future eczema PCOR and CER applicable to a diverse patient population.” Full

Journals

It’s Time to Reconsider the Three Percent Discount Rate

Joshua T. Cohen, PhD.

March 8, 2024, Value in Health

Value in Health

 

Comparative Effectiveness of Bimekizumab and Guselkumab in Patients with Psoriatic Arthritis at 52 Weeks Assessed Using a Matching-Adjusted Indirect Comparison

Richard B Warren, et al.

March 15, 2024, Rheumatology and Therapy

PubMed

 

Studying How Patient Engagement Influences Research: A Mixed Methods Study

Deborah A. Marshall, et al.

March 15, 2024, The Patient - Patient-Centered Outcomes Research

Springer

News from Friday, March 15, 2024

Articles

Reducing Low-Value Care Is Hard, but What About Just Not Paying for It?

(3/14, Christina Mattina, The American Journal of Managed Care) comments “...A new approach is to implement performance guarantees in the contracts between carriers and payers that specify payers will not pay for LVC. There has been pushback from carriers on this idea and skepticism from those who say the potential savings would be too small, but ‘if we throw out all the ideas where you’re only going to save half a percent or a quarter of a percent, you’re going to have no ideas,’ [Michael E. Chernew, PhD] said. He also mentioned that these performance guarantees could be titrated to address concerns about imperfection in measurement.” Full

 

Drug Price Shenanigans

(3/15, Grace-Marie Turner, RealClearHealth) comments “...Former White House Economist Tomas Philipson estimates President Biden’s price control scheme will mean 135 fewer drugs over the life cycle of drug development, resulting in 330 million lost life years in the U.S. from patients who could have been treated or cured from drugs that were never developed. Investors won’t fund research if there is little chance the company can receive a return. And where does the money the government saves from getting cheaper drugs go? To funding electric cars and other green projects.” Full

 

Former U.S. Patent Head On Why Federal March-In Rights for Drugs Would Be a 'Devastating' Mistake

(3/14, Michael Gibney, PharmaVoice) reports “...[Andrei Iancu, co-chair of the Council for Innovation Promotion:] Here’s the reality: If the government threatens to take away these patents, the consumers will have zero access to that technology if the industry doesn’t bring it to market. The price of no technology on the market is infinite, and there is no amount of money you can give to buy a product that doesn’t exist.” Full

 

Vertex Resolves To Reverse English Funding Rejection For CRISPR Sickle Cell Gene Therapy

(3/15, Neena Brizmohun, Pink Sheet) reports “...The health technology assessment institute, NICE, is not yet ready to recommend Casgevy for sickle cell disease and says it wants more data. Meanwhile, an access agreement relating to the treatment’s use for transfusion-dependent β-thalassemia is making progress in England, as are reimbursement talks for SCD in other European countries.” Subscription Required

Journals

Specialty Drug Use for Autoimmune Conditions Varies by Race and Wage Among Employees with Employer-Sponsored Health Insurance

Bruce W Sherman, Rochelle Henderson, Leah Kamin, Sharon Phares

March 14, 2024, Journal of Managed Care & Specialty Pharmacy

JMCP

 

Comparative Effectiveness and Safety of Prasugrel and Ticagrelor in Patients of Acute Coronary Syndrome Undergoing Percutaneous Transluminal Coronary Angioplasty: A Propensity Score-Matched Analysis

Avik Ray, et al.

March 12, 2024, Indian Heart Journal

PubMed

 

Principles for Stakeholder Engagement in Observational Health Research

Tainayah W. Thomas, PhD, MPH; Stephanie A. Hooker, PhD, MPH; Julie A. Schmittdiel, PhD

March 15, 2024, JAMA Health Forum

JAMA Health Forum

 

Accountable Care Organization Leader Perspectives on the Medicare Shared Savings Program: A Qualitative Study

Dhruv Khullar, MD, MPP, et al.

March 15, 2024, JAMA Health Forum

JAMA Health Forum

News from Thursday, March 14, 2024

Articles

Evolution of HDHPs With VBID Components Can Improve Care for Chronic Disease

(3/14, Laura Joszt, MA, The American Journal of Managed Care) reports “...The theme of the University of Michigan’s Center for Value-Based Insurance Design (V-BID)’s 2024 V-BID Summit was ‘Overcoming Financial Toxicity,’ starting with a discussion around the successes and challenges of HDHPs based on research from the past 20 years and the evolutions to the products...The high-level takeaway was that ‘very high out-of-pocket [OOP] cost obligations were sort of a blunt instrument [that reduced] all types of care whether they were needed and high value or less needed and low value,’ [Frank Wharam, MD, MPH] explained.” Full

 

Bringing Real World Data to Clinical Studies: Q&A With Dr. David Cosgrove of Compass Oncology

(3/14, Mike Hollan, Pharmaceutical Executive) reports “Dr. David Cosgrove, a medical oncologist with Compass Oncology, presented the results of a study on the first-line use of atezolizumab plus bevacizumab on patients with a certain type of liver cancer at ASCO GI earlier this year. He recently spoke with Pharmaceutical Executive about some of the methods used in the study to bring real-world data into the fold.” Full

 

Update On The Medicare Value-Based Care Strategy: Alignment, Growth, Equity

(3/14, Douglas Jacobs, et al., Health Affairs Forefront) comments “...CMS recently finalized a rule that requires certain health insurers to make specified data readily available to providers in standardized application programming interfaces. This standardization complements existing Medicare Part A, B, and D monthly claims data sharing that CMS provides to ACOs and will help practitioners and ACOs understand the full medical claims history of their patients. We are also focused on sending ACOs more data on specialist performance, consistent with the Innovation Center’s Specialty Strategy. We believe access to this claims data will help lower barriers to participation in value-based arrangements...” Full

 

Passing the Baton – The Future of HSR is in Good Hands, but Much Work Remains

(3/14, Lisa Simpson, M.B., B.Ch., M.P.H., FAAP, AcademyHealth Blog) comments “...We must move the incentives toward social impact – and the biggest incentive is money. Federal research funders, including the National Institutes of Health (NIH), the Patient Centered Outcomes Research Institute, the Agency for Healthcare Research and Quality, and the Veterans Administration Health Services Research Program have a critical leadership role to play, and can affect significant change through their funding practices. Each has launched initiatives to drive implementation and impact, yet the proportion of total funding on these is small compared to total research funding. This must change.” Full

 

NICE Not Sold on Vertex, CRISPR's Groundbreaking Sickle Cell Gene Therapy Casgevy Without More Data

(3/14, Zoey Becker, Fierce Pharma) reports “Ever since Vertex and CRISPR therapeutics scored a groundbreaking U.K. authorization for their sickle cell gene therapy Casgevy in November, the looming hurdle to commercialization was securing a recommendation from the region’s cost watchdog...In its draft guidance, NICE did not recommend Casgevy but is ‘exploring further data collection’ on the therapy’s effectiveness and a potential commercial arrangement, the regulator said in an emailed press release.” Full

 

German HTA: Clinical Cancer Registries Could Hold Key To Better Comparative Data For Benefit Assessments

(3/14, Francesca Bruce, Pink Sheet) reports “...Better data linkage and data entry systems could transform Germany’s clinical cancer registries into a reliable data source for health technology assessments.” Subscription Required

Press Releases

Institute for Clinical and Economic Review Publishes Final Evidence Report on Treatments for Paroxysmal Nocturnal Hemoglobinuria

(3/13, ICER Press Release) “The Institute for Clinical and Economic Review today released a Final Evidence Report assessing the comparative clinical effectiveness and value of iptacopan (Novartis) and danicopan (Alexion Pharmaceuticals) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). ICER’s report on these therapies was the subject of the February 2024 public meeting of the of the CTAF, one of ICER’s three independent evidence appraisal committees.” Full

Journals

Real-World Effectiveness of Ustekinumab and Vedolizumab in TNF-Exposed Pediatric Patients with Ulcerative Colitis

Perseus V Patel, et al.

March 14, 2024, Journal of Pediatric Gastroenterology and Nutrition

PubMed

Reports

Iptacopan and Danicopan for Paroxysmal Nocturnal Hemoglobinuria

March 13, 2024

ICER

News from Wednesday, March 13, 2024

Articles

Review Finds JAK Inhibitors More Effective for Moderate-to-Severe AA Compared With Established Therapies

(3/13, Cameron Santoro, The American Journal of Managed Care) reports “Patients with moderate-to-severe alopecia areata (AA) identified baricitinib and other oral Janus kinase (JAK) inhibitors as effective treatment routes compared with systemic corticosteroids, topical immunotherapy, cyclosporine A, methotrexate, and azathioprine.” Full

 

Guselkumab Outperforms Ustekinumab in Indirect Comparison PsA Analysis

(3/12, Lana Pine, HCP Live) reports “Individual patient-level data (IPD) analysis showed patients with psoriatic arthritis (PsA) receiving treatment with guselkumab were more likely to achieve American College of Rheumatology 20% improvement (ACR20) when compared with those treated with ustekinumab, according to a study published in Rheumatology and Therapy. These results were superior from week 16 in biologic-naïve patients and week 24 in biologic-experience patients.” Full

 

Unlock the Power of Real World Data (RWD) to Drive Innovation in the Healthcare Industry

(3/13, Jamia Moss, MD, IQVIA Blog) comments “...After real-world data is curated, harmonized, and made analytically-ready, it can be leveraged. This includes planning for real-world evidence strategy, adopting appropriate clinical trial designs and comparators, and performing thorough post-marketing evaluations. Pharma companies and regulatory bodies are investing in building internal knowledge and adopting RWE-based strategies to support effective and safe product launches, new development needs, and drug use assessment in real-world settings. This RWD guide showcases examples of how RWE is leveraged throughout the product lifecycle, from preclinical to post-approval uses for RWD.” Full

 

Insurer Prior-Authorization Delays Put Lives at Risk

(3/12, Lucie Turcotte, Minneapolis Star-Tribune) comments “...Critical services, including cancer treatments aligned with national oncological guidelines, medications for substance use disorder treatment, generic drugs deemed therapeutically equivalent by the FDA, preventive treatments, pediatric hospice services and neonatal abstinence programs, should not be subject to unnecessary bureaucratic hurdles. Private and public insurers must be held accountable for the disparities they perpetuate and the risks they impose on vulnerable patients when they overuse prior authorization processes.” Full

Journals

Comparative Effectiveness of Baricitinib and Alternative Biological DMARDs in a Swiss Cohort Study of Patients with RA

Benoit Thomas P Gilbert, et al.

March 12, 2024, BMJ Open

BMJ Open

 

Assessing the Properties of Patient-Specific Treatment Effect Estimates from Causal Forest Algorithms Under Essential Heterogeneity

John M. Brooks, et al.

March 13, 2024, BMC Medical Research Methodology

BMC Medical Research Methodology

Reports

White Paper: The Cost of the 340B Program Part 1: Self-Insured Employers

March 12, 2024

IQVIA

News from Tuesday, March 12, 2024

Articles

Other IRA Cases Could Be Influenced by New Jersey Judge's Decisions, Expert Says

(3/11, Nicole DeFeudis, Endpoints News) reports “A New Jersey federal judge's skepticism of drugmakers' arguments against the Inflation Reduction Act's drug price negotiations could become influential in other courts as pharma's challenges to the law continue...‘You see judges in different districts looking at decisions of other judges and either explaining why they agree with that analysis, or why they don't,’ Zachary Baron, director of the Health Policy and the Law Initiative at Georgetown University's O'Neill Institute, told Endpoints News on Monday.” Full

 

EU’s DARWIN Project To Expand Geographical Reach With 10 New Data Partners

(3/12, Eliza Slawther, Pink Sheet) reports “...The EU DARWIN initiative is expected to deliver more than 70 real-world data studies this year and twice this figure from 2025 onwards to enhance European decisions on medicines.” Subscription Required

Press Releases

Institute for Clinical and Economic Review Publishes Final Evidence Report on Treatment for Schizophrenia

(3/11, ICER Press Release) “The Institute for Clinical and Economic Review today released a Final Evidence Report assessing the comparative clinical effectiveness and value of xanomeline tartrate/trospium chloride (KarXT, Karuna Therapeutics) for the treatment of schizophrenia. ICER’s report on these therapies was the subject of the February 2024 public meeting of the of the New England CEPAC, one of ICER’s three independent evidence appraisal committees.” Full

 

Institute for Clinical and Economic Review Names Henry Claypool to Governance Board

(3/12, ICER Press Release) “...Henry Claypool has 30+ years of experience developing and implementing disability policy at the federal, state, and local level. His career in public service includes positions as the Director of the Health and Human Services Office on Disability and the founding Principal Deputy Administrator of the Administration for Community Living. He also served as a presidentially-appointed member of the Federal Commission on Long-Term Care, advising Congress on how long-term care can be better provided and financed for the nation’s older adults and people with disabilities, now and in the future.” Full

Journals

Editorial: The Limitations of EQ-5D as a Clinical Outcome Assessment Tool

Ari Gnanasakthy & Carla Romano DeMuro

March 11, 2024, The Patient - Patient-Centered Outcomes Research

Springer

 

One-Year Comparative Effectiveness of Upadacitinib Versus Tofacitinib For Ulcerative Colitis: A Multicenter Cohort Study

Rahul S Dalal, et al.

March 12, 2024, The American Journal of Gastroenterology

PubMed

 

Association Between Expedited Review Designations and the US or Global Burden of Disease for Drugs Approved by the US Food and Drug Administration, 2010–2019: A Cross-Sectional Analysis

Matthew J Jackson, Gregory Vaughan, Fred D Ledley

March 12, 2024, BMJ Open

BMJ Open

News from Monday, March 11, 2024

Articles

Value Viewpoint: 3-8-24

(3/8, Kimberly Westrich, LinkedIn) comments “...Last week, Specialty Pharmacy Continuum published an article highlighting my former colleague Jane Ha’s AMCP platinum award-winning research on ICER’s ‘Unsupported Price Increase’ report. The research found that ICER rejects 97% of submitted evidence, a troubling finding against the backdrop of ‘significant critique and concern from stakeholders regarding restrictive criteria and lack of transparency.’ As I wrote in December on this topic, NPC continues to be concerned that the UPI report uses flawed methods and lacks balance, which is especially troubling as ICER markets this report for use by payers and policymakers.” Full

 

Biden's $7.3 Trillion Budget Sets Spending, Tax Fight With Trump

(3/11, Justin Sink and Erik Wasson, Bloomberg) reports “...While the administration is currently bargaining with drugmakers to reduce the cost of ten popular drugs for seniors on Medicare, Biden envisions expanding that program over the next decade. The administration projects it would save $200 billion in taxpayer money that would otherwise go to drug companies. Drug companies take another hit in Biden's budget, which would raise taxes on multinational corporations to 21%.” Subscription Required

 

Aligning Accountable Care Models With The Goal Of Improving Population Health

(3/11, Elliott S. Fisher, Andrew B. Bindman and Michael Kopko, Health Affairs Forefront) comments “...If we want to improve population health and lower costs, we must complete the transition to payment models that provide much stronger incentives to improve health, coordinate care, improve efficiency, and avoid unneeded care and preventable complications. Capitation, a prospective monthly per-patient payment to the practice or health care delivery organization for the total cost of care, is the most effective way to do this.” Full

 

Bluebird Bio Inks First Medicaid Coverage Agreement for Sickle Cell Gene Therapy, Signing on with Michigan

(3/11, Kevin Dunleavy, Fierce Pharma) reports “Bluebird bio has secured its first Medicaid outcomes-based agreement for its sickle cell disease gene therapy Lyfgenia (lovo-cel), signing on with the state of Michigan...The outcomes-based framework offered by bluebird tracks treatment results for three years and provides rebates if a patient is hospitalized because of a painful episode of vaso-occlusion.” Full

 

UK: Ipsen’s Cabometyx and BMS’ Opdivo Recommended by NICE for Advanced Kidney Cancer

(3/11, Emily Kimber, PMLiVE) reports “...The agency has specifically recommended the combination as a first-line therapy for adults with untreated advanced renal cell carcinoma (RCC) whose disease is intermediate or poor risk and if Opdivo plus BMS’ Yervoy (ipilimumab) or Eisai’s Lenvima (lenvatinib) plus Merck & Co’s Keytruda (pembrolizumab) would otherwise be offered.” Full

Press Releases

The Light Treatment Effectiveness (LITE) Study Reveals Groundbreaking Data on the Effectiveness of Phototherapy

(3/9, National Psoriasis Foundation Press Release) “Groundbreaking data presented today at the American Academy of Dermatology Annual Meeting reveal new findings on the effectiveness of home phototherapy. Results from the Light Treatment Effectiveness (LITE) study, the largest study of its kind, demonstrate that home phototherapy is non-inferior to office phototherapy, with both home and office phototherapy demonstrating excellent effectiveness and safety in real world settings. The findings support the use of home phototherapy as a first line treatment option for psoriasis, including individuals with no prior phototherapy experience, and advocates for efforts to make phototherapy more available to people living with psoriasis.” Full

 

25,000 People to Benefit after NICE Recommends New Ulcerative Colitis Treatment

(3/11, NICE Press Release) “Thousands of people could be set to benefit after NICE today (Monday, 11 March) recommended a new one-a-day pill as an option for treating severe ulcerative colitis. NICE’s final guidance on etrasimod (also known as Velsipity and made by Pfizer) is being published on the same day that the treatment was granted a licence by the Medicines and Healthcare Products Regulatory Agency (MHRA).” Full

Journals

Perceived Barriers Toward Patient-Reported Outcome Implementation in Cancer Care: An International Scoping Survey

Lawson Eng, MD, SM, FRCPC, et al.

March 8, 2024, JCO Oncology Practice

JCO Oncology Practice

News from Friday, March 8, 2024

Articles

Biden's Pharma Plans Keep Industry Fighting On All Fronts

(3/7, Sarah Karlin-Smith, Pink Sheet) reports “...Drug manufacturers still have unanswered questions about the current IRA implementation such as what evidence [CMS] is considering when determining a price for a drug, said John O’Brien, CEO of the National Pharmaceutical Council. ‘If I’m a manufacturer, I won’t know what evidence CMS valued until it releases what they call a “concise justification” early next year – after manufacturers of the next batch of drugs have already submitted their evidence before the next round’s deadline,’ O'Brien said. ‘Drug development is risky business, and layering more uncertainty on top of the uncertainty caused by the IRA will make it even harder.’” Subscription Required

 

PhRMA Seeks to Revive Medicare Price Negotiations Lawsuit, Blasts Biden's Request for IRA Expansion

(3/8, Fraiser Kansteiner, Fierce Pharma) reports “...The trade group cited bipartisan support for fixing ‘some of the flaws of the IRA,’ but argued that the government is nevertheless ‘rushing to make this bad law worse.’ ‘Letting government bureaucrats arbitrarily set the price of medicines allows politics to dictate which cures are worth investing in and who should get access to them,’ PhRMA said in a statement late Thursday. ‘It puts the government between patients and their doctors.’” Full

 

Value-Based-Care: Making Primary Care More Cost-Effective

(3/8, Web Golinkin, Forbes.com) comments “...Because VBC models incentivize providers to reduce costs while also improving quality, it is clear that the system should continue to move in this direction and away from the FFS model, whose misaligned incentives have resulted in a healthcare system that is unsustainable in its current form while providing sub-optimal care for many of the patients it is intended to serve. VBC is on the move, and justifiably so!” Full

 

European Commission Proposes Framework for Joint Clinical Assessments

(3/7, Ferdous Al-Faruque, Regulatory Focus) reports “The European Commission has proposed a framework for joint clinical assessments as part of its effort to implement the EU health technology assessment regulation. The document outlines how to determine the scope of joint clinical assessments, how to share information between the relevant entities, how to select experts to help make joint clinical assessments, and more.” Full

Press Releases

Institute for Clinical and Economic Review to Assess Treatments for Transthyretin Amyloidosis Cardiomyopathy

(3/8, ICER Press Release) “The Institute for Clinical and Economic Review announced today that it will assess the comparative clinical effectiveness and value of acoramidis (BridgeBio Pharma, Inc), tafamidis (Pfizer Inc.), and vutrisiran (Alnylam Pharmaceuticals, Inc.) for the treatment of transthyretin amyloidosis cardiomyopathy (ATTR-CM). The assessment will be publicly discussed during a meeting of the Midwest CEPAC (Midwest CEPAC) in September 2024, where the independent evidence review panel will deliberate and vote on evidence presented in ICER’s report.” Full

Journals

Delivering on the Promise of Accountable and Value-Based Care

Mohamed Diab

March 2024, The American Journal of Accountable Care

AJMC

 

Comparing Ocrelizumab to Interferon/Glatiramer Acetate in People with Multiple Sclerosis Over Age 60

Yi Chao Foong, et al.

March 7, 2024, Journal of Neurology, Neurosurgery and Psychiatry

PubMed

News from Thursday, March 7, 2024

Articles

Drugmakers Gear Up for Joint IRA Arguments as PhRMA Appeals Texas Verdict

(3/6, Nicole DeFeudis, Endpoints News) reports “...[O]n Wednesday, experts said the drugmakers will likely focus on one argument in particular: that Medicare negotiations violate the Fifth Amendment’s takings clause. The clause states that private property shouldn’t be taken without just compensation. While it’s often used in the context of land taken by eminent domain, the companies say it also applies to their prescription drugs...[University of California College of the Law, San Francisco professor Robin Feldman] told Endpoints News via email on Wednesday that the takings arguments would be a ‘knockout blow’ to the IRA.” Subscription Required

 

Biden Proposes Strengthening Medicare’s Drug Pricing Power

(3/7, Kristin Jensen, BioPharma Dive) reports “...In a fact sheet released Wednesday, the White House said Medicare should be allowed to negotiate prices for 50 medications every year...While raising that figure to 50 would undoubtedly hurt pharmaceutical profits, analysts are skeptical that Biden’s proposals will become reality under what’s likely to be a divided government in Washington.” Full

 

Republicans Consider a New Attack On Biden 'March-In' Plan for Lower Drug Prices

(3/6, John Wilkerson, STAT Plus) reports “...Republicans in Congress might try to use a technical maneuver to block the Biden administration from using so-called march-in rights to seize pharmaceutical patents and lower drug prices...Sen. Bill Cassidy (R-La.) is trying to change that. He asked the Government Accountability Office to designate the draft framework as a formal regulation. If GAO determines that the proposal is indeed a rule, opponents could then challenge it under the Congressional Review Act.” Subscription Required

Press Releases

Boston Medical Center Researchers Receive $5 Million Grant to Evaluate Interventions That Target Disparities in Healthcare

(3/6, Boston Medical Center Press Release) “Boston Medical Center researchers have established Boston Health Equity & community-Aligned Learning Health System (Boston HEALHS), a new program that will support comparative effectiveness research to accelerate enhanced health equity, patient care, and community engagement. Boston HEALHS will be supported by a $5 million grant from the federal Agency for Healthcare Research and Quality and the Patient-Centered Outcomes Research Institute.” Full

 

Columvi® (Glofitamab for Injection) Receives Positive CADTH and INESSS Recommendations for Adult Patients with Relapsed or Refractory Diffuse Large B-Cell Lymphoma (DLBCL)

(3/7, Roche Canada Press Release) “Hoffmann-La Roche Limited (Roche Canada) is pleased to announce that on March 5, 2024, the Institut National d'Excellence en Santé et en Services Sociaux (INESSS) recommended Columvi® (glofitamab for injection) for public reimbursement. This recommendation follows the recent positive recommendation from the Canadian Agency for Drugs and Technologies in Health (CADTH) Canadian Drug Expert Committee (CDEC) issued on February 21, 2024.” Full

Journals

Comparative Effectiveness Research Trial for Antidepressant Incomplete and Non-Responders with Treatment Resistant Depression (ASCERTAIN-TRD) a Randomized Clinical Trial

George I. Papakostas, et al.

March 7, 2024, Molecular Psychiatry

PubMed

 

Editorial: Overly Complex Methods May Impair Pragmatic Use of Core Evidence-Based Medicine Principles

Rebecca Kuehn, Ying Wang, Gordon Guyatt

March 7, 2024, BMJ Evidence-Based Medicine

BMJ Evidence-Based Medicine

 

Comparison of Prior Authorization Across Insurers: Cross Sectional Evidence from Medicare Advantage

Ravi Gupta, et al.

March 7, 2024, BMJ

BMJ

 

Comparative Effectiveness of Natalizumab, Fingolimod, and Injectable Therapies in Pediatric-Onset Multiple Sclerosis: A Registry-Based Study

Tim Spelman, et al.

April 9, 2024, Neurology

PubMed

News from Wednesday, March 6, 2024

Articles

Drug Price Negotiations Could Discourage Additional Approvals: Study

(3/6, Adrianna Nine, BioSpace) reports “...Experts at the National Pharmaceutical Council...investigated 50 drugs with the highest gross spending by Medicare Part D in 2020 and found that when additional clinical trials or real-world evidence was needed, subsequent approvals took an average of 7.5 years—meaning they would occur after the drug could be targeted for price negotiations...Study co-author and NPC senior director of research Julie Patterson told BioSpace that, had a policy like the IRA been enacted earlier, some of the subsequent indications found in her team’s study might have been affected by the IRA. ‘Rivaroxaban’s indication to reduce the risk of major cardiovascular events in patients with coronary artery disease was approved over seven years after the drug’s initial approval,’ she said. ‘Empagliflozin’s indications in heart failure were approved over seven years after the drug was first approved.’” Full

 

Hospitals’ Drug Price Markups Incentivize Consolidation And Reduce Funding For Pharmaceutical Innovation

(3/6, James C. Robinson, Health Affairs Forefront) comments “...The ‘buy and bill’ method of reimbursement is inherently inflationary in a market context where the ‘bill’ price can be leveraged higher by hospital systems that dominate their local markets...Outright replacement of ‘buy and bill’ by a system in which payments to hospitals are based on actual inventory costs would provide an even more logical solution. For its part, the 340(B) program should be returned to its original purpose of helping hospitals obtain price discounts on the drugs they administer to indigent patients.” Full

 

England’s NICE Asks For Feedback On New Approach To Prioritizing HTAs

(3/6, Eliza Slawther, Pink Sheet) reports “...NICE, the health technology assessment body for England and Wales, plans to reform how it prioritizes therapies and topics for appraisal by introducing a single prioritization board and common framework.” Subscription Required

 

UK: Sanofi’s Xenpozyme Not Recommended by NICE for Ultra-Rare Disease ASMD

(3/6, Emily Kimber, PMLiVE) reports “Sanofi’s Xenpozyme (olipudase alfa) enzyme replacement therapy has not been recommended by the National Institute for Health and Care Excellence to treat acid sphingomyelinase deficiency (ASMD) types AB or B.” Full

Press Releases

AHRQ National Advisory Council to Meet Virtually on March 14

(3/6, AHRQ Press Release) “...The meeting will feature several presentations, including...Update from the NAC Subcommittee for AHRQ’s Patient-Centered Outcomes Research Trust Fund Investments, Karin Rhodes, M.D., M.S., Chief Implementation Officer, AHRQ.” Full

 

The American Cancer Society & Flatiron Health Announce Recipients of Fourth Annual Real-World Data Impact Award

(3/6, American Cancer Society Press Release) “...‘Flatiron Health is incredibly excited to continue our partnership with the American Cancer Society, promoting the use of real-world data to advance innovative cancer research that supports more effective and more equitable treatments and outcomes for patients.’ said Neal Meropol, MD, Vice President of Research Oncology at Flatiron Health. ‘Flatiron’s EHR-derived real-world data offers researchers a unique opportunity to drive inclusive research that has the potential to answer key questions relevant to cancer care delivery.’” Full

 

It’s Been Four Years Since COVID-19 Was Declared a Pandemic. What Have We Learned from Research Powered by PCORnet®?

(3/6, PCORnet Press Release) “From supporting a key collaboration with the U.S. Centers for Disease Control and Prevention to enabling the swift establishment of a research-ready community of healthcare workers, PCORnet resources were critical to powering key COVID-19 insights in the acute phase of the pandemic. Today, PCORnet resources continue to serve at the forefront of learning on important topics like long COVID and the effectiveness of repurposed medications to treat COVID-19.” Full

Journals

Comparative Effectiveness of Bimekizumab and Secukinumab in Patients with Psoriatic Arthritis at 52 Weeks Using a Matching-Adjusted Indirect Comparison

Philip J Mease, et al.

March 6, 2024, Rheumatology and Therapy

PubMed

 

Comparison of Effectiveness of Programmed Death Protein 1 and Programmed Death Ligand 1 Inhibitors in Extensive-Stage Small-Cell Lung Cancer: A Meta-Analysis of Randomized Controlled Trials and Observational Studies

Nanush Damarpally, et al.

March 6, 2024, Cureus

Cureus

News from Tuesday, March 5, 2024

Articles

Additional Benefit Seen for Colonoscopy Versus Sigmoidoscopy

(3/5, Elana Gotkine, HealthDay) reports “There may be a limited additional colorectal cancer (CRC) screening benefit for colonoscopy compared with sigmoidoscopy, according to a study published online Feb. 29 in JAMA Network Open.” Full

 

As Medicare Price Negotiations Roll On, Drugmakers Counter HHS' Initial Offers

(3/4, Zoey Becker, Fierce Pharma) reports “...Every drugmaker with a product on the list has responded to the government’s Feb. 1 pricing offer with a counteroffer, [HHS] said in a Monday press release. The agency is pleased with the ‘good-faith, up front negotiations,’ HHS Secretary Xavier Becerra said in the release. ‘We are committed to constructive dialogue and are glad the drug companies are coming to the table.’” Full

 

Republican Senators Join Attack On Biden's Proposal to Seize Drug Patents Based On Price

(3/4, Zachary Brennan, Endpoints News) reports “...As President Joe Biden continues to tout his administration's efforts to lower drug prices, 16 Republican senators on Monday raised concerns with NIH director Monica Bertagnolli over plans to ‘march-in’ and seize pharma company patents if the price of a drug is too high.” Full

 

Romney, Colleagues Oppose Biden Proposal to Illegally Seize Drug Patents

(3/4, U.S. Senator for Utah Mitt Romney) comments “U.S. Senator Mitt Romney (R-UT), member of the Senate Health, Education, Labor, and Pensions Committee, today joined HELP Ranking Member Bill Cassidy, M.D. (R-LA) and 15 Republican colleagues in sending a letter to National Institutes of Health (NIH) Director Monica Bertagnolli, M.D. raising serious concerns with the Biden Administration's proposal that attempts to allow agencies to seize drug patents from companies under the Bayh-Dole Act if the Administration views that the price the company is charging is too high.” Full

 

Regulatory Tracker: England's NICE Rejects AZ and Daiichi's Enhertu in HER2-Low Breast Cancer

(3/5, Fraiser Kansteiner, et al., Fierce Pharma) reports “...England's cost watchdogs at the National Institute for Health and Care Excellence (NICE) opted to not recommended AstraZeneca and Daiichi Sankyo’s Enhertu in certain patients over pricing concerns. Specifically, Enhertu is not recommended for NHS coverage in the treatment of HER2-low metastatic or unresectable breast cancer after chemotherapy.” Full

 

BioRationality: EMA Announces Readiness to Waive Comparative Efficacy Studies of Biosimilars

(3/4, Sarfaraz K. Niazi, PhD, The Center For Biosimilars) ...Sarfaraz K. Niazi, PhD, takes a look at the European Medicines Agency's announcement that it will investigate whether comparative efficacy tests should be needed for a biosimilar to receive regulatory approval. It did not take long for the European Medicines Agency to act after the FDA held a conference on ‘Increasing the efficiency of biosimilar development’ in September 2023, which was participated by several regulatory agencies and other stakeholders, debating issues relating to removing hurdles in the approval of biosimilars... Full

Press Releases

Nair Appointed to ICER California Technology Assessment Forum Evidence Appraisal Council

(3/5, University of Colorado Skaggs School of Pharmacy and Pharmaceutical Sciences Press Release) “Dr. Kavita Nair, PhD, Professor, has been appointed to the Institute for Clinical and Economic Review (ICER) California Technology Assessment Forum independent evidence appraisal council. Over the last decade her primary research interests lie in evaluating the treatment of neurological diseases. Primarily in examining the comparative effectiveness of disease modifying agents used in the management of multiple sclerosis using a combination of several measures to include brain atrophy measurement, clinical cognition, blood biomarkers and patient reported outcomes.” Full

 

New Treatment Option Available Today for Womb Cancer

(3/5, NICE Press Release) “A new treatment option given with chemotherapy is recommended for some types of advanced or recurrent womb cancer in final draft NICE guidance published today. 540 adults will have access to Dostarlimab (also called Jemperli and made by GSK). Dostarlimab is a monoclonal antibody and helps the immune system to fight cancer. It is given through a drip over 30 minutes in hospital.” Full