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
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
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
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
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
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
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
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
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
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
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
‘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
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
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
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
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
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
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
News from Wednesday, November 15, 2023
Experts Update Guidance on COVID-19 Treatment and Therapies
(11/15, Kennedy Ferruggia, Pharmacy Times) reports “...‘This living guideline responds to emerging evidence from RCTs on existing and new drug treatments for COVID-19. The guideline leverages several living networks' meta-analyses which iteratively incorporate newly available trial data and allow for analysis of comparative effectiveness of multiple covid-19 treatments,’ said the study authors.” Full
UK: RWE Accepted As ‘Primary Evidence’ In 20% Of NICE Appraisals Over Past Year
(11/15, Eliza Slawther, Pink Sheet) reports “...NICE, the health technology assessment body for England and Wales, says it has seen ‘good uptake’ of its new evaluation methods manual and real-world evidence framework, both introduced last year.” Subscription Required
Special Issue of Medicare Care Supports the Need to Study Economic Impacts on Patient Outcomes
(11/14, Wolters Kluwer Health Press Release) “A special supplemental issue of Medical Care, sponsored by the Assistant Secretary for Planning and Evaluation (ASPE) in the U.S. Department of Health and Human Services, supports the growing recognition that economic factors often affect health outcomes, patient decision-making, and equity in health care.” Full
Visualisation of Evidence for Shared Decsion Making
Marie-Anne Durand, et al.
November 15, 2023, BMJ Evidence-Based Medicine
Avenues for Strengthening PCORnet’s Capacity to Advance Patient-Centered Economic Outcomes in Patient-Centered Outcomes Research (PCOR)
Waitman, Lemuel R., et al.
December 2023, Medical Care
Economic Outcomes in Patient-Centered Outcomes Research: A Paradigm Shift
Smith, Scott R., et al.
December 2023, Medical Care
Medicare Data Linkages for Conducting Patient-Centered Outcomes Research on Economic Outcomes
Brown, Derek S., et al.
December 2023, Medical Care
Linking Medicare-Medicaid Claims for Patient-Centered Outcomes Research Among Dual-Eligible Beneficiaries
Jones, Kelley A., et al.
December 2023, Medical Care
News from Tuesday, November 14, 2023
Podcast: POC for Verve? Plus IRA & East-West Deals: A BioCentury Podcast
(11/13, Jeff Cramer, BioCentury) comments “...The editors also assess the latest developments related to the Inflation Reduction Act, including a National Pharmaceutical Council study showing how the law could impede collection of evidence about new indications for drugs, and assess a selection of biopharma deals, including a pair of sizable cross-border deals for Korea biotechs, a partnership between Legend Biotech Corp. and Novartis AG and the IPO by Cargo Therapeutics Inc.” Full
FDA Quietly Announces Office Of Pharmaceutical Quality Restructure
(11/13, Jessica Karins, InsideHealthPolicy) reports “...All research will be consolidated under a new Office of Pharmaceutical Quality Research, and all quality assessment activities will be consolidated under the Deputy Director of Science. A new Office of Quality Assurance will include the center’s Quality Management System, Enterprise Risk Management, and training and development activities, as well as project management not related to drug applications.” Subscription Required
EU Joint Clinical Assessment (JCA) – Implications for Pharma and Medtech
(11/14, PMLiVE) reports “Maria Dimitrova (Consultant – HTA) and Samantha Gillard (Director – HTA) provide an overview of the EU Joint Clinical Assessment (JCA) and what it means for Pharma and Medical Device manufacturers launching new technologies from 2025.” Full
NICE Launches ‘Concierge System’ To Guide Companies Through HTA Process
(11/14, Eliza Slawther, Pink Sheet) reports “...The health technology assessment body for England and Wales has launched an integrated, flexible advice portal to help guide companies through its potentially ‘bewildering’ health system.” Subscription Required
ICER Publishes Evidence Report on Treatment for Pulmonary Arterial Hypertension
(11/14, ICER Press Release) “...‘Pulmonary arterial hypertension is a progressive disease that can lead to debilitating shortness of breath, fatigue, and lightheadedness as the heart struggles to pump blood through the lungs,’ said ICER’s Chief Medical Officer, David Rind, MD. ‘Current drug treatments are primarily combinations of vasodilators, and these can be very burdensome and cause significant side effects. Sotatercept has a novel mechanism of action and it is administered subcutaneously every few weeks, which reduces burdens. Current evidence suggests that sotatercept has fewer side effects and improves short-term outcomes, but we have some uncertainties about long-term efficacy and safety.’” Full
Alternative Approaches to Measuring Value: An Update on Innovative Methods in the Context of the United States Medicare Drug Price Negotiation Program
Michael J DiStefano, et al.
November 14, 2023, Expert Review of Pharmacoeconomics & Outcomes Research