CER Daily Newsfeed

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

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

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

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

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

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

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

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

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

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