US FDA Introduces the Oncology QCARD Initiative
Although real-world data (RWD) is increasingly being used in oncology for clinical development, safety evaluation, and regulatory decision-making, the widely varying data sources make quality evaluation difficult. To address this issue, the US Food and Drug Administration (FDA) has announced the Oncology QCARD Initiative.
Medicare Drug Pricing Negotiations Loom Large Over Biotech Industry
At two recent biotech conferences, a consistent theme of presentations and conversations was the upcoming Medicare drug pricing negotiations from the Inflation Reduction Act. Representatives from smaller biotechs expressed concern that the negotiations will hamper partnership opportunities while companies large and small decried the legislation as a roadblock to innovation.
Real-World Evidence Study Demonstrates Efficacy of CAR T-Cell Therapy in Older Lymphoma Patients
Real-world evidence (RWE) published in Blood demonstrated the efficacy of CAR T-cell therapy in treating relapsed or refractory diffuse large B-cell lymphoma (DLBCL) in older patients. While median overall survival was similar between adults 65 and up and adults over 75, median event-free survival was better in the adults over 65 but younger than 75.
Study Finds Americans with Health Issues Sticking with Managed Care Plans
A recently published study in JAMA finds that sicker patients of advanced age tend to stick with managed care plans. This is a change of pace from recent years, when the industry was decried for preferentially selecting healthier patients. The study was conducted using Medicare enrollment data taken from 45,000 patients.
Looking Past Amyloid – The Future of Alzheimer’s Therapeutics
One of the key therapeutic targets in Alzheimer's disease is amyloid, a sticky protein that builds up in clumps outside neurons, causing cell death and cognitive decline. However, new therapeutics targeting the protein have only shown mild efficacy in delaying disease progression. As a result, drug developers are looking for other biological targets.
7 Key Drug Shortages to Watch
Drug shortages have rocked the US, imperiling access to therapeutics for millions of patients with a wide array of diseases and conditions. A new Becker’s Hospital Review article lays out 7 of the most prominent drug shortages to watch. One is the attention deficit hyperactivity disorder (ADHD) drug Vyvanse (lisdexamphetamine dimesylate).
Pharma’s Legal Challenges Against US Drug Pricing Negotiations Face an Uphill Battle
Merck, BMS, and three industry groups filing lawsuits against the Department of Health and Human Services (HHS) and the Centers for Medicare and Medicaid Services (CMS) may face an uphill battle in stopping upcoming Medicare drug pricing negotiations. The suits filed by the first two are based on the notion that the negotiations violate the First Amendment, freedom of speech, and the Fifth Amendment, the right against the government seizing property without just compensation.
NICE Requests More Data Before Covering Mounjaro
The UK’s National Institute for Health and Care Excellence (NICE) has sent a request to Eli Lilly asking for more data regarding Mounjaro (tirzepatide) before it will cover it for NHS patients. The decision comes based on data comparing the drug’s efficacy to Novo Nordisk’s rival drug Ozempic and standard insulin therapy. While the data showed Mounjaro was more effective at weight loss and glucose control, NICE needs more information before making a coverage decision.
UCB Gets FDA Approval for Generalized Myasthenia Gravis
UCB’s Rystiggo (rozanolixizumab-noli), a treatment for two subtypes of generalized myasthenia gravis (gMG), has been approved by the US Food and Drug Administration (FDA). Muscle weakness episodes in the rare autoimmune disease is caused by antibodies that cling to proteins at the connection site between nerves and muscles. Rystiggo, an antibody-based treatment, reduces circulating antibodies and reduces symptoms.
NEW! MALCOM e-learning: up-skilling your HEOR and market access teams
A new on-demand e-learning platform is now available for HEOR and market access teams. MALCOM (Market Access Learning Compendium) helps [...]
Examining the Challenges in Implementing Value-Based Care at Academic Medical Centers
A new report in Health Affairs Scholars looks into the challenges that academic health centers (AMCs) face in implementing value-based care models. AMCs provide care that exceeds that of non-AMCs, but features that help them excel may hinder value-based payment models. Key challenges they encounter are rigidity of workflows and the financial agreements they have with practitioners.
Education as a Social Determinant of Cardiovascular Disease Outcomes
A newly published study in BMC Public Health reports that education level may be a critical independent social determinant of health (SDoH) influencing cardiovascular disease (CVD) outcomes. The study, which drew upon data from over 210,000 participants, found that people without a high school degree had a 47.9% higher mortality rate in atherosclerotic CVD than those with college degrees.
FDA Approves Pfizer’s Treatment for Alopecia Areata in Adolescents
The US Food and Drug Administration (FDA) has approved Pfizer’s Litfulo (ritlecitinib) for use in adolescents with severe alopecia areata, an autoimmune condition that causes hair loss. The approval marks the first treatment for the psychologically damaging condition in teens. Positive results from a phase 2b/3 study, which included over 700 patients, supported the FDA’s decision..
New £21m Fund to Expand Artificial Intelligence in NHS Trusts
A newly announced fund will help implement artificial intelligence (AI) in NHS Trusts. The AI Diagnostic Fund will help promote the use of AI solutions for imaging and diagnostics for conditions like cancer, cardiovascular disease, and stroke. One key goal is to get AI tools for stroke diagnosis to all stroke centers by the end of this year.
EMA Releases Report on Real-World Evidence
The European Medicines Agency published a report last week discussing the use of real-world evidence (RWE) in clinical trials and regulatory decisionmaking, the benefits it can provide, and the challenges in doing so. RWE, according to the report, is widely used in pharmacovigilance studies, but its use in early drug development still faces uncertainties.
FDA Grants Roche Accelerated Approval for Lymphoma Therapeutics
The US Food and Drug Administration (FDA) has granted Roche accelerated approval for Columvi, its antibody-based therapeutics for two aggressive forms of lymphoma. Results from a phase 1/2 study found the drug provided durable remission for over half of patients and a complete response for 43%, with 66% continuing to respond for 9 months or longer.
FDA Grants Full Approval of Amgen’s Supplemental Biologics License Application for Leukemia Treatment
Amgen’s supplemental biologics license application (sBLA) for its leukemia treatment Blincyto has received full approval by the US Food and Drug Administration (FDA). The move comes based on new data from two phase 3 trials, upgrading the treatment’s accelerated approval to a full approval in the treatment of CD19-positive B-cell precursor acute lymphoblastic leukaemia (B-ALL).
Verana Launches Real-World Evidence-Driven Clinical Trial Site Selection Platform
Verana Health has launched VeraSite, a new platform that uses real-world evidence (RWE) to help clinical trial sponsors select clinical trial sites. The tool uses electronic health records (EHR) to identify locations with large eligible patient populations. With this platform, Verana hopes to address one of the biggest challenges facing clinical trials – recruitment.
Biden Administration Meets with Drugmakers to Discuss Naloxone Prices
Officials from the Biden Administration met with drugmakers this week to discuss prices of naloxone, a drug used to immediately reverse opioid overdoses. Emergent Biosolutions, the manufacturer of brand name Narcan, took heat for setting its price for 2 doses at $50. Concerns were also raised about the high cost of other formulations, which can reach $100 for uninsured people.
Sarepta’s New Duchenne Gene Therapy to Cost $3.2 Million
Hot on the heels of approval by the US Food and Drug Administration (FDA), Sarepta has announced the price of its Elevidys, its new gene therapy for Duchenne muscular dystrophy, at $3.2 million. This makes it one of the most expensive therapeutics in history. However, Sarepta claims this price is far below it’s initial cost-effectiveness estimates.
FDA Approves First Gene Therapy for Duchenne Muscular Dystrophy
In a first for the rare disease, the FDA has approved Sarepta’s gene therapy for Duchenne Muscular Dystrophy, a genetic condition that causes progressive muscle wasting in childhood, primarily affecting boys. The approval covers children between the ages of 4 and 5, contrary to the request from advocacy groups to open up the treatment to all children with the disorder who can still walk.
From Analysis to Action: Exploring Health Equity with Aletha Maybank
In a new interview with Nature, senior vice president and chief health equity officer at the American Medical Association (AMA) Aletha Maybank, MD, MPH, speaks about her work analyzing the processes underlying health disparities and how they can be targeted to promote better outcomes in marginalized populations.
CDC Adcomm Hesitantly Recommends RSV Vaccines for Older Adults
GSK’s Arexy and Pfizer’s Abrysvo vaccines for respiratory syncytial virus (RSV) have received recommendations from the US Centers for Disease Control and Prevention’s (CDC’s) Advisory Committee on Immunization Practices (ACIP). While the committee voted almost unanimously in support of the vaccines for adults aged 60-64, the recommendation for adults over 65 was far more contentious, sitting at 9-5 for and against, respectively.
FDA Grants Priority Review for Sickle Cell Gene Therapy by Bluebird Bio
The US Food and Drug Administration (FDA) has accepted bluebird bio’s application for priority review for its sickle cell gene therapy lovotibeglogene autotemcel (lovo-cel). The decision was made based on efficacy data from 36 patients and safety results from 50. Sickle cell disease (SCD) is an inherited blood disorder caused by a mutation in the gene encoding for hemoglobin that sticks together inside red blood cells.
PhRMA Enters the Legal Fight against US Medicare Drug Pricing Negotiations
The pharmaceutical trade group PhRMA has entered the fray, joining BMS and Merck in filing legal challenges against the upcoming US Medicare drug pricing negotiations. Unlike the arguments raised by the two pharma giants’ complaints, which centered on the First Amendment, PhRMA’s argument hinges on fines imposed on manufacturers who do not work with the negotiation process violates the Eighth Amendment. In addition to pharma, the Global Colon Cancer Association and the National Infusion Center Association have filed their own suits.
