Healthcare Costs to Jump by 7% in 2024
A report by PwC projects that healthcare costs will rise by 7% next year. The report was based on interviews with major payer organizations covering over 110 million patients in the US. Estimated increases in costs are attributed to labor shortages, rising drug prices, provider contracts, and more.
BioMarin Nets First FDA Approval for Severe Hemophilia A Gene Therapy
BioMarin Pharmaceuticals has received full approval by the US Food and Drug Administration (FDA) for its gene therapy Roctavian (valoctocogene roxaparvovec-rvox) for severe hemophilia A in adults. This marks the first gene therapy for patients with severe cases of the disease, which causes bleeding and progressive joint damage.
NPPA Caps Prices for 51 Medications
India’s National Pharmaceutical Pricing Authority (NPPA) has announced its latest round of ceiling prices for medications, this time capping the prices of 51 medications and 2 formulations for anti-coagulant therapeutics. The other drugs covered include 24 diabetes treatments. The NPPA is India’s key drug pricing regulatory body, in charge of setting ceiling prices for medications and formulations.
Canadian Agency for Drugs and Technologies in Health Publishes Real-World Evidence Guidance
As real-world evidence (RWE) and real-world data (RWD) increasingly supplement data from randomized controlled trials (RCTs) in regulatory decision-making in Canada, the Canadian Agency for Drugs and Technologies in Health (CADTH) has issued a guidance for RWE reporting. The guidance, published in coordination with the Institut national d’excellence en santé et en services sociaux (INESSS) and Canada Health, will help align RWE and RWD usage with regulatory bodies and health technology assessment (HGA) agencies.
The Next US Presidency Doesn’t Look Friendly to Pharma
No matter who wins the next US presidency, it doesn’t look that there will be a friend to the pharma and biotech industries in the White House, according to a new Axios article. Donald Trump, the current Republican front-runner has already repeated his previous campaign promise of not letting the US pay more for medications than other countries, while his rival Ron DeSantis launched a Florida state grand jury to scrutinize COVID-19 vaccine producers over alleged misrepresentation of safety information.
Looking at How Medicaid Expansion Affect Sexual and Reproductive Health
Extensive research has explored how Medicaid expansion has impacted health outcomes in 40 US states, generally finding that it improves health outcomes for patients and provides financial benefits for state governments. A new report by the Kaiser Family Foundation reviews the recent literature exploring the impact of Medicaid expansion on reproductive and sexual health, beginning with coverage.
Study Explores Real-World Data Reporting Quality According to RECORD Statement
With the rise of real-world data (RWD) and real-world evidence (RWE), a lack of transparency impairs reproducibility and quality assessment. The Reporting of studies Conducted using Observational Routinely-collected health Data (RECORD) statement was drafted to help address this issue. A newly published study in BMC Medical Research Methodology explores reporting quality in RWD studies using the RECORD checklist.
CMS Will Cover New Alzheimer’s Drugs with Real-World Data Registry Participation
The US Centers for Medicare and Medicaid Services (CMS) has provided more insights into coverage details regarding new therapeutics for Alzheimer’s disease. Once a drug receives approval by the Food and Drug Administration (FDA), Medicare will cover them if clinicians participate in a real-world data (RWD) registry.
Turning Real-World Data into Action in the NHS
Despite recent high-profile examples, biopharma startups face difficulties partnering with the UK’s NHS. This represents a missed opportunity due to the agency’s vast repository of healthcare real-world data (RWD), which could be used to accelerate drug development, value-assessment, and regulatory approval applications.
ICER Founder and President to Leave Position This Year
The Institute for Clinical and Economic Review (ICER) has announced that its founder, Steve Pearson, MD, will step down from his role as president later this year. ICER’s COO Sarah Edmond will take over the position as president, while Pearson will move to an advisory position for the organization, which performs value-assessments on therapeutics.
Pfizer and OPKO Get FDA Approval for Childhood Growth Hormone Deficiency Treatment
OPKO Health and Pfizer’s Ngenla, a treatment for childhood growth hormone deficiency, has received approval by the US Food and Drug Administration (FDA). The treatment for the rare disease, which causes short stature and delayed puberty, had been rejected once by the agency with a request for more information.
Tips for Managed Care to Approach Value-Based Care
The shift towards value-based care has the potential to benefit patients, providers, and payers alike, but it brings about unique challenges for managed care plans. In a new Managed Healthcare Executive article, learn more about value-based care payment models and strategies to apply them successfully in managed care organizations.
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.
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.
