UK Public Okay with NHS Data Storage with Some Reservations
The UK’s NHS generates massive amounts of real-world data (RWD) every day, but recently data security has become a concern. However, a recent survey found that 59% of participants feeling comfortable with the agency storing their data, although most (89%) felt it was important that the data was stored in the UK. A majority, however, do not feel comfortable with their data being processed using artificial intelligence (AI).
US Senator Bernie Sanders Looks to Cap Prices on Publicly Funded Drugs
US Senator Bernie Sanders (I – VT) is looking to include price caps on drugs developed with federal funding into a reauthorization of the 2006 Pandemic and All-Hazards Preparedness Act. The proposal would place a ceiling price that matches the lowest price for a given drug in France, Germany, Japan, Canada, the UK, or Italy.
ICER to Perform Value-Assessment for KarXT Schizophrenia Treatment
The Institute for Clinical and Economic Review (ICER) will perform a value-assessment on KarXT (xanomeline tartrate/trospium chloride), Karuna Therapeutics’ experimental therapeutic for schizophrenia. Public discussion of the assessment will take place next February at the 2024 New England CEPAC meeting.
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Amicus Therapeutics Gets EU Approval for Pompe Disease Drug
Amicus Therapeutics has received approval from the European Commission (EC) for Opfolda (miglustat), its treatment for Pompe disease, a rare disorder that causes progressive muscle weakness. The approval specifically covers the drug in combination with Pombiliti (cipaglucosidase alfa). Results from a recent phase 3 study showing positive changes in mobility and breathing, both affected by the disorder, supported the EC’s decision.
Examining Representation Disparities in aRCC Clinical Trials
A recently published abstract in the Journal of Clinical Oncology examined patient populations in clinical trials for advanced renal cell carcinoma (aRCC), finding that significant underrepresentation based on age, ethnicity, and racial backgrounds. The study examined data from multiple real-world evidence (RWE) and randomized controlled trials (RCTs) covering over 4800 patients.
A Wave of Humira Biosimilars Hit the US Market
Several new biosimilars to AbbVie’s Humira (adalimumab) have just hit the US market in the past week, bringing a new wave of competition for the expensive top-selling therapeutic of all time. The copycats come from companies like Organon, Samsung Bioepsis, Sandoz, and Boehringer Ingelheim. Hadlima, a product of Samsung and Organon, will retail for 85% less than brand name Humira.
Using Patient Registries to Accelerate Rare Disease Research
In a recent interview with Clinical Leader, Harsha Rajasimha, PhD, of the Indo US Organization for Rare Diseases (IndoUSrare) talks about the challenges facing rare disease research and how the use of patient registries can accelerate research and clinical development. Patient registries allow the collection of real-world data (RWD) that can be used in research, but doing so requires collaboration between all stakeholders.
Celltrans Secures FDA Approval for First Type 1 Diabetes Cell Therapy
The US Food and Drug Administration (FDA) has approved Lantrida (donislecel), the first cell therapy for diabetes type 1. Celltrans’ treatment is now approved for use in adults with “brittle” diabetes type I, a rare form of the disease wherein patients have recurrent severe hypoglycemic episodes. The approval is based on two open-label clinical trials with 30 patients.
Exploring Health Disparities in Childhood Epilepsy
A recently published article in Clinical Psychology in Medical Settings finds that health disparities faced by children and youth with epilepsy (CYE) are poorly explored in the scientific literature. The authors of the paper noted that very few epilepsy studies actually focused on identifying health disparities, with even fewer focusing on CYE.
CMS Publishes Revised Guidance for Medicare Drug Pricing Negotiations
As the upcoming Medicare drug pricing negotiations approach, the US Centers for Medicare and Medicaid Services (CMS) have released a revised guidance that better outlines the process. Pharma companies and industry groups have been anxiously awaiting such clarification, but the new information has done little to quell their concerns.
Looking to the Future of AI in Healthcare
Artificial Intelligence (AI) is increasingly being incorporated into every aspect of society, including healthcare. From diagnostics to reimbursement, AI is taking a foothold in healthcare and medicine from drug development to the clinic. However, significant barriers to realizing the potential of the technology still remain.
CMS Makes Changes to Upcoming Medicare Drug Pricing Negotiations
In response to a series of lawsuits from the pharma industry, the US Centers for Medicare and Medicaid Services (CMS) have announced changes to upcoming Medicare drug pricing negotiations. The agency dialed back a confidentiality policy associated with the negotiations, a sticking point for some plaintiffs who claimed it violated their First Amendment rights.
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.
