22 February 2025
Identifying key randomised clinical trials that could transform clinical care and public health
Clinical trials have been pivotal to the improvements in public health and clinical care, leading to many millions of additional years of healthy life globally. Some examples of disease areas in which outcomes have improved include primary and secondary prevention of cardiovascular disease, breast cancer, some forms of leukaemia, and HIV treatment and prevention. In all of these disease domains, clinical practitioners have embraced the centrality of evidence from clinical trials and large-scale randomised clinical trial (RCT) collaborations have been implemented in partnership with patients, health systems, industry, and other partners.
21 February 2025
F.D.A. Firings Decimated Teams Reviewing A.I. and Food Safety
The Trump administration has implemented significant layoffs at the FDA, targeting recently hired employees in the food safety, medical devices, and tobacco products divisions. These cuts are part of a broader effort to reduce the federal workforce, impacting the FDA's ability to review the safety of food additives and medical devices. The layoffs have raised concerns about the agency's capacity to protect public health, especially given the FDA's reliance on newer staff with up-to-date technical skills.
21 February 2025
FDA Approves First Treatment for Cerebrotendinous Xanthomatosis, a Rare Lipid Storage Disease
The FDA has approved Ctexli (chenodiol) as the first treatment for cerebrotendinous xanthomatosis (CTX), a rare lipid storage disease. This approval provides a new therapeutic option for adults suffering from CTX, which can lead to neurological and systemic complications if left untreated.
21 February 2025
US FDA approves Mirum Pharma's genetic disorder drug
The U.S. Food and Drug Administration (FDA) has approved Mirum Pharmaceuticals' drug, Livmarli, for treating progressive familial intrahepatic cholestasis (PFIC), a rare genetic liver disorder. This approval allows the drug to be used for reducing severe itching in patients aged five years and older. The decision was based on positive results from a late-stage trial involving 93 patients.
13 February 2025
How Trump’s Medical Research Cuts Would Hit Colleges and Hospitals in Every State
Changes to a key funding formula will reduce research grants
at hospitals and universities by billions — and may discourage future research.
The Trump administration's decision to cut funding for the
National Institutes of Health (NIH) is causing significant concern in the
scientific community. By capping indirect costs at 15%, the administration has
effectively reduced the budget for essential administrative and facility
expenses. This move has led to lawsuits and fears that major research
institutions will struggle to continue their work, potentially stalling
scientific progress and impacting the careers of young researchers.
19 February 2025
Artificial intelligence for modelling infectious disease epidemics
Infectious disease threats to individual and public health
are numerous, varied and frequently unexpected. Artificial intelligence (AI)
and related technologies, which are already supporting human decision making in
economics, medicine and social science, have the potential to transform the
scope and power of infectious disease epidemiology. Here we consider the
application to infectious disease modelling of AI systems that combine machine
learning, computational statistics, information retrieval and data science. We
first outline how recent advances in AI can accelerate breakthroughs in
answering key epidemiological questions and we discuss specific AI methods that
can be applied to routinely collected infectious disease surveillance data.
Second, we elaborate on the social context of AI for infectious disease
epidemiology, including issues such as explainability, safety, accountability
and ethics. Finally, we summarize some limitations of AI applications in this
field and provide recommendations for how infectious disease epidemiology can
harness most effectively current and future developments in AI.
20 January 2025
Young people are crucial to research — how can they be paid fairly?
Young people are playing a crucial role in research, but
compensating them fairly presents ethical and practical challenges. Kids and
teens, aged 12 to 24, are increasingly involved as advisers and co-researchers,
not just participants. This shift, supported by major funders, aims to empower
youth through participatory action research, where they identify research
questions and analyze data themselves. Their involvement has led to significant
contributions, such as policy recommendations for climate adaptation and
blocking child marriages in Bangladesh.
19 February 2025
Vaccine targeting pancreatic cancer shows promise in new study of clinical trial
A clinical trial at Memorial Sloan Kettering Cancer Center
is showing promise for a new mRNA vaccine targeting pancreatic cancer. The
trial, led by Dr. Vinod Balachandran, combines standard treatments with a
personalized vaccine designed from the patient's tumor. This approach aims to
teach the immune system to recognize and attack cancer cells, inspired by the
immune responses seen in long-term pancreatic cancer survivors. Early results
from the phase 1 trial, which included 16 patients, suggest that the vaccine
could potentially extend survival times for patients with this typically deadly
cancer.
18 February 2025
Can large, simple trials bring drug developers back to common diseases?
Martin Landray, a physician and epidemiologist at Oxford
University, argues that large, simple clinical trials can make drug development
for common diseases more affordable and efficient. He highlights the success of
the UK's RECOVERY trial during the COVID-19 pandemic, which tested multiple
treatments on a modest budget. Landray's non-profit, Protas, aims to replicate
this model for other common diseases, reducing the cost and complexity of phase
III trials. This approach could encourage drug developers to invest more in
treatments for widespread health issues like heart disease and stroke.
1.6 Million Clinical Research Records With PII and Patient Medical Info Exposed in Data Breach
A data breach at DM Clinical Research exposed over 1.6 million records containing sensitive personal and medical information. The unprotected database included names, dates of birth, contact details, vaccination statuses, and other health-related data. Discovered by cybersecurity researcher Jeremiah Fowler, the breach was quickly reported and secured. However, the incident raises significant concerns about data privacy and the protection of patient information.
18 February 2025
2024's top 10 clinical trial flops
Fierce Biotech's report on the top 10 clinical trial flops
of 2024 highlights some significant setbacks in drug development. Notable
failures include AbbVie's emraclidine for schizophrenia, which flunked phase 2
trials, and GSK's HSV vaccine, which failed in phase 2, ceding the race to
Moderna. These high-profile flops underscore the inherent risks in
clinical-stage drug development, particularly in challenging therapeutic areas
like Alzheimer's disease, schizophrenia, and herpes simplex virus.
30 January 2025
FDA Approves Novel Non-Opioid Treatment for Moderate to Severe Acute Pain
The FDA has approved Journavx (suzetrigine), a new
non-opioid medication for treating moderate to severe acute pain in adults.
This first-in-class drug targets pain-signaling pathways involving sodium
channels in the peripheral nervous system, reducing pain before it reaches the
brain. The approval is based on successful trials showing significant pain
reduction compared to placebo. This new option aims to provide effective pain
relief while mitigating the risks associated with opioid use.
Introducing JOURNAVX™ (suzetrigine) for Moderate-to-Severe Acute Pain
31 January 2025
European Medicines Agency Clinical Trials Regulation becomes fully applicable
The Clinical Trials Regulation (CTR) is now fully applicable
across the European Union (EU) as of January 31, 2025. This regulation replaces
the previous Clinical Trials Directive and aims to streamline the submission,
assessment, and oversight of clinical trials through the Clinical Trials
Information System (CTIS). All ongoing trials must now comply with the CTR, and
any that haven't transitioned may face corrective measures. The CTR is designed
to enhance transparency and make Europe a more attractive location for clinical
research.
