Study Finds Many New Drug Indications Don’t Add Value
A newly published study in the BMJ finds that almost 60% of drugs receiving new indications from the US Food and Drug Administration (FDA) do not add high therapeutic value to patients. As a result, the researchers argue that patients and providers should be aware and that prices should be set accordingly.
Clinical Trial Diversity on the Decline
According to a recent IQVIA report, clinical trial diversity is on the downswing despite recent efforts to the contrary. The research specifically identified significant under-representation for Black and Hispanic patients. Industry action is needed, according to IQVIA, as clinical trial under-representation is systemic and negatively impacts people from marginalized racial and ethnic backgrounds.
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.
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.
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.
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.
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.
Real-World Data Study Finds Breztri Reduces COPD Exacerbation Risk
A real-world data (RWD) study presented at this year’s ATS International Conference shows that Breztri Aerosphere reduces the risk of COPD exacerbations. The study, which looked at RWD from over 2,400 patients, showed that early treatment with the combination therapy reduced the risk of exacerbations by nearly a quarter.
Atropos Health Shares Real-World Evidence Examining High vs Low-Cost Medications
In a poster session at this year’s American Society of Health-System Pharmacists (ASHP) meeting, Atropos Health presented real-world evidence examining the impact of prescribing high-cost vs low-cost medications on patient health outcomes. The study, which looked at electronic health records (EHR) taken between 2015 and 2021, found no difference in outcomes between drugs at different price brackets.
Talking Fast-Track Designation and Drug Development with Dr Courtney DiNardo
In a new interview with AJMC, Courtney DiNardo, MD, MSCE, of the University of Texas MD Anderson Cancer Center detailed how FDA fast track designation can help with the drug development process. She argues that it can help guide trial development in ways that align with agency preferences and priorities. In addition, she spoke about patient recruitment and retention.
340B Eligibility Associated with Reduced Biosimilar Uptake
A recently published study in Health Affairs found reduced biosimilar uptake in hospitals in the 340B program, which provides drugs at reduced cost to certain care centers. The study examined nearly 600 hospitals using claims data, the Healthcare Cost Report Information System, and a 340 participation database. 340B-eligible care centers had a biosimilar usage rate that was 66% lower than non-eligible hospitals.
AstraZeneca Looking Toward Early Full Approval for Anticoagulant Reversal Medication
Following positive results in a phase IV clinical trial, Astrazeneca is making moves to apply for full approval for its anticoagulant reversal drug Andexxa earlier than expected. Data from the trial, which is being ended early based on outside recommendations, showed that the drug was effective in patients on certain anticoagulants who are experiencing an intracranial hemorrhage.
Real-World Data Study Finds Two Year Immunotherapy Regimens Appropriate for Advanced Lung Cancer
A real-world data (RWD) study published in JAMA Oncology finds that administering immunotherapy to patients with advanced non-small cell lung cancer (NSCLC) is appropriate. The study, which followed 1091 patients at Penn Medicine’s Abramson Cancer Center, found that patients whose cancer hasn’t progressed after two years of immunotherapy did not gain any clinical benefits by prolonging treatment past a 2-year period.
EMA Calls for Novartis’ Sickle Cell Treatment to Lose Market Authorization
Novartis took a hit this week as the European Medicines Agency (EMA) has recommended that its market authorization for Adakveo, a sickle cell medication, be revoked. Although initial data suggested the drug could help reduce pain crises in patients 16 and older, the agency argues that new data showing increased levels of serious side effects do not outweigh the modest treatment effect.
Accelerating and Improving Clinical Trials with Real-World Data
The ubiquitous adoption of electronic health records (EHR) has supported the explosion of real-world data (RWD) applications in clinical trials. RWD has the potential to accelerate and optimize clinical trials while expanding their reach. In a new Life Science Leader article, learn more about the many ways that RWD can improve clinical trials and accelerate clinical development and market access.
Yuhan Corp. Releases Real-World Data Supporting Leclaza in Lung Cancer
Researchers at Korea’s National Cancer Center have released new real-world data (RWD) supporting the use of Yuhan Corp’s Leclaza (lazertinib) in the treatment of a subset of non-small cell lung cancer (NSCLC). The study, basefd on data from 103 patients who used Leclaza as a second-line treatment, showed that the drug improved median progression-free survival (mPFS) out to 13.9 months, an improvement on the 11.1 months mPFS demonstrated in a randomized clinical trial.
ISPOR Investigates HEOR Research in LMICs with New Report
In a newly released report, ISPOR breaks down the state of health economics and outcomes research (HEOR) in low- and middle-income countries (LMICs). The report, entitled “Advancing HEOR in Low- and Middle-Income Countries” covers the need to better implement HEOR research in LMICs and ways to do so that will advance healthcare decision-making and patient outcomes.
NICE Recommends Expanding Approval for AstraZeneca’s Heart Failure Med
The UK’s National Institute for Health and Care Excellence (NICE) has recommended that Astrazeneca’s heart failure med Forixga (dapagliflozin) should have its indication expanded to adults regardless of the degree of their left ventricular ejection fraction (LVEF). The drug was previously indicated with patients with reduced LVEF of 40% or lower.
Real-World Data Backs SPRINT® PNS System for Pain Relief
Real-world data (RWD) published in Pain Physician found that SPR Therapeutics’ percutaneous peripheral nerve stimulation (PNS) device significantly reduced pain in patients. The data, which came from over 6,100 patients, showed that more than 70% of patients saw a 50% or greater reduction in their pain symptoms.
PTC’s Phenylketonuria Treatment Meets Phase III Endpoint
PTC Therapeutics announced positive results from a phase III clinical trial for its drug sepiapterin in the treatment of phenylketonuria (PKU), a rare disease causing excessive accumulation of the amino acid phenylalanine. The drug met the primary endpoint of reducing blood levels of phenylalanine in both adult and pediatric PKU patients in the trial.
Drugmakers Are Putting Family-Funded Rare Disease Therapeutic Research on Hold
Families of patients have often led funding for rare disease therapeutic development through donation campaigns. However, many drugmakers have put these programs on hold, with 26 rare disease projects shelved in the past 16 months. This has caused an outcry from patient advocates. Pharma analysts argue financial reasons are to blame.
Monica Bertagnolli Nominated for Director of NIH
The Biden administration has nominated Monica Bertagnolli for the next director of the US National Institutes of Health (NIH). Bertagnolli formerly worked as the chief of surgical oncology at the Dana-Farber Brigham Cancer Center and is currently the director of the National Cancer Institute (NCI). Her appointment is expected to make it through the narrowly Democratic-leaning senate some time later this year.
Real-World Evidence Backs Venetoclax-Based Treatment in Chronic Lymphocytic Lymphoma
A real-world evidence (RWE) study presented at this year’s ISPOR meeting shows that venetoclax-based treatment regimens improve patient outcomes in chronic lymphocytic lymphoma (CLL). Patients on venetoclax (Venclexta) had reduced rates of treatment initiation and had a longer time before new treatments or death. The study was based off electronic health records (EHR) from CLL patients across the US.
Real-World Data Study Examines the Past Decade of Biomarker Testing for Lung Cancer
A real-world data study presented at this year’s ISPOR meeting showed that biomarker testing for early-stage non-small cell lung cancer (NSCLC) increased steadily over the past ten years. Data from over one thousand patients with early-stage NSCLC from 2011 to 2021, finding that 74.1% of patients had biomarker testing performed within the first six months after being diagnosed with the disease.
Lung Cancer Detection AI Accurately Identifies Pre-Cancerous Growths
An artificial intelligence (AI) model developed by UK researchers identified abnormal lung growths that would later become cancerous with a high degree of accuracy. The AI model was developed in the LIBRA study using lung imaging data from 500 patients with large abonormal lung nodules. 82% of the tumors the AI model identified as high-risk were later found to be cancerous.
