Using Real-World Data to Implement External Comparator Arm Studies
Single-arm trials (SATs) have entered the clinical trial space thanks to the growing abundance of real-world data (RWD) from sources like electronic health records (HER) that allows for the use of external comparator arms (ECAs). These trial formats help accelerate and enable research, especially when randomized controlled trials are infeasible. In a new article on PharmaPhorum, Dr. Gerd Rippin of IQVIA discusses the use of RWD in SATs.
Eiger’s Scraps Emergency Use Authorization Submission for COVID-19 Treatment
The US Food and Drug Administration has rejected Eiger BioPharmaceuticals’ request to meet with the agency regarding peginterferon lambda, its repurposed hepatitis D treatment for use in COVID-19. The company asked for the meeting to discuss an emergency use authorization in September based on data from the TOGETHER Phase III trial. However, it appears the data was insufficient for the agency.
The Disconnect Between Clinical Trial and Real-World Populations Holds Back Oncology Care
Randomized controlled trials (RCTs) are the gold standard for regulatory approval. However, cancer trial participants are often not representative of real-world patient populations. As a result, patient outcomes may not mirror those found in trials. In a new article published on OncLive, Maurie Markman, MD, discusses the shortcomings of traditional RCTs and how unrepresentative patient populations hold back therapeutic development.
External Control Arms as a Substitute for Placebo Groups
While randomized controlled trials (RCTs) are the gold standard format of clinical trials, the need for a placebo group can extend development or even render their use impossible for rare diseases with small patient populations. The use of existing real-world data (RWD) and real-world evidence (RWE) in the form of electronic health records (EHR) and previous study data as an external control arm can help accelerate development, especially in rare diseases.
Lecanemab May Avoid Aduhelm’s Fate With CMS
New data from Eisei suggests that its Alzheimer's drug lecanemab may not share the same fate as aduhelm, which was pulled from the market after the Centers for Medicare and Medicaid Services (CMS) agreed to only reimburse the drug for patients in randomized clinical trials. The results show that the drug was able to slow cognitive decline by 27% over 18 months, which may meet the agency’s requirements. However, Eisei is being cautious with its pricing.
Webinar: Avoid The Valley of Death – Increase Confidence in Translational Research Using Biosimulation – September 12th
Only a third of preclinical programs make it to Phase 1 trials. Honing translational research is key to reducing the substantial financial risk associated with drug development. A new tool to help narrow the focus of preclinical programs is physiologically-based pharmacokinetic modeling (PBPK) modeling and simulation. Correct implementation can help predict human doses, find prime targets, and identify interactions.
Using Real-World Data to Boost Clinical Trial Patient Diversity
The patient population of clinical trials currently and historically have rarely represented the demographics of the general population. Real-world data (RWD) is increasingly being used to help address these disparities in trial participation. RWD sources are often far more diverse than patient pools available for physical clinical trial sites due to geographical and economic constraints.
Webinar: Overcoming Patient Recruitment Challenges in Rare Disease Trials
Nearly 450 million people live with rare diseases globally. This diverse population needs treatment options, but clinical trials are made [...]
Webinar: Overcoming Patient Recruitment Challenges in Rare Disease Trials – September 28th
Patient recruitment is one of the most difficult parts of any clinical trial, but it’s even tougher when studying therapeutics for rare diseases. In an upcoming webinar taking place on September 28th, speakers will discuss the unique challenges of patient recruitment in the field, how to speed up patient recruitment for rare diseases, and the lived experience of someone living with a rare disease.
Whitepaper: Making APAC the Oncology Clinical Trial Destination
A new whitepaper from Premier Research makes the case that the Asia-Pacific (APAC) Region is the hot spot for oncology clinical trials. Currently, APAC stands as the region with the most ongoing oncology trials worldwide, with the number of active trials growing 138% over the last 10 years. In the whitepaper, the authors break down what factors are driving this growth and why clinical research organizations (CROs) should consider hosting their trials in the region. Chief among these reasons is the massive potential patient pool.
Using Just-in-Time Clinical Trials to Address Disparities in Clinical Trial Representation
Finding and enrolling patients for clinical trials is one of the most difficult, time-consuming, and expensive parts of the drug development process. Ensuring that the patients represent the diversity of the population is even harder. In a new Targeted Oncology article, Kashyap Patel, MD, and colleagues discuss how just-in-time (JIT) clinical trial design can make the patient recruitment process cheaper and reduce disparities in clinical trial representation.
Whitepaper – Adaptive Trial Designs in Early Oncology: Minimizing Risk & Accelerating Timelinesv
Despite a growing understanding of cancer biology, only 10% of cancer treatments approved by the FDA have an overall survival benefit for patients. A newly available white paper lays out the current precision medicine landscape and how the shift towards adaptive trial designs could accelerate market access and improve patient outcomes
Free Ebook: Real-World Evidence in Oncology
Health economics and outcomes research (HEOR) and real-world evidence (RWE) are changing the clinical trial landscape in oncology. Oncology Central has released a free eBook about HEOR and RWE in oncology that includes peer-reviewed research articles and reviews, as well as access to a panel discussion.
How Decentralized Clinical Trials Can Center Patient Care
The COVID-19 pandemic drove decentralized care models to new prominence, allowing patients to safely access care from home through telehealth. [...]
Bavarian Nordic’s RSV Vaccine Candidate Granted PRIME Eligibility
The PRIME scheme helps accelerate assessment by the European Medicines Agency for therapeutics that treat otherwise unmet medical needs. Bavarian [...]
New Data Shows Bristol Meyers Squib’s Zeposia Improves Cognitive Outcomes in Multiple Sclerosis Patients
Results from Bristol Meyers Squib’s (BMS) Phase 3 DAYBREAK and SUNBEAM trials show that Zeposia helps improve and preserve cognitive [...]
Biogen Ends Aduhelm Study Due to Medicare Restrictions
Biogen has ended its observational ICARE AD real-world data (RWD) trial for Aduhelm, another in a long series of setbacks [...]
Clovis Withdraws Approval Applications for Ovarian Cancer Drug Rubraca
Clovis Oncology has announced it will withdraw its FDA approval application for Rubraca, its third-line BRCA-mutated ovarian cancer drug. The [...]
Roche’s Amyloid-Targeting Alzheimer’s Drug Fails in Phase II Trial
Roche and Genentech released data from their Phase II API-ADAD trial showing their experimental Alzheimer’s disease drug crenezumab failed to [...]
ICER Signals Concern About Price for Amylyx’s ALS Drug
The Institute for Clinical and Economic Review (ICER) has released a draft report detailing their concerns surrounding Amylyx Pharmaceutical’s new [...]
FDA Decisions on China-based Trials Speeds Transition to Multiregional Clinical Trials
The US Food and Drug Administration (FDA) has taken an increasingly tough stance on clinical trials performed primarily in China [...]
Two Women Execs from Takeda Talk Industry and Inclusion
Ramona Sequeira and Julie Kim took on new positions in Takeda’s executive leadership team early this April. Women are drastically [...]
Achieving Health Equity Requires More than Just Good Science
People with marginalized identities face undue difficulties accessing healthcare that is often lower quality than that received by majority groups. [...]
The Potential of Sleep Tracking Devices for Clinical Trials
As the public increasingly adopts apps and devices meant to track sleep habits, researchers have taken notice of the trend’s [...]
Synklino Nets €29.8M Series A Funding for Cytomegalovirus Drug Trial
Synklino, a Danish biotech, announced on Monday, June 6th, that it received €29.8 in a Series A funding round for [...]
