In a new draft guidance, the US FDA proposes two strategies to make clinical trials for cancer drugs better support accelerated approval applications for cancer drugs. Such applications are expected to be followed by confirmatory studies that often don’t materialize or yield negative results. The […]
Improving Clinical Trials for Rare Disease Therapeutics
The decades old Orphan Drug Act helped spur a renaissance in rare disease research, incentivizing development therapeutics that may otherwise be deemed unprofitable. However, factors like small, spread-out patient populations and funding considerations make running clinical trials for rare diseases […]
Improving Clinical Trials for Rare Disease Therapeutics
With millions worldwide living with rare diseases that have few or no current therapeutic options, clinical trials are essential in treating the conditions faced by this diverse patient population. However, running these trials are expensive, hard to recruit for, and often require novel trial […]
FDA Allows Dose Increase in Dravet Syndrome Clinical Trial
The US Food and Drug Administration (FDA) has released a hold blocking a Phase I/IIa clinical trial testing an increased dose of STK-001, Stoke Therapeutics’ drug candidate for Dravet syndrome. Dravet syndrome is a rare form of severe childhood epilepsy that comes with a short life expectancy and […]
Positive Trial Data for AstraZeneca Lung Cancer Immunotherapy Sets the Stage for Battle with Merck’s Keytruda
Astrazeneca reported promising data for its non-small cell lung cancer (NSCLC) immunotherapy Imfinzi, setting the stage for a market showdown with Merk’s Keytruda. The therapy, when added to chemotherapy, improved event-free survival in patients undergoing surgery for early stage NSCLC. Once […]
New Trial Data Sinks Astellas’ Hopes to Expand Indication for Leukemia Drug
Astellas reported new trial data for Xospata that bodes poorly for its chances at an expanded indication as an add-on in the treatment of acute myeloid leukemia (AML), an aggressive blood cancer. The data showed that addition of Xospata after stem cell therapy did not improve relapse-free survival […]
Transparency, Oversight, and Opt-in Consent Makes Patients More Open to Sharing Data
A newly published paper in JAMA Network Open found that patients are more likely to participate in patient data sharing programs if certain conditions are met. Survey data from 3,500 hundred patients showed that patients valued transparency, opt-in informed consent, oversight mechanisms, and […]
Using Real-world Data to Take Better Baseline Patient Measurements
After recruitment and on-boarding, patients in clinical trials undergo a battery of baseline measurements. However, measurements can vary significantly from moment to moment and “normal” ranges are often wide. Therefore, an approach that uses multiple measurements could help reduce […]
Improving Health Equity Through More Representative Clinical Trials
Patients from marginalized or rural populations often have less access to clinical trials or distrust based on a long-standing history of medical exploitation. However, this disparity in patient populations is detrimental to health equity efforts, biasing research by skewing heavily toward primarily […]
EMA Committee Recommends Regeneron’s Libtayo + Chemotherapy Combo for Lung Cancer
Regeneron’s Libtayo (cemplimab-rwlc) has received a recommendation from the European Medicine’s Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) for use in forms of non-small-cell lung cancer (NSCLC) as a combination therapy with a traditional chemotherapy agent. The […]
