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.
Looking Past Amyloid – The Future of Alzheimer’s Therapeutics
One of the key therapeutic targets in Alzheimer's disease is amyloid, a sticky protein that builds up in clumps outside neurons, causing cell death and cognitive decline. However, new therapeutics targeting the protein have only shown mild efficacy in delaying disease progression. As a result, drug developers are looking for other biological targets.
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.
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.
Molecular Templates’ Multiple Myeloma Trial Partial Hold Lifted
Molecular Templates’ clinical trial for MT-0169, its experimental multiple myeloma therapeutic, is back on, following the US Food and Drug Administration (FDA) lifting a partial hold. The hold was placed on the trial following two adverse events experienced by patients, who both recovered. The move comes after the company laid off half its employees to focus on this drug, along with two others.
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.
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.
EQRx Dashes Plans to Drive US Drug Pricing Reform
EQRx is moving on from its previous goal to change the drug pricing landscape in the US, letting go of over half its employees in the process. The company’s mission was to cut costs through developing drugs in categories with known safety profiles, cutting research timelines and expenses by up to 50%. They are now moving towards a market-based pricing strategy moving forward.
How to Overcome Challenges When Backing Regulatory Submissions with Real-World Data
Clinical developers and regulatory approval agencies are increasingly relying on real-world data (RWD) and the real-world evidence (RWE) generated from it in decision-making. However, clinical research organizations (CROs) often run into significant pitfalls when using RWD, complicating the process and driving up expenses. In a new Applied Clinical Trials article, learn what these challenges are and how to overcome them.
Potential Partnerships Between Retailers and Real-World Data Firms Could Advance Clinical Trials
Major retail chains like CVS, Walgreens, and Kroger are increasingly entering the clinical trials space, effectively creating a swath of new recruitment sites for clinical research partners. According to COTA CEO and president Miruna Sasu, PhD, there is fertile ground for partnerships between retailers and real-world data (RWD) companies which, if capitalized upon, could accelerate clinical research.
FDA Panel Votes for Limited Approval of AstraZeneca and Merck’s Lynparza
AstraZeneca and Merck’s Lynparza looks set for a narrow approval as part of a combination therapy for metastatic castration-resistant prostate cancer (mCRPC). In an 11 to 1 vote, a Food and Drug Administration (FDA) panel supported approval of the drug in combination with a corticosteroid and J&J’s Zytiga only for a small subset of mCRPC patients.
ICER Issues Final Report on Alzheimer’s Drug Leqembi
Following a session of public comments, the Institute for Clinical and Economic Review (ICER) has issued its final evidence report on Eisai’s Alzheimer’s drug Leqembi (lecanemab). The independent organization valued the treatment between $8,900 - $21,500 per year. This is notably lower than the current list price of $26,500 per year set by the manufacturer.
FDA Gives Abbvie’s Qulipta the Nod for Chronic Migraine Prevention
The US Food and Drug Administration (FDA) approved the use of Abbvie’s Quilipta as a preventive treatment for chronic migraines. This approval gives AbbVie a leg up on Pfizer, whose migraine drug Nurtec ODT is not officially approved for chronic migraines. The decision was made based on data from a phase 3 trial that found the drug significantly reduced migraine day for patients.
Patient Recruitment Dodges Older Adults to Their Detriment
Despite representing the largest market for new and existing medications, a recent study by the UK-based International Longevity Centre (ILC) finds that adults over 60 are starkly underrepresented or outright excluded from clinical trials. Costs are commonly credited for this disparity, which imperils health outcomes for older adults.
Embracing Patient-Centered Clinical Trials to Facilitate More Representative Recruitment
Despite recent efforts, patient recruitment for clinical trials is expensive, difficult, and time-consuming. Moreover, the vast majority of clinical trials are far from representative of real patient populations, often severely lacking in people from marginalized ethnic and racial backgrounds. In a new sponsored Biopharma Dive article, learn how a patient-centered approach may facilitate cheaper and more representative recruitment.
FDA Advisory Committee Recommends Rexulti for Alzheimer’s-Related Agitation
Agitation is a common symptom of Alzheimer’s disease, negatively impacting the quality of life for patients and their caregivers. An advisory committee for the US Food and Drug Administration (FDA) has given the nod to expand the indication of Takeda and Lundbeck’s Rexulti, a widely used atypical antipsychotic, to include the treatment Alzheimer’s disease-related agitation (AAD).
US FDA Shoots Down Eli Lilly’s Application for Ulcerative Colitis Drug
The US Food and Drug Administration (FDA) has declined to approve Eli Lilly’s ulcerative colitis drug mirikuzimab. The agency credits manufacturing issues for the decision, noting that the drugs efficacy and safety are not in question. A Lilly spokesperson said that they are working closely with the agency to address these problems and bring the drug to market.
Most Accelerated Approval Confirmatory Studies are Turned in Late
Accelerated approval by the US Food and Drug Administration (FDA) requires drug and device manufacturers to conduct confirmatory studies to support full approval. A newly published paper in JAMA Health Forum found that over half of these studies are turned in late to the agency. These delays were more common in trials for small molecule drugs and non-oncologic therapeutics
Blueprint’s Experimental Cancer Drug Back in the Clinic After FDA Hold Lifted
New patients will receive Blueprint Medicine’s experimental cancer drug BLU-222 after the US Food and Drug Administration (FDA) lifted a partial hold. The agency paused dosing new patients after some participants reported vision-related adverse events. Blueprint said its updates to its protocols on adverse events were responsible for the hold being lifted.
US FDA Releases Draft Guidance on Oncology Clinical Trials Seeking Accelerated Approval
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 first suggestion is to move to a single trial for accelerated approval that also confirms potential health benefits.
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 formats to account for small participant pools. Regardless, hope is on the horizon for patients.
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 developmental delays.
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 entering the market, the drug is set to be a strong competitor with Keytruda.
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 at 2-year followup.
