Clinical Trial Design Challenges and Opportunities for Emerging Treatments for Opioid Use Disorder: A Review.
Sign Up to like & getrecommendations! Published in 2022 at "JAMA psychiatry"
DOI: 10.1001/jamapsychiatry.2022.4020
Abstract: Importance Novel treatments for opioid use disorder (OUD) are needed to address both the ongoing opioid epidemic and long-standing barriers to existing OUD treatments that target the endogenous μ-opioid receptor (MOR) system. The goal of… read more here.
Keywords: clinical trial; trial; design; opioid use ... See more keywords
Issues in Clinical Trial Design-Lessons From the FDA's Rejection of MDMA.
Sign Up to like & getrecommendations! Published in 2025 at "JAMA psychiatry"
DOI: 10.1001/jamapsychiatry.2025.0442
Abstract: This Viewpoint explores potential justifications underlying the US Food and Drug Administration (FDA)’s rejection of methylenedioxymethamphetamine (MDMA) as a treatment for posttraumatic stress disorder, including lack of blinding and issues around the integrity of relevant… read more here.
Keywords: mdma; clinical trial; rejection; fda rejection ... See more keywords
A roadmap for clinical trial design in marginal zone lymphoma
Sign Up to like & getrecommendations! Published in 2022 at "American Journal of Hematology"
DOI: 10.1002/ajh.26706
Abstract: Significant advances in the drug development of indolent non-Hodg-kin lymphoma (iNHL) occurred over the last years expanding the landscape of treatment options. Several novel agents have been approved by the US Food and Drug Administration… read more here.
Keywords: lymphoma; design marginal; roadmap clinical; clinical trial ... See more keywords
Interpreting clinical trial results by deductive reasoning: In search of improved trial design
Sign Up to like & getrecommendations! Published in 2017 at "BioEssays"
DOI: 10.1002/bies.201700103
Abstract: Clinical trial results are often interpreted by inductive reasoning, in a trial design-limited manner, directed toward modifications of the current clinical practice. Deductive reasoning is an alternative in which results of relevant trials are combined… read more here.
Keywords: clinical trial; deductive reasoning; trial; trial results ... See more keywords
Analyses of seizure responses supportive of a novel trial design to assess efficacy of antiepileptic drugs in infants and young children with epilepsy: Post hoc analyses of pediatric levetiracetam and lacosamide trials
Sign Up to like & getrecommendations! Published in 2021 at "Epilepsia Open"
DOI: 10.1002/epi4.12482
Abstract: Recently, a novel trial design has been proposed to overcome challenges with traditional placebo‐controlled trials of antiepileptic drugs in infants and young children (≥1 month of age) (Auvin S, et al. Epilepsia Open 2019;4:537‐43). The… read more here.
Keywords: infants young; young children; trial design; seizure ... See more keywords
Clinical trial design essentials for genetic counselors.
Sign Up to like & getrecommendations! Published in 2025 at "Journal of genetic counseling"
DOI: 10.1002/jgc4.70150
Abstract: Genetic counseling often raises important questions: Does a new service delivery model improve patient outcomes? Will delivering results in a certain way reduce anxiety? Does a genetic counseling intervention in certain populations change health outcomes?… read more here.
Keywords: clinical trial; genetic counseling; design essentials; genetic counselors ... See more keywords
The Personalised Randomized Controlled Trial: Evaluation of a new trial design
Sign Up to like & getrecommendations! Published in 2023 at "Statistics in Medicine"
DOI: 10.1002/sim.9663
Abstract: In some clinical scenarios, for example, severe sepsis caused by extensively drug resistant bacteria, there is uncertainty between many common treatments, but a conventional multiarm randomized trial is not possible because individual participants may not… read more here.
Keywords: randomized controlled; trial; treatment; controlled trial ... See more keywords
The Road to Remyelination in Multiple Sclerosis: Breakthroughs, Challenges, and Considerations for Future Trial Design
Sign Up to like & getrecommendations! Published in 2025 at "Drugs"
DOI: 10.1007/s40265-025-02212-x
Abstract: Despite major advances in multiple sclerosis (MS) treatment, disability accumulation independent of relapse activity remains a significant challenge. Chronic demyelination is a key driver of neurodegeneration and disease progression, highlighting remyelination as a promising therapeutic… read more here.
Keywords: trial design; multiple sclerosis; trial; remyelination ... See more keywords
swCRTdesign: An RPackage for Stepped Wedge Trial Design and Analysis
Sign Up to like & getrecommendations! Published in 2020 at "Computer methods and programs in biomedicine"
DOI: 10.1016/j.cmpb.2020.105514
Abstract: BACKGROUND AND OBJECTIVE Stepped wedge trials (SWTs) are a type of cluster-randomized trial that are commonly used to evaluate health care interventions. Most SWT-related software packages have restrictive assumptions about the study design and correlation… read more here.
Keywords: design analysis; correlation; trial design; stepped wedge ... See more keywords
Statistical properties of Continuous Composite Outcomes: Implications for clinical trial design
Sign Up to like & getrecommendations! Published in 2020 at "Contemporary Clinical Trials Communications"
DOI: 10.1016/j.conctc.2020.100655
Abstract: Statistical efficiency can be gained in clinical trials by using composites of time-to-event outcomes when the individual component outcomes have low event rates. However, the utility of continuous composite outcome measures is not as clear.… read more here.
Keywords: clinical trial; trial; continuous composite; statistical properties ... See more keywords
Phase II Trials in Drug Development and Adaptive Trial Design.
Sign Up to like & getrecommendations! Published in 2019 at "JACC: Basic to Translational Science"
DOI: 10.1016/j.jacbts.2019.02.005
Abstract: Summary Phase II clinical studies represent a critical point in determining drug costs, and phase II is a poor predictor of drug success: >30% of drugs entering phase II studies fail to progress, and >58%… read more here.
Keywords: drug; trial design; phase; drug development ... See more keywords