Statistical Challenges in the Evaluation of Treatments for Small Patient Populations

Adaptive Randomization Versus Interim Monitoring

(2013) Boris Freidlin et al.   JOURNAL OF CLINICAL ONCOLOGY

Sustained response and prevention of damage progression in patients with neonatal-onset multisystem inflammatory disease treated with anakinra: A cohort study to determine three- and five-year outcomes

(2012) Cailin H. Sibley et al.   ARTHRITIS AND RHEUMATISM

Design Issues in Randomized Phase II/III Trials

(2012) Edward L. Korn et al.   JOURNAL OF CLINICAL ONCOLOGY

Prospective Trial of a Pediatric Ventricular Assist Device

(2012) Charles D. Fraser et al.   NEW ENGLAND JOURNAL OF MEDICINE

Clinical trial of a farnesyltransferase inhibitor in children with Hutchinson-Gilford progeria syndrome

(2012) L. B. Gordon et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Berlin Heart EXCOR Pediatric ventricular assist device Investigational Device Exemption study: Study design and rationale

(2011) Christopher S. Almond et al.   AMERICAN HEART JOURNAL

Reply to M. Buyse et al

(2011) Edward L. Korn et al.   JOURNAL OF CLINICAL ONCOLOGY

Reply to Y. Yuan et al

(2011) Edward L. Korn et al.   JOURNAL OF CLINICAL ONCOLOGY

Overall Survival As the Outcome for Randomized Clinical Trials With Effective Subsequent Therapies

(2011) Edward L. Korn et al.   JOURNAL OF CLINICAL ONCOLOGY

A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation

(2011) Bonnie W. Ramsey et al.   NEW ENGLAND JOURNAL OF MEDICINE

Testing and interval estimation for two-sample survival comparisons with small sample sizes and unequal censoring

(2010) Rui Wang et al.   BIOSTATISTICS

Efficacy and tolerability of a new formulation of pancrelipase delayed-release capsules in children aged 7 to 11 years with exocrine pancreatic insufficiency and cystic fibrosis: A multicenter, randomized, double-blind, placebo-controlled, two-period crossover, superiority study

(2010) Gavin R. Graff et al.   CLINICAL THERAPEUTICS

A general inefficacy interim monitoring rule for randomized clinical trials

(2010) Boris Freidlin et al.   Clinical Trials

Outcome-Adaptive Randomization: Is It Useful?

(2010) Edward L. Korn et al.   JOURNAL OF CLINICAL ONCOLOGY

Trastuzumab in combination with chemotherapy versus chemotherapy alone for treatment of HER2-positive advanced gastric or gastro-oesophageal junction cancer (ToGA): a phase 3, open-label, randomised controlled trial

(2010) Yung-Jue Bang et al.   LANCET

Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTRMutation

(2010) Frank J. Accurso et al.   NEW ENGLAND JOURNAL OF MEDICINE

Anaplastic Lymphoma Kinase Inhibition in Non–Small-Cell Lung Cancer

(2010) Eunice L. Kwak et al.   NEW ENGLAND JOURNAL OF MEDICINE

The Path to Personalized Medicine

(2010) Margaret A. Hamburg et al.   NEW ENGLAND JOURNAL OF MEDICINE

Effective Incorporation of Biomarkers into Phase II Trials

(2009) L. M. McShane et al.   CLINICAL CANCER RESEARCH

Improved Early Event-Free Survival With Imatinib in Philadelphia Chromosome–Positive Acute Lymphoblastic Leukemia: A Children's Oncology Group Study

(2009) Kirk R. Schultz et al.   JOURNAL OF CLINICAL ONCOLOGY

Multi-Arm Clinical Trials of New Agents: Some Design Considerations

(2008) B. Freidlin et al.   CLINICAL CANCER RESEARCH

Monitoring for Lack of Benefit: A Critical Component of a Randomized Clinical Trial

(2008) Boris Freidlin et al.   JOURNAL OF CLINICAL ONCOLOGY