Related Experiment Video
Updated: Jan 21, 2026
Laboratory Scale Slow Cook-Off Testing of Rocket Propellants: The Combustion Rate Analysis of a Slowly Heated Propellant CRASH-P Test
Published on: February 6, 2021
Strategy for Assessing New Drug Value in Orphan Diseases: An International Case Match Control Analysis of the PROPEL
Owen A O'Connor1, Enrica Marchi1, Weining Volinn2
1Department of Medicine, Center for Lymphoid Malignancies, Columbia University Medical Center, College of Physicians and Surgeons, New York, NY.
Background:
Although randomized studies are designed to assess overall survival (OS) benefit, the conduct of regulatory studies in patients with orphan diseases can be timely and costly without offering the same commercial return on the investment. The peripheral T-cell lymphomas (PTCL) represent a rare group of heterogeneous lymphoid malignancies with very poor prognosis. PROPEL was a pivotal phase II study that led to the accelerated approval of pralatrexate for patients with relapsed or refractory PTCL.
Methods:
An international database of 859 patients was assembled from four institutions with an interest in PTCL, of which 386 were considered eligible for matching against the PROPEL criteria. Using a rigorous propensity score matching algorithm, a unique 1:1 case match of 80 patients was performed.
Results:
The analysis demonstrated an OS benefit for the PROPEL population with a median OS of 4.07 and 15.24 months (hazard ratio = 0.432, 95% confidence interval = 0.298 to 0.626), respectively, for the control and PROPEL populations. Highly statistically significant improvements in OS were noted for the PROPEL population about the subtype of PTCL (save anaplastic large cell lymphoma) and all age groups, including the elderly (>65 years of age). For patients on PROPEL, there was a statistically significant prolongation in progression free survival compared with the line of prior therapy, including those with refractory disease.
Conclusion:
In the context of this case-match-control study, patients treated on PROPEL experienced an OS advantage compared with an international database of historical controls. This information can help inform critical decision-making regarding clinical studies in PTCL.
Related Concept Videos
01:02Prescription, Nonprescription and Orphan Drugs
The misuse and addiction to prescription drugs is a growing problem that can affect people of all age groups, specifically teenagers. This can happen when prescription medications are used in ways not intended by the prescriber, such as taking someone else's prescription or using medication for...
01:17Sign Test for Matched Pairs
To conduct the sign test, we first calculate the differences in...
01:25Strategies for Assessing and Addressing Confounding
Confounding can be addressed at both the design phase of a study and through analytical methods after data...
01:27Chronic Obstructive Pulmonary Disease-IV: Assessement and Diagnostic Studies
Medical History
02:00Internal Energy
01:23Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...