Tufts Center for the Study of Drug Development

About: Tufts Center for the Study of Drug Development is a based out in . It is known for research contribution in the topics: Drug development & Clinical trial. The organization has 78 authors who have published 258 publications receiving 16047 citations.

Finding value in the U.S. Food and Drug Administration's Fast Track program.

Abstract: The U.S. Food and Drug Administration's (FDA) Fast Track program, created in 1997, was designed to facilitate the development and expedite the review of drugs and biologics intended to treat serious or life-threatening conditions, and that demonstrate the potential to address unmet medical needs. Although the intent is laudable, the significance of designations and effectiveness of the program have recently come into question. Tufts Center for the Study of Drug Development has collected data on fast track candidates since 1998. We analyzed the current dataset of 344 fast track candidates granted nearly 400 designations, representing approximately 70% of the fast track designations granted by FDA, to address questions regarding common metrics. We found that fast track candidates were widely diverse in characteristics and development histories. The complexity and limitations of the data introduced biases in metrics such as clinical phase lengths and phase transition probabilities, although these could be determined for subsets of the candidates. Our results suggest that evaluation of the Fast Track program requires a nuanced approach, and estimates of the program's value should include assessment of the resulting marketed products.

Cost-effectiveness analysis of three algorithms for diagnosing primary ciliary dyskinesia: a simulation study

Abstract: Primary Ciliary Dyskinesia (PCD) diagnosis relies on a combination of tests which may include (a) nasal Nitric Oxide (nNO), (b) High Speed Video Microscopy (HSVM) and (c) Transmission Electron Microscopy (TEM). There is variability in the availability of these tests and lack of universal agreement whether diagnostic tests should be performed in sequence or in parallel. We assessed three combinations of tests for PCD diagnosis and estimated net sensitivity and specificity as well as cost-effectiveness (CE) and incremental cost-effectiveness (ICE) ratios. A hypothetical initial population of 1000 referrals (expected 320 PCD patients) was followed through a probabilistic decision analysis model which was created to assess the CE of three diagnostic algorithms (a) nNO + TEM in sequence, (b) nNO + HSVM in sequence and (c) nNO/HSVM in parallel followed, in cases with conflicting results, by confirmatory TEM (nNO/HSVM+TEM). Number of PCD patients identified, CE and ICE ratios were calculated using Monte Carlo simulations. Out of 320 expected PCD patients, 313 were identified by nNO/HSVM+TEM, 274 with nNO + HSVM and 198 with nNO + TEM. The nNO/HSVM+TEM had the highest mean annual cost (€209 K) followed by nNO + TEM (€150 K) and nNO + HSVM (€136 K). The nNO + HSVM algorithm dominated the nNO + TEM algorithm (less costly and more effective). The ICE ratio for nNO/HSVM+TEM was €2.1 K per additional PCD patient identified. The diagnostic algorithm (nNO/HSVM+TEM) with parallel testing outperforms algorithms with tests in sequence. These findings, can inform the dialogue on the development of evidence-based guidelines for PCD diagnostic testing. Future research in understudied aspects of the disease, such as PCD-related quality of life and PCD-associated costs, is needed to help the better implementation of these guidelines across various healthcare systems.