The expedited approval pathways implemented by the FDA in the past 30 years have shortened many drugs’ road to market, but a new study from Harvard University researchers questions whether the time saved outweighs the risks of approving new drugs without putting them through the traditional clinical trial process.
While it would be impractical — and unnecessary — to try to conduct trials in every single recognized patient population, researchers should design trials, choose study sites and recruit patients in a way that generates data about the safety and efficacy of drugs for as much of the population as possible. This could mean conducting studies in particular places or merely increasing the numbers of patients recruited to include less-represented populations.
Spurred in part by a disappointing 11th place ranking in a 2017 CenterWatch survey of how trial sites view their sponsors, Sanofi launched a program to reduce sites’ management burden. Sanofi now credits the program with its No. 1 ranking in the 2019 version of the CenterWatch survey.
The global drugmaker is seeing faster startup times, improved patient recruitment and retention, and overall increased efficiencies as a result of the new program. Sanofi unveiled its new program — dubbed Sanofi Makes Investigators’ Lives Easier (SMILE) — in May 2018.
A host of pressures — patient recruitment and retention, cost and efficiency, and regulatory — have researchers seeking out novel approaches to clinical trials. Among them are virtual trials, which are often touted as a solution to many of these problems. Still, challenges and concerns remain, resulting in slow industry adoption.
Patient-centeredness has become a hallmark of high-quality research, patient care and drug development — and patient engagement has become a major focus for clinical trial managers (CTMs). Study participants themselves are calling for more input into their care and satisfaction and quality outcomes are clearly tied to one another.
Adaptive trial designs can produce results more quickly and identify patient populations more clearly. But the savings in time and effort during the trial is balanced by the work that must be done in the design stage.
Unlike conventional studies, adaptive clinical trial design calls for ongoing analysis of data and modification of key trial parameters in response to the results. The adaptive trial model requires more extensive protocol planning — regular data analysis points need to be identified and rules created for making decisions about modifications before the trial begins.
Adaptive clinical trial design has been making its way from oncology and other specialty fields into the broader world of pharmaceutical and biologic studies. While adaptive design can offer many benefits for clinical investigators and sponsors, those conducting such trials can be easily tripped up in two areas: adequate, detailed planning of the design; and data control during the interim analysis that is the hallmark of adaptive design.
The PI and designated research staff are responsible for identifying, investigating, classifying and reporting adverse events (AE) under U.S. and international regulations, as well as IRB policies and specific study protocols.
Despite more than six years of regulators’ encouragement to adopt risk-based monitoring (RBM) of trials, the industry still has concerns about compliance.
Attendees at a recent FDA workshop question whether RBM is appropriate for small and complex studies, the appropriateness of sampling techniques for source data review (SDR) and source data verification (SDV) and how data privacy regulations might impact transmission and review of trial subject data.