Study Reveals One-Third of Clinical Trial Data May Be Unnecessary, Highlighting Burden on Patients and Sites

A groundbreaking study conducted by the Tufts Center for the Study of Drug Development (Tufts CSDD) in collaboration with 14 biopharmaceutical companies has found that approximately one-third of all data points collected in phase 2 and 3 clinical trials may be unnecessary. This revelation comes as the pharmaceutical industry grapples with increasingly complex trial protocols and mounting concerns over patient and site burden.

Unnecessary Data Collection and Its Impact

The working paper, which analyzed 105 phase 2 and 3 trials, identified two types of potentially superfluous data:

1. "Non-core" data: Information not required by regulators or used to support key primary and secondary endpoints.
2. "Non-essential" data: Excess data collected beyond what is necessary for important data types.

Kenneth Getz, executive director of Tufts CSDD and lead author of the study, emphasized the significance of these findings: "It gives a lot of ammunition to clinical teams to really discuss and look at how they can optimize the collection of that data so that they can minimize patient burden, site burden and all of those downstream effects."

The study revealed that between 25% and 30% of the burden placed on trial participants was attributed to the collection of non-core and non-essential data. This burden includes factors such as travel time and lifestyle changes required by study participation.

Escalating Complexity in Clinical Trials

Over the past 25 years, trial protocols have become increasingly intricate, with more customized designs, additional eligibility criteria and endpoints, and larger patient enrollments across multiple sites. This trend has led to poorer trial performance, making patient recruitment, screening, and retention more challenging, while also complicating data cleaning and analysis processes.

Previous research from Tufts CSDD highlighted a dramatic increase in data collection, with the number of datapoints in phase 3 trials surging from 929,203 in 2012 to 3,560,201 in 2020. Getz illustrated the practical implications of this trend: "We have studies where patients have to go to three or four different locations within an institution just to have different assessments performed the same day. All of that adds time, adds burden."

Industry Response and Future Directions

In light of these findings, TransCelerate member companies are developing a framework to optimize their data collection practices. This framework will be published and shared with the broader industry, potentially leading to significant changes in clinical trial design and execution.

The study's results align with recent regulatory guidelines, particularly the January 2025 guidelines for good clinical practice from the International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use (ICH). These guidelines advocate for "fit-for-purpose, intentional protocol designs that weigh patient and site burden, that really look to optimize data volume," according to Getz.

As the pharmaceutical industry continues to evolve, this study serves as a crucial reminder of the need to balance scientific curiosity with practical considerations of patient and site burden. By optimizing data collection and streamlining trial protocols, companies may be able to improve trial efficiency, reduce costs, and ultimately accelerate the development of new therapies.