Designing New Trials with Technology

Technology enables advances in trial design, but it is the creativity of people that really moves things forward.

If asked how computers, clinical trial software, and related technologies are changing clinical trials, most would probably think about the efficiencies that these technologies bring to the process. Faster data entry, more efficient communications between participants, and improved process management would be what springs to mind. While these are real and important benefits of clinical trial technology, they miss a key, driving benefit-changing the design of the clinical trial itself. (I am using the term "design" to cover both data- and operations-related factors.)

As I have discussed in previous columns, technology is the great enabler of process change. The value that any technology brings is often related to the creativity that users bring to the process. Through a thorough understanding of the capabilities of a given technology or set of technologies, creative people can change the fundamental process that the technology is applied to. A real-world example may help demonstrate this.

Not very long ago, our options for watching a movie or television program were limited. We could see the films that played in the movie theater at fixed times or watch television programs when they were broadcast.

New technology has led to a process change in movie distribution. The emergence of videotape, followed by DVD, made it possible to buy or rent almost any movie you wanted by going to the store. The arrival of the Internet enabled another leap forward--NetFlix created a business where anybody could order movies from an online database to be shipped for watching at home. Most recently, a Web site called Cin-o-matic¹ has made it possible to look at reviews from hundreds of movies available on video (or in the theater). Any movie of interest can be added to your list of NetFlix "orders" with one click.

Beyond this, the availability of broadband in many homes makes it possible to have movies distributed to your home immediately, without leaving your home, by your cable company.

In the course of 20 years, technology has enabled a revolutionary change in the process of watching movies. Yet, none of the advances in technology were created specifically for the process of viewing films--they were created for general purposes such as with the Internet, or for related purposes such as with cable. It was the creativity of people, using the enabling technology, who created these remarkable ways that we can get movies. Now, let's turn to clinical trials and technology.

Clinical trial design and technology
When considering clinical trial design, most of us think first about clinical endpoints used for statistical analysis. There are several important ways in which technology enables a dramatic change in clinical trial endpoints.

First is the collection of clinical endpoint data directly from the patient, today known as electronic patient reported outcomes (ePROs). Most clinical endpoints are collected directly by a physician or study nurse--accurate, objective reporters of quantitative data who understand the principles of scientific study. Certainly, for the measurement of blood pressure or ECG changes this makes sense.

However, the clinically meaningful endpoints for many clinical trials are observations that the patient makes and reports to the physician. For example, number of cigarettes smoked, frequency of headaches, episodes of nausea, etc. We have come to accept that these endpoints are collected at periodic, scheduled clinic visits during questioning by study staff. Patients are notoriously inaccurate at collecting data into paper diaries, and the data are usually of insufficient quality to be used as a clinical endpoint.