Patient reported outcomes (ePRO) are a newly hot topic for the industry, with a second CBI conference dedicated to the topic barely half a year after an earlier event.

There is a limited number of technology firms competing in the ePRO niche. Perhaps half a dozen. We have no idea which company is the leader. It would not surprise us if Philadelphia’s CRF  or Pittsburgh’s invivodata would stake a claim to that title. As would PHT of Boston.

We hasten to note that several interactive voice response (IVR) companies (say, Almac or ClinPhone) insist rather vigorously that handheld devices aren’t the best way to deliver ePRO, citing cost and shipping hassles.

Sizing The ePRO Market

Be that as it may, CRF, invivodata and PHT have generally been in a friendly race in which all have prospered by selling Palm or Windows handheld devices with proprietary software. There’s no question some studies are too complex for IVR-based ePRO. The three handheld patient diary firms all track a variety of metrics showing their strengths—numbers of trials started, number of languages supported, devices shipped into the field, even peer-reviewed journal publications.

But now the 190-employee PHT is trying to make the case that it is the top firm in the sector with a 40 percent market share.

As ClinPage recently heard from Phil Lee, PHT’s president and CEO, he’s eager to reveal a bit about the privately held company to bolster its claims. So PHT says it has supported 340 trials worldwide. The firm’s most impressive statement is that its software has been used in nine new drug application (NDA) submissions, five of which resulted in approvals.

Top Spot?

On the financial front, PHT says bookings through the third quarter of 2007 were $34 million. The unaudited figure for the full year might be $40 million. “That makes us a rather substantial company, not just in the ePRO space but in the entire clinical space,” says Lee. If our calculator is working properly, that would appear to put the size of the entire ePRO niche at $100 million last year. “Bookings,” of course, is not a rigorously defined term regulated by the accounting profession, nor is a booked contract necessarily one that materializes as revenue.

“We wanted to make a public statement about where we were,” Lee added. “There is a lot of discussion about who the market leader is. We wanted to put our numbers out there, and let competitors do the same. We’ll see who comes out at number one.”

New Users

But it still sounds like the ePRO niche is on a role. Lee says PHT recently added ten new customers, three of which are among the 15 largest sponsors in the industry. “A lot of them had not done ePRO before,” notes Lee. “That’s very exciting.”

Why is the company doing well? There is a new program to assist contract research organizations (CROs), to which Lee hopes to assign additional resources. Beyond that, the complexity and scope of the work for existing clients is growing. “We are working on a lot of larger programs—large,  global, longer-term trials,” he says.

In-house Help

“We provide a strong regulatory approach,” Lee says. Such services may include assessing a trial protocol and offering counsel on the likely compliance of patients and other potential glitches. Notes Lee: “That type of expertise is very well received. We’ve done a really good job with our clients. When you do a good job in this space, they are good at giving you more studies.” PHT says one top-five pharma is now using its ePRO technology exclusively.

On a more mundane level, Lee says the company’s help desk—yes, its humble call center—is a competitive advantage. It has not been outsourced. “I believe we are the only company [in the ePRO sector] that has a 100 percent internal help desk,” says Lee. In many cases, he says, the PHT help desk is trained on specific protocols. That can help the people answering the phone to provide an informed answer quickly. “It’s about quality and responsiveness,” says Lee. “It’s about delivering [assistance] rapidly.”

Electronic Data

Lee says that the percentage of trials using electronic diaries is up dramatically. The usage of electronic data capture is helping drive adoption of ePRO, he says. For if a trial is collecting most of its data electronically, one system using paper (the patient diaries) is a relatively obvious bottleneck. Says Lee: “Paper becomes a limiting step in closing the trial and analyzing the data. That’s one thing that will drive adoption in 2008 and beyond.”

Still, Lee does concede that in some quarters of the sponsor community, there is significant uncertainty about the regulatory status of electronic diaries. Yes, there is existing guidance on the topic. But sponsors are worrying about how to validate a particular instrument. Lee says that validating paper patient diaries can also be a challenge or become an issue with regulators. And he says electronic ePRO systems have a host of other benefits that paper diaries lack.

Asked about the PHT annual user meeting, Lee says there were many best-practice lessons shared by customers. One example: tantalizing insights into how patient diaries might be used in a context of monitoring drug safety during a trial. Email alerts can be generated automatically according to any predefined set of circumstances, such as a patient who has not made a diary entry recently or an investigative site that has slipped in enrolling patients.

Adaptive Era

As with any new technology, Lee says PHT customers are trying to avoid collecting exactly the same clinical data using different buckets. “It creates an opportunity for there to be a discrepancy. It becomes a significant process to try to figure our which system is correct. It just creates more headaches than you really want,” says Lee. His advice: find the best system to collect a particular type of data. Use only that.

As the industry tip-toes into adaptive or flexible trials, Lee says, it will mean fewer studies using paper patient diaries. “As adaptive trials become more significant, that will require ePRO and electronic data capture,” he says. “You can’t do adaptive trials on paper. It can’t be done.”