High Cost a Primary Obstacle; Sites/Patient Recruitment Remains Problematic; Presence of Dedicated CRO Analgesia Team Important

A new survey of analgesia clinical trial decision makers commissioned by Premier Research and involving 51 pharmaceutical and biotech firms primarily in North America and Europe reveals that while three out of four respondents (75%) have some recent experience with the use of ePRO (electronic patient-reported outcomes) technologies in clinical trials, about the same number (76%) are confused or even unfamiliar with the regulatory guidance for the use of the technology.

Despite almost all respondents (98%), saying that they were likely or definitely going to use ePRO technology for their future clinical trials, 45% also cited ePRO’s high cost as the primary obstacle to its actual adoption.

ePRO Option Far and Away the Most Popular

Handheld devices that feature ePRO technology are preferred by a greater than four-to-one margin among survey respondents and the vast majority (90%) are familiar with ePRO. Electronic patient-reported outcomes are collected via a mobile handheld device, like a tablet.

“Pain studies rely on subjective data points which reflect the way study participants perceive pain—as to intensity, frequency, duration, and so on,” Premier Research VP, Analgesia Michael Kuss said. “Because pain studies lack objective measurements, such as a blood test, methodologies that optimize the objectivity of inherently subjective data are highly sought after.”

Time-stamped for verification, ePRO data, Kuss noted, “can be monitored for compliance in real time, versus, say, old-fashioned paper diaries, which are often presented to investigators days or weeks later. The immediacy and thus the accuracy of ePRO is just huge.”

Indeed, seven in 10 respondents (71%) acknowledge that ePRO applications are a valuable method for collecting data for analgesia clinical trials.

Only 6% of respondents prefer the telephone (IVR-interactive voice response) collection method, while 16% prefer web-based survey tools (IWR –interactive web response). Traditional paper diaries are still preferred by 12% of respondents.

Still, noting that immediacy alone does not compensate for variability among different patients’ perceptions of pain, Kuss emphasized that consistency can be ensured across subjective patient feedback by:

Familiarity with ePRO does not Translate into Familiarity with Guidance

Kuss observed that despite the popularity of ePRO technology among sponsors, about the same number are unfamiliar with the guidance (37%) or just plain unaware that the FDA has issued its final ePRO guidance (35%).

In fact, only one-quarter (24%) of respondents are familiar with, indeed, have a complete understanding of the FDA’s final guidance on ePRO.

“Keeping up with guidance is crucial,” Kuss said. “We are as familiar with the process as most companies are unfamiliar with guidance,” adding that Premier Research has gained that experience in managing more than 600 pain trials in which more than 90,000 patients were treated. Kuss added that Premier Research has conducted clinical trials for virtually every major class of analgesic, including all of the NSAID analgesics on the market today.

Site/Patient Recruitment an Obstacle; Dedicated CRO Analgesia Team Important

Even though most respondents (71%) select investigative sites from a combination of their own site lists and those of their contract research organization (CRO) partners, two-thirds (65%) say recruiting investigative sites for analgesia studies is problematic, even challenging.

Two out of three respondents (67%) say that recruiting patients is their biggest obstacle and six in 10 (63%) indicated the trait most highly valued when outsourcing an analgesia clinical trial is the ability of the CRO to recruit study participants.

“Perhaps this is why almost all respondents believe that the presence of a separate, dedicated CRO team solely responsible for analgesia studies is a compelling factor in deciding which service provider to choose,” Kuss said. “Without the right sites and motivated investigators, patient recruitment inevitably falls short—especially in pain trials.”

Respondents were split as to how to remedy patient recruitment woes: 51% would opt for a study-specific patient recruitment and retention program to increase enrollment; 47% would add more investigational sites.

Thus finding patients quickly is respondents’ top priority—“even when,” Kuss pointed out, “the potential tradeoff was increased data variability across investigational sites. Perhaps in the sponsors’ minds,” he mused, “data variability can be mitigated through the use of ePRO technology.”

Kuss noted that when asked about the benefits of using ePRO technology on an analgesia study, 53% of respondents indicated it would contribute to better data quality and improved patient compliance.

Getting to PoC is Top Priority in Analgesia Trials

Four in five respondents (80%) say that getting to “proof of concept” (PoC) as fast as possible is their top priority in developing analgesia compounds.

The tactic that six in 10 (61%) biopharma sponsors of analgesia clinical trial studies say works best in accelerating the delivery of PoC for novel pain medications is the use of validated indication-specific development models.

At the same time, 55% of respondents say that combining Phase I and Phase II studies also works well, while 43% say that reducing the signal-to-noise ratio in study data by limiting the number of investigational sites is important. Also, 37% say that conducting a global study to reduce patient recruitment time works best in speeding up the PoC process.

Doability vs. Variability

Patient recruitment is such a crucial issue in biopharma companies’ ability to complete clinical trials that those companies by a two-to-one margin are willing to sacrifice data variability for increased patient recruitment by working with more investigational sites.

“Pain is an unforgiving master, and, because of nausea and other side effects of many pain medications, we see people drop out of analgesia clinical trials more frequently than in any other type of clinical drug trial,” Kuss explained. “Our mission is to balance the scientific design of analgesia protocols against the need to keep pain patients from dropping out. Study doability versus data variability is where the rubber meets the road in analgesia clinical trials.”

“The question becomes, what’s the boundary between variability and doability,” Kuss added. “How does the ability to recruit the right patients and the right number of patients start to be a data issue? That’s where therapeutic expertise becomes an art.”

Outsourcing of Analgesia Clinical Trials is Preferred Option
Biopharma sponsors frequently turn to CROs for their expertise in running analgesia clinical studies. In fact, eight in 10 (80%) say they retain CROs to develop their regulatory strategies in complying with pediatric requirements for their pain drugs.

About the Survey
For this survey, 51 high-level clinical development decision-makers were interviewed during August 2012.

About Premier Research
Premier Research is a leading global contract research organization (CRO) serving biotech, pharmaceutical, and medical device corporations. Its services include clinical research and regulatory outsourcing in the areas of analgesia; neurology; infectious, cardiovascular, and respiratory disease; dermatology; and oncology. The company also has a wealth of experience in medical device and pediatric research.

Premier Research has managed more than 600 pain trials treating over 90,000 patients since our company’s inception and has conducted trials for virtually every major class of analgesic including all of the NSAID analgesics on the market today.

Premier Research has 21 offices (seven in North America, 14 in Europe) and operates in 23 countries. It employs 1,000 clinical professionals dedicated first and foremost to fulfilling each customer’s requirements in a timely, accurate, and cost-effective manner. This includes a strong international network of monitors and project management professionals combined with regulatory, data management, statistical, scientific, and medical experts, and staff at its well-established network of dedicated clinical sites.